Core Insights - Encaleret demonstrated significant efficacy in normalizing blood and urine calcium levels in post-surgical hypoparathyroidism patients, with 80% achieving normal levels within 5 days of treatment initiation compared to 0% on conventional therapy at baseline [1][6] - The treatment was well-tolerated, with no serious adverse events reported during the study [1] - BridgeBio plans to initiate a registrational clinical study for encaleret in chronic hypoparathyroidism in 2026 based on the promising Phase 2 results [1][3] Company Overview - BridgeBio Pharma, Inc. is focused on developing transformative medicines for genetic diseases, with a pipeline that includes early science to advanced clinical trials [5] - The company was founded in 2015 and aims to leverage advances in genetic medicine to expedite patient care [5] Study Details - The Phase 2 proof-of-concept study evaluated the PTH-independent effects of encaleret on renal calcium handling in ten participants with post-surgical hypoparathyroidism, administered at 162 mg twice daily for up to 5 days [2] - Calcitriol was stopped one day prior to the first dose, and calcium levels were titrated based on blood calcium after starting encaleret [2] Additional Findings - Encaleret treatment resulted in a rapid and sustained reduction in fractional excretion of calcium in nine participants, supporting its potential as an orally-administered treatment option for chronic hypoparathyroidism [6] - Data from preclinical studies of infigratinib were also presented, showing significant improvements in bone growth and skull development in relevant mouse models, indicating broad potential for treating skeletal conditions [4]

BridgeBio Pharma (BBIO) 2025 Conference September 04, 2025 02:10 PM ET Company ParticipantsJosh Schimmer - Managing DirectorChinmay Shukla - SVP - Strategic Finance & IRChristine Siu - CEO - ML Bio SolutionsAnanth Sridhar - COOJosh SchimmerAll right, we're ready to get started. Welcome, everyone. I'm Josh Shummer from the Kantar Biotech Equity Research Team. Very pleased to introduce some of the management team from BridgeBio Pharma. We have Christine Xu, CEO of ML Bio Solutions. That's the limb-girdle musc ...

BridgeBio Pharma (BBIO) FY 2025 Conference September 03, 2025 09:30 AM ET Company ParticipantsTiago Fauth - Director - Equity Research Small/Mid Cap BiotechnologyNone - ExecutiveAnanth Sridhar - COOTiago FauthAll right, perfect. Thanks, everyone, for joining us. I'm Chad Lefauche, I'm a biotech analyst here at Wells Fargo. We're joined today by the BridgeBio team. We're going to have a fireside chat, focus a little bit on the pipeline and some upcoming catalysts that we have. Towards the end, we'll talk a l ...

Financial Data and Key Metrics Changes - The company reported a significant increase in the number of treatment-naive patients on acoramidis, indicating a positive trend in commercial execution [47][53] - The gross-to-net ratio is expected to stabilize in the range of 30% to 40%, reflecting a more representative payer mix as of Q2 [55][56] Business Line Data and Key Metrics Changes - The ADH-1 program is transitioning from a single-center phase two study to a global phase three study with over 25 sites, which may lead to a more heterogeneous patient population [3][4] - In the phase two study of ADH-1, approximately 70% of patients responded to encaleret, and the company aims to replicate this success in phase three [16][38] Market Data and Key Metrics Changes - The prevalence of ADH-1 has been estimated at about 1 in 25,000 based on data from multiple biobanks, indicating a consistent finding across different cohorts [18][19] - The company is comparing the patient identification and market development strategies for ADH-1 to those used in the ATTR field, which has seen significant improvements in diagnosis rates [22] Company Strategy and Development Direction - The company is focused on driving disease education and the utilization of genetic testing to improve diagnosis rates for ADH-1 [19] - The strategy for encaleret includes differentiating the product through urine calcium reduction and long-term safety, aiming to establish it as a standard of care [27][28] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the launch of acoramidis, noting that the share of treatment-naive patients continues to increase, which is a positive indicator for long-term success [47][48] - The company is optimistic about the upcoming data presentations that may address skepticism regarding the efficacy of encaleret in chronic hypoparathyroidism [25][26] Other Important Information - The company is preparing for a webinar to provide more details on the ADH-1 program, indicating a commitment to transparency and stakeholder engagement [3] - Management highlighted the importance of compelling clinical data and new treatment guidelines in driving diagnosis rates for ADH-1 [13][14] Q&A Session Summary Question: What are the risks in translating phase two findings to phase three for ADH-1? - The main difference is the number of sites and patients, with phase three being a global study involving over 25 sites [3] Question: Why is PTH replacement therapy not optimal for ADH-1 patients? - PTH replacement does not address the root cause of the condition, and urine calcium levels remain high even with treatment [10][11] Question: How is the company addressing patient identification for ADH-1? - The company is focusing on compelling clinical data, new treatment guidelines recommending genetic testing, and increased awareness through ICD-10 coding [13][14][18] Question: What is the expected impact of competition on acoramidis? - The company has not seen significant pressure from competitors and maintains a strategy of parity access without needing to offer larger rebates [57][59] Question: What is the confidence level regarding the safety profile of infigratinib? - The phase two data provided confidence in a clean safety profile, and the company expects to continue monitoring safety closely in phase three [65][66]

