Drug Development, Setbacks

Summit execs sharpen the budget ax as Duchenne MD study flops — shares routed at Sarepta partner

Summit Therapeutics $SMMT has crushed high expectations for its Duchenne muscular dystrophy drug, reporting today that their therapy completely failed a key trial. Execs at the biotech say they’re now dumping the drug — the focus of an $842 million deal with Sarepta — and prepping a restructuring needed to survive the setback.

Glyn Edwards

The 48-week Phase II trial failed at the primary as well as secondary level, researchers say, leaving nothing worthwhile to pursue in a late-stage study. The entire program is being scrapped now, and the news hit shares hard, with the stock plunging 77%.

Sarepta $SRPT had partnered with Summit on the drug — providing $40 million upfront with R&D support — looking to move beyond its fragile success with their experimental Exondys51. That drug was approved for DMD after a bruising internal dispute at the FDA — but on the market with a label that says there’s still no solid evidence of its efficacy.

The Oxford-based Summit saw its shares surge back at the beginning of the year on positive preliminary results, with their utrophin modulator ezutromid demonstrating a 23% decrease in myosin. But biomarker activity is no stand-in for a disease like DMD.

Sarepta too has relied heavily on biomarker data for its therapies, bagging a notable but very early success with a gene therapy for the disease.

Summt is now planning some painful cost cutting, which likely means a reduction in headcount.

“While we believe utrophin modulation could still have a place in the treatment of DMD, it is clear that ezutromid is not providing a benefit for patients,” said CEO Glyn Edwards. “We therefore feel that our resources are better focused on the development of our promising pipeline of new mechanism antibiotics.”

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Sr. Manager, Regulatory Affairs, CMC
CytomX Therapeutics San Francisco, CA
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Catalytic Data Science Charleston, SC
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Flatiron Health New York City or San Francisco

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