Receptos Doses First Patient in Phase 2 Trial for RPC1063 in Ulcerative Colitis

SAN DIEGO, Jan. 4, 2013 /PRNewswire/ -- Receptos, Inc., announced today that its selective sphingosine‐1‐ phosphate 1 receptor (S1P1R) modulator, RPC1063, has been administered to the first patient in a Phase 2 study examining the clinical efficacy and safety of induction therapy in patients with moderately to severely active ulcerative colitis (UC). Leading to the initiation of the RPC01-202 study, called TOUCHSTONE, Receptos successfully filed an Investigational New Drug (IND) Application with the Division of Gastroenterology and Inborn Errors Products (DGIEP) of the United States Food and Drug Administration (USFDA). Receptos also has an active IND for RPC1063 with the Division of Neurology Products (DNP), under which an ongoing Phase 2/3 study for RPC1063 in Relapsing Multiple Sclerosis (RMS) is being enrolled.

TOUCHSTONE is a multi-national, multi-center, double-blind, randomized, placebo-controlled study investigating the effect of two active doses of RPC1063 versus placebo. The study is designed to enroll approximately 180 patients in North America, Europe and Asia Pacific. The primary objective of TOUCHSTONE is to compare the efficacy of RPC1063 for the induction of clinical remission in patients with moderately to severely active UC at eight weeks of treatment.

Receptos has completed a Phase 1 study with RPC1063 which tested single ascending doses, multiple ascending doses and dose titration regimens in healthy volunteers. The Phase 1 results confirmed optimal pharmacokinetic, pharmacodynamic and safety features, which provide supportive data for the differentiation strategy for RPC1063 as a potential best-in-class second generation S1P1R modulator. In particular, robust pharmacodynamic effects of peripheral lymphocyte count reduction were achieved in the Phase 1 study. Clinical efficacy in UC has been demonstrated by several marketed or clinical stage lymphocyte trafficking agents with different mechanism of action to S1P1R modulation but that impact similar lymphocyte populations (naive T cells [CD4⁺, CD8⁺], memory T cell subsets, and B cells). These data, in addition to preclinical efficacy data generated at Receptos, provide rationale for anticipating clinical efficacy for S1P1R modulation in inflammatory bowel disease (IBD). New targeted immunotherapeutic options such as S1P1R modulation are expected to provide treating physicians with more choices in their treatment algorithm for IBD.

"Patients suffering from ulcerative colitis need more effective maintenance therapies with favorable safety profiles and oral administration for convenient long-term, chronic administration," said Faheem Hasnain, President and Chief Executive Officer of Receptos. "RPC1063 not only has the potential to demonstrate efficacy in both induction and maintenance therapy for ulcerative colitis, but could represent a first-in-class offering for S1P1R modulation as a novel mechanism of action to treat inflammatory bowel disease."

The UC market has significant unmet medical need for over 1.5 million patients, since all currently approved drugs have therapeutic, safety and logistic limitations. First-line therapies for UC have disadvantages ranging from limited efficacy (5-Aminosalicylic acid, also known as 5-ASA or mesalamine) to unacceptable side effects pursuant to chronic administration and the loss of patient response over time (corticosteroids). Azathioprine (AZA) and 6-Mercaptopurine (6-MP), used to maintain remission, have a slow onset of action and multiple notable toxicities. Approved anti-TNF (tumor necrosis factor) therapies Remicade^® (infliximab) and Humira^® (adalimumab), though effective in inducing and maintaining remission in a portion of UC patients, require repeated IV infusions, result in long-term immune suppression lasting up to 3 months, and have the potential for increased risk for infection. Finally, despite receiving standard therapy, more than half of all patients will experience an exacerbation of their disease with proctocolectomy (surgical removal of the rectum and all or parts of the colon) as their only alternative.

About RPC1063 and S1P1R Modulators 
RPC1063 is a novel, differentiated sphingosine 1-phosphate 1 receptor (S1P1R) selective modulator exhibiting picomolar potency that is effective in rodent models of both multiple sclerosis (MS) and IBD, and possesses an excellent safety profile in non-clinical toxicology studies. Receptos completed a Phase 1 study with RPC1063 in 2012 which tested single ascending doses, multiple ascending doses and dose titration regimens in healthy volunteers. The Phase 1 results confirmed optimal pharmacokinetic, pharmacodynamic and safety features, which provide supportive data for the differentiation strategy for RPC1063 as a potential best-in-class second generation S1P1R modulator.

About Receptos 
Receptos is a biopharmaceutical company developing therapeutic candidates for the treatment of autoimmune and metabolic diseases. The company's lead program, RPC1063, is a potential best-in-class S1P1R small molecule modulator candidate for autoimmune indications. In addition to the Phase 2 trial for UC, a Phase 2/3 study is ongoing examining the efficacy of RPC1063 in the indication of relapsing multiple sclerosis (RMS). Patents supporting RPC1063 were exclusively licensed to Receptos from The Scripps Research Institute (TSRI). The discoveries originated in the NIH Molecular Libraries Probe Production Center at TSRI, which is part of the NIH Common Fund Molecular Libraries initiative (http://commonfund.nih.gov/molecularlibraries/) . Receptos' expertise in S1P1 biology has been informed by the company's high resolution protein crystal structure of the S1P1 receptor, published in Science in 2012. Receptos has established partnerships for its GPCR structure determination technology platform with Eli Lilly, Ono Pharmaceutical and Janssen Pharmaceuticals, Inc. For more information visit www.receptos.com.

Media and Investor Contacts:
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SOURCE Receptos, Inc.