In Development for Acute Treatment and Prevention of Migraine
Rimegepant, the lead product candidate in our CGRP platform, is an orally available small molecule CGRP receptor antagonist. In March 2018, rimegepant met both registrational co-primary endpoints (pain freedom and freedom from most bothersome symptom at 2 hours) in two pivotal Phase 3 trials. In each trial, a single dose of rimegepant, without any rescue medications, was superior to placebo for pain freedom and pain relief at 2 hours post-dosing, and showed a profile of increasing improvement throughout the first eight hours that was sustained compared to placebo out to 24 and 48 hours. The vast majority of rimegepant treated patients did not take rescue medications during the 24 hour period after dosing. Rimegepant-treated patients also showed improvement on measures of functional disability with a greater proportion of patients achieving normal function. More than 3 times the number of subjects who responded to treatment preferred rimegepant over their previous therapy.
In addition to the durability of clinical effect seen across multiple outcome measures, rimegepant was also observed to be generally safe and well-tolerated in these Phase 3 trials, with a safety profile similar to placebo including liver safety, and favorable compared to historical triptan experience. The efficacy and safety profile of rimegepant has now been observed across three randomized controlled trials, including a Phase 2b study.
An open-label long-term safety study of rimegepant in patients with migraine that allows up to daily dosing is ongoing. A third Phase 3 clinical trial with a bioequivalent Zydis® orally dissolving tablet formulation is also underway. Results from both studies are expected in the fourth quarter of 2018.
The co-primary endpoints achieved in the Phase 3 trials are consistent with regulatory guidance from the U.S. Food and Drug Administration (FDA) and, together with the long-term safety data, provide the basis for a planned submission of a new drug application (NDA) to the FDA in 2019.
Zydis is a registered trademark of R.P. Scherer Technologies, Inc.