Gene therapy specialist Meiragtx Holdings plc got a market bump courtesy of newly released top-line data from its phase II bridging study in Parkinson’s disease. The six-month, three-arm randomized, double-blind, sham controlled trial of AAV-GAD, a one-time infusion, demonstrated significant and clinically meaningful improvements in key efficacy endpoints. The primary objective was evaluating the therapy’s safety and tolerability. The study of participants with idiopathic disease showed the therapy was safe and well-tolerated with no serious adverse events. Meiragtx is pursuing approvals in the U.S., Europe and Japan.
This summer’s IPO by Artiva Biotherapeutics Inc. highlighted early stage efforts with natural killer cells in autoimmune disease, where a handful of companies are advancing programs. The firm raised $167 million through an upsized financing, with funds aimed at the development of its lead AlloNK program for systemic lupus erythematosus and other indications in the same category.
Third-party litigation funding has been a source of controversy in the U.S. over the past decade, but the practice drew little national scrutiny up to now.
The U.S. FDA has approved the second hemophilia drug in nearly six months from Pfizer Inc. This one, Hympavzi (marstacimab), is for preventing or reducing bleeding in those age 12 and older with hemophilia A and B. Hympavzi heralds a couple of market boundary breakers: it’s the first and only anti-tissue factor pathway inhibitor approved in the U.S. for hemophilia A or B and the first hemophilia medicine approved in the U.S. to be administered using a pre-filled, auto-injector pen.
Continuing its streak of promising early clinical data, Jasper Therapeutics Inc.’s briquilimab impressed in a preliminary readout from a phase Ib/IIa study in chronic inducible urticaria (CIndU), showing a clinical response of 93%. CIndU, an inflammatory skin condition causing hives that is often induced by physical or environmental stimuli, is commonly treated with antihistamines, though some patients are refractory. Beyond antihistamines, there is no treatment available globally, explained Edwin Tucker, Jasper’s chief medical officer, so briquilimab has the potential to be “a new treatment paradigm for patients,” both in reducing disease burden and in improving quality of life.
H. Lundbeck A/S is to acquire Longboard Pharmaceuticals Inc. in an all-cash deal valuing the epilepsy specialist at $2.6 billion. The agreed price of $60 per share is a 54% premium to the closing price of Longboard stock (NASDAQ:LBPH) on Oct. 11 and represents the biggest deal in Lundbeck’s 110-year history. The acquisition will give the Copenhagen, Denmark-based pharma company ownership of bexicaserin, which in September entered phase III development in Dravet syndrome. The 5-HT2C agonist has the potential to treat this and other rare developmental and epileptic encephalopathies, for most of which there are no approved therapies.
Hologic Inc. signed an agreement to acquire Gynesonics Inc. for $350 million. The acquisition will significantly boost Hologic’s revenues from surgical gynecology and provide an option not met by the company’s current portfolio of products.
The U.S. FDA’s device center disclosed its guidance ambitions for this new fiscal year – a list that includes the usual A and B lists for draft and final guidances. However, the agency now has an “under construction” list of guidance ambitions, the status of which is entirely reliant on the agency’s resources.
The founding CEO of Alnylam Pharmaceuticals Inc. is now leading the charge with newly launched City Therapeutics Inc., which just completed a $135 million series A financing. City’s executive chair, John Maraganore, will be in familiar territory as the new company plans to develop RNAi-based medicines using next-generation siRNA engineering. He expects dozens of these therapies to reach the market in a relatively short period of time, not just from City Therapeutics but from other companies. It’s a period in the development timeline that he finds reminiscent of the rise and development of monoclonal antibodies.
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