- In Cohort 5 of PROPEL 2 (0.25 mg/kg/day), oral treatment with infigratinib resulted in a statistically significant and sustained increase in annualized height velocity (AHV), with a mean change from baseline of +2.51cm/yr at Month 12, and +2.50 cm/yr at Month 18 (p=0.0015) - At Month 18, there was a statistically significant improvement in body proportionality (p-value of 0.001). The mean upper to lower body segment ratio was 1.88 at Month 18, as compared to 2.02 at baseline - Infigratinib continues to b ...

Core Insights - BridgeBio Pharma announced sustained positive results from the PROPEL 2 Phase 2 trial of infigratinib in children with achondroplasia, indicating potential best-in-class efficacy and a favorable safety profile [1][2] Group 1: Clinical Trial Results - In Cohort 5 of PROPEL 2, infigratinib treatment resulted in 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 (p=0.0015) [1][2] - There was a statistically significant improvement in body proportionality, with the mean upper to lower body segment ratio changing from 2.02 at baseline to 1.88 at Month 18 (p=0.001) [1][2] - Infigratinib was well-tolerated, with no treatment-related adverse events reported in Cohort 5 [1][2] Group 2: Ongoing Studies and Future Plans - PROPEL 3, the global Phase 3 registrational study of infigratinib in achondroplasia, is on schedule for enrollment completion by the end of 2024 [1][3] - BridgeBio is expanding the development of infigratinib for hypochondroplasia, with the first child consented in the ACCEL observational study [1][4] - The ACCEL program will include a Phase 2/3 multicenter study to evaluate the efficacy and safety of infigratinib in children with hypochondroplasia [4] Group 3: Company Commitment and Community Impact - BridgeBio is committed to exploring the potential of infigratinib for broader medical and functional impacts on skeletal dysplasias, addressing significant unmet needs for families [7][11] - The company has received positive feedback from the U.S. FDA and EMA regarding the development of infigratinib for hypochondroplasia [4][5] - The Chandler Project, a patient advocacy organization, emphasizes the importance of addressing the needs of the skeletal dysplasia community through collaborative research efforts [6]

Group 1 - BridgeBio Pharma, Inc. is a commercial-stage biopharmaceutical company focused on genetic diseases [2] - The company will host a call on June 4, 2024, to share results from Month 12 and Month 18 of Cohort 5 of the PROPEL 2 trial [1] - The PROPEL 2 trial is a Phase 2 study investigating the oral therapy infigratinib in children with achondroplasia [1] Group 2 - BridgeBio was founded in 2015 and aims to discover, create, test, and deliver transformative medicines for genetic diseases [2] - The company's pipeline includes programs ranging from early science to advanced clinical trials [2] - BridgeBio's team is committed to applying advances in genetic medicine to help patients quickly [2]

PALO ALTO, Calif., June 03, 2024 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio), a commercial-stage biopharmaceutical company focused on genetic diseases, today announced that members of its management team, along with Ravi Savarirayan, M.D., Ph.D., of Murdoch Children’s Research Institute in Melbourne, Australia, and the global lead investigator for PROPEL 2, will host a call on June 4, 2024, at 8:00 am ET to share results from Month 12 and Month 18 from Cohort 5 of PROPEL 2, a Phase ...

- Acoramidis treatment resulted in increased serum transthyretin (TTR) levels by Day 28 that were sustained and were correlated with a reduced risk of all-cause mortality (ACM), cardiovascular mortality (CVM), and cardiovascular-related hospitalization (CVH) in ATTR-CM participants through month 30 - Acoramidis treatment resulted in a significant improvement in the composite endpoint of CVM and CVH in ATTR-CM participants, with benefit evident as early as Month 3 - In ATTRibute-CM, participants with at lea ...

- Acoramidis treatment resulted in increased serum transthyretin (TTR) levels by Day 28 that were sustained and were correlated with a reduced risk of all-cause mortality (ACM), cardiovascular mortality (CVM), and cardiovascular-related hospitalization (CVH) in ATTR-CM participants through month 30 - Acoramidis treatment resulted in a significant improvement in the composite endpoint of CVM and CVH in ATTR-CM participants, with benefit evident as early as Month 3 - In ATTRibute-CM, participants with at lea ...

- BridgeBio to host investor call on Wednesday, May 29, 2024 at 5:30 pm ET, with presentations from Mathew Maurer, M.D. of Columbia University Irving Medical Center, U.S. and Ahmad Masri, M.D., M.S. of Oregon Health & Science University, U.S. PALO ALTO, Calif., May 24, 2024 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, announced today that 12 oral and moderated poster presentatio ...

- BridgeBio to host investor call on Wednesday, May 29, 2024 at 5:30 pm ET, with presentations from Mathew Maurer, M.D. of Columbia University Irving Medical Center, U.S. and Ahmad Masri, M.D., M.S. of Oregon Health & Science University, U.S. PALO ALTO, Calif., May 24, 2024 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (NASDAQ:BBIO) ("BridgeBio" or the "Company"), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, announced today that 12 oral and moderated poster presentation ...

PALO ALTO, Calif., May 14, 2024 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (NASDAQ:BBIO) ("BridgeBio" or the "Company"), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced that members of its management team will present at the Bank of America Merrill Lynch Global Healthcare Conference 2024 in Las Vegas, NV on Wednesday, May 15 at 3:00 pm PT. To access the live webcast of BridgeBio's presentation, please visit the "Events & Presentations" page within the In ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 Title of each class Trading Symbol(s) Name of each exchange on which registered Common Stock, par value $0.001 per share BBIO The Nasdaq Global Select Market FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2024 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ...