Catabasis Pharmaceuticals, Inc. CATB announced that it has presented results from the part A of the MoveDMD study (phase I/II) on edasalonexent at the congress of the World Muscle Society.
Edasalonexent, a molecule that inhibits a protein called NF-kB is being developed for the treatment of patients with Duchenne muscular dystrophy (DMD).
The part A results revealed that a pre-selected NF-kB gene set was significantly inhibited in whole blood mRNA in boys with DMD, who were dosed with edasalonexent (67 mg/kg and 100 mg/kg) for one week.
Currently, edasalonexent is being evaluated in the part B (phase II) of the study to assess the safety and efficacy of the candidate for 12 weeks. Top-line data from the study are expected in the first half of 2017.
The candidate is also being assessed in an open-label extension (part C) of the phase II portion of the study. Patients completing part B is eligible to be enrolled in the extension study. They will receive edasalonexent for 36 weeks.
In the U.S., edasalonexent enjoys Orphan drug, Fast Track and Rare Pediatric Disease designations for the treatment of DMD. Edasalonexent also enjoys Orphan Medicinal Product designation for this indication in the EU.
DMD, a devastating and debilitating disease, represents a market with significant unmet medical need.
We remind investors that last month the FDA has granted accelerated approval to Sarepta Therapeutics, Inc.’s SRPT EXONDYS 51, the first DMD drug to be approved in the U.S.
Meanwhile, earlier this year, BioMarin Pharmaceutical Inc. BMRN had received a Complete Response Letter from the FDA for its DMD candidate, Kyndrisa.
Also, Mallinckrodt Public Limited Company MNK is developing Synacthen Depot for the treatment of DMD. In Aug 2016, the company received Fast Track designation in the U.S. for its Investigational New Drug (IND) application for Synacthen Depot in this indication.
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