Biohaven Ltd. · NYSE:BHVN
Biohaven — 43rd Annual J.P. Morgan Healthcare Conference (January 2025)
52 slides from Biohaven's conference presentation, Jan 13, 2025
Slide 1: 43rd Annual J.P. Morgan Healthcare Conference
© 2025 Biohaven, Ltd. All rights reserved. 43rd Annual J.P. Morgan Healthcare Conference January 13, 2025 Vlad Coric, M.D. Chairman and Chief Executive Officer JENNIFER Living with SCA3 Participant in the Troriluzole Clinical Study
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 clinical trials for our taldefgrobep alfa, troriluzole, BHV
Slide 3: Integrated discovery engine advancing therapies across top areas of innovation
Integrated DISCOVERY ENGINE ADVANCING CANCER TREATMENTS with strategic partnerships PIONEERING therapies for rare diseases including SMA & SCA Focused on Days Matter COMMERCIALIZATION OF NOVEL TXs POSITIONED FOR FUTURE VALUE CREATION DIVERSIFIED into top areas of INNOVATION INCREASED MARKET CAP 10X
Slide 4: Biohaven 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 5: Extracellular degraders: rapid and selective removal of disease-causing proteins
DEGRADATION TARGET BIFUNCTIONAL MoDE DEGRADER ASGPR RECEPTOR ON HEPATOCYTE EXTRACELLULAR DEGRADERS RAPID AND SELECTIVE REMOVAL OF DISEASE-CAUSING PROTEINS
Slide 6: MoDE Platform: Degraders Designed to Preserve Healthy Immune Functioning
January 2025 J.P. Morgan Healthcare Conference6 DEGRADERS MoDE Platform: Degraders Designed for Real-life and to Preserve Healthy Immune Functioning • Maximizes selectivity to treat disease while minimizing side effects • Short half-life enables concomitant administration with Fc-biologics • Allows
Slide 7: MoDE Degrader Platform Technology: Targeted Removal of Disease-Causing Proteins
TSHR AUTOANTIBODY DEGRADER Graves’ disease PLA2R AUTOANTIBODY DEGRADER Membranous nephropathy (MN) Other indications MoDE Degrader Platform β1AR AUTOANTIBODY DEGRADER Dilated cardiomyopathy (DCM) IgG4 DEGRADER • Membranous nephropathy (MN) • Pemphigus vulgaris • Autoimmune encephalitis (AE) • Muscle
Slide 8: Degrader Platform Technology
P A T IE N T C E N TRIC L IF E A LT E R ING 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 TUNABLE • Level of degradation carefully modulated by dose level and frequency • Employs b
Slide 9: IgA Nephropathy: BHV-1400
IgA NEPHROPATHY ✓ BHV-1400: Potential to treat by removing pathogenic species without chronic immunosuppression ✓ Robust science indicating disease is galactose-deficient IgA1-driven ✓ Biomarker endpoint with well-established accelerated approval pathway January 2025 J.P. Morgan Healthcare Conferenc
Slide 10: IgA Nephropathy Is Caused by Excess Gd-IgA1
IgA Nephropathy Is Caused by Excess Production of Galactose-Deficient IgA1 (Gd-IgA1) January 2025 J.P. Morgan Healthcare Conference10 Gd-IgA1 immune complexes deposit into the kidney, recruiting immune cells and causing the release of pro- inflammatory and profibrotic mediators No therapy selectivel
Slide 11: BHV-1400 Rapidly Removes Gd-IgA1 in Pre-Clinical Studies
BHV-1400 Rapidly Removes Galactose-Deficient IgA1 from Circulation and from the Renal Glomerular Mesangium in vivo in Pre-Clinical Studies January 2025 J.P. Morgan Healthcare Conference11 BHV-1400 Rapidly Clears Galactose-Deficient IgA1 1 HOUR 6 HOUR Gd-IgA1 + Vehicle Gd-IgA1 + BHV-1400 R E N A L C
Slide 12: Preliminary Phase 1: Selective and Deep Removal of Gd-IgA1 Within Hours
Preliminary Phase 1: Selective and Deep Removal of Gd-IgA1 Within Hours January 2025 J.P. Morgan Healthcare Conference12 BHV-1400 at the lowest SAD cohort rapidly and selectively removes 60% of Gd- IgA1 while preserving normal immunoglobulins (IgG, IgE, IgA, IgM)NEWS BREAKING -80 -60 -40 -20 0 Hours
Slide 13: BHV-1400 Selectively Degrades Only Gd-IgA1 Compared to Market Competitors
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 Gd-IgA1 without Immunosuppressio
