The U.S. FDA has approved Cobenfy, a dual M1/M4 muscarinic agonist that offers a fundamentally different approach to treating schizophrenia. The fixed dose combination of xanomeline-trospium is the first to act via a novel mechanism for the serious psychiatric disorder in over 50 years, finally expanding the treatment options beyond dopamine-targeted therapies. Bristol Myers Squibb Co., which acquired Cobenfy developer Karuna Therapeutics Inc. for $14 billion in 2024, said the drug will be available in the U.S. from late October at a price of $1,850 per month, more than the annual cost of most currently available generic antipsychotics. Notably, in relation to likely uptake, Cobenfy does not have atypical antipsychotic class warning or a boxed warning.

Regeneron, Sanofi add COPD to Dupixent label

The U.S. FDA cleared Dupixent (dupilumab), an IL-4 receptor alpha antagonist partnered between Regeneron Pharmaceuticals Inc. and Sanofi SA, as an add-on maintenance treatment for inadequately controlled chronic obstructive pulmonary disorder (COPD) with an eosinophilic phenotype. Dupixent, the first-ever biologic for COPD, gained its first nod from regulators in March 2017. The latest approval is based on data from two phase III trials, Boreas and Notus, that evaluated the efficacy and safety of Dupixent vs. placebo in adults currently on maximal standard-of-care inhaled therapy (almost all on triple therapy) with blood eosinophils ≥300 cells per µL.

Biomea’s diabetes studies back in business as FDA lifts hold

The U.S. FDA has lifted the full clinical hold it imposed in June on Biomea Fusion Inc.’ s phase I/II studies in types 1 and 2 diabetes. A safety review of the phase IIb expansion study was encouraging and none of the elevated lab values confirmed serious liver injury or impairment, said Biomea’s CEO, Thomas Butler. When the clinical hold went into effect during the summer, the FDA said the hold was based on the possibility of drug-induced elevated liver enzymes in type 2 patients who had completed the dose-escalation portion of the study. The company’s stock (NASDAQ:BMEA) had swollen around 10% about the time the market opened but had settled at midday to a more modest 4.3% rise at $9.98 per share.

Asabys’ €180M fund to support Europe’s life sciences ecosystem

Spanish VC firm Asabys Partners has closed its second fund at €180 million (US$201.3 million), to be invested in seed to series B rounds in 12 to 15 biotech, med-tech and digital health companies. Of these, 50% will be biotechs, which will be allocated 60% of the fund. In common with the firm’s Fund I, much of the focus will be on Spain, where a recent revival in public spending on research is generating significant IP, and where there is limited competition. “[We] want to really support the industry here, and the ecosystem in Spain is pretty good right now,” said Maite Malet, head of investment and corporate development at Asabys. “We’re even bringing in scientists from Belgium and Switzerland to Spain, because R&D here is very strong,” she told BioWorld.

China’s NMPA accepts NDAs from Innovent, Lepu

China’s National Medical Products Administration (NMPA) has accepted NDAs for Innovent Biologics Inc.’s IL-23p19 antibody picankibart to treat moderate to severe plaque psoriasis, and Lepu Biopharma Co. Ltd.’s antibody-drug conjugate MRG-003, to treat recurrent or metastatic nasopharyngeal cancer. Innovent’s IBI-112 is a monoclonal antibody that selectively targets the p19 subunit of IL-23 but not the p40 subunit. Lepu’s MRG-003 is an antibody-drug conjugate targeting epidermal growth factor receptor.

Phase III successes in August: Izokibep, Yorvipath and multiple cardio trials

In August 2024, BioWorld tracked 92 clinical trial updates, including nine successful phase III trials, three with mixed outcomes and three failures. Of note, data from two phase III studies showed that George Medicines Pty Ltd.’s GMRx2, a combination of the generic drugs telmisartan, amlodipine and indapamide, proved more effective than any of the three dual therapies.

BioWorld Insider podcast: Capricor’s CEO pursues a BLA and talks rare disease

Capricor Therapeutics Inc. just wrapped up a visit with the U.S. FDA and is prepping to file a BLA in October for its Duchenne muscular dystrophy treatment. Linda Marbán, Capricor’s CEO, is the guest on the newest BioWorld Insider podcast and she talks about deramiocel (CAP-1002), the company’s allogeneic cardiac-derived cell therapy, for treating the rare disease and how the FDA has made strong efforts in helping lay the groundwork for deramiocel. Marbán has been working on the Duchenne treatment for many years and she has strong insights into how to tackle a rare disease development program and how the FDA has changed its ways over time to help out. She has been in the biopharma space for more than 20 years and also is a co-founder of Capricor.

Also in the news

858 Therapeutics, Abbvie, Acumen, Acurx, Ani, Apimeds, Arch, Astrazeneca, Bayer, Bioage Labs, Biocity, Biogen, Emergent, Enanta, Engene, Enterprise, Evaxion, Evotec, Eydisbio, Genentech, Genevant, Inmune, Innovent, Leo, Marius, Mirai, MSD, Novo Nordisk, Nubioage, Nuhx, Odyssey, Pasithea, Pharming, PTC, Qualigen, Repair, Sage, Scisparc, Takeda, Tempus, Union, Vertex, Wave