BridgeBio Pharma Inc.’s drug to treat a devastating muscle-wasting disorder met its goal in a late-stage trial, paving the way for the biotech company to seek US approval https://t.co/3px4OBe0LQ ...

Core Insights - The FORTIFY Phase 3 study of BBP-418 has successfully achieved all primary and secondary interim analysis endpoints with a well-tolerated safety profile consistent with prior studies [1] - BBP-418 treatment resulted in a significant increase in glycosylated αDG by 1.8 times from baseline at 3 months, with sustained improvements at 12 months [1][4] - The average reduction in serum CK, a marker of muscle damage, was 82% from baseline at 12 months in BBP-418 treated individuals, showing a statistically significant difference versus placebo [1][4] - The company plans to file a New Drug Application (NDA) with the FDA in the first half of 2026 [1][5] Study Results - The FORTIFY study is a randomized, double-blind, placebo-controlled Phase 3 trial evaluating BBP-418 for LGMD2I/R9 [3] - Key results at 12 months include: - Ambulatory function (100MTT): Increase in velocity of 0.14 m/s from baseline and 0.27 m/s versus placebo (p<0.0001) [3][4] - Pulmonary function (FVC): Increase of approximately 3% predicted volume from baseline and a difference of about 5% versus placebo (p=0.0071) [3][10] Safety and Regulatory Designations - BBP-418 was well-tolerated with no new or unexpected safety findings observed [10] - The drug has received Orphan Drug, Fast Track, and Rare Pediatric Disease Designations from the FDA, as well as Orphan Drug Designation from the EMA [6] Disease Background - LGMD2I/R9 is a monogenic autosomal recessive disease caused by mutations in the FKRP gene, leading to impaired glycosylation of αDG [7] - Clinical manifestations include skeletal myopathy, pulmonary muscle involvement, and cardiac issues, with significant morbidity in affected individuals [7] Company Overview - BridgeBio Pharma, Inc. is focused on discovering and delivering transformative medicines for genetic diseases, with a commitment to applying advances in genetic medicine [8]

PALO ALTO, Calif., Oct. 24, 2025 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a new type of biopharmaceutical company focused on genetic diseases, today announced that members of its management team will host two separate business update calls to share the following: Limb-girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9) FORTIFY Phase 3 Interim Analysis Topline Results Webinar: Monday, October 27 at 8:00 am ETQ3 2025 Earnings: Wednesday, October 29 at 4:30 pm ET T ...

