Retrophin (RTRX) announced positive results from a mid-stage clinical trial Wednesday in which patients with a rare kidney disease responded to treatment with its experimental drug sparsentan at more than twice the rate of patients receiving a control drug.
The company's next step will be to meet with the U.S. Food and Drug Administration to map out plans for a pivotal phase III study of sparsentan, with the goal of securing the drug's approved for the treatment of focal segmental glomerulosclerosis, or FSGS.
There are no currently approved drugs for the rare disease, which causes progressive scarring of the kidneys, leading to end-stage kidney disease.
Retrophin shares rose 41% to $22.98 in Wednesday trading.
Sparsentan is the first drug to be developed internally by Retrophin, an orphan disease company founded by Martin Shkreli in 2011. As CEO of Retrophin, Shkreli in-licensed sparsentan from Ligand Pharmaceuticals (LGND) in 2012 and helped design the successful clinical trial announced Wednesday.
Shkreli left Retrophin in 2014 to start up another drug company, Turing Pharma, and then became infamous as the reviled "Pharma Bro" who raised the price of an antiparasitic drug and was later arrested and indicted on federal charges of securities fraud.
The criticism, even hatred, of Shkreli might be justified, but if sparsentan is eventually approved, patients with FSGS will have him to thank for bringing the drug into clinical development.
The phase II study enrolled 109 patients with FSGS and randomized them to treatment with one of three doses of sparsentan or an active control arm treatment using another drug, irbesartan.