BridgeBio Pharma Shares Positive Results of Single-Arm Phase 3 Study of Acoramidis in Japanese Patients with Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Including No Mortality Reported in the Trial at 30 Months
– Phase 3 open-label, single-arm study conducted in Japan by BridgeBio licensing partner Alexion, AstraZeneca Rare Disease showed consistency with global ATTRibute-CM Phase III trial
– No mortality was reported over the 30 month acoramidis treatment period
– Acoramidis was well-tolerated, with no safety signals of potential clinical concern identified
– These results support local regulatory submission in Japan
– The findings from this study build upon positive results from BridgeBio’s global ATTRibute-CM Phase 3 trial, where the primary endpoint was met (Win Ratio of 1.8) with a highly statistically significant p-value (p<0.0001) and an 81% survival rate at 30 months and a 0.29 annualized cardiovascular hospitalization rate were observed on acoramidis treatment
PALO ALTO, Calif., Feb. 02, 2024 (GLOBE NEWSWIRE) — BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today shared positive results from the Japan Phase III trial of acoramidis in adults with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), conducted by Alexion, AstraZeneca Rare Disease. Results showed consistency to those in the global BridgeBio ATTRibute-CM Phase III trial (NCT03860935), including survival, cardiovascular-related hospitalizations and other measures of improved functions (measured by six-minute walk test) and quality of life (measured by the Kansas City Cardiomyopathy Questionnaire Overall Summary Score) at 30 months. This trial in Japan was conducted to support local registration.
In this single-arm study where patients were on acoramidis treatment for 30 months, acoramidis was well-tolerated, with no safety signals of potential clinical concern identified, and no mortality was reported. The data will be presented at a forthcoming medical meeting and submitted to Japan’s health authority for regulatory review.
Alexion, AstraZeneca Rare Disease, maintains an exclusive license with BridgeBio’s affiliate, Eidos Therapeutics, Inc. to develop and commercialize acoramidis in Japan. Acoramidis is an investigational, next-generation, oral, highly potent small molecule stabilizer of transthyretin (TTR), designed to achieve maximal stabilization and preserve native TTR.
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 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.
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
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usually identified by the use of words such as “anticipates,”
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27A of the Securities Act and Section 21E of the Exchange Act.
These forward-looking statements, including statements relating
to the clinical and therapeutic potential of our clinical
development program for acoramidis for patients with
transthyretin amyloid cardiomyopathy, including the plans to
present data from the single-arm, open-label Phase 3 study of
acoramidis conducted in Japan by our partner Alexion,
AstraZeneca Rare Disease, at a forthcoming medical meeting and
to submit such data to Japan’s health authority for regulatory
review, 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 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