Core Insights - The article highlights stocks reaching new 52-week highs, indicating market recognition of strong fundamentals and potential catalysts for growth [1][2]. Company Summaries Cogent Biosciences Inc. (COGT) - Cogent Biosciences plans to file its first New Drug Application for Bezuclastinib by the end of 2025, targeting non-advanced systemic mastocytosis [3]. - A phase III trial comparing Bezuclastinib with Sutent is ongoing, with results expected in the second half of 2025 [4]. - The stock reached a 52-week high of $16.99, up from $7.25 when last featured [5]. Assembly Biosciences Inc. (ASMB) - Assembly Biosciences is advancing four key development programs, with ABI-5366 expected to enter phase 2 studies in mid-2026 [6]. - Interim data for ABI-1179 is anticipated this fall, while ABI-6250 is in a phase 1a trial [7]. - The stock hit a 52-week high of $28, up from $14.53 when last featured [8]. Compass Therapeutics Inc. (CMPX) - Compass Therapeutics is conducting a phase 2/3 study of Tovecimig for advanced biliary tract cancer, with analyses of secondary endpoints expected in Q1 2026 [9][10]. - The stock reached a 52-week high of $4.39, up from $2.91 when last featured [11]. NewAmsterdam Pharma Company N.V. (NAMS) - NewAmsterdam Pharma is developing Obicetrapib as a cholesterol-lowering therapy, with positive data from the BROADWAY trial [12][13]. - The company has completed two additional phase III trials and submitted marketing applications to the EMA [16]. - The stock reached a 52-week high of $39.76, up from $21.56 when last featured [17]. Mineralys Therapeutics Inc. (MLYS) - Mineralys is developing Lorundrostat for uncontrolled hypertension, with a pivotal phase III trial achieving its primary endpoint [19]. - A phase II trial for overweight participants with OSA is ongoing, with topline results expected in 1H 2026 [20]. - The stock hit a 52-week high of $43.88, up from $10.34 when last featured [20]. Kymera Therapeutics Inc. (KYMR) - Kymera is set to report data from its phase I trial of KT-621 this quarter, with phase 2b studies planned for late 2025 and early 2026 [21][22]. - The stock reached a 52-week high of $60, up from $40 when last featured [22]. Insmed Inc. (INSM) - Insmed has two approved drugs and is conducting a phase 3 trial of Arikayce, with topline results expected in 1H 2026 [24][25]. - The stock hit a high of $166.54, up from $76.54 when last featured [26]. Adaptive Biotechnologies Corp. (ADPT) - Adaptive Biotechnologies expects MRD revenue between $190 million and $200 million for 2025, up from $145.5 million in 2024 [28][29]. - The stock reached a 52-week high of $15.94, up from $9.80 when last featured [29]. BridgeBio Pharma Inc. (BBIO) - BridgeBio has upcoming topline results from the FORTIFY and CALIBRATE studies expected in Fall 2025 [30]. - The stock reached a 3-year high of $56.24, up from $25.10 when last featured [31]. Tarsus Pharmaceuticals Inc. (TARS) - Tarsus reported strong sales for Xdemvy, with Q2 2025 sales of $102.7 million, compared to $40.8 million in Q2 2024 [32]. - The stock hit an all-time high of $70.15, up from $25.01 when last featured [34]. Palvella Therapeutics Inc. (PVLA) - Palvella's QTORIN is under development for various skin diseases, with a phase 2 trial expected to report data in mid-December 2025 [35][36]. - The stock reached a 52-week high of $76.76, up from $25 when last featured [36]. Merus N.V. (MRUS) - Merus agreed to be acquired by Genmab for $97 per share, with the deal expected to close in early Q1 2026 [37]. - The stock was at $39.71 when last featured [39]. Nephros Inc. (NEPH) - Nephros reported net revenue of $4.4 million for Q2 2025, marking its third consecutive quarter of profitability [40][41]. - The stock hit a 52-week high of $5.98, up from $2.93 when last featured [42].

Core Insights - BridgeBio Pharma, Inc. (NASDAQ:BBIO) is recognized as one of the top biotech stocks to consider for investment, particularly due to the launch of its medication, Attruby [1] - UBS has maintained a Buy rating for BridgeBio, expressing optimism about the launch trajectory of Attruby, supported by a significant number of new patient diagnoses [1] Financial Potential - UBS estimates that Attruby could generate over $4 billion in long-term revenue, with its competitive edge highlighted by data showing its differences from the rival medication tafamidis [2] - The firm also notes that BridgeBio's pipeline beyond Attruby is "underappreciated," with three Phase 3 readouts expected in the next six months, which could collectively match Attruby's revenue potential [2] Company Overview - BridgeBio Pharma, Inc. is a biopharmaceutical company focused on discovering, developing, evaluating, and marketing innovative medications aimed at curing genetic diseases [3]

Core Insights - BridgeBio Pharma, Inc. (NASDAQ:BBIO) is recognized as a promising biotech stock by hedge funds, with Piper Sandler analysts maintaining an 'Overweight' rating and a price target of $68, indicating a potential upside of nearly 33% [1] - The company's third-quarter performance is expected to mirror the second quarter in terms of free-drug distribution and gross-to-net adjustments, with the German launch of Beyonttra (Attruby) showing stronger results than the initial U.S. launch due to wide access at approval [2] - BridgeBio has demonstrated impressive financial performance, achieving one-year and three-year returns of 100.67% and 413.98%, respectively, significantly outperforming the market returns of 15.34% and 85.36% [3] Company Overview - BridgeBio Pharma, Inc. is a California-based commercial-stage biopharmaceutical company focused on developing transformative medicines for genetic diseases and cancers, founded in 2015, and offers products such as Attruby and low-dose infigratinib [4]

