Biomarin Pharmaceutical Inc. plans to divest its pioneering gene therapy, Roctavian (valoctocogene roxaparvovec), and remove it from the portfolio in order to grow the company. A year ago, Biomarin expected to bring in $4 billion in 2027 but has now adjusted its goal since its third quarter 2025 numbers underwhelmed investors. One of the factors to the change of tack was competition to Biomarin’s biggest seller, Voxzogo (vosoritide), which underperformed during the quarter. Another was sluggish Roctavian sales. Out-licensing opportunities in addition to the divestment is now part of the plan. The first gene therapy approved for hemophilia A, Roctavian received the U.S. FDA’s blessing in June 2023.
Levicept data show neurotrophin inhibitor may be disease modifying in osteoarthritis
An examination of X-ray and MRI scans of 518 patients before and after treatment with Levicept Ltd.’s Levi-04 has shown that, in addition to providing significant analgesia, the selective neurotrophin-3 inhibitor may have a disease-modifying effect in osteoarthritis of the knee. The new data from the randomized, double-blind and placebo-controlled phase II trial demonstrate there was a dose-dependent reduction in bone marrow lesions, which are associated with pain, cartilage loss and disease progression. In 40% of patients in the active arm, the lesions, which occur in the subchondral bone beneath the cartilage in the knee joint, disappeared completely.
Hanmi’s efpeglenatide hits phase III endpoints in obesity
Hanmi Pharmaceutical Co. Ltd. announced Oct. 27 that its glucagon-like peptide-1 (GLP-1) receptor agonist, efpeglenatide (HM-11260C), met the co-primary endpoints in a phase III study of obese adults without diabetes. Hanmi plans to file an NDA to South Korea’s Ministry of Food and Drug Safety within the year, ideally obtain clearance in mid-2026 and launch the product in the second half of 2026. If cleared, efpeglenatide will become the first GLP-1 drug developed by a local pharmaceutical company and tailored to the Korean population.
Another M1/M4 drug takes aim at schizophrenia, AD, as Maplight chalks up $250M IPO
Another entry into the M1/M4 muscarinic agonist space gained airtime as Maplight Therapeutics Inc. went public by way of an IPO that priced about 14.7 million shares at $17 each for proceeds of about $250.7 million, granting underwriters an option to buy as many as about 2.2 million more units. A concurrent private placement was expected to close today. Maplight’s lead prospect, ML-007C-MA, is an oral, extended-release, fixed-dose combination of an M1/M4 drug ML-007, co-formulated with a peripherally acting anticholinergic. The compound is undergoing phase II trials in Alzheimer’s disease and schizophrenia. Bristol Myers Squibb Co.’s Cobenfy (xanomeline and trospium chloride), bearing the same mechanism of action, was approved for the latter indication by the U.S. FDA in September of last year. Shares of Maplight, which priced Oct. 27 (NASDAQ:MPLT), were trading at $18.65.
Zag Bio launches with $80 million to dampen immune response via the thymus
Zag Bio came out of stealth mode with $80 million in funding so far, including a recently closed series A financing, to develop its platform for autoimmune diseases using drugs targeted to the thymus where thymic regulatory cells (Tregs) are made. By upregulating Tregs, Zag hopes to dampen the immune response in autoimmune diseases. The startup will initially focus on type 1 diabetes with expectations to enter the clinic in late 2026. The series A funding will also help the company discover additional therapies for autoimmune diseases of the skin, nervous system and gut.
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Adaptam, Annovis, Artelo, Bayer, Biogen, BMS, Bridgebio, Cabaletta, Cellectar, Clearmind Medicine, Cristcot, Eisai, Enable Injections, Evaxion, Genenta, Grand Life, GSK, Halda, Hemab, Hyloris, Immatics, Incyte, Innovent, Intellia, ITM, J&J, Jupiter, Kadimastem, Kivu, Kyverna, Levicept, Maplight, Merck, NLS, Oncoc4, Organon, Revolution, Sobi, Syndax, Syndivia, Targetgene, Vivani, Xencor, Zenas, Zenith