Slide 14: Timeline of Earliest Reported Gd-IgA1 Lowering Across Competition
BHV-1400 Degrades Gd-IgA1 Rapidly: Timeline of Earliest Reported Gd-IgA1 Lowering Across Key Market Competition January 2025 J.P. Morgan Healthcare Conference14 Lowest dose of BHV-1400 tested shows deep reductions of Gd-IgA1 within hoursKEY POINT 1. Lowering numbers reported for the median from the
Slide 15: BHV-1400 Phase 2/3 Study Concept
9-month 104-Week Treatment Phase (SOC +) BHV-1400 (SOC +) Placebo Screening Harnessing Efficient Trial Design to Address a High Unmet Need BHV-1400 Phase 2/3 Study Concept January 2025 J.P. Morgan Healthcare Conference15 Pivotal Trial UPCR, Surrogate Endpoint for Accelerated Approval DATA READOUT DA
Slide 16: Peripartum Cardiomyopathy: BHV-1600
PERIPARTUM CARDIOMYOPATHY ✓ BHV-1600: Potential to treat through selective removal of pathogenic autoantibody without chronic immunosuppression ✓ High unmet need: rare disease affecting new mothers with no approved treatment ✓ Robust science highlighting ß1AR- autoantibodies as pathogenic ✓ Biomarke
Slide 17: BHV-1600, a Novel Investigational Treatment for Peripartum Cardiomyopathy
PERIPARTUM CARDIOMYOPATHY: • A rare disease with high unmet need • Maternal mortality highest since 1965 and primary contributor is PPCM with mortality rates reported up to 20% • 10% go on to require mechanical support (LVAD or heart transplant) • BHV-1600 degrades ß1AR autoantibodies to potentially
Slide 18: BHV-1600 selectively targets ß1AR autoantibodies with optimal safety profile
BHV-1600 selectively targets ß1AR autoantibodies to treat PPCM with Optimal Safety Profile KEY POINT Ongoing Phase 1 Preliminary Clinical Data • First-in-human dosing with BHV-1600 has been safe and well-tolerated to date with two cohorts dosed • All AEs have been mild, with no SAEs • Laboratory dat
Slide 19: BHV-1600 Trial Design and FDA INTERACT meeting
12-Week Treatment Phase 52-Week Treatment Phase (SOC +) BHV-1600 (SOC +) Placebo Screening Harnessing Efficient Trial Design to Address a High Unmet Need January 2025 J.P. Morgan Healthcare Conference19 Pivotal Trial Surrogate Endpoint for Accelerated Approval DATA READOUT DATA READOUT Confirmatory
Slide 20: Graves' Disease: BHV-1300
✓ BHV-1300: Potential to transform clinical paradigm to improve patient lives ✓ Robust science indicating disease is IgG1 antibody-mediated ✓ Easily measured biomarker endpoint ✓ Potential first or second to market with strong commercial opportunity GRAVES’ DISEASE January 2025 J.P. Morgan Healthcar
Slide 21: Seventy Years of Research Demonstrate Pathogenicity of TSH Receptor Autoantibodies
1970s Passive transfer of TSH receptor autoantibodies into healthies prove definitively to stimulate the thyroid Seventy Years of Research Demonstrate the Pathogenicity of TSH Receptor Autoantibodies in Graves’ Disease (GD) 1950s “Long-Acting Thyroid Stimulator,” (TSH receptor autoantibody) identifi
Slide 22: Biohaven IgG Degrader Platform for Graves' Disease
O V E R P R O D U C T I O N O F H O R M O N E S TSH-mimicking IgG1 antibody T 3 T 4 SYMPTOMS Biohaven IgG1,2,4 Degrader Platform: A Novel Therapeutic for the Treatment of Graves’ Disease Graves’ disease Insomnia Exophthalmos Muscle Weakness Tremor Oligomenorrhea Dyspnea Heat Intolerance Arrhythmia W
Slide 23: Redefining Possibilities in Graves' Disease Treatment
Redefining Possibilities in Graves' Disease Treatment: Treat the Mechanism of Disease, Spare Patients their Thyroid January 2025 J.P. Morgan Healthcare Conference23 “A drug that causes fatal agranulocytosis and liver failure is probably not one I want to take.” “Why lose my thyroid?” “Why trade HYPE
Slide 24: Broad Market Strategy to Modify Graves' Disease
