BBP-631 / BridgeBio - LARVOL DELTA

Innovations in pharmacotherapy of CAH (ESPE 2024) - "Circadian delivery of hydrocortisone improves control of 21OHD compared to conventional hydrocortisone and over time results in lower glucocorticoid exposure...The P450 17A1 inhibitor abiraterone acetate directly blocks androgen production but at the expense of progesterone accumulation. The corticotropin-releasing factor type 1 receptor (CRF1) antagonists crinecerfont and tildacerfont have shown efficacy in suppressing ACTH and adrenal biomarkers in phase 2 trials...Gene therapy is also under study with an AAV5 vector (BBP-631). These modern approaches to deliver cortisol replacement and the suppress adrenal androgen production should simplify management, improve disease control, and mitigate the long-term consequences of 21OHD and chronic non-physiologic glucocorticoid exposure."

Congenital Adrenal Hyperplasia • Endocrine Disorders • Gene Therapies

A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH) (clinicaltrials.gov) - P1/2 | N=8 | Active, not recruiting | Sponsor: Adrenas Therapeutics Inc | Recruiting ➔ Active, not recruiting | N=25 ➔ 8 | Trial completion date: Nov 2028 ➔ Feb 2029 | Trial primary completion date: Dec 2024 ➔ Feb 2029

Enrollment change • Enrollment closed • Gene therapy • Trial completion date • Trial primary completion date • Congenital Adrenal Hyperplasia • Endocrine Disorders • Gene Therapies

A Study to Determine Eligibility for CAH-301 (A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH) [NCT04783181]) (clinicaltrials.gov) - P=N/A | N=65 | Completed | Sponsor: Adrenas Therapeutics Inc | Recruiting ➔ Completed | N=300 ➔ 65 | Trial completion date: Dec 2024 ➔ Aug 2024 | Trial primary completion date: Dec 2024 ➔ Aug 2024

Enrollment change • Gene therapy • Trial completion • Trial completion date • Trial primary completion date • Congenital Adrenal Hyperplasia • Endocrine Disorders • Gene Therapies

A Study to Determine Eligibility for CAH-301 (A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH) [NCT04783181]) (clinicaltrials.gov) - P=N/A | N=300 | Recruiting | Sponsor: Adrenas Therapeutics Inc | Trial completion date: Jun 2024 ➔ Dec 2024 | Trial primary completion date: Jun 2024 ➔ Dec 2024

Gene therapy • Trial completion date • Trial primary completion date • Congenital Adrenal Hyperplasia • Endocrine Disorders • Gene Therapies

A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH) (clinicaltrials.gov) - P1/2 | N=25 | Recruiting | Sponsor: Adrenas Therapeutics Inc | Trial primary completion date: Jun 2024 ➔ Dec 2024

Gene therapy • Trial primary completion date • Congenital Adrenal Hyperplasia • Endocrine Disorders • Gene Therapies

BridgeBio Pharma Reports Third Quarter 2023 Financial Results and Business Update (GlobeNewswire) - "The Phase 1/2 gene therapy trial of BBP-631 for CAH continues to progress, with the dose-escalation portion of the study (N=6) fully enrolled; the Company plans to share data from the program in early 2024....The Company and Resilience signed an agreement to transfer the manufacturing process for the Company’s lead AAV-based gene therapy candidates, BBP-631 and BBP-812, to Resilience’s network of gene therapy manufacturing sites. Resilience will also be the primary manufacturer for future clinical projects across the Company’s gene therapy portfolio."

Enrollment closed • Licensing / partnership • P1/2 data • Congenital Adrenal Hyperplasia