BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio) and Kyowa Kirin
Co., Ltd (TSE:4151) (Kyowa Kirin) today announced a partnership
wherein BridgeBio’s affiliate, QED Therapeutics, grants Kyowa Kirin
an exclusive license to develop and commercialize infigratinib for
achondroplasia, hypochondroplasia, and other skeletal dysplasias in
Japan. In exchange, BridgeBio will receive an upfront payment
of USD 100 million as well as royalties up to the high-twenties
percent on sales of infigratinib in Japan, with the potential for
additional milestone-based payments. Infigratinib is an oral small
molecule designed to inhibit FGFR3 and thus target FGFR3-driven
skeletal dysplasias at their source, including achondroplasia and
hypochondroplasia.
“We are pleased to partner with Kyowa Kirin given their
community-oriented core mission to bring smiles to faces by
providing innovative medicines with life-changing value, which has
seen success with their commercialization of Crysvita and other
rare disease drugs. The BridgeBio vision is to help bring people
access to novel treatments and trials, and so we are pleased that
this collaboration will ensure the development of infigratinib for
children living with achondroplasia, and eventually for other
skeletal dysplasias in Japan,” said Neil Kumar, Ph.D., BridgeBio’s
CEO and founder.
“By partnering with Kyowa Kirin, we hope to significantly
accelerate the development of infigratinib to potentially provide
options for children with achondroplasia, hypochondroplasia, and
eventually skeletal dysplasias in Japan. We hear the need from the
community for a once-daily oral treatment option and are looking
forward to initiating our trials in Japan at a later date,” said
Justin To, CEO of QED Therapeutics, BridgeBio’s affiliate for
skeletal dysplasias.
“We are thrilled to announce that we have signed the License
Agreement with BridgeBio,” said Yasuo Fujii, MBA., Chief Strategy
Officer, Managing Executive Officer, Vice President, Head of
Strategy Division at Kyowa Kirin. “It is important for us to
strengthen our portfolio by introducing pipelines in the fields of
bone & mineral disorders, including achondroplasia. Based on
the results from the latest clinical trials, we believe BridgeBio’s
infigratinib has high potential for treating achondroplasia. We
will steadily advance the development in Japan and aim to deliver
life-changing value to people with skeletal dysplasias including
achondroplasia.”
In December 2023, BridgeBio dosed the first child in PROPEL 3, a
one-year, 2:1 randomized, placebo-controlled Phase 3 pivotal trial,
evaluating the efficacy and safety of infigratinib in children with
achondroplasia aged 3 to <18 years with open growth plates. The
Phase 3 builds on the success of PROPEL 2, a Phase 2 trial of
infigratinib in achondroplasia which demonstrated a +3.38cm/yr
increase in annualized height velocity, the strongest clinical
result published to date. In Japan, Kyowa Kirin will start to
discuss with Pharmaceuticals and Medical Devices Agency (PMDA) of
Japan in 2024, with the aim of initiating a Japanese registrational
trial in 2025.
“As the father of a child with achondroplasia, I am keenly aware
of the health and mental health issues of people affected by the
physical characteristics of living with achondroplasia. GLORY
TO ACHONDROPLASIA is deeply grateful for the time spent by
researchers and practitioners who support progress in medicine and
society, as well as by people affected by the condition and their
families. When considering the future of children with
achondroplasia, we as a support organization believe it is our
absolute responsibility to improve treatment options and social
recognition. We are pleased for BridgeBio to partner
with Kyowa Kirin and look forward to collaborating on our
future responsibilities,” said Eihaku Itooka, founder and president
of GLORY TO ACHONDROPLASIA, a Japanese bone dysplasia patient
advocacy organization.
Information about PROPEL 3 (NCT06164951) 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.
About AchondroplasiaAchondroplasia is the most
common cause of disproportionate short stature, affecting
approximately 55,000 people in the U.S. and EU, and 6,000 in Japan.
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.
About Kyowa KirinKyowa Kirin aims to discover
novel medicines with life-changing value. As a Japan-based Global
Specialty Pharmaceutical Company, we have invested in drug
discovery and biotechnology innovation for more than 70 years and
are currently working to engineer the next generation
of antibodies and cell and gene therapies with the
potential to help patients affected by a severe or rare
disease. A shared commitment to our values, to sustainable growth,
and to making people smile unites us across our four regions –
Japan, Asia Pacific, North America, and EMEA/International. You can
learn more about the business of Kyowa Kirin at:
https://www.kyowakirin.com.
BridgeBio Pharma, Inc. Forward-looking
Statements This 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
expectations, plans, and prospects regarding the success of our
exclusive license agreement with Kyowa Kirin to develop and
commercialize infigratinib for achondroplasia, hypochondroplasia,
and other skeletal dysplasias in Japan; BridgeBio’s eligibility to
receive an upfront payment, future royalty payments, and
potentially additional milestone-based payments under the license
agreement; the clinical, therapeutic and market potential of our
clinical development program for infigratinib in achondroplasia;
the progress of the clinical trials of infigratinib in
achondroplasia and hypochondroplasia, including the continuation of
PROPEL 3, our Phase 3 pivotal trial evaluating the efficacy and
safety of infigratinib in children with achondroplasia aged 3 to
<18 years with open growth plates, the expectation of Kyowa
Kirin’s starting discussions with PMDA of Japan in 2024, with the
aim of initiating a Japanese Phase 3 trial in 2025, and the
expectation of our initiation of ACCEL, an observational lead-in
study for infigratinib in hypochondroplasia, in the first half of
2024; the statements regarding the expectations for the partnership
between BridgeBio and Kyowa Kirin and the potential benefits of
infigratinib for achondroplasia, hypochondroplasia, and other
skeletal dysplasias in Japan, including such statements in the
quotes of Dr. Kumar, Mr. To, Mr. Fujii and Mr. Itooka, 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, the success of our license agreement with Kyowa Kirin,
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 the Middle East and 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
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