BridgeBio Pharma and Kyowa Kirin Announce Partnership with an Upfront Payment of $100 Million for an Exclusive License on Infigratinib in Skeletal Dysplasias in Japan
– BridgeBio grants Kyowa Kirin exclusive license to develop and commercialize infigratinib for skeletal dysplasias in Japan
– BridgeBio to receive upfront payment of USD 100 million with royalties up to the high-twenties percent, with additional milestone-based payments
– Infigratinib is a once daily oral treatment option under development for achondroplasia, which was well-tolerated and demonstrated potentially best-in-class efficacy in PROPEL 2, a Phase 2 study in children with achondroplasia
– PROPEL 3, a Phase 3 study of infigratinib in achondroplasia is underway globally outside of Japan with the first child dosed in December 2023
– Infigratinib adds to Kyowa Kirin’s successful portfolio in the therapeutic areas of bone & mineral diseases
PALO ALTO, Calif. and TOKYO, Feb. 07, 2024 (GLOBE NEWSWIRE) — 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 Achondroplasia
Achondroplasia 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 bridgebiodev.wpengine.com and follow us on LinkedIn and Twitter.
About Kyowa Kirin
Kyowa 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 Bali
[email protected]
(650)-789-8220