The debate over Sarepta Therapeutics Inc.’s gene transfer therapy, SRP-9001 (delandistrogene moxeparvovec), in Duchenne muscular dystrophy (DMD) proved as thorny as expected during a closely watched meeting of the U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee. Panelists voted on a single question: “Do the overall considerations of benefit and risk, taking into account the existing uncertainties, support accelerated approval of SRP-9001, using as a surrogate endpoint expression of Sarepta’s microdystrophin at week 12 after administration, for the treatment of ambulatory patients with DMD with a confirmed mutation in the DMD gene?” Balloting turned out 8 yes, 6 no. Read More
Biopharma financings are pacing more than $300 million higher than at the end of April last year, but are well behind the total through April 2021 when the value of financings more than doubled any recent year due to the response to the COVID-19 pandemic. Through the end of April, biopharmas have raised $19.83 billion this year, compared to $17.5 billion through the same month in 2022, $53.7 billion in 2021, $24.04 billion in 2020 and $19.53 billion in 2019. Read More
Despite congressional concerns about accelerated approval, the U.S. FDA’s use of the pathway is not slowing down. If anything, it’s picked up pace since Congress gave the agency stronger authority last year to monitor drugs approved based on a surrogate endpoint and to ensure that confirmatory trials are progressing in a timely way. Read More
Korean biopharmas in the antibody-drug conjugate (ADC) space can leverage the strength of the Korean ecosystem when partnering with global pharma companies, said investors during the BIO Korea 2023 conference in Seoul on May 12. Read More
Swiss biopharma startup Aphaia Pharma AG is taking the concept of “location, location, location” to its extreme. The company started dosing patients in a phase II trial of its lead candidate, Aph-012, in late April, 2023. The trial is a randomized, double-blind, placebo-controlled, multicenter proof-of-concept study to evaluate Aph-012’s ability to improve glucose tolerance in individuals with prediabetes, as measured by a pathological oral glucose tolerance test. In another phase II trial, Aph-012 is being tested as a weight loss drug for individuals with a BMI between 30 and 40. Aph-012’s active ingredient? Glucose. But delivered exactly to the right place. Read More
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Abeona, Bellerophon, Cassava, Cytokinetics, Extend, First Wave, Innovent, Mirum, Mithra, Olema, Onconova, Palisade, Praxis, Vir. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Cytoagents, Immunitybio, Lundbeck, Mindbio, Otsuka, Soligenix. Read More
