Slide 1: Corporate Presentation June 2025
© 2025 Biohaven, Ltd. All rights reserved. JENNIFER Living with SCA3 Participant in the Troriluzole Clinical Study Corporate Presentation June 2025
Slide 2: Forward-Looking Statement
Forward-Looking Statement This presentation includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements about Biohaven Ltd. (the “Company”) and our planned and ongoing trials for our troriluzole, taldefgrobep alfa, BHV-7000, BH
Slide 3: Working Towards Commercialization of Novel Therapeutics
Working Towards Commercialization of NOVEL THERAPEUTICS INNOVATING EXECUTING CREATING VALUE PIONEERING THERAPIES FOR RARE DISEASES Integrated DISCOVERY ENGINE CLINICALLY VALIDATED MoDE AND TRAP DEGRADERS ADVANCING CANCER TREATMENTS DIVERSIFIED PORTFOLIO TOP AREAS OF INNOVATION IMMUNOLOGY & INFLAMMAT
Slide 4: Creating Near-Term Value
CREATING NEAR-TERM VALUE Kv7 Program Expecting Pivotal Topline Results Depression — 2H 2025 Epilepsy — 1H 2026 Neuroinflammation Initiated Early Parkinson’s — 1H 2025 SCA NDA Under Priority Review PDUFA — 2H 2025 Pioneering Degrader Platform With 3 Assets in the Clinic Initiating Pivotal Trials Grav
Slide 5: Pipeline
ARIA, Amyloid-related imaging abnormalities; AAb, Autoantibody. PRECLINICAL PHASE 1 PHASE 2 PHASE 3 MARKET GLUTAMATE Troriluzole BHV-4157 Spinocerebellar Ataxia Obsessive-Compulsive Disorder MYOSTATIN Taldefgrobep Alfa BHV-2000 Spinal Muscular Atrophy Obesity ION CHANNEL Kv7 Activator BHV-7000 Focal
Slide 6: MoDE and TRAP Degraders
DEGRADATION TARGET BIFUNCTIONAL MoDE DEGRADER ASGPR RECEPTOR ON HEPATOCYTE MoDE and TRAP Degraders
Slide 7: Our Novel Degrader Platform
Gd-IgA1 IgG antibody BHV-1300 TRAPMoDE OUR NOVEL DEGRADER PLATFORM Revolutionary Yale-licensed technology to remove disease-causing proteins from the bodyKEY POINT Target a class of proteins implicated in pathogenesis of disease Remove specific disease-causing proteins and leave rest of immune syste
Slide 8: A Novel Mechanism: Hepatic ASGPR Receptor Harnessed
A Novel Mechanism: Hepatic ASGPR Receptor Harnessed for Efficient and Safe Removal of Circulating Pathogenic Targets subcutaneous or intravenous injection MoDE are administered via *Stylistic representation ASGPR, asialoglycoprotein receptor; MoDE , molecular degraders of extracellular proteins • In
Slide 9: A Transformational MoDE Drug Platform
A Transformational MoDE Drug Platform: Molecular Degraders of Extracellular Proteins (MoDE) Precisely balanced components selected for optimal efficacy, safety, and product profile TARGET BINDER STABLE LINKER ASGPR BINDER COMPOUND LIBRARY BINDER COMPOUND LIBRARY MoDE Platform IND, Investigational Ne
Slide 10: Catalyzing Innovation for Patients
Catalyzing Innovation for Patients and Early Demonstration of Removal of Disease-Causing Protein June 2025 Biohaven Investor Presentation10 C R E A T I N G V A L U EE X E C U T I N GI N N O V A T I N G Follow the Science Understand the Need PRECISION IMMUNOLOGY TARGET THE DISEASE, NOT THE PATIENT KE
Slide 11: Degraders Validated in the Clinic
BHV-1600 PERIPARTUM CARDIOMYOPATHY Biohaven’s Novel Degraders Validated in the Clinic: 166 Individuals Dosed With MoDE and TRAP Degraders BHV-1400 IgA NEPHROPATHY BHV-1300 GRAVES’ DISEASE PHASE 1 DATA DEMONSTRATES BIOHAVEN’S MoDE AND TRAP DEGRADERS: ✓Safe ✓Well-tolerated ✓Highly selective ✓Deep and
Slide 12: Degrader Platform Technology
P A T IE N T CENTRIC LIFE ALTE R IN G FAST AND DEEP Removes disease-causing proteins within hours EASY-TO-USE • Easy-to-use autoinjector for self-administration • Allows for concomitant use of biologics SELECTIVE Designed to target specific pathogenic species for maximal efficacy and minimal side ef
Slide 13: Degrader Clinical Milestones
1H 2025 BHV-1300 Graves’ Disease 2025 Study 2026 Topline Data BHV-1310 Myasthenia Gravis Study BHV-1600 Cardiomyopathy Study New MoDE and TRAP Degraders Enter Phase 1 IgG4 degrader, PLA2R autoantibody degrader, TSHR autoantibody degrader 2H 2025–2026 BHV-1400 IgA Nephropathy Study BHV-1300 Phase 1 B
Slide 14: Market Potential of Degrader Platform
IgG DEGRADERS Graves’ disease Rheumatoid arthritis (RA) Myasthenia gravis (MG) IgG DEGRADERS Gd-IgA1 DEGRADER β1AR AUTOANTIBODY DEGRADER IgG4 DEGRADER PROINSULIN AUTOANTIBODY DEGRADER PLA2R AUTOANTIBODY DEGRADER TSHR AUTOANTIBODY DEGRADER ~ $8B* > $15B* Market Potential of Biohaven’s Degrader Platfo
Slide 15: Gd-IgA1 TRAP Degrader
Onc A. Gd-IgA1 TRAP™ Degrader biohaven’
Slide 16: BHV-1400 Selectively Removes Gd-IgA1
BHV-1400 Selectively Removes Gd-IgA1, the Pathogenic Antibody in IgAN While Sparing Healthy Immunoglobulins Phase 1 Pharmacodynamic endpoint predicts success, enables speed; sparing of healthy immune components predicts improved safety Demonstrate Early Efficacy Create Value PIVOTAL TRIAL TO INITIAT
Slide 17: IgA Nephropathy Caused by Excess Gd-IgA1
Gd-IgA1 immune complexes deposit into the kidney, recruiting immune cells and causing the release of pro- inflammatory and profibrotic mediators S Y S T E M I C C I R C U L A T I O N Aberrant form of immunoglobulin, galactose-deficient IgA1 forms in excess Immunoglobulin (IgG, IgM or IgA) target Gd-
Slide 18: BHV-1400 Single Dose Removal of Gd-IgA1
-80 -70 -60 -50 -40 -30 -20 -10 0 BHV-1400: Single Subcutaneous Dose Delivers Rapid, Selective, Deep and Sustained Removal of Gd-IgA1 G d -I g A 1 % C h a n g e f ro m B a s e li n e IgG IgA IgE IgM Gd-IgA1 18 DAYS 2 3 4 5 108 H O U R S DEGRADERS June 2025 Biohaven Investor Presentation A single SC
Slide 19: BHV-1400 Targets Gd-IgA1 Without Reducing Healthy Immunoglobulins
BHV-1400 TRAP Degrader Targets Gd-IgA1 with Precision Without Reducing Healthy Immunoglobulins June 2025 Biohaven Investor Presentation19 IgG IgA IgE IgM BHV-1400 Single Dose SC Sibeprenlimab Ph12Povetacicept Ph1 IV1 Povetacicept Ph1 SC1 Atacicept3 DEGRADERS K E E P T H E P A T I E N T A T T H E C E
Slide 20: High Selectivity Predicts Improved Safety
High Selectivity Predicts Improved Safety DEGRADERS BHV-1400 Tarpeyo® (budesonide) Vanrafia® (astrasentan) Filspari® (sparsentan) Fabhalta® (iptacopan) Risk Evaluation and Mitigation Strategies (REMS) Not anticipated NO NO YES YES Black Box Not anticipated NO YES embryo-fetal toxicity YES hepatotoxi
