CNS

In this week's podcast edition of Five Must-Know Things: global CEO remuneration; another late-stage loss for Merch KgaA; deals shift to smaller M&A, partnering; Bayer’s Parkinson’s bets bear fruit; and India looks to innovate CAR-T models.

The companies are set to present Phase III results from their rival myostatin inhibitor drugs for spinal muscular atrophy in Q4, while also fast-tracking the candidates in obesity studies.

The German major is kicking off Phase II trials for a cell therapy and a gene therapy that could be gamechangers for the progressive neurodegenerative disorder that affects more than 10 million people worldwide.

The biotech said it plans to engage with regulators before the end of the year for the Takeda-partnered antisense oligonucleotide, WVE-003, to discuss a pivotal trial that would support accelerated approval.

The gene therapy is now approved for all ambulatory boys with Duchenne muscular dystrophy ages 4 and older. The revised label also includes accelerated approval for non-ambulatory patients.

A Phase IIb trial evaluating suvecaltamide failed to show a statistically significant benefit versus placebo on an assessment scale for essential tremor.

A first big pharma partnership for Ascidian, headed up by ex-Biogen research head Michael Ehlers, as the Swiss firm expands its gene editing and neuroscience efforts.

Caplyta succeeded in a second Phase III study as adjunctive therapy for major depression, putting the schizophrenia/bipolar disorder drug on track for sNDA filing later in 2024.

Ganaxolone met one of two endpoints in its Phase III refractory status epilepticus trial, but next steps are uncertain. Analysts say the firm should focus on a more promising trial, but Marinus thinks conditional approval is possible.

Two Phase III studies testing soticlestat, licensed from Ovid, in Dravet and Lennox-Gastaut syndromes failed to meet their primary endpoints on improvement in seizure frequency.

Several Japanese firms are facing R&D challenges and setbacks in the US, in the form of missed Phase III endpoints, trial recruitment difficulties and a Complete Response Letter.

With several new drugs progressing for the estimated 70,000 patients in China with DMD, a recent setback for Nippon Shinyaku's contender and progress for Chinese gene therapy developers are adding both uncertainty and likely competition in an untapped market with pressing medical need.