- Case study co-authored by members of BridgeBio senior management and BridgeBio co-founder and MIT professor, Andrew W. Lo, Ph.D. - Case study features BridgeBio’s unique model that uses portfolio theory to enable early-stage research for genetic diseases and conditions that are not typically addressed by the pharmaceutical industry PALO ALTO, Calif., Nov. 05, 2024 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a new type of biopharmaceutical company focused on ge ...

Core Viewpoint - Upcoming FDA decisions for BridgeBio Pharma, PTC Therapeutics, and Vertex Pharmaceuticals could lead to significant stock price movements before the end of 2024 [1] Group 1: BridgeBio Pharma - BridgeBio Pharma's acoramidis is under FDA review for treating transthyretin amyloidosis cardiomyopathy (ATTR-CM), with a decision expected by Nov. 29 [2] - Recent evidence shows acoramidis reduced the risk of death by 42% and hospitalization due to cardiovascular events by 50% compared to a placebo [2] - BridgeBio has a market cap of $4.7 billion, and a positive FDA decision could significantly increase its stock value [3] Group 2: PTC Therapeutics - PTC Therapeutics has two approved therapies and receives royalties from Roche for Evrysdi, generating $118 million in product sales and $53 million in royalties in Q2 [4] - The FDA is reviewing Upstaza, a gene therapy for AADC deficiency, with a decision expected by Nov. 13 [4] - PTC Therapeutics has a market cap of $2.8 billion, and a positive decision on Upstaza could enhance its stock performance [5] Group 3: Vertex Pharmaceuticals - Vertex Pharmaceuticals' lead drug, Trikafta, is projected to generate over $10.5 billion in sales this year, treating over 68,000 cystic fibrosis patients [6] - The FDA is reviewing a new once-daily dosing regimen for Vertex, with a decision expected by Jan. 2, 2025 [6] - Trikafta patents are expected to maintain market exclusivity until 2037, and a new approval could further solidify Vertex's market position [7]

Core Insights - BridgeBio Pharma, Inc. announced that outcomes data from the ongoing long-term open-label extension of its Phase 3 study of acoramidis in ATTR-CM will be presented at the AHA Scientific Sessions in November 2024 [1] - The company will also share three moderated digital posters related to ATTR-CM during the conference [1] Presentation Details - The featured science oral presentation will focus on how acoramidis reduces all-cause mortality and cardiovascular-related hospitalization, presented by Dr. Daniel Judge on November 18, 2024 [2] - The moderated digital posters will cover various topics, including healthcare resource utilization in transthyretin amyloid cardiomyopathy and evolving baseline risk in patients [2] Company Overview - BridgeBio Pharma is a commercial-stage biopharmaceutical company founded in 2015, dedicated to developing transformative medicines for genetic diseases [3] - The company’s pipeline includes a range of development programs from early science to advanced clinical trials [3]

Core Insights - BridgeBio Pharma presented a post-hoc analysis of acoramidis in the Phase 3 ATTRibute-CM study, showing significant reductions in all-cause mortality and recurrent cardiovascular-related hospitalizations [1][2] - The analysis demonstrated a 42% reduction in composite ACM and recurrent CVH events at 30 months with acoramidis compared to placebo [1] - The FDA has accepted BridgeBio's marketing application for acoramidis, with a PDUFA action date set for November 29, 2024 [2] Group 1 - The post-hoc analysis indicated a 42% reduction in composite ACM and recurrent CVH events at 30 months with acoramidis treatment compared to placebo, with a p-value of 0.0005 [1] - A 30.5% hazard reduction in ACM and recurrent CVH events at 30 months was observed using the Andersen-Gill model, with a p-value of 0.0008 [1] - The results support acoramidis as a potential first-line therapy for patients with ATTR-CM, improving overall quality of life [2] Group 2 - Transthyretin amyloidosis (ATTR-CM) is caused by the liver producing faulty transthyretin proteins, leading to abnormal protein clumps in the heart [2] - BridgeBio also presented additional data from the ATTRibute-CM study at the European Society of Cardiology 2024 [3] - Following the positive results, BBIO stock increased by 2.39% to $25.54 [3]

- Acoramidis treatment led to a highly significant reduction in all-cause mortality (ACM) and recurrent cardiovascular-related hospitalizations (CVH) at Month 30 compared with placebo - Additionally, results from a Phase 3 trial in adults with ATTR-CM conducted in Japan were presented, with no ACM events reported over the 30 month treatment period in participants administered acoramidis - Greater transthyretin (TTR) stabilization has been associated with improved clinical outcomes for patients, and in ATTRi ...

