BridgeBio Completes Enrollment of FORTIFY, Phase 3 Registrational Study of BBP-418 in Limb-girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9)
– Study exceeded target enrollment, with an expected topline data readout from the interim analysis expected in 2025
– BridgeBio believes there is an opportunity to pursue Accelerated Approval in the U.S. for BBP-418 in LGMD2I/R9 based on a potential biomarker surrogate endpoint of glycosylated alpha-dystroglycan (αDG) at time of the interim analysis
– If successful, BBP-418 could be the first approved therapy for individuals living with LGMD2I/R9 in the U.S.
– Enrollment completion announced on 10th annual LGMD Awareness Day, a collaborative yearly effort on September 30th to globally raise awareness of individuals living with LGMD
PALO ALTO, Calif., Sept. 30, 2024 (GLOBE NEWSWIRE) — BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases, announced, on LGMD Awareness Day, the completion of enrollment of FORTIFY, the Company’s Phase 3 registrational study of BBP-418 in individuals with LGMD2I/R9.
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 potentially 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 supporting the efficacy of BBP-418. More information about the Phase 3 clinical trial of BBP-418 (NCT05775848) can be found here on clinicaltrials.gov. A topline data readout from the interim analysis is expected in 2025.
“Living with a progressive muscle wasting condition like LGMD2I/R9 means that individuals are continually losing the ability to perform daily activities independently and relying on friends and family to support them as they experience a continued decline in strength and health. For patients with LGMD2I/R9, there are currently no approved treatment options, but the promise seen in the rapid enrollment of this clinical trial provides hope for patients and their families that this may change in the future,” said Kelly Brazzo, CEO of CureLGMD2i Foundation.
“Completing rapid enrollment of FORTIFY is an important milestone and underscores the need for a therapeutic option for patients,” said Douglas Sproule, M.D., M.Sc., Chief Medical Officer of ML Bio Solutions, a BridgeBio company developing BBP-418 for LGMD2I/R9. “Based on multiple encouraging discussions with the FDA, we believe there is an opportunity to pursue Accelerated Approval in the U.S. for BBP-418 in LGMD2I/R9 based on a potential surrogate endpoint biomarker of glycosylated αDG at time of the interim analysis. If successful, BBP-418 could be the first approved disease-modifying therapy for individuals living with LGMD2I/R9 in the U.S. We’re immensely grateful to the patients, their families, and the trial site investigators participating in our study and look forward to sharing pivotal data with the community.”
BBP-418 has previously received Orphan Drug, Fast Track, and Rare Pediatric Disease Designations from the FDA and Orphan Drug Designation from the European Medicines Agency (EMA). Consistent with the Rare Pediatric Designation from the FDA, if BBP-418 is approved, BridgeBio may qualify for a Priority Review Voucher.
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 bridgebiodev.wpengine.com and follow us on LinkedIn, Twitter and Facebook.
BridgeBio Pharma, Inc. Forward-Looking Statements
This 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 potential benefits of
BBP-418, including its potential to address 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 that BridgeBio may qualify for a
Priority Review Voucher based on receipt of the Rare Pediatric
Designation from the FDA, the expected timeline of announcing
the topline data from the interim analysis of FORTIFY in
individuals with LGMD2I/R9 in 2025, the statements regarding the
potential benefit of our clinical trial or of our product
candidate in the quotes of Dr. Sproule and Kelly Brazzo, and the
progress, timeline and success of BridgeBio’s ongoing and
planned clinical trials of BBP-418, 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, the ability of BBP-418 to retain
Orphan Drug, Fast Track, and Rare Pediatric Disease Designations
from the FDA and Orphan Drug Designation from the European
Medicines Agency and 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 Bali
[email protected]
(650)-789-8220