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Near-Term Launches

High-Value Pipeline Day:

Scientific Posters

December 12, 2022

Forward-Looking Statements, Non-GAAP Financial Information and Other Notices

FLS

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Near-Term Launches High-Value Pipeline Day | FLS

1

Introduction

• In the acute treatment of migraine, nonoral therapies are recommended for:
• • Attacks that include severe nausea or vomiting or rapidly escalating headache pain
  • Patients in whom oral forms are associated with inadequate response, slow onset of action, or poor tolerability1
• Although people with migraine commonly cite rapid relief as a high-priority treatment goal, lack of efficacy, adverse events (AEs), and cardiovascular complications limit the clinical utility of existing nonoral alternatives (triptans, ergot alkaloids)2
• Zavegepant is the only small molecule CGRP receptor antagonist delivered by nasal spray in late-stage development for the acute treatment of migraine
• A Phase 1 study found that the 10 mg nasal spray was rapidly absorbed (Tmax ~30 minutes)3; a dose-ranging study (NCT03872453) found that zavegepant 10 mg was the optimal dose4

Objective

• The objective of this study was to compare the efficacy and safety of zavegepant nasal spray with placebo in the acute treatment of migraine

Results

Subjects

• In total, 1978 subjects were recruited and screened for eligibility between 27 October 2020 and 20 August 2021; of these, 1405 were randomized, and 1269 were evaluable for efficacy (zavegepant n=623, placebo n=646)
• Demographics and other baseline characteristics are described in Table 1

Table 1. Demographics and baseline characteristics

			Zavegepant	Placebo	Overall
			Nasal Spray 10 mg
			(n=646)	N=1269
			(n=623)
		Age, years, mean (SD)	40.9 (13.22)	40.8 (13.46)	40.8 (13.34)
	Female, n (%)	506	(81.2)	546	(84.5)	1052 (82.9)
	Race, n (%)	511	(82.0)	540	(83.6)	1051 (82.8)
		White
		Black or African American	85 (13.6)	83 (12.8)	168	(13.2)
		Other	27	(4.3)	23	(3.6)	50	(3.9)
		Moderate-severe attacks per month, n, mean (SD)	4.6 (1.81)	4.7 (1.81)	4.7 (1.81)
	Using preventive migraine medication, n (%)	88 (14.1)	82 (12.7)	170	(13.4)

Efficacy

• Zavegepant was superior to placebo for the coprimary endpoints of freedom from pain (23.6% vs 14.9%, P<0.0001) and freedom from the MBS (39.6% vs 31.1%, P=0.0012) at 2 hours postdose
• Zavegepantwas also superior to placebo on multiple secondary endpoints (Table 2), including pain relief at 15 minutes, return to normal function at 30 minutes, and sustained pain relief from 2 to 48 hours postdose
• Zavegepant was more effective than placebo for pain relief from 15 minutes through 2 hours postdose (Figure 1)

Figure 1. Pain relief at 15, 30, 60 and 120 minutes postdose.

Methods

Design

• This was a phase 3, double-blind, randomized, placebo-controlled multicenter trial (NCT04571060)
• Subjects self-administered 1 dose of zavegepant 10 mg nasal spray or placebo to treat 1 migraine attack of moderate or severe pain intensity; they used an eDiary to record data from predose through 48 hours postdose
• Subjects were permitted to use only the following rescue medications: aspirin, ibuprofen, acetaminophen [up to 1000 mg/d](including Excedrin Migraine), naproxen [or any other nonsteroidal anti-inflammatory], antiemetics, or baclofen) 2 hours after study medication dose

Subjects

• Adults aged ≥18 years with ≥ 1-year history of migraine and, in the 3 months prior to screening, 2-8 moderate to severe monthly migraine attacks and >15 monthly headache days (migraine or nonmigraine)
• If using preventive medication, the dose had to be stable for ≥3 months

Assessments

• The coprimary efficacy endpoints were freedom from pain at 2 hours postdose and freedom from the most bothersome symptom (MBS) at 2 hours postdose; safety assessments included adverse events (AEs), ECGs, vital signs, physical measurements, clinical laboratory tests, Sheehan Suicidality Tracking Scale
• Randomization (1:1) was stratified by use of preventive migraine medication (yes or no)
• A hierarchical gatekeeper approach was used to control for multiple comparisons

