clinical pipeline
RPC1063
RPC1063, developed in the labs of Receptos, is a novel, orally available, potent and highly elective S1P1 agonist that induces rapid, reversible lymphopenia in rodents, dogs and nonhuman primates. Pharmacology studies in a mouse model of MS demonstrate dose proportional pharmacodynamic effects (lymphopenia) and significant reduction in EAE disease severity scores. Extensive non clinical in vivo safety studies have shown that RPC1063 is well tolerated in animals at doses that generate robust lymphopenia, suggesting that the compound is safe and has a broad therapeutic window.
RPC1063 has entered a single-ascending and multiple-ascending dose design Phase 1 clinical safety study under a US Investigational New Drug (IND) application. The study will generate data to confirm that the characteristics of RPC1063 meet pre-specified pharmacokinetic, pharmacodynamic, and safety criteria. These include half-life determination to support once-per-day dosing and measures that will focus on extent and speed of reversibility of lymphopenia. An understanding of the pharmacokinetic/pharmacodynamic relationship of RPC1063 will also allow accurate dose selection for Phase 2 evaluation. The Phase 1 study is anticipated to conclude in early 2012, paving the way for a Phase 2 proof-of-concept study in patients with relapsing-remitting MS.