Blueprint Medicines Corp. found a new U.S. commercialization home for Gavreto (pralsetinib) through an agreement with Rigel Pharmaceuticals Inc. potentially worth $117.5 million, now that Roche Holding AG has relinquished all rights. The product, a once-daily oral small-molecule kinase inhibitor of wild-type RET (rearranged during transfection) and oncogenic RET fusions, received accelerated approval in 2020 by the U.S. FDA, under priority review and with orphan drug designation, to treat adults with metastatic RET fusion-positive non-small-cell lung cancer.
Alnylam Pharmaceuticals Inc.’s decision to change its analysis plan for the phase III Helios-B trial of RNAi therapeutic Amvuttra (vutrisiran) to treat transthyretin amyloidosis with cardiomyopathy, pushing top-line results back by three months, pressured its share price (NASDAQ:ALNY) down by 10% on Feb. 15, while also boosting shares of competitor Bridgebio Pharma Inc. (NASDAQ:BBIO) by 14%.
Raising $94 million in a series A round, South San Francisco-based Firefly Bio Inc. has emerged from stealth to advance its Firelink linker platform technology to develop degrader-antibody conjugates (DACs) to treat cancer. A combination of antibody-drug conjugates (ADCs) and targeted protein degradation therapies, DACs are a new class of medicines that have recently gained attention for their ability to replace toxic ADC payloads and to eliminate cancer-driven proteins.
Weeks after Roche Holding AG paid a $40 million milestone payment to partner Repare Therapeutics Inc. when the first cancer patient in the phase II Tapistry trial was dosed with camonsertib, the Basel, Switzerland-based pharma backed out of the 20-month-old deal.
A disease-modifying gene therapy for Sanfilippo syndrome type A has demonstrated reductions in heparin sulfate within cerebrospinal fluid, as well as increases in the cognitive function of young patients, arming Ultragenyx Pharmaceutical Inc. with data needed to support an accelerated BLA with the U.S. FDA.
A rare pediatric disease designation for its achondroplasia treatment and a subsequent $200 million private placement boosted shares of Tyra Biosciences Inc. on Feb. 2 by 29.3%.
A company focused on regulating immune response through nanoparticle technology, Cour Pharmaceuticals Development Co. Inc. has raised $105 million in a series A round to move its lead autoimmune disease products into phase IIa trials.
Be it viral, nucleic acid or protein vaccines, recent efforts that led to the first regulatory approvals for not only COVID-19, but also for malaria and respiratory syncytial virus, positioned infectious diseases in the headlines for much of the last four years.
Becoming the first treatment for children ages 1 to 11 with eosinophilic esophagitis (EE), Sanofi SA and Regeneron Pharmaceuticals Inc.’s IL-4/IL-13 inhibitor Dupixent (dupilumab) was cleared by the U.S. FDA on Jan. 25.
Be it viral, nucleic acid or protein vaccines, recent efforts that led to the first regulatory approvals for not only COVID-19, but also for malaria and respiratory syncytial virus, positioned infectious diseases in the headlines for much of the last four years. But despite that attention, or the threat of future pandemics, or the numerous infectious diseases for which there are no preventable vaccines and very little development activity, the level of private and public funding for biopharma companies working in the space is dismal – at least compared with that of oncology products, according to a new analysis report released by the Biotechnology Innovation Organization (BIO) on Jan. 25.
