SCYNEXIS (SCYX) Corporate presentation summary

Portfolio overview

• Pipeline targets severe rare diseases: SCY-770 for ADPKD, SCY-247 for invasive fungal infections, and Fungerp analogs for resistant fungi; BREXAFEMME is partnered with GSK and approved.

• SCY-770 is a first-in-class, selective oral direct AMPK activator with FDA Orphan Drug Designation; its Phase 2 ADPKD study is planned to start in Q4 2026.

• SCY-247 is a next-generation Fungerp with QIDP, Fast Track, and Orphan Drug designations; oral and IV Phase 1 studies are complete, with Phase 2 expected in 2027.

• BREXAFEMME is licensed to GSK; the presentation cites up to ~$146M in sales milestones plus royalties following relaunch.

SCY-770 in ADPKD

• ADPKD affects >140K U.S. patients, with ~6K new cases yearly; half reach end-stage kidney disease by age 60, while Jynarque had ~11K U.S. patients and $1.5B in 2024 U.S. sales.

• The proposed unmet need is an oral therapy that preserves kidney function without liver risk or heavy urine output; Jynarque carries a boxed warning for serious and fatal liver injury and requires a REMS monitoring program.

• SCY-770 preferentially binds kidney-common β1-containing AMPK heterotrimers; the presentation cites a strong preclinical rationale and FDA-endorsed total kidney volume (TKV) surrogate endpoint for potential accelerated approval.

• Phase 1 enrolled 25 healthy participants across three cohorts; SCY-770 was well tolerated at all doses, with loose stools the most common adverse event, and exposures reached the efficacy target for dose selection.

• Phase 2 initiation is planned for Q4 2026, with interim TKV analysis expected in Q4 2027 and study completion in 2H 2028; the illustrative plan targets accelerated approval using TKV and full approval based on eGFR.

SCY-247 and antifungal opportunity

• Invasive candidiasis treatment carries a 20–40% mortality rate and ~70K U.S. cases treated annually; the presentation estimates a U.S. commercial opportunity above $700M annually across treatment and prophylaxis.

• SCY-247 is described as fungicidal against Candida, including multidrug-resistant C. auris and echinocandin-resistant C. glabrata, with activity against azole-resistant Aspergillus and IV plus oral formulations.

• The profile highlights deep-tissue distribution, low drug-interaction risk, and favorable safety in ~148 trial participants; its glucan synthase