- Spinocerebellar Ataxia (SCA) is a rare, genetic,
life-threatening neurodegenerative disease with no available
treatment.
- Troriluzole demonstrated a 50-70% slowing of SCA disease
progression on the primary and secondary outcome measures at the
3-year endpoint in a real-world evidence (RWE) study.
- Troriluzole has a well-established safety profile and if
approved, would be the first and only FDA-approved treatment for
SCA; subject to receipt of FDA approval, Biohaven is
prepared to commercialize troriluzole for SCA in the US in
2025.
NEW
HAVEN, Conn., Feb. 11,
2025 /PRNewswire/ -- Biohaven Ltd. (NYSE: BHVN)
(Biohaven or the Company), today announced that the US Food
and Drug Administration (FDA) has accepted for review the Company's
New Drug Application (NDA) for troriluzole for the treatment of
adult patients with spinocerebellar ataxia (SCA) and has granted
Priority Review. This designation is assigned to applications for
drugs that would offer a significant improvement over other
available treatments for a given disorder or would provide a
treatment option where none exists. In the case of SCA, a rare,
genetic, neurodegenerative disease, troriluzole would be the first
and only FDA-approved treatment for this life-threatening disorder.
The FDA's decision regarding the NDA is expected within 6 months of
filing (during 3Q2025). Based on FDA Priority Review timelines and
if ultimately approved, Biohaven is prepared to commercialize
troriluzole for SCA in the US in 2025.
Melissa Beiner, M.D., SCA
Clinical Development Lead at Biohaven, commented, "Our NDA filing
is the culmination of over 8 years of clinical research and
represents an important collaboration across the SCA community. The
troriluzole NDA reflects rigorous scientific collaborations between
advocacy groups, patients and their families, clinical experts in
SCA and Biohaven. The FDA decision to grant Priority Review
demonstrates the extremely high unmet need in this rare
neurodegenerative disease. Time is of the essence for patients with
SCA, who are suffering relentless and irreversible functional
decline including impairments in coordination and balance leading
to falls, loss of ambulation, and difficulties with vision, speech
and swallowing." Dr. Beiner added, "The robust clinical data
presented in the NDA demonstrate sustained and compelling treatment
benefit in SCA patients treated with troriluzole, a once-daily,
oral pill. We look forward to working closely with the FDA
throughout the review process to bring the very first treatment to
patients and families suffering from SCA."
The NDA submission was based, in part, on positive topline
results from Study BHV4157-206-RWE (NCT06529146), in which
troriluzole 200 mg dosed orally in patients with SCA met the
study's primary endpoint of change from baseline on the functional
Scale for the Assessment and Rating of Ataxia (f-SARA), in all SCA
genotypes, at 3 years compared to an external control arm.
Troriluzole showed statistically significant superiority across 9
consecutive, prespecified primary and secondary endpoints with
highly consistent, sustained, robust and clinically meaningful
treatment effects. SCA patients treated with troriluzole showed a
50-70% slower rate of decline, representing 1.5-2.2 years delay in
disease progression, over the 3-year study period (Figures 1-3 and
Table 1). The NDA also includes confirmatory and supportive data
from Studies BHV4157-201 and BHV4157-206, the first large,
multi-center registrational trials in SCA. Notably, these data
include disease stabilization in the SCA3 genotype (Figure 4) and a
reduction in falls in all SCA genotypes (Figure 5), both compared
to placebo over 48 weeks in Study BHV4157-206.
Jeremy Schmahmann, M.D.,
Professor of Neurology at Harvard Medical
School and Founding Director of the Ataxia Center and the
Martha and Robert Fogelman Endowed Chair in Ataxia and Cerebellar
Neurology at Massachusetts General Hospital commented, "The FDA
acceptance for review of this NDA represents a critical milestone
for SCA patients. Since the discovery of the first gene for SCA in
1993, patients and families affected by SCA have watched generation
after generation suffer severe, progressive disability and
premature death with no treatment options. The need for an
intervention that can slow disease progression and help patients
maintain their independence is urgent. The delay in disease decline
shown in the real-world evidence study is a watershed in the
history of the SCAs. This is what patients have been waiting
for. It is what the doctors who have been powerless, have been
waiting for. Additionally, the importance of troriluzole's effects
on reducing falls in this patient population cannot be overstated.
I applaud the FDA for recognizing this urgency by granting a
Priority Review and look forward to using troriluzole in the clinic
if approved."
Biohaven's troriluzole clinical development program in SCA
collected data over 8 years, including a robust long-term safety
profile, and was the first industry trial conducted in SCA. The
external control arms used in Biohaven's BHV4157-206-RWE Study were
provided from objective, third-party data gathered from two
independent natural history cohorts: one in the United States and one in Europe (EUROSCA Natural History Study).
