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Pharma & Biotech

Catabasis Pharmaceuticals reports positive pre-clinical data for DMD treatment

Dr H Lee Sweeney, who led the research, said: "There remains a large unmet need in Duchenne for therapies that can treat all affected boys and slow disease progression.

Shares in Catabasis Pharmaceuticals Inc (NASDAQ:CATB) advanced as it reported positive pre-clinical data for the treatment of muscle-wasting disease Duchenne muscular dystrophy (DMD).

The data comes from the edasalonexent programme, which showed two inhibitors edasalonexent (CAT-1004) and CAT-1041 are effective in making better the dystrophic process in two animal models.

Dr H Lee Sweeney, who led the research, said: "There remains a large unmet need in Duchenne for therapies that can treat all affected boys and slow disease progression.

"The orally bioavailable NF-kB inhibitors, edasalonexent and CAT-1041, improve the severe dystrophic phenotype found in both mechanically-damaged mdx mice and a GRMD dog and create an environment that can support more successful muscle regeneration."

Catabasis's chief scientific officer Dr Andrew Nichols, added: "We very much appreciate the research performed by Dr Sweeney and his colleagues.

"We agree that these data support edasalonexent as a potential treatment to improve both the quantity and quality of muscle fibers in boys affected by DMD and look forward to the Phase 2 clinical trial results with edasalonexent in the first half of Q1 2017."

Another DMD firm - Summit Therapeutics (LON:SUMM) is in a phase II trial with its potentially breakthrough treatment for the disease.

In October, it inked a licensing deal with US biotech Sarepta Therapeutics (NASDAQ:SRPT) worth up to US$522mln.

Shares added 2.49% to US$3.70.

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