BridgeBio JPM 2025

Industry and Company Overview * Industry: Biotechnology and pharmaceuticals, focusing on rare and orphan diseases. * Company: BridgeBio Pharma, a clinical-stage biopharmaceutical company developing therapies for rare genetic diseases. Key Recent Achievements * Clinical Impact: * Publication of positive clinical trial results for Acoramidis (acoramidis) in Transthyretin Amyloid Cardiomyopathy (ATTR-CM) in the New England Journal of Medicine. * Publication of positive clinical trial results for Infigratinib in Achondroplasia in the New England Journal of Medicine. * Publication of positive clinical trial results for Encaleret in Autosomal Dominant Hypocalcemia Type 1 in the New England Journal of Medicine. * Regulatory Advancement: * Infigratinib received Breakthrough Therapy Designation (BTD) for Achondroplasia. * BBP-418 received Rare Pediatric Disease Designation (RPDD) for Limb-Girdle Muscular Dystrophy 2I/R9. * BBP-812 received Regenerative Medicine Advanced Therapy Designation (RMAT) for Canavan Disease. * Approval of Acoramidis (acoramidis) for the treatment of adult patients with ATTR-CM. * Commercial Momentum: * Acoramidis (acoramidis) scripts have been written to date. * Positive feedback from payers and healthcare providers regarding Acoramidis (acoramidis) market access. Pipeline Programs * Attruby (acoramidis): A treatment for ATTR-CM, with a global annual market sales of over $6.4 billion. * **Infigratinib**: A treatment for Achondroplasia and Hypochondroplasia, with a market opportunity of over$4 billion. * BBP-418: A treatment for Limb-Girdle Muscular Dystrophy 2I/R9, with a market opportunity of over $1 billion. * **Encaleret**: A treatment for Autosomal Dominant Hypocalcemia Type 1, with a market opportunity of over$2 billion. * BBP-812: A treatment for Canavan Disease, with a potential to change the disease trajectory for affected patients. * BridgeBio Oncology Therapeutics: A separate company focused on oncology therapies. * GondolaBio: A separate company focused on rare disease therapies. Financials and Future Outlook * BridgeBio Pharma is well-financed and expects to hit numerous milestones in 2025. * The company's vision for 2030 includes de-risking its pipeline, impacting over 100,000 lives, and achieving a market capitalization of over $8 billion.