“We’ve seen some tremendous acceleration in science over the past few years and that science is the driving force behind new medicines. The fundamental need for these new medicines is the underpinning of the sector’s longevity and will be what leads the recovery from the current position,” declared Chris Hollowood, CEO of UK life sciences investment firm Syncona Investment Management Limited, summing up a view that is held by many in the sector.
“This optimism is rooted in our belief that the industry is now at a critical juncture, where the tools we have available, and the advancement of biological understanding, are presenting opportunities to tackle disease at an unprecedented cadence,” he said. “We believe the next wave of medical innovation is being driven by a more holistic understanding of disease from genetics through to fundamental biology, paired with an incomparable number of modalities with which we can now attack a disease with even greater precision. We’ve got more tools available to us than ever before and picking the right modality, for the right target, in the right setting will be key for therapeutic advancements in 2024 and beyond.”
Cancer is one area where these advances are particularly notable.
“2024 could be the year of breakthroughs in oncology and novel modalities,” Tadaaki Taniguchi, chief medical officer, head of medical and development at Astellas Pharma, Inc., told Scrip. “Oncology continues to dominate the global R&D pipeline and exciting, new treatment options could be significantly augmented this time next year. Leaps forward in our understanding of cancer biology are raising the possibility of treating more cancer, more effectively, in more patients, with advances in small molecules, antibodies, immune cell engagers, oncolytic viruses, targeted protein degraders and novel cancer cell therapies.”
For Daphne Zohar, CEO of PureTech Health plc, on the other hand, “the areas that have been attracting interest include metabolic disease, neuroscience and immunology and additional deals could further solidify this trend.”
“Attention returns to chronic conditions based on significant unmet need, large patient populations, and pricing pressure forcing lower price points that require large volume markets,” noted Jon Congleton, CEO of Mineralys Therapeutics, Inc.. “Immunology and inflammation, CNS/neuropsychiatry and cardiorenal metabolic – all former hallmarks of the pharma industry – will see a resurgence in interest and value,” he added.
John Tsai, executive partner at Syncona, CEO of Forcefield Therapeutics Ltd. and former head of global drug development at Novartis AG, took a similar view: “There will be further momentum for a return to large disease populations of unmet need such as cardiovascular disease targeting novel biology to have an inflection on improved outcomes of large populations. We’re starting to see this changing now, and suddenly there’s wealth of extremely exciting innovation in this space, such as in metabolic and renal disease, which is fantastic,” he said. “It’s a great time to be evaluating exciting targets which could have an impact on these diseases. Novel clinical trial approaches using novel endpoints and AI-assisted tools will facilitate exploration of difficult to treat patient populations.”
John Lepore, CEO of ProFound Therapeutics and CEO-partner at Flagship Pioneering, also saw new possibilities in chronic disease treatment. “The therapeutic choices in many diseases – particularly common chronic diseases like renal failure, heart failure, and neurodegenerative diseases – have been relatively stagnant, in part because of a lack of mechanistic disease understanding and availability of new drug targets,” he commented. “This has also led to many companies competing to drug the same small number of targets. One promising area is the exploration of the ‘dark genome’ – segments of DNA that were not previously known to produce functional proteins. It is now becoming apparent that there are thousands of such proteins, opening up opportunities to find new drug targets and to position them in specific subsets of disease for greater treatment effect.”
“The cardiorenal risk reduction demonstrated with the new obesity therapeutics is highlighting the unique underlying biology of millions of patients. Despite the enthusiasm for these new therapeutics, there remains significant residual risk that may be addressed by a whole new wave of innovation in the CVRM [cardiovascular/renal/metabolic] space,” said Congleton. “These are some of the largest market opportunities for pharma and represent an opportunity to counterbalance the growing pricing pressure that is mounting in the US. While the IRA [Inflation Reduction Act] may face challenges and even be struck down, there are even more draconian measures potentially waiting in the wings that will ultimately make high-volume, low-price markets attractive to biotech and pharmas alike.”
“We must become more efficient at developing therapies that go beyond incremental benefits to supersede standards of care.”
Robert Plenge, Bristol Myers Squibb
Robert Plenge, executive vice president, chief research officer and head of research at Bristol Myers Squibb Company, also flagged up the importance of making a real difference with new therapies at the same time as addressing cost issues. “The biopharma industry is reaching an inflection point, reckoning with productivity challenges that impact patients. We must become more efficient at developing therapies that go beyond incremental benefits to supersede standards of care,” he said. “To accomplish this, we must increase the likelihood of clinical success and address the novelty dilemma – the need to identify new therapeutic targets that will make meaningful differences in improving, if not stopping, disease.”
He explained that BMS uses three research strategies to address these needs: “selecting targets with strong causal human biology, aided by human genetics; matching therapeutic modality to disease mechanism of action, leveraging expertise across modalities; and defining the path from research to clinical proof of concept, increasing success rates.”
Plenge predicted: “With a deep understanding of the cause-and-effect relationships between targets and outcomes, and developing more efficient clinical trials, we can discover new therapies to benefit patients in 2024 and beyond.”
Cost-effectiveness was on the mind of Eliot Forster, CEO of pain-focused drug developer Levicept Ltd. “Given the ever-rising healthcare budgets driven by an aging population, drugs that can significantly move the needle by improving patient care whilst at the same time reducing the cost burden will remain the Holy Grail,” he said. “Approaches like early diagnosis and more cost-effective therapies that improve patients’ health span as well as life span are critical, as are non-addictive drugs to treat chronic pain.”
Judith Klimovsky, executive vice president and chief development officer at Genmab A/S, flagged up the importance of our growing understanding of the immune system to therapeutic advances. “As a physician and biotech professional, I’m driven by a passion to harness the power of the human immune system to make a real impact on patients’ lives. It’s exciting to see innovation in this space, including the continued exploration of new modalities such as bispecifics and antibody-drug conjugates,” she said. “And in 2024, we will continue to see an increase in the number of innovative antibody medicines explored in clinical trials and potentially approved by regulatory agencies as we move toward using a more targeted approach to address difficult-to-treat diseases, like advanced cancer.”
Klimovsky also observed that “the COVID-19 pandemic has accelerated the embrace of patient-centric clinical trial models. This trend is likely to continue, offering greater flexibility and access to trials for patients, potentially improving recruitment and retention rates.”
The use of artificial intelligence and machine learning to accelerate therapeutic advancements via drug discovery was highlighted by Nicolas Tilmans, CEO of Anagenex. “The key marker for success, however, is the ability to experimentally generate the kinds of datasets that AI needs to be successful,” he pointed out. “Looking ahead to 2024 I predict that momentum will continue to grow behind those types of companies as we continue to join bench science and computation ever more tightly to dramatically shorten timelines to find molecules for the toughest problems in medicine, ultimately improving patients’ lives.”
More than 100 industry leaders shared their thoughts on likely areas for therapeutic progress in 2024; their insights continue below.
Cell And Gene Therapies, Genetic Medicines
Several executives flagged cell and gene therapies and genetic medicines as important areas to watch for therapeutic advances. “We continue to believe in the potential of this ground-breaking science, which has started to deliver transformational benefits for patients and even cures for previously uncurable diseases,” said Chris Hollowood, of Syncona, which has been investing in the area for the past decade.
In 2017, the US FDA approved the first chimeric antigen receptor (CAR)-T cell therapy, Novartis’s CD19-targeting Kymriah (tisagenlecleucel), for acute lymphoblastic leukemia. Since then, other products have been approved for additional indications, but there remain many avenues for further development.
“Aiming to address the existing limitations of cell therapies, the next evolution of research will focus on genetic engineering to innovate CAR therapies and enhance the innate characteristics of the cells themselves.”
Nicholas Siciliano, Vittoria Biotherapeutics
“The CAR-T cell therapy pioneers spent decades optimizing the ‘CARs’ used in today’s therapies, resulting in remarkable responses for hematologic malignancies, especially for patients with relapsed/refractory B-cell leukemia and lymphoma. However, CAR-T cell therapies have yet to be approved for indications beyond B-cell malignancies, and challenges still exist to move beyond hematological malignancies that may require a more innovative approach to CAR-T cell design,” said Nicholas Siciliano, CEO of cell therapy company Vittoria Biotherapeutics. “Aiming to address the existing limitations of cell therapies, the next evolution of research will focus on genetic engineering to innovate CAR therapies and enhance the innate characteristics of the cells themselves. Using modern gene editing approaches, we target key pathways to enhance attributes like stemness and durability. Improving these factors could yield therapies that have superior activity and broader applicability – unlocking the full potential of cell therapies and improving patient outcomes across more difficult to treat disease indications, like T-cell malignancies and solid tumors.”
“CAR-T cells, immune cells boosted by genetic modification, were initially used to treat blood cancers in patients refractory to the usual treatments or in relapse, after two attempts at ‘standard of care’. These produced remarkable results. In 2022, with the results of clinical trials confirming the long-term effects, some countries moved these very special drugs from third- to second-line treatment. CAR-T cells have themselves become a standard of care,” said Guillaume Wallart, CEO of French CAR-T cell production specialist CellQuest. “2023 saw CAR-T cells emerge from blood cancers, with extraordinary results against lupus and encouraging results against certain cancers with a very poor prognosis, including ovary and glioblastomas. 2024 will certainly see confirmation that CAR- T cell therapy is capable of curing the most aggressive cancers. All that remains is to find out how to mass-produce them.”
“Thanks to regulatory approvals throughout 2023, cell and gene therapies have significant momentum going forward. These include a range of new technologies – including the first CRISPR-Cas9 edited therapy and the first allogeneic CD34+ cell therapy – and expansion into new indications like sickle cell disease, beta-thalassemia, dystrophic epidermolysis bullosa and type 1 diabetes,” said Veerle d’Haenens, general manager, global therapeutic systems and cell therapy technologies, Terumo Blood and Cell Technologies.
“We expect to see more regulatory approvals and more novel technologies progressing through the clinic in 2024, but equally important to being the first is making the best. We believe there will be an increase in collaborative efforts aimed at standardizing and optimizing each phase of development and manufacturing. We work with developers to optimize the collection of cells that form the basis of these advanced therapies. And as partnerships like these continue to make their mark, interest will grow in additional collaborations to overcome the remaining obstacles to access for patients to the treatment they need,” said d’Haenens.
Carole Nicco, chief scientific officer at BioSenic SA, which is developing cell therapy products as well as an arsenic trioxide-based treatment for autoimmune diseases, underlined that “harnessing the immune system has revolutionized cancer treatment in recent years” but noted that “on 28 November, the FDA received reports of malignancies in patients who received modified autologous CAR-T cell immunotherapies. There are currently six approved products in this class on the market. Benefits of these treatments must continue to outweigh risks. In 2024, there will be more regulatory action regarding cell gene therapies (CGT) and their impact on developing cancers, immune and autoimmune responses.” (Also see "CAR-T Malignancy Reports Could Temper Excitement For Use Beyond Oncology" - Pink Sheet, 29 November, 2023.)
For Kristin Yarema, CEO of Poseida Therapeutics, Inc., “Today, far too many patients are unable to benefit from autologous CAR-T therapy due to its limited supply, lengthy timelines, complex logistics, and cost. In 2024, I believe we will see continued progress in the development of readily produced, off-the-shelf allogeneic CAR-T products that have the potential to offer a compelling efficacy and safety profile while also supporting patient access. It’s absolutely critical to use the right manufacturing approach and platform technologies to deliver scalable and cost-effective allogeneic therapies for patients in need.”
