Catabasis Pharmaceuticals, Inc. announced that it has initiated an open-label extension (Part C) of the phase II portion of its MoveDMD study (phase I/II) on edasalonexent (CAT-1004).

 

Edasalonexent, a molecule that inhibits a protein called – NF-kB – is being developed for the treatment of patients with Duchenne muscular dystrophy (DMD).

 

Patients who complete the 12-week phase II portion (Part B) of the MoveDMD study will be eligible to enroll in the open-label extension. They will be given edasalonexent for 36 weeks. The open-label extension study will evaluate the longer-term safety and efficacy of the candidate with the same clinical endpoints as Part B.

We note that Part B was initiated in Apr 2016 to evaluate the safety and efficacy of oral edasalonexent (67 mg/kg/day and 100 mg/kg/day) for 12 weeks, with the primary endpoint being the change in magnetic resonance imaging (MRI). Top-line results from Part B are expected in late 2016.

 

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 the DMD indication in the EU.

 

DMD, a devastating and debilitating disease, represents a market where there is currently no approved drug, indicating significant unmet need for treatments.

 

Note that Sarepta Therapeutics, Inc. (SRPT Quick QuoteSRPT - Free Report) is looking for an approval of its DMD drug, eteplirsen. A New Drug Application is currently under review in the U.S.

 

BioMarin Pharmaceutical Inc. (BMRN Quick QuoteBMRN - Free Report) was also developing its pipeline candidate, Kyndrisa, for the DMD indication. However, last month, the company announced that it has withdrawn a Marketing Authorisation Application (MAA) for the product from the EU, following discussions with the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP), which hinted at the issuance of a negative opinion. Kyndrisa has also received a Complete Response Letter from the FDA. Consequently, BioMarin had stopped developing Kyndrisa.

 

Catabasis currently carries a Zacks Rank #2 (Buy). Another favorably placed stock in the health care sector is GW Pharmaceuticals plc , sporting a Zacks Rank #1 (Strong Buy).