SCYNEXIS (SCYX) 3rd Annual Guggenheim Healthcare Innovation Conference summary

Portfolio strategy

• Portfolio now spans SCY-770 for ADPKD and SCY-247 for invasive fungal disease, following expansion beyond antifungal development into rare disease.

• BREXAFEMME is fully out-licensed to GSK; future sales milestones may be payable.

SCY-770 and ADPKD

• SCY-770 is a selective AMPK activator intended to affect cyst-growth pathways, fluid secretion and inflammation; preferential binding to kidney-associated AMPK forms may support safety.

• About 300 people have received the molecule in clinical trials; recently completed Phase I doses were well tolerated and informed population PK modeling for Phase II.

• Phase II is planned to start before year-end 2026, subject to final FDA design discussions; proposed design is one year, two SCY-770 dose groups and placebo, enrolling rapid progressors.

• Interim analysis is targeted for Q4 2027, with total kidney volume (TKV) as a key early efficacy measure; full study completion is expected in 2028.

• Placebo patients in comparable studies typically show ~5% annual TKV growth; tolvaptan cut this by 50% relative, a reduction viewed as a potential efficacy benchmark for SCY-770.

ADPKD market and development path

• Tolvaptan is the only marketed ADPKD therapy, with $1.5B in 2024 sales despite safety and tolerability limitations; multiple approaches are in development.

• SCY-770 acts downstream of genetic mutations and could theoretically complement other therapies, although combination development is not the primary plan.

• Phase II and Phase III are expected to assess both TKV and GFR; TKV may support potential accelerated approval after one year in Phase III, with GFR evidence ultimately needed.

• Current development outline is one Phase II study followed by one Phase III study, with Phase II results guiding Phase III planning.