Small Molecules for Neurology Indications

Biohaven, Ltd. is a biopharmaceutical company dedicated to the discovery and development of innovative treatments across various therapeutic areas, including neuroscience, immunology, and oncology. The company has established a strong reputation for scientific innovation, particularly through its proprietary drug development platforms. These platforms include Kv7 ion channel modulation, which targets epilepsy and mood disorders, and TRPM3 antagonism, aimed at treating migraine and neuropathic pain. Additionally, Biohaven is advancing its MoDE™ platform for addressing antibody-mediated diseases and the MATE™ platform for developing next-generation antibody-drug conjugates, showcasing its commitment to creating best-in-class therapies for conditions with significant unmet medical needs.

Biohaven's pipeline reflects its focus on addressing complex diseases through a combination of novel mechanisms and established scientific principles. For instance, the company is exploring myostatin inhibition for neuromuscular and metabolic diseases, including obesity, and utilizing TYK2/JAK1 inhibition for neuroinflammatory disorders. The company’s approach emphasizes collaboration and innovation, integrating internal research with licensed intellectual property from leading global institutions. This strategy not only enhances Biohaven's drug development capabilities but also aims to deliver transformative therapies that improve patient outcomes and create shareholder value.

United States

Biohaven Pharmaceutical Holding Company Ltd., a clinical-stage biopharmaceutical company, develops late-stage products candidates targeting neurological diseases and rare disorders in the United States. Its clinical stage products include Rimegepant, a product that has completed phase III trials for acute treatment and prevention of migraine; Vazegepant that has completed Phase 2/3 trial for acute treatment and prevention of migraine; and Troriluzole that has completed II/III randomization phase and is ongoing extension trial for ataxias. The company also offers Troriluzole that is in phase II/III clinical stage for obsessive compulsive disorders and Alzheimer diseases; BHV-0223, a product for amyotrophic lateral sclerosis; BHV-5000, a product that has completed phase I clinical trial for neuropsychiatric disorders; and Verdiperstat, a product that is in phase III trial for multiple system atrophy. Biohaven Pharmaceutical Holding Company Ltd. has a collaboration agreement with Cove to facilitate telemedicine evaluation for migraine sufferers. The company was founded in 2013 and is headquartered in New Haven, Connecticut.

Cancer

Central Nervous System (CNS)

Immune System Diseases

Inflammatory disorders

Kidney Diseases

Metabolic Diseases

Musculoskeletal Diseases

Pathological Conditions

Psychological Diseases

Biohaven, Ltd.

Spinocerebellar Ataxia

Our glutamate modulation platform is comprised of two product candidates: troriluzole (BHV-4157) and BHV-5000/5500. These work in the body to balance the glutamate system via two distinct mechanisms: glutamate transporter modulation (troriluzole) and N-methyl-D-aspartate, or NMDA, glutamate receptor antagonism (BHV-5000/5500). Troriluzole is currently in late-stage development for spinocerebellar ataxia (SCA) and obsessive compulsive disorder (OCD).

Troriluzole 

Obsessive compulsive disorder

Troriluzole (BHV-4157)

Spinal muscular atrophy

Taldefgrobep targets myostatin, a natural protein that limits skeletal muscle growth, through two mechanisms: lowering myostatin directly and blocking key downstream signaling mechanisms. Taldefgrobep is an investigational, muscle-targeted recombinant protein with the potential to enhance muscle mass and strength in people living with SMA when used in combination with other approved treatments.

Taldefgrobep alfa (BHV-2000)

Autoimmune disease

Graves’ disease

Rheumatoid arthritis

BHV-1300

IgA nephropathy

BHV-1400 is a novel IgA nephropathy (IgAN) investigational therapy designed to selectively degrade galactose deficient IgA1 (Gd-IgA1) without immunosuppression. BHV-1400 is highly differentiated, engineered to clear the pathogenic nidus of disease, Gd-IgA1, and preserve the individual's immunoglobulins (IgG, IgA, IgE, IgM), immune cells, and complement system.

BHV-1400

Bipolar disease

Epilepsy

BHV-7000

Major depressive disorder

BHV-7000 

Obesity

Taldefgrobep alfa (BHV-2000) 

Migraines

BHV-2100 [TRPM3 Antagonist]

Central nervous system illness

Parkinson's disease

TYK2/JAK1 Inhibitor

BHV-8000

Epithelial Tumors

BHV-1510 is a next-generation, fully optimized ADC that consists of a Trop-2 directed antibody conjugated to a proprietary best-in-class Topoisomerase 1 (TopoIx) payload at a homogeneous drug-antibody ratio (DAR) of 4. BHV-1510 incorporates a unique site-specific conjugation methodology and highly stable and irreversible linker chemistry designed by GeneQuantum Healthcare Co. (Suzhou) Ltd. Preclinically BHV-1510 has shown superior cellular cytotoxicity, bystander killing, and immunogenic cell death resulting in improved efficacy as monotherapy, and synergistic efficacy in combination with anti-PD-1 therapy. In IND-enabling studies, BHV-1510 also showed a broader therapeutic margin relative to more advanced Trop-2 ADCs, including a lack of lung toxicity, that may translate to an improved clinical efficacy and safety profile.

BHV-1510

Focal Onset Seizures

Cardiomyopathy

BHV-1600 is a MoDE engineered to rapidly degrade pathogenic autoantibodies to the b1-adrenergic receptor (b1AR). BHV-1600 is the first rationally designed investigative treatment in development for peripartum cardiomyopathy (PPCM), a rare type of heart failure that occurs during pregnancy or soon after birth that has no currently approved therapy. In b1AR autoantibody-driven cardiomyopathy, autoantibodies bind b1AR and induce cardiomyocyte toxicity and heart failure.

BHV-1600

IgG4-related disease

BHV-1310 is an optimized and selective IgG1, IgG2, and IgG4 degrader.

Biohaven, Ltd.

Company Details