Table 2. Summary of results on coprimary and secondary efficacy endpoints.a

		Zavegepant	Placebo		P-valueb
		Nasal Spray 10 mg
	Pain freedom at 2 hours postdose	147/623	(23.6)	96/646 (14.9)		<0.0001
	Freedom from the MBS at 2 hours postdose	247/623	(39.6)	201/646	(31.1)		0.0012
	Pain relief at 2 hours postdose	366/623	(58.7)	321/646	(49.7)		0.0012
	Return to normal function at 2 hours postdosec	204/570	(35.8)	152/593	(25.6)		0.0001
	Sustained pain relief from 2 to 24 hours postdose	253/623	(40.6)	213/646	(33.0)		0.0048
	Sustained pain relief from 2 to 48 hours postdose	225/623	(36.1)	191/646	(29.6)		0.0130
	Sustained pain freedom from 2 to 24 hours postdose	91/623 (14.6)	63/646	(9.8)		0.0076
	Sustained pain freedom from 2 to 48 hours postdose	77/623 (12.4)	56/646 (8.7)		0.0308
	Phonophobia freedom at 2 hours postdosed	167/407	(41.0)	139/425	(32.7)		0.0123
	Photophobia freedom at 2 hours postdosed	207/558	(37.1)	167/585	(28.5)		0.0018
	Pain relief at 60 minutes postdose	270/623 (43.3)	241/646	(37.3)		0.0293
	Return to normal function at 60 minutes postdosec	115/570	(20.2)	92/593 (15.5)		0.0362
	Pain relief at 30 minutes postdose	190/623	(30.5)	131/646	(20.3)		<0.0001
	Return to normal function at 30 minutes postdosec	60/570 (10.5)	36/593	(6.1)		0.0059
	Pain relief at 15 minutes postdose	99/623 (15.9)	52/646 (8.0)		<0.0001
	Return to normal function at 15 minutes postdosec	19/570	(3.3)	12/593	(2.0)		0.1826
	Rescue medication use within 24 hours postdosee	184/620	(29.7)	230/643	(35.8)		0.0194
	Nausea freedom at 2 hours postdosed	199/380	(52.4)	206/405	(50.9)		0.6753
	Pain relapse from 2 to 48 hours postdosef	60/147 (40.8)	34/96 (35.4)		0.3944

aSubjectstaking rescue medication at orbefore the time point were imputed asfailuresfor all endpointsexcept rescue medication use within 24 hourspostdose; bCochran-Mantel-Haenszel test stratified by preventive migraine medication use at randomization; cAmong subjectswith functional disability at time of dosing; dAmong subjectswith the symptom present at time of dosing; eSubjectswith rescue medication start date on or before the study drug start date + 1 dayand missing rescue medication start time were excluded; fAmong subjectswith pain freedom at 2 hourspostdose.

Safety and Tolerability

• Table 3 presents a summary of safety for treated subjects
• There were no serious AEs, and most AEs were mild or moderate in intensity; no signal of hepatotoxicity was observed
• The most common (≥2% of subjects) AEs were dysgeusia (20.5% vs 4.7%), nasal discomfort (3.7% vs 0.8%), and nausea (3.2% vs 1.1%)

Table 3. Treatment-emergent adverse events (≥2% of subjects)

	Zavegepant	Placeboa
	Nasal Spray 10 mga
	(n=653)
	(n=629)
Subjects with ≥1 AE, n (%)	186	(29.6)	102 (15.6)
AEs reported by ≥2% of subjects, n (%)
Dysgeusia	129	(20.5)	31 (4.7)
Nasal discomfort	23	(3.7)	5 (0.8)
Nausea	20	(3.2)	7 (1.1)

Conclusions

1. A 10 mg dose of zavegepant nasal spray was more effective than placebo in the acute treatment of migraine
2. Zavegepant nasal spray achieved its coprimary endpoints and provided a rapid onset of pain relief as early as 15 minutes postdose, sustained benefits to 48 hours postdose, and favorable safety and tolerability

Author Disclosures: RC, JM, JH, LM, ML, and VC are employed by and own stock/stock options in Biohaven Pharmaceuticals. RBL has received honoraria and research support from Biohaven Pharmaceuticals; he is also a stockholder.

