BridgeBio Pharma, Inc. · NASDAQ:BBIO
BridgeBio Pharma — 2025 NTSAD Family Conference
14 slides from BridgeBio Pharma's conference presentation, Apr 25, 2025
Slide 1: Aspa Therapeutics' BBP-812 Gene Therapy Program for Canavan Disease: Updates
Aspa Therapeutics’ BBP-812 Gene Therapy Program for Canavan Disease: Updates Mitra Tavakkoli, MD, PharmD Aspa Program Global Medical Lead April 25, 2025 MAT-US-BBP812-0008 20250418 © 2025 BridgeBio Gene Therapy, LLC. Courtesy from and proprietary to Aspa Therapeutics, a BridgeBio company
Slide 2: Natural History Study (CVN-101)
Natural History Study (CVN-101) © 2025 BridgeBio Gene Therapy, LLC. Courtesy from and proprietary to Aspa Therapeutics, a BridgeBio company
Slide 3: Study Updates: Natural History Study
Study Updates Natural History Study • 67 participants enrolled from 17 countries • Current sites: Mass. General Hospital (MGH), Boston, MA; University Clinic (UKE), Hamburg, Germany; University of California, San Francisco (UCSF) Oakland, CA UKE UCSF MGH 3 © 2025 BridgeBio Gene Therapy, LLC. Courtes
Slide 4: Aspa Published Data from Natural History Study
Aspa Published Data from Natural History Natural History Study Study Mild phenotype is defined as achieved the CDC milestone “pulls to stand/walks holding on”; Cr, creatinine; NAA, N-acetylaspartic acid. Nagy A, et al, Urine N-Acetylaspartate Distinguishes Phenotypes in Canavan Disease. Hum Gene The
Slide 5: Key Learnings
Key Learnings Natural History Study • Thank you! • Natural history data show little to no gains in motor function in individuals with Canavan between the age of 0-60 months • This highlights the difficulty in gaining and maintaining skills in this population. HINE – 2 Sitting Age (Months) As of Sept
Slide 6: Gene Therapy Clinical Trial (CVN-102)
Gene Therapy Clinical Trial (CVN-102) © 2025 BridgeBio Gene Therapy, LLC. Courtesy from and proprietary to Aspa Therapeutics, a BridgeBio company
Slide 7: Important Disclaimer
Important Disclaimer Gene Therapy Clinical Trial The following slides discuss Aspa’s investigational gene therapy BBP-812 which has not been approved by the FDA or other regulatory authorities. 7
Slide 8: Individual Experience Study Overview
Individual Experience Study investigators review potential participants for screening on an ongoing basis 6 wks. 1 year 4 years Participant Screen Initial Follow-Up Long-Term Follow-Up Dosing Study Overview Dose Level 1 Gene Therapy Clinical Trial Dose Level 2 Dose Selection and Expansion 8 © 2025 B
Slide 9: Updates – Enrollment and Safety
Updates – Enrollment and Safety Gene Therapy Clinical Trial Dosed Participants 8 6 14 Participants at Participants at Total Participants Low Dose High Dose Safety Intravenous (IV) infusions were well-tolerated with no infusion reactions All participants experienced at least 1 adverse event (AE); mos
Slide 10: Emerging dose-dependent reductions in urine NAA
Emerging dose-dependent reductions in Gene Therapy Clinical Trial urine NAA • Post-dose urine NAA levels drop to range seen in milder clinical presentation (phenotypes) • High dose urine NAA levels are beginning to separate from low dose levels. 10 © 2025 BridgeBio Gene Therapy, LLC. Courtesy from a
Slide 11: Change in HINE-2 Total Score
Preliminary Data Show That Children Dosed With BBP-812 Have Improved Motor Function Over Time Compared to Natural Gene Therapy Clinical Trial History (HINE-2) Change in HINE-2 Total Score Months since Baseline Natural Treatment - Low Treatment - High History As of December 2024 11 © 2025 BridgeBio G
Slide 12: More Understanding to Come
More Understanding to Come Gene Therapy Clinical Trial • While these early results are encouraging, more participants will need to receive the investigational gene therapy and longer duration data are needed in order to fully understand the safety and efficacy of BBP-812. • The results shown may not
Slide 13: Thank you!
Thank you! 13 © 2025 BridgeBio Gene Therapy, LLC. Courtesy from and proprietary to Aspa Therapeutics, a BridgeBio company
Slide 14: Human Gene Therapy NTSAD
clinicaltrials.gov/study/NCT04126005 clinicaltrials.gov/study/NCT04998396 treatcanavan.com Human Gene Therapy NTSAD Urine NAA Publication 2024 Presentation MAT-US--162 14 © 2025 BridgeBio Gene Therapy, LLC. Courtesy from and proprietary to Aspa Therapeutics, a BridgeBio company