Top-line data from a phase II trial of Italfarmaco SpA's givinostat in men with Becker muscular dystrophy found it failed to affect a significant change in total muscle fibrosis vs. placebo, the study's primary endpoint. But whether the outcome reflected a shortcoming of the drug or a function of the rare condition's clinical profile remained uncertain, with the company placing its bets on the latter.
Fulcrum Therapeutics Inc.’s phase IIb data with losmapimod in facioscapulohumeral muscular dystrophy (FSHD) brought renewed hope for patients in what historically has proved a challenging therapeutic space. Though the firm’s oral p38 mitogen-activated protein kinase inhibitor missed its primary biomarker endpoint – changes in DUX4-driven gene expression – other indicators of benefit in the study called ReDUX4 painted a bright picture.
Blueprint Medicines Corp. gained the FDA’s nod for Ayvakit (avapritinib) to treat systemic mastocytosis (SM), adding another indication to the KIT inhibitor’s label. For the first time, patients have available a targeted therapy designed to block D816V mutant KIT, the central driver of the disease.
Trading in shares in AB Science SA was suspended Tuesday after a safety signal prompted a voluntary hold on two late-stage clinical studies of its masitinib, in mastocytosis and amyotrophic lateral sclerosis, respectively.
DUBLIN – Shares in Santhera Pharmaceuticals Holding AG surged by as much as 71% June 1 on news that the high-dose arm of a phase IIb pivotal trial of vamorolone hit the primary endpoint of an improvement vs. placebo in the time-to-stand velocity attained by ambulatory boys with Duchenne muscular dystrophy (DMD).
Reneo Pharmaceuticals Inc.’s $93.8 million IPO last month brought renewed attention to primary mitochondrial myopathy (PMM), a genetic disorder that impairs oxidative phosphorylation, affecting mainly muscles. The San Diego-based firm sold about 6.2 million shares at $15 each, but the stock (NASDAQ:RPHM) has since taken a dive, closing May 19 at $8.83.
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.
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