Slide 21: Selective Removal Compared to Competitors
P R E C IS I O N TA R G E T IN G BHV-1400 SELECTIVELY DEGRADES ONLY Gd-IgA1 Targeting the pathogenesis of disease without immunosuppression TARGET B CELLS with global immunoglobulin suppression Povetacicept Atacicept Sibeprenlimab Zigakibart Felzartamab BHV-1400: Selective Removal of Disease-Causing
Slide 22: IgAN Phase 2/3 Study Design
Harnessing Efficient IgAN Trial Design to Address a High Unmet Need BHV-1400 Phase 2/3 Study Design 22 9 months 24-month Treatment Phase (SOC +) BHV-1400 (SOC +) Placebo Screening Pivotal Trial UPCR, Surrogate Endpoint for Accelerated Approval DATA READOUT DATA READOUT eGFR Confirmatory Endpoint (24
Slide 23: IgG MoDE Degrader
APPAR. © IgG MoDE™ Degrader biohaven’
Slide 24: BHV-1300 Deep, Rapid and Tunable IgG Reductions
-90 -80 -70 -60 -50 -40 -30 -20 -10 0 BHV-1300: Differentiated Small Molecule Degrader Achieves Deep, Rapid and Tunable IgG Reductions Customized to the Needs of Specific Diseases 24 June 2025 Biohaven Investor Presentation Ig G % C h a n g e f ro m B a s e li n e WEEK 1 (n=8) (n=8) WEEK 2 (n=8) (n=
Slide 25: BHV-1300 vs Plasma Exchange
-100 -80 -60 -40 -20 0 20 IgG BHV-1300: Offers a New Potential Paradigm for Management of Acute Disease, Lowering IgG as Rapidly and Deeply as Plasma Exchange June 2025 Biohaven Investor Presentation25 Two doses of BHV-1300 lower IgG as deeply and quickly as these invasive methods including plasma e
Slide 26: Replacement Plasma Exchange in an Auto-Injector
BHV-1300 Plasma Exchange BHV-1300 Could Represent Replacement Plasma Exchange (PLEX) in an Auto-Injector DEGRADERS K E E P T H E P A T I E N T A T T H E C E N T E R VS 26 June 2025 Biohaven Investor Presentation
Slide 27: Not Just Another FcRn Inhibitor
• 74.6% IgG lowering after load, 68.8% in maintenance in Vivacity MG-31,2 • IV infusion • Increased cholesterol (24%), muscle spasms (12%), edema (12%) • Approximately 61% IgG lowering @ week 4 (VYVGART Hytrulo® in MG trial)3 (Average 75% in MAD)4 • Prefilled Syringe • Cyclical dosing can lead to sy
Slide 28: Graves' Disease: Precision Target Selection
Graves’ Disease: Precision Target Selection Enables Speed and Facilitates Confidence of Success Follow the Science Understand the Need Phase 1 Pharmacodynamic endpoint predicts success and enables speed in Pivotal Studies PRECISION TARGETING OF DISEASE BIOLOGY 270M US ADULT POPULATION1 2.7M NEW ONSE
Slide 29: IgG Degrader Targets Root Cause of Graves'
Hepatocytes Biohaven IgG Degrader Targets the Root Cause of a Broad Autoimmune Disease to Treat and Prevent Multi-Organ Complications June 2025 Biohaven Investor Presentation29 Redirects Disease Causing Target to Liver for Removal + Biohaven IgG degrader Biohaven IgG degrader removes IgG to eliminat
Slide 30: Graves' Disease Pivotal Trial
Initiate 2H 2025 2H 2025 Graves’ Disease Pivotal Trial DESIGN Randomized, double-blind, placebo-controlled trial POPULATION Male and female adults with Graves’ disease TREATMENT DURATION 24-week treatment period KEY ENDPOINTS Normalization of T3, T4 and TSH without rescue at week 24 Biomarker driven
Slide 31: Myasthenia Gravis: Tunable IgG Degraders
Create Value PIVOTAL TRIAL TO INITIATE 2026 Myasthenia Gravis: Biohaven’s IgG Degraders are Tunable Therapy for Acute and Chronic Phases of Disease Phase 1 Pharmacodynamic endpoint predicts success and enables speed in Pivotal Studies Multi-billion dollar nationally and internationally growing marke
Slide 32: In Vivo Validation of IgG Degrader Mechanism
In Vivo Pre-Clinical Validation of the IgG Degrader Mechanism of Action Biohaven IgG degraders can rapidly clear IgG from the interstitial space and neuromuscular junction KEY POINT Ig G Ig G + B H V N D e g ra d e r IgG Reduction in Neuromuscular Junction with Degrader Treatment 4 hr 24 hr 48 hr DE
Slide 33: Cycles of Dosing with FcRns Lead to Symptom Rebound
MG-ADL scores rebound in between cycles Cycles of Dosing with FcRns Lead to Cycles of Recovery Punctuated by Periods of Symptom Rebound • Chronic diseases like MG require sustained therapy to prevent symptom fluctuations and worsening • Drug holidays or cyclic dosing can lead to MG-ADL score rebound
Slide 34: IgG Degrader MG Clinical Trial Design
Biohaven IgG Degrader: Clinical Trial Design Highlights Potential Efficacy in Acute and Maintenance MG Therapy QMG (within 2 weeks) DESIGN Randomized, double-blind, placebo-controlled trial POPULATION Male and female adults with gMG TREATMENT DURATION 24-week treatment period KEY ENDPOINTS QMG (with
Slide 35: Targeting Autoimmunity in PPCM
Targeting the Role of Autoimmunity in PPCM Current treatment for PPCM • Guideline-Directed Medical Therapy: No disease specific therapy Treatment goals in PPCM • Intervene early • Treat acute heart failure • Prevent further myocardial injury and restore baseline normal cardiac function β1AR AAbs pre
Slide 36: BHV-1600 Targets β1AR Autoantibodies in PPCM
Create Value PHASE 1 SAD/MAD completed dosing: IV and SC dosing up to 500mg IV 2026 STUDY in PPCM patients PPCM: BHV-1600 Targets β1AR Autoantibodies for First Potential Disease-Specific Therapy Follow the Science Understand the Need ✓ PHASE 1 SAD/MAD Completed dosing, 1AR PD biomarker results pend
Slide 37: BHV-1600 Phase 2/3 Pivotal Study Design
12-Week Treatment Phase 52-Week Treatment Phase (SOC +) BHV-1600 (SOC +) Placebo Screening BHV-1600: Phase 2/3 Pivotal Study Design Based Upon FDA INTERACT 37 Pivotal Trial Surrogate Endpoint for Accelerated Approval DATA READOUT DATA READOUT Confirmatory Endpoint (Up to 52 weeks) DEGRADERS Pivotal
Slide 38: Next-Gen Degraders
biohaven’ Next-Gen Degraders
Slide 39: Biohaven Discovery Provides Pipeline Sustainability
preDC DC* IgG4: BHV-1450 IgG-Driven Disease: BHV-1320 Anti-PLA2r: Nephropathy Anti-Proinsulin: Type I Diabetes Anti-TSHR: Graves’ Disease Multiple Early Targets Technology Build Biohaven Discovery Provides Pipeline Sustainability and Optionality May 28, 2025 Biohaven R&D Day39 DEGRADER PORTFOLIO MoD
Slide 40: Expanding the Degrader Platform Into Focused Research Areas
