Despite two recent clinical trial failures, Orphazyme A/S’s arimoclomol is still on track for its June 17 PDUFA date in treating Niemann-Pick disease type C. The newest stumble is in the pivotal study of arimoclomol for treating amyotrophic lateral sclerosis (ALS) as it failed to hit its primary and secondary endpoints.
CEO Dipal Doshi of Boston-based Entrada Therapeutics Inc. said the field of Duchenne muscular dystrophy (DMD) therapeutics has seen “a lot of first-generation, interesting programs that have kickstarted more focus” on the disease, “but no one really is fundamentally moving the needle in a robust clinical way.” His firm, with $116 million in new series B money, wants to change that. “Our focus on DMD is very direct and very specific,” he told BioWorld.
Top-line from Orphazyme A/S’ phase II/III trial of arimoclomol for treating inclusion body myositis, a muscle-wasting disease, failed to hit its primary and secondary endpoints. The data caused investors to pull back sharply as shares of Copenhagen-based Orphazyme (NASDAQ:ORPH) had dropped 28.97% on March 29 to close at $8.80 per share.
Shares of Israeli drug delivery specialist Entera Bio Ltd. (NASDAQ:ENTX) led the U.S. market March 11, in both percentage gain and volume, climbing 152.5% to $4.04 after a report that its oral parathyroid hormone candidate, EB-613, significantly boosted levels of P1NP, a biomarker indicating new bone formation vs. placebo at three months in an ongoing phase II osteoporosis study.
In the wake of Study 045’s failure with Translarna (ataluren) in nonsense mutation Duchenne muscular dystrophy, PTC Therapeutics Inc. is “trying to thread the needle between the notion of getting Study 041 completed in the third quarter of 2022 vs. getting accelerated approval now,” CEO Stuart Peltz said.
Sarepta Therapeutics Inc.’s miss on a key phase II ambulatory endpoint in its Duchenne muscular dystrophy (DMD) trial may have been caused by a dramatic disparity in functional ability at baseline among older vs. younger patients afflicted with the progressively worsening disorder. In any case, Wall Street had knives out, carving 51% of the value away from shares (NASDAQ:SRPT) of the Cambridge, Mass.-based firm, which closed at $82.29, a loss of $86.66, or 51%.
Precision Biosciences Inc. CEO Matthew Kane said the company is in “active discussions around additional partnerships in vivo and in other areas across our organization,” after scoring a deal with Eli Lilly and Co. centered on the firm’s Arcus genome-editing platform. “There’s no conceivable way in the near term that we’re going to advance all of the possibilities of Arcus on our own,” he said.
Solid Biosciences Inc. already has its own Duchenne muscular dystrophy program, but with some new investment money it is plunging into a collaboration with Ultragenyx Pharmaceutical Inc. to co-create another program. The two will collaborate on developing and commercializing new gene therapies for treating Duchenne muscular dystrophy as Novato, Calif.-based Ultragenyx is investing $40 million in Solid.