BridgeBio Pharma Shares Positive Long-Term Data from an Ongoing Phase 2 Study, which Support the Potential Use of Glycosylated Alpha-dystroglycan (⍺DG) Levels as a Surrogate Endpoint in Limb-girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9)
October 09 2023 - 7:30AM
BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the
“Company”), a commercial-stage biopharmaceutical company
focused on genetic diseases and cancers, shared new long-term data
from its Phase 2 trial in patients with limb-girdle muscular
dystrophy type 2I/R9 (LGMD2I/R9) at the Annual Congress of World
Muscle Society (WMS) in Charleston, South Carolina. The new
long-term data remains consistent with earlier data from the Phase
2 study showing a well-tolerated safety profile and encouraging
preliminary efficacy. Additionally, early changes in glycosylated
⍺DG levels at 3 months appear to be associated with ambulatory
improvements at 9 months, providing support for the possible use of
glycosylated ⍺DG levels as a surrogate endpoint in the ongoing
Phase 3 study for accelerated approval.
BridgeBio presented 21-month results from its ongoing Phase 2
trial, including:
- Large (≥80%), sustained reduction in creatine kinase observed
over an extended (up to 21-months) treatment period
- Stabilization in NSAD scores and ambulatory
measures observed over 21-months of BBP-418 treatment
- BBP-418 continues to be well-tolerated with longer-term
treatment
- No treatment-related SAEs or dose limiting toxicities observed
with 21-months of BBP-418 dosing
“There is a serious unmet need for those with LGMD2I/R9. The
consistent improvement observed in biomarkers as well as
stabilization in ambulatory measures are encouraging and help
predict the clinical benefit for patients. The data for BBP-418
give me hope that similar results will be carried through in the
currently enrolling Phase 3 trial, FORTIFY, for patients with
LGMD2I/R9,” said Amy Harper, M.D., professor in the department of
neurology at Virginia Commonwealth University (VCU) and primary
investigator of the Phase 2 clinical trial in LGMD2I/R9.
BridgeBio recently announced the first patient dosed in FORTIFY,
its Phase 3 registrational study in patients with LGMD2I/R9 and is
continuing to enroll throughout the U.S. with clinical trial sites
planned for Europe and Australia. The Phase 3 FORTIFY
registrational study is a randomized, double-blind,
placebo-controlled study evaluating the safety and efficacy of
BBP-418. FORTIFY has a planned interim analysis at 12 months
focused on assessing glycosylated αDG as a surrogate endpoint to
potentially support an accelerated approval. The NSAD and secondary
endpoints will be evaluated at 36 months, and results are expected
to provide confirmatory clinical data.
“To see that our therapy is potentially providing clinical
improvements to people who otherwise could be non-ambulatory is
deeply heartening for us,” 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. “LGMD2I/R9 is a
severely progressive disease and, inevitably, the weakening of the
muscles causes many affected people to become fully dependent on a
caregiver. Current treatments for LGMD2I/R9 are purely supportive.
BBP-418 has the potential to be the first disease-modifying orally
administered therapy available for patients.”
More information about FORTIFY, the ongoing Phase 3 clinical
trial of BBP-418 (NCT05775848) can be found here on the
ClinicalTrials.gov website.
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 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. Patients who harbor a L276I homozygous 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). Patients with other
(non-L276I homozygous) genotypes 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 and
cancers with clear genetic drivers. 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
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,” “estimates,” “expects,” “intends,”
“may,” “plans,” “projects,” “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 potential of BridgeBio’s
programs and product candidates, including BBP-418 for the
treatment of LGMD2I, the potential benefits of BBP-418, including
that the early assessment of increased glycosylated ⍺DG levels at 3
months predicted subsequent ambulatory improvements at 9 months,
supporting the use of glycosylated ⍺DG levels as a potential
surrogate endpoint in LGDM2I/R9 and that BBP-418 continues to be
well-tolerated with longer-term treatment , the progress of
BridgeBio’s ongoing and planned clinical trials of BBP-418,
including the continued enrollment of the Phase 3 study in the U.S.
with clinical trial sites planned for Europe, UK, and Australia,
the potential and the opportunity to pursue Accelerated Approval
Pathway for BBP-418 in LGMD2I/R9 in the U.S. based on recent
interactions with the FDA on the use of glycosylated αDG levels as
a surrogate endpoint, the statement regarding the potential benefit
of FORTIFY trial for patients with LGMD2I/R9 and of BBP-418 in the
quotes of Dr. Harper and Dr. Sproule, the expectation that the
results of the evaluation of secondary endpoints at 36 months will
provide confirmatory clinical data, and the timing and success of
BridgeBio’s clinical trials and development pipeline, 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, initial and ongoing data from clinical trials not being
indicative of final data, the design and success of BridgeBio’s
ongoing and planned 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, as well as those risks set forth in the Risk Factors
section of BridgeBio’s Annual Report on Form 10-K for the year
ended December 31, 2022, and BridgeBio’s other SEC filings.
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 Media Contact:Vikram
Balicontact@bridgebio.com (650)-789-8220
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