Expanding the Degrader Platform Into Focused Research Areas • Neuro and Neuromuscular: MG, MuSK, CIDP, LRP4, Guillain Barré, MS, MOGAD, NMO* • Renal: IgAN, PLA2r, IgG4, GBM, nephrin-1, cortico-interstitial fibrosis, ADAs* • Hematology: Gammopathies, TTP, wAIHA, CAG, IgA1 and ANCA-associated vasculit
Slide 41: BHV-1450: Deep Removal of IgG4
BHV-1450: Deep Removal of IgG4 for IgG4-Mediated Diseases May 28, 2025 Biohaven R&D Day41 IgG4-related diseases result in target organ inflammation and fibrosis • Pancreatitis • Sclerosing cholangitis • Retroperitoneal fibrosis • Tubulointerstitial nephritis • Mikulicz’s disease and sclerosing sialo
Slide 42: Removal of Proinsulin Autoantibodies in Type 1 Diabetes
Removal of Proinsulin Autoantibodies Halts Progression of Nascent Type 1 Diabetes (T1D) June 2025 Biohaven Investor Presentation42 Islet of Langerhans PANCREAS β-cell depletion results in T1D and hypoinsulinemia ADCC-mediates killing of insulin producing β-cells UNTREATED viral infection idiopathic
Slide 43: Targeting Anti-PLA2r Antibodies in Membranous Nephropathy
Selective Targeting of Anti-Phospholipase A2 Receptor (PLA2r) Antibodies for Idiopathic Membranous Nephropathy (IMN) HEALTHY KIDNEY Currently no specific therapies to treat IMN2 • Rituximab or cyclophosphamide + glucocorticoids are first-line therapies but have serious side effects • Combination of
Slide 44: Removal of Neutralizing Antibodies to Capsids
Removal of Neutralizing Antibodies to Capsids to Optimize Gene Therapy Uptake and Allow Repeat Administration Discovery of Dominant Epitopes for Anti-AAV Antibodies 1 10 100 1000 0 100000 200000 300000 400000 500000 BH3746 nM 6 4 7 a re a ( u m 2 /i m a g e ) Internalization anti-AAV9 antibody IgG1
Slide 45: BHV-7000 Kv7.2/7.3 Activator
BHV-7000 Kv7.2/7.3 Activator Potassium (K+) Ion Channel Ion Channel Platform
Slide 46: BHV-7000: Kv7 Activator for Epilepsy and Depression
BHV-7000: Kv7 Activator for Epilepsy and Depression
Slide 47: Kv7 Is a Transformational Target
June 2025 Biohaven Investor Presentation47 Kv7 Is a Transformational Target in Neurology and Neuropsychiatry • Selective Kv7 activation avoids unwanted CNS side effects • Clinically validated in epilepsy, depression and pain BHV-7000 Is Potential Best-in-Class Selective Kv7 Activator with Blockbuste
Slide 48: BHV-7000 Nears Completion of Pivotal Trials
BHV-7000: Potential Best-in-Clinic Selective Kv7 Activator Nears Completion of Pivotal Trials with Blockbuster Potential Epilepsy 3.5M Patients • Clinically validated MOA for epilepsy • Global Phase 2/3 program ongoing in focal epilepsy (2 trials) and idiopathic generalized epilepsy (1 trial) Major
Slide 49: BHV-7000 Med-Chem Designed to Leverage Kv7
BHV-7000 Med-Chem Designed to Fully Leverage Kv7 Target Potential Key areas of differentiation to discover and develop best-in-class Kv7 activator 49 Pharmacophore-based design principles Screening and Tier I ADME Advanced bridging in vitro and in vivo assays Clinical Novel scaffolds Functional prim
Slide 50: BHV-7000 Equipotent With Azetukalner Preclinically
BHV-7000 potency consistently ~ 120 nM in complex neuronal system assaysKEY POINT BHV-7000 Equipotent With Azetukalner in Preclinical MES Assay Efficacy of BHV-7000, Azetukalner and Ezogabine in the Rat AC-MES Assay 0.001 0.01 0.1 1 10 100 0 25 50 75 100 Maximal Electroshock Model (rat) Brain concen
Slide 51: Superior Tolerability vs Ezogabine and Azetukalner
No effect of BHV-7000 on rotarod fall latency at 60x the ED50 — TI not calculable KEY POINT BHV-7000 Differentiated Preclinically From Ezogabine and Azetukalner With Superior Tolerability 51 BHV-7000 Efficacy/Tolerability Index Rotarod MES m g /k g 1 0 0 8 0 6 0 5 0 4 0 2 7 2 0 4 0 Tested 1h Post Do
Slide 52: Single Doses of BHV-7000 Well-Tolerated
Single Doses of BHV-7000 IR Achieve Multiples Above MES EC50 and Are Well-Tolerated in Healthy Volunteers Dose (mg) Cmax (ng/mL) MES EC50 3 (ng/mL) Cmax: MES EC50 Nervous System AEs, n (%) Somnolence Dizziness Headache 25 45.8 (14.3) 81 0.57x 2 (33.3) 3 (50.0) 0 Azetukalner2,a Dose (mg) Cmax (ng/mL)
Slide 53: Multiple Doses of BHV-7000 ER Well-Tolerated
Multiple Doses of BHV-7000 ER Exceed MES EC50 Over Entire Dosing Interval and Are Well-Tolerated in Healthy Volunteers QD x 7 days (mg) Cmax (ng/mL) Ctau (ng/mL) MES EC50 (ng/mL) Cmax: MES EC50 Ctau: MES EC50 Nervous System AEs1 Somnolence Dizziness Headache 50 406 (21.8) 265 (26.0) 186 2.18x 1.42x
Slide 54: BHV-7000 CNS Activity Demonstrated in EEG Study
Spectrum at Broadband Max Response BHV-7000 50 mg vs Baseline1 Spectrum at Broadband Max Response BHV-7000 25 mg vs Baseline1 Dose-Response BHV-7000 CNS Activity Demonstrated in EEG Study Single Dose (mg) Cmax (ng/mL) Nervous System AEs Somnolence Dizziness Headache 25 309 (35.7) 0 0 0 50 537 (36.4)
Slide 55: Excellent CNS Tolerability in Focal Epilepsy Studies
Excellent CNS Tolerability Observed in Ongoing Phase 2/3 Focal Epilepsy Studies (Blinded Data) Consistent With Phase 1 Profile BHV-7000 well-tolerated in focal epilepsy at exposures predicted to be effective based on preclinical data and associated with CNS activity on EEG in healthy volunteers KEY
Slide 56: Two Phase 2/3 Studies in Focal Epilepsy
BHV-7000: Two Phase 2/3 Studies in Focal Epilepsy Ongoing DESIGN Randomized, double-blind, placebo-controlled trials POPULATION Subjects 18-75 years of age with refractory focal epilepsy SAMPLE SIZE 390 subjects in each study (randomized 1:1:1) KEY ENTRY CRITERIA Average of ≥4 observable focal seizu
Slide 57: Phase 2/3 Study in Idiopathic Generalized Epilepsy
OLEScreening BHV-7000 75 mg Placebo BHV-7000: Phase 2/3 Study in Idiopathic Generalized Epilepsy Ongoing DESIGN Randomized, double-blind, placebo-controlled trial POPULATION Subjects 18-75 years old with idiopathic generalized epilepsy with intractable generalized tonic-clonic seizures SAMPLE SIZE 2
Slide 58: Patient Case Report From OLE
4.8 0.8 0 1 2 3 4 5 6 Observation Open Label BHV-7000: Patient Case Report From OLE Sample Patient Case Report • Subject: 71-year-old male • Type of seizure: focal impaired awareness • Seizure description: tingling and twitching of cheek spreading to neck and arm followed by arm rigidity and impaire
Slide 59: Clinical Evidence Supports Kv7 Activation in MDD
