Gene therapy developer Kate Therapeutics Inc. (KateTx), which is developing next-generation adeno-associated virus (AAV) vectors that target skeletal and cardiac muscle, has unveiled $51 million series A round and a licensing deal with Astellas Pharma Inc.
Lack of efficacy brought the development of two investigational agents for amyotrophic lateral sclerosis (ASL) to a halt over the past week. On May 23, Wave Life Sciences Inc. disclosed that its stereopure antisense oligonucleotide WVE-004 failed to demonstrate clinical benefit after 24 weeks of treatment on a phase Ib/IIa trial in familial ALS patients or frontotemporal dementia patients. And on May 25, Apellis Pharmaceuticals Inc. and its partner, Swedish Orphan Biovitrum International AB, said that pegcetacoplan failed to meet its primary endpoint of a one-year phase II trial in patients with sporadic disease.
Blueprint Medicines Corp. scored a broader label from the U.S. FDA for Ayvakit (avapritinib), which became the first approved therapy to treat adults with indolent systemic mastocytosis (ISM).
Sarepta Therapeutics Inc.’s balloting March 12 from the U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee (OTAT) in favor of gene transfer therapy SRP-9001 (delandistrogene moxeparvovec) in Duchenne muscular dystrophy (DMD) had Wall Street mulling the odds for others in the space.
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.
How grave they might be remains unknown, but regulatory questions have surfaced in briefing documents related to the soon-to-happen panel meeting on Sarepta Therapeutics Inc.’s gene transfer therapy delandistrogene moxeparvovec in Duchenne muscular dystrophy (DMD). The U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee will meet May 12 to discuss the compound, also known as SRP-9001.
Despite the approval of Biogen Inc.’s Qalsody (tofersen) for treating amyotrophic lateral sclerosis (ALS), there are still hurdles for the drug to clear, including a confirmatory study and setting a price. Because the U.S. FDA granted Qalsody accelerated, not full approval, there are plenty more data to collect in the ongoing confirmatory phase III Atlas study of those who develop ALS symptoms during the trial compared to placebo. The randomized, double-blind, placebo-controlled study is being conducted with those carrying the superoxide dismutase 1 gene genetic mutation but are symptom free.
A twisted, uncertain path has led to a U.S. FDA approval for Biogen Inc.’s Qalsody (tofersen), the first drug targeting a genetic cause of amyotrophic lateral sclerosis (ALS). Qalsody is for ALS associated with a mutation in the superoxide dismutase 1 (SOD1) gene. Patients with SOD-1 mutations account for 2% of ALS cases.
Dupuytren’s disease is often referred to as “the most common crippling hand condition that people have never heard of,” but Kurt Harrington is on a mission to change that. A seasoned biotech and pharma consultant, Harrington has had the condition himself for over a decade and, acutely aware of the lack of available treatment options, has founded Ventoux Biosciences Inc. to bring additional therapies forward.
The editing in human cells and in mice of the survival motor neuron 1 gene (SMN1) restored the levels of SMN protein that the mutation of the SMN2 gene produces in spinal muscular atrophy. Scientists from the Broad Institute in Boston and The Ohio State University reversed the mutation using the base editing technique.