“Previously CAR-T has revolutionized the hematologic malignancy space but now cell therapy is moving firmly into solid tumors with many trials reading out in 2024.”
Sandeep “Bobby” Reddy, ImmunityBio
“2024 could be the year for the second wave in cell therapy. We are expecting the first TIL approval and off-the-shelf T-cell, NK cell, and dendritic cell candidates are advancing in many biotech pipelines, particularly in new targets,” said Sandeep “Bobby” Reddy, chief medical officer of ImmunityBio. “Previously CAR-T has revolutionized the hematologic malignancy space but now cell therapy is moving firmly into solid tumors with many trials reading out in 2024.”
“One of the big changes to watch in cell therapy is the expansion into autoimmune disease by emerging biotech companies and big pharma,” said Paul Hastings, CEO of Nkarta, Inc., which develops allogeneic natural killer (NK) cells. “This activity has been catalyzed by a growing body of academic research suggesting cell therapy holds the potential to transform the treatment of autoimmune disease just as it has done for the treatment of blood cancers.”
“This is a critically important moment in the evolution of cell therapy,” Hastings went on. “Some of the most commonly underserved disease areas are now being investigated. Most lupus patients, for example, are young women of color. The FDA has approved only three medications to treat lupus in the past 70 years – symptomatic therapies that do not stop irreversible tissue and organ damage. Cell therapy companies believe we have the potential to achieve what decades of conventional R&D could not and attack, possibly eradicating, the underlying cause of the disease.”
As for gene therapy, Janice Cruz Rowe, Johnson & Johnson Innovative Medicine’s worldwide vice president, global commercial strategy organization, retina therapeutic area, sees both promise and challenge.
“Gene therapy holds promising potential to not only treat a wide range of diseases (eg, rare inherited retinal disorders, spinal muscular atrophy, cystic fibrosis, heart disease and hemophilia) but also potentially reverse and eliminate them. With exciting new advances and approved drugs over the past 40 years, there’s been significant progress to unlock this potential – but as we look ahead, two critical challenges remain,” she warned. “First is the scientific challenge to optimize gene therapy delivery and demonstrate long term effectiveness across a broad set of diseases. Concurrent with this is the challenge of ensuring that patients across the world can gain access to these transformational new therapies – and this includes managing the complexities of manufacturing, end-to-end distribution, patient support and market access and reimbursement. Success in these areas will require new technologies and capabilities, integrated planning with internal teams, and external partnerships that maximize resources and reach.”
“Novel modalities will remain firmly in the spotlight, in oncology and further afield. For cell and gene therapies alone, there could be over 20 approved therapies by 2024 as these approaches are applied to an expanding array of diseases for which no, or limited approved treatment options exist,” said Astellas Pharma’s Tadaaki Taniguchi. “With significant scientific, development and access challenges to overcome, commitment and long-term therapeutic strategies will be essential for delivering the value of these innovations to patients, prescribers and health systems.”
Vertex Achieves Gene-Editing Milestone With First Ever CRISPR Therapy Approval
Eric Rhodes, CEO of ERS Genomics Limited, highlighted gene editing and the recent approval of Vertex Pharmaceuticals Incorporated/CRISPR Therapeutics AG’s Casgevy (exagamglogene autotemcel) in the UK and US. “In the years since the discovery of CRISPR/Cas9, the research sector and applications of this revolutionary gene editing technology have grown considerably,” he said. The first approved clinical application of CRISPR by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) is testament to the power of the technology to cure previously untreatable diseases. The US FDA has also followed suit.”
Rhodes told Scrip: “With this precedent set, 2024 could be the year that we see a rapid increase in the number of clinical trials testing CRISPR-based therapies. Further consideration will also need to be given to pricing for CRISPR-based therapies, along with discussions around establishing and refining regulatory frameworks.”
Meanwhile, access, cost and other challenges particularly around blood stem cell transplantation were flagged by Dhvanit Shah, CEO of Garuda Therapeutics, Inc. “Today, patients in need of a blood stem cell transplant are dependent on finding viable human donor material as a source of blood stem cells. While recent advances in gene therapy have led to innovative new treatment options for patients, significant challenges associated with these approaches persist including the need for broader access to the product, the high cost of drug production, timeliness of collection, durability of therapeutic benefit, and quality and safety of blood stem cell material,” he said. “As we progress into 2024, I expect to see a push toward off-the-shelf offerings which have the potential to overcome many of the challenges associated with existing personalized treatments, broadening access to medicines for patients across the globe.”
Oncology
There is no sign that oncology will be toppled from its position as the therapeutic area with the most pipeline investment and diversity, and this was reflected in the wide range of predictions referring to cancer R&D.
“In the evolving landscape of cancer treatment, targeted therapies continue to be a transformative technology driving innovation,” said Steve Worland, CEO of eFFECTOR Therapeutics, Inc.. “These therapies are designed to home in on specific molecular or genetic features of cancer cells, disrupting their growth mechanisms while sparing healthy tissues. By homing in on precise disease drivers, targeted therapies hold the potential to maximize treatment efficacy while minimizing side effects, thus improving the overall quality of life for cancer patients.
“Approaches that enhance the body’s immune response against cancer, such as immunotherapies and immune checkpoint inhibitors, are gaining prominence. These treatments harness the immune system’s natural defenses to combat cancer cells. As these therapies continue to develop, they offer new hope for durable responses and even potential cures. In 2024, I look forward to further improvements in these technologies, with particular interest in seeing updates on how to use targeted therapies in combinations that delay or prevent drug resistance.”
Henrik Jespersen works as a clinical oncologist at Oslo University Hospital where he is head of melanoma oncology: “I am just old enough to remember when ineffective chemotherapy was standard of care, and most patients didn’t survive a year after diagnosis,” he said. “With the introduction of immune checkpoint inhibitors, melanoma suddenly became the model disease of a completely new way of treating advanced cancer that could induce long-lasting, deep responses, and I think it is safe to say that we are now curing a proportion of patients with metastatic melanoma. This has set the bar of what is possible with innovative drugs, even in the most aggressive metastatic diseases. I am convinced that the ever-increasing insights into tumor immunology will eventually lead to even more effective immunotherapies, so that even more patients can benefit from the powers of their own immune system.”
“While we now have the proof of principle that the immune system can recognize and eliminate cancer and, in some cases, deliver long-term control and even cures, most cancer patients are not yet benefiting from the approved immunotherapies.”
Jill O’Donnell-Tormey, Cancer Research Institute
“Key trends in the cancer immunotherapy space in 2024 all focus on various ways to create an effective anti-cancer immune response in every patient,” noted Jill O’Donnell-Tormey, CEO and director of scientific affairs at Cancer Research Institute (CRI). But she cautioned: “While we now have the proof of principle that the immune system can recognize and eliminate cancer and, in some cases, deliver long-term control and even cures, most cancer patients are not yet benefiting from the approved immunotherapies.”
Robert Iannone, head of R&D at Jazz Pharmaceuticals plc, was of a similar mind. “Even with all the recent advances in immuno-oncology and targeted therapies, there is still a vital need to improve care for people with cancer,” he said. “From our perspective, continued R&D is essential to transform current standards of care and address treatment options across multiple cancer types with significant unmet needs and historically poor outcomes. Because the treatment of cancer often requires a multi-prong approach, we are focused on pioneering new pathways into novel mechanisms and platform technologies that we believe can address and overcome some of the greatest unmet needs in cancer care to extend patient life with the goal of providing cures. For example, we are exploring novel ways to expand the applicability of targeting the HER2 protein, that is present in many solid tumor types — hopefully providing new treatments needed for difficult-to-treat cancers such as breast cancer or those arising in the stomach and biliary tract.”
“In 2023, we continued to see an increased interest in T-cell engagers for solid cancers. The modality remains promising, in particular as T-cell receptor-based or mimicking therapeutics (TCR) offer a solution to the off-tumor related limitations of current immunotherapies,” said CDR-Life, Inc.’s CEO Christian Leisner. “I believe that we will see T-cell engager companies continue to develop and leverage the major histocompatibility complex (MHC) for targeting cancer testis antigens such as MAGE-A4. With high expression levels in head and neck, gastric, bladder and non-small cell lung cancer, and close to none in normal tissues, a target like MAGE-A4 has the potential to produce tumor-targeted T-cell responses that maximize cancer cell elimination. In fact, we may see T-cell receptor based or mimicking therapeutics among the next wave of T-cell engager approvals for solid cancers.”
For John Scarlett, CEO ofGeron Corporation, blood cancers are top of mind. “I believe we’ll see continued momentum around new modalities and new classes of medicines for the treatment of hematologic malignancies as our understanding of the biology in this area improves significantly. Today, CAR-Ts, immunotherapies and other novel treatment approaches have the potential to revolutionize care for the millions of Americans impacted by blood disorders,” he said. “However, despite the advances represented by these new therapeutic approaches, there are still a number of hematologic disorders with substantial patient populations that continue to have unmet medical needs. That is why the lead program for our novel, first-in-class investigational telomerase inhibitor is designed to address the needs of patients with lower-risk MDS, a serious blood cancer that has seen only modest drug development innovation over the past decade, and where a new treatment option could be very meaningful for these patients.”
Mai-Britt Zocca, CEO of IO Biotech ApS, emphasized the importance of checkpoint inhibitor-based combination approaches: “When it comes to cancer, checkpoint inhibitors have probably been the biggest single story of the last decade. But on their own they have limitations, in terms of patient response rates. Over the last couple of years there have been numerous trials looking to boost response rates by combining checkpoint inhibitors with new therapies. For example, combining them with small molecules, antibody-drug conjugates and therapeutic cancer vaccines, which also can target the tumor micro-environment, to make it less immuno-suppressive and thereby improve efficacy results. Over the next year or two, some of these trials should start to bear fruit.”
Detlev Biniszkiewicz, chairman of NextPoint Therapeutics and managing director of biotech investment firm MPM BioImpact, was also thinking about checkpoint inhibitors: “Over the last four years, the dominant play in oncology has been PD-1/L1 plus something else. We are learning that PD-1/L1 is not the only backbone checkpoint in immuno-oncology (IO) and we are just starting to scratch the surface of understanding resistance mechanisms to standard-of-care treatments. I expect we will continue to see momentum build in the antibody-drug conjugate space as well as a revival of oncology targets such as CD3. The expansion of oncology targets to immunology and the merging of targets in these fields will also continue. In 2024, the most exciting development will be in advancing precision IO, both in terms of defining the patients who are most likely to respond and progress in combining tumor targeting with immune checkpoint inhibitors.”
“Following success in curbing the COVID-19 pandemic, I think we are likely to see advancements in RNA delivery of cancer vaccines.”
Christopher Haqq, Elicio Therapeutics
“The innovation in the immunotherapy space has been nothing short of transformative as researchers continue to push boundaries with technologies that empower the immune system to fight cancers,” said Christopher Haqq, head of R&D and chief medical officer at Elicio Therapeutics, a Boston, MA-based company operating that is developing an amphiphile platform to deliver therapeutic payloads directly to lymph nodes.
“In 2024, I expect we’ll see developments in technologies that increase the potency of T cell-based immunotherapies,” he went on. “I’m particularly excited about cancer vaccines because after years of setbacks in trying to develop vaccines that are safe and can also mount a durable immune response, I believe we’re finally making headway. Following success in curbing the COVID-19 pandemic, I think we are likely to see advancements in RNA delivery of cancer vaccines.”
