• In total, 3553 subjects were evaluated for safety (rimegepant n=1771, placebo n=1782), and 3507 subjects were evaluated for efficacy (rimegepant n=1749, placebo n=1758)

• The sex, age, and race subgroups were demographically balanced (Table 1)• The most common historical MBS was photophobia (57%)• About 15% of subjects were using preventive treatment

Safety

EndpointRimegepant 75 mg

N=1749n (%)

PlaceboN=1758n (%)

Pain relief, 2 h 1013 (57.9) 771 (43.9)

Ability to function normally, 2 h 611 (34.9) 419 (23.8)

Sustained pain relief, 2–24 h 760 (43.5) 482 (27.4)

Used rescue medication within 24 h 319 (18.2) 569 (32.4)

Sustained pain relief, 2–48 h 660 (37.8) 422 (24.0)

Freedom from photophobia,a 2 h 545 (35.1) 376 (23.9)

Sustained pain freedom, 2–24 h 247 (14.1) 120 (6.8)

Freedom from phonophobia,b 2 h 454 (39.2) 348 (29.3)

Sustained pain freedom, 2–48 h 206 (11.8) 108 (6.2)

Freedom from nausea,c 2 h 523 (48.8) 473 (43.5)

Pain relapse,d 2–48 h 145 (41.7) 107 (50.0)MBS, most bothersome symptomaRimegepant (n=1552), placebo (n=1571)bRimegepant (n=1158), placebo (n=1187)cRimegepant (n=1070), placebo (n=1088)dRimegepant (n=351), placebo (n=215)

Results cont.

• Pooled efficacy results were similar to the individual trials and showed that rimegepant was moreeffective than placebo for the coprimary efficacy endpoints of freedom from pain (20.1% vs 12.2%) andfreedom from the MBS (36.4% vs 26.6%) at 2 hours postdose

• As shown in Table 4, the pooled analysis found that a single dose of rimegepant 75 mg was moreeffective than placebo on multiple secondary efficacy endpoints, including:–Pain relief and functional disability freedom– Freedom from nausea, photophobia, and phonophobia–Sustained efficacy endpoints from 2 through 48 hours postdose–Use of rescue medication

Safety

Table 4. Secondary Endpoints in the Pooled Population

• In selecting pharmacotherapy for acute treatment of migraine, the sex, age, and race of patients cancomplicate well known safety and tolerability issues, such as adverse events causing delays oravoidance of treatment and cardiovascular contraindications with triptans

• Rimegepant is an orally administered small molecule calcitonin gene-related peptide receptorantagonist that has demonstrated efficacy and safety in the acute treatment of migraine in 3 separatePhase 3 clinical trials1-3 — 2 using an oral tablet and 1 with a novel orally disintegrating tablet (ODT)utilizing the Zydis® fast-dissolve technology to optimize speed of onset and ease of use; rimegepantODT demonstrated statistically significant pain relief and return to normal function at 60 minutes andthrough 48 hours postdose

• The objective of this pooled analysis was to evaluate the effects of subject sex, age, and race on thesafety and tolerability of rimegepant 75 mg and placebo

Objective

• Three double-blind, randomized, placebo-controlled, multicenter trials of identical design (Figure 1)were conducted utilizing an oral tablet (Study 301, NCT03235479; Study 302, NCT03237845) and anODT (Study 303, NCT03461757)

Methods

Figure 1. Study Design

• Subjects used an eDiary to record data from predose through 48 hours postdose

• Aged ≥18 years, with ≥1-year history of ICHD-3 beta migraine• Two to 8 moderate or severe monthly migraine attacks; <15 monthly headache days (migraine or

nonmigraine) over the last 3 months• Preventive migraine medication dose stable for ≥3 months (if using)

Subjects

• Safety assessments: adverse events (AEs), ECGs, vital signs, physical measurements, routinelaboratory tests, including liver function tests

• Coprimary efficacy endpoints: pain freedom at 2 hours postdose and freedom from the mostbothersome symptom (MBS) at 2 hours postdose

• A range of secondary endpoints were assessed to determine efficacy at timepoints out to 48 hours postdose

Assessments

• Safety analyses were conducted on enrolled subjects who took any dose of study medication(rimegepant or placebo)

• Subjects were grouped by sex (female, male), age (<40, ≥40, <65, ≥65 years), and race (White, non-White) for analyses of AEs

• Efficacy analyses were performed on the modified intent-to-treat population• Efficacy endpoints were evaluated using Cochran-Mantel Haenszel tests and stratified by use of

preventive medication; noncompleters were classified as failures

Statistical Analysis

Introduction

ResultsSubjects

Conclusions•Pooled results from 3 Phase 3 randomized controlled clinical trials in the acutetreatment of migraine demonstrate that the safety and tolerability of rimegepant wassimilar to placebo

•No drug-related liver safety signals were identified•There were no meaningful differences in safety and tolerability between rimegepant andplacebo in the sex, age, and race subgroups

•These results confirm that rimegepant has a favorable benefit/risk profile for the acutetreatment of migraine

Results cont.