Broad Market Strategy to Modify Graves’ Disease January 13, 2025 J.P. Morgan Healthcare Conference24 80M GLOBAL PREVALENCE3 IgG Degrader Opportunity Degraders redefine care, targeting the autoimmune pathogenesis of disease with the potential to treat across the course of disease KEY POINT 1. Forian
Slide 25: Phase 2 Refractory Graves' Disease Trial Design
Screening Phase 3 Graves’ Disease Mid-2025 with Biomarker Endpoint Phase 2 Refractory Graves’ Disease 12-Week Treatment Phase T 3 T 4 DATA READOUT Newly Diagnosed Graves’ Disease Refractory Graves’ Disease Biomarker endpoint Biomarker Diagnosis (ATD +) IgG Degrader (ATD +) Placebo Phase 2 Refractory
Slide 26: Biohaven's Goal Is to Change the Treatment Paradigm in Graves' Disease
Biohaven’s Goal Is to Change the Treatment Paradigm in Graves’ Disease January 2025 J.P. Morgan Healthcare Conference26 1790s DeSault performs first successful partial thyroidectomy 1940s Antithyroid drugs and radioactive iodine (RAI) used as alternative to surgery, chronic thyroid replacement 2020s
Slide 27: Market Potential of Biohaven's Degrader Platform
January 2025 J.P. Morgan Healthcare Conference27 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* > $15
Slide 28: Oncology: Next-Generation ADCs
Antibody Cancer cell MATE Payload Targeted cancer cell antigen Oncology: Next-Generation ADCs
Slide 29: Novel ADC Conjugation Technology and Strategic Collaborations
Novel mAbs • Validated and emerging targets • Merus collaboration leverages differentiated dual-targeting antibody platform Exclusivity to TopoIx payload • Superior preclinical anti-PD/L1 synergy and immunogenic cell death • GeneQuantum collaboration provides broad target exclusivity to the payload
Slide 30: BHV-1510 is a Highly Differentiated Trop2 ADC
BHV-1510 is a Highly Differentiated Trop2 ADC • Ideally positioned for fast-to-market strategy with anti-PD-1 combo Novel TopoIx Payload Synergy with Anti-PD-1 In Vivo • Induces immunogenic cell death and complete tumor regressions • Superior to datopotamab deruxtecan (DS-1062) plus anti-PD-1 Fully
Slide 31: BHV-1510 with Early Clinical Activity in Phase 1
BHV-1510 (Trop2 ADC with Topolx) with Early Clinical Activity in Phase 1 • Clinical activity across doses starting at the lowest dose (2 mg/kg, Q3W) • Tumor reduction observed in tumor types including ovarian, SCLC, NSCLC • Favorable preliminary safety and PK profile • No payload-associated ILD, dia
Slide 32: TopoIx Payload Is a Novel Topoisomerase Inhibitor
TopoIx Payload Is a Novel Topoisomerase 1 Inhibitor With a Superior Pre-clinical Profile Compared to DXd and SN-38 January 13, 2025 J.P. Morgan Healthcare Conference32 SN-38 DXd TopoIx In vitro cytotoxicity ++ ++ +++ ICD* + + ++ Transported by ABCG2 n/a Y N Bystander killing n/a ++ +++ In vivo effic
Slide 33: BHV-1530 Clinic-Ready FGFR3 ADC
Advancing TopoIx Payload in Next-Gen ADC to Target Urothelial Cancer and Other Solid Tumors • Novel and proprietary FGFR3 mAb • Enzymatic, site-specific conjugation • Favorable nonclinical tox profile Validated target with limited competition • No ADCs approved or in advanced development • Core oppo
Slide 34: BHV-1530: Potential to Address Unmet Need in Metastatic Urothelial Cancer
P ro b a b il it y o f S u rv iv a l (% ) Days after cell implantation BHV-1530: Potential to Address Unmet Need in Metastatic Urothelial Cancer (mUC) and other FGFR3-driven Tumors BHV-1530 shows synergistic activity in vivo with anti-PD-L1 combination G1. vehicle G2. anti-PD-L1 G3. BHV-1530 G4. BHV
Slide 35: Biohaven-Merus Collaboration for Bispecific ADCs
A leader in developing differentiated bispecific mAbs for oncology • Clinically validated platform • Lead program (Zenocutuzumab) granted US FDA accelerated approval in December 2024 Next-generation ADC conjugation and payload platform technologies Biohaven-Merus Collaboration Represents a Leading-E
Slide 36: BHV-7000 Kv7.2/7.3 Activator: Ion Channel Platforms
BHV-7000 Kv7.2/7.3 Activator Potassium (K+) Ion Channel Ion Channel Platforms
