• Of the 2867 subjects who entered the observation period, 1894 were enrolled in the treatment period, and 1784 were treated with rimegepant (PRN [n=1498], QOD+PRN [n=286])

• Most subjects (90%) were female; median age was 43 years, and 3.6% of subjects were ≥65 years of age; and the median number of moderate to severe attacks per month was 6.0

• Rimegepant 4-week exposure data are shown in Table 1; overall, 105,192 doses of rimegepant 75 mg were administered

Safety

Long-Term, Open-Label Safety Study of Rimegepant 75 mg for the Treatment of Migraine (Study 201): Interim Analysis of Safety and Exploratory EfficacyRichard B. Lipton, MD1; Gary Berman, MD2; David Kudrow, MD3; Kathleen Mullin, MD4; Alexandra C. Thiry, PhD5; Meghan Lovegren, BS5; Vladimir Coric, MD5; Robert Croop, MD5

1 Albert Einstein College of Medicine, Bronx, NY; 2 Clinical Research Institute, Minneapolis, MN; 3 California Medical Clinic for Headache, Santa Monica, CA; 4 New England Institute for Neurology and Headache, Stamford, CT; 5 Biohaven Pharmaceuticals, New Haven, CT

Poster No. P235LB

References: 1. Resnik DB. Drug Des Devel Ther. 2007;1:1–5; 2. Lipton RB et al. Cephalalgia. 2018;38(1 suppl):123-55 (abstract MTIS2018-171); 3. Lipton RB et al. AAN 2019 Emerging Science presentation (Data Blitz #005, Poster #075). Disclosures This study was sponsored by Biohaven Pharmaceuticals. RBL, GB, DK, and have received honoraria and research support from Biohaven Pharmaceuticals; RBL is also a stockholder. ACT, ML, VC, and RC are employed by and own stock/stock options in Biohaven Pharmaceuticals. Zydis is a registered trademark of R.P. Scherer Technologies, Inc.

Results cont.

• A Phase 3 randomized, placebo-controlled trial (NCT03732638) is ongoing to confirm these preliminary findings regarding rimegepant as a preventive treatment for migraine

Efficacy

Ongoing Clinical Research

• Rimegepant is an orally administered small molecule calcitonin gene-related peptide receptor antagonist that has demonstrated efficacy and safety in the acute treatment of migraine in 3 separate Phase 3 clinical trials2,3 — 2 using an oral tablet and 1 with a novel orally disintegrating tablet (ODT) utilizing the Zydis® fast-dissolve technology; rimegepant ODT demonstrated statistically significant pain relief and return to normal function at 60 minutes and through 48 hours postdose

• The long-term safety of rimegepant and potential benefits of repeated dosing on monthly migraine days have not previously been assessed

• The objective of this study was to evaluate the long-term safety of rimegepant 75 mg dosed up to once daily; an exploratory objective was to evaluate the effects of repeated dosing of rimegepant on migraine day frequency

Objectives

• This is an ongoing multicenter, open-label, long-term safety study (Study 201, NCT03266588) of rimegepant 75 mg oral tablet (Figure 1)

Methods

Figure 1. Study Design

• Aged ≥18 years, with ≥1-year history of ICHD-3 beta migraine• Two to 14 moderate or severe monthly migraine attacks; a subgroup with 4 to 14

moderate to severe monthly attacks per month was assigned to QOD+PRN• If using preventive medication, stable dose for ≥3 months

Subjects

• Safety assessments: AEs, ECGs, vital signs, physical measurements, routine laboratory tests, including liver function tests

• Exploratory efficacy: reduction in monthly migraine days over time

Assessments

• Safety analyses were conducted on enrolled subjects who took any dose of study medication

• Preventive effects analysis was based on treated subjects with ≥14 days of data in the observation period and ≥1 interval of 4 weeks at any point in the treatment period

• This poster describes an interim analysis of data performed on 21 February 2019; dosing in the 12-week QOD+PRN enrollment group was complete

Statistical Analysis

Introduction ResultsSubjects

• Subjects with ≥14 migraine days per month during the observation period had reductions in monthly median migraine days over 1 year, even with PRN-only dosing for acute attacks (Figure 2)

• As shown in in Figure 3, in subjects in the QOD+PRN enrollment group with ≥14 migraine days/month during observation period, there was a mean reduction of 5.3 migraine days/month at month 3 (ie, weeks 9-12)

• A total of 48.4% of subjects in QOD+PRN enrollment group experienced ≥50% reduction from baseline during month 3 (ie, weeks 9-12) in the frequency of monthly migraine days with moderate to severe pain intensity (Figure 4); results were consistent regardless of if subjects had <14 or ≥14 migraine days during the observation period

