Analysts from the hosting firm talked up their takeaways on biopharma from the J.P. Morgan (JPM) Healthcare Conference and looked ahead to 2024, anticipating a generally better year than those in the recent past.
“This is a tough business. It’s never a straight line from start to success.” Those words, from Exelixis Inc. CEO Michael Morrissey, during a presentation at the J.P. Morgan Healthcare Conference (JPM), could easily sum up any aspect of the biopharma industry. But with more biopharma firms than ever having reached commercial status, along with the introduction of new therapeutic modalities into the health care market, many are finding the toughest part comes after regulatory approval, whether it’s navigating a competitive landscape, getting payers and physicians on board, or satisfying regulators’ stringent postmarketing requirements. As industry players and observers head home after a busy week in San Francisco, BioWorld offers a brief glimpse at a few firms taking on those post-approval challenges in 2024.
Investigators from Biomarin Pharmaceutical Inc. have presented the first preclinical data for BMN-293, a novel adeno-associated virus (AAV) gene transfer vector carrying the MYBPC3 gene. MYBPC3 mutations can cause hypertrophic cardiomyopathy (HCM), a heart medical condition characterized by an abnormally thick myocardium, which makes it more difficult for the heart to pump blood.
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.
Becoming the first gene therapy approved for hemophilia A, Roctavian (valoctocogene roxaparvovec) finally received the U.S. FDA’s blessing on June 29, after developer Biomarin Pharmaceutical Inc. spent nearly three years working to address issues raised in a 2020 complete response letter. The approval came a day prior to the June 30 PDUFA date.
After almost 30 years in business, Sangamo Therapeutics Inc. is finally nearing a BLA filing for one of its programs. But the company, wounded by the recent loss of alliances with Biogen Inc. and Novartis AG, is also running out of cash and investor interest – and it badly needs a new deal to stay afloat.
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.
An uptick in positive clinical data reported in recent months has helped nudge the BioWorld Biopharmaceutical Index (BBI) to its highest point this year. BBI is up by 15.58%, significantly higher than the 1.36% increase recorded at the end of August, just as statistically significant clinical results began to roll in from a number of biopharma companies.
After winning the backing of European regulators, Biomarin Pharmaceutical Inc. is returning to the U.S. FDA with its hemophilia A gene therapy, valoctocogene roxaparvovec, following a rejection in August 2020. It's armed with data it hopes will assuage concerns about long-term safety and benefits.