BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the
“Company”), a commercial-stage biopharmaceutical company focused on
genetic diseases, today announced it has surpassed its interim
analysis enrollment target and expects topline interim data from
its Phase 3 registrational study (FORTIFY) in individuals with
LGMD2I/R9 in 2025.
FORTIFY is a randomized, double-blind, placebo-controlled Phase
3 study evaluating the safety and efficacy of BBP-418, an
investigational oral therapy in development for the treatment of
individuals living with LGMD2I/R9. The study includes a planned
interim analysis at 12 months focused on assessing glycosylated αDG
as a surrogate endpoint to support Accelerated Approval. The
primary endpoint to be evaluated at 36 months is the North Star
Assessment (NSAD) for limb-girdle type muscular dystrophies and is
designed to provide confirmatory clinical data. BridgeBio is
continuing to enroll FORTIFY in the U.S., UK, Europe, and
Australia. More information about the ongoing Phase 3 clinical
trial of BBP-418 (NCT05775848) can be found here on
clinicaltrials.gov.
Multiple encouraging discussions with the FDA in 2024, focused
on the validated glycosylated αDG bioassay and our interim analysis
plans, continue to support the Company’s plan to pursue Accelerated
Approval for BBP-418 based on the use of glycosylated αDG levels as
a surrogate endpoint in LGMD2I/R9. Furthermore, the Agency
indicated that the Company’s approach to measure glycosylated αDG
levels via a novel, validated muscle tissue-based bioassay appears
reasonable. A peer-reviewed manuscript providing details on the
novel, multiplexed, fluorescence-based Western Blot assay was
recently published in the Journal of Muscle Research and Cell
Motility.
“Reduced glycosylated αDG is the primary molecular driver of
LGMD2I/R9, a serious, progressively debilitating disease that
weakens the muscles causing many affected people to become fully
dependent on a caregiver, while also threatening their cardiac and
respiratory function,” said Douglas Sproule, M.D., M.Sc., chief
medical officer of ML Bio Solutions, a BridgeBio affiliate that is
focused on developing BBP-418 for LGMD2I/R9. “We hope to continue
to serve individuals through expeditious enrollment of the Phase 3
FORTIFY study and look forward to continuing to partner with the
FDA to accelerate development of a potential new therapeutic option
for people living with LGMD2I/R9.”
The Company also announced that the FDA has granted Rare
Pediatric Disease Designation (RPDD) for BBP-418 in the treatment
of LGMD2I/R9, recognizing the rarity of this disease is
characterized by serious manifestations primarily affecting
children. If BBP-418 is approved, BridgeBio may qualify for a
Priority Review Voucher based on receipt of the RPDD. A Priority
Review Voucher can be applied to another therapy in the Company’s
pipeline for a shorter timeline during the review process of a New
Drug Application or can be sold and transferred to another company
looking to receive priority review for one of its applications.
About Limb-girdle Muscular Dystrophy Type 2I/R9
(LGMD2I/R9)LGMD2I/R9 is a monogenic autosomal recessive
disease caused by partial loss of function mutations in the
fukutin-related protein (FKRP) gene, and FKRP mutations impair
glycosylation of alpha-dystroglycan (αDG), a protein associated
with stabilizing muscle cells. Clinical manifestations typically
present as a skeletal myopathy affecting the lower and then upper
limbs, which is commonly later accompanied by respiratory muscle
and cardiac muscle involvement. Individuals who harbor a homozygous
L276I genotype typically develop disease manifestations during late
childhood with progression to loss of independent ambulation (25%),
assisted ventilation (10%), and cardiomyopathy (30%) in adulthood.
Cardiomyopathy is progressive, with an annual loss of 0.4% of left
ventricular ejection fraction (LVEF). Individuals with other FKRP
genotypes typically have an earlier childhood onset with a more
severe clinical course, rapid loss of mobility by 20 years of age,
more frequent cardiac involvement (60%), and eventual respiratory
failure by 30 years of age in nearly all cases.
About BridgeBio Pharma, Inc.BridgeBio Pharma,
Inc. (BridgeBio) is a commercial-stage biopharmaceutical company
founded to discover, create, test and deliver transformative
medicines to treat patients who suffer from genetic diseases.
