Gilead To Be More Selective About M&A Going Forward
Gilead Sciences CEO Daniel O’Day said during a media briefing on 15 January on the sidelines of the J.P. Morgan Healthcare Conference that the company will be more selective going forward in terms of mergers and acquisitions now that the company has been through a more active period of both internal and external investment, during which it built up significant capabilities across its three main therapeutic areas of virology, oncology and inflammation.
“Now, at this stage, we are at a stage where we will continue to generate significant cash moving forward because that build has occurred and we have strong growing franchises across all three areas,” O’Day said. “We will look to be more selective, however, on our M&A moving forward. And I think that that’s appropriate given the fact that we have a full, robust pipeline and really the build out on the competency side already in all three therapeutic areas.”
First and foremost, he noted, Gilead is focused on partnerships and acquisitions for assets in late research or early development, such as the company’s deal with LEO Pharma announced on 13 January, in which it paid $250m up front for a preclinical oral small molecule STAT6 inhibitor program.
“I think that’s something that a growing healthy business like ours does routinely to complement their internal innovation,” O’Day said. “And I think we’ve said publicly we spend roughly $1bn a year, plus or minus, on those types of things to feed the pipeline. And then what we’ve also said is that we have a lot going on within our current portfolio, but on occasion we’ll see something in a later stage of development – or potentially it’s already on market – that could complement our portfolio,” such as last year’s acquisition of the liver disease drug developer CymaBay for $4.3bn.
“Those types of acquisitions on a later stage, every one to two years, we may do something like that, but the bar is very, very high,” O’Day said. That bar is high, he added, because in addition to Gilead’s “robust” existing drug development portfolio, “we don’t really have any major products that are going off patent until the end of 2033, so nine years from now, and that medicine is Biktarvy.”
Roche Sticking To BD Playbook
Roche will continue to follow a similar business development strategy in 2025 as it has in recent years, head of corporate business development Boris Zaïtra said in an interview.
“If you take 2023 and 2024, I think that presents a relatively good mix of what we are looking after,” he said. Zaïtra highlighted deals like Roche’s $7.1bn acquisition of Telavant, a Roivant Science spin off, in 2023 for a TL1a directed antibody, the $2.7bn acquisition of Carmot Therapeutics in obesity, and the $1.5bn buyout of the cell therapy company Poseida, gaining a CAR-T platform.
Zaïtra, who has overseen M&A at Roche, added pharma partnering to his remit last year after James Sabry left to join BioMarin. Zaïtra worked in M&A outside of pharma before joining Roche in 2012.
“James was a scientist. You can see me more as a deal maker,” Zaïtra said. “We’ve got thousands of scientists at Roche, so that’s perhaps not where I can add the most value. I really come in on the execution side, on delivering the deals, of course working end-to-end with the rest of the organization.”
“With the one pharma strategy, we’ve defined very clearly what we aspire to achieve; the ambition is very high, and to achieve those ambitions, we have to be very proactive, very determined, very systematic,” he added.
The company’s BD is focused on five core therapeutic areas: oncology, neuroscience, ophthalmology, cardiovascular/metabolic and immunology.
“I would say they’re all important because you have to match the aspiration of the playbook to what’s available,” he said. So Roche screens widely for new assets across all five areas, but Zaïtra said the company is also looks outside of those core therapeutic areas, based on the science, for opportunities with exciting potential.
Bayer Hopeful About Good Access To Elinzanetant In US
Scrip spoke with Bayer’s global head of commercialization Christine Roth at J.P. Morgan about upcoming product launches in 2025, including elinzanetant for vasomotor symptoms (hot flashes) associated with menopause. Roth expressed optimism about access to the drug – a dual antagonist of the neurokinin 1 and 3 (NK-1,3) receptors – in the US, where elinzanetant is expected to launch around mid-year. If approved, it will compete directly with Astellas Pharma’s first-in-class NK-3 antagonist Veozah (fezolinetant), which has had a slow initial launch and recently added a liver injury warning to its label.
“We’re cautiously optimistic about our position in the class,” Roth said. “The class did not get off to the best start. I think there are a lot of reasons why,” she said, and it wasn’t expected to take off immediately. “There’s a lot of education that’s going to be required.”
