BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the
“Company”), a commercial-stage biopharmaceutical company focused on
genetic diseases, today announced topline results from the Phase
1/2 open-label ADventure study investigating BBP-631, the Company’s
investigational adeno-associated virus (AAV) 5 gene therapy, for
the treatment of congenital adrenal hyperplasia (CAH).
The Phase 1/2 open-label ADventure study was designed to
evaluate the safety, tolerability and pharmacodynamic activity of
BBP-631 in adults with classic CAH. To date, key results from the
study include:
- Increased endogenous cortisol production was achieved in all
patients at higher doses. A maximum change from baseline post-ACTH
stimulation test of 4.7 μg/dL and 6.6 μg/dL was observed at the two
highest dose levels, respectively, with cortisol levels as high as
11 μg/dL achieved.
- Substantial and durable increases in 11-deoxycortisol, the
product of 21-hydroxylase, and reductions in 17-hydroxyprogesterone
(17-OHP), the substrate of 21-hydroxylase, provide compelling
evidence of durable BBP-631 transgene activity.
- At the highest dose levels, sustained 11-deoxycortisol averaged
a 55-fold increase from baseline with a maximum of 99-fold increase
from baseline. These represent an average maximum of 23-fold the
upper-limit of normal.
- Robust reduction in 17-hydroxyprogesterone, with the majority
of patients reaching a reduction of ≥50%, with a max reduction of
95%.
- BBP-631 has been well tolerated with only mild to moderate
treatment-emergent adverse events (TEAEs) and no treatment-related
SAEs reported.
“While the data to date are not yet transformational, the study
showed for the first time that people living with CAH can indeed
make their own cortisol, and that gene therapy can be safely
administered in this patient population. We remain committed to
finding the right partner for those in the CAH community and are
grateful to the participants and those who expressed interest in
both the pre-screening study and the ADventure study. We also want
to thank the ADventure study investigators and staff, the CAH
patient advocacy organizations and the broader CAH community,” said
Neil Kumar, Ph.D., CEO and Founder of BridgeBio.
“Given that the results of the trial did not meet the threshold
to warrant additional capital investment at this time, BridgeBio
will be reducing the gene therapy budget more than $50M, consistent
with our capital allocation principles, and reserving gene therapy
for priority targets that we cannot treat any other way,” said
Brian Stephenson, Ph.D., CFA, Chief Financial Officer of BridgeBio.
“We believe that gene therapies have the potential to fulfill a
significant unmet need and are eager to work closely with the FDA
and the Canavan community with the goal of bringing our therapy to
families living with Canavan disease as fast as possible.”
BridgeBio will no longer be pursuing development of BBP-631 for
CAH and the Company is actively seeking partnership opportunities
to support future development of BBP-631 or next-generation gene
therapies for the treatment of CAH, a very prevalent genetic
disease that still has significant unmet need, with more than
75,000 cases estimated in the United States and European Union.
About BBP-631BBP-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 Congenital Adrenal Hyperplasia
(CAH)Affecting approximately 75,000 people in the United
States and European Union, CAH is a group of genetic disorders that
affect the adrenal glands, which is caused by a mutation in the
gene encoding for 21-hydroxylase, an enzyme essential for making
the hormones cortisol and aldosterone which are critical for
various physiologic functions. Cortisol is necessary for the body
to respond to injury, stress or illness, and aldosterone is
required to maintain proper blood pressure and sodium levels.
Unable to produce cortisol and aldosterone, people with classic CAH
cannot mount the healthy physiological response to stressors, such
as illnesses, that allows their heart, lungs, kidneys and other
organs to compensate for the stress, which can be life-threatening.
These adrenal crises can be particularly dangerous for young
children.
About BridgeBio Pharma, Inc.BridgeBio Pharma,
Inc. (BridgeBio) is a commercial-stage biopharmaceutical company
founded to discover, create, test and deliver transformative
medicines to treat patients who suffer from genetic diseases.
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, Twitter
and Facebook.
BridgeBio Pharma, Inc. Forward-Looking
StatementsThis 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 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,” “continues,” “estimates,” “expects,”
“hopes,” “intends,” “may,” “plans,” “projects,” “remains,” “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 in Dr. Kumar’s quote relating to the
expectations, plans and prospects regarding BBP-631, the statements
in Dr. Stephenson’s quote relating to the Company’s financial
performance, capitalization status, strategy, business plans and
goals and the potential for gene therapy and future partnership
opportunities for BBP-631, reflect our current views about our
plans, intentions, expectations, strategies and prospects, which
are based on the information currently available to us and on
assumptions we have 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, risks inherent in developing
therapeutic products, the success, cost, and timing of the
Company’s product candidate research and development activities and
ongoing and planned preclinical studies and clinical trials, the
success and timing of preclinical study and clinical trial results,
the success of its clinical trial designs, the fact that successful
preliminary preclinical study or clinical trial results may not
result in future clinical trial successes and/or product approvals,
trends in the industry, the legal and regulatory framework for the
industry, the accuracy of the Company’s estimates regarding
expenses, future revenue, future expenditures and needs for and
ability to obtain additional financing, the Company’s ability to be
a sustainable genetic medicine innovation engine and to build the
next great genetic medicine company, the Company’s ability to
obtain and maintain intellectual property protection for its
product candidates and approved products, the competitive
environment and clinical and therapeutic potential of the Company’s
product candidates and FDA-approved products, potential adverse
impacts due to global health emergencies, including 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 hostilities in Ukraine
and in Israel and the Gaza Strip, increasing rates of inflation and
rising interest rates, on our business operations and expectations
as well as those risks set forth in the Risk Factors section of
BridgeBio’s most recent Annual Report on Form 10-K, and BridgeBio’s
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, 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 Pharma (NASDAQ:BBIO)
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