BridgeBio Pharma Announces Publication of Positive Results from Phase 3 ATTRibute-CM Study of Acoramidis for Patients with Transthyretin Amyloid Cardiomyopathy (ATTR-CM) in the New England Journal of Medicine
11 1월 2024 - 7:15AM
BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the
“Company”), a commercial-stage biopharmaceutical company
focused on genetic diseases and cancers, announced that positive
results from its Phase 3 ATTRibute-CM study of acoramidis for
patients with ATTR-CM were published in the New England Journal of
Medicine (NEJM). ATTRibute-CM was designed to study the efficacy
and safety of acoramidis, an investigational, next-generation,
orally-administered, small molecule stabilizer
of transthyretin (TTR).
“The consistent benefits of acoramidis treatment demonstrated by
the ATTRibute-CM results, especially in the context of contemporary
ATTR-CM care, are striking and encourage its potential use,” said
Professor Julian Gillmore, M.D., Ph.D., head of University College
London’s Centre for Amyloidosis and research lead at the UK
National Amyloidosis Centre. “Given the efficacy and safety of
acoramidis demonstrated in this trial, I am hopeful that it will
soon be available to the benefit of the growing global population
of patients diagnosed with ATTR-CM.”
The ATTRibute-CM study demonstrated a significant treatment
effect of acoramidis in the primary analysis that compared, in a
hierarchical manner, all-cause mortality (ACM),
cardiovascular-related hospitalization (CVH), N-terminal prohormone
of brain natriuretic peptide (NT-proBNP), and 6-minute walk
distance (6MWD). Findings presented in the NEJM support acoramidis
as an effective and safe treatment option for patients with ATTR-CM
and reinforce the hypothesis that greater stabilization of TTR may
be associated with improved clinical outcomes. Additional findings
in the publication include:
- The majority of comparisons in the primary hierarchical
analysis (58% in the associated Win Ratio) were determined by the
first two components of ACM and CVH; statistical significance was
also achieved on a F-S test with those two cardiovascular outcomes
parameters alone
- Statistically significant treatment benefit was observed for
change from baseline in 6MWD, Kansas City Cardiomyopathy
Questionnaire (KCCQ), and serum TTR
- The observed 30-month survival rate of 74.3% in the placebo arm
of ATTRibute-CM is greater than the observed 30-month survival rate
of 70.5% in the combined tafamidis treatment arms of ATTR-ACT, the
only previously reported cardiovascular outcomes study in ATTR-CM
- As a contemporary benchmark for placing the survival rate of
80.7% in the treatment arm of ATTRibute-CM into context, recent
data from the U.S. Social Security Administration estimated
30-month survival at 85% in an age-matched cohort of the general
population
- Similarly, the annualized CVH rate in the treatment arm of
ATTRibute-CM of 0.29 can be viewed in the context of data on the
annual overall hospitalization rate of 0.26 in the U.S. Medicare
population.
- Serum TTR was promptly and
consistently elevated throughout the study in patients receiving
acoramidis as a result of near-complete stabilization of the
protein
- Acoramidis was well-tolerated, with no safety signals of
potential clinical concern identified
“These results add to the totality of the available data
supporting the concept that the greater the degree of stabilization
of tetrameric TTR, the greater the observed clinical benefit,” said
Jonathan Fox, M.D., Ph.D., president and chief medical officer of
BridgeBio Cardiorenal. “With the submission of our NDA to register
acoramidis with the FDA, and with additional regional and national
regulatory submissions planned, we look forward to making
acoramidis available to patients who might benefit from its
demonstrated efficacy and safety.”
In July, BridgeBio announced positive topline results from
ATTRibute-CM. BridgeBio has also presented additional detailed
results from ATTRibute-CM at the European Society of Cardiology
Congress 2023 in August and at the American Heart Association
Scientific Sessions 2023 in November.
The Company submitted a New Drug Application to the U.S. FDA in
2023 and intends to submit additional marketing authorization
applications to regulatory bodies in 2024.
About BridgeBio 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.
BridgeBio Forward-Looking StatementsThis 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,”
“continue,” “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 the clinical, therapeutic and
market potential of our programs and product candidates, including
our clinical development program for acoramidis for patients with
transthyretin amyloid cardiomyopathy, the timing and success of our
clinical development programs, the progress of our ongoing and
planned clinical trials of acoramidis for patients with
transthyretin amyloid cardiomyopathy, including our planned
interactions with regulatory authorities, our intention to submit
additional marketing authorization applications in 2024, the
statements regarding the potential benefit of our clinical trial or
of our product candidate or the potential availability of our
product candidate to patients who might benefit from it in the
quotes of Dr. Gillmore and Dr. Fox, 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, 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 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
BridgeBio Pharma (NASDAQ:BBIO)
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