BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the
“Company”), a commercial-stage biopharmaceutical company
focused on genetic diseases and cancers, announced positive results
of five new analyses of clinical endpoint events from its Phase 3
ATTRibute-CM study of acoramidis in ATTR-CM. These new analyses
were shared in oral presentations and posters at the 2024
International Symposium of Amyloidosis (ISA). 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.
BridgeBio will host an investor call on Wednesday,
May 29th at 5:30 pm ET to discuss these results.
“The data presented at ISA confirm that improved
stabilization as reflected in higher serum TTR levels is directly
correlated with improved clinical outcomes. Prior analyses from
ATTRibute-CM demonstrated that the near-complete stabilization by
acoramidis rapidly and durably increased serum TTR
levels. Clinically, we now have evidence that
acoramidis-mediated increase in serum TTR independently predicted
statistically significant improvement in survival, and risk
reduction of CVM and CVH,” said Mathew Maurer M.D. of Columbia
University Irving Medical Center, U.S.
Three analyses presented from ATTRibute-CM
emphasized the correlation between increased serum TTR and improved
clinical outcomes, including the reduced risk of both all-cause
mortality and cardiovascular death as well as
cardiovascular-related hospitalization:
- Early increase in serum
transthyretin level is an independent predictor of improved
survival in ATTR cardiomyopathy: Insights from acoramidis Phase 3
study ATTRibute-CM, presented by Mathew Maurer, M.D., Columbia
University Irving Medical Center, U.S.
- Statistically significant
correlation between increasing serum TTR and decreasing risk of
death: for every 5mg/dL increase in serum TTR level at day 28 after
treatment initiation, the risk of death through Month 30 was
reduced by 30.9% (by the logistic model) and 26.1% (by the Cox
proportional hazards model)
- Statistical modeling suggests that
the acoramidis-mediated increase in serum TTR at Day 28 is an
independent predictor of survival
- Treatment-related early increase in
serum TTR is associated with lower cardiovascular mortality in
ATTR-CM: Insights from ATTRibute-CM, presented by Nitasha Sarswat,
M.D., University of Chicago Medicine, U.S.
- For each 1 mg/dL increase in serum
TTR on day 28 after treatment initiation, there was a 5.5% risk
reduction in cardiovascular death observed through Month 30
- To the company’s knowledge, this is
the first prospective demonstration of the relationship between
change from baseline in serum TTR and subsequent risk of
cardiovascular death in ATTR-CM
- Acoramidis treatment-related
increase in serum TTR is associated with a lower risk of
cardiovascular-related hospitalization in ATTR-CM Patients:
Insights from the ATTRibute-CM trial, presented by Margot Davis,
M.D., Vancouver General Hospital, CA
- Each 1 mg/dL serum TTR increase at
Day 28 after treatment initiation was associated with a 4.7% lower
risk of a first cardiovascular hospitalization over 30 months
- The analysis, the first from a
prospective study of the relationship between change from baseline
in TTR and subsequent risk of first CVH in ATTR, demonstrated that
a greater increase in TTR is significantly associated with a lower
risk of CVH
- The Kaplan-Meier curves for time to
first CVH were also presented; the curves separated early, showing
treatment benefit at Month 3 and continuing to separate through
Month 30
The results from the analysis highlighting the
early reduction in cardiovascular mortality (CVM) or CVH in ATTR in
the ATTRibute-CM trial were shared by Kevin M. Alexander, M.D.,
Stanford University School of Medicine, U.S., in an oral
presentation. Key findings included:
- Acoramidis time-to-first event
Kaplan-Meier (K-M) curves for a composite of CVM and CVH in ATTR-CM
patients separated beginning at Month 3, representing an early and
profound reduction on the composite endpoint of CVM and CVH in
ATTR-CM patients, with a 15.2% absolute risk reduction and a 38.2%
hazard reduction by Month 30 (p=0.0003).
