Biohaven Ltd. · NYSE:BHVN
Biohaven — 41st Annual J.P. Morgan Healthcare Conference (January 2023)
28 slides from Biohaven's conference presentation, Jan 10, 2023
Slide 1: 41st Annual J.P. Morgan Healthcare Conference
41st Annual J.P. Morgan Healthcare Conference Vlad Coric, M.D., Chairman and Chief Executive Officer January 10, 2023
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 BHV-2100, troriluzole, BHV-5500, BHV-1100, B
Slide 3: Pfizer Acquisition of CGRP Platform $13B
Pfizer Acquisition of CGRP Platform $13B
Slide 4: Because we are passionate about the work we do everyday
Because we are passionate about the work we do everyday — FRANCINE Through state- of-the-art drug discovery and passionate scientists — LARRY With a powerful R&D engine — JAVIER We never forget patients need us. — JEREMY With a commitment to transforming patients’ lives — KATRINA
Slide 5: High Value Platforms, Innovative Portfolio, Proven Business Formula
HIGH VALUE PLATFORMS INNOVATIVE PORTFOLIO PROVEN BUSINESS FORMULA Pursuing novel paths of science to transform the treatment of neurological and neuropsychiatric diseases A broad therapeutic portfolio addressing patient needs with intention. Formula for continued growth built upon past success of ex
Slide 6: Drug or platform pipeline
January 10, 2023Biohaven | 41st Annual J.P. Morgan Healthcare Conference6 DRUG OR PLATFORM PRECLINICAL PHASE 1 PHASE 2 PHASE 3 MARKET ION CHANNEL Kv7 ACTIVATOR BHV-7000 BHV-7010 ION CHANNEL TRPM3 BHV-2100 GLUTAMATE Troriluzole MYOSTATIN CD38 Bispecific targeted cell therapy DISCOVERY RESEARCH IgG de
Slide 7: Key Programs
¥ TAIYE | Associate Scientist : / ew. Key Programs
Slide 8: Kv7 Ion Channels: Key Regulators of Neuronal Function
Kv7 Ion Channels: Key Regulators of Neuronal Function 5 FAMILY SUBTYPES Primary localizations: • Kv7.1: cardiac • Kv7.2: CNS BHV-7000/7010 activator • Kv7.3: CNS BHV-7000/7010 activator • Kv7.4: smooth muscle and inner ear • Kv7.5: vascular tissue, neurons, skeletal muscle Kv7 Stria Vascularis Organ
Slide 9: Kv7 Potassium Channels Regulate Neuronal Excitability and Loss of Function Causes Epilepsy
Kv7 Potassium Channels Regulate Neuronal Excitability and Loss of Function Causes Epilepsy January 10, 2023Biohaven | 41st Annual J.P. Morgan Healthcare Conference9 Impaired Kv7 Channel Activity Causes Certain Types of Epilepsy Kv7 Activation Normalizes Action Potential Firing Loss-of-function mutat
Slide 10: Kv7 Platform: Focal Epilepsy
January 10, 2023Biohaven | 41st Annual J.P. Morgan Healthcare Conference10 Kv7 PLATFORM Focal Epilepsy Potential Best-in-Class, Fast-Follower Approach Clinically validated mechanism of action Pursuing Differentiation Preclinical data suggests wide therapeutic index in the clinic by dialing out GABA
Slide 11: BHV-7000 selective for Kv7 over GABAA; third-generation differentiation
BHV-7000 is chemically stable to photo-oxidation BHV-7000 is selective for Kv7 over GABAA receptors a BHV-7000 has a wide therapeutic index preclinically a 1st and 2nd Gen Kv7 Activators Show Clinical Anti-seizure POC, But Off-target Activities, Opportunity for 3rd Gen Kv7 Differentiation XEN1101 •
Slide 12: BHV-7000 Exhibits Highly Differentiated Preclinical Profile
BHV-7000 Exhibits Highly Differentiated Preclinical Profile January 10, 2023Biohaven | 41st Annual J.P. Morgan Healthcare Conference12 Wide Therapeutic Index Kv7.2/7.3 Activator GABAA Activity “dialed-out” Ezogabine XEN1101 BHV-7000 Activator, clinical and preclinical anti-seizure activity Activator
Slide 13: BHV-7000 Phase I SAD/MAD Study Update: Dosing Completed
BHV-7000 Phase I SAD/MAD Study Update: Dosing Completed Preliminary Safety • Single doses up to 100 mg and multiple doses up to 40 mg daily x15 days generally well-tolerated • Most AEs mild and resolved spontaneously • No serious or severe AEs • No dose limiting toxicities Preliminary Pharmacokineti
Slide 14: Kv7 Role in Bipolar Disorder and Depression
Kv7 In mania models, Kv7 activation reduces hypermetabolism and hyperactive behavior1 Robust MDD efficacy likely to translate to bipolar disorder2 fMRI study: ezogabine effects mediated by the limbic system Preclinical and Clinical Data Suggest a Role for Kv7 in Bipolar Disorder and Depression Janua
Slide 15: Blockbuster Peak Sales Potential for Kv7 Platform
Epilepsy Blockbuster Peak Sales Potential for Kv7 Platform January 10, 2023 Mood Disorders Biohaven | 41st Annual J.P. Morgan Healthcare Conference15 ✓ Easy to Prescribe Convenient dosing, no titration, no food effect ✓ Safe/Tolerable Side effects negatively impacted other recent epilepsy launches ✓
