BridgeBio (BBIO) 12th Annual Cantor Fitzgerald Global Healthcare Conference summary

Strategic overview and pipeline updates

• Positioned at an inflection point with $8 billion in peak year sales assets, including four major indications: ATTR-CM, limb-girdle, achondroplasia, and ADH1, with launches spanning late 2024 to early 2025.

• Expansion opportunities include hypoparathyroidism and early-stage assets via a sister company, GondolaBio, broadening the genetic disease portfolio.

• Strong financial position with $1.7 billion in cash as of July 1, supporting global commercialization plans.

ATTR-CM (Attruby) market positioning and outlook

• Attruby, a second-generation stabilizer, is differentiated by rapid onset, kidney protection, and superior outcomes versus first-generation therapies.

• Holds over 25% share in frontline patients, targeting 30–40% at peak in the U.S., with higher penetration already seen in Europe.

• Anticipates $25–30 million quarterly sales growth for the next 3–4 quarters, with acceleration expected by late 2027 or early 2028.

• Ongoing claims and real-world data analyses are expected to further support clinical differentiation, especially at upcoming HFSA meetings.

• Confident in maintaining market share post-generic entry due to specialty pharmacy dynamics and clinical superiority.

BBP-418 (Limb-girdle muscular dystrophy) development and commercialization

• PDUFA date set for November 27, 2024, with strong clinical data supporting approval and potential for traditional approval without confirmatory studies.

• U.S. patient population estimated at 2,000–2,500, with 500 genetically confirmed and identified; ongoing efforts to expand identification.

• Majority of identified patients are candidates for therapy, with broad inclusion criteria and strong anticipated adoption.

• Pricing expected to be at a premium to exon-skipping therapies due to demonstrated functional benefits.