Conclusions• Rimegepant 75 mg administered in multiple doses for up to 52

weeks was well tolerated, with a favorable safety profile consistent with previous clinical trial results

• Overall, only 2.7% of subjects discontinued due to an AE• High frequency dosing of rimegepant 75 mg at least every other

day appears to be safe, with no signal of hepatotoxicity• Rimegepant dosing led to substantial decreases from baseline in

migraine days per month when taken as needed for acute treatment and when taken every other day plus as needed

• In aggregate these data, alongside data from 3 Phase 3 acute treatment trials, suggest that rimegepant has the potential for benefit in both acute and preventive treatment for migraine

Results cont.Efficacy

PRN (2-8)n (%)

(n=1,017)

PRN (9-14)n (%)

(n=481)

ScheduledQOD+PRN

(n=286)

Overalln (%)

(N = 1,784)Subjects with ≥1 AE 659 (64.8) 294 (61.1) 109 (38.1) 1,062 (59.5)AE leading to discontinuation 24 (2.4) 15 (3.1) 9 (3.1) 48 (2.7)AEs reported in ≥ 2% overallUpper respiratory tract infection 108 (10.6) 31 (6.4) 12 (4.2) 151 (8.5)Nasopharyngitis 70 (6.9) 36 (7.5) 9 (3.1) 115 (6.4)Sinusitis 56 (5.5) 23 (4.8) 7 (2.4) 86 (4.8)Urinary tract infection 40 (3.9) 19 (4.0) 8 (2.8) 67 (3.8)Influenza 49 (4.8) 3 (0.6) 1 (0.3) 53 (3.0)Bronchitis 35 (3.4) 10 (2.1) 4 (1.4) 49 (2.7)Nausea 33 (3.2) 15 (3.1) 3 (1.0) 51 (2.9)Back pain 35 (3.4) 11 (2.3) 6 (2.1) 52 (2.9)Dizziness 25 (2.5) 11 (2.3) 3 (1.0) 39 (2.2)AE, adverse event

• As shown in Table 2, 59.5% of subjects experienced ≥1 AE, the majority of AEs were mild or moderate in intensity and were considered to be unrelated to rimegepant

• Overall, only 2.7% of subjects discontinued due to an AE• The most common AEs were upper respiratory tract infection (8.5%), nasopharyngitis

(6.4%), and sinusitis (4.8%)• No deaths have been reported in this study• SAEs were reported in 45 (2.5%) subjects

– SAEs considered by the investigator to be possibly (1 SAE) or unlikely (8 SAEs) related to study drug were reported in 9 (0.5%) subjects

– All other SAEs were considered by the investigator to be unrelated to rimegepant.• No clinically relevant laboratory abnormalities were observed• In the QOD+PRN enrollment group, no subject experienced ALT or AST levels >3x the

upper limit of normal (ULN) or bilirubin elevations >2x ULN during treatment with rimegepant (n=281 subjects with liver function test data)

Table 2. On-Treatment Adverse Events Reported in ≥ 2% of Subjects Overall

Figure 2. Reduction in Migraine Days per Month in All Subjects With ≥14 Migraine Days per Month

Figure 3. Moderate or Severe Migraine Days per Month in QOD+PRN Subjects With ≥14 Migraine Days per Month

• Subjects were instructed to self-administer treatment as follows:– As needed (PRN) enrollment groups: rimegepant 75 mg up to once daily as

needed to treat attacks of any pain intensity for 52 weeks – Scheduled dosing group (QOD+PRN): rimegepant 75 mg every other day (QOD)

supplemented by PRN dosing on nonscheduled dosing days to treat attacks of any pain intensity for 12 weeks

−5.3 days/month

−4.0 days/month

Figure 4. Percentage of Subjects With ≥50% Reduction in Moderate or Severe Migraine Days per Month in QOD+PRN Subjects

PRN (2-8)n (%)

(n=1,017)

PRN (9-14)n (%)

(n=481)

ScheduledQOD+PRN

(n=286)

Overalln (%)

(N = 1,784)

Mean (SD) 5.6 (3.46) 8.5 (4.12) 13.7 (2.93) 7.7 (4.60)Median (range) 4.9 (.2, 27.6) 7.9 (.7, 26.1) 14.2 (.3, 22.0) 6.5 (.2, 27.6)

Table 1. Average Rimegepant Exposure (Tablets per 4 Weeks)

American Headache Society 61st Annual Scientific Meeting | July 11-14, 2019 | Philadelphia, PA To download a copy of this poster, scan QR code.

Rimegepant is an investigational new drug, not approved or authorized for marketingin the U.S. or any country for any indication or treatment of any disease or condition.This material is being made available through Biohaven’s Medical Affairs Department.