“2023 has been a promising year for therapeutic cancer vaccines, bringing positive results and renewed investment to a rapidly expanding repertoire of advanced platforms. With over 200 cancer vaccines in clinical development for solid tumors, pioneering and ambitious therapies are reaching late clinical phase development,” said Lindy Durrant, CEO of Scancell.
“Personalized approaches targeting patient-specific tumor neoantigens, like Moderna, Inc.’s, have demonstrated durable responses. However, questions remain on streamlining timescales to meet patient demand in the future,” Durrant continued. “Building on the success of these approaches, much attention now focuses on ‘off-the-shelf’ vaccines that can address tumor heterogeneity through multiple tumor-specific and tumor-associated antigen combinations. Also, targeting antigen-presenting cells to generate potent T-cells that kill tumors and how this has a greater impact than the choice of antigen. Over 2024, readouts of cancer vaccines in randomized trials will further inform if these advances in antigen targeting, vaccine potency, and patient selection will provide a runway for more novel therapeutic cancer vaccines to overcome historical challenges.”
“Cellular immunotherapy has shown great potential in treating cancer in recent years,” said Michael Klichinsky, chief scientific officer of Carisma Therapeutics Inc. “Developing therapies based on engineered macrophages is a promising addition to the immune cell therapy arsenal. Such approaches have the potential to make a positive impact on patients dealing with cancer and other serious disorders and to transform the therapeutic landscape.”
In 2024, Loïc Vincent, chief scientific officer of Affini-T Therapeutics, Inc., expected that “we will see more companies leveraging a variety of gene-editing techniques in the quest to develop safer and more effective cell therapies for patients living with the most devastating solid tumor cancers.” He added: “We’ll also continue to see data from these next-generation approaches using non-viral CRISPR gene edited TCR-T cell therapies designed to target the most prevalent cancer-driving mutations in solid tumors. Non-viral gene editing may not only address potential safety concerns that could arise with more traditional cell therapy engineering approaches, but also enhance the speed and efficiency of manufacturing these medicines, which means in time, a treatment that could be much more accessible for patients.”
“Precision oncology is set to witness significant developments in 2024.”
Laksh Aithani, CHARM Therapeutics
For Laksh Aithani, CEO of CHARM Therapeutics, “precision oncology is set to witness significant developments in 2024. The evolving landscape will likely feature the rise of PRMT5 inhibitors for MTAP-deleted cancers, with early data from Mirati Therapeutics, Inc. and Amgen, Inc. showing promise. The field will also see advancements in treating lung cancers with next-generation ROS1 and ALK inhibitors, such as the newly approved Augtyro (repotrectinib), and impressive results from Nuvalent. Additionally, innovative strategies like targeting the active state of KRAS, as pursued by Revolution Medicines, Inc., are anticipated to improve the efficacy of KRAS inhibitors, addressing a historically challenging target in oncology.”
“In oncology, where unprecedented innovations are reshaping drug development, a pivotal player emerging in this space is steroid receptor coactivator 3 (SRC-3),” said Steve Gorlin, CEO of CoRegen, which is developing anticancer therapies using a gene regulator platform. “Initially, its role in the immune system was nebulous, but recent revelations highlight its abundance in regulatory T-cells (Tregs), influencing their function. Recognizing SRC-3’s significance unveils a potential cornerstone for cancer immunotherapies. Studies indicate that inhibiting SRC-3, either through drugs or gene knockouts, significantly impacts tumor size and growth in animal models. This discovery potentially indicates a future where manipulating SRC-3 expression unlocks the immune system’s formidable anti-cancer potential, ushering in a new era of targeted therapies against solid tumors in 2024 and beyond.”
“We can expect major advancements in precision medicine in 2024 with the integrated use of recently available technologies to improve the odds of clinical success for innovative cancer medicines. These novel methodologies may spare patients in clinical trials from ineffective therapy and usher precision medicine into mainstream clinical practice,” said Spiro Rombotis, CEO of Cyclacel Pharmaceuticals, Inc. “For example, the broad use of sequencing modalities (from NGS to RNA seq to epigenetic ATAQ seq) allows interrogation of biospecimens from responding patients early in development. This in turn may influence trial design for mid- and late-stage development and potentially improve probability of success. Another example is the use of organoid PDX models to study antitumor activity before IND and even during early development. These represent a step-wise change from xenografts of old to identify sensitive cancer cell types and evaluate synthetic lethal relationships for combinations. They may also suggest potential tumor vulnerabilities to be studied in the clinic.”
“Precision oncology approaches have made great strides in recent years, but traditionally target pathways that are deregulated in tumor cells only. The reality is that cancer cells are just a small proportion of the total cells that support the tumor to grow,” Fiona McLaughlin, chief scientific officer at Avacta Group plc, said. “These non-tumor cells, which make up the tumor microenvironment (TME), consist of tumor-associated macrophages, T- and B-cells, natural killer cells, myeloid-derived suppressor cells and cancer-associated fibroblasts (CAFs). In pancreatic cancer, for example, CAFs make up 90% of the tumor mass. Newer approaches showing promise directly target drugs to the TME, including via an enzyme such as FAP on the surface of CAFs, targeting both tumor and non-tumor cells, potentially leading to widespread tumor reduction, whilst sparing normal tissue.”
“What patients really need are high response rates AND durability. 2023 saw important steps in that direction, which could bear fruit in 2024.”
Ben Zeskind, Immuneering Corporation
“Targeted therapies (eg, KRAS-G12C inhibitors, BRAF inhibitors) have high response rates in tumors with specific mutations, but struggle with durability: tumors become resistant by developing new mutations,” commented Ben Zeskind, CEO of Immuneering Corporation. “Immunotherapies (eg, checkpoint inhibitors) can provide more durable responses, but only for a subset of patients. What patients really need are high response rates AND durability. 2023 saw important steps in that direction, which could bear fruit in 2024.”
“First,” he said, “several pan-RAS programs are in development, designed for broader activity which makes it harder for new mutations to cause resistance. Second, FDA’s Project Optimus has increased focus on improving tolerability by prioritizing the most effective dose, not the maximum tolerated dose. Better tolerability enables combinations, and combinations improve durability by making it harder for tumors to mutate their way to resistance. Together, these trends could help make 2024 the year that targeted cancer therapy demonstrates clinically durable responses.”
Kenji Hashimoto, chief medical officer of Crescendo Biologics Ltd., noted “significant progress” for the treatment of metastatic castration-resistant prostate cancer with the approval of Pluvicto (Novartis’s targeted radiopharmaceutical lutetium Lu177 vipivotide tetraxetan), but observed that “there remains an unmet medical need for treatments that offer a long duration of response or, ultimately, can cure the underlying cancer.” He went on: “Immunotherapy has not been well established in mCRPC apart from microsatellite instability (MSI)-high specific disease, where checkpoint inhibitors are approved. Recently, encouraging efficacy was seen with the CD3 x STEAP1 bispecific molecule, AMG509. T-cell agonistic immunotherapy and other emerging treatments may alter the tumor microenvironment, potentially unlocking new approaches. Understanding the intricacies of tumor biology is crucial in identifying the optimal positioning of novel drugs within the sequence of treatment. As the treatment landscape in mCRPC evolves, we need to understand the mechanism of action of these new investigational therapies but also their impact on tumor biology. We look forward to seeing emerging biomarker data of these new agents in 2024.”
“The shift in oncology has been toward multitargeted approaches as we work to overcome resistance and reflect the complexity of the tumor microenvironment,” said Leena Gandhi, chiefmedical officer of NextPoint Therapeutics. “We’ve recently seen an explosion of interest in antibody-drug conjugates (ADCs) as the field embraces precision targeting for existing therapeutics. Although ADCs are not new, their potential continues to grow and many will enter the clinic in 2024. I am particularly eager to see progress in tumor targeting dovetail with immuno-oncology, allowing us to tailor therapies and expand the pool of patients able to benefit from these treatments.”
Several executives referred to ADCs.
“We can anticipate in 2024 that ADCs will expand into new indications and earlier lines of treatment, as well as use more diversified payloads, offering potential for increased efficacy and tolerability.”
David Epstein, Seagen Inc.
“Antibody-drug conjugates for the treatment of multiple cancer types are at an inflection point. We are seeing unprecedented growth in the ADC market, which will only continue to advance more rapidly in 2024,” said David Epstein, CEO of Seagen Inc., which was acquired by Pfizer Inc. for $43bn in 2023.
“Just in the last year, we have witnessed incredible breakthroughs with ADCs, particularly in bladder and cervical cancer, with positive overall and progression-free survival data. Targeted cancer treatments like ADCs provide patients with opportunities to receive therapies that offer greater cancer targeting precision and reduced treatment burden. ADCs are gaining momentum and recognition industry wide, and Seagen’s merger with Pfizer speaks to the increased interest many companies have in this technology. We can anticipate in 2024 that ADCs will expand into new indications and earlier lines of treatment, as well as use more diversified payloads, offering potential for increased efficacy and tolerability.”
“ADCs in oncology represent one of the biggest sources of growth and most promising areas for breakthrough treatments in the entire biopharma industry. Biopharma companies will continue to expand their development and M&A activity across a wide variety of tumor types and disease areas as clinical development pipelines expand to unlock the true potential of ADCs,” said Ken Keller, president and CEO of Daiichi Sankyo Inc. and global head of the Japanese group’s oncology business. “Advancement of pipelines and new indications for successful ADCs will firmly establish groundbreaking therapies as a transformed pillar of cancer treatment.” Daiichi Sankyo (with partner AstraZeneca PLC) sells the blockbuster breast cancer ADC, Enhertu (trastuzumab deruxtecan).
“Historically, the antibody-drug conjugate (ADC) field has been limited due to unstable conjugation resulting in systemic off-target toxicity,” said Daniel O’Connor, CEO of Ambrx Biopharma, Inc., which has “developed a technology to incorporate synthetic amino acids (SAAs) into proteins at selected sites, which enable us to utilize highly stable novel conjugation chemistry specific to our SAA, instead of only being able to use conjugation chemistry amenable to amino acids at random positions.” He noted: “With stable conjugation technology, the field is now poised to move beyond traditional cytotoxic payloads. For example, immune-stimulating agents and protein degraders are currently being incorporated into antibody-conjugates and tested in combo with ADCs. In 2024 we will see a new ADC expansion into more targets and indications and antibody-conjugates that will continue to drive strong interest across the industry.”
Acknowledging that “the potential of ADCs as an effective alternative to chemotherapy will continue to be in the spotlight during 2024, as the number of assets entering and moving through the clinic continues to increase at pace,” Cristian Massacesi, AstraZeneca PLC’s chief medical officer and oncology chief development officer, highlighted patient selection as an area to watch. “What will also start to be realized in 2024 are novel, sophisticated biomarkers to identify the right patients for ADCs, such as TROP2-targeted agents, using automated computational pathology solutions, like Quantitative Continuous Scoring (QCS), to more precisely – and at scale – continuously assess biomarkers on a single-cell level, using cutting-edge imaging technology and AI.
“QCS has potential to uncover novel ways of selecting patients – identifying patients likely to be most responsive to certain treatments as well as new patient populations, such as patients who express low levels of a biomarker that may not be detected using traditional pathologist scoring,” he explained, adding, “It’s an exciting time to be in oncology R&D!”
