Retrophin Inc (NASDAQ:RTRX) announced positive top-line results from the Phase 2 DUET study of sparsentan for the treatment of focal segmental glomerulosclerosis (FSGS), a rare kidney disorder without an approved pharmacologic treatment that often leads to end-stage renal disease. The study achieved statistical significance in the primary efficacy endpoint for the overall sparsentan treatment group, demonstrating a greater than two-fold reduction of proteinuria compared to irbesartan after the eight-week, double-blind treatment period.

“We are very pleased with the robust top-line results from DUET, which suggest sparsentan could be a significant advancement in the treatment of FSGS,” said Stephen Aselage, chief executive officer of Retrophin. “FSGS patients today face poor outcomes with limited medical options; we look forward to working with the FDA to find the most expeditious path forward that would deliver the first approved pharmacologic treatment to the FSGS community.”

In the DUET study, the mean reduction of proteinuria from baseline after eight weeks of treatment for all patients treated with 200, 400, and 800 mg/day of sparsentan (n=64) was 44.8 percent, compared to a mean reduction of proteinuria for all patients receiving 300 mg/day of irbesartan (n=32) of 18.5 percent (p=0.006). Further, the mean reduction of proteinuria from baseline after eight weeks of treatment for all patients treated with 400 mg and 800 mg doses of sparsentan (n=51) was 47.4 percent, compared to a mean proteinuria reduction of 19.0 percent for patients receiving 300 mg of irbesartan (n=25) in these two dose cohorts (p=0.011). The comparison of individual sparsentan dose cohorts to irbesartan showed clear signals of relative improvement, but did not reach statistical significance.

“The results from DUET serve as proof of concept for sparsentan’s novel approach of combining endothelin receptor type A blockade with angiotensin II inhibition for the treatment of FSGS,” said Alvin Shih, M.D., executive vice president and head of research & development for Retrophin. “Significant reductions in proteinuria, along with a well-tolerated preliminary safety profile have us excited about being one step closer to providing a new treatment option for patients with FSGS.”

Top-line results suggest sparsentan was generally safe and well-tolerated in the DUET study. One serious adverse event, anemia, classified as potentially related to treatment occurred in the sparsentan group but did not result in study discontinuation during the eight-week blinded treatment period. There were no withdrawals due to fluid retention during the eight-week blinded treatment period. All patients who completed the eight-week treatment period entered the ongoing open label extension study, and the vast majority of these patients continue to receive therapy.

The Company plans to present detailed study results, including data from the open label extension, at an upcoming medical meeting or in a peer-reviewed publication. (Original Source)

Shares of Retrophin jumped nearly 28% to $20.95 in pre-market trading. RTRX has a 1-year high of $29.64 and a 1-year low of $11.60. The stock’s 50-day moving average is $17.44 and its 200-day moving average is $16.27.

On the ratings front, Retrophin has been the subject of a number of recent research reports. In a report issued on August 7, Leerink analyst Joseph Schwartz reiterated a Buy rating on RTRX, with a price target of $27, which represents a potential upside of 65% from where the stock is currently trading. Separately, on August 5, BMO’s Do Kim reiterated a Buy rating on the stock and has a price target of $28.

According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Joseph Schwartz and Do Kim have a total average return of 9.3% and 14.2% respectively. Schwartz has a success rate of 49% and is ranked #327 out of 4147 analysts, while Kim has a success rate of 50% and is ranked #816.

Retrophin, Inc. is a fully integrated biopharmaceutical company, which develops pharmaceutical products for the treatment of rare diseases. It is focused on the development, acquisition and commercialization of therapies for the treatment of serious, catastrophic or rare diseases.