Benefits

• A total of 14.2% (252/1771) and 11.7% (209/1782) of subjects in the rimegepant and placebo groups,respectively, experienced at least 1 AE

• The most common AEs were nausea and urinary tract infection (≤1.5%)• No serious treatment-related AEs were reported• Serum alanine transaminase (ALT) and aspartate transaminase (AST) levels greater than the upper

limit of normal (ULN) were seen in 2.7% (48/1771) and 2.9% (52/1782) of subjects treated withrimegepant and placebo, respectively; 2 subjects (.1%) in each treatment group had ALT or AST >3x ULN

• Tables 2 and 3 show that sex, age, and race had no effect on safety outcomes

Table 2. Pooled On-Treatment Adverse Events (by Sex)

Rimegepant 75 mgN=1771n (%)

PlaceboN=1782n (%)

<40 YEARS N=888 N=896≥1 AE 98 (11.0) 81 (9.0)

Nausea 16 (1.8) 9 (1.0)Urinary tract infection 7 (.8) 6 (.7)

Treatment-related SAEs 0 0≥40 YEARS N=883 N=886≥1 AE 94 (10.6) 73 (8.2)

Nausea 10 (1.1) 5 (.6)Urinary tract infection 7 (.8) 0

Treatment-related SAEs 0 0<65 YEARS N=1735 N=1730≥1 AE 187 (10.8) 149 (8.6)

Nausea 25 (1.4) 12 (.7)Urinary tract infection 13 (.7) 6 (.3)

Treatment-related SAEs 0 0≥65 YEARS N=36 N=52≥1 AE 5 (13.9) 5 (9.6)

Nausea 1 (2.8) 2 (3.8)Urinary tract infection 1 (2.8) 0

Treatment-related SAEs 0 0WHITE N=1322 N=1378≥1 AE 138 (10.4) 121 (8.8)

Nausea 18 (1.4) 12 (.9)Urinary tract infection 10 (.8) 4 (.3)

Treatment-related SAEs 0 0NON-WHITE N=449 N=404≥1 AE 54 (12.1) 33 (8.2)

Nausea 8 (1.8) 2 (.1)Urinary tract infection 4 (.9) 2 (.1)

Treatment-related SAEs 0 0AE, adverse event; SAE, serious adverse event

Table 3. Pooled On-Treatment Adverse Events (by Age and Race)

Rimegepant 75 mgN=1771n (%)

PlaceboN=1782n (%)

FEMALE N=1529 N=1534≥1 AE 171 (11.2) 136 (8.9)

Nausea 23 (1.5) 11 (.7)Urinary tract infection 14 (.9) 6 (.4)

Treatment-related SAEs 0 0MALE N=242 N=248≥1 AE 21 (8.7) 18 (7.3)

Nausea 3 (1.2) 3 (1.2)Nasopharyngitis 2 (.8) 0

Treatment-related SAEs 0 0AE, adverse event; SAE, serious adverse event

Rimegepant 75 mgN=1771n (%)

PlaceboN=1782n (%)

SEXFemale 1529 (86.3) 1534 (86.1)Male 242 (13.7) 248 (13.9)

AGE, YEARS<40 888 (50.1) 896 (50.3)≥40 883 (49.9) 886 (49.7)<65 1735 (98.0) 1730 (97.1)≥65 36 (2.0) 52 (2.9)

RACEWhite 1322 (74.6) 1378 (77.3)Non-Whitea 449 (25.4) 404 (22.7)

aIncludes Black/African-American, Asian, and other

Table 1. Demographics of the Pooled Population by Sex, Age, and Race

Safety of Rimegepant 75 mg in Adults With Migraine: No Effects of Age, Sex, or Race in 3 Phase 3 TrialsSusan Hutchinson, MD1; Jack Schim, MD2; Richard B. Lipton, MD3; Alexandra Thiry, PhD4; Beth Morris, BS4; Vladimir Coric, MD4; Robert Croop, MD4

1 Orange County Migraine & Headache Center, Irvine, CA; 2 Headache Center of Southern California, Carlsbad, CA; 3 Albert Einstein College of Medicine, Bronx, NY; 4 Biohaven Pharmaceuticals, Inc., New Haven, CT

Poster No. IHC-PO-136

References: 1. Lipton RB et al. Headache. 2018;58:1336–37 (Poster #PS123LB); 5; Lipton RB et al. N Engl J Med. 2019;381:142-49; 6. Croop R et al. Lancet. 2019. doi: 10.1016/S0140-6736(19)31606-X.Disclosures This study was sponsored by Biohaven Pharmaceuticals. SH and RBL have received honoraria and research support from Biohaven Pharmaceuticals; RBL is also a stockholder. AT, BAM, 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.

19th International Headache Congress | September 5-8, 2019 | Dublin, IrelandTo 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.