FULFILLING OUR COMMITMENT TO PATIENTS

Biohaven has earned its reputation as a leader in scientific innovation. Our portfolio includes treatments for a range of diseases with unmet medical needs including focal epilepsy, obsessive compulsive disorder (OCD), spinocerebellar ataxia (SCA), spinal muscular atrophy (SMA) and more. We are proud of our pioneering science and a growing pipeline built by our own discoveries and collaborations with others. We continue to apply scientific breakthroughs to make a difference in the lives of people living with debilitating diseases around the world.

RESEARCH PIPELINE

Our broad and growing R&D pipeline includes an array of novel therapies for people suffering from neurological and neuropsychiatric diseases, rare disorders and other strategic therapeutic adjacencies. Our diverse portfolio includes numerous product candidates across all stages of development.

Kv7 ACTIVATOR PLATFORM

DRUG NAME

INDICATION

PRECLINICAL

PHASE 1

PHASE 2

PHASE 3

FILED

MORE INFO

Focal Epilepsy

BHV-7000

PC
P 1
P 2
P 3
FILE

INDICATION:

The lead indication for BHV-7000 is refractory focal epilepsy. Epilepsy affects approximately 3.5 million Americans and more than 50 million people worldwide. Sixty percent of patients with epilepsy have focal epilepsy, and of those, approximately one in three are refractory to available antiseizure medications. Epilepsy can also be lethal. SUDEP is the sudden, unexpected death of someone with epilepsy, who was otherwise healthy. Each year, more than one in 1,000 people with epilepsy die from SUDEP.

RATIONALE:

BHV-7000 is a potent activator of voltage-gated Kv7.2/7.3 potassium channels, key subunits involved in neuronal signaling and in regulating the hyperexcitable state in focal epilepsy. BHV-7000 is highly selective for Kv7.2/7.3 channels, avoiding activation of the GABAA receptor in vitro, thereby reducing the potential for off-target effects. BHV-7000 is structurally distinct from other Kv7 activators, demonstrates potent anti-seizure effects and is well-tolerated in preclinical seizure models, and has shown potential to be a best-in-class Kv7 activator.

KCNQ2-DEE

BHV-7000

PC
P 1
P 2
P 3
FILE

INDICATION:

KCNQ2 Developmental and Epileptic Encephalopathy (DEE) is a rare, serious, and debilitating disease that manifests in infants and children as early-onset seizures and encephalopathy due to a defect in the function of the Kv7.2 channel caused by pathogenic mutations in the KCNQ2 gene.  KCNQ2-DEE has a characteristic newborn presentation including hypotonia, treatment-resistant tonic seizures, a profoundly abnormal EEG, and most often with moderate-to-profound developmental impairment, with limited or no achievement of normal language, motor, or cognition milestones.

RATIONALE:

In addition to being a potent activator of normal Kv7.2/Kv7.3 channels, BHV-7000 has been shown to normalize the impaired channel function resulting from the KCNQ2 mutations that cause KCNQ2-DEE in neonates and infants. In in vitro studies of Kv7.2/7.3 channels poisoned by KCNQ2-DEE mutations, BHV-7000 was able to restore pathologically reduced channel currents to normal levels, providing evidence of the drug’s potential as a precision treatment directed at the root cause of the disease.

BHV-7000 has received rare pediatric disease designation from the U.S. Food and Drug Administration for the treatment of KCNQ2-DEE.

Mood Disorders

BHV-7000

PC
P 1
P 2
P 3
FILE

Approximately 1 in 5 adults in the U.S. are living with neuropsychiatric illnesses that are, in turn, associated with inadequate treatment, poor quality of life, disability, and considerable direct and indirect costs. There is significant unmet need for novel and effective therapeutic options that are not limited by long latency periods to clinical effects, low response rates, and significant risks and side effects. Increasing evidence from animal models and clinical trials now suggests that Kv7.2/7.3 targeting drugs offer the potential to treat a spectrum of these neuropsychiatric diseases including, but not limited to, mood disorders such as major depressive disorder, bipolar disorder, and anxiety.

