BridgeBio (BBIO) Barclays 28th Annual Global Healthcare Conference summary

Patent litigation and market dynamics

• Ongoing litigation over Vyndamax patents focuses on infringement and validity, with strong scientific evidence supporting the infringement case and expert testimony expected to be influential.

• Vyndaqel generics are not expected to significantly impact the market due to brand discontinuation, lack of substitutability, and payer dynamics, with Vyndamax remaining the key brand.

• Patient copays are uniform due to Medicare, and branded products are preferred by ISPs, supporting continued research and brand dominance.

Pipeline progress and clinical data

• ADH1 presents a large unmet need with compelling Phase III data, showing over 75% normalization and a significant proportion of patients achieving normal PTH levels.

• The ADH1 patient population is sizable but underdiagnosed, with 3,000–4,000 identified patients ready for launch and a safe, highly effective oral therapy available.

• Limb-girdle 2I (LGMD2I) interim Phase III data show significant biomarker and functional improvements, with consistent results across subpopulations and a U.S. patient base of 1,200–1,500.

• Regulatory discussions for LGMD2I are oriented toward full approval, with European engagement expected in two months.

Commercial strategy and launch dynamics

• Commercial build-out leverages a decentralized affiliate model connected to a centralized U.S. infrastructure, aiming for efficient launches without large field forces.

• Ex-U.S. launches benefit from concentrated centers of excellence and global trial experience, facilitating patient identification and market entry.

• New patient starts for ATTR therapies accelerated due to increased physician education, EMR tools, and compelling subpopulation data, especially in high-volume heart failure practices.

• Price evolution in the U.S. remains upward due to channel dynamics, with generous access programs supporting patient and physician adoption; no dramatic near-term price changes expected.