BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio), a
commercial-stage biopharmaceutical company focused on genetic
diseases, today announced sustained positive results from PROPEL 2,
a Phase 2 trial of the investigational therapy infigratinib in
children with achondroplasia, demonstrating continued potential
best-in-class efficacy and an encouraging safety profile.
Infigratinib is an oral small molecule designed to inhibit FGFR3
signaling and target achondroplasia and hypochondroplasia at their
source. BridgeBio will also host an investor call on June 4,
2024, at 8:00 am ET with Ravi Savarirayan, M.D., Ph.D., of Murdoch
Children’s Research Institute in Melbourne, Australia, and the
global lead investigator for PROPEL 2, to discuss the results from
the Phase 2 study.
To date, key results from the Cohort 5 dose escalation cohort in
PROPEL 2 trial include:
- Sustained and statistically significant mean increase in AHV of
+2.51cm/year from baseline at 12 months, and +2.50 cm/yr at 18
months (p=0.0015)
- Statistically significant improvement in body proportionality
(mean upper to lower body segment ratio), from 2.02 at baseline to
1.88 at Month 18 (mean change from baseline, p=0.001)
- A continued well-tolerated safety profile, with no
treatment-related adverse events assessed as related to
infigratinib
“These data indicate that treatment with infigratinib is
continuing to show increased growth velocity and improvements in
body proportionality in children with achondroplasia. This is
encouraging and suggests that infigratinib has the potential to
enhance functionality for people living with achondroplasia in
addition to increasing growth. We hope to see these improvements
reflected in the ongoing PROPEL 3 pivotal study that will build
toward providing a safe and effective oral therapy to those in the
achondroplasia community who are seeking treatment,” said Dr.
Savarirayan.
PROPEL 3, the global Phase 3 registrational study of
infigratinib in achondroplasia, continues to enroll on schedule,
with completion of enrollment anticipated by the end of the
year.
Given the promising results from PROPEL 2, BridgeBio is
committed to expanding the FGFR3-related skeletal dysplasias
franchise for infigratinib by accelerating development in
hypochondroplasia. Positive interactions with both the U.S. FDA and
EMA support development in children with hypochondroplasia, with a
small open-label Phase 2 portion testing a single dose of 0.25
mg/kg/day, leading into a double-blinded, placebo-controlled Phase
3 study. ACCEL, the observational lead-in program for
hypochondroplasia, was initiated with the first participant
consented in May 2024. The interventional program, ACCEL 2/3, will
be a global Phase 2/3 multicenter, single-dose study, to evaluate
the efficacy and safety of 0.25mg/kg/day of infigratinib in
children living with hypochondroplasia. The open-label Phase 2
portion in children aged 5 to 11 years old will be followed by a
pivotal Phase 3, one-year, 2:1 randomized, double-blinded,
placebo-controlled study in children aged 3 to < 18 years old
with growth potential. In addition to changes from baseline in AHV
measurements, the study will evaluate changes in other indicators
of growth, body proportions, medical complications associated with
hypochondroplasia, and changes in quality-of-life measures.
BridgeBio has previously presented promising preclinical data for
hypochondroplasia at ENDO 2023 and ASHG 2022.
“We are very excited to see a persistence of response to
infigratinib in linear growth. We are especially encouraged by the
promising effect on body proportions, which supports infigratinib’s
potential to provide benefits that could impact the lives of
children with achondroplasia. These results motivate us to continue
evaluating infigratinib in other FGFR-related skeletal dysplasias
and genetic conditions. The initiation of our observational study
in hypochondroplasia and the obtainment of FDA and EMA alignment on
the interventional study underlie our excitement for the potential
of infigratinib as a treatment option for children with
hypochondroplasia,” said Daniela Rogoff, M.D., Ph.D., Chief Medical
Officer, Skeletal Dysplasias at BridgeBio.
“The journey of living with skeletal dysplasia varies from
person to person, but many are impacted by functional limitations,
social stigma and medical complications due to their condition and
the way their bones develop. We are encouraged to see
infigratinib’s potential to improve body proportionality, which
could help address functional complications meaningful to people
living with skeletal dysplasia. The Chandler Project values the
collaborative partnership we’ve developed with BridgeBio and QED,
to ensure that the community’s true needs are prioritized
throughout the discovery and development process. We are also
thrilled for the launch of an observational study in
hypochondroplasia, a community that has been eager for further
research and development of treatment options,” said Chandler
Crews, founder of The Chandler Project, a patient advocacy
organization based in Baltimore, MD.
Information about PROPEL 3 (NCT06164951) can be found
here on clinicaltrials.gov. Information about
PROPEL (NCT04035811), BridgeBio’s observational lead-in study in
achondroplasia for PROPEL 3 and other studies, can be found
here on clinicaltrials.gov. Information about
ACCEL (NCT06410976), BridgeBio’s observational lead-in study in
hypochondroplasia can be found here on
clinicaltrials.gov. BridgeBio is committed to exploring the
potential of infigratinib on wider medical and functional impacts
of achondroplasia, hypochondroplasia and other skeletal dysplasias,
which hold significant unmet needs for families.
Webcast InformationBridgeBio will host an
investor call and simultaneous webcast to discuss the Phase 2 data
at Months 12 and 18 of infigratinib in children with achondroplasia
on June 4, 2024 at 8:00 am ET. A link to the webcast may be
accessed from the event calendar page of BridgeBio’s website at
https://investor.bridgebio.com/. A replay of the conference call
and webcast will be archived on the Company’s website and will be
available for at least 30 days following the event.
About AchondroplasiaAchondroplasia is the most
common cause of disproportionate short stature, affecting
approximately 55,000 people in the United States (US) and European
Union (EU), including up to 10,000 children and adolescents with
open growth plates. Achondroplasia impacts overall health and
quality of life, leading to medical complications such as
obstructive sleep apnea, middle ear dysfunction, kyphosis, and
spinal stenosis. The condition is uniformly caused by an activating
variant in FGFR3.
About HypochondroplasiaHypochondroplasia is
also an FGFR3-associated skeletal dysplasia and is a rare condition
with similar prevalence in achondroplasia. Hypochondroplasia
presents with a wide spectrum of phenotypes including
disproportionate short stature, mild joint laxity and macrocephaly.
Currently, no treatments for hypochondroplasia are approved in the
United States.
About BridgeBio Pharma, Inc.BridgeBio Pharma
(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 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. We intend 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, therapeutic and
market potential of our programs and product candidates, including
our clinical development program for infigratinib in
achondroplasia, the timing and success of our clinical development
programs, the progress of our ongoing and planned clinical trials
of infigratinib in achondroplasia and in hypochondroplasia , our
planned interactions with regulatory authorities, the availability
of data from our clinical trials of infigratinib, and the timing of
these events, reflect our current views about our plans,
intentions, expectations and strategies, which are based on the
information currently available to us and on assumptions we have
made. Although we believe that our plans, intentions, expectations
and strategies as reflected in or suggested by those
forward-looking statements are reasonable, we 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, initial and ongoing
data from our 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 our 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 our most recent Annual
Report on Form 10-K and our other filings with the U.S. Securities
and Exchange Commission. Moreover, we operate 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 our 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
Medical Information
Contact:MedInfo@QEDTx.com1-877-280-5655
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