Fibrodysplasia ossificans progressiva is a rare and life-threatening genetic disease caused by gain-of-function mutations in the ALK2 gene, which encodes activin receptor-like kinase 2. Blueprint Medicines Corp. has elucidated the discovery of their ALK2 inhibitor BLU-782, which is now in phase II studies at Ipsen for the treatment of FOP.
Bridgebio Pharma Inc.’s data from the phase II study with infigratinib in achondroplasia “swings the pendulum of debate sharply in favor” of the firm, competing with Biomarin Pharmaceutical Inc. in the space, Leerink analyst Mani Foroohar said in a report.
The EMA has been sent back to the drawing board to re-evaluate PTC Therapeutics Inc.’s Duchenne muscular dystrophy therapy Translarna (ataluren), after failing to get the usual rubber stamp following its recommendation in January that the drug’s conditional approval be withdrawn.
Shares of Dyne Therapeutics Inc. (NASDAQ:DYN) closed May 20 at $35.38, up $7.70, or 28%, on word of positive data from the phase I/II Achieve trial of DYNE-101 in myotonic dystrophy type 1 (DM1) and the phase I/II Deliver effort with DYNE-251 in Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. CEO John Cox, who joined Waltham, Mass.-based Dyne eight weeks ago, said he “couldn’t be more proud to be part of this team.” Studies are ongoing, but new data with regard to DM1 as well as DMD showed a “compelling” impact, Dyne said, plus satisfying safety profiles.
Following a cumbersome process, the U.S. FDA is withdrawing its accelerated approval for Truseltiq (infigratinib) as a second-line treatment for patients with unresectable locally advanced or metastatic cholangiocarcinoma harboring an FGFR2 fusion or rearrangement as detected by an FDA-approved test.
A non-traditional route for financing has been the path to success for Fibrobiologics Inc. In the newest BioWorld Insider podcast, CEO Pete O’Heeron offers insight into the company’s unusual path to a Nasdaq listing in January.
Aqilion AB has expanded its pipeline through the nomination of protein kinase C θ (PKCθ) as the target of a new immunology project. The PKCθ kinase is believed to be centrally involved in the pathogenesis of T-cell-mediated inflammatory and autoimmune diseases.
Fulcrum Therapeutics Inc.’s deal with Sanofi SA to develop and commercialize oral losmapimod shone more light on facioscapulohumeral muscular dystrophy (FSHD), a rare genetic disease where Avidity Biosciences Inc. also has an earlier-stage but high-profile program.
A boy participating in the phase II Daylight study of Duchenne muscular dystrophy (DMD) “has passed away suddenly,” according to Pfizer Inc. The participant had received fordadistrogene movaparvovec, a mini-dystrophin gene therapy, in early 2023. The fatal serious adverse event was reported May 3 as a cardiac arrest, Pfizer told BioWorld. Pfizer, together with the independent external data monitoring committee, is reviewing the data to understand the potential cause, the company added.
Vyne Therapeutics Inc. has received IND clearance from the FDA allowing it to initiate a first-in-human phase Ia study of VYN-202, an oral small-molecule BD2-selective BET inhibitor for autoimmune diseases.
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