BridgeBio and Resilience Announce Strategic Multi-Year Partnership to Advance BBP-631, BBP-812 and Future Gene Therapy Treatments
- Resilience to manufacture BridgeBio’s lead investigational gene therapy treatments, BBP-631 and BBP-812
- Partnership provides an innovative, capital efficient, sustainable model for BridgeBio to develop, test and deliver transformative medicines for patients more quickly
PALO ALTO, Calif. and SAN DIEGO, Oct. 03, 2023 (GLOBE NEWSWIRE) — BridgeBio Pharma, Inc. (BridgeBio), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, and National Resilience, Inc. (Resilience), a technology-focused biomanufacturing company dedicated to broadening access to complex medicines, today announced a strategic collaboration to manufacture and advance BBP-812, an investigational adeno-associated virus (AAV) 9 gene therapy for Canavan disease, and BBP-631, an investigational AAV 5 gene therapy for congenital adrenal hyperplasia (CAH).
The two companies have developed a novel manufacturing and aligned incentive business model to drive these gene therapies forward with an emphasis on sustainability and capital-efficiency. Under the terms of the collaboration, BridgeBio will transfer its manufacturing process for its lead AAV-based gene therapy candidates to Resilience’s network of gene therapy sites. As part of an innovative cost and risk-sharing framework, Resilience will provide in-kind manufacturing services and will receive future development and approval milestones and low-to-mid single digit royalties on BBP-631 and BBP-812. Resilience will support the ongoing clinical development manufacturing needs and will serve as the primary commercial manufacturer for both programs if successful.
“Our partnership with BridgeBio seeks to accelerate development of innovative therapeutic options for patients in need,” said Rahul Singhvi, Sc.D., Chief Executive Officer of Resilience. “We are pleased to partner with a gene therapy and rare disease leader, and we are inspired by their passion to deliver medicines to patients.”
Beyond BBP-812 and BBP-631, Resilience will also be the primary manufacturer for future clinical projects across BridgeBio’s gene therapy portfolio. The agreement will reduce manufacturing uncertainty for these programs and is expected to help BridgeBio expedite development of gene therapies going forward.
“Manufacturing is the most critical and costly aspect of developing gene therapy for patients with a serious unmet need. We conduct process development, analytical development, and optimization in our own labs, and with this partnership, we can now hand these programs off to one of the most trusted partners in the industry for scale up and commercial manufacturing. This allows us to accelerate the development of our gene therapy portfolio in a capital-efficient and sustainable way with the hope of providing medicines more quickly,” said Eric David, M.D., J.D., chief executive officer of BridgeBio Gene Therapy.
“We appreciate the opportunity to collaborate with the experienced and knowledgeable team at Resilience on the manufacturing of our gene therapies. We hope this expedites our path to benefiting as many patients as possible, as soon as possible,” said Neil Kumar, Ph.D., founder and CEO of BridgeBio.
About BridgeBio Pharma, Inc.
BridgeBio Pharma, Inc. 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 BBP-812
BBP-812 is an investigational AAV9 gene therapy for Canavan
disease. Using AAV gene therapy, BridgeBio seeks to deliver
functional copies of the ASPA gene throughout the body and into
the brain, potentially correcting the disease at its source.
Preclinical proof-of-concept results have shown the approach
restores survival and normal motor function in Canavan disease
models. BBP-812 was granted Fast Track Designation, Rare
Pediatric Drug Designation, and Orphan Drug Designation by the
U.S. Food and Drug Administration. BBP-812 was also granted
Orphan Drug Designation by the European Medicines Agency.
About BBP-631
BBP-631 is an AAV5 gene therapy developed to treat CAH due to
21-hydroxylase deficiency at its source. BBP-631 is designed to
deliver a functional copy of the 21-hydroxylase gene and has
been shown through multiple preclinical studies to result in
efficient and persistent delivery to the adrenal gland, where
hormones are naturally made. If successful, BBP-631 may restore
the body’s hormone and steroid balance by enabling people with
CAH to naturally make their own cortisol and aldosterone. It
could also allow for people with CAH to eliminate or
significantly reduce their daily glucocorticoid or
mineralocorticoid doses, which is the current standard of care
for patients.
About Resilience
Resilience is a technology-focused biomanufacturing company
dedicated to broadening access to complex medicines. Founded in
2020, the company is building a sustainable network of
high-tech, end-to-end manufacturing solutions to ensure the
treatments of today and tomorrow can be made quickly, safely,
and at scale. By continuously advancing the science of
biopharmaceutical manufacturing and development, Resilience
seeks to free its partners to focus on the discoveries that
improve patients’ lives and protect biopharmaceutical supply
chains against future disruptions. For more information,
visit https://resilience.com/ and follow us on social media: @IncResilience on Twitter and Resilience on LinkedIn.
BridgeBio Pharma, Inc. Forward-Looking Statements
This press release contains forward-looking statements.
Statements BridgeBio makes in this press release may include
statements that are not historical facts and are considered
forward-looking within the meaning of the Private Securities
Litigation Reform Act of 1995, as amended, 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.
BridgeBio intends 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 BridgeBio’s collaboration agreement with
Resilience and the milestone and royalty payments thereunder,
the ability of BridgeBio and Resilience’s model to drive
BridgeBio’s gene therapies forward in a capital-efficient and
sustainable way, the success and impact of the collaboration
with Resilience, including on reducing manufacturing uncertainty
and expediting development of BridgeBio’s gene therapy
portfolio; and the potential of our related gene therapy
programs, if successful, to benefit patients with CAH or Canavan
disease, reflect our current views about our plans, intentions,
expectations and strategies, which are based on the information
currently available to us and on assumptions BridgeBio has made.
Although BridgeBio believes that its plans, intentions,
expectations, strategies and prospects as reflected in or
suggested by those forward-looking statements are reasonable,
BridgeBio 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, BridgeBio’s ability to continue and complete
its ongoing and planned clinical trials of BBP-631 for the
treatment of CAH disease and of BBP-812 for the treatment of
Canavan disease, initial and ongoing data from clinical trials
not being indicative of final data; the design and success of
BridgeBio’s ongoing and planned clinical trials, difficulties
with enrollment in BridgeBio’s clinical trials, adverse events
that may be encountered in BridgeBio’s clinical trials, the FDA
or other regulatory agencies not agreeing with BridgeBio’s
regulatory approval strategies, components of BridgeBio’s
filings, such as clinical trial designs, conduct and
methodologies, or the sufficiency of data submitted, as well as
those risks set forth in the Risk Factors section of BridgeBio’s
Annual Report on Form 10-K for the year ended December 31, 2022
and its other filings with the U.S Securities and Exchange
Commission. Moreover, BridgeBio operates 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 BridgeBio’s 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, BridgeBio
assumes no obligation to update publicly any forward-looking
statements, whether as a result of new information, future
events or otherwise. No representations or warranties (expressed
or implied) are made about the accuracy of any such
forward-looking statements.
Media Contacts:
BridgeBio
Vikram Bali
[email protected]
(650) 789-8220
Resilience
Michele Roberts
Head of Communications
[email protected]
(888) 737-2460
Tatjana Heller
Head of Investor Relations and Analytics
[email protected]
(888) 737-2460