Pharmaceutical Pipeline

Our lead candidate, INZ-701, is in clinical development for the potential treatment of ENPP1 Deficiency and ABCC6 Deficiency, which are driven by low levels of inorganic pyrophosphate (PPi) andadenosine. Currently, there are no therapeutic options for patients suffering from ENPP1 Deficiency and ABCC6 Deficiency –  two rare disorders. We are pioneering the development of effective therapies for these patients and their families.

INDICATION: Dilated Cardiomyopathy

RATIONALE:

Cardiac beta-1 adrenergic receptors (β1-AR), when activated, increase heart rate, contractility, and cardiac output. There is a high prevalence of agonistic autoantibodies that activate cardiac β1-AR in multiple cardiomyopathies, which lead to dilation of the heart and heart failure. The presence of β1-AR autoantibodies correlates with a poor prognosis, and removing or neutralizing β1-AR autoantibodies has shown potential therapeutic benefits. BHV-1600 is a bifunctional MoDE designed to selectively degrade pathogenic β1-AR autoantibodies and should augment the limited efficacy of beta-blockers, providing a novel approach to treating multiple cardiomyopathies.

Myasthenia Gravis

RATIONALE:

Molecular Degraders of Extracellular Proteins (“MoDE™”) are bispecific molecules that target pathologic circulating proteins and direct them to the liver (or other organ systems) for degradation by the endosomal/lysosomal pathway. Hepatic asialoglycoprotein receptor (ASGPR) ligand degraders able to recognize all potentially pathogenic isoforms of IgG represent a novel, competitive platform with a differentiated profile relative to FcRN inhibitors. Specifically, high circulating levels of antibodies (monoclonal or polyclonal gammopathy) drive conditions such as myasthenia gravis, rheumatoid arthritis, systemic lupus erythematosus, pemphigus vulgaris, and many other diseases. It is hypothesized that rapid and sustained lowering of pathogenic antibody titers in blood will significantly reduce disease symptoms. 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.

Molecular Degraders of Extracellular Proteins (“MoDE™”) are bispecific molecules that target pathologic circulating proteins and direct them to the liver (or other organ systems) for degradation by the endosomal/lysosomal pathway---a novel, competitive platform with a differentiated profile relative to FcRN inhibitors. Specifically, high circulating levels of antibodies (monoclonal or polyclonal gammopathy) drive conditions such as myasthenia gravis, rheumatoid arthritis, systemic lupus erythematosus, pemphigus vulgaris, and many other diseases. 

IgA nephropathy (“IgAN”) is the most common primary glomerulonephritis that can progress to renal failure and is characterized by immunoglobulin deposits in the renal mesangium comprised exclusively of the IgA1 subclass. Currently, no IgAN-specific therapies are available. Patients are managed with the aim of controlling blood pressure and maintaining renal function.

Cancer

B Cell Malignancies and AML

Cancer

Multiple Sclerosis

Cancer

Cancer

Ovarian Cancer

Mitochondrial Protonophores

Oncology

Oncology

Oncology

Covid

Infectious Disease / Oncology

Canine cancer

Cancer

SickleCell, CysticFibrosis

Peanut Allergy

Chikungunya

HSV,  HBV, HCV, HPV, RSV

Antibacterial

Lassa

RSV, EquineEncephalitis

Cancer