She noted that among women who suffer with vasomotor symptoms, “a very small percentage of them actually get hormone-based therapy, even though it’s indicated for most, but yet the uptake of the non-hormonals has not been that strong. So, I think it’s an issue of awareness for both patients and physicians and making sure people understand the value proposition of a non-hormonal in this space.”
Despite the slow initial uptake for Veozah, Roth said the Astellas product has gained momentum recently, which should benefit the broader NK receptor antagonist class.
“It’s great for women, it’s good for the class. Obviously having a second entry into the market will increase the noise for this option and I think that a rising tide lifts all boats,” Roth said. “We anticipate that Astellas came into the market without a lot of experience in women’s health and Bayer’s building on a 100-year heritage in this space. And so, we know our customers, we know their needs. We have more than just elinzanetant to offer when we walk into that OB/Gyn’s office and there’s a very high degree of trust amongst our women’s health team and our customers.”
She conceded that Bayer will have to make a concerted effort to educate payers about elinzanetant’s value in terms of pricing and access, but noted that physicians have made public statements in support of gaining access to elinzanetant since the company first reported Phase III results for the drug.
“I think maybe it’s a little unfortunate that [US president Joe Biden’s wife] Jill Biden will no longer be our First Lady, because she was just getting into her stride about the importance of women’s health, both from a innovation – research and development – as well as an access perspective,” Roth said. “But I think with the incoming [FDA] commissioner [nominee Martin Makary], he is also very focused on that. And I think we look forward to working with the new administration in whatever way we can to ensure access to innovation in a fast and cost-effective manner regardless of what therapeutic area it is."
AbbVie Looks To Make Big Expansion Into Oncology
AbbVie CEO Rob Michael told the company’s 15 January presentation to expect 17% sales growth in 2024 despite the declines for Humira (adalimumab) due to biosimilar erosion and a “return to robust growth in 2025.” Part of that will come from a significant push into oncology. He pointed for example to ABBV-383 (etentamig), a bispecific antibody targeting BCMAxCD3 that is in Phase III development for multiple myeloma. “I think it’s very compelling in myeloma – we’ll see as the data reads out, but I don’t think it’s getting as much credit,” he said.
But the company has a wider range of oncology assets in the pipeline, including in immuno-oncology, with Michael listing trispecifics, multispecifics, T-cell engagers and CAR-T cell therapies. “So, we think about next-generation platforms for oncology – that’s where we’ve been focused,” he said.
On 13 January, AbbVie announced a partnership with Shanghai-based Simcere Zaiming, a subsidiary of Simcere Pharmaceutical, to develop SIM0500, a GPRC5DxBCMAxCD3-directed trispecific antibody in Phase I for multiple myeloma. Under the deal, Simcere Zaiming will be eligible to receive option fees and milestone payments of up to $1.055bn on top of an undisclosed upfront payment.
The current market leaders in multiple myeloma are Johnson & Johnson – which markets a BCMA-directed CAR-T as well as bispecific T-cell engagers targeting BCMA and GPRC5D – and Bristol Myers Squibb, which has a BCMA-directed CAR-T on the market as well as a GPRC5D-directed CAR-T in the clinic. Hitting both targets with a single drug would put AbbVie in a potentially advantageous competitive position.
In solid tumors, chief scientific officer Roopal Thakkar also highlighted ABBV-399 (telisotuzumab vedotin), a cMet-targeting antibody-drug conjugate for non-small cell lung cancer that is currently under FDA review for accelerated approval, along with follow-on drug ABBV-400, which has a more stable linker.
Ionis Anticipates Rapid Expansion Of Independent Commercial Business
For many years, Ionis Pharmaceuticals was a company with a rich, deep pipeline of antisense oligonucleotide product candidates, but no approved products, at least not that it had developed independently. That changed on 19 December when the FDA approved Tryngolza (olezarsen) for familial chylomicronemia syndrome (FCS), making it the first drug approved for the genetic metabolic disease and the first approval for a drug that Ionis had developed by itself, apart from the drugs it had developed under partnerships with other companies.
And that, CEO Brett Monia told J.P. Morgan on 15 January, is just the beginning.
“We’ve built a highly innovative, highly experienced and scalable commercial organization that is in place to launch our products from our wholly owned pipeline,” Monia said. “We’ve expanded and diversified our technology in many ways through medicinal chemistry and in many other ways, and a lot of the advancements we’ve made in technology are now bearing fruit.”