“In 2024 and beyond, we will see the benefits of decades of fundamental research starting to unravel the complex structure and activities of a functioning cell. We can now piece together the impact of mislocation of single enzymatic and factor activities on the cell seen as an organization,” said Max Herzberg, founder and chair of Vidac Pharma. “A perfect example is the abnormal metabolism of cancer cells, which causes high proliferation and an acid environment. This principle, the Warburg effect, ie, hyper-glycolysis, has been known since the 1920s and is only used in PET scanning. We now have a view of its principal causative factors: the HK2 binding to the VDAC mitochondrial pore.
“Vidac Pharma has created molecules, now at the clinical stage, that dissociate the HK2-VDAC complex, which renormalizes cellular metabolism and reactivates apoptosis. We believe this is a real breakthrough in controlling cancer cells.”
“I see 2024 as the year the industry makes meaningful progress in shifting standard of care expectations for pediatric oncology patients and their families through accelerated infrastructure, more efficient drug development and anticipated approvals.”
Jeremy Bender, Day One Bio
Jeremy Bender, CEO of Day One Biopharmaceuticals, LLC, noted that “cancer is the number one cause of death by disease among children, and while there has been increased innovation in oncology therapies, children with cancer are often left waiting.”
Inflammation And Immunology
“One of the biggest shifts we expect to see in in 2024 is a greater push to identify novel treatment paradigms in complex therapeutic areas such as immunology,” said Jesper Lange, CEO of MC2 Therapeutics A/Ss, a Danish company developing candidates for inflammation and immunology indications.
“We have seen huge advances in understanding of immune-mediated disease in recent years and there’s scope for significant innovation in the future,” Lange said. “A multi-disciplinary, entrepreneurial approach will be key to this, with a better understanding of the space contributing to the identification of new treatment opportunities for immune-mediated diseases with excellent pharmacological properties that are truly differentiated. We’re already starting to see evidence of this in the clinic as companies explore drug candidates that have the potential to unlock further innovation and we expect to see this trend continue into 2024.”
“The trend of novel small molecule successes for immuno-inflammatory diseases is expected to continue into 2024 and will result in more treatment options for patients with diseases that have historically been hard to treat.”
Clive Dix, C4X Discovery
“The inflammation and immunology space has been gaining impressive traction with small-molecule medicines being approved in 2023, including Velsipity [Pfizer’s etrasimod, an S1P inhibitor] for inflammatory bowel disease and deucravacitinib [Bristol Myers Squibb Company’s TYK2 inhibitor Sotyktu] for psoriasis,” said Clive Dix, CEO of UK-based C4X Discovery Holdings plc.
“The trend of novel small molecule successes for immuno-inflammatory diseases is expected to continue into 2024 and will result in more treatment options for patients with diseases that have historically been hard to treat. The small-molecule candidates that show potential best-in-class profiles when compared to already approved biologic therapeutic options are highly exciting and could prove to be game changers for patients around the world,” he continued.
“We are witnessing exciting breakthroughs in our understanding of the role that the energetic status of immune cells plays in driving autoimmune and inflammatory disease,” said Neil Weir, CEO of Sitryx Therapeutics. “This is leading to the identification of novel targets for future therapies that could drive lasting disease remission for patients – addressing a major unmet need and offering significant improvements over existing therapies. We look forward to seeing how our industry unlocks the potential of these new targets and advances them into the clinic.”
“In 2024, I look forward to therapeutic advancements for people living with rheumatic diseases, particularly ones in which patients still do not experience optimal disease outcomes like psoriatic arthritis (PsA), non-radiographic axial spondyloarthritis and ankylosing spondylitis,” said Jeffrey Stark, head of medical immunology at UCB S.A. “To achieve this, I anticipate we will see more routine use of more stringent outcome measures and higher treatment targets in clinical research.”
Stark added: “In PsA, for example, researchers have historically used standard endpoints like ACR20, which represents only a 20% improvement in patient disease symptoms and severity since entering the trial. By raising the bar to design trials with more stringent endpoints, such as ACR50, meaning a 50% improvement, we can encourage the community to target a higher standard of care, making a difference for patients and ultimately reshaping future rheumatology treatment.”
“As our understanding of the immune system advances, therapeutics in the autoimmune and allergic disease spaces will continue to be a focus for biopharma in 2024. The increasing prevalence of allergic disease, like eosinophilic esophagitis, is propelling the development of therapeutics aiming to orchestrate a balanced immune response and avoid immune system overreaction,” said Jonathan Rigby, CEO of Revolo Biotherapeutics. “In the realm of autoimmune diseases, the focus is on solutions providing longer-term remission while mitigating the substantial side-effects of current treatments. Intriguingly, new data suggests that many allergic or autoimmune diseases don’t come alone but co-occur with one another. This is prompting the development of therapeutics that reset the immune system to a balanced homeostatic state. These innovations, addressing multiple conditions simultaneously and moving beyond symptomatic relief, will continue to reshape therapeutic pipelines.”
Checkpoint inhibitors are commonly associated with cancer therapeutics, but they are also relevant in autoimmune and other conditions. “After 10-15 years of research and early development, in 2024 the biopharmaceutical industry will begin to see robust Phase II data readouts from novel checkpoint agonist classes, such as PD-1 and BTLA,” said Daniel Faga, CEO of AnaptysBio, Inc., which is developing immune cell modulators for rheumatological, gastrointestinal and dermatological conditions.
“These therapeutics take a top-down approach by specifically targeting activated immune cells to broadly drive immunological outcomes with the potential to restore immune balance in diseases like rheumatoid arthritis and atopic dermatitis. I am hopeful that these collective data will be game-changing by breaking through the long-standing efficacy ceiling to raise the standard of care for the millions of patients living with these systemic, heterogeneous diseases,” he said.
“The world of inflammation and immunology is highly competitive and changing rapidly. There is a significant need for new treatments to address lesser understood indications with high unmet patient need. Recent new scientific insights have supported new innovations in terms of molecular targets and treatment modalities, particularly in dermatological conditions, such as hidradenitis suppurativa,” said Kristian Reich, chief scientific officer of MoonLake Immunotherapeutics AG. “AbbVie Inc.’s Humira (adalimumab), which targets tumor necrosis factor-alpha (TNF-alpha), remains the standard of care for many indications. Novartis AG’s Cosentyx (secukinumab), an antibody targeting IL-17A, is approved and making a commercial impact. Most recent, UCB S.A.’s Bimzelx (bimekizumab), a traditional antibody that targets both IL-17A and IL-17F, has received approvals in the US and Europe and is being launched. New innovations such as MoonLake’s sonelokimab, a Nanobody targeting IL-17A and IL-17F, is poised to enter Phase III trials this year. This competitive area is one to watch as it continues to evolve during 2024.”
“As we gain insight to how dysregulation of the immune system can result in diseases like type 1 diabetes and multiple sclerosis, we are able to use this knowledge to develop better therapies and identify patients who will benefit the most.”
Andrew Mackie, Imcyse
“Momentum is building with novel, targeted approaches being developed to treat autoimmune diseases,” said Andrew Mackie, chief business officer of ImCyse S.A. “In the near future, many proof-of-concept clinical trials will show how new drugs can treat and potentially cure diseases. Whatever their results, the data from individual trials will help industry and medical communities to better understand the patients and diseases. As we gain insight to how dysregulation of the immune system can result in diseases like type 1 diabetes and multiple sclerosis, we are able to use this knowledge to develop better therapies and identify patients who will benefit the most.”
He was not the only one to mention the increasing ability to identify the right patients for a particular treatment approach. “Patient stratification based on predictive markers (genetic signatures or other biomarkers) is expected to play a much larger role in inflammation and immunology space, following the successful implementation of a companion diagnostics strategy by Prometheus Biosciences, Inc (now Merck & Co., Inc.) in their ARTEMIS-UC anti-TL1a Phase II study,” commented C4X’s Dix.
“A key trend for the biopharma industry in 2024 will be the development and initiation of further clinical trials and therapeutics testing various pathways and treatment strategies targeting inflammation to reduce cardiovascular disease.”
Michael Garshick, New York University Langone Health
Richard Philipson, chief medical officer of Calliditas Therapeutics AB, focused on immunoglobulin A nephropathy in particular. “There are many different approaches being tested in IgAN, including autoimmune suppression focused on [targets] APRIL and BAFF, as well as investigational products targeting various complement activation pathways. We would expect there to be data provided from a variety of clinical trials over the next several years.”
Michael Garshick, assistant professor of medicine and dermatology and director, cardio-rheumatology program at the center for the prevention of cardiovascular disease, Leon H. Charney Division of Cardiology, New York University Langone Health, drew attention to the fact that “systemic inflammation is a key driver of cardiovascular disease.”
He said: “Cardiovascular disease is a leading cause of death in the US despite highly effective prevention strategies including new advances in cholesterol lowering, diabetes, and obesity therapies. Recent clinical trials as well as the FDA approval of specific anti-inflammatory therapies suggest that targeting inflammation reduces cardiovascular events. Thus, a key trend for the biopharma industry in 2024 will be the development and initiation of further clinical trials and therapeutics testing various pathways and treatment strategies targeting inflammation to reduce cardiovascular disease.”
Finally, Saad Harti, CEO of Legacy Healthcare SA, highlighted the potential for botanicals in autoimmune disease treatment. “While we should witness more cell therapy, genome editing, mRNA, microbiome, targeted etc therapies, I am particularly excited about potential advances in botanical drugs,” he said. “Instead of addressing a single druggable target, the multiple molecules/metabolites a botanical drug contains deliver a pleiotropic effect, allowing potential a disease-modifying effect and a durable response in chronic diseases, especially autoimmune diseases.”
Obesity/Cardiometabolic
“Novel breakthroughs in the metabolic space have resulted in next-generation treatments for obesity which we expect will significantly expand biotech market growth in 2024.”
Sam Clark, Terran Biosciences
The ongoing commercial growth and therapeutic area expansion of Novo Nordisk’s glucagon-like peptide-1 (GLP-1) agonist semaglutide and Eli Lilly’s GLP-1/GIP (gastric inhibitory polypeptide) agonist tirzepatide have reignited interest in this field. As Sam Clark, CEO of CNS-focused therapeutics company Terran Biosciences, Inc., noted, “novel breakthroughs in the metabolic space have resulted in next-generation treatments for obesity which we expect will significantly expand biotech market growth in 2024.”
“GLP-1s, supported by compelling cardiovascular and chronic kidney disease (CKD) trial data, will continue to attain widespread prescription status once cost-effectiveness is well established in the payer sector,” said Marc Samuels, CEO of ADVI Health, a health policy consulting company specializing in market access, reimbursement, and data analytics for life sciences. “The introduction of oral formulations in 5-7 years could further solidify GLP-1s’ prominence, even if it is in the face of intense competition.”
“Obesity will remain in the spotlight in 2024 when we think market sales may almost double to at least $11bn, driven by Novo Nordisk as Eli Lilly’s Zepbound makes its debut,” said Michael Shah, a pharmaceutical industry analyst at Bloomberg Intelligence. “While both are investing to expand capacity, supply will likely remain a constraining factor, near term. Obesity-related label expansion for Novo’s Wegovy is anticipated, with supplemental approvals for heart failure, and the prevention of major adverse cardiovascular events in patients with established heart disease, as supported by the SELECT trial. SELECT could drive a more proactive approach toward weight management versus the current treatment paradigm, which focuses on hypertension, lipids and diabetes. Novo could also win approval for the first GLP-1 pill for obesity, though its launch hinges on supply. Key clinical catalysts include mid-stage readouts for Amgen and Viking Therapeutics, Inc.’s combination drugs, while Zealand Pharma A/Scould report Phase II data for dapiglutide, which appears to be the only GLP-1/GLP-2 in development.”
“In 2024, I believe our industry will see exciting new developments in the treatment of obesity and type 2 diabetes. While glucagon-like peptide-1 agonists have emerged as an important therapeutic intervention for these conditions, they have also sparked increasing interest in other types of incretin-based therapies,” said Jay Galeota, CEO of Kallyope Inc. “Despite the efficacy of the current drugs, it is estimated that only two percent of patients with obesity in the US receive medical treatment with therapeutics due in part to limitations such as cost, supply disruptions, issues with tolerability, reimbursement, and patient and practitioner resistance to the use of injectables. With a clear need for new classes of effective, easy to use, and well tolerated oral agents with unique and potentially complementary mechanisms of action, I look forward to seeing how therapies like nutrient receptor agonists are further advanced to meet patients’ needs more broadly in the coming year.”
“2024 will stand in the history of humans as an important moment in stemming the tide of global obesity.”
John Amos, Vivus
“The science and treatment of obesity will continue to undergo significant advances for the next three to five years,” stated John Amos, CEO of Vivus, Inc., which obtained US FDA approval for its obesity drug Qsymia (phentermine/topiramate) in 2012. “2023 marked the point in human history where a significant portion of the clinical and general population realized that obesity and all the associated health implications cannot be treated by diet and exercise alone. Therapeutic intervention is required to modify both behaviors and manage our genetically inherited desires to consume food. 2024 will mark a significant expansion for researchers, health policy executives, health insurers both government and private to develop a deeper understanding of how to use the current and future collection of obesity centered pharmaceuticals to create a series of protocols for population health management.”
He concluded: “As these protocols are developed and propagated to the clinicians and ultimately the public, 2024 will stand in the history of humans as an important moment in stemming the tide of global obesity.”
Meanwhile, Jay Backstrom, CEO of Scholar Rock, LLC, saw opportunities where the current obesity drugs still fall short. “As GLP-1 therapies for obesity continue to gain traction, the substantial loss of lean muscle mass poses a challenge toward achieving sustainable and healthy weight loss,” he said. “Therapies that can preserve muscle mass will be of utmost importance as GLP-1 uptake accelerates and patients undergo longer durations of treatment. In 2024, after decades of long interest, I anticipate our industry will realize the market opportunity for novel muscle-targeted therapies that block myostatin across a spectrum of cardiometabolic disorders and neuromuscular diseases.”
“The advent of incretin agonists as a new highly potent class of obesity drugs, demonstrating considerable weight-loss benefits, has created both an enormous demand and unprecedented opportunities,” declared Steffen-Sebastian Bolz, CSO of Aphaia Pharma AG, which is developing a coated bead formulation of glucose to treat obesity and prevent diabetes. “We are currently witnessing the development and clinical testing of dual and triple incretin agonist combinations, accompanied by a shift from injections to oral solutions. Innovative treatment concepts within the rapidly evolving obesity drug landscape specifically target the underlying pathologies of obesity, promising to combine long-lasting efficacy with minimal side effects. In the years to come, fresh ideas and multiple mechanism-based treatment solutions will unfold benefits beyond weight loss. The immense clinical value of reducing obesity primarily results from inherently addressing associated sequelae like diabetes and heart and liver disease. In that sense, breakthrough advancements will continue to transform the treatment landscape for the 1 billion people worldwide living with obesity. We are excited to make meaningful contributions.”
“Obesity was the trending therapeutic area in 2023 and rightfully so given its impact on more than 100 million people in the US alone,” said Vipin Garg, CEO of Altimmune Inc. “With a population so vast and diverse, patients will need a variety of therapeutic options to address the numerous health issues associated with obesity such as dyslipidemia, high blood pressure, high liver fat and cardiovascular conditions. In 2024, we believe we’ll see more companies looking beyond weight loss to address the broad health impact of obesity by developing differentiated therapeutics with the potential to treat the broader health impact of obesity, unlocking unprecedented benefits for patients and our healthcare system.”
Small-molecule approaches were not the only area of interest among our commentators. “For 100+ years, diabetes has been treated with insulin injections and patients have been offered limited treatment innovation,” observed Cynthia Pussinen, CEO of regenerative medicine company Sernova Corp. “Looking to the future, cell and gene therapy technologies have the potential to transform how people live with diabetes. In 2023, the industry saw the approval of the first cellular therapy to treat type 1 diabetes. This was a step in the right direction, but further innovation is needed. Several companies are developing novel platforms that utilize medical devices, stem-cell-based therapeutic cells, and immune protection technologies. The goal is to provide a scalable commercial diabetes treatment that eliminates the need for daily insulin injections. Recent clinical data has shown that a ‘functional cure’ for diabetes may be possible. In 2024, I anticipate that we will see additional validating clinical data for these approaches in diabetes, as well as interesting findings on how cell therapies can be utilized to treat other diseases.”
Daphne Zohar, CEO of PureTech Health plc, meanwhile, warned: “I personally think the GLP-1 space is getting very crowded and is overhyped right now, being positioned as a panacea.”
“We need a paradigm shift in cardiometabolic disease, including type 2 diabetes, and 2024 could be the year.”
Martin Ridderstråle, Novo Nordisk Foundation
But Martin Ridderstråle, head of medical science at Novo Nordisk Foundation, thought medical science should aim even higher: “We need a paradigm shift in cardiometabolic disease, including type 2 diabetes, and 2024 could be the year. With cancer, we work on prevention and cure, but with diabetes and other cardiometabolic conditions, we expect people to be content with managing their condition. Why this disparity, when cardiometabolic disease is the number one cause of death globally?” he asked.
“Research into the biological mechanisms of type 2 diabetes is starting to yield new knowledge that can move us towards preventive or curative measures, while promising results in the GLP-1 receptor agonist space suggest exciting possibilities for reducing the risk of major adverse cardiovascular events. A new focus on how climate change can exacerbate global health problems such as cardiometabolic disease – including at the recent COP28 – could also take us to a tipping point where we see the mindset shift and therapeutic advances that millions of people around the world desperately need,” Ridderstråle said.
How payers will address the cost challenges of the wave of new therapeutic developments remains in question. “Nearly 42% of the United States is obese and if left unaddressed, this challenge can lead to dire and expensive health consequences given the relationship between cardiovascular disease, type-2 diabetes and obesity – or cardiodiabesity. While GLP-1 therapies offer significant hope for many, they also bring additional cost, complexity and lack of predictability for health plans and employers grappling with how to provide coverage,” observed Adam Kautzner, president of pharmacy benefit management group Express Scripts. “We anticipate health plans and sponsors will move towards a more coordinated approach to managing this trifecta of diseases that prioritizes preventative health measures, including mental health and health coaching, to promote long-term cost savings while also carefully weighing clinical considerations so that the right patients are treated with the right approach at the right time.”
CNS
“CNS [central nervous system] advancements have been significant in recent times. Typically seen as one of the more challenging diseases areas with multiple specificities with hard-to-treat diseases, CNS has had a number of breakthroughs in the last few years,” noted James West, managing director at investment bank Lincoln International. “AI drug design and discovery has been instrumental in these developments. Global spend across CNS is predicted to rise 8.6%, which places it third amongst other therapeutic areas, only behind oncology and immunoregulators.”
“The way we saw major advances in oncology in the 2000s and 2010s, I believe we will see major progress in treatments for central nervous system and neurodegenerative diseases in the 2020s.”
Rachel Lenington, Athira Pharma
“The way we saw major advances in oncology in the 2000s and 2010s, I believe we will see major progress in treatments for central nervous system and neurodegenerative diseases in the 2020s,” predicted Rachel Lenington, chief operating officer of Athira Pharma, Inc., which develops small-molecule drugs for neurological disease. “The recent advances and current standard approach of targeting amyloid-beta or tau is promising, but that is just the tip of the iceberg. Patients are still declining and need more innovative and disease-modifying treatment options. We’re starting to see the shift, with targets having pleiotropic effects in Alzheimer’s to tackle more than one component of the disease via new therapeutics or combination approaches. Together, these encouraging new approaches may help us treat these multifactorial disease pathologies more holistically.”
“I’m particularly excited about neuroscience where the unmet need is massive and [there are] some exciting new therapeutics in the pipeline,” said PureTech Health’s Zohar. “There is a huge unmet need for patients struggling with mental health disorders. For example, generalized anxiety disorder (GAD) impacts 6.8 million adults, social anxiety disorder affects 15 million adults and panic disorder impacts 6 million adults. It’s really debilitating and devastating.”
Mark Rus, CEO of Delix Therapeutics, Inc., was similarly hopeful. “The industry is continuing to expand its understanding of how many CNS indications are treated, as we seek to address the underlying pathophysiology of these diseases,” he said. “Traditional treatments for conditions such as major depressive disorder or substance use disorder, including ketamine and serotonergic psychedelics, promote neuroplasticity to help repair damaged neurons in the brain; however, this can take anywhere from weeks to years, limiting the efficacy of these treatments. Moreover, they have been associated with troublesome side effects and cardiac risks. As we enter 2024, I expect we will continue to see a greater focus on mental health and increased research into novel compounds, such as the neuroplastogens, which are engineered with scientific rigor to produce functional neuroplasticity on a much faster time scale and effectively mitigate harmful effects, and physically repair the brain.”
“With better understanding of disease biology, new approaches for the potential treatment of chronic diseases of the CNS, recent approvals by the US FDA and ongoing interest in acquisitions and partnerships related to CNS therapies, 2024 could be the year for this field.”
Cory Nicholas, Neurona Therapeutics
“For years, the dream of creating disease-modifying therapies has stimulated development in the central nervous system disease field by biopharma companies. However, such initiatives have regularly ended with clinical trial failures and companies exiting the area despite the continued unmet medical need,” commented Cory Nicholas, CEO of Neurona Therapeutics. “With better understanding of disease biology, new approaches for the potential treatment of chronic diseases of the CNS, recent approvals by the US FDA and ongoing interest in acquisitions and partnerships related to CNS therapies, 2024 could be the year for this field.”
Nicholas highlighted cell therapies as an area to watch in this therapeutic space. “First-in-human data from trials of cell therapy approaches for both mesial temporal lobe epilepsy, the most common form of focal epilepsy, and Parkinson’s disease demonstrated that the proprietary cell treatments were well-tolerated and provided some early signs of efficacy in patients. 2024 will bring further progress with additional data as these investigative therapies advance through clinical development,” he said.
“The resurgence of interest in neuroscience has continued during 2023 and we foresee further advancement in 2024,” said Andrea Chicca, CEO of Synendos Therapeutics,which is developing endocannabinoid modulators for indications including post-traumatic stress disorder (PTSD).
“This year has seen the filing of the first new potential treatment for schizophrenia in decades, KarXT from Karuna Therapeutics, Inc., utilizing a mode of action that may deliver much needed improvements on the side-effect profile seen with current antipsychotics. This milestone leads the way together with other positive clinical data in neuropsychiatric disorders such as PTSD,”he said, adding: “This provides an important inspiration for companies such as ours.”
“For the biopharma industry tides to turn in 2024 it will require transformative technology, innovative approaches, and solutions that not only replace existing treatments but also enable the treatment of diseases previously thought to be untreatable. For example, we’re now starting to see a renaissance in neuropsychiatry with an entirely new class of compounds and technologies, including novel biomarker approaches and psychedelic-based therapeutics, which could drive a paradigm shift in the treatment of diseases that have traditionally been difficult to target, such as treatment-resistant depression and PTSD,” said Terran Biosciences’ Sam Clark.