Core Insights - The FDA granted Breakthrough Therapy Designation to infigratinib for children with achondroplasia, based on preliminary clinical evidence from the PROPEL 2 trial, indicating potential substantial improvement over existing therapies [1][2][3] - Infigratinib could become the first oral therapeutic option for children with achondroplasia if approved, which would significantly benefit the affected community [1][4] - The PROPEL 2 trial data showed a statistically significant increase in annualized height velocity (AHV) of +2.51 cm/year at Month 12 and +2.50 cm/year at Month 18, with p-values indicating strong statistical significance [1][3] Regulatory Designations - In addition to Breakthrough Therapy Designation, infigratinib has received Orphan Drug Designation, Fast Track Designation, and Rare Pediatric Disease Designation from the FDA [5] - The Breakthrough Therapy Designation is aimed at expediting the development and regulatory review process for drugs that show substantial improvement over existing therapies [2] Clinical Development - The ongoing PROPEL 3 Phase 3 registrational study of infigratinib is on schedule, with enrollment expected to be completed by the end of the year [4] - The company is committed to exploring the broader medical and functional impacts of infigratinib on achondroplasia and other skeletal dysplasias, addressing significant unmet needs [4]

Core Viewpoint - BridgeBio's stock performance has been disappointing despite positive developments such as the upcoming FDA approval for acoramidis, strong phase 2 data for infigratinib, and the spinout of its oncology unit and early-stage rare disease drugs into GondolaBio [2][9] Company Developments - Acoramidis is positioned to capture market share in the growing ATTR cardiomyopathy market, with recent data showing it has a 42% reduction in all-cause mortality and cardiovascular hospitalizations at month 30, compared to 25% for tafamidis and 28-33% for Amvuttra [4][6] - The HELIOS-B trial results have alleviated concerns regarding acoramidis' competitive position against Amvuttra, allowing investors to focus on BridgeBio's own pipeline and upcoming regulatory events [9][13] - The company has a strong pipeline with late-stage candidates including infigratinib for achondroplasia and encaleret for autosomal dominant hypocalcemia type 1, both of which are expected to deliver positive results in upcoming trials [10][13] Market Context - The ATTR cardiomyopathy market is projected to exceed $15 billion globally by the early 2030s, with acoramidis and Amvuttra potentially generating $2-3 billion in annual net sales by the late 2020s [8] - Tafamidis is expected to remain the market leader initially due to its first-mover advantage, but acoramidis and Amvuttra are anticipated to compete closely and may surpass tafamidis by the early 2030s [8] Regulatory and Financial Outlook - The key upcoming event for BridgeBio is the PDUFA date for acoramidis in late November, which poses a significant risk but also an opportunity for revenue growth if approved [9][12] - If acoramidis is approved, it is expected to exceed the revenue consensus of $236 million for the following year, driven by its strong clinical profile and the size of the ATTR CM market [9][10] - The company has a solid financial position with $587 million in cash and equivalents, and additional funding contingent on acoramidis' FDA approval [12]

Core Insights - The study presented by BridgeBio Pharma indicates that acoramidis, a TTR stabilizer, shows significant improvements in serum TTR levels and clinical outcomes for patients with ATTR-CM [1][2][3] Group 1: Clinical Study Results - In the ATTRibute-CM study, participants switching to acoramidis from tafamidis and placebo experienced a mean increase of 3.0 mg/dL in serum TTR at Month 1 and 3.4 mg/dL at Month 6 of the open-label extension [1][2] - Increased serum TTR levels at Day 28 were correlated with reduced risks of all-cause mortality, cardiovascular mortality, and cardiovascular-related hospitalization [1][3] - Acoramidis demonstrated a 42% reduction in composite cardiovascular hospitalization and all-cause mortality events relative to placebo at Month 30 [1] - A 50% reduction in cumulative frequency of cardiovascular hospitalization events was observed at Month 30 compared to placebo [1] Group 2: Regulatory and Commercial Developments - BridgeBio submitted a New Drug Application to the U.S. FDA, accepted with a PDUFA action date of November 29, 2024, and a Marketing Authorization Application to the European Medicines Agency, with a decision expected in 2025 [4] - The company granted Bayer exclusive rights to commercialize acoramidis for ATTR-CM in Europe, leveraging Bayer's expertise in cardiovascular disease [6] Group 3: Future Directions and Collaborations - The ongoing partnership with the Cardiovascular Data Science Lab aims to utilize AI tools for screening ATTR-CM across diverse health systems, focusing on undiagnosed cases [3] - The data presented may support the rationale for monitoring serum TTR levels in patients and inform treatment decisions in the context of multiple disease-modifying therapies for ATTR-CM [3]

Core Insights - BridgeBio Pharma, Inc. announced additional data on clinical outcomes from its Phase 3 study of acoramidis in ATTR-CM, to be presented at two major upcoming medical conferences [1][2] Group 1: Clinical Study and Presentations - The Phase 3 study, ATTRibute-CM, focuses on the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM) with acoramidis [1] - Presentations will occur at the European Society of Cardiology (ESC) Congress 2024 in London from August 30 to September 2, and at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2024 in Atlanta from September 27 to 30 [1][2] - Dr. Mathew S. Maurer will present findings on serum TTR levels observed with acoramidis treatment at the ESC Congress [2] - Dr. Daniel P. Judge will present a post hoc recurrent event analysis of clinical outcomes at the HFSA meeting [2] Group 2: Company Overview - BridgeBio Pharma is a commercial-stage biopharmaceutical company focused on developing transformative medicines for genetic diseases [3] - The company was founded in 2015 and has a pipeline that includes programs from early science to advanced clinical trials [3] - BridgeBio aims to leverage advances in genetic medicine to expedite treatment for patients [3]

PALO ALTO, Calif., Aug. 28, 2024 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio), a commercial-stage biopharmaceutical company focused on genetic diseases, today announced that members of the management team will participate in the following upcoming investor conferences: Morgan Stanley 22nd Annual Global Health Care Conference, New York, NY: Fireside Conversation on September 4th at 10:00 am ET Wells Fargo 19th Annual Healthcare Conference 2024, Boston, MA, September 5th Cantor Global ...