Financial Data and Key Metrics Changes - The company reported a significant increase in the number of treatment-naive patients on acoramidis, indicating a positive trend in commercial execution [47][53] - The gross-to-net ratio is expected to stabilize in the range of 30% to 40%, reflecting a more representative payer mix as of Q2 [55][56] Business Line Data and Key Metrics Changes - The ADH-1 program is transitioning from a single-center phase two study to a global phase three study with over 25 sites, which may lead to a more heterogeneous patient population [3][4] - In the phase two study of ADH-1, approximately 70% of patients responded to encaleret, and the company aims to replicate this success in phase three [16][38] Market Data and Key Metrics Changes - The prevalence of ADH-1 has been estimated at about 1 in 25,000, consistent across multiple biobanks, indicating a stable understanding of the patient population [18][19] - The company is comparing its market potential to that of ATTR and X-linked hypophosphatemia, suggesting a similar trajectory in patient identification and market development [22][23] Company Strategy and Development Direction - The company is focused on driving disease education and the utilization of genetic testing to improve diagnosis rates for ADH-1 [19] - The strategy includes leveraging compelling clinical data and new treatment guidelines to enhance awareness and diagnosis of the condition [13][14] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the launch of acoramidis, noting that the share of treatment-naive patients continues to increase, which is a positive indicator for long-term success [47][52] - The company is optimistic about the upcoming data presentations that may address skepticism regarding the efficacy of encaleret in chronic hypoparathyroidism [25][66] Other Important Information - The company is preparing for a webinar to provide more details on the ADH-1 program, indicating a commitment to transparency and stakeholder engagement [3] - Management highlighted the importance of long-term safety and efficacy data for encaleret, particularly in relation to urine calcium reduction, which could differentiate it in the market [27][66] Q&A Session Summary Question: What are the risks in translating phase two findings to phase three for ADH-1? - The main difference is the number of sites and patients, with phase three being a global study involving over 25 sites [3] Question: Why is PTH replacement therapy not optimal for ADH-1 patients? - PTH replacement does not address the root cause of the condition, and urine calcium levels remain high even with treatment [10][11] Question: How is the company addressing patient identification for ADH-1? - The company is focusing on compelling clinical data, new treatment guidelines recommending genetic testing, and increased awareness through ICD-10 coding [13][14][18] Question: What is the expected impact of competition on acoramidis? - The company has not seen significant pressure from competitors and maintains a strategy of parity access without needing to offer larger rebates [57][58] Question: How does the company view the potential for generic competition for tafamidis? - The company believes that the existing patents provide strong protection against generics, and the discontinuation of Vyndaqel complicates entry for potential competitors [60][61]

PALO ALTO, Calif., Sept. 03, 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 Wednesday, September 10, 2025 at 8:00 am ET with Rachel Gafni, M.D., Senior Research Physician at the National Institute of Dental and Craniofacial Research, part of the National Institutes of Health (NIH) and Principal Investigator and Steering Committee Co ...

Pipeline and Approvals - Attruby (acoramidis) is now FDA approved in the US for Transthyretin Amyloidosis (ATTR-CM)[8, 40, 42] - Beyonttra (acoramidis) is approved in the EU, Japan, and UK[11, 32] - Three Phase 3 trials are fully enrolled: BBP-418 for Limb-Girdle Muscular Dystrophy 2I/R9 (112 patients), encaleret for Autosomal Dominant Hypocalcemia Type 1 (71 patients), and infigratinib for Achondroplasia (114 participants)[12] Attruby (acoramidis) Clinical and Commercial Performance - Attruby demonstrated a 42% reduction in the composite of all-cause mortality and recurrent cardiovascular-related hospitalization events at Month 30[44] - Attruby showed a 50% reduction in the cumulative frequency of cardiovascular-related hospitalization events at Month 30[44] - In Q1 2025, Attruby generated $36.7 million in total U S revenue[51] - Attruby has achieved 77% Medicare lives in equal formulary position to tafamidis[51] Market and Financial Outlook - The global annual ATTR market is projected to reach $15-$20 billion at its peak[30, 66] - BridgeBio envisions a vision for 2030 with >$8 billion in revenue[35] - The company estimates the market opportunity for Infigratinib in Achondroplasia and Hypochondroplasia to be $2B+ each[30] - The company estimates the market opportunity for BBP-418 in Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9) to be $1B+[30, 146] - The company estimates the market opportunity for Encaleret in Autosomal Dominant Hypocalcemia Type 1 (ADH1) to be $1B+[30, 121]