Receptos shares spike as oral drug reduces brain lesions in PhII MS trial

Last year Receptos pulled off one of the most successful biotech IPOs in a red-hot market for new offerings, largely on the promise of a midstage study underway for its oral multiple sclerosis drug. And this morning investors cheered the biotech on, spurring a 35% spike in its share price as investigators spelled out promising data from that Phase II trial.

The trial recruited 258 patients with relapsing MS. In the drug arm, patients responded with an 86% reduction in brain lesions for both doses that were studied--a statistically significant effect compared to placebo. And at least one analyst was quick to call it out as a potential improvement over Gilenya, one of the leaders in that segment of the market.

"(The drug) remains differentiated relative to Gilenya and that RCP1063 can be a notable drug in the growing oral MS market place," Credit Suisse analysts noted, according to a report from Reuters.

Receptos ($RCPT) is targeting a blockbuster MS market that has been undergoing a rapid transition to oral drugs. The once-daily Gilenya from Novartis ($NVS) recently won expanded approval in Europe, while Biogen Idec's ($BIIB) twice-daily Tecfidera has been taking the market by storm. Sanofi ($SNY), meanwhile, has introduced Aubagio, which has limited market appeal, and is trying to get an FDA approval for Lemtrada, which was initially spurned by the agency.

RPC1063, now in an ongoing Phase III trial, is a sphingosine 1-phosphate 1 receptor (S1P1R) small molecule which is also in the clinic for inflammatory bowel disease. The study wasn't powered to deliver a clear read on its impact on the rate of relapses, a major goal in this field, but investigators claimed that they tracked a "trend" in its favor. On the safety side, investigators have been carefully tracking its impact on QT--the body's electrically charged pattern of heartbeats--but they say that the trial only demonstrated a slight reduction in heartbeats among patients.

"The positive results of the Phase II portion of RADIANCE exceeded our expectations with respect to the differentiation thesis for RPC1063," said San Diego-based Receptos CEO Faheem Hasnain in a statement. "Based on our analysis of the Phase II data set, we believe that RPC1063 has the opportunity to be the best-in-class S1P receptor modulator. Our Phase III program is now well underway, positioning the program as the most advanced S1P receptor modulator in development for relapsing multiple sclerosis. In addition, we believe that RPC1063 may have promise in other therapeutic areas, and we continue to look forward to the results of TOUCHSTONE, our Phase II trial of RPC1063 in ulcerative colitis, in the fourth quarter of 2014."

- here's the release
- here's the Reuters report

Special Report: The top 10 biotech IPOs of 2013 - Receptos