On behalf of the authors, John Whang, M.D., Chief
Medical Affairs Officer of BridgeBio Cardiorenal, presented data
showing a higher risk of mortality in previously hospitalized
participants. Insights included:
- Participants with no CVH during the study had a 30-month
survival rate of 86.7%, vs 60.1% in participants who had at least
one CVH during the study
- To the company’s knowledge, this is the first time a
prospective trial demonstrates that CVH portends a higher
subsequent mortality in ATTR-CM patients
- This suggests that effective treatments to reduce CVH risk are
critically important, and a targeted therapy for ATTR-CM that
reduces CVH can improve the prognosis of patients with ATTR-CM
The rationale and design of ACT-EARLY, the
acoramidis TTR amyloidosis prevention trial, was also presented by
Pablo Garcia-Pavia, M.D., Ph.D., Iron Gate Majadahonda University
Hospital, ES. ACT-EARLY will be the first Phase 3 trial to evaluate
prophylactic therapy for the prevention or delay of ATTR
amyloidosis in asymptomatic pathogenic TTR variant carriers with
study initiation planned in 2024.
Jonathan Fox, M.D., Ph.D., President and Chief
Medical Officer of BridgeBio Cardiorenal, shared the following:
“The totality of acoramidis data across clinical outcomes,
biomarkers, quality of life, and cardiac imaging continues to
expand with the analyses shared at ISA and the data recently
presented at ACC and ESC-HF. We remain encouraged by the potential
benefits of targeting near-complete TTR stabilization, the
resulting increases in serum TTR, and the corresponding
statistically significant benefits on clinical event
outcomes. We are committed to bringing acoramidis to the
ATTR-CM community as quickly as possible, working toward our
November 29th PDUFA date.”
Additionally, BridgeBio presented six encore poster
presentations on its ATTRibute-CM data, which were previously
shared at the European Society of Cardiology Heart Failure (ESC-HF)
Congress 2024, the American College of Cardiology (ACC) Annual
Scientific Sessions & Expo 2024 and the American Heart
Association (AHA) Scientific Sessions 2023.
Based on the positive results from ATTRibute-CM,
BridgeBio submitted a New Drug Application (NDA) to the U.S. Food
and Drug Administration, which has been accepted with a
Prescription Drug User Fee Act (PDUFA) action date of November 29,
2024, and a Marketing Authorization Application (MAA) to the
European Medicines Agency, with a decision expected in 2025.
Webcast InformationBridgeBio will
host an investor call and simultaneous webcast to discuss the
recent analyses and positive data from the ATTRibute-CM Phase 3
trial and emerging real-world evidence in ATTR-CM presented at the
2024 ISA, ESC Heart Failure 2024 and the 2024 ACC Annual Scientific
Sessions & Expo on Wednesday, May 29 at 5:30 pm ET. A link to
the webcast may be accessed from the event calendar page of
BridgeBio’s website at https://investor.bridgebio.com/. A replay of
the conference call and webcast will be archived on the Company’s
website and will be available for at least 30 days following the
event.
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
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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,” “continues,” “estimates,”
“expects,” “hopes,” “intends,” “may,” “plans,” “projects,”
“remains,” “seeks,” “should,” “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 impact of acoramidis on
clinical outcomes, including survival, mortality and
hospitalization rates and risks, quality of life, biomarkers and
magnetic resonace imaging; potential benefits of acoramidis,
including near-complete TTR stabilization, increases in serum TTR,
and the corresponding statistically significant benefits on
clinical event outcomes; anticipated timeline to bring acoramidis
to the ATTR-CM community; and the clinical, therapeutic and market
potential of our clinical development program and timeline for
acoramidis 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
preclinical studies and clinical trials not being indicative of
final data, the potential size of the target patient populations
our product candidates are designed to treat not being as large as
anticipated, the design and success of ongoing and planned clinical
trials, future regulatory filings, approvals and/or sales, the FDA
or such 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, the continuing success of our collaborations,
potential volatility in our share price, uncertainty regarding any
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 our
most recent Annual Report on Form 10-K 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
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