“Additionally, with recent advances in the treatment of Alzheimer’s and Parkinson’s disease, technologies that may have been once less useful, such as early diagnostics, now become essential, as earlier detection enables earlier treatment.”
“We have seen promising advancements in scientific and clinical research in neurodegenerative diseases in 2023. This is essential, especially when you realize that approximately 55 million patients worldwide are suffering from various forms of dementia alone, not even including other neurodegenerative diseases such as multiple sclerosis, Parkinson’s disease, or amyotrophic lateral sclerosis (ALS). I strongly believe there will be more innovation in this area in the near future and I look forward to seeing the field progress,” commented Immunic Inc’s CEO Daniel Vitt. “Small molecule drugs have been and will continue to play a major role in the drug markets in 2024 as they have clear advantages over more complex molecules due to their lower molecular weight and simpler chemical structures, both making pharmacokinetics (PK) and pharmacodynamics (PD) more predictable and thereby dosing and administration simpler. Maybe most importantly, small molecules lead to lower production costs, which is critical especially in times of particularly cost-sensitive markets. This trend is in clear contrast to the rise of personalized medicine as an emerging new practice, which is very promising but also very costly.”
“Figuring out the key to delivering genes intravenously that are capable of crossing the blood-brain barrier and reaching the brain will unlock an incredibly promising path for treating neurological diseases using genetic medicine.”
Joseph La Barge, Apertura Gene Therapy
But according to Joseph La Barge, CEO of Apertura Gene Therapy, his field holds promise for neurological disorders. “Pharma continues to express interest in the development of next-generation gene therapies as there is an urgent need for improvement in the technology to address the needs of patients. I am particularly excited to see advances in gene therapy delivery. Figuring out the key to delivering genes intravenously that are capable of crossing the blood-brain barrier and reaching the brain will unlock an incredibly promising path for treating neurological diseases using genetic medicine,” he said.
“The rise in prevalence of neurodegenerative diseases and a growing geriatric population warrants the increased demand for safe and effective therapeutics. While existing treatments are designed to alleviate symptoms, there is a more pressing unmet need for a treatment that could potentially halt or slow the progression of disease,”said Anker Lundemose, CEO of Mission Therapeutics Ltd.
“Passive immunotherapy, treatment using externally produced monoclonal antibodies, has shown clear progress in slowing Alzheimer’s disease progression. The clinical successes of the two monoclonal antibodies, lecanemab and donanemab, are already providing much needed impetus to the entire neurodegeneration field, demonstrating the potential value of effective products,” said Andrea Pfeifer, CEO of AC Immune SA.
“This first step opens the future possibility of both halting progression of Alzheimer’s and perhaps preventing it from developing in the first place,” she continued. “For this, I believe we need active immunotherapies, a vaccine-based approach whereby the body produces its own antibodies, as well as combination treatments. Several active immunotherapies, which offer improved convenience and safety for patients and potentially lower cost, are moving through clinical development, showing the desired signs of immune activation and good safety profiles. This progress inspires optimism that the first active immunotherapies for Alzheimer’s disease could reach the market by 2030, transforming the way we address and hopefully prevent this devastating disease.”
“We’ll continue to see advancements in understanding basic neuroscience biology and novel ways to diagnose and treat Alzheimer’s disease,” also forecast Julie Gerberding, president and CEO of the Foundation for the National Institutes of Health. “From new drugs to brain-computer interfaces, the research is fascinating and consistently breaks new ground. At the Foundation for the National Institutes of Health (FNIH), our Biomarkers Consortium is working to accelerate the development of cost-effective, non-invasive tools to diagnose Alzheimer’s disease earlier in the course of illness, and the Accelerating Medicines Partnership for Alzheimer’s is using a data-driven approach to identify and validate promising new targets for drug development.”
“The advancement and approvals of new and innovative treatments for Alzheimer’s disease that we saw in 2023 will lead to more effective therapies in 2024 and beyond. It takes a village to make progress in this space – and with the complexities of Alzheimer’s disease being unraveled with each day, patients and families alike have good reason to be optimistic and look forward to continued progress,” said Gene Kinney, CEO of Prothena Corporation plc. “As we look ahead, we need to ensure that all patients that can benefit from these treatments have access to them, and in a way that avoids undue burden on patients, and I hope 2024 will be marked by a continued collaborative, industry-wide effort to ensure this happens.”
Treating Addiction
“With the rise of fentanyl and other new illicit substances adulterating the drug supply, addressing the full spectrum of substance use from harm reduction to recovery requires creative collaborations with industry and non-industry partners,” commented Joshua Cohen, chief medical officer at Braeburn Inc., which obtained US FDA approval for a long-acting buprenorphine injectable in 2023. “To learn how we may best advance drug development in substance use disorders, Braeburn is in discussions with public and private entities in an effort to transform these conversations into actionable plans. In 2024, I expect to see the advancement of molecules in the preclinical and clinical stages focused on new approaches to treating this chronic brain disease beyond the brain’s opioid receptor. Therapeutic advances are most impactful when they are available to those who need them most.”
“Advancements in smoking cessation tools are imperative as smoking remains the leading cause of preventable death, with more than 28.3 million adults in the US currently smoking cigarettes and more than 16 million Americans living with a smoking-related disease. While there has not been FDA-approved smoking cessation treatment in nearly two decades, we have seen positive results and data from smoking cessation trials over the past few years, and this progress shows that the void of new treatment options has the potential to be filled,” said John Bencich, CEO of Achieve Life Sciences, which is focused on developing a product for nicotine dependence. “I am optimistic that the smoking cessation landscape will continue to evolve in the coming years to improve and save lives, which in turn, will transform the health industry by reducing the resource utilization, personnel burden, and cost associated with smoking-related diseases.”
Infectious Diseases
“It is important to stay prepared and to ask the question – what is the next virus?”
Anu Osinusi, Gilead Sciences
“The COVID pandemic brought infectious diseases to the forefront of global health and has led to huge investment in this area of therapeutic research,” commented Anu Osinusi, vice president, clinical development, hepatitis, respiratory and emerging viruses, at Gilead Sciences, Inc. “COVID-19 remains a serious public health threat and there is ongoing work underway to develop new COVID antiviral therapies and to study the use of antivirals in treating and preventing long-COVID.”
“The COVID-19 treatment landscape requires continuing innovation. While we’ve come to accept that the virus will remain with us for the foreseeable future, we have more to learn about how the virus affects people, irrespective of risk factors in both the short-term and long-term,” said Shionogi & Co. Ltd.’s head of clinical development, Simon Portsmouth. “The scientific community must remain vigilantly focused on continuing to better understand how rapid elimination of the virus could affect transmission, symptom improvement and long COVID in individuals and the community.”
Meanwhile, Osinusi urged further vigilance beyond addressing COVID-19. “It is important to stay prepared and to ask the question – what is the next virus? The World Health Organization keeps a list of viruses and bacteria with pandemic-causing potential to help guide scientists, governments and academic institutions in investing energy and funds to study the pathogens most likely to cause the greatest devastation to humans. It is an ongoing pursuit, but it remains critical to bring to bear our collective scientific expertise to make sure we are ready to address future threats,” she said. “For any resulting innovations, ensuring access in resource-limited countries will be a critical step in combating future health challenges and will require building on past successes to widely distribute important medicines.”
Advances in the field of vaccines were flagged up by several commentators – as well as the need to facilitate access and uptake.
“Vaccination is one of the most cost-effective preventative health investments we can make as an industry and a society. As I look to 2024, I am excited to see years of innovation and hard work on the development of new vaccines pay off even more with a profound impact on public health,” said Robert Walker, senior vice president and chief medical officer at Novavax, Inc. “In 2023, the industry celebrated key wins, including the first licensed RSV (respiratory syncytia virus) vaccines and a new malaria vaccine – representing advances that will protect the very young to the very old.”
Walker noted the R21/Matrix-M vaccine developed by the University of Oxford and containing Novavax’s Matrix-M adjuvant as “an additional tool in the malaria prevention box that has the potential to help control a disease that causes more than 600,000 deaths annually, mostly in children”, and said that “similar collaborations to identify new ways of harnessing combinations of antigens, adjuvants, and novel technologies are likely to continue to pay dividends in 2024. I’m encouraged with this progress and excited about advances that can make a difference when looking to dramatically improve global public health.”
Gary Dubin, president of Takeda Pharmaceutical Co. Ltd.’s global vaccine business unit, also highlighted vaccines against mosquito-borne infections: “While small, mosquitoes prove to be among the deadliest creatures, spreading illnesses that affect hundreds of millions of people worldwide. In recent years, the rise of mosquito-borne diseases, like malaria, dengue fever and chikungunya, has posed a significant global health threat, which is exacerbated by the impacts of climate change as disease outbreaks continue to cause widespread concern. However, we’ve seen a glimmer of hope this year with significant advancements in prevention, including vaccination,” he said.
“The World Health Organization recommended a new vaccine for the prevention of malaria in children, and just in November, the US Food and Drug Administration approved the world’s first vaccine against chikungunya, with regulatory decisions in other countries also anticipated soon. We also have seen important progress this year at Takeda for our dengue vaccine, which brought launches in 20 countries worldwide in 2023 and more activities anticipated in 2024.
“Having spent over 30 years in vaccine development, it has been incredible to see how far we’ve come as an industry in our journey to combat some of the world’s toughest diseases. I have no doubt that our industry will continue to make meaningful progress in 2024 with first-in-class advancements in new vaccine technologies and disease targets.”
Gary Dubin, Takeda
“And 2023 also saw the first vaccines targeting RSV licensed, another impressive achievement. Having spent over 30 years in vaccine development, it has been incredible to see how far we’ve come as an industry in our journey to combat some of the world’s toughest diseases. I have no doubt that our industry will continue to make meaningful progress in 2024 with first-in-class advancements in new vaccine technologies and disease targets.”
“In 2024, we expect to see continued innovation in vaccines, building on recent advances in RSV, COVID-19 and hepatitis B, and to reap the benefits of having improved community immunity – a direct result of having broadened vaccine options and availability for adults,” said Ryan Spencer, CEO of Dynavax Technologies Corporation. “But we will also experience the challenges that come with a more complex vaccine landscape – including the need to address inequitable access, misinformation, and vaccine fatigue. We have responsibility to continue education efforts, so adults know which vaccines to get when and how to access them, and to advocate for equitable and sustained vaccine funding to support all adults – particularly the uninsured. While there is more work to do, 2024 will be another momentous year as it relates to enabling healthy communities and preventing outbreaks of infectious diseases.”
“Sustained vaccine innovation is critical as seasonal respiratory viruses continue to threaten at-risk populations and finite healthcare resources,” said Stefan Merlo, vice president of commercial development at CSL Seqirus. “This commitment to public health protection is at the heart of what drives us at CSL Seqirus. Our ambition is to evolve beyond our achievements in seasonal influenza to a future where we expect our innovative technologies, including self-amplifying mRNA, to improve public health protection against many burdensome respiratory viruses.”
Others recalled the pressing need to address antimicrobial resistance (AMR).