References: 1. Marmura et al. Headache. 2015;55:3-20.2. Ailani et al. Headache. 2021;61:1021- 1039. 3. Lipton et al. Headache. 2002;42 Suppl 1:3-9.4. Ferrari et al. Lancet. 2001;358:1668-1675.5. Buse et al. Headache. 2017;57:31-44.6. Lipton et al. Headache. 2017;57:1507-1521.7. Boureau et al. Int J Clin Pract. 2000;54:281-286.8. Silberstein and Kori. CNS Drugs. 2013;27:385-394.9. Bertz et al. Headache. 2022;62(S1):122.10. Croop et al. Headache. 2021;61(S1):104-105.

To download a copy of this poster, scan QR code.

Presented at The 16th European Headache Congress

December 7-10, 2022 • Vienna, Austria and Virtual

aSafety was analyzed in subjectswho tookany dose of study medication (zavegepant or placebo).

IntroductionResults

Results cont.

Results cont.

• Rimegepant is an FDA-approved,orally-administered calcitonin gene-related peptide receptor antagonist that has demonstrated efficacy and safety in the acute treatment of migraine across multiple randomized, placebo-controlled clinical trials1-4
• In a long-term,open-label safety study, a reduction in monthly migraine days (MMDs) was observed with rimegepant 75 mg dosed every other day for 12 weeks, with no signs of medication-overuse headache5
• The reduction in MMDs generated the hypothesis that an every-other-day dosing regimen of rimegepant might be safe and effective for preventive treatment of migraine
• This was the first randomized, placebo-controlled clinical trial designed to evaluate rimegepant for the preventive treatment of migraine6

Objective

• Compare the efficacy, safety, and tolerability of rimegepant with placebo for the preventive treatment of migraine

Methods

• This randomized, double-blind,placebo-controlled trial (NCT03732638) was conducted at 92 study centers in the United States

Figure 1. Study Design

Assessments

• Primary efficacy endpoint: mean change from the 4-week observation period in the number of migraine days per month in the last 4 weeks of the double- blind treatment phase (weeks 9-12)
• Safety assessments: adverse events (AEs), including hepatic-related, and laboratory tests, including liver function tests

Statistical Analysis

• Efficacy population: randomized subjects who received ≥1 dose of rimegepant or placebo and had ≥14 days of electronic diary efficacy data from the 4-week observation period and for ≥1 of the 4-week intervals during double-blind treatment
• The primary efficacy endpoint was tested for superiority versus placebo using a generalized linear mixed-effect model; secondary endpoints were tested hierarchically to control Type I error
• A post-hoc analysis assessed mean percentage changes from the observation period in weekly migraine days at each week during the first 4 weeks of double-blind treatment; weekly migraine frequency in the observation period was computed using the number of migraine days over the 4-week period prorated to 7 days
• Safety population: subjects who received ≥1 dose of rimegepant or placebo

Subjects

• In total, 747 subjects were randomized (rimegepant n=373, placebo n=374); 741 were treated (rimegepant n=370, placebo n=371); and 695 were evaluated for efficacy (rimegepant n=348, placebo n=347)
• Most subjects (82.7%) were female, with a mean (SD) age of 41.2 (13.1) years
• The treated population had a mean (SD) history of 7.8 (2.7) moderate or severe attacks per month; 23.3% had a history of chronic migraine (rimegepant n=78 [21.1%], placebo n=95 [25.6%])
• During the observation period, efficacy-evaluable subjects in the rimegepant (n=348) and placebo (n=347) groups had a mean (SD) of 10.3 (3.2) and 9.9 (3.0) MMDs, respectively

Efficacy

• Rimegepant 75 mg was superior to placebo for the primary endpoint of mean change in MMDs during Weeks 9 through 12 (−4.3 vs −3.5, p=0.0099), as shown in Table 1