Ionis has several products on the market developed under partnerships, including AstraZeneca’s Wainua (eplontersen) for polyneuropathy in hereditary transthyretin-mediated amyloidosis (hATTR-PN), which won FDA approval in December 2023. But now it is anticipating a rapid expansion as a standalone commercial organization as well.
In the second half of this year, it anticipates topline Phase III data and an FDA submission for olezarsen in severe hypertriglyceridemia, while its potential second independent commercial launch could come in the form of donidalorsen for hereditary angioedema, which has an FDA action date of 21 August. Launches that it expects to take place further in the future include the wholly owned zilganersen for Alexander disease in the 2026-2027 timeframe – with Phase III data for that drug expected in the second half of 2025 – as well as partnered assets like the Roche-partnered IONIS-FBLRx for immunoglobulin A nephropathy.
SMA Pioneers Pave The Road For Scholar Rock
Launching a new rare disease product can be daunting, but it holds no fears for Scholar Rock CEO Jay Backstrom, who told attendees at J.P. Morgan that the groundwork for its spinal muscular atrophy (SMA) candidate apitegromab had already been done by the three current standard of care medicines, Biogen’s Spinraza (nusinersen), Roche’s Evrysdi (risdiplam) and Novartis’s Zolgensma (onasemnogene abeparvovec).
Unlike many other rare disease markets, “where there is a need to improve diagnosis and educate and identify patients,” SMA patients are already identified, he said, “with treatment highly concentrated among 250 expert US physicians.” There is “already clear recognition of the unmet need,” Backstrom added, noting that the space can boast “an incredibly organized and motivated caregiver and patient community who are looking for additional treatments.”
Payers are also familiar with SMA, he said, “there’s established value for improving motor function, they recognize the continued need and are covering sequential treatment. All of these dynamics point to an optimal opportunity for a highly successful launch to usher in a new treatment paradigm in SMA.” Apitegromab will be filed in the US and EU later this quarter.
“We should tip our caps to the groups that launched the SMA correctors,” Backstrom said. “They really set the market for us and do great things for patients, they really paved the road. Now we know where the patients are, and we know how to get to them. The neuro piece is very well taken care of, but the muscular piece needs a lot of help, so that’s where we’re going to go.”
Edgewise Rises After Setting Out 2025 Plans
J.P. Morgan presentations generally don’t impact stock market sentiment as much as trial readouts at medical congresses, but Edgewise Therapeutics saw its share price rise by 9% (to around $28.50 a share) after CEO Kevin Koch presented its plans at the meeting on 13 January.
The company was already riding high thanks to two positive readouts for sevasemten in Becker muscular dystrophy last year – the open-label ARCH study and Phase II CANYON study – which helped double the company’s market cap over the course of 2024.
Based on the data, the company plans to talk to the US and EU regulators about filing strategies for the first-in-class fast skeletal myosin inhibitor, which could become the first treatment approved for Becker disease.
The share price rise rests partly to the chance that Edgewise could gain an accelerated approval based on its existing data: Koch said he was encouraged by the US Food and Drug Administration’s latest guidance on accelerated approvals.
That said, analysts are still assuming that the company will need to complete its Phase III GRAND CANYON study, expected in Q4 2026, to gain approval.
Also encouraging was an update on the company’s plans for advancing sevasemten in Duchenne muscular dystrophy (DMD), which could challenge Sarepta’s dominant position in the field. Edgewise will report data from the twin Phase II LYNX and FOX studies of sevasemten in in children aged 4 to 9 years and 6 to 17 years, respectively, in the first half. This will set out how the treatment could be used throughout the DMD population, including patients on background steroids and those who have already been treated with gene therapy, namely Sarepta’s Elevidys (delandistrogene moxeparvovec).
Analysts at Leerink Partners said in a same-day note that, if approved, sevasemten could earn around $2.1bn in DMD and $1.3bn in Becker.
Edgewise also presented initial Phase I results from EDG-7500, a selective cardiac sarcomere modulator, in patients with obstructive hypertrophic cardiomyopathy. Leerink said this could reach peak annual sales of $2.5bn.
[Editor’s note: This article has been updated to correct a misspelled name.]