Slide 37: BHV-7000 Nears Completion of Pivotal Trials
BHV-7000, Potential Best-in-Clinic Selective Kv7 Activator, Nears Completion of Pivotal Trials with Blockbuster Potential January 13, 2025 J.P. Morgan Healthcare Conference37 Epilepsy 3.5M Patients • Clinically validated MOA for epilepsy • Global Phase 2/3 program ongoing in focal epilepsy (2 trials
Slide 38: BHV-2100: 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 39: Troriluzole — SCA
PRESYNAPTIC NEURON POSTSYNAPTIC NEURON GLUTAMATE TRORILUZOLE Troriluzole — SCA
Slide 40: Troriluzole Glutamate Modulator
January 13, 2025 J.P. Morgan Healthcare Conference40 TRORILUZOLE GLUTAMATE MODULATOR • Submitted NDA for treatment of all SCA genotypes (potential Priority Review) • Preparing for commercial launch in 2025NEWS BREAKING Troriluzole Is First Treatment to Slow SCA Disease Progression • Long-term RWE st
Slide 41: BHV-8000
biohaven’ BHV-8000
Slide 42: BHV-8000 TYK2/JAK1 Inhibitor (brain-penetrant)
January 13, 2025 J.P. Morgan Healthcare Conference42 BHV-8000 TYK2/JAK1 INHIBITOR (brain-penetrant) First-in-Clinic, Oral, Selective, Brain-Penetrant TYK2/JAK1 Inhibitor • Uniquely potent, TYK2/JAK1 selective, brain-penetrant inhibitor • Selectivity profile avoids class risks associated with JAK2/3
Slide 43: Real-World Analytics Show Parkinson's Disease Risk Reduction With TNF/IL-17 Targeting
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 44: BHV-8000 Demonstrates a Promising Phase 1 Profile
BHV-8000 Demonstrates a Promising Phase 1 Profile STUDY COMPLETED: 3 SAD cohorts and 3 MAD cohorts • SAD dose cohorts (10, 20 and 30 mg); MAD dose cohorts (6, 10 and 20 mg) • 8 healthy subjects per cohort (6 active: 2 placebo) SAFETY PROFILE: Safe and well-tolerated to date • No SAEs or severe AEs;
Slide 45: BHV-8000: Unique Phase 2/3 Study Design for Parkinson's Disease
Provides a highly-sensitive supportive secondary efficacy endpoint Novel Composite Endpoint Parkinson’s Disease Composite Score (PARCOMS) • Based on established methodology (i.e., Alzheimer’s Disease Composite Score [ADCOMS]) • Leverages PPMI and placebo-arm clinical trial data (C-Path) • Comprises
Slide 46: Myostatin — SMA and Obesity
TALDEFGROBEP / MYOSTATIN COMPLEX ACTIVIN TYPE II RECEPTOR ACTIVIN TYPE I RECEPTOR SKELETAL MUSCLE CELL SURFACE Myostatin — SMA and Obesity
Slide 47: Taldefgrobep Efficacy Results in SMA
Efficacy Results: Clinically Meaningful Improvements Enhanced In Myostatin-Positive Caucasian Participants ADDITIONAL SUPPORTIVE DATA • Responder Analysis* 50% of taldefgrobep-treated participants responded vs. 30% on placebo • Open-label Extension** Motor function continues to improve Taldefgrobep
Slide 48: Optimal Management of 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 49: Taldefgrobep Phase 2 Study in Obesity
Taldefgrobep Phase 2 Study in Obesity DESIGN Randomized, double-blind, placebo-controlled trial POPULATION Male and female adults living with overweight or obesity (BMI 27 - 40) without comorbid diabetes mellitus SAMPLE SIZE 80 participants randomized 1:1 (Sex [M/F] and BMI [<35, ≥35-40]) TREATMENT
Slide 50: Company Capitalization Updates
Company Capitalization Updates January 13, 2025 J.P. Morgan Healthcare Conference50 1. Cap reached if aggregate annual U.S. net sales of rimegepant and zavegepant amount to $8.15B. Royalty payments would be in respect of years ended on or before 12/31/40. 2. As of November 8, 2024; excludes outstand
Slide 51: Biohaven's Pipeline Working to Help Millions of Patients
PATIENTS 1 INDICATION Gd-IgA1 Degrader 140K IgA NEPHROPATHY RARE DISEASE RENAL CARDIOVASCULAR ONCOLOGY OBESITY NEUROLOGY IMMUNOLOGY & INFLAMMATION β1AR Degrader 200K DCM / PERIPARTUM CARDIOMYOPATHY Trop2 316K EPITHELIAL TUMORS CD30 95K HODGKIN LYMPHOMA Taldefgrobep Alfa 10K SPINAL MUSCULAR ATROPHY 1
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