Biohaven Pharmaceutical Holding Company Ltd. is presenting expanded data from two randomized, pivotal Phase 3 clinical trials of rimegepant, Biohaven's small molecule calcitonin gene-related peptide (CGRP) receptor antagonist, on June 30, 2018 at the American Headache Society (AHS) Annual Scientific Meeting 2018.

These data support rimegepant's potential to provide patients with pain freedom and freedom from the most bothersome migraine-associated symptom in the acute treatment of migraine using a single dose.

"The two acute treatment Phase 3 clinical trials demonstrate that a single dose of rimegepant can relieve pain and restore function for people with migraine," said Richard B. Lipton, M.D., Professor and Vice Chair of Neurology at the Albert Einstein College of Medicine and Montefiore Health System, Director of the Montefiore Headache Center, and Chair of Biohaven's CGRP Scientific Advisory Board. "Most importantly, rimegepant has the potential to help millions of people with migraine get back to work and back to their families."

In the two completed pivotal Phase 3 trials of rimegepant, 1,162 and 1,186 patients were randomized to receive a single dose of either rimegepant or placebo. A full spectrum of clinically meaningful benefit compared to placebo was seen across multiple outcome measures. These included the co-primary endpoints of pain freedom and freedom from the most bothersome symptom at two hours post-dosing. These gains were durable, with rimegepant outperforming placebo in terms of sustained pain freedom and sustained freedom from the most bothersome symptom from 2 to 24 hours, and from 2 to 48 hours. In terms of function, rimegepant-treated patients saw meaningful gains over placebo in both pain relief and freedom from functional disability at 2 hours post-dose. These gains were also durable, with rimegepant outperforming placebo in sustained pain relief and sustained freedom from functional disability from 2 to 24 hours and 2 to 48 hours. In addition, there was a low use of rescue medications in patients treated with rimegepant.

The Phase 3 trial results showed increasing benefit in relief from migraine-associated symptoms after dosing, with a greater proportion of rimegepant patients achieving freedom from photophobia (light sensitivity), phonophobia (sound sensitivity) and nausea over eight hours as compared to placebo. Rimegepant was generally well tolerated, with the most frequent adverse event, nausea, occurring in only 1.4% of rimegepant patients, as compared to 1.1% of patients on placebo. Additionally, rimegepant demonstrated a liver safety profile similar to placebo.

Biohaven is also presenting new data from a preclinical study of rimegepant and BHV-3500, a second small molecule product candidate from Biohaven's CGRP receptor antagonist platform, in which no active vasoconstrictive properties with either product candidates were observed. This differentiates the two CGRP receptor antagonists from the current standard of care for the acute treatment of migraine, the triptans, which have contraindications for use by patients with cardiovascular conditions such as coronary artery disease, and warnings about use by patients with multiple cardiovascular risk factors such as hypertension and diabetes. Patients with absolute cardiovascular contraindications to triptans are currently being enrolled in Biohaven's long-term safety study which began in 2017.

"We are excited about these positive Phase 3 trials and the consistency of benefit of rimegepant that we've seen across the program. These clinical results, coupled with the new preclinical data which support the potential for a favorable cardiovascular profile, highlight the potential for rimegepant to be an effective treatment for migraine, including for those who are not currently able to take the standard of care due to safety concerns," said Vlad Coric, M.D., CEO of Biohaven. "More than 10% of the U.S. adult population suffers from migraine, yet few meaningful advancements in treatment have been made in the last 25 years. We are committed to providing new options for those who suffer from migraine with the ongoing development of rimegepant and other therapies in our novel CGRP platform."

The Company is also presenting new data from a Phase 1 study evaluating the bioequivalence of oral tablet and ODT formulations of rimegepant. These data show that the fast-dissolve Zydis formulation has a favorable pharmacokinetic profile that might translate into an earlier onset of action, an important feature for patients in pain.