Drug Development, Licensing

Clozel hands over a rival to PTC’s controversial steroid deflazacort — now in a pivotal study for Duchenne MD

Back when Jean-Paul Clozel sold Actelion to J&J, he kept a pipeline of experimental meds for his new company Idorsia. One of those therapies was vamorolon, a mid-stage compound which he had in-licensed from Rockville, MD-based ReveraGen BioPharma.

Jean-Paul Clozel

Today, Idorsia is spinning the compound out to Santhera (SIX: SANN) in a sub-license, handing over a treatment that’s now in a pivotal Phase IIb with hopes that it can replace the current glucocorticoids used to help young boys with Duchenne muscular dystrophy.

Idorsia gets $20 million, most of which is intended to cover its costs so far in a Phase IIb trial. It’s also getting a million shares in Santhera, which makes it a big shareholder with 13.3% of the equity.

The drug is billed as a dissociative steroid, designed to enhance strength like any steroid but without the severe side effects — including stunted growth — that is tied to steroids.

If Santhera now proves successful in the pivotal study, they could overtake deflazacort, a controversial steroid that Marathon was able to pivot to a quick and economical FDA approval specifically for Duchenne muscular dystrophy — before the subsequent controversy caused Marathon to sell the drug to PTC Therapeutics.

PTC is now selling deflazacort in the US at a big multiple to the $1,000 a year that DMD parents had been paying to import the cheap, old steroid from Europe. The parents preferred it to prednisone, deeming it better at helping preserve their child’s strength without the kind of weight gain associated with prednisone.

PTC has managed to avoid the controversy and keep the growing revenue — $22.6 million in Q3.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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