Announcement • 1m

BridgeBio Pharma Submits NDA For Encaleret For Individuals Living With ADH1

BridgeBio Pharma submitted NDA to FDA for encaleret for individuals living with ADH1. Phase 3 CALIBRATE primary results were presented in an oral presentation at the 2026 ECE, demonstrating the rapid and durable benefit of encaleret across key clinical parameters in ADH1. All pre-specified primary and key secondary efficacy endpoints were met in the Phase 3 CALIBRATE trial; 76% of participants administered encaleret achieved both serum and urine calcium within the respective target ranges at Week 24 compared to 4% when on conventional therapy at Week 4 (p. Rapid and sustained improvements in calcium metabolism for participants randomized to encaleret, with increases in serum calcium observed by Day 3 and reductions in urine calcium by Week 3, maintained through Week 24. At Week 24, more participants randomized to encaleret achieved both target serum and urine calcium levels than participants who remained on standard of care (76% on encaleret vs. 19% on standard of care; p < 0.0001). Encaleret was observed to restore endogenous parathyroid hormone (91.1% on encaleret vs. 0% of participants on standard of care at Week 24). Favorable safety and tolerability profile, with no discontinuations in the encaleret arm and low rates of serious adverse events with frequency similar between treatment arms. BridgeBio anticipates a U.S. launch in early 2027. Nearly 2,000 individuals have been diagnosed in the U.S. with autosomal dominant hypocalcemia (ADH) since October 2023 based on claims data, suggestive of a growing marketplace and elevated diagnostic suspicion. The Company also intends to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the use of encaleret in ADH1 in the second half of 2026. BridgeBio is currently enrolling CALIBRATE-PEDS (NCT07080385), a global registrational Phase 2/3 study of encaleret in pediatric ADH1. The Company also plans to initiate RECLAIM-HP, a global Phase 3 study of encaleret in chronic hypoparathyroidism in Summer 2026, building on the Phase 2 proof-of-concept findings of PTH-independent effects of encaleret on renal calcium handling and expanding the potential applications of encaleret beyond ADH1. Encaleret is an investigational, orally administered small molecule under investigation to treat ADH1 and chronic hypoparathyroidism, that is designed to selectively negatively modulate the calcium sensing receptor. Encaleret has been granted Fast Track Designation by the U.S. FDA and Orphan Drug Designation in the U.S., European Union, and Japan.

Announcement • May 12

BridgeBio Pharma Announces Disease-Modifying Effects of Acoramidis in ATTR-CM

BridgeBio Pharma, Inc. announced new data from the Phase 3 ATTRibute-CM study at Heart Failure 2026, organized by the Heart Failure Association of the European Society of Cardiology (ESC-HF), further demonstrating acoramidis’ consistent and clinically meaningful benefit across the transthyretin amyloid cardiomyopathy (ATTR-CM) disease spectrum, with new data demonstrating disease modifying effects across clinical outcomes, biomarkers, and functional capacity. Acoramidis is the only selective small molecule, orally administered, near-complete (=90%) transthyretin (TTR) stabilizer. Acoramidis showed an association between the treatment-related increase in serum transthyretin (sTTR) and a significant reduction of sTTR variability over time, which is independently associated with reduced all-cause mortality (ACM). Findings included: Lower intraindividual sTTR variability and higher achieved sTTR levels were each independently associated with reduced risk of all-cause mortality (HR: 0.56; p=0.014 and HR: 0.46; p=0.014, respectively). Participants with both higher achieved sTTR levels and less variable sTTR levels experienced the greatest survival benefit, while higher sTTR variability was associated with adverse clinical features of ATTR-CM. Acoramidis increased sTTR levels early (Day 28) and sustained them through Month 30, while significantly reducing sTTR variability versus placebo (9.5% vs. 12.8%; p). Acoramidis further demonstrated early separation of outpatient worsening heart failure seen within 30 days and sustained through Month 30, showing the fastest time to impact of any disease modifying treatment in ATTR-CM. Acoramidis demonstrated a statistically significant, clinically meaningful 34% reduction in cardiovascular hospitalizations versus tafamidis in a matching-adjusted indirect comparison, with a favorable mortality trend and comparable safety. Acoramidis demonstrated a statistically significant, 34% reduction in cardiovascular hospitalizations versus tafamidis (RRR: 0.66; 95% CI: 0.46–0.95). Acoramidis showed a favorable mortality trend with 28% hazard reduction in all-cause mortality versus tafamidis, with consistent benefit across sensitivity analyses. Comparable safety profile observed between acoramidis and tafamidis. Acoramidis Treatment Attenuates the Rise in NT-proBNP from Baseline to Month 30 Compared to Placebo Across all Subgroups. In ATTRibute-CM, acoramidis consistently blunted the 30-month increase in NT-proBNP by about 50% across all participant subgroups assessed, including advanced disease, compared with placebo, demonstrating its robust efficacy on a key biomarker of ATTR-CM disease progression. Consistent Benefit on Kansas City Cardiomyopathy Questionnaire Overall Summary Score (KCCQ-OS) with Acoramidis Treatment Compared with Placebo Across Participant Subgroups in ATTRibute-CM. Acoramidis significantly attenuated the decline in heart failure-related health status compared with placebo in individuals with ATTR-CM. This effect was observed consistently across all pre-specified participant subgroups, including those with advanced heart failure symptoms. Effect of Acoramidis on Improvement or Maintenance of Heart Failure-Related Health Status as Assessed by KCCQ-OS Score in ATTRibute-CM. In individuals with ATTR-CM, acoramidis treatment was associated with a significantly greater likelihood of maintenance or improvement in health status compared with placebo, suggesting a clinically relevant modification of disease trajectory. These data provide an integrated assessment of health status and survival to inform medical decision making. Acoramidis is approved as Attruby® by the U.S. FDA and is approved as BEYONTTRA® by the European Medicines Agency (EMA), Japanese Pharmaceuticals and Medical Devices Agency, Swissmedic, the Swiss Agency for Therapeutic Products, the UK Medicines and Healthcare Products Regulatory Agency, and the Brazilian Health Regulatory Agency (ANVISA) with all labels specifying near-complete stabilization of TTR. Additional data on the benefit of Attruby for individuals with ATTR-CM is planned for future medical meetings. Attruby is a transthyretin stabilizer indicated for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization. Diarrhea (11.6% vs 7.6%) and upper abdominal pain (5.5% vs 1.4%) were reported in patients treated with Attruby versus placebo, respectively. The majority of these adverse reactions were mild and resolved without drug discontinuation. Discontinuation rates due to adverse events were similar between patients treated with Attruby versus placebo (9.3% and 8.5%, respectively).

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BridgeBio Pharma Submits NDA For Encaleret For Individuals Living With ADH1

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