“In 2024, biopharma leaders will allow growing public health trends to influence their strategies and continue to spur innovation. For example, antimicrobial resistance continues to increase, with deaths from AMR infections projected to outpace cancer deaths by 2050,” pointed out Joe Todisco, CEO of CorMedix Inc., which received US FDA approval of its non-antibiotic antimicrobial product to reduce catheter-related bloodstream infections in adult patients with kidney failure on hemodialysis in November 2023. “The overuse of antibiotics can supercharge resistance, which is a boon to perpetuating AMR. This means biopharma, policymakers, regulators, as well as hospitals and health systems, will look to innovation to help prevent healthcare-associated infections that are typically treated with antibiotics. This is an environment ripe for cross-sector collaboration, investment and innovation – with biopharma leading the way after several major breakthroughs in the space over the past year.”
“Antimicrobial resistance is an escalating global threat, and there is an urgent need to develop therapies that target resistant microbes before they cascade into a range of conditions that require innovative treatment strategies,” said James Graham, CEO of Recce Pharmaceuticals Ltd., an Australian firm developing synthetic anti-infective drugs. “Notably, urinary tract infections (UTIs) have demonstrated resistance to the current standards of care and often require several rounds of treatment. This leads to recurrent or complicated UTIs, where the infection may spread to other organs and into the bloodstream and result in sepsis or urosepsis. Looking ahead, I am most excited about seeing the intensified efforts, technological advancements, and collaborative initiatives of the biopharma industry. In 2024, I believe we will witness significant strides in the development of efficacious solutions to combat AMR and improve the quality of life of patients impacted by these unmet medical needs.”
“In 2024, we will see collaborations across sectors and disciplines play a role in preserving the efficacy of existing and new antifungals.”
Francesco Maria Lavino, F2G
“The HBO series The Last of Us propelled fungi into the mainstream spotlight, sparking conversations about their role in infections, the disconcerting trend of developing resistance to current antifungal therapies, and the pressing issue of limited drug classes available for treatment,” said Francesco Maria Lavino, CEO of antifungal developer F2G Ltd. “One of the factors driving resistance is the dual use of antifungals in medicine and agriculture. This interconnectivity has prompted the urgent need for a One Health approach to combat fungal diseases, overcome resistance, and ensure the protection of humans, plants, and animals. In 2024, we will see collaborations across sectors and disciplines play a role in preserving the efficacy of existing and new antifungals.”
Meanwhile, Jeff Boyle, chief science officer of Addimmune, an HIV gene therapy developer spun out of American Gene Technologies International Inc., is hopeful about advances in HIV. “Over the past 35 years, we’ve witnessed advancements in treating HIV transforming it from a death sentence to a chronic illness with antiretroviral therapy (ART). Nonetheless, people living with HIV still face challenges including treatment side-effects, failure and difficulty with adherence. Natural resistance to HIV is rare and centered on mutations in CCR5, which is a coreceptor on the surface of CD4 T-cells crucial for HIV’s entry. Bone marrow transplants in HIV infected patients using donors with this rare CCR5 mutation have shown that a cure is possible and driven research to find a better way,” he said. “This includes genetic interventions targeting CCR5 in all cells or CD4 T-cells. Looking ahead, researchers are seeking to pair CCR5 targeting including HIV specific CD4 T-cells with a variety of backup strategies to hopefully prevent HIV infection and enable the immune system to establish long-term viral suppression.”
Fibrosis And Respiratory
“There is a big focus on healthier aging to enable patients suffering from chronic, long-term diseases to have a much-improved quality of life. We are seeing a renewed interest from big pharma in respiratory diseases,” said Peter Jackson, CEO of UK anti-infectives specialist Infex Therapeutics Holdings plc, adding that “there will be a number of novel respiratory assets entering/progressing through the clinic targeting different/new mechanisms of action.”
“Fibrosis is increasingly becoming an area of focus given the current limited treatment options and large, growing unmet need,” stated Helen Timmins, vice president, senior medical director, at Redx Pharma Plc. “In particular, there are several novel programmes in development for idiopathic pulmonary fibrosis (IPF) with data readouts in 2024, which will be interesting given IPF is a historically challenging area in drug discovery due to problems with tolerability and side-effects.”
“Idiopathic pulmonary fibrosis remains an area of high unmet need with a limited number of programs in clinical development and significant commercial opportunity.”
Ahmed Mousa, Vicore Pharma
“I believe that heavy historical investment in oncology, intense competition in the disease, and other factors will drive a continued diversification to other therapeutic areas, including in obesity, immunology, inflammation, fibrosis as well as rare indications,” predicted Ahmed Mousa, CEO of Vicore Pharma. “While the overall financing environment remains difficult, because of successes in these diseases and large commercial opportunities, these spaces will be ripe for continued growth. For example, idiopathic pulmonary fibrosis remains an area of high unmet need with a limited number of programs in clinical development and significant commercial opportunity. That the disease’s orphan status provides regulatory advantages and a commercial edge in view of the IRA [the Inflation Reduction Act in the US, which mandates price negotiation for some drugs under Medicare] will continue to make it an attractive therapeutic area in the future.”
Women’s Health
“I hope to see more shareholders, analysts and journalists asking CEOs how they plan to be part of this emerging field.”
Jessica Federer, Bayer
“2023 marked the 30th anniversary of the NIH revitalization act requiring women and minorities to be included in clinical research, creating significant momentum going into 2024 to improve the investment and focus on women’s health,” said Jessica Federer, Bayer’s former chief digital officer and a board member and investor for companies in the health sector.
“In the coming year, we will see a concerted effort to advance investment in women’s health at every major event starting with J.P. Morgan and Davos,” she told Scrip in December 2023. “This includes the newly announced White House initiative on women’s health, as well as the women’s health roadmap from the National Institute of Health and Gates Foundations, the Milken Institute and Manatt Health project for women’s health financing, the McKinsey and the World Economic Forum women’s health report, and the NYSE Women’s Health Investor Summit. I expect the market to better understand the breadth of the economic opportunity from the still nascent field of women’s health, with implications spanning brain health, autoimmune, cardiovascular, oncology and more. And I hope to see more shareholders, analysts and journalists asking CEOs how they plan to be part of this emerging field.”
“Efforts to improve women’s health research will be front and center in 2024,” said Ramita Tandon, chief clinical trials officer at Walgreens. “Despite making up more than half of the population, issues predominately impacting women continue to be under-researched, which has had significant consequences on women nationally. Both the public and private sectors will be involved in this movement, as President Biden recently announced a new White House Initiative to close gaps in women-focused health research emphasizing collective action from across industries. Trusted healthcare organizations with roots in local communities and female consumer bases, like Walgreens, will be instrumental to furthering the Initiative’s goals through targeted outreach, education, and tailored clinical trial recruitment.”
“It’s time for a billion-dollar reproductive health fund,” declared Agnès Arbat, CEO and chief medical officer of Oxolife, which is developing a drug to enhance embryo implantation. “Recognizing the urgency, women’s health has achieved impressive momentum in recent times. New initiatives are emerging to foster innovation and address unmet medical needs, like the first-ever White House initiative on women’s health research launched in November 2023. New treatments are poised to contribute to transformative change during 2024, such as the expectation that significant progress will be made in enhancing embryo implantation, the main cause today of ART [assisted reproductive technology] failure. Late-stage clinical developments are advancing in this area and pioneering approaches are expected to be announced, playing a pivotal role in reshaping the future of fertility in the years to come.”
Arbat’s colleague at Oxolife, chief scientific officer Ignasi Canals, said: “In the realm of women’s health and fertility, innovation has been under-represented in R&D relative to the burden. Despite making up more than half of the population, women’s health has been chronically underserved. The femtech sector is estimated to be worth $1tn by 2027, but the ‘taboo’ nature of women’s health and gender imbalances in investment are providing obstacles to the industry. Contrary to other fields, the regulatory path is underexplored, with no established endpoints and biomarkers, or accelerated tracks for approval. Consequently, women’s health has been flooded with a lot of early clinical or non-clinical ideas but there is a scarcity of innovations among products in late-stage development.”
Nevertheless, “exciting advancements are on the horizon for women’s health, addressing long-standing and dangerous gaps that impact women at nearly all stages of life,” according to Colby Holtshouse, global medtech commercial lead at women’s health company Organon. “For instance, a device designed to treat postpartum hemorrhage, a leading cause of maternal morbidity, is bringing much-needed innovation to an area that has remained stagnant for over two decades. Interesting new technologies are in development for monitoring pregnant women and predicting complications. And, for the first time ever, there’s an oral medication for postpartum depression [Sage Therapeutics/Biogen, Inc.’s Zurzuvae], a condition affecting one in seven new moms. It is exciting to think that there are millions of women having babies in 2024 who could benefit from new innovations like these, if the situation calls for it.”
Reprioritization On Cards After Sage/Biogen’s Zurzuvae Receives CRL For MDD
“Recent advancements in maternal mental health reinforce the importance of treating mental and physical health as equally critical. In 2023, the FDA approved the first oral treatment for women with postpartum depression, signaling a new era of hope and opportunity,” said Laura Gault, Sage Therapeutics, Inc.’s chief medical officer. “Not only must we recognize PPD as a serious medical condition, but we must also expand our thinking to overcome barriers that prevent women from reaping the benefits of innovative medicines. Today, almost half of all PPD cases go undiagnosed, leaving mothers to grapple with the devastating consequences. We can do better for mothers through consistent screening, diagnosis, and access to treatment options that support them during this critical time.
“Considering the commercial opportunity this market represents, and innovative research being conducted by certain companies, I expect to see women’s health take centre stage in the second half of this decade.”
David Solomon, Mithra
“My hope and projection for 2024 is that the industry work together with women and their families, advocates, clinicians and legislators to foster continued progress in maternal mental health to enact meaningful and enduring change,” concluded Gault.
“Women’s health is a market that has been underserved and is often characterized by lack of innovation. As women increasingly build their careers throughout their fifties, many suffer in silence as they enter menopause – a defined set of changes in all women’s bodies – and that needs to change,” said David Solomon, CEO of Mithra Pharmaceuticals SA, which specializes in products for contraception and menopause. “I would like to see governments working hand in hand with academia and the private sector to incentivize greater investment in women’s health research, leading to breakthroughs that could address health disparities and inequities and ultimately improve the lives of millions of women.
“The economic and wider societal benefits of investing in women’s health are slowly being acknowledged. Considering the commercial opportunity this market represents, and innovative research being conducted by certain companies, I expect to see women’s health take center stage in the second half of this decade with new contraceptive options being developed, and also importantly, treatments for the symptoms of menopause, as well as new products that could even delay its onset,” Solomon added.
Rare Diseases
“Rare disease research, including advances in gene and cell therapy, RNA targeting and next-generation therapies, has increasingly become an area of focus in drug development and will continue on this trajectory into 2024,” predicted Giacomo Chiesi, head of Chiesi Global Rare Diseases.
“There has been an uptick in M&A and licensing deals related to promising rare disease drugs and new regulatory and policy frameworks have emerged to incentivize drug development in the sector, such as the FDA’s START pilot program and new draft guidance regarding confirmatory evidence of clinical trials,” he went on. “If these efforts are any indication, support for R&D of new rare disease drugs is only poised to continue. Despite the unique challenges in rare disease research, related to small patient populations, trial endpoints, and the heterogeneity of diseases, there seems a willingness for stakeholders to collaborate to try to overcome these challenges for the benefit of patients and their families who depend on the work we do.”
“Without continued engagement and partnership across the healthcare ecosystem I fear we may see a decline in rare disease development in 2024 that will impact the advances of the future.”
