BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the
“Company”), a commercial-stage biopharmaceutical company
focused on genetic diseases and cancers, today presented results
from the exploratory CMR imaging substudy of ATTRibute-CM, its
Phase 3 trial of acoramidis in ATTR-CM. These data were presented
at the American College of Cardiology (ACC) Annual Scientific
Sessions & Expo in a moderated poster session by Yousef Razvi,
M.D. of University College London. ATTRibute-CM was designed to
study the efficacy and safety of acoramidis, an investigational,
next-generation, orally-administered, highly potent, small molecule
stabilizer of TTR. Based on the positive results from ATTRibute-CM,
BridgeBio submitted an NDA to the U.S. FDA, which has been accepted
with a PDUFA action date of November 29, 2024, and an MAA to the
European Medicines Agency, with a decision expected in 2025.
“CMR is the reference non-invasive imaging method to evaluate
cardiac structure, function, and amyloid burden in patients with
ATTR-CM. The results shown in the imaging substudy are indicative
of potential improvement of cardiac structure and function in
patients with ATTR-CM, consistent with the clinical outcomes with
acoramidis treatment observed in the ATTRibute-CM Phase 3 clinical
trial. The impact on cardiac amyloid load highlights the potential
that acoramidis could lead to cardiac amyloid regression in a
proportion of patients with ATTR-CM, which we have not observed in
such a controlled clinical trial to date,” said Marianna Fontana,
M.D., Ph.D., Professor of Cardiology and Honorary Consultant
Cardiologist at the National Amyloidosis Centre, Division of
Medicine, University College London, and Principal Investigator of
the Substudy.
CMR imaging was performed at baseline before the first dose in
35 participants or within three months after the first dose in 17
participants (range, 14-105 days); subsequent CMR imaging was
performed at months 12, 24, and 30 in those individuals who were
available to undergo imaging, which was influenced by survival.
Images were read centrally at the National Amyloidosis Centre in a
fashion blinded to other clinical data. Findings included:
- Key CMR imaging parameters measuring cardiac structure,
including mean indexed left ventricular mass, were found to be
stable or have a trend towards improvement on acoramidis vs.
deteriorated on placebo over 30 months
- Treatment with acoramidis preserved or was associated with a
trend towards improvement in measures of cardiac function including
left ventricular ejection fraction and stroke volume compared to
initial measures, and relative to placebo
In July 2023, BridgeBio announced positive results from
ATTRibute-CM, reporting a highly statistically significant result,
demonstrated by a Win Ratio of 1.8 (p<0.0001) on the primary
endpoint (a hierarchical analysis prioritizing in order: all-cause
mortality, then frequency of cardiovascular hospitalization, then
change from baseline in N-terminal prohormone of brain natriuretic
peptide, then change from baseline in 6-minute walk distance).
Acoramidis was well-tolerated, with no safety signals of potential
clinical concern identified. BridgeBio has also presented analyses
from ATTRibute-CM at the European Society of Cardiology Congress
2023 and at the American Heart Association Scientific Sessions
2023. In February 2024, BridgeBio shared positive results of a
single-arm Phase 3 study of acoramidis in Japanese patients with
ATTR-CM, including no mortality observed in the trial at 30
months.
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 bridgebio.com and follow us
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BridgeBio Pharma, Inc. 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 and therapeutic 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
the expectations of receiving a PDUFA action from the FDA on
November 29, 2024 and receiving a decision from the EMA on the MAA
for acoramidis in 2025, the expected intellectual property
protection of acoramidis, our planned interactions with regulatory
authorities, the statements regarding the potential clinical
benefits or of potential benefits for ATTR-CM patients in the
quotes of Dr. Fontana, 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 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, 2023 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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