Table 1. Efficacy of Rimegepant in the Modified Intention-To- Treat Populationa

		Rimegepant	Placebo	Difference	P value
		75 mg
		n=348	n=347	(95% CI)
	PRIMARY EFFICACY ENDPOINT
	Mean change in number of	-4.3	-3.5	-0.8	0.0099
	MMDs during Weeks 9-12b	(-1.5 to -0.2)
	SECONDARY EFFICACY ENDPOINTS
	≥50% reduction in mean number			8
	of moderate or severe MMDs	49	41	0.0438
	(0 to 15)
	during Weeks 9-12, %
	Mean change in number of total	−3.6	−2.7	-0.8	0.0017
	MMDs during Weeks 1-12b	(-1.3 to -0.3)
	Rescue medication days per	3.7	4.0	-0.2	0.3868c
	month during Weeks 9-12b	(-0.8 to 0.3)
	Change in number of total	−2.9	−1.7	-1.2	<0.0001c
	MMDs during Weeks 1-4b	(-1.7 to -0.6)
	Change in MSQ restrictive role	18.0	14.6	3.5	0.0358c
	function at Week 12b.d	(0.2 to 6.7)
	Change in MIDAS total score at	−11.8	−11.7	-0.1	0.9616c
	Week 12b,d	(-4.7 to 4.5)

CI,Figureconfidence2.interval;SubjectsMMD,Withmonthly≥50%migraineReductionday; MSQ,inmigrainethe Mean-specificNumberquality of oflife

questionnaire;ModerateMIDAS,to SevereMigraineMigraineDisability AssessmentDays Per Month, Weeks 9 Through 12

aEvaluable subjects had ≥14 days of electronic diary efficacy data (not necessarily consecutive) in the observation period and ≥1 month (4-week interval) in the double-blind treatment phase. To control the type I statistical error rate at 0.05, a pre-planned hierarchical testing procedure was applied; endpoints are presented in the sequence in which they were evaluated; bReported as least squares mean and analyzed using a generalized linear mixed-effects model with treatment group, preventive migraine medication use at randomization, study month, and month-by-treatment group interaction as fixed effects and subject as random effect.; cNominal p-value in hierarchical testing; dReported as least squares mean; analysis only included subjects who completed the MIDAS or MSQ questionnaire within the prespecified on-treatment efficacy analysis window (Weeks 10-13; rimegepant n=269, placebo n=266).

Efficacy

• Rimegepant was also superior to placebo on the secondary endpoints of ≥50% reduction in the number of moderate or severe MMDs during Weeks 9-12 (Figure 2) and mean change in the number of total MMDs during Weeks 1-12 (Table 1)
• A post-hoc analysis assessed mean percentage changes from the observation period in weekly migraine days over the first 4 weeks of double-blind treatment (Figure 3); preventive effects of rimegepant were observed within the first week

Figure 2. Subjects With ≥50% Reduction in the Number of Moderate to Severe Migraine Days Per Month During Weeks 9-12

Figure 3. Percentage Change from the Observation Period in Migraine Days per Week During the First 4 Weeks of Double- Blind Treatmenta

Safety

• Subjects in the rimegepant and placebo treatment groups were equally likely to experience an AE, as shown in Table 2
• Nearly all AEs were mild or moderate in intensity
• No treatment-related serious AEs were reported in the rimegepant group
• The rate of discontinuations due to an AE was low in both treatment groups
• Four (1.1%) subjects who were treated with rimegepant and 2 (0.5%) subjects who were treated with placebo had aspartate aminotransferase or alanine aminotransferase elevations >3x ULN
• One (0.3%) subject in the rimegepant group had asymptomatic elevation of transaminases with ALT greater than 10x ULN, and alkaline phosphatase and bilirubin levels were always within normal limits; the site principal investigator deemed the increases not related to study drug, and an independent panel of liver experts concluded that the relation to study medication was not probable
• One (0.3%) subject in the rimegepant group had bilirubin levels greater than 2x ULN and was diagnosed with Gilbert's syndrome after genotyping

