NEW
HAVEN, Conn., July 27,
2023 /PRNewswire/ -- Biohaven Ltd. (NYSE: BHVN)
("Biohaven" or the "Company"), a global clinical-stage
biopharmaceutical company focused on the discovery, development and
commercialization of life-changing therapies to treat a broad range
of rare and common diseases, today announced multiple updates on
key programs including: preliminary EEG data from its lead
investigational agent, BHV-7000, from the Kv7 activator program;
Phase 1 update on its brain-penetrant dual TYK2/JAK1 inhibitor,
BHV-8000; development update on its IgG degrader, BHV-1300, from
the bispecific platform; and a regulatory update on
troriluzole.
Biohaven reported positive, interim data from an
electroencephalogram (EEG) biomarker study with the initial,
low-dose of BHV-7000 studied in healthy volunteers. Preliminary
Phase 1 data confirmed evidence of target engagement in the central
nervous system for subjects with projected therapeutic
concentrations of BHV-7000 (based on the EC50 from
preclinical models), measured by changes from baseline in EEG
spectral power that occurred after dosing. These pharmacodynamic
(PD) effects were similar to those reported in the literature for
antiseizure medicines (ASMs), including Kv7 activators in
development that are clinically effective in treating epilepsy.
BHV-7000's PD effects were also differentiated from those reported
for other Kv7 activators including, specifically, the absence of
increases in EEG spectral power in frequency bands associated with
drowsiness and somnolence. While additional, higher-dose groups of
BHV-7000 are still being evaluated in the EEG analysis, the results
from the low-dose group validate the preclinical hypothesis,
confirm the Phase 1 SAD/MAD clinical data, and provide strong
support for Biohaven's plans to initiate pivotal studies with
BHV-7000 in focal epilepsy and bipolar disorder in the second half
of 2023. The preliminary data highlight BHV-7000's differentiation
and potentially favorable clinical profile compared to other ASMs,
and Biohaven expects to present the complete EEG results by the end
of the year. Additionally, new pharmacokinetic data from multiple
clinical formulations being studied has now confirmed a once daily
extended-release formulation that will be used in the Phase 2/3
clinical programs.
Michael Bozik, M.D., President,
Ion Channel Research & Development at Biohaven, commented,
"These EEG data provide further evidence of the paradigm changing
potential of BHV-7000 to deliver robust antiseizure efficacy,
without the burdensome CNS adverse effects typical of antiseizure
medicines. While other Kv7 modulators have exhibited clear efficacy
in the clinic, they have been limited by CNS effects such as
somnolence, speech disorder, and memory impairment. Demonstrating
target engagement without affecting the power spectra of low
frequency bands in the EEG is consistent with the absence of
somnolence and fatigue seen in the Phase 1 SAD/MAD studies of
BHV-7000. We are extremely pleased that BHV-7000 continues to
demonstrate an exceptional and differentiated clinical
profile."
Biohaven has now successfully dosed three dose cohorts with
single ascending doses of its brain penetrant TYK2/JAK1 agent,
BHV-8000, in the ongoing Phase 1 study. The ongoing Phase 1
study is designed to evaluate the safety, tolerability,
pharmacokinetics and pharmacodynamics of single and multiple
ascending doses of BHV-8000 in healthy volunteers. Based on the
preliminary data that are available, projected therapeutic
concentrations of BHV-8000 were achieved, and BHV-8000 was well
tolerated with only mild adverse events reported. These data
support further development of BHV-8000, and Biohaven anticipates
beginning a Phase 2 clinical trial with BHV-8000 in Parkinson's
disease and potentially other neuroinflammatory diseases in
2024.
Biohaven's first-in-class bispecific IgG degrader, BHV-1300,
is demonstrating a highly competitive safety, manufacturable and
pharmacodynamic profile as it advances to IND in 2H2023.
BHV-1300 sits atop a deep pipeline of partially derisked, follow-on
IgG degraders as well as antigen-specific degraders providing both
optionality and a sustainable output of drug candidates for several
years. Currently approved FcRn inhibitors are limited by the need
for healthcare provider administration and some have the potential
for effects on albumin and cholesterol. Additionally, FcRn
inhibitors preclude the coadministration of biologic
immunosuppressive therapies – this mechanism clears all Fc
containing drugs – while BHV-1300 dosage regimens will allow
coadministration of existing standards of care.
Biohaven anticipates completing enrollment in a Phase 3 study
of troriluzole in OCD by the end of the year. Two Phase 3
randomized, double-blind, placebo-controlled studies are expected
to enroll up to 700 patients (in each trial) across nearly 200
global study sites.
On its SCA program, the FDA informed Biohaven that it would
not review the recently submitted NDA application for troriluzole
given that the study's primary endpoint was not met and thus, would
not permit a substantive review. The communication from the FDA
indicated that the Company may request a Type A meeting within 30
days. Biohaven is committed to working closely with the FDA to
bring troriluzole to people with SCA3 as quickly as possible given
no therapy is currently approved for this ultra-rare genetic
disorder and is requesting a Type A meeting to comprehensively
address FDA's concerns cited in the refusal to file letter. Any
updates regarding the Type A meeting will be provided subsequent to
the upcoming regulatory interaction.
Vlad Coric, M.D. CEO and Chairman
commented, "As a physician, I am deeply disappointed by the FDA's
decision not to review the submitted NDA, and not to give complete
consideration of all available data that we believe show disease
modifying effects for this genetic disorder that has no approved
treatments. Troriluzole's active metabolite has a known safety
profile and is well-tolerated; and, troriluzole was submitted under
a 505(b)(2) application with data suggesting an 80% reduction or
7-month benefit in disease progression over the 1-year study
period. The risk-benefit profile for troriluzole warranted careful
consideration by the FDA for this ultra-rare disorder. SCA is
clearly a severely debilitating, life-threatening disease with
substantial unmet need. The approximately 6,000 patients in
North America at least deserved a
thorough review of the data package submitted. Approximately 200
patients have been treated with troriluzole for up to 3 years
(whose diagnosis has been confirmed with genetic testing), and the
troriluzole treated cohorts have remained stable compared to the
untreated natural history cohorts who clearly show marked disease
progression over that similar time period." Dr. Coric added, "We
stand committed to serving people suffering from SCA and will
continue to work with the FDA to request further consideration of
all the available data. We believe the NDA package is compelling
and shows that treatment with troriluzole leads to clinically
meaningful treatment benefits, including significantly delaying
disease progression and reduction in falls. We stand by these data
and analyses."
About Biohaven
Biohaven is a global
clinical-stage biopharmaceutical company focused on the discovery,
development and commercialization of life-changing therapies to
treat a broad range of rare and common
diseases. 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. Biohaven 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 (MoDEs™ platform) 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.