Azetukalner RCT3 Rapid-onset, dose-related reduction of depressive and anhedonic symptoms vs pbo: MADRS (-3, p=.135) and SHAPS (-2.5, p<.05) in 20 mg group Efficacy not optimized likely due to dose limiting tolerability concerns: CNS AEs (43%) including dizziness (18%) in 20 mg group Azetukalner Mt
Slide 60: Phase 2/3 Study in Major Depressive Disorder
BHV-7000: Phase 2 Study in Major Depressive Disorder Ongoing DESIGN Randomized, double-blind, placebo-controlled trial POPULATION Subjects with Major Depressive Disorder (MDD) with at least one prior depressive episode who are currently experiencing a depressive episode with anhedonia (HAM-D ≥ 20, S
Slide 61: BHV-7000 Potential to Overcome Limitations of Existing Therapies
BHV-7000 Has Potential to Overcome Limitations of Existing Therapies SSRIs/SNRIs TYPICAL TREATMENT PATTERN FOR MDD Atypical antidepressants Augmentation strategies ✓ Novel mechanism ✓ Favorable tolerability ✓ Addresses anhedonia ✓ Rapid onset of effect ✓ Ease of use and administration BHV-7000 61 Ju
Slide 62: BHV-2100: TRPM3 Antagonist for Pain
BHV-2100: TRPM3 Antagonist for Pain
Slide 63: First-in-Clinic TRPM3 Antagonist for Pain
June 2025 Biohaven Investor Presentation63 First-in-Clinic TRPM3 Antagonist — Novel Mechanism for the Treatment of Pain • BHV-2100 is an orally administered, peripherally-restricted and selective TRPM3 antagonist • Preclinical data shows robust efficacy in pain models • Highly differentiated from pr
Slide 64: Targeting the Unmet Medical Need in Pain
BHV-2100: Targeting the Unmet Medical Need in Pain ACUTE PAIN & NEUROPATHIC PAIN Emerging role of novel mechanisms: ion channels in the periphery BHV-2100 is a selective, peripherally-restricted TRPM3 antagonist that is a potentially highly-effective, non-sedating, non-opioid treatment for pain KEY
Slide 65: BHV-2100 Reduces Acute and Nerve Injury Pain
BHV-2100: Potently Reduces Acute Chemogenic Pain and Pain Following Nerve Injury Encouraging evidence of pain reduction without the sedation observed with high-dose pregabalin Drug administered 30 minutes prior to TRPM3 agonist injection in a hind paw of rats Drug administered 14 days after unilater
Slide 66: Reverses Established Pain in Neuropathic Models
BHV-2100: Reverses Established Pain States in Peripheral Neuropathic Pain Models Drug administered 6 days after oxaliplatin treatment in mice Chemotherapy-Induced Neuropathic Pain Model Diabetic Neuropathy Model Drug administered 7 days after STZ treatment in rats 3 4 5 6 7 8 9 10 11 * ** *** 2 h1 h
Slide 67: Ideal Pharmacokinetic Profile for Treating Pain
BHV-2100: Ideal Pharmacokinetic Profile for Treating Pain EC90 represents the estimated plasma concentration threshold based on a preclinical model Phase 1 data in healthy subjects shows plasma PK exceeds EC90 by 20 min at all doses tested and is sustained above EC50 for 24 hours at doses > 25 mg QD
Slide 68: Safe and Well-Tolerated in Healthy Subjects
BHV-2100: Safe and Well-Tolerated in Healthy Subjects SAFETY AND TOLERABILITY • No dose limiting toxicities in studies • No SAEs • No severe TEAEs; most TEAEs were mild • No clinically significant trends in vital signs (including body temperature), laboratory values, or ECGs DOSING • SAD: single dos
Slide 69: Proof of Concept Pain Study
BHV-2100: Proof of Concept Pain Study Demonstrates Anti‐Nociceptive and Anti‐Hyperalgesic Effects Efficacy • Lowering in self-reported VAS pain rating scale • Clinically meaningful reductions in laser-evoked potentials in normal and UVB-inflamed skin Safety • Well-tolerated • No effects observed on
Slide 70: TRPM3 Platform Expanding Range of Diseases
BHV-2100 TRPM3 Platform Offers Potential to Treat an Expanding Range of Diseases • Chronic Neuropathic Pain • Nociceptive Pain (Osteoarthritis) • Mixed Neuropathic and Nociceptive Pain (Back Pain) • Acute Pain ✓Mechanistic Data ✓Human Genetic Validation ✓ Safety and Tolerability ✓Human Proof of Conc
Slide 71: BHV-8000: Brain-Penetrant TYK2/JAK1 Inhibitor for Parkinson's
BHV-8000: Brain-Penetrant TYK2/JAK1 Inhibitor for Parkinson’s Disease
Slide 72: First-in-Clinic Brain-Penetrant TYK2/JAK1 Inhibitor
June 2025 Biohaven Investor Presentation72 First-in-Clinic, Brain-Penetrant, Selective TYK2/JAK1 Inhibitor for the Treatment of Neuroinflammatory and Neurodegenerative Diseases • TYK2 and JAK1 inhibition target key inflammatory signaling pathways • Selectivity profile avoids safety liabilities of JA
Slide 73: TYK2/JAK1 Inhibitor for Neuroinflammatory Disorders
BHV-8000: Brain-Penetrant TYK2/JAK1 Inhibitor With Potential to Treat Neuroinflammatory & Neurodegenerative Disorders Cytokine Receptor JAK FAMILY Microglia IFN-γ, IFN-⍺, IFN-β Astrocytes IFN-γ, IFN-⍺ and IFN-β Lymphocytes and Other Leukocytes IL-23, IL-17 downstream of IL-23 CELLULAR DRIVERS IN NEU
Slide 74: Approved JAK Inhibitors Safety Risks
Approved JAK Inhibitors — Significant Safety Risks Associated with JAK2 and/or JAK3 Inhibition Inhibitor Status IC50 in nM Safety JAK1 (autoimmune) JAK2 (hematology) JAK3 (leucocyte) TYK2 (inflammation) Tofacitinib1 Approved 15 77 55 489 Boxed Warning (MACE, malignancy, thrombosis, serious infection
Slide 75: BHV-8000 Phase 1 Profile
BHV-8000: Demonstrates a Promising Phase 1 Profile Brain exposure sustained above target EC50s for 24 hours at clinically relevant dosesKEY POINT BHV-8000 Brain Exposures at 20 mg QD EC50 Modelling data Phase 1 program completed • Includes SAD/MAD study in healthy adults Well-tolerated • No SAEs or
Slide 76: Evidence of Pharmacodynamic Effects
BHV-8000: Shows Evidence of Pharmacodynamic Effects Preliminary Phase 1 data. hs-CRP, high-sensitivity C-reactive protein; IFN-beta, Interferon beta. * p<0.05 IFN-betahs-CRP Day 14 -60 -40 -20 0 20 40 Study Day M e a n % C h a n g e IFNb Placebo 20mg Day 14 -100 -50 0 50 Study Day M e a n % C h a n
Slide 77: BHV-8000 Demonstrates CNS Exposure
Time after dosing 6, CSF 6, Plasma 24, CSF 24, Plasma BHV-8000: Demonstrates CNS Exposure CSF, Cerebro Spinal Fluid Expected to have sustained brain exposures above EC50 (target engagement)KEY POINT CSF and Plasma Concentrations (20 mg QD) Brain PK sustained above EC50 for 24 hours at 20 mg QD B H V
Slide 78: Parkinson's Disease Is an Autoimmune Disorder
