Biohaven Provides Overview of Clinical Progress, Regulatory Updates, and Pipeline Developments at R&D Day
- Submitted a new drug application (NDA) for troriluzole in Spinocerebellar Ataxia Type 3 (SCA3) to
U.S.FDA in 2Q2023, marking the team's fourth NDA in approximately 3 years
- Released additional data from Kv7 platform, including Phase 1 safety data by dose groups for BHV-7000 that further validates differentiated profile
- Projected Phase 3 Spinal Muscular Atrophy trial to complete enrollment in 2023
- Initiated Phase 1 study of brain penetrant TYK2/JAK1 inhibitor, BHV-8000, and anticipate beginning Phase 2 trial in Parkinson's disease next year
- Highlighted robust pipeline with multiple INDs planned to be filed within the next year, including pan IgG degrader for multiple immune-mediated diseases in 2023
The clinical progress, regulatory updates, and pipeline developments to include:
- Troriluzole in SCA: Submitted New Drug Application (NDA) to the
U.S.FDA for troriluzole for the treatment of spinocerebellar ataxia type 3 (SCA3), an ultra-rare, genetically-defined, neurodegenerative disease associated with progressive disability, frequent falls, loss of ambulation, speech and swallowing impairment, and premature death that is the most common SCA genotype worldwide.
- NDA supported by consistent treatment benefits observed in patients with genotype SCA3 in Study BHV4157-206 across multiple outcome measures including the change from baseline f-SARA at Week 48, CGI-I total score at Week 48, and a robust reduction in fall risk over the study period. A rigorous analysis by genotype was possible as patients were randomized by genotype strata at baseline prior to randomization in the pivotal Phase 3 48-week, double-blind, placebo-controlled phase of Study BHV4157-206.
- SCA3 represented 41% of study participants, consistent with being the most common subtype. SCA3 affects approximately 10,600 people in
North Americaand in the EU and Japan.
- NDA further supported by a composite scale development (SCACOMS) and analysis of Study BHV4157-206, and confirmatory evidence of efficacy provided by data from the 3-year, long-term open-label (OLE) extension phase of two studies (BHV4157-206 and BHV4157-201) using a Matching Adjusted Indirect Comparison (MAIC) to an external control group.
Biohavenhas previously received Fast-Track and Orphan drug designation (ODD) from the FDA, and ODD from the European Medicines Agency, for troriluzole in SCA.
- Kv7 Platform: Highlighted progress and broad potential of Kv7 platform, including ongoing and planned development of BHV-7000 (Kv7.2/3 activator):
- Phase 1 EEG study with BHV-7000 in 1H2023.
- Pivotal studies with BHV-7000 in focal epilepsy and bipolar disorder planned to initiate in 2H2023.
- Burgeoning evidence for therapeutic benefits of targeting Kv7 in diverse, high unmet need indications.
- Bispecific Platform: Provided updates regarding planned INDs for targeted extracellular protein degradation franchise (including IgG, IgA, and autoantibody-specific degrader programs) and next-generation antibody-drug conjugate (ADC) technologies;
- IND application for novel IgG degrader, BHV-1300, on track for submission in 2023.
- TYK2/JAK1 Inhibition in Immune-Mediated Brain Disorders: Began dosing with BHV-8000 (an oral, brain-penetrant, dual TYK2/JAK1 inhibitor) in a Phase 1 study in normal healthy volunteers.
- Taldefgrobep Alfa:
- In Spinal Muscular Atrophy: Enrollment of approximately 225 patients in global Phase 3 trial now anticipated to complete in 2023.
- In Metabolic Disorders: Planned Phase 2 trial initiation in 2H2023.
"The NDA we submitted for troriluzole for SCA3 represents approximately seven years of effort by the
"We are pleased that our decades-long investment in ataxia research and understanding of disease progression has accelerated treatment development for SCA," said
The Research & Development Day presentations will be made available following the conclusion of the program on https://ir.biohaven.com/events-presentations/events.
Biohaven is a global clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of life-changing therapies for people with debilitating diseases, including rare disorders. Biohaven's experienced management team brings with it a track record of delivering new drug approvals for products for diseases such as migraine, depression, bipolar disorder and schizophrenia. The company is advancing a pipeline of therapies for diseases with little or no treatment options, leveraging its proven drug development capabilities and proprietary platforms, including Kv7 ion channel modulation for epilepsy and neuronal hyperexcitability, glutamate modulation for obsessive-compulsive disorder and spinocerebellar ataxia, myostatin inhibition for neuromuscular diseases, and brain-penetrant TYK2/JAK1 inhibition for immune-mediated brain disorders. Biohaven's portfolio of early- and late-stage product candidates also includes discovery research programs focused on TRPM3 channel activation for neuropathic pain, CD-38 antibody recruiting, bispecific molecules for multiple myeloma, antibody drug conjugates (ADCs), and targeted extracellular protein degrader platform technology (MoDE™) with potential application in neurological disorders, cancer, and autoimmune diseases. For more information, visit www.biohaven.com.
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