Roivant Sciences Ltd. CEO Matt Cline said the firm’s unit Immunovant Inc. with FcRn blocker batoclimab has established “frankly a new bar” in myasthenia gravis (MG) as the New York-based firm reported top-line results from its phase III study and first data from period 1 of the phase IIb study with the same drug in chronic inflammatory demyelinating polyneuropathy. The data look promising, and Immunovant intends to use the findings to help advance second-generation FcRn prospect IMVT-1402 in both indications. Potentially registrational trials are planned. The U.S. FDA has granted IND clearance.
Tandem Diabetes Care Inc.’s Control-IQ+ automated insulin delivery system improved multiple measures of glycemic control in individuals with type 2 diabetes who use insulin, a study presented March 19 at the 18th International Conference on Advanced Technologies & Treatments for Diabetes and published in The New England Journal of Medicine demonstrated.
Latest findings on Healios K.K.’s stem cell therapy to treat acute respiratory distress syndrome (ARDS), coined Multistem (invimestrocel; HLCM-051), found the regenerative medicine effective in reducing the number of patient days on ventilator treatment, as well as mortality benefits.
Wall Street was weighing the gravity of the death from acute liver failure of a patient who was treated for Duchenne muscular dystrophy (DMD) with Sarepta Therapeutics Inc.’s gene therapy, Elevidys (delandistrogene moxeparvovec). Liver injury is a known possible side effect of the product, first approved by the U.S. FDA in June 2023 for DMD, as well as other AAV-mediated gene therapies, and the potential problem is highlighted in Elevidys’ prescribing information.
While data on functional endpoints are still to come, Avidity Biosciences Inc. executives said the firm is moving ahead with plans for a BLA filing by the end of 2025 for del-zota, an antibody-oligonucleotide conjugate, in Duchenne muscular dystrophy with mutations amenable to exon 44 skipping (DMD44), based on positive top-line data that analysts say bode well for Avidity’s other late-stage programs targeting rare neuromuscular diseases.
Investors wanted more from Incyte Corp.’s top-line results in hidradenitis suppurativa (HS) from its pivotal phase III Stop-HS trial program with oral small-molecule JAK1 inhibitor povorcitinib in adults with moderate to severe disease. Shares of the Wilmington, Del.-based firm (NASDAQ:INCY) closed March 17 at $62.01, down $5.85, after the company made public that Stop-HS1 and Stop-HS2 met the primary endpoint at both tested doses (45 mg and 75 mg)
Latest findings on Healios K.K.’s stem cell therapy to treat acute respiratory distress syndrome (ARDS), coined Multistem (invimestrocel; HLCM-051), found the regenerative medicine effective in reducing the number of patient days on ventilator treatment, as well as mortality benefits.
Term sheets are being drawn up with potential pharma partners after Torqur AG reported positive interim results from an ongoing phase II trial of bimiralisib topical gel in the treatment of actinic keratosis.
Bristol Myers Squibb Co.’s decision this week to snag Bluebird Bio Inc. spinout 2seventy Bio Inc. for $102 million net – just weeks after investors bid $30 million for Bluebird itself – seemed to place a final blow on what was once a promising gene therapy company. The space in general has struggled to make business sense out of the one-time therapies that often involve complicated manufacturing and exorbitant prices, despite the life-changing value that gene therapies bring to patients. But despite some recent setbacks, biopharmas continue to plow forward with promising research in the field.
Boehringer Ingelheim GmbH terminated its second metabolic dysfunction-associated steatohepatitis (MASH) alliance on March 6, ending an $870 million license agreement inked with Yuhan Corp. for dual GLP-1/FGF21 agonist, BI-3006337 (YH-25724). Yuhan said March 7 that Boehringer, of Ingelheim, Germany, returned rights to YH-25724, a dual-acting glucagon-like peptide-1 and fibroblast growth factor 21 receptor agonist, based on the counterparty’s “strategic judgement” on developing MASH therapeutics.
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