Longboard Pharmaceuticals (LBPH) H.C. Wainwright 26th Annual Global Investment Conference 2024 summary

Company formation and strategic partnerships

• Spun out from Arena in 2020 to focus on neuro assets with a specialized team.

• Arena was acquired by Pfizer in 2022; Pfizer receives royalties but holds no equity.

• Pipeline is based on highly selective GPCR-targeting compounds for neurological diseases.

Bexicaserin (LP352) program highlights

• Bexicaserin is a selective 5-HT2C super agonist, designed to avoid 2B and 2A receptor side effects.

• Oral delivery chosen for suitability in patients, many of whom use feeding tubes.

• Targets developmental and epileptic encephalopathies (DEEs), a group with significant unmet need.

• Estimated 150,000–250,000 DEE patients in the U.S., with a $6 billion market opportunity.

Clinical data and regulatory progress

• PACIFIC phase Ib/IIa study showed 60% seizure reduction for bexicaserin vs. 17% for placebo; 42% placebo-adjusted reduction.

• Open label extension showed 58% seizure reduction and high patient retention at nine months.

• Received FDA breakthrough therapy designation for DEEs, enabling a broad phase III program.

• Phase III will include studies in Dravet, LGS, and other DEEs, with details to be shared at R&D Day.