- By examining over 100 unique variants associated with the CASR gene causative of ADH1, the average frequency of gain-of-function CASR variants was ~3.7 per 100,000, closely aligned to previously published estimates1 (3.9 per 100,000), which equates to approximately 25,000 carriers of ADH1-causing variants in the US and EU - Only ~20% of individuals with genetic variants linked to ADH1 were found to have an established diagnosis, highlighting a major gap in disease recognition and care - Nine novel gain-of ...

PALO ALTO, Calif., July 22, 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 it will release its second quarter financial results and business updates after the market closes on Tuesday, August 5, 2025. BridgeBio will host a conference call to discuss the financial results and program updates at 4:30 pm ET the same day. To access the live webcast of BridgeBio’s presentation ...

Summary of BridgeBio Pharma (BBIO) Update / Briefing July 11, 2025 Company Overview - **Company**: BridgeBio Pharma (BBIO) - **Focus**: Development of therapies for genetic diseases, specifically Limb Girdle Muscular Dystrophy (LGMD) Industry Context - **Disease**: Limb Girdle Muscular Dystrophy (LGMD), particularly LGMD2IR9FKRP related - **Patient Population**: Approximately 7,000 patients in the U.S. and EU with a focus on those with significant cardiac and respiratory involvement Key Points and Arguments 1. **Therapeutic Development**: The Phase III trial for BVP-four eighteen is fully enrolled, with interim data expected later this year to support an accelerated approval strategy [9][10][24] 2. **Clinical Trials**: The Phase II study has shown promising results, with a focus on increasing glycosylated alpha dystroglycan levels and reducing serum creatine kinase levels, indicating muscle injury reduction [30][40] 3. **FDA Engagement**: Positive discussions with the FDA regarding the use of glycosylated alpha dystroglycan as a surrogate endpoint for accelerated approval [45][48] 4. **Market Opportunity**: The company aims to be the first to market with a disease-modifying oral therapy, addressing a significant unmet need in the LGMD patient population [51][55] 5. **Patient Engagement**: Efforts to increase patient identification through partnerships with advocacy organizations and genetic testing programs [53][54] Additional Important Content 1. **Genetic Understanding**: The understanding of LGMD has evolved from clinical symptoms to identifying specific genetic mutations, particularly the FKRP gene associated with LGMD2IR9 [24][19] 2. **Clinical Impact**: The therapy aims to stabilize muscle function and potentially improve quality of life for patients, with a focus on slowing disease progression rather than just symptomatic relief [62][75] 3. **Safety Profile**: BVP-four eighteen has been well tolerated in trials, with only minor gastrointestinal side effects reported [38][82] 4. **Regulatory Strategy**: The company maintains a conservative approach to regulatory approval, emphasizing the need for flexibility given the lack of existing therapies for this patient population [80][81] Conclusion - BridgeBio Pharma is positioned to potentially deliver a first-of-its-kind therapy for LGMD, with ongoing clinical trials and strong regulatory engagement paving the way for future developments in the treatment of this genetic disorder. The focus on patient outcomes and safety, combined with a clear understanding of the disease's genetic basis, underscores the company's commitment to addressing significant unmet medical needs in the LGMD community.

Core Viewpoint - BridgeBio Pharma has entered into a royalty financing agreement, selling a portion of its royalties from BEYONTTRA sales in Europe for $300 million, which will provide immediate capital to support its operations and product launches [1][2][4]. Financial Details - The agreement allows BridgeBio to receive $300 million in exchange for 60% of royalties on the first $500 million of annual BEYONTTRA net sales in Europe, with total payments capped at 1.45 times the initial investment [3][4]. - BridgeBio has previously received $210 million in upfront and regulatory milestone payments from Bayer for BEYONTTRA and anticipates an additional $75 million in near-term milestone payments [4]. Product Information - BEYONTTRA is an orally administered stabilizer of transthyretin (TTR) for treating wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM) [6][8]. - Acoramidis, the active ingredient in BEYONTTRA, has shown significant clinical benefits, including a 42% reduction in composite adverse cardiovascular events and a 50% reduction in cumulative frequency of cardiovascular events at 30 months compared to placebo [6]. Strategic Partnerships - The partnership with HealthCare Royalty and Blue Owl Capital is aimed at strengthening BridgeBio's balance sheet to support the launch of Attruby and its pipeline of genetic medicines [2][3]. - HCRx has expressed confidence in the commercial potential of BEYONTTRA and is committed to supporting innovation in the biopharmaceutical industry through this investment [3].