BridgeBio’s pipeline of development programs ranges from early
science to advanced clinical trials. BridgeBio was founded in 2015
and its team of experienced drug discoverers, developers and
innovators are committed to applying advances in genetic medicine
to help patients as quickly as possible. For more information
visit bridgebio.com and follow us
on LinkedIn and Twitter.
BridgeBio Pharma, Inc. Forward-Looking
StatementsThis press release contains forward-looking
statements. Statements BridgeBio makes in this press release may
include statements that are not historical facts and are considered
forward-looking within the meaning of Section 27A of the Securities
Act of 1933, as amended (the “Securities Act”), and Section 21E of
the Securities Exchange Act of 1934, as amended (the “Exchange
Act”), which are usually identified by the use of words such as
“anticipates,” “believes,” “continues,” “estimates,” “expects,”
“hopes,” “intends,” “may,” “plans,” “projects,” “remains,” “seeks,”
“should,” “will,” and variations of such words or similar
expressions. BridgeBio intends these forward-looking statements to
be covered by the safe harbor provisions for forward-looking
statements contained in Section 27A of the Securities Act and
Section 21E of the Exchange Act. These forward-looking statements,
including statements relating to the clinical and therapeutic and
market potential of BridgeBio’s programs and product candidates,
including BBP-418 for the treatment of LGMD2I/R9, the drug design
and the potential benefits of BBP-418, including its potential to
address serious unmet need for patients with LGMD2I/R9, the
potential and the opportunity to pursue Accelerated Approval
Pathway for BBP-418 in LGMD2I/R9 in the U.S., the potential
BridgeBio may qualify for a Priority Review Voucher based on
receipt of the RPDD, the expected timeline of announcing the
topline data from the interim analysis of FORTIFY in individuals
with LGMD2I/R9 in 2025, and the statements regarding the potential
benefit of our clinical trial or of our product candidate in the
quotes of Dr. Sproule; the progress, timeline and success of
BridgeBio’s ongoing and planned clinical trials of BBP-418, the
timeline of evaluation of the NSAD and secondary endpoints at 36
months, and the expectation of providing confirmatory clinical data
from such results, the plans of engaging with regulatory
authorities, including the collaboration and interaction with the
FDA to address the challenges associated with LGMD2I/R9 drug
development, among others, reflect BridgeBio’s current views about
its plans, intentions, expectations, strategies and prospects,
which are based on the information currently available to BridgeBio
and on assumptions BridgeBio has made. Although BridgeBio believes
that its plans, intentions, expectations, strategies and prospects
as reflected in or suggested by those forward-looking statements
are reasonable, BridgeBio can give no assurance that the plans,
intentions, expectations or strategies will be attained or
achieved. Furthermore, actual results may differ materially from
those described in the forward-looking statements and will be
affected by a number of risks, uncertainties and assumptions,
including, but not limited to, BridgeBio’s ability to continue and
complete its ongoing and planned clinical trials of BBP-418 for the
treatment of LGMD2I/R9, initial and ongoing data from its clinical
trials not being indicative of final data, the design and success
of ongoing and planned clinical trials, difficulties with
enrollment in our clinical trials, adverse events that may be
encountered in our clinical trials, the FDA or other regulatory
agencies not agreeing with BridgeBio’s regulatory approval
strategies, components of our filings, such as clinical trial
designs, conduct and methodologies, or the sufficiency of data
submitted, potential adverse impacts due to global health
emergencies, including delays in regulatory review, manufacturing
and supply chain interruptions, adverse effects on healthcare
systems and disruption of the global economy, the impacts of
current macroeconomic and geopolitical events, including changing
conditions from hostilities in Ukraine and in Israel and the Gaza
Strip, increasing rates of inflation and rising interest rates, on
our business operations and expectations as well as those risks set
forth in the Risk Factors section of BridgeBio’s most recent Annual
Report on Form 10-K, and BridgeBio’s other filings with the U.S.
Securities and Exchange Commission. Moreover, BridgeBio operates in
a very competitive and rapidly changing environment in which new
risks emerge from time to time. These forward-looking statements
are based upon the current expectations and beliefs of BridgeBio’s
management as of the date of this press release and are subject to
certain risks and uncertainties that could cause actual results to
differ materially from those described in the forward-looking
statements. Except as required by applicable law, we assume no
obligation to update publicly any forward-looking statements,
whether as a result of new information, future events or
otherwise.
BridgeBio Contact:Vikram
Balicontact@bridgebio.com(650)-789-8220
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