Slide 16: TRPM3: A Novel Peripheral Target for Neuropathic Pain
TRPM3: A Novel Peripheral Target for Neuropathic Pain January 10, 2023Biohaven | 41st Annual J.P. Morgan Healthcare Conference16 Differentiated from existing programs targeting TRPV1 and TRPA1 • Current SoC comes with liabilities — addiction (opioids), sedation • Pre-clinical data shows reversal of
Slide 17: BHV-2100 Reduces Pain in Preclinical Models
BHV-2100 Reduces Pain in Preclinical Models of Chemotherapy and Diabetic Neuropathy Encouraging evidence of pain reduction without the sedation observed with high dose tramadol/gabapentin January 10, 2023Biohaven | 41st Annual J.P. Morgan Healthcare Conference17 BHV-2100 5 mg/kg BHV-2100 25 mg/kg Tr
Slide 18: Troriluzole: SCA and OCD
January 10, 2023Biohaven | 41st Annual J.P. Morgan Healthcare Conference18 TRORILUZOLE SCA and OCD SPINOCEREBELLAR ATAXIA (SCA) • Reduction in fall risk + treatment effect in SCA3 genotype • Safe and well-tolerated profile • Regulatory engagement planned for H1 2023 OBSESSIVE COMPULSIVE DISORDER (OC
Slide 19: Taldefgrobep Alfa: SMA Pivotal Phase Study
January 10, 2023Biohaven | 41st Annual J.P. Morgan Healthcare Conference19 TALDEFGROBEP ALFA SMA Pivotal Phase 3 Study Initiated July 2022 • Large pre-clinical and clinical safety package licensed from BMS • Limited additional work needed to support BLA submission • 34 sites total, 13 open POC for M
Slide 20: Bispecific Platform: IgG Degradation via MoDE
• Harnesses the body’s own machinery for degrading proteins • Extracellular protein targets are eliminated via the asialoglycoprotein receptor (ASGPR) • Protein targets are degraded via endolysosomal proteolysis * formerly BH 2640 Bispecific Platform: IgG Degradation via MoDE™ January 10, 2023Biohav
Slide 21: Pan-IgG Degrader (BHV-1300) Shows Superiority Over SoC
Pan-IgG Degrader (BHV-1300) Shows Superiority Over SoC (Efgartigimod) DISCOVERY RESEARCH Efgartigimod: Cyno PD 0 5 10 15 0 25 50 75 100 125 Days % Ig G , ba se lin e vehicle 10 mg/kg 75 mg/kg 250 mg/kg BHV-1300: Cyno PD 3 days 75% depletion 5–7 days 50% depletion January 10, 2023Biohaven | 41st Annu
Slide 22: Bispecific Platform: IgA Degrader for IgA Nephropathy
DISCOVERY RESEARCH Bispecific Platform: IgA Degrader for IgA Nephropathy Therapeutic Hypothesis: Galactosyl deficient-IgA (Gd-IgA) containing-immune complexes drive glomerular injury in IgA nephropathy.1 Specific removal of this pathogenic Gd-IgA with preservation of “normal” IgA (and other Ig’s) pe
Slide 23: Bispecific Platform: CD38 Targeted Cell Therapy for Multiple Myeloma
Bispecific Platform: CD38 Targeted Cell Therapy for Multiple Myeloma CD38 Antibody Recruiting Molecule (ARM™) • Modular bispecific molecules with two moieties, each designed for non-covalent binding to a specific target • Redirect endogenous antibodies to target cancerous or virally infected cells f
Slide 24: Ex Vivo Patient Plasma Study in Multiple Myeloma
Ex Vivo Patient Plasma Study: 4 Out of 5 Patients Showed Reduction in Plasma Cells (Multiple Myeloma) January 10, 2023Biohaven | 41st Annual J.P. Morgan Healthcare Conference24 ARM Using patient bone marrow and a combination therapy % reduction in plasma cells in patient bone marrow +/- autologous C
Slide 25: Bispecific Platform: Next-Generation Site-Specific ADCs
Second-generation ADC-linker technologiesBiohaven third-generation technology DISCOVERY RESEARCH Bispecific Platform: Advancing Next-Generation, Site-Specific Antibody Drug Conjugates (ADCs) Potential for best-in-class A single residue per heavy chain is available for conjugation • Controlled DAR* r
Slide 26: Capitalization Considerations
Capitalization Considerations CGRP royalties: Pfizer will make royalty payments in low- to mid-teens% in respect of annual U.S. net sales of rimegepant and zavegepant >$5.25B, subject to annual cap ($400M/year)3 ~$540 million2 SHARE O/S CASH POTENTIAL ROYALTIES 1. Excludes 9.1 million options ($7.00
Slide 27: Near-Term Milestones (Anticipated)
File IND Near-Term Milestones (Anticipated) January 10, 2023Biohaven | 41st Annual J.P. Morgan Healthcare Conference27 DRUG NAME INDICATIONS 2H 2022 1H 2023 2H 2023 BHV-7000 Kv7 channel activator Focal epilepsy Bipolar disorder BHV-7010 Kv7 channel activator Epilepsy and mood disorder BHV-2100 TRPM3
Slide 28: Pursuing novel paths of science
Pursuing novel paths of science A broad therapeutic portfolio Formula for continued growth HIGH VALUE PLATFORMS INNOVATIVE PORTFOLIO PROVEN BUSINESS FORMULA
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