BridgeBio (BBIO) 2024 Cantor Fitzgerald Global Healthcare Conference summary

Commercial and clinical pipeline updates

• Focused on commercial preparation for acoramidis in ATTR cardiomyopathy and advancing late-stage phase III programs including achondroplasia, ADH1, and LGMD2I.

• Acoramidis data shows significant survival benefit, with up to 100% survival at 30 months in some settings, and a strong correlation between increased serum TTR and reduced mortality.

• Early commercial strategy targets new and undiagnosed patients, with efforts to expand diagnosis using AI and partnerships with cardiology labs.

• Sales force and market access teams are fully hired, drawing on rare disease commercialization experience and industry talent.

• Competitive landscape includes tafamidis and vutrisiran, with acoramidis positioned as a potent stabilizer; future market may see combination therapies.

Market dynamics and education

• Education efforts are in early stages, with initial adopters expected from clinical trial sites and experienced practitioners.

• 15%-40% of tafamidis patients are considered progressors, representing a key opportunity for conversion to acoramidis.

• AI-driven diagnostic initiatives aim to identify undiagnosed patients, especially in underserved populations such as the African American community.

• Emphasis on data-driven differentiation and ease of access for prescribers and patients to compete with established players.

• Patent landscape for tafamidis may extend exclusivity to 2035, impacting generic competition timing.

Pipeline highlights and future outlook

• Infigratinib received FDA breakthrough designation for achondroplasia, with data showing improvements in growth velocity and proportionality; pivotal data expected by end of 2025.

• Dose optimization for infigratinib is ongoing, with most patients responding at current levels; higher doses may be explored post-approval.

• Hypochondroplasia phase II is underway, with potential expansion into other FGFR-driven skeletal dysplasias and rare growth disorders.

• Encaleret for ADH1 is a first-in-class program with a tight efficacy profile, targeting 3,000-5,000 identified U.S. patients and broader outreach through EMR and AI-based identification.

• BBP-418 for LGMD2I aims for interim data next year, focusing on glycosylation of alpha dystroglycan as a primary endpoint for accelerated approval.