Christelle Huguet, Ipsen
However, Christelle Huguet, head of R&D at Ipsen SA, sounded a note of caution on the regulatory front, at least in the EU. “There are shared concerns over the revision of the EU regulation incentivizing companies to invest in orphan medicines and the increased unpredictability it could present for companies investigating in this space,” she warned. “Previously, a scientific breakthrough (eg, biomarker identification) would drive investment by numerous companies to develop new treatments. However, in the future, we may see companies moving away from investing in some rare diseases where other treatments are in development. Sadly, the biggest impact of this will be felt by patients. With 95% of rare diseases currently having no effective treatment and attracting significantly less research funding there is already huge unmet need. Without continued engagement and partnership across the healthcare ecosystem I fear we may see a decline in rare disease development in 2024 that will impact the advances of the future.”
Most commentators were positive about the space, though. “The combination of increased awareness of rare diseases, better understanding of the biological causes of rare diseases, and dramatic advances in new treatment modalities will continue to drive increased rates of rare disease approvals,” foresaw David Goldstein, CEO of Actio Biosciences. “Beyond the increased number of approvals for rare indications, we expect to see a sharp transition toward treatments that are targeted to proximate causes of disease yielding substantial clinical benefits.”
“It’s encouraging to see that therapeutic advances in rare diseases have remained a focus for the industry given the high unmet need that persists for this incredibly diverse and underserved patient population,” said Dipal Doshi, CEO of Entrada Therapeutics, Inc. “As we progress into 2024, I anticipate that we’ll see a continued appetite for treatments that employ more flexible therapeutic approaches for patients with rare diseases, including those with inherited neuromuscular diseases such as Duchenne muscular dystrophy (DMD). DMD is one of the many rare diseases that have a profound unmet clinical need. Affecting about 30,000 people in the US and Europe, DMD is a devastating disease which causes progressive loss of muscle function throughout the body and requires treatment approaches rooted in flexibility – a shortcoming that exists today with the current therapeutic options. I believe we will continue to see an effort to advance medicines for these patients to close the gap between DMD patient needs and the availability of impactful treatments.”
For Jörg Schüttrumpf, chief scientific innovation officer of Grifols, S.A., “the ongoing innovation in treatments, in addition to new indications, will increasingly address therapeutic administration and patient convenience, particularly in the area of rare and chronic conditions.”
He added: “This is particularly relevant for well-established healthcare companies with extensive expertise in specific diseases. We will see advancements towards more convenient administration routes and less frequent dosing.”
Aging
“In 2024, we will see an influx of interest in the science behind aging which will fuel advancements in medicine that are less focused on extending the years of one’s life but, more specifically, on extending the number of healthy years, or health span.”
Jerry McLaughlin, Life Biosciences
“We know that the progressive deterioration of our health as we age has significant impact not only on our individual livelihood, but also on society by placing a heavy burden on the care and support of aging adults,” said Jerry McLaughlin, CEO of Life Biosciences LLC.
“In 2024, we will see an influx of interest in the science behind aging which will fuel advancements in medicine that are less focused on extending the years of one’s life but, more specifically, on extending the number of healthy years, or health span. This is a very important distinction and shift in mindset that will be emphasized in 2024.
“The first step to accomplish this will be the development of therapeutics that can prevent, treat, and/or reverse aging-related conditions in areas where aging biology has a clear link to disease pathogenesis, and we now have companies making great strides in this space such as achieving cellular rejuvenation through partial epigenetic reprogramming.”
Cuong Do, CEO of BioVie, Inc., declared: “We’re on the cusp of being able to reverse the biological aging process. We can’t stop aging, but we can keep people healthier as we age. Longevity research has largely focused on understanding the molecular and cellular bases of aging up until now, but I expect in 2024 we will see an acceleration of progress as additional interest and funding pour into the sector.
“While the length of the human life span makes testing longevity drugs in vivo a long and complex process, there’s been great progress made in identifying DNA methylation as a biomarker of aging. I predict that this year we will see some later-stage candidates in this space produce promising early clinical results on reducing DNA methylation (or ‘age deceleration’) along with continued efforts from companies to validate this biomarker of aging as a driver of age-related diseases like Alzheimer’s disease and Parkinson’s disease.”
Organ Transplantation
“The past 20+ years in organ transplantation have seen little progress. However, 2023 marked a turning point, with the groundbreaking xenotransplantation of humanized pig hearts into two patients,” said David-Alexandre Gros, CEO of Eledon Pharmaceuticals, Inc. “These procedures, driven by innovative gene editing techniques and a novel class of immunosuppressive therapy, signal the beginning of a new era in transplantation medicine. We are now at the technological forefront of achieving two key objectives: significantly extended organ viability post-transplant (‘one organ for life’) and universal availability of organs (‘one organ for all’). These developments not only offer renewed hope to organ failure patients worldwide but also present burgeoning market opportunities.
“Looking to 2024, I anticipate a leap in allograft patients having their transplanted organs protected by new medicines in clinical trials, alongside a parallel expansion in the types of xenotransplants performed, key next steps in our path to revolutionize transplant patient outcomes.”
Supportive Care
“With the recent and remarkable breakthroughs that have led to the development of numerous new cancer therapies, I believe there will be a tremendous need in the near future for novel products and mechanisms focusing on supportive care and quality of life for the patients on these chronic, lifesaving therapies,” said Lisa Conte, CEO of Jaguar Health, Inc., a commercial-stage biopharma company developing novel, plant-based medicines for gastrointestinal distress.
“These new cancer therapies are wonderful but come with a side-effect ‘cost.’ The publications describing many breakthrough therapies reference the associated ‘tolerable’ and ‘manageable’ toxicities. When patients read these publications, they often ask, ‘Tolerable for whom?’ The need for supportive care extends to rare disease patient populations as well. Rare diseases, when taken together, are not that rare at all. In fact, 30 million Americans – 10% of the population – have one of the approximately 7,000 known rare diseases. Breakthroughs to extend the life of rare disease patients, who, like cancer patients, are typically managed chronically, also demand a focus on quality of life, side-effect management, and patient comfort and dignity.”
Diagnostics And Screening
Several executives spoke about breakthroughs in diagnostics and screening.
Yves Dubaquie, senior vice president, diagnostics, of Revvity, PerkinElmer Inc.‘s rebranded diagnostics and life sciences business, announced: “The era of a one-size-fits-all approach to healthcare is over as personalized medicine makes it possible to match specific patient groups with tailor-made treatments that can improve clinical outcomes, and in some cases, save lives.”
But he called for better collaboration. “However, siloes and technological gaps stand in the way of personalized medicine reaching its fullest potential. As science becomes both more complex and more personalized, we will see even greater collaboration between life sciences and diagnostics. This convergence is key to helping drive new scientific breakthroughs in laboratories, bringing a new product through the clinic, and ultimately tailoring a unique diagnostic which can help identify individuals who stand to benefit the most from this new therapeutic or cure. This full ‘continuum’ involving research to development, diagnosis to cure, will improve healthcare for everyone, everywhere.”
“Instead of the traditional one-size-fits-all approaches that have long dominated healthcare, advancements in medicine are evolving towards therapies that consider individual factors like genes, environment, lifestyle, and more.”
Luca Dezzani, Johnson & Johnson
Luca Dezzani, vice president, US oncology medical affairs at Johnson & Johnson, also highlighted personalized medicine advances. “Personalized treatments are generating increased interest from patients and healthcare providers alike. Instead of the traditional one-size-fits-all approaches that have long dominated healthcare, advancements in medicine are evolving towards therapies that consider individual factors like genes, environment, lifestyle, and more.”
In particular, he saw its application in oncology. “Genetic testing plays an increasingly significant role in the shift towards precision medicine, especially in cancer care. By analyzing a patient’s genetic makeup – identifying specific genetic mutations or variations that may influence their response to treatment – healthcare providers can tailor therapies to target specific molecules or pathways responsible for cancer growth and proliferation,” he told Scrip. “These advances will help us continue to move towards treatments that are unique to each patient, making health care strategies more effective and outcomes better.”
Josh Ofman, president of GRAIL, Inc., meanwhile, highlighted the detection of cancer. “Oncology continues to be an area of both high need and significant advances, a trend that will undoubtedly continue in 2024. While 2023 predictions focused on therapeutic approaches, I am most encouraged by the progress in cancer screening,” he said.
“Cancer remains a leading cause of death, as we still find most cancers too late. To address this, innovations in screening technologies – including multi-cancer early detection – have dramatically changed our ability to detect cancer earlier, often before it has spread and presents symptoms. I see tremendous opportunity to continue expanding cancer screening in 2024, building on key advances in human genomics, AI, and understanding of cancer biology. By successfully incorporating these technologies into standard clinical practice, we can potentially help doctors take steps to improve chances of cancer survival, prevent premature suffering and death, and improve quality of life. My hope for next year is to finally bend the cancer mortality curve.”
Screening in a different context was highlighted by Revvity’s chief scientific officer Madhuri Hegde. “Next-generation sequencing (NGS) is providing efficient, holistic insights into the human genome, which has become a viable option for population-wide screening, including within newborn screening (NBS). A recent study conducted by Revvity Omics has shown the clinical value of proactive, sequencing-based screening in apparently healthy newborns. The use of proactive genomic screening would enable healthcare professionals to uncover a wide range of risks for looming pediatric onset conditions, allowing for earlier interventions and personalized treatment plans based on individual genetic make-up. As NGS becomes more accessible to the general public, harnessing this tool could have a profound impact on the health and wellbeing of families and future generations.”
“I think 2024 we will unlock new potential within liquid biopsy technology and its ability to diagnose diseases at the earliest stage possible, when treatment has the greatest potential for real results,” said Matthew Baker, chief technical officer at Dxcover Limited. “To date, most research focuses on solely genomic data, which can only identify disease based on information from a tumor, inherently indicting a later stage disease. Those that will win in 2024 and beyond will utilize methods like multi-omic analysis, which combines data from multiple sources to create a more global view, to be able to look at the entire signal from the sample. This means not only looking at tumor markers, but also the human response, which can then be teased apart by machine learning.”
Biosimilars
In 2024, I believe that patient access and affordability will continue to be issues facing biopharma,” said Jon Martin, head of Organon’s US biosimilar business unit. “Considerable potential remains for biosimilars to help address these unmet needs, especially following the long-awaited launch of several biosimilars to Humira (adalimumab) in 2023. We cannot afford to miss the opportunity to accelerate adoption of biosimilars in the US.”
“While adoption of biosimilar blockbuster medicines has been slower than anticipated, 2024 will usher in an era of significant biosimilar adoption – and financial savings.”
Matthew Erick, Biocon Biologics Ltd
Matthew Erick, chief commercial officer, advanced markets, at Biocon Biologics, agreed. “With billions of dollars in branded pharmaceuticals coming off patent, the healthcare system can save significant amounts of money, while improving accessibility and affordability for patients. While adoption of biosimilar blockbuster medicines has been slower than anticipated, 2024 will usher in an era of significant biosimilar adoption – and financial savings.”
“Greater biosimilar adoption can help reduce the affordability burden of high-cost brand biologics on the health care system, and, above all else, potentially help with the affordability burden on patients,” Martin went on. “A significant number of patients who need a biosimilar to Humira suffer from a chronic disease that requires lifelong management that historically comes with a high price tag. The high price tag is because co-insurance payments are often based on the list price of a medication. When formularies choose to prioritize coverage for a lower-cost biosimilar, instead of an expensive branded biologic or a higher-cost biosimilar, the financial savings to individual patients and to the health care system can be considerable. It’s my view that in 2024, payers, physicians and patients will not be able to ignore the economic value of biosimilars, and we’ll see greater adoption overall.”
Additional reporting by Joseph Haas.
