Neurological
The US biotech’s Duchenne muscular dystrophy drug is “undoubtedly the most successful gene therapy launch in all of history,” according to CEO Doug Ingram. “We have barely scratched the surface of the opportunity in front of us.”
XGene's pain candidate has shown positive results in a US bunionectomy trial and the Chinese company is also eyeing chronic and cancer pain indications for its contender, which may provide a competitor to Vertex's suzetrigine.
Johnson & Johnson started the M&A year with a bang at the J.P. Morgan meeting, unveiling its $14.6bn bid for Intra-Cellular. The deal would bolster its neuroscience portfolio and pipeline.
Productive talks with the US FDA after securing Regenerative Medicine Advanced Therapy designation haves speeded up development of bemdaneprocel, which is about to go into a registrational trial to see if it can slow down or even stop progression of the neurodegenerative disease.
With positive Phase II data for its amisulpride derivative, LB is planning a Phase III program to show safety and convenience with a lower dose that passes more easily through the blood-brain barrier.
Two treatments that target elF2B, a regulator of the integrated stress response that appears to be overactive in the progressive motor neurone disease, have missed their primary and secondary endpoints in a landmark platform trial.
While quieter than some recent years, 2024 did produce several clinical trial readouts destined to have an impact on their respective markets. Here, in roughly chronological order, Scrip takes a look at the 10 clinical stories that got readers clicking the most over the past 12 months.
Neumora, one of two firms testing kappa opioid receptor antagonism in depression, failed its first of three Phase III studies. The company’s share price crashed sharply.
BMS's Sotyktu succeeded in two Phase III psoriatic arthritis trials, Novo's Alhemo (concizumab) was approved in the US for hemophilia A and B with inhibitors, Pfizer returned a hemophilia gene therapy to Sangamo ahead of US and EU filings, and more late December news.
The Swiss giant’s blockbuster gene therapy is currently only administered intravenously into patients with spinal muscular atrophy under two years of age. Its intrathecal candidate, called OAV-101, has just passed a Phase III test which could expand its use in children and young adults.
The Phase IIb failure was expected, but Roche believes consistent trends towards efficacy could be confirmed with ongoing open-label studies, leaving life in the program yet.
Japanese major highlights at R&D Day key late-stage pipeline assets set to be filed for approval over the next few years, which it says have a combined peak sales potential of $10-20bn, and which could help it weather the expiry of current blockbuster Entyvio.
Almost three years to the day, Novartis and UCB heralded a pact that could have been worth $1.5bn to the Brussels-based group for a first-in-class, small-molecule, alpha-synuclein misfolding inhibitor for Parkinson’s disease. However, after a Phase IIa fail, the development of minzasolmin has been terminated.
Shares in the CNS disease-focused biotech crashed by nearly 80% after its lead product, neflamapimodin, failed in a Phase IIb study in Lewy body dementia. Investors are pondering what went wrong.
The firm said it will file the D1/D5 dopamine receptor agonist for US FDA approval in 2025. The drug may salvage value from AbbVie’s Cerevel buyout.
The big pharma is tapping into Muna’s MiND-MAP platform to gain extra validation for its own early-stage work in Alzheimer’s and other neurodegenerative conditions.
China’s fresh approval of a sublingual form of Simcere's edaravone/dexborneol brain cytoprotective agent for acute ischemic stroke is paving the way for an international Phase III study in this indication.
Novartis will also pay up to $1.9bn for milestones and share US profits on the Phase II RNA-splicing candidate. PTC is discussing potential accelerated approval with the US FDA.
While most drug development in the multiple sclerosis space has focused just on relapse prevention, Immunic’s dual-acting therapy vidofludimus calcium also offers a novel approach for neurodegeneration, its CEO Daniel Vitt tells Scrip.
Deal Snapshot: The essential tremor space has not seen innovation in treatment for decades. Acadia believes Saniona's GABAA-α3 positive allosteric modulator could be the answer.