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Santhera’s Raxone fails NIH’s MS study in latest setback, shares plunge

Just a few weeks after being shot down (again) by European regulators who were opposed to letting Santhera (SIX: SANN) extend its marketing approval for Raxone to include Duchenne muscular dystrophy, the biotech reported that an early-stage study of the drug failed to demonstrate any value for muscular sclerosis, either.

The NIH ran this study, dividing 77 patients between the drug and a placebo group (66 finished the trial), but found no difference between the two in controlling serious adverse events for patients with a primary progressive form of the disease.

Thomas Meier, CEO of Santhera

The Swiss biotech blamed the failure on the small size of the trial for idebenone. CEO Thomas Meier noted that “the small sample size is a limitation when studying a therapeutic intervention in such a complex, relentlessly progressing neurological disease.”

Raxone is approved in Europe to treat Leber’s hereditary optic neuropathy. Santhera’s shares plunged 15% on the news of the latest setback, which follows the EMA’s rejection of Santhera’s appeal of the Duchenne MD slap down.

The biotech has had a lot of trouble with clinical trial designs. Santhera had based its marketing pitch for DMD on data from a Phase III trial, but regulators say there wasn’t enough to prove its value — in a field where PTC obtained an accelerated approval following failed studies. And now the biotech has the drug back in another late-stage study.


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Biotech Investment Analyst
SV Health Investors Boston, MA
Research Scientist - Disease Biology
Recursion Pharmaceuticals Salt Lake City, UT
Senior Scientist - Predictive Pharmacology
Recursion Pharmaceuticals Salt Lake City, UT
Director, Translational Sciences
Cadent Therapeutics Cambridge, MA
Director of Marketing
twoXAR Mountain View, CA

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