The National Ataxia Foundation (NAF) sponsored the Clinical
Research Consortium for the Study of Cerebellar Ataxia (CRC-SCA)
that served as the basis for the US natural history cohort. The
data from the CRC-SCA is managed by the University of South Florida Health Informatics
Institute. A total of 35 clinical sites provided data in the US and
European natural history cohorts that served as the external
controls in BHV4157-206-RWE. As per instructions from FDA on the
real-world evidence study design and statistical analysis plan, the
external control arm was determined using a Propensity Score
Matching (PSM) method to ensure that untreated subjects from the
comparator natural history cohort were rigorously matched to
treated subjects from the troriluzole arm of Study BHV4157-206. PSM
was used on all prognostic, demographic, and baseline
characteristics known to be associated with disease progression in
SCA, including baseline f-SARA, age, sex, age at symptom onset,
genotype, and trinucleotide repeat length (by genotype).
Andrew Rosen, Chief Executive
Officer of the National Ataxia Foundation (NAF), stated,
"Biohaven's SCA program reflects years of dedicated clinical
research and collaboration with leading world experts and advocacy
groups to advance the ataxia field. We are proud that our
multi-year effort to fund and support the Clinical Research
Consortium for the Study of Cerebellar Ataxia played such a
critical role in providing the external control arm of Biohaven's
study. The goal of CRC-SCA is to improve our understanding of
SCA disease progression and to promote the development of
disease-modifying therapies for SCA." Mr. Rosen added, "On behalf
of patients and families, who have watched generations of family
members succumb to this devastating disease and have been waiting
for decades for a treatment that could slow disease progression, I
thank the FDA for not only accepting this NDA for review, but
recognizing the need for urgency for our community in the form of a
Priority Review."
Biohaven previously received both Fast-Track and Orphan Drug
Designation (ODD) from the FDA, and ODD from the European Medicines
Agency, where a troriluzole MAA is currently under review.
An expanded access protocol (EAP) is currently enrolling
patients with SCA who are eligible. EAPs are designed to give early
access to potential therapies before they are approved by the FDA.
More information about can be found at
https://clinicaltrials.gov/study/NCT06034886.
About Spinocerebellar Ataxia (SCA)
Spinocerebellar
ataxia is a group of dominantly inherited neurodegenerative
disorders characterized by progressive loss of voluntary motor
control and atrophy of the cerebellum and brainstem. SCA affects
approximately 15,000 people in the United States and 24,000 in Europe and the United Kingdom. Patients experience
significant morbidity, including impaired gait leading to falls,
loss of ambulation and progression to a wheelchair, inability to
communicate due to speech impairment, difficulty swallowing, and
premature death. While signs and symptoms can appear anytime from
childhood to late adulthood, SCA typically presents in early
adulthood and progresses over a number of years. Currently, there
are no FDA-approved treatments and no cure for SCA.
About Troriluzole
Troriluzole is a new chemical entity
(NCE) and third-generation novel prodrug that modulates glutamate,
the most abundant excitatory neurotransmitter in the human body.
The primary mode of action of troriluzole is reducing synaptic
levels of glutamate. Troriluzole increases glutamate uptake from
the synapse, by augmenting the expression and function of
excitatory amino acid transporters located on glial cells that play
a key role in clearing glutamate from the synapse. The glutamate
modulating activity of troriluzole addresses the widely documented
glutamate deregulation that underlies neurodegeneration and
Purkinje cell dysfunction in patients with SCA. Troriluzole also
has the potential to be developed in a number of other diseases
associated with excessive glutamate. More information about
troriluzole can be found at the Biohaven's website:
https://www.biohaven.com/pipeline/clinical-programs/glutamate/.
About Biohaven
Biohaven is a
biopharmaceutical company focused on the discovery, development,
and commercialization of life-changing treatments in key
therapeutic areas, including immunology, neuroscience, and
oncology. The company is advancing its innovative portfolio of
therapeutics, leveraging its proven drug development experience and
multiple proprietary drug development
platforms. Biohaven's extensive clinical and preclinical
programs include Kv7 ion channel modulation for epilepsy and mood
disorders; extracellular protein degradation for immunological
diseases; TRPM3 antagonism for migraine and neuropathic pain;
TYK2/JAK1 inhibition for neuroinflammatory disorders; glutamate
modulation for OCD and SCA ; myostatin inhibition for neuromuscular
and metabolic diseases, including SMA and obesity; antibody
recruiting bispecific molecules and antibody drug conjugates for
cancer. 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, , including the
potential approval and commercialization of troriluzole for SCA,
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 FDA; 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 approved 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 news 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.
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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