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
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Insights
The recent findings from the Japan Phase III trial of acoramidis present important implications for BridgeBio Pharma and its stakeholders. The consistency of the trial's results with the global ATTRibute-CM Phase III trial emphasizes the potential of acoramidis as a stabilizer of transthyretin (TTR) in the treatment of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). The absence of mortality and the well-tolerated nature of the treatment over a 30-month period are significant, as they suggest a favorable safety profile which is a critical factor in the drug approval process. The reported survival rate and reduction in cardiovascular hospitalizations could translate to reduced healthcare costs and improved patient outcomes, which is a key consideration for healthcare providers and insurance companies.
Furthermore, the positive outcome of this trial is likely to bolster investor confidence in BridgeBio Pharma, potentially affecting its stock valuation positively. The data also supports the company's strategy to expand its commercial footprint in Japan, a significant market for pharmaceuticals. The successful commercialization of acoramidis in Japan could lead to increased revenue streams for the company and its licensing partner, Alexion, AstraZeneca Rare Disease.
The announcement of successful Phase III trial results in Japan is likely to have a considerable impact on BridgeBio Pharma's financial outlook. The trial's success paves the way for regulatory submission in Japan, which could result in a new revenue source upon approval. The market for ATTR-CM treatments is currently underserved and the introduction of a novel therapy like acoramidis could capture significant market share. The high statistical significance of the trial's primary endpoint and the impressive survival rate at 30 months are metrics that investors will closely monitor, as they are indicative of the drug's efficacy and market potential. These factors are likely to influence the company's stock performance in the short term, with the anticipation of regulatory approval and market entry offering potential long-term financial benefits.
From a market perspective, the successful results of acoramidis in Japan represent a strategic milestone for BridgeBio Pharma. Japan is one of the largest pharmaceutical markets globally and a local regulatory submission backed by positive trial data could facilitate swift market penetration. The global ATTR-CM market is expected to grow and BridgeBio's entry into this space, particularly in a high-value market like Japan, could position the company favorably against competitors. The therapeutic's oral administration and potency could provide a competitive edge over existing treatments, which may involve more invasive administration routes or have less efficacy. Market adoption will depend on factors such as pricing strategy, reimbursement policies and physician awareness, which will need to be carefully managed to optimize market uptake.
- 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
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 bridgebio.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 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 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
contact@bridgebio.com
(650)-789-8220
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