PALO ALTO, Calif., June 26, 2025 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a new type of biopharmaceutical company focused on genetic diseases, today announced the Company will host an investor webinar on Friday, July 11, 2025 at 8:00 am ET with Matthew Wicklund, M.D., FAAN, Professor of Neurology and Vice Chair for Research, Department of Neurology at the University of Texas (UT) Health Science Center San Antonio. Dr. Wicklund will provide an overview of limb ...

Corporate Presentation April 29th, 2025 Forward Looking Statements and Disclaimer The presentation contains forward-looking statements. Statements made or presented 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, and Section 21E of the Securities Exchange Act of 1934, as amended. Words such as "believe," "anticipate," "plan," "expect," "intend," "will," "may," "goal," "potential," "should," " ...

Group 1 - BridgeBio Pharma (NASDAQ: BBIO) reported a significant earnings beat since the last coverage in April, yet the stock has remained relatively flat, underperforming major indexes [1] - The investment strategy focuses on identifying key information from earnings reports and transcripts that indicate potential growth opportunities [1] Group 2 - The article does not provide any additional relevant information regarding the company or industry [2][3]

Company Overview - BridgeBio Pharma, Inc. is a biopharmaceutical company focused on discovering, creating, testing, and delivering transformative medicines for genetic diseases [3] - Founded in 2015, the company has a pipeline that includes programs from early science to advanced clinical trials [3] - The team consists of experienced drug discoverers, developers, and innovators dedicated to applying advances in genetic medicine [3] Upcoming Event - Members of BridgeBio's management team will participate in a fireside chat at the Goldman Sachs 46th Annual Global Healthcare Conference 2025 on June 9 at 8:40 am ET [1] - A live webcast of the presentation will be available on the company's website, with a replay accessible for 30 days post-event [2]

Company Overview - Alignment Healthcare (ALHC) is currently ranked 5 in the Zacks Sector Rank within the Medical group, which consists of 999 companies [2] - The Zacks Rank system, which focuses on earnings estimates and revisions, has assigned ALHC a Zacks Rank of 2 (Buy) [3] Performance Metrics - The Zacks Consensus Estimate for ALHC's full-year earnings has increased by 34.1% over the past quarter, indicating improved analyst sentiment and a stronger earnings outlook [4] - Year-to-date, ALHC has returned 44.4%, significantly outperforming the average loss of 4.8% in the Medical group [4] Industry Context - Alignment Healthcare operates within the Medical Services industry, which includes 58 stocks and currently ranks 52 in the Zacks Industry Rank; this industry has seen an average loss of 1.9% this year, highlighting ALHC's relative strength [6] - In contrast, BridgeBio Pharma (BBIO), another outperforming stock in the Medical sector, belongs to the Medical - Generic Drugs industry, which has a lower ranking (198) and has declined by 15.1% this year [6] Future Outlook - Investors interested in Medical stocks should monitor Alignment Healthcare and BridgeBio Pharma for potential continued strong performance [7]

Core Insights - Acoramidis has shown significant clinical benefits in reducing the incidence of atrial fibrillation (AF) and cardiovascular-related hospitalizations in patients with transthyretin amyloid cardiomyopathy (ATTR-CM) [1][4][5] - The drug has received regulatory approvals in multiple regions, including the U.S. and Europe, indicating its potential as a treatment option for ATTR-CM [1][6] Group 1: Clinical Outcomes - In a post-hoc analysis of the ATTRibute-CM study, acoramidis reduced the annual frequency of cardiovascular hospitalizations (CVH) due to AF/atrial flutter (AFL) by 43% compared to placebo [1][4] - The incidence of new-onset AF/AFL was reduced by 17% in patients with no prior history of AF when treated with acoramidis [1][4] - A 42% reduction in composite all-cause mortality (ACM) and recurrent CVH events was observed at Month 30 relative to placebo [3] - Acoramidis treatment led to a greater than 50% reduction in ACM and CVH in participants with variant ATTR-CM compared to placebo [7] Group 2: Functional Capacity and Quality of Life - Patients with variant ATTR-CM treated with acoramidis showed a clinically significant slower decline in functional capacity and quality of life compared to placebo [7] - At Month 30, the mean difference in the 6-minute walk distance was 86.7 meters in favor of acoramidis (p = 0.0048) [7] - The KCCQ-OS score change from baseline at Month 30 was 20.3 points higher in the acoramidis group compared to placebo (p = 0.0019) [7] Group 3: Regulatory Approvals and Future Directions - Acoramidis is approved as Attruby™ by the U.S. FDA and as BEYONTTRA by the European Commission and other regulatory agencies [1][6] - The company plans to present more data on the benefits of acoramidis for ATTR-CM patients at future medical meetings [6]