Bridgebio Pharma Inc. kept the phase III wins coming, this time with positive top-line results from Propel 3, the global phase III pivotal study of oral infigratinib, designed to inhibit FGFR3 signaling in children with achondroplasia.
Biomarin Pharmaceutical Inc. plans to divest its pioneering gene therapy for hemophilia, Roctavian (valoctocogene roxaparvovec), and remove what had been considered a potential blockbuster from the portfolio in order to grow the company.
It’s been a big week for Inozyme Pharma Inc. On the heels of a promising interim readout for phase III-stage enzyme replacement therapy (ERT) candidate INZ-701 in ENPP1 deficiency, the firm agreed to be acquired by Biomarin Pharmaceuticals Inc. in a deal valued at about $270 million, putting the rare disease ERT in the hands of an experienced commercial team.
Predictably, weekly vs. daily subcutaneous injections made a big difference in the achondroplasia space, where shares of Copenhagen, Denmark-based Ascendis Pharma A/S (NASDAQ:ASND) closed Sept. 16 at $139.57, up $20.35, or 17%, on favorable, pivotal top-line data with Transcon CNP (navepegritide), as competitor Biomarin Pharmaceutical Inc.’s stock (NASDAQ:BMRN) ended the day at $69.86, down $15.04, or 17.7%.
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.
Four other companies want to replicate Biomarin Pharmaceuticals Inc.’s recent success as they seek approvals to treat the rare but most prevalent genetic form of dwarfism, achondroplasia. The U.S. FDA approved the sNDA for injectable Voxzogo (vosoritide), fibroblast growth factor receptor 3 (FGFR3) inhibitor from Biomarin on Oct. 20.
Positive results from Bridgebio Pharma Inc.’s phase II study of infigratinib in children with achondroplasia, a genetic disease that inhibits bone length and leads to short stature, prompted the company stock to surge. Participants receiving the highest dosage, which was the fifth cohort getting 0.25 mg/kg daily, saw a 3.03-centimeter increase, about 1.19 inch, in their height annually, which produced a “p” value of 0.0022.
Wall Street apparently wants to see longer-term data from Bridgebio Pharma Inc. with oral infigratinib in children with achondroplasia (ACH) before deciding about the drug’s chances against the approved therapy Voxzogo (vosoritide) from Biomarin Pharmaceutical Inc.
Biomarin Pharmaceutical Inc.’s data supporting the use of Voxzogo (vosoritide) in children with the most common form of dwarfism proved compelling for the FDA, which cleared the modified C-type natriuretic peptide as the first treatment for the rare genetic disease affecting bone growth.