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Core Insights - BridgeBio Pharma's heart-disease drug, Attruby, has shown promising results in reducing the risk of death by nearly 50% in an exploratory study, which could significantly impact treatment approaches for cardiomyopathy [1][2][4] Group 1: Drug Efficacy and Approval - Attruby is approved for treating cardiomyopathy due to transthyretin amyloidosis, with a 49% lower risk of death observed in patients after 30 months of treatment [2][5] - The drug's effects were noticeable within just one month of treatment, suggesting the need for earlier diagnosis and intervention [3][7] - Attruby has been marketed since late 2024, generating $5.9 million in its first quarter, which increased to $71.5 million in subsequent quarters, with a forecast of $94.9 million for the current quarter [5][6] Group 2: Market Potential and Competition - Analysts predict Attruby could become a blockbuster drug by 2027, competing against Pfizer's Vyndaqel, which generated $5.45 billion in sales last year [5][6] - The difference in outcomes between Attruby and placebo was significant, with 53 events prevented per 100 treated patients after 30 months [8] Group 3: Clinical Implications - The cardiologist Dr. Ahmad Masri emphasizes that Attruby could shift the treatment paradigm for TTR-related cardiomyopathy, focusing on preventing serious cardiovascular events [9][10] - Continuous treatment with Attruby is highlighted as crucial for improving patient longevity and quality of life, as recovery from heart failure episodes can be challenging [10]

Core Insights - Acoramidis has shown significant early and sustained clinical efficacy in reducing cumulative cardiovascular outcomes in patients with transthyretin amyloidosis cardiomyopathy (ATTR-CM) [1][2][3] Group 1: Clinical Efficacy - Acoramidis reduced cumulative cardiovascular mortality (CVM) or recurrent cardiovascular-related hospitalizations (CVH) by 49% through Month 30 compared to placebo, with a p-value of less than 0.0001 [1][3] - At Month 30, 53 events were avoided per 100 treated participants, with a 95% confidence interval of 29 to 79 [1][3] - Continuous treatment with acoramidis at Month 42 resulted in a 45% reduction in CVM compared to a switch from placebo to acoramidis, with a p-value of 0.0011 [3] Group 2: Patient Population Insights - Acoramidis demonstrated a consistent mitigation of NT-proBNP levels in patients with variant ATTR-CM, starting from Month 3 and continuing through Month 30 [3] - The findings highlight the importance of early and continuous treatment with acoramidis, regardless of TTR genotype, in both wild-type and variant ATTR-CM patients [3] Group 3: Regulatory Approvals - Acoramidis is approved as Attruby® by the U.S. FDA and as BEYONTTRA® by the European Commission, Japanese Pharmaceuticals and Medical Devices Agency, and the UK Medicines and Healthcare Products Regulatory Agency [4][5] Group 4: Future Developments - Additional data on the benefits of Attruby for ATTR-CM patients is planned for future medical meetings [4]

Core Insights - BridgeBio Pharma, Inc. is set to present significant findings related to its drug Acoramidis at the Heart Failure Society of America Annual Scientific Meeting 2025, highlighting its focus on genetic diseases [1][6]. Group 1: Clinical Presentations - A late-breaking clinical trial presentation will discuss the effect of Acoramidis on cardiovascular outcomes in patients with ATTR-CM, presented by Dr. Ahmad Masri on September 28 [2]. - An oral presentation will reveal that continuous treatment with Acoramidis significantly reduced the risk of all-cause mortality and cardiovascular-related hospitalization at month 42 for patients with transthyretin amyloidosis, presented by Dr. Lily Stern on September 27 [2]. Group 2: Poster Sessions - A poster session will present results showing that Acoramidis mitigates the rise in NT-proBNP levels compared to placebo in patients with variant transthyretin amyloid cardiomyopathy, presented by Dr. Nitasha Sarswat on September 27 [3]. - Another poster will discuss the effect of Acoramidis on cardiac conduction abnormalities in transthyretin amyloid cardiomyopathy, presented by Dr. Brett W. Sperry on September 27 [3]. - A poster will address state-level differences in the incidence of transthyretin amyloid cardiomyopathy among U.S. veterans after the introduction of disease-modifying therapy, presented by Dr. Sandesh Dev on September 28 [3]. Group 3: Product Information - Attruby™ (acoramidis) is indicated for treating cardiomyopathy associated with wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults, aimed at reducing cardiovascular death and related hospitalizations [4]. Group 4: Safety Information - Adverse reactions reported for Attruby include diarrhea (11.6% vs 7.6% for placebo) and upper abdominal pain (5.5% vs 1.4% for placebo), with most being mild and resolving without discontinuation of the drug [5].