BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio), a
commercial-stage biopharmaceutical company focused on genetic
diseases and cancers, today announced positive results from
PROPEL2, a Phase 2 trial of the investigational therapy
infigratinib in children with achondroplasia, demonstrating
potential best-in-class efficacy and a clean safety profile.
Infigratinib is an oral small molecule designed to inhibit FGFR3
and target achondroplasia at its source. BridgeBio will also host
an investor call on March 6, 2023, at 7:30 am ET to discuss the
results from the Phase 2 study.
To date, key results from the clinical trial include:
- At the highest dose level evaluated to date (Cohort 5, 0.25
mg/kg once daily), the mean increase from baseline in annualized
height velocity (AHV) for the 10 children that have had six-month
visits was +3.03 cm/yr (p = 0.0022). Individual data can be found
in Figure 1 below
- The baseline AHV for the 10 children with six-month visits was
in the expected range for children with achondroplasia at 3.73
cm/yr, rising to 6.77 cm/yr after treatment
- The two remaining children who have not yet had six months of
follow-up have a mean change from baseline in AHV of +8.8 cm/yr at
three months. The mean age for the cohort was 7.24 years
- 80% of the 10 children with six-month visits were responders,
with a change from baseline AHV of at least 25%. Among the
responders, the average change from baseline in AHV was +3.81
cm/yr
- Preliminary analysis of Collagen X (CXM) levels also saw a
statistically significant increase from baseline in Cohort 5
(p=.03). CXM is the gold-standard biomarker of chondrocyte-driven
growth and further validates the robust response to
infigratinib
- Combined with the previously reported Cohort 4 change from
baseline in AHV value of +1.52 cm/yr, the Cohort 5 data demonstrate
a strong dose response for infigratinib
- Median follow-up across all cohorts is 71.1 weeks. To date, the
study has shown a well-tolerated safety profile, with no study drug
related treatment emergent adverse events (TEAEs) in Cohort 5. No
serious adverse events (SAEs) or discontinuations due to AEs were
reported in any cohort
Figure 1
“The data from Cohort 5 has shown a major impact on annualized
height velocity for children with achondroplasia and an excellent
safety profile to date. We are thrilled to see these promising
results and consider that AHV increases of this magnitude will
translate to improvements in the medical and functional
complications of achondroplasia. We are excited about taking the
next steps towards initiating a Phase 3, pivotal clinical trial,”
said Professor Ravi Savarirayan, M.D., Ph.D., clinical geneticist
and group leader of molecular therapies research at the Murdoch
Children’s Research Institute in Australia, the lead investigator
for PROPEL2.
“I am encouraged by these efficacy and safety results and
thankful for our partnership with the physicians, community
advocates, children, and families in this study. These results
reach a new tier of efficacy, and coupled with our differentiated
safety and convenience profile, provide us the opportunity to serve
children with achondroplasia and other skeletal dysplasias. We look
forward to exploring the potential of infigratinib on the wider
medical and functional impacts of achondroplasia, hypochondroplasia
and other skeletal dysplasias, which hold significant unmet needs
for families,” said Neil Kumar, Ph.D., founder and CEO of
BridgeBio.
Based on the positive results to date, BridgeBio has started
enrolling children in the run-in for a Phase 3 trial. Additionally,
BridgeBio expects to initiate clinical development for infigratinib
in hypochondroplasia, a skeletal dysplasia closely related to
achondroplasia and similarly driven by FGFR3 gain-of-function
variants. BridgeBio has previously presented promising preclinical
data for hypochondroplasia at ENDO 2022 and ASHG 2022.
“Achondroplasia can have broad impact that affects the whole
person. People can experience a range of medical complications,
including foramen magnum stenosis, spinal stenosis, cardiovascular
complications, sleep-disordered breathing, obesity, and sometimes,
individuals may need surgical intervention. In addition to the
potential medical and physical complications, people with
achondroplasia may also experience social and emotional impacts as
a result of living with the condition. We are encouraged by
BridgeBio’s mission to develop a therapy with the potential to
address this as a whole-person condition that affects the overall
health, independent function, and quality of life of those with
achondroplasia,” said Dianne Kremidas, executive director of The
MAGIC Foundation.
Infigratinib has IP protection out to at least 2041.
Webcast InformationBridgeBio will host an
investor call and simultaneous webcast to discuss the Phase 2 data
from Cohort 5 of infigratinib in children with achondroplasia on
March 6, 2023 at 7:30 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
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
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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,” “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 our plans to initiate a Phase 3 trial for infigratinib in
achondroplasia and to initiate clinical development 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 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 Contact:Vikram
Balicontact@bridgebio.com(650)-789-8220
BridgeBio Skeletal Dysplasia Advocacy
Contact:Anne LeeAnne.Lee@bridgebio.com(415)-694-0979
A photo accompanying this announcement is available at
https://www.globenewswire.com/NewsRoom/AttachmentNg/a43bfd09-35ca-4f7a-925f-4e5b114b7837
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