Table 2. Adverse Events With Rimegepant 75 mg and Placebo

	Rimegepant 75 mg	Placebo
	n=370	n=371
	n (%)	n (%)
Subjects with any AE	133	(35.9)	133	(35.8)
AEs (≥2% of subjects)
Nasopharyngitis	13	(3.5)	9	(2.4)
Nausea	10	(2.7)	3	(0.8)
Urinary tract infection	9	(2.4)	8	(2.2)
Upper respiratory tract infection	8	(2.2)	10	(2.7)
Subjects with mild AE	92	(24.9)	91	(24.5)
Subjects with moderate AE	64	(17.3)	62	(16.7)
Subjects with AEs related to	40	(10.8)	32	(8.6)
treatment
Serious AEs	3	(0.8)	4	(1.1)
Serious AEs related to treatment		0	1	(0.3)
AEs leading to discontinuation	7	(1.9)	4	(1.1)
AE, adverse event

Conclusions

• Oral rimegepant 75 mg taken every other day was effective for the preventive treatment of migraine
• Preventive efficacy was observed within the first week of rimegepant treatment
• Tolerability was similar to placebo, and there were no unexpected or serious safety issues
• These preventive effects, along with previously established acute treatment efficacy, suggest that rimegepant may provide a new approach to treat migraine in an adaptive way depending on an individual's treatment needs from acute to prevention

References: 1. Marcus R et al. Cephalalgia. 2014;34(2):114-25; 2. Lipton R et al. Headache. 2018;58:1336-37 (Poster #PS123LB); 3. Lipton RB et al. N Engl J Med. 2019;381:142-49; 4. Croop R et al. Lancet. 2019;394(10200):737-745; 5. Lipton RB et al. Cephalalgia. 2019; 39 (suppl): 1-337(IHC-PO-127); 6. Croop R et al.Lancet. 2021;397(10268):51-60.Disclosures This study was sponsored by Biohaven Pharmaceuticals. RC, DAS, LK, CMC, EGS, and VC are employed by and own stock/stock options in Biohaven Pharmaceuticals. RBL has received honoraria and research support from Biohaven Pharmaceuticals; RBL is also a stockholder. DK reports advisory board fees and research support from Biohaven Pharmaceuticals. PJG reports grants and personal fees from Biohaven Pharmaceuticals.

American Academy of Neurology 2021 Annual Meeting | Virtual Poster	To download a copy of
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Efficacy, Safety, and Tolerability of Rimegepant 75 mg Orally DissolvingTablet for the Acute Treatment of Migraine: A Phase 3, Double-Blind, Randomized, Placebo-Controlled Trial (Study 303)

Richard B. Lipton, MD1; Vladimir Coric, MD2; Elyse G. Stock, MD2; David A. Stock, PhD2; Charles M. Conway, PhD2; Christopher M. Jensen, PharmD2; Ralph Gosden, MSc3; Micaela Forshaw, MPH2; Robert Croop, MD2

1Albert Einstein College of Medicine, Bronx, NY, USA; 2Biohaven Pharmaceuticals, Inc., New Haven, CT, USA; 3Catalent UK Swindon Zydis Limited, Swindon, UK

Introduction

Results

Results cont.

Results cont.

• Rimegepant is an orally administered small molecule calcitonin gene- related peptide receptor antagonist
• Currently available acute treatments for migraine may be ineffective, poorly tolerated, or contraindicated in many patients1
• People with migraine prefer orally dissolving tablets (ODTs) to oral tablets, mainly for their convenience, onset of action, and ability to be taken without drinking liquids2-4
• Two Phase 3 clinical trials demonstrated efficacy and safety of rimegepant 75 mg oral tablet for the acute treatment of migraine (Study 301 and Study 302)
• A novel ODT formulation of rimegepant was developed utilizing the Zydis® fast-dissolve technology to optimize its speed of onset and ease of use
• Unlike other ODTs, rimegepant 75 mg ODT has a shorter Tmax (1.5 h) than the tablet (1.99 h; P=.0028), suggesting that the ODT may have a faster onset of action

Objective

• Assess rimegepant 75 mg ODT versus placebo on efficacy, safety, and tolerability in the acute treatment of migraine

Methods

• This was a double-blind, randomized, placebo-controlled, multicenter trial (Study 303, NCT03461757)

Figure 1. Study Design

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

Subjects

• 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)

Assessments

•	Coprimary efficacy endpoints: pain freedom at 2 hours postdose and
	freedom from the most bothersome symptom (MBS [ie, photophobia,
	phonophobia, or nausea]) at 2 hours postdose
•	Safety assessments: AEs, ECGs, vital signs, physical measurements,