Pain Disorders

BHV-7000

PC
P 1
P 2
P 3
FILE

In the United States, over 30 million adults are estimated to be living with chronic pain. Pharmacological treatments for pain vary according to patient needs. Initial or first line treatment often includes agents that reduce neuronal hyperexcitability, such as antiseizure medicines. Second line treatment of persistent, severe pain may require escalation to opioids. Thus, an urgent need exists for effective, non-addictive pain therapies. Selective Kv7 potassium channel activators represent a new approach in the development of non-opioid therapeutic options for pain.

In addition to reduced abuse and addiction risk potential, our Kv7 potassium channel platform addresses the complexities of channel subtype physiology through targeted pharmacology to overcome the limitations inherent in unbiased Kv7 activators and is intended to deliver a well-tolerated, highly effective, non-opioid treatment for pain.

Our Kv7 program research was supported in part with funding from the National Institutes of Health (“NIH”) through the NIH Helping to End Addiction Long-term Initiative (“NIH HEAL Initiative”).

GLUTAMATE PLATFORM

DRUG NAME

INDICATION

PRECLINICAL

PHASE 1

PHASE 2

PHASE 3

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Spinocerebellar Ataxia (SCA)

Troriluzole

PC
P 1
P 2
P 3
FILE

INDICATION:

Spinocerebellar ataxias (SCA) are a group of rare and ultra-rare progressively debilitating and fatal genetic conditions with no treatments available. SCA are characterized by progressive ataxia affecting coordination of hands, arms and legs as well as balance and speech. Patients experience significant morbidity, including progression to a wheelchair, impaired gait leading to falls, inability to communicate due to speech impairment, difficulty swallowing, and premature death. The range of symptoms and rate of progression of disease depend on the type of SCA, age of onset, and other factors. More than 40 different types of SCA are now recognized. SCA3 (Machado-Joseph Disease) is the most prevalent form of SCA worldwide, representing 30 to 50% of patients.

RATIONALE:

Troriluzole is a new chemical entity (“NCE”) and tripeptide prodrug of the active metabolite, riluzole. Troriluzole is optimized for improved bioavailability, pharmacokinetics, tolerability, and dosing compared to its active metabolite. Based on its mechanism of action, preclinical data and clinical studies, troriluzole has potential for therapeutic benefit in a range of neurological and neuropsychiatric illnesses. Initial development focused on SCA, based on two prior randomized controlled trials conducted by third parties that demonstrated preliminary efficacy of riluzole in the treatment of subjects with ataxia.

Troriluzole has received orphan drug designation from the U.S. Food and Drug Administration for the treatment of SCA.

STATUS

Top-line results from a Phase 3 clinical trial evaluating the efficacy and safety of troriluzole in SCA were announced in May 2022.

Obsessive-Compulsive Disorder (OCD)

Troriluzole

PC
P 1
P 2
P 3
FILE

INDICATION:

Obsessive-Compulsive Disorder (OCD) is a chronic neuropsychiatric disorder characterized by symptoms of obsessions (intrusive thoughts) and compulsions (repetitive behaviors) that can interfere with patients’ functional abilities. According to the National Institute of Mental Health, the 12-month prevalence of OCD is 1% of the U.S. adult population, and approximately half of these cases are characterized as severe. Despite the significant public health burden, no novel mechanisms of action have been approved by the FDA for OCD in over two decades. First-line treatment for OCD includes cognitive behavioral therapy, selective serotonin reuptake inhibitors and adjunctive use of atypical antipsychotics. Nonetheless, up to 60% of patients have an inadequate response to conventional intervention strategies and some seek invasive neurosurgical procedures to ameliorate symptoms.

RATIONALE:

Troriluzole is a new chemical entity (“NCE”) and tripeptide prodrug of the active metabolite, riluzole. Troriluzole is optimized for improved bioavailability, pharmacokinetics, tolerability, and dosing compared to its active metabolite. Based on its mechanism of action, preclinical data and clinical studies, troriluzole has potential for therapeutic benefit in a range of neurological and neuropsychiatric illnesses. The rationale for use of troriluzole in OCD is supported by clinical data with its active metabolite, riluzole, in populations with OCD in open-label and placebo-controlled clinical trials as well as in preclinical, genetic and neuroimaging studies implicating the glutamatergic hyperactivity in the pathogenesis of OCD.