Parkinson’s Disease Is an Autoimmune Disorder 1. Sulzer, Nature 2017; 2. Wissemann, Am J Hum Genet 2013; 3. Li, Front Immunol 2023; 4. Tansey, Nat Rev Immunol 2022; 5. Potashman, Parkinsonism Relat Disord 2025; 6. Roodveldt, Brain 2024; 7. Karikari, Brain Behav Immun 2022; 8. Williams, Brain 2021; 9
Slide 79: Clinical Data Supports Targeting Neuroinflammation in Parkinson's
` BHV-8000: Clinical Data Supports Targeting Neuroinflammation in Parkinson’s Disease In Vivo Cytokine Levels3 Elevated levels of pro-inflammatory cytokines (e.g., IL-1β, IL-6, TNF-⍺, IFN-Ɣ) found in the CSF and blood of PD patients In Vivo Imaging2 18F-DPA-714 TSPO imaging increased in early PD rel
Slide 80: Real-World Analytics Show PD Risk Reduction
Real-World Analytics of Large Healthcare Database Show Parkinson's Disease Risk Reduction With TNF/IL-17 Targeting Therapies • Biohaven conducted analysis using Komodo Health database (over 320 million patients since 2012) examining treatment with anti-TNF or anti-IL17 and incidence of PD • Millions
Slide 81: TYK2/JAK1 Signaling Fuels Inflammation in Parkinson's
TYK2/JAK1 Signaling Fuels Inflammation Driving Disease Progression in Parkinson’s Disease Alpha-synuclein T cells IFN-γ IFN-⍺ IFN-β IFN-γ IFN-⍺ IFN-β Reactive Astrocytes Activated Microglia Cytokine Receptor JAK2 JAK1 TYK2 JAK3 S T A T S IG N A L IN G JAK2 JAK1 TYK2 JAK3 S T A T S IG N A L IN G VICI
Slide 82: BHV-8000 Targets Both TYK2 and JAK1
BHV-8000 Targets Both TYK2 and JAK1 to Control Immune Dysregulation in Parkinson’s Disease 82 1. Main, Glia. 2016; 2. Waters, bioRxIv. Accessed May 15, 2025. https://www.biorxiv.org/content/10.1101/2024.05.05.592614v1. 3. Chen, Front Aging Neurosci. 2020; 4. Hong, J Neuroinflammation. 2024 STAT ACTI
Slide 83: Efficacious in AAV-α-synuclein Mouse Model
BHV-8000: Efficacious in an AAV-α-synuclein Mouse Model of Parkinson’s BHV-8000 Shows Dose Proportional Increase in Total And Corner Movement in an Open Field BHV-8000 Shows an Increase in Grip Strength P<0.001 P<0.01 P<0.001 BHV-8000 Treatment Significantly Decreased IL-6 Level in Str and SN Tissue
Slide 84: Phase 2/3 Parkinson's Study Design
MDS-UPDRS, Movement Disorder Society – Unified Parkinson’s Disease Rating Scale; CGI-S, Clinician Global Impression of Severity scale; PARCOMS, Parkinson’s composite scale that includes most sensitive items from MDS-UPDRS and PDQ-39. 48-Week Time-to-event BHV-8000 20 mg PO Once Daily BHV-8000 10 mg
Slide 85: ARIA: Potential Target for TYK2/JAK1 Inhibition
BHV-8000: ARIA—A Potential Therapeutic Target for TYK2/JAK1 Inhibition • ARIA events typically occur early after initiation of anti-amyloid mAb therapy.1 The occurrence of ARIA can complicate the benefit-risk assessment in certain patient groups • Reduction or elimination of ARIA should improve the
Slide 86: Incidence of ARIA-E With Anti-Amyloid Therapy
• APOE4 carriers at increased risk for ARIA and accelerated progression of AD6,7 • Risk of ARIA can complicate the benefit-risk assessment of anti-amyloid mAbs, the only approved disease- modifying treatment for AD8 BHV-8000: Incidence of ARIA-E With Anti-Amyloid Therapy Anti-Amyloid mAb, (n) Overal
Slide 87: Phase 2/3 Prevention of ARIA Study Design
Anti-Amyloid mAb BHV-8000 (High Dose) + Anti-amyloid mAb BHV-8000 (Low Dose) + Anti-amyloid mAb Placebo + Anti-amyloid mAb BHV-8000: Phase 2/3 Prevention of ARIA Study Design DESIGN Randomized, double-blind, placebo-controlled trial POPULATION Male and female adults with early Alzheimer’s disease wh
Slide 88: Evidence of TYK2/JAK1 Signaling in AD
Transcriptional Profiling Study of AD Patient Brains With Drug Mechanism Reveals Strongest Association For JAK Family Kinases BHV-8000: Patient-Derived Evidence of TYK2/JAK1 Signaling in AD TYK2 Is The Most Prominent Blood Leukocyte Gene In AD Patients g e n e Nevado-Holgado et al, Cells. 2019 May 8
Slide 89: Phase 2/3 Disease-Modifying Study in Early AD
BHV-8000: Phase 2/3 Disease-Modifying Therapy Study in Early AD DESIGN Randomized, double-blind, placebo-controlled trial POPULATION Male and female adults with early symptomatic Alzheimer’s disease SAMPLE SIZE 2,000 participants (randomized 1:1:1 across 2 active and 1 placebo arm) TREATMENT DBT Pha
Slide 90: TYK2/JAK1 Inhibition for Multiple Sclerosis
BHV-8000: TYK2/JAK1 Inhibition Is a Potential Treatment for Multiple Sclerosis • Genetic evidence: Recent study found a protective genetic variation in the TYK2 gene that decreased signaling capacity in response to IL-12 and IL-23, reducing the function of TYK2, resulting in reduction in risk for de
Slide 91: Phase 2 Imaging POC Study in Relapsing MS
BHV-8000 (High Dose) BHV-8000 (Low Dose) Placebo Screening BHV-8000: Phase 2 Imaging POC Study in Relapsing Multiple Sclerosis Preliminary clinical trial design DESIGN Randomized, double-blind, placebo-controlled Phase 2 imaging proof-of-concept study POPULATION Adults with relapsing multiple sclero
Slide 92: Myostatin Platform
TALDEFGROBEP / MYOSTATIN COMPLEX ACTIVIN TYPE II RECEPTOR ACTIVIN TYPE I RECEPTOR SKELETAL MUSCLE CELL SURFACE Myostatin Platform
Slide 93: Taldefgrobep Alfa for Obesity
Taldefgrobep Alfa for Obesity
Slide 94: Differentiated Pharmacology Key in Obesity
June 2025 Biohaven Investor Presentation94 Differentiated Pharmacology Key to Optimizing Benefit in Overweight and Obesity • Taldefgrobep-myostatin complex competitively inhibits multiple key ligands from signaling through Activin II receptors (ActRII) • Unique MOA leads to direct beneficial effects
Slide 95: Obesity Remains a Critical Unmet Medical Need
Optimal Management of Obesity Remains a Critical Unmet Medical Need • By 2030, 1 billion people worldwide will be living with obesity, including 50% of American adults1 • Obesity is a disease of excess and/or abnormal adipose tissue, not excess mass • Incretin mimetics have revolutionized management
Slide 96: Novel MOA Optimizes Metabolic and Body Composition Change
Taldefgrobep Alfa: Novel MOA Optimizes Metabolic and Body Composition Change Important to those with Obesity 1. Illustration adapted from Severinsen et al. Endocr Rev. 2020 Aug 1;41(4):594–609. 2. Daghlas et al. BMJ Med. 2023;2(1):e000354. 3 Lee et al. Exp Biol Med. 2018;243:1275-85. 4. Chen et al.
