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

Rhea-AI Summary

BridgeBio Pharma, Inc. (BBIO) announced positive results from its Phase 3 ATTRibute-CM study of acoramidis for patients with ATTR-CM, demonstrating a significant treatment effect with a 1.8 Win Ratio and 80.7% survival rate. Acoramidis was well-tolerated, with no safety concerns identified. BridgeBio submitted a New Drug Application to the FDA and plans to submit marketing authorization applications to additional regulatory bodies in 2024.

Medical Research Analyst

The publication of the Phase 3 ATTRibute-CM study results in the New England Journal of Medicine marks a significant milestone for BridgeBio Pharma and the medical community treating ATTR-CM, a form of cardiac amyloidosis. The reported Win Ratio of 1.8 and a survival rate of 80.7% at 30 months for patients treated with acoramidis are critical data points. These figures suggest a notable improvement in treatment outcomes compared to the current standard of care, potentially establishing acoramidis as a new benchmark in ATTR-CM therapy.

In the context of medical research, the hierarchical analysis method used in the trial is noteworthy for its robustness in evaluating multiple endpoints, such as all-cause mortality and cardiovascular-related hospitalization. The observed benefits in secondary endpoints, including 6-minute walk distance and serum TTR levels, further corroborate the drug's efficacy. The consistent elevation of serum TTR indicates effective stabilization of the transthyretin protein, which is implicated in the pathophysiology of ATTR-CM.

The safety profile of acoramidis, as indicated by the absence of concerning safety signals, is equally important for patient care. Tolerability is a critical factor in chronic conditions where long-term treatment adherence is necessary.

Financial Analyst

The announcement by BridgeBio Pharma regarding the positive outcomes of the acoramidis study is poised to have significant financial implications. The submission of a New Drug Application (NDA) to the FDA is the first step towards commercialization, which could lead to a new revenue stream for the company. Given the high unmet medical need in ATTR-CM and the lack of alternative treatments demonstrating similar benefits, acoramidis could capture a substantial market share.

Investors should pay attention to the FDA's decision on the NDA, as approval would likely have a positive impact on BridgeBio's stock value. Additionally, the intention to submit marketing authorization applications to other regulatory bodies in 2024 hints at the company's strategy for global market penetration, which could further enhance its growth prospects and investor confidence.

However, it is important to monitor the competitive landscape, as other companies may be developing similar treatments that could affect market dynamics. Furthermore, the drug's pricing strategy, reimbursement potential and market adoption rates will be crucial factors determining its financial success.

Market Research Analyst

The data presented in the ATTRibute-CM study not only has clinical significance but also provides insights into the market dynamics of therapies for ATTR-CM. The comparison of the 30-month survival rate in the treatment arm with an age-matched cohort from the general population provides a context for evaluating the drug's potential impact on patient outcomes. Moreover, the annualized cardiovascular-related hospitalization rate in the treatment arm being close to the overall hospitalization rate in the U.S. Medicare population suggests that acoramidis may help reduce the burden of hospitalizations, a significant cost driver in healthcare.

As the only intervention to demonstrate a cardiovascular outcomes benefit in a prospective clinical trial for the contemporary ATTR-CM population, acoramidis could become the treatment of choice, influencing prescribing patterns. Market adoption will likely be influenced by factors such as clinician awareness, patient advocacy and the strength of clinical data. BridgeBio's efforts in presenting detailed results at major cardiology congresses are indicative of a strategic approach to building awareness and credibility within the medical community.

Understanding the patient population's size and growth trajectory, as well as the potential for label expansion, will be key in forecasting the market potential for acoramidis. The drug's profile suggests a strong value proposition, which could drive market uptake and support a favorable reimbursement environment.

- ATTRibute-CM demonstrated a significant treatment effect of acoramidis on the primary endpoint (a hierarchical analysis inclusive of all-cause mortality (ACM) and frequency of cardiovascular-related hospitalization (CVH)), with a Win Ratio of 1.8 (p<0.0001)

- Acoramidis demonstrated an observed 30-month survival rate of 80.7% in the treatment arm of ATTRibute-CM; 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 of 0.29 in the treatment arm can be viewed in the context of the annual overall hospitalization rate of 0.26 in the U.S. Medicare population

- Acoramidis is the only intervention to demonstrate cardiovascular outcomes benefit in a prospective clinical trial in the contemporary ATTR-CM population to the Company's knowledge

- Acoramidis was well-tolerated, with no safety signals of potential clinical concern identified

-   BridgeBio has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) and intends to submit marketing authorization applications to additional regulatory bodies in 2024

PALO ALTO, Calif., Jan. 10, 2024 (GLOBE NEWSWIRE) -- 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 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 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 Bali
contact@bridgebio.com
(650)-789-8220

FAQ

What are the results of the Phase 3 ATTRibute-CM study by BridgeBio Pharma, Inc. (BBIO)?

The study demonstrated a significant treatment effect of acoramidis with a 1.8 Win Ratio and an 80.7% survival rate.

What is acoramidis and its potential use according to the study results?

Acoramidis is an investigational, next-generation, orally-administered, small molecule stabilizer of transthyretin (TTR) and has shown consistent benefits in the treatment of ATTR-CM.

What is the New Drug Application (NDA) submitted by BridgeBio Pharma, Inc. (BBIO)?

BridgeBio submitted an NDA to the U.S. FDA for acoramidis and plans to submit marketing authorization applications to additional regulatory bodies in 2024.

What were the additional findings in the publication from the Phase 3 ATTRibute-CM study?

Additional findings include a 30-month survival rate of 74.3% in the placebo arm and the well-tolerated nature of acoramidis with no safety concerns identified.

Where have the detailed results from ATTRibute-CM been presented?

The detailed results have been presented at the European Society of Cardiology Congress 2023 and the American Heart Association Scientific Sessions 2023.