Phase 2/3 study results demonstrated that troriluzole administered once daily as adjunctive therapy in OCD patients with inadequate response to standard of care showed consistent numerical improvement over placebo on the Yale-Brown Obsessive Compulsive Scale (Y-BOCS) at all study timepoints (weeks 4 to 12). While the primary outcome measure at week 12 (p = 0.22 at week 12) was not met, it was significant at week 8 (p = 0.05). Given the strong signal in the Phase 2/3 proof of concept study, a Phase 3 program was initiated.

STATUS

Two Phase 3 studies evaluating the efficacy and safety of troriluzole as adjunctive therapy in OCD are currently ongoing.

MYOSTATIN PLATFORM

DRUG NAME

INDICATION

PRECLINICAL

PHASE 1

PHASE 2

PHASE 3

FILED

MORE INFO

Spinal Muscular Atrophy (SMA)

Taldefgrobep

PC
P 1
P 2
P 3
FILE

INDICATION:

Spinal muscular atrophy (SMA) is a rare genetic neurodegenerative disorder characterized by the loss of motor neurons, atrophy of the voluntary muscles of the limbs and trunk, and progressive muscle weakness that is often fatal and typically diagnosed in young children. The underlying pathology of SMA is caused by insufficient production of the SMN (survival of motor neuron) protein, essential for the survival of motor neurons, and is encoded by two genes, SMN1 and SMN2. In the U.S., SMA affects approximately 1 in 11,000 births, and about 1 in every 50 Americans is a genetic carrier.

RATIONALE:

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 targets myostatin, a natural protein that limits skeletal muscle growth, through two mechanisms: lowering myostatin directly and blocking key downstream signaling mechanisms. Myostatin inhibition is a potential therapeutic strategy for children and adults with a range of neuromuscular conditions for whom active myostatin can limit the skeletal muscle growth needed to achieve developmental and functional milestones.

STATUS

A Phase 3 trial to evaluate the efficacy and safety of taldefgrobep in SMA is currently ongoing.

ARM™ PLATFORM

DRUG NAME

INDICATION

PRECLINICAL

PHASE 1

PHASE 2

PHASE 3

FILED

MORE INFO

Multiple Myeloma

BHV-1100

PC
P 1
P 2
P 3
FILE

INDICATION:

BHV-1100 is being studied to provide antigen target specificity to natural killer (NK) cell therapies with the goal of enhancing efficacy and safety in the combination treatment of multiple myeloma.

RATIONALE:

Antibody Recruiting Molecules (ARM™) are bispecific molecules that recruit endogenous antibodies to target cancer, virally infected cells, and disease-causing microorganisms for immune-mediated clearance. Similar to biologics, ARMs directly engage the immune system to destroy disease cells by connecting target disease cells with components of the immune system. ARMs are engineered as modular components that are readily interchangeable, giving the platform tremendous flexibility for a variety of indications and therapeutic areas.

STATUS

A Phase 1 trial to evaluate the safety and tolerability as well as exploratory efficacy endpoints in newly diagnosed multiple myeloma patients is currently ongoing.

DISCOVERY RESEARCH

PLATFORM / PROGRAM

PRECLINICAL

PHASE 1

PHASE 2

PHASE 3

FILED

MORE INFO

Kv7 Platform

PC
P 1
P 2
P 3
FILE

Kv7 is a voltage-gated potassium channel present in the brain and is a critical regulator of neuronal excitability.

TRPM3

PC
P 1
P 2
P 3
FILE

Biohaven’s first-in-class molecule directed against TRPM3, a novel druggable calcium channel in the transient receptor potential (TRP) cation channel family.

MATE™ Platform

PC
P 1
P 2
P 3
FILE

Multimodal Antibody Therapy Enhancers or (MATE™) are a next-generation antibody conjugation technology that enables site-directed pairing with off-the-shelf therapeutic monoclonal antibodies (mAbs), or therapeutic commercial immunoglobulin (IG) pooled from donors.

MoDE™ Platform

PC
P 1
P 2
P 3
FILE

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.

ARM™ Platform

PC
P 1
P 2
P 3
FILE

Antibody Recruiting Molecules (ARMs) are bispecific molecules that recruit endogenous antibodies to target cancer, virally infected cells, and disease-causing microorganisms for immune-mediated clearance.

UC1MT

PC
P 1
P 2
P 3
FILE

The UC1MT discovery program is focused on the development of a therapeutic antibody targeting extracellular metallothinein (MT), a stress response protein synthesized during infection, inflammation, and autoimmune diseases.



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