Slide 97: Direct Effects on Muscle and Fat
Taldefgrobep Has Direct Effects on Muscle and Fat — Optimizing Quality of Weight Loss June 2025 Biohaven Investor Presentation97 BINDS active myostatin (GDF-8), GDF-11 FORMS COMPLEX that attaches to ActRIIA/B prevents ActRII binding (ALK4,5,7) INHIBITS ActRIIA/B signaling by key ligands including Ac
Slide 98: Differentiated Approach Balances Efficacy and Safety
Taldefgrobep’s Differentiated Approach Balances Efficacy and Safety June 2025 Biohaven Investor Presentation98 ALK4 ALK5 ActRIIA/B SKELETAL MYOCYTE SMAD2/3/4 SIGNALING ActRII signaling inhibits muscle growth and suppresses lipolysis ALK4 ALK5 ActRIIA/B ALK4 ALK5 ActRIIA/B M M G A A M M TARGETS pro-
Slide 99: Differentiated Profile Within Myostatin Class
Taldefgrobep: Highly Favorable and Differentiated Profile Within the “Myostatin Pharmacologic Class” Pure Myostatin Agent • Inhibits latent myostatin • No direct ActRIIB receptor effects, so activity limited to PK of drug (limited PK/PD) • Claims better safety due to selectivity • Likely associated
Slide 100: Improves Body Composition in Non-Obese Adults
Taldefgrobep Improves Body Composition in Non-Obese Adults with Potential for Monthly Dosing in Obesity June 2025 Biohaven Investor Presentation100 Muntoni F. et al, Neurol Ther. 2024 Feb;13(1):183-219. Taldefgrobep Continued to Decrease Total Fat Mass Beyond the Dosing Period Placebo 45 mg Q1W Last
Slide 101: ALK7 Inhibition Reduces Fat Mass
ALK7 Inhibition Can Meaningfully Reduce Fat Mass in a DIO Mouse Model1 Taldefgrobep Stimulates Lipolysis and Decreases Fat Mass June 2025 Biohaven Investor Presentation101 • ALK7 activation blunts β-adrenoreceptor-mediated lipolysis and lipid oxidation in people with obesity1 • Reduction in ALK7 sig
Slide 102: Greater Effect With Semaglutide in DIO Mice
Taldefgrobep: Shows Greater Effect in Combination With Semaglutide than Semaglutide Alone in DIO Mice T-alfa Shows Weight Loss, and Combination with Semaglutide Shows Higher Reduction in Body Weight Than Semaglutide Alone Weeks T-alfa and Combination Show Greater Reduction in Fat Mass Than Semagluti
Slide 103: Monotherapy and Combination With NuSH Therapies
Taldefgrobep Monotherapy Offers Attractive Differentiation; but Also Potential for Combination With NuSH Therapies to Improve Quality of Weight Loss June 2025 Biohaven Investor Presentation103 MITIGATE AGAINST ✓ Excess loss of lean mass ✓ Excess loss of bone mass ✓ Rapid weight rebound following int
Slide 104: Populations That Can Benefit From Combination
Populations That Can Benefit From Taldefgrobep + NuSH Combination June 2025 Biohaven Investor Presentation104 People living with overweight and obesity + comorbid T2DM Males living with overweight and obesity +/- T2DM Older individualsPeople living with extreme obesity — BMI ≥40 T-ALFA
Slide 105: Phase 2 Combination Therapy Study in Obesity
Design provides insight into key questions • Impact of taldefgrobep monotherapy on body composition, TBW, metabolic endpoints in representative population • Ability of taldefgrobep to provide additional benefits on top of NuSH therapies alone • Ability of taldefgrobep to mitigate against reductions
Slide 106: Taldefgrobep Alfa for SMA
Taldefgrobep Alfa for Spinal Muscular Atrophy (SMA)
Slide 107: Differentiated Pharmacology in SMA
June 2025 Biohaven Investor Presentation107 Differentiated Pharmacology Balancing Efficacy and Safety in SMA • Taldefgrobep-myostatin complex competitively inhibits multiple key ligands from signaling through Activin II receptors (ActRII) • Unique MOA leads to direct beneficial effects on both muscl
Slide 108: High Unmet Need Remains for SMA
High Unmet Need Still Remains For SMA 1. Mercuri E, et al. Nat Rev Dis Primers. 2022 Aug 4;8(1):52 . 2. Day JW, et al. BMC Pediatr. 2022;22(1):632. 3. Darras BT, et al. N Engl J Med. 2021;385(5):427-435. 4. Finkel RS, et al. N Engl J Med. 2017;377(18):1723-1732 5. Cure SMA. Accessed November 2024. h
Slide 109: 97% of Adults With SMA Hope for Muscle Strength
“What are your most significant current unmet needs that you hope new therapies would address?”1 97% of Adults With SMA Hope New Therapies Will Help Them Gain Muscle Strength 1. Cure SMA. Accessed November 2024. https://www.curesma.org/wp-content/uploads/2024/06/9042024_State-of-SMA_vWeb.pdf. 97% 90
Slide 110: Well-Characterized Clinical Profile
Taldefgrobep Alfa: Well-Characterized Clinical Profile CLINICAL DEVELOPMENT >700 trial participants treated (adults and children) across 6 studies • Completed: 3 Phase 1 trials (SAD/MAD, SC BA, autoinjector BE); 1 Phase 1b/2 trial in DMD and 1 Phase 2/3 trial in DMD • Ongoing: Phase 3 trial in SMA P
Slide 111: Robust Target Engagement
Taldefgrobep Alfa Achieved Robust Target Engagement One third of taldefgrobep alfa participants had undetectable myostatin at baseline (pre-treatment) Free Myostatin Mean ng/mL, (SD) Baseline Week 12 Week 48 Taldefgrobep alfa 0.233 (0.118) undetectable undetectable Placebo 0.259 (0.147) 0.267 (0.133
Slide 112: Treatment Effect on MFM-32 Additive to SOC
Taldefgrobep Alfa Treatment Effect on MFM-32 Is Additive to SOC MFM-32 TALDEFGROBEP ALFA + SOC Caucasian population with quantifiable myostatin 1.361 2.4 RISDIPLAM L e a s t s q u a re s m e a n c h a n g e ( 9 5 % C I) fr o m b a s e li n e i n M F M 3 2 t o ta l s c o re Risdiplam group Placebo gr
Slide 113: Treatment Difference in Caucasian Population Subgroups
Treatment Difference Favors Taldefgrobep Alfa in Caucasian Population: MFM-32 Subgroups and RULM at Week 48 Caucasian 2 3 4 Risdiplam Nusinersen Onasemnogene or Combination Prior Onasemnogene No Prior Onasemnogene Younger Older Ambulatory Non-ambulatory SOC Initiation <6yo SOC Initiation ≥6yo F A V
Slide 114: Treatment Effect Increases After 1 Year
Taldefgrobep Alfa Clinically Meaningful Treatment Effect Continues to Increase After 1 Year of Treatment in Overall Population MFM-32 Total Score Change from Baseline 48-Week Double-Blind Phase and Open-Label Extension 0 2 4 6 0 12 24 36 48 60 72 84 96 M F M -3 2 M e a n C h a n g e Weeks Taldefgrop
Slide 115: MFM-32 Trajectory Expected to Decline on SOC
