BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio), a
commercial-stage biopharmaceutical company focused on genetic
diseases and cancers, today announced that the first child has been
dosed in PROPEL 3, a Phase 3 clinical trial studying the efficacy
and safety of infigratinib in children with achondroplasia. Both
the U.S. FDA and the EU EMA indicated the trial design for PROPEL 3
would be acceptable as a registrational study to support a
marketing application for the treatment of children with
achondroplasia.
“The Phase 2 data for infigratinib has been very promising and
suggests potential to increase growth, improve functionality and
reduce the associated medical complications in children with
achondroplasia. With the recent dosing of the first child in the
Phase 3 trial, I am hopeful that we are one step closer to
providing a safe, effective oral therapy to the people in the
achondroplasia community who are seeking treatment,” said Dr. Ravi
Savarirayan, M.D., Ph.D., clinical geneticist and leader of the
molecular therapies research group at the Murdoch Children’s
Research Institute in Melbourne, Australia and the global lead
investigator for PROPEL 3.
PROPEL 3 is a global, one-year, 2:1 randomized, double-blinded
placebo-controlled clinical trial, which will evaluate the efficacy
and safety of infigratinib in children with achondroplasia aged 3
to <18 years with open growth plates. The primary endpoint will
be change from baseline in AHV, with secondary endpoints including
proportionality, height Z-score (a measure of variance and
dispersion from the average height of children with
achondroplasia), and impact on medical complications and quality of
life.
“We are thrilled about this major milestone for our program and
are hopeful about what this means for those families seeking a
therapeutic option. We are grateful to the children and their
families for participating in our Phase 3 trial as well as the open
collaboration we have built with physicians and advocacy groups to
best understand the needs of those living with this condition. We
look forward to continuing to explore the benefits of infigratinib
not only in growth, but in the functional improvements and the
alleviation of medical complications that are most meaningful to
the community,” said Daniela Rogoff, M.D., Ph.D., senior vice
president of clinical development for skeletal dysplasias at
BridgeBio.
In June 2023, the Company presented updated six-month results
from Cohort 5 of PROPEL 2, its Phase 2 dose-finding study, at ENDO
2023. Cohort 5 demonstrated that the dose level of 0.25 mg/kg/day
resulted in a significant and robust increase in AHV, with a mean
change of +3.38 cm/year from baseline at six months. The findings
also highlighted early but promising trends towards improvement in
proportionality, as measured by the upper and lower body segment
ratio. Additionally, the results showed a well-tolerated safety
profile, with no study drug related treatment emergent adverse
events, serious adverse events or discontinuations due to adverse
events at the 0.25 mg/kg dose.
“Achondroplasia can impact the overall health and wellbeing of a
person, including functional limitations, social stigma and medical
complications. We appreciate the opportunity to work with BridgeBio
to ensure the needs of our community are heard, and we are excited
by what the Phase 3 study could bring for those looking for options
to help themselves or their loved ones,” said Susana Noval,
director of Fundación ALPE Acondroplasia. Fundación ALPE
Acondroplasia, based in Gijón, Spain, is an advocacy organization
for people with achondroplasia and other skeletal dysplasias and
their families.
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. Additionally, BridgeBio expects to initiate
ACCEL, an observational lead-in study for infigratinib in
hypochondroplasia, a skeletal dysplasia closely related to
achondroplasia and similarly driven by FGFR3 gain-of-function
variants, in the first half of 2024. BridgeBio has previously
presented promising preclinical data for hypochondroplasia at ENDO
2023 and ASHG 2022. 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.
About AchondroplasiaAchondroplasia is the most
common cause of disproportionate short stature, affecting
approximately 55,000 people in the U.S. and 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 mutation in FGFR3.
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 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 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,” “estimates,” “expects,”
“intends,” “may,” “plans,” “projects,” “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, including the design of PROPEL3, including the
expectation that PROPEL3 will evaluate the safety and efficacy of
infigratinib in children with achondroplasia aged 3 to 18 years
with open growth plates, the primary endpoint and the secondary
endpoints of such trial; the expectations of our interactions with
regulatory authorities, including the indications by both the U.S.
FDA and the EU EMA that the trial design for PROPEL 3 would be
acceptable as a registrational study to support a marketing
application for the treatment of children with achondroplasia; the
statements regarding the potential benefits of infigratinib and the
benefits of the Phase 3 study, including such statements in the
quotes of Dr. Savarirayan, Dr. Rogoff and Ms. Noval; our
exploration of the potential of infigratinib on wider medical and
functional impacts of achondroplasia, hypochondroplasia and other
skeletal dysplasias; and the expectation to begin development of
infigratinib in hypochondroplasia, with the initiation of a
observational lead-in study in the first half of 2024, 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 the global COVID-19
pandemic such as 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 the COVID-19 pandemic, hostilities in Ukraine,
increasing rates of inflation and rising interest rates, on our
overall business operations and expectations, as well as those
risks set forth in the Risk Factors section of our Annual Report on
Form 10-K for the year ended December 31, 2022 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 Media Contact:Vikram
Balicontact@bridgebio.com(650)-789-8220
BridgeBio Pharma (NASDAQ:BBIO)
Gráfica de Acción Histórica
De Abr 2024 a May 2024
BridgeBio Pharma (NASDAQ:BBIO)
Gráfica de Acción Histórica
De May 2023 a May 2024