- 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
NEW
HAVEN, Conn., May 31, 2023
/PRNewswire/ -- Biohaven Ltd. (NYSE: BHVN) ("Biohaven"), a global
clinical-stage biopharmaceutical company focused on the discovery,
development and commercialization of life-changing therapies for
people with debilitating diseases, including ultra-rare disorders,
will provide an overview of clinical progress, regulatory updates,
and pipeline developments at an in-person R&D Day today,
concurrently with the Yale Ventures' Innovation Summit 2023 taking
place on May 31-June 1. Members of
Biohaven's senior management team and key opinion leaders will meet
with investors and research analysts.
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 America and 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.
- Biohaven has 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.
Vlad Coric M.D., CEO and Chairman
of Biohaven, commented, "Today's R&D Day review of our robust
pipeline highlights Biohaven's continued dedication to advance
novel therapeutics for brain disorders and builds upon our team's
successes with the prior approvals of Nurtec® ODT and Zavzpret™
that have changed the treatment paradigm in migraine."
"The NDA we submitted for troriluzole for SCA3 represents
approximately seven years of effort by the Biohaven team to bring
forward a potentially new treatment for this ultra-rare disease.
Advancing a new investigational drug for a rare disease that has no
current therapy is a multi-year process that is the culmination of
not only our own internal efforts but also close coordination with
patient advocacy groups including the National Ataxia Foundation
(NAF), leading academic researchers and regulatory agencies. Rare
brain diseases are particularly challenging to research given
relatively small populations of patients to run / participate in
clinical trials, the need to rely on real-world data and often a
lack of standardized ratings scales or biomarkers. We could not
have advanced the SCA program this far without the leadership from
the National Ataxia Foundation that has supported basic science
research as well as the development of natural history cohorts in
SCA that serve as an external control for longitudinal studies. The
Biohaven clinical trials in SCA were a first of its kind in this
area and utilized a newly developed rating scale (the functional
SARA or f-SARA) that was developed in close consultation with the
FDA using standard regulatory pathways to elucidate this new scale.
We look forward to interactions with the US FDA, EMA and other
regulatory agencies across the globe as our submissions advance in
the review process," Dr. Coric added.
"We are pleased that our decades-long investment in ataxia
research and understanding of disease progression has accelerated
treatment development for SCA," said Andrew
Rosen, Executive Director of the National Ataxia Foundation.
"Thank you to all NAF members who participated in these important
trials."
Bruce Car PhD, Biohaven's CSO, stated, "Given the lack of
efficacious therapies for many brain disorders, we must urgently
evaluate new mechanisms and approaches to change the treatment
paradigm in this therapeutic area. Our R&D Day today
demonstrates the commitment that we have given to this effort with
exciting new approaches ranging from our submission of a glutamate
modulator in SCA to immune modulation using small molecule
degraders to ion channels targeting agents for epilepsy and mood
disorders, as well as a biologic treatment to enhance muscle growth
in SMA. Our scientific and clinical team is poised to file multiple
new INDs from our deep pipeline and complete late-stage clinical
trials in the next couple of years. It is an exciting time at
Biohaven for our passionate employees, patients and investors as we
advance these novel investigational drugs in our
pipeline."
The Research & Development Day presentations will be made
available following the conclusion of the program on
https://ir.biohaven.com/events-presentations/events.
About Biohaven
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.
Forward-looking Statements
This news release includes
forward-looking statements within the meaning of the Private
Securities Litigation Reform Act of 1995. The use of certain words,
including "continue", "plan", "will", "believe", "may", "expect",
"anticipate" and similar expressions, is intended to identify
forward-looking statements. Investors are cautioned that any
forward-looking statements, including statements regarding the
future development, timing and potential marketing approval and
commercialization of development candidates are not guarantees of
future performance or results and involve substantial risks and
uncertainties. Actual results, developments and events may differ
materially from those in the forward-looking statements as a result
of various factors including: the expected timing, commencement and
outcomes of Biohaven's planned and ongoing clinical trials; the
timing of planned interactions and filings with the Food and Drug
Administration; the timing and outcome of expected regulatory
filings; complying with applicable U.S. regulatory requirements;
the potential commercialization of Biohaven's product candidates;
the potential for Biohaven's product candidates to be first in
class therapies; and the effectiveness and safety of Biohaven's
product candidates. Additional important factors to be considered
in connection with forward-looking statements are described in
Biohaven's filings with the Securities and Exchange Commission,
including within the sections titled "Risk Factors" and
"Management's Discussion and Analysis of Financial Condition and
Results of Operations". The forward-looking statements are made as
of the date of this new release, and Biohaven does not undertake
any obligation to update any forward-looking statements, whether as
a result of new information, future events or otherwise, except as
required by law.
MoDEs is a trademark of Biohaven Therapeutics Ltd.
Investor Contact:
Jennifer
Porcelli
Vice President, Investor Relations
jennifer.porcelli@biohavenpharma.com
+1 (201) 248-0741
Media Contact:
Mike
Beyer
Sam Brown Inc.
mikebeyer@sambrown.com
+1 (312) 961-2502
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SOURCE Biohaven Ltd.