MFM-32 Trajectory Expected to Decline on Stable SOC Treatment MFM-32 Total Score Change from Baseline Risdiplam SUNFISH Registrational Trial with OLE1 MFM-32 change at end of double- blind phase M e a n c h a n g e f ro m b a s e li n e i n M F M -3 2 t o ta l s c o re ( 9 5 % C I) MFM-32, 32-Item M
Slide 116: Safe and Well-Tolerated in Phase 3 SMA Study
Taldefgrobep Alfa Safe and Well-Tolerated in Phase 3 SMA Study: TEAEs Reported in ≥ 10% of Participants Overall During DB Phase • All SAEs judged not related to study drug • Low rate of TEAEs leading to discontinuation: 4 participants on taldefgrobep (2.8%); 1 on placebo (1.4%) KEY POINTS Placebo (n
Slide 117: Glutamate Modulation
PRESYNAPTIC NEURON POSTSYNAPTIC NEURON AMPA NMDA GLUTAMATE Glutamate
Slide 118: Significant Unmet Need in SCA
June 2025 Biohaven Investor Presentation118 Significant Unmet Need in SCA With No Available Treatment • Rare, relentlessly progressive, inherited neurodegenerative disease • ~15,000 patients in US US Priority Review of NDA With FDA Is Underway • Completed mid-cycle review and inspections of HQ and c
Slide 119: Troriluzole Rationally Designed to Optimize Therapy
Troriluzole Was Rationally Designed to Optimize Therapy ✓ Improved absorption ✓ Enhanced bioavailability ✓ Reduced drug burden ✓ Reduced first pass metabolism ✓ Favorable safety profile ✓ Once-daily dosing Stable in saliva, intestinal fluids and liver microsomes No negative food effect Actively abso
Slide 120: SCA: Rare Progressive Neurodegenerative Disorder
• Autosomal dominant, progressive, neurodegenerative disease with multiple genotypes1–3 • Onset in early adulthood with symptoms leading to severe disability and premature death3 • High unmet need and no approved therapies1,2 SCA: Rare Progressively Debilitating and Fatal Neurodegenerative Disorder
Slide 121: f-SARA Tracks SCA Disease Progression
f-SARA: Neurologist-Assessed Scale that Tracks SCA Disease Progression • Measures 4 core functional items that are clinically meaningful and reflect hallmark symptoms of SCA5 • Individual items rated 0–4 with total score range 0–16 • Generally increases (worsens) 0.5 points annually • Developed with
Slide 122: Troriluzole Delays Disease Progression Across SCA Genotypes
Troriluzole Restores Glutamate Homeostasis and Delays Disease Progression Across SCA Genotypes Clinically Meaningful Effect on f-SARA Strong Mechanistic Rationale Preclinical and clinical evidence for role of glutamate in SCA Reduction in Falls Falling is cardinal feature of SCAs leading to injury a
Slide 123: Studies 201, 206 and 206-RWE
‘201 data leads to new validated f-SARA scale to focus on functional changes over time; also provides long-term OLE and challenge/dechallenge data 1-year RCT insufficient duration to show efficacy in all genotypes Longer, multi-year RWE study is necessary to assess treatment effect in all genotypes
Slide 124: RWE Study Designed With FDA Input
RWE Study Was Designed With FDA Input & Following RWE Guidance 3-year real-world evidence protocol with external control using propensity score matching YEAR 3YEAR 1 YEAR 2 Troriluzole (n=101)Study 206 EXTERNAL CONTROL ARM - US US CRC-SCA (n=202) EXTERNAL CONTROL ARM - Europe EUROSCA (n=170) INDEPEN
Slide 125: RWE Study Designed With FDA Feedback
RWE Study Designed With FDA Feedback to Reduce Bias Biohaven Proposal FDA Feedback BHV4157-206-RWE Protocol Analyze new data from subjects completing 3-years of treatment Follow Industry Guidance for RWE* Regulatory precedent for NDA approval based on RWE and external control for analysis of 3-year
Slide 126: Demographic and Baseline Characteristics
Demographic and Baseline Characteristics BHV4157-206 CRC-SCA EUROSCA n 105 446 358 Age (years), n 105 434 358 mean (SD) 47.6 (13.1) 51.6 (13.8) 47.3 (12.7) median (range) 49.0 (18, 73) 52.0 (0, 89) 47 (18, 84) Sex, n 105 446 358 Male (%) 47 (45) 200 (45) 171 (48) Female (%) 58 (55) 246 (55) 187 (52)
Slide 127: Troriluzole vs US Natural History Control
Troriluzole vs Matched US Natural History External Control Shows Slowing of Disease Progression out to Year 3 CRC-SCA, Clinical Research Consortium for SCA; EUROSCA, European registry of SCA; f-SARA, Functional Scale for the Assessment and Rating of Ataxia; LSM, least squares mean; PSM, Propensity S
Slide 128: Troriluzole vs EU Natural History Control
Troriluzole vs Independent Matched EU Natural History External Control Shows Slowing of Disease Progression out to Year 3 0.0 0.5 1.0 1.5 2.0 2.5 0 1 2 3 f- S A R A ( ± S E ) Years * * * TRO EU 85 170 78 157 65 146 54 112 *p-values < .0001 Troriluzole EUROSCA 2.2 year delay in progression Troriluzol
Slide 129: Troriluzole vs Global Natural History Control
0.0 0.5 1.0 1.5 2.0 2.5 0 1 2 3 f- S A R A ( ± S E ) Years * * * TRO Global 101 303 90 272 75 172 61 129 *p-values < .01 Troriluzole Global (CRC-SCA & EUROSCA) 1.9 year delay in progression Troriluzole reduced SCA disease progression by 60%KEY POINT TRORILUZOLE June 2025 Biohaven Investor Presentati
Slide 130: Met Prespecified Hierarchical Endpoints
Troriluzole Met 9 Prespecified Consecutive Hierarchical Endpoints Demonstrating Robust and Durable Treatment Benefit Over 3 Years f-SARA at Year p-value US External Control (CRC-SCA) 3 <0.05 2 <0.05 1 <0.05 Europe External Control (EUROSCA) 3 <0.0001 2 <0.0001 1 <0.0001 Global External Control (CRC+
Slide 131: Untreated Patients Higher Likelihood of Worsening
Untreated SCA Patients Have Higher Likelihood of Significant Worsening *Prespecified Odds Ratio of f-SARA ≥2-Point Worsening in Untreated p-value US External Control vs Troriluzole* 2.4 0.0359 EU External Control vs Troriluzole 6.1 <0.0001 Global External Control vs Troriluzole 4.1 <0.0001 f-SARA ≥2
Slide 132: Anchoring Analysis Confirms Need for 3-Year Study
Anchoring Analysis Confirms Need for 3 Year Study to Overcome Heterogeneity in SCA Progression Patterns • External control was anchored to Study 206 placebo progression rate over 1-year using PSM • Anchored analysis shows efficacy at Years 2 and 3 consistent with primary results from Study 206-RWE •
Slide 133: New Data From 3-Year Completers Consistent
New Data From Subjects Completing 3 Years of Treatment Was Consistent With Primary Results in Each External Control Arm 0.0 0.5 1.0 1.5 2.0 2.5 0 1 2 3 f- S A R A ( ± S E ) Years Troriluzole CRC-SCA 0.0 0.5 1.0 1.5 2.0 2.5 0 1 2 3 f- S A R A ( ± S E ) Years Troriluzole EUROSCA 0.0 0.5 1.0 1.5 2.0 2.
