Pipeline

INDICATION:

Epilepsy affects approximately 3.5 million Americans and more than 50 million people worldwide; approximately 1/3 are refractory to available anti-seizure medications (ASMs). After starting an ASM, 80% of patients will experience burdensome adverse events, which can include somnolence, dizziness, cognitive dysfunction, and mood disturbances. Epilepsy can also be lethal; each year, more than one in 1,000 people with epilepsy die from SUDEP (sudden, unexpected death of someone with epilepsy).

RATIONALE:

Opakalim (BHV-7000) is a potent, selective activator of Kv7.2/7.3 potassium channels, a clinically validated target to regulate the hyperexcitable state in epilepsy. Opakalim (BHV-7000) is structurally and pharmacologically distinct from other potassium channel activators and demonstrates minimal GABA_A receptor activation, potentially providing better tolerability compared to other ASMs. Opakalim (BHV-7000) was potent in the maximal electroshock (MES) preclinical epilepsy model without adverse neurobehavioral or motor effects and was well-tolerated in a Phase 1 SAD/MAD clinical study without the central nervous system (CNS) adverse events typical of anti-seizure medications. A Phase 1 EEG biomarker study confirmed evidence of target engagement in the CNS.

INDICATION:

Pain Disorders

RATIONALE:

BHV-2100 is a potential first-in-class, orally administered, highly selective TRPM3 antagonist that is being developed for the treatment of pain disorders. TRPM3 is a novel druggable calcium-permeable ion channel in the transient receptor potential (TRP) channel family. TRPM3 is expressed in the relevant human tissue types for pain, and both preclinical models and human genetics implicate TRPM3 in pain. BHV-2100 offers a novel, non-addictive treatment approach to pain and has demonstrated potent pain reversal in preclinical models; favorable safety, tolerability, and pharmacokinetic properties in Phase 1 clinical studies; and early clinical proof of concept in pain.

INDICATION:

Obesity is a disease of excess and/or abnormal deposits of adipose tissue and a current global public health crisis. By 2030, it is expected that nearly one billion people will be living with obesity, including 50% of the adult and 25% of the adolescent US population. The primary driver of obesity-related morbidity and mortality is metabolically active visceral adipose tissues and associated deposits in and around organs such as the heart, liver, kidneys, and muscle.

RATIONALE:

Taldefgrobep is an investigational fusion protein with the potential to induce physical and metabolic changes that are highly relevant to individuals living with overweight and obesity. Taldefgrobep targets myostatin, a natural protein that limits skeletal muscle growth. Taldefgrobep’s binding of myostatin prevents activin receptor signaling, resulting in muscle hypertrophy. Current anti-obesity therapies achieve weight loss through the reduction in both fat mass and lean muscle mass. Taldefgrobep offers a potential novel approach to addressing the primary pathology of obesity. Through its unique mechanism of action, taldefgrobep may be able to meaningfully reduce total body and visceral adipose tissue volumes and improve insulin sensitivity, while increasing lean muscle mass.

INDICATION:

Rare Disease (Myasthenia Gravis)

RATIONALE:

Biohaven’s Molecular Degraders of Extracellular Proteins (“MoDE™”) Platform is the first clinically-validated technology enabling degradation of extracellular disease-causing proteins. Each MoDE is a bispecific molecule that targets pathogenic proteins and directs them to the liver (or other organ systems) for rapid and deep degradation by the endosomal/lysosomal pathway. High circulating levels of IgG antibodies (monoclonal or polyclonal gammopathy) drive conditions such as myasthenia gravis, CIDP, thyroid eye disease, Graves’ disease, rheumatoid arthritis, systemic lupus erythematosus, pemphigus vulgaris, and many other diseases. In preclinical studies, BHV-1310 demonstrates 90% IgG depletion with a single dose. Therapeutic pan-IgG depletion using Biohaven’s proprietary MoDE™ platform technology is expected to have significant potential benefit for multiple diseases.

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