Duchenne muscular dystrophy
Dyne’s exon-skipping therapy zeleciment rostudirsen resulted in an approximately sevenfold increase in dystrophin levels at six months and elicited functional improvements that are the “best ever” for this treatment class, Stifel analysts said.
Six months after receiving a surprise rejection due to what the FDA called “lack of substantial evidence of effectiveness,” Capricor’s cell therapy deramiocel showed significant benefits in upper-limb function and slowed decline in cardiac function in a Phase III trial.
Sarepta nevertheless plans to push for full FDA approval of Vyondys 53 and Amondys 45 based on what it said are “encouraging trends” in efficacy.
The takeover of its competitor, announced Sunday, could also bring some attention to Dyne Therapeutics, which has a similar RNA-based pipeline in rare muscle diseases.
A new analyst survey suggests that doctors are still prescribing Sarepta’s Elevidys, even after a series of deaths in certain populations marred the gene therapy’s record.
Analysts at BMO Capital Markets expect the lack of other exon-44-skiping therapies to facilitate a “smooth” approval process for Avidity’s del-zota.
As AAV9 and CRISPR programs navigate safety, delivery and scalability hurdles, small molecules offer a deployable, scalable bridge, complementing genetic approaches and accelerating meaningful impact for patients with Duchenne muscular dystrophy.
Capricor Therapeutics met with the FDA last week for a type A meeting, during which CEO Linda Marbán aimed to explain to the regulator that it got it wrong. Capricor plans to resubmit the application based on deramiocel’s existing dataset.
Prasad Returns, Delany Departs, Lilly’s Weight Loss Pill Disappoints and Sarepta’s Fallout Continues
CBER Chief Vinay Prasad reclaimed his job less than two weeks after his mysterious exit; MAHA implementor Gray Delany is out after reportedly sparring with other agency officials over communications strategy; Eli Lilly’s first Phase III readout for oral obesity drug orforglipron missed analyst expectations; and Arrowhead Pharmaceuticals addresses the recent woes of its of partner Sarepta.
Jefferies analysts said these detailed safety outcomes confirm the gene therapy’s positive risk/benefit profile in ambulatory patients with Duchenne muscular dystrophy.
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