Subjects

• In total, 1375 subjects were randomized and treated (rimegepant n=682, placebo n=693), and 1351 were evaluated for efficacy (rimegepant n=669, placebo n=682)
• Most subjects (84.9%) were female, with a mean (SD) age of 40.2 (12.0) years
• Mean (SD) history of attacks per month was 4.6 (1.8), and mean (SD) duration of untreated attacks was 29.5 (21.6) hours
• Historically, the most common MBS was photophobia (57.0%), followed by nausea (23.5%) and phonophobia (19.3%)
• Approximately 14% were using preventive treatment

Efficacy

• A single dose of rimegepant 75 mg ODT was superior to placebo for pain freedom (21.2% vs 10.9%, P<.0001) and freedom from the MBS (35.1% vs 26.8%, P=.0009) at 2 hours potdose, as well as on 19 prespecified, hierarchically-tested secondary endpoints (Figures 2 and 3), including:
• • Pain relief and functional disability freedom at 60 minutes postdose
  • Pain freedom and MBS freedom at 90 minutes postdose
  • Sustained efficacy endpoints from 2 through 48 hours postdose

Figure 2. Efficacy on Coprimary and Secondary Endpoints

Figure 3. Efficacy Summary: Pain and Return to Normal Function

Efficacy

• Rimegepant ODT was numerically separated from placebo on pain relief beginning at 15 minutes postdose; statistically significant at 60 minutes (Figure 4)

Figure 4. Pain Relief Through 2 Hours Postdose

Conclusions

• Rimegepant ODT was superior to placebo on rescue medication use (Figure 5); only 14.2% of rimegepant-treated subjects took rescue medication within 24 hours

Figure 5. Rescue Medication Use Up to 24 Hours Postdosea

Safety

• The most common AEs were nausea and urinary tract infection (≤1.6%)
• One rimegepant-treated and 1 placebo-treated subject had a transaminase level >3x ULN, but neither was assessed by the principal investigator as related to treatment
• No subjects had elevations in bilirubin >2 times the ULN
• Safety results are shown in Table 2

Table 2. Adverse Events

	Rimegepant	Placebo
	75 mg ODT
	N=693
	N=682
	n (%)
	n (%)
Participants with AEs	90 (13.2)	73 (10.5)
AEs reported by ≥1% of subjects
Nausea	11	(1.6)	3	(.4)
Urinary tract infection	10	(1.5)	4	(.6)
AEs related to treatment	47	(6.9)	36	(5.2)
Serious AEs	0	(.0)	0	(.0)

routine laboratory tests, including liver function tests

Statistical Analysis

• Efficacy analyses were performed on the modified intent-to-treat population: randomized subjects who had a qualifying migraine attack, took study medication, and provided ≥1 evaluable postbaseline efficacy data point
• Safety analyses were conducted on enrolled subjects who took any dose of study medication (rimegepant or placebo)
• A hierarchical gatekeeper approach was used to control for multiple comparisons

• In the acute treatment of migraine, a single dose of rimegepant 75 mg ODT was more effective than placebo
• Statistically significant and clinically meaningful effects on pain relief and functional disability freedom were observed at 60 minutes and sustained from 2 through 48 hours postdose
• Safety and tolerability were similar to placebo
• A single dose of rimegepant ODT has potential for early and sustained therapeutic action in the acute treatment of migraine

References: 1. Ferrari MD et al. Lancet. 2001;358:1668-1675; 2. Loder E et al. Headache. 2001;41:745-753; 3. Dowson AJ et al. Curr Med Res Opin. 2005;21 Suppl 3:S13-17; 4. Dowson AJ et al. Int J Clin Pract. 2003;57:573-576.Disclosures This study was sponsored by Biohaven Pharmaceuticals. RBL has received honoraria and research support from Biohaven Pharmaceuticals; he is also a stockholder. VC, EGS, DAS, CMC, CMJ, MF, and RC are employed by and own stock/stock options in Biohaven Pharmaceuticals.; RG is employed by Catalent Pharma Solutions. Zydis is a registered trademark of R.P. Scherer Technologies, Inc.

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