Slide 134: Burden of Falls and Fall Risk Reduction
Burden of Falls in SCA9–10 • Most SCA patients (74–84%) report falling in the preceding 12 months • Falling is associated with a high rate of injury (74%) • Frequent fallers report more fall-related injuries • Fall frequency decreases when patients become wheelchair dependent or immobile Falls Risk
Slide 135: Troriluzole Well-Tolerated in Clinical Trials
Troriluzole Was Well-Tolerated in Clinical Trials 13 6.5 12 8.3 4.6 6.5 5.6 3.7 9.3 6.5 4.6 22 6.4 7.3 10.1 6.4 0.9 8.3 6.4 8.3 2.8 6.4 0 5 10 15 20 25 Fall Contusion Headache Dizziness UTI Nausea Muscle spasms Arthralgia Fatigue COVID-19 URTI Troriluzole Placebo Troriluzole N=108 Placebo N=109 Seri
Slide 136: Compelling Treatment Effect in SCA
Troriluzole Demonstrates Compelling Treatment Effect and Meaningful Delay in SCA Disease Progression • Efficacy data from Studies 201, 206 and RWE • 53% risk reduction in falls • 50–70% slowing of disease progression, representing 1.5–2.2 years delay in disease progression over 3-year RWE Study • Mu
Slide 137: SCA Significant Commercial Opportunity
SCA Represents a Significant Commercial Opportunity • 6,000 diagnosed US patients • No currently approved SCA treatments • Availability of genetic testing and advent of approved treatment will facilitate diagnosis • Engaged, connected SCA patient community • Strong patient advocacy support • KOLs, H
Slide 138: Market Dynamics Create Opportunities for VYGLXIA
Existing SCA Market Dynamics Creates Opportunities for VYGLXIA® and Biohaven • VYGLXIA® name conditionally accepted by the FDA pending product approval • VYGLXIA® would be the first and only approved treatment for SCA KEY POINT No approved therapies for SCA and no therapies in pivotal development S
Slide 139: Strategic Imperatives to Drive Successful Launch
Strategic Imperatives to Drive Successful Launch in SCA Identify patients, drive early diagnosis Establish VYGLXIA® as SOC in SCA Create access and reimbursement Ensure ongoing treatment 01 02 03 04 SCA Launch Priorities SCA launch relies on identifying patients and driving diagnosisKEY POINT VYGLXI
Slide 140: Patient Identification and Diagnosis Critical to Launch
Driving Patient Identification and Diagnosis Are Critical to Launch Success and Long-Term Growth FAMILY HISTOR Y 2. DIAGNOSIS 3 . S Y M PTOM PROGRESS IO N 1. SYMPTOM ONSET 1–10 YEARS before diagnosis is prompted Neurologist / ataxia expert & testing Symptom management/ alternate care Neurologist vis
Slide 141: Centralized Treatment Allows Targeted Commercialization
SCA Centralized Treatment Allows for Targeted and Efficient Commercialization Plan June 2025 Biohaven Investor Presentation141 Key centers in US by state US distribution of people with SCA 1. National Ataxia Foundation. Accessed May 12, 2025. https://www.ataxia.org/neurologists-and-specialty-clinics
Slide 142: VYGLXIA in SCA Commercial Opportunity
VYGLXIA® in SCA Represents a Significant Commercial Opportunity • HUB will be a central point of contact for the SCA community • Strive to ensure all SCA patients will have access to VYGLXIA® • High-volume ataxia experts and people with SCA interested or involved in clinical research programs/regist
Slide 143: Oncology: Next-Generation ADCs
Antibody Cancer Cell MATE Payload Targeted Cancer Cell Antigen Oncology: Next-Generation ADCs
Slide 144: Biohaven's Technologies Modernizing Next-Gen ADCs
Merus (undisclosed) STRATEGIC COLLABORATIONS Biohaven’s Innovative Technologies Modernizing Next-Gen ADCs June 2025 Biohaven Investor Presentation144 BHV-1510 (Trop2 TopoIx) Phase 1 mono and anti-PD-1 combo BHV-1500 (CD30 MMAE) IND planned 2H 2026 BHV-1530 (FGFR3 TopoIx) Phase 1 EXPANDING CLINICAL S
Slide 145: BHV-1510 Highly Differentiated Trop2 ADC
BHV-1510 Is a Highly Differentiated Trop2 ADC • Site-specific, highly stable conjugation-linker • TopoIx payload ideally positions for fast-to-market strategy with anti-PD-1 combo • Remains competitive as fast-follower Emerging Clinical Data Shows Predicted Profile and Potential of Proprietary TopoI
Slide 146: BHV-1510 Favorable PK and Safety Profile
BHV-1510: Favorable PK and Safety Profile With Very Low Free Payload • Mean serum exposure of BHV-1510 increased with increasing doses • Very low levels of serum free payload, demonstrating high ADC stability June 2025 Biohaven Investor Presentation146 Prelim data from ongoing study ADC and Payload,
Slide 147: Monotherapy Shows PRs in Patients Failing Standard Therapies
- Monotherapy Shows PRs in Patients Failing Standard Therapies June 2025 Biohaven Investor Presentation147 Case: 60 y/o female with endometrial cancer Prior therapies included dostarlimab, carboplatin and paclitaxel ONCOLOGY Case: 72 y/o with EGFRwt NSCLC Prior therapies include carboplatin and pacl
Slide 148: BHV-1510 Combination With Anti-PD-1
BHV-1510 Combination With Anti-PD-1
Slide 149: BHV-1510 + Anti-PD-1 vs DS-1062
SN-38 ** vs DXd ** vs SN-38 Concentration (µM) H M G 1 r e le a s e (p g /m l/ 1 0 ^ 4 c e ll s ) Concentration (µM) A T P r e le a s e (L u m in e s c e n c e ) SN-38 ** vs DXd ** vs SN-38 ** vs DXd ** vs SN-38 ** vs DXd BHV-1510+Anti-PD-1: Syngeneic Models Suggest Superior to DS-1062 June 2025 Bio
Slide 150: BHV-1510 + Cemiplimab Preliminary Activity
Change in Tumor Size BHV-1510 + Cemiplimab: Preliminary Activity Observed With Tumor Reduction Seen in First 6 Patients Treated • Encouraging preliminary efficacy with tumor reductions seen on first scan for all patients • Partial responses starting at lowest dose tested • Clinical activity demonstr
Slide 151: Partial Response in Patients Failing SOC Therapy
Partial Response in Patients Failing SOC Therapy Including Targeted Therapy and Immunotherapy June 2025 Biohaven Investor Presentation151 Red arrows: tumor metastasis Week 6 Baseline Week 6 Baseline Case: 53 y/o with urothelial cancer PR (33% reduction) at 6 weeks after 1510+cemiplimab combo • Prior
Slide 152: Tumor Reduction in Difficult to Treat Tumors
BHV-1510: Tumor Reduction Seen in Difficult to Treat Tumors, in Patients Progressing on Standard and Approved Therapies • Clinical activity seen across doses starting at the lowest dose • As monotherapy partial response seen in tumor types (SCLC, Endometrial, NSCLC) in patients that are heavily pret
Slide 153: BHV-1530
biohaven’ BHV-1530
Slide 154: Novel FGFR3 mAb With Proprietary TopoIx Payload
Novel FGFR3 mAb With Proprietary TopoIx Payload • Only FGFR3 directed ADC in clinic • Site-specific conjugation with favorable nonclinical tox profile FGFR3 Is a Validated Target With Limited Competition • No current FGFR3 ADCs approved or in advanced development Core opportunity in FGFR3-altered me
Slide 155: FGFR3 Promising Therapeutic Target in Several Tumors
FGFR3 Is a Promising Therapeutic Target in Several Tumors June 2025 Biohaven Investor Presentation155 ONCOLOGY 5% of endometrial cancers have FGFR3 alterations, potentially indicating more widespread overexpression 4% of cervical cancer cases exhibit the FGFR3- TACC3 fusion, which is associated with
Slide 156: BHV-1530 Potential in Metastatic Urothelial Cancer
BHV-1530: Potential to Address Unmet Need in Metastatic Urothelial Cancer (mUC) and other FGFR3-Driven Tumors June 2025 Biohaven Investor Presentation156 P ro b a b il it y o f S u rv iv a l (% ) Days after cell implantation BHV-1530 shows synergistic activity in vivo with anti-PD-L1 combination G1.
Slide 157: BHV-1530 Phase 1 Study in Advanced Tumors
BHV-1530: Phase 1 Study in Advanced Tumors June 2025 Biohaven Investor Presentation157 DESIGN Open label, dose escalation (Ph1) POPULATION Advanced tumors having failed SOC therapy SAMPLE SIZE 95 patients TREATMENT BHV-1530 TREATMENT DURATION Until disease progression or toxicity KEY ENDPOINTS Safet
Slide 158: Liquidity Update
Liquidity Update CASH SHARES OUTSTANDING POTENTIAL ROYALTIES Pfizer will make royalty payments in low- to mid-teens% in respect of annual US net sales of rimegepant and zavegepant >$5.25B, subject to annual cap ($400M/year)1 ~$518M3 102M2 CGRP June 2025 Biohaven Investor Presentation158 1. Cap reach
Slide 159: Pipeline Milestones 2025-2026
1H 2025 2H 2025 2026 GLUTAMATE Troriluzole | BHV 4157 Spinocerebellar Ataxia Obsessive-Compulsive Disorder MYOSTATIN Taldefgrobep Alfa | BHV-2000 Spinal Muscular Atrophy Obesity ION CHANNEL Kv7 Activator | BHV-7000 Focal Epilepsy Major Depressive Disorder TRPM3 Antagonist | BHV-2100 Pain Disorders I
Slide 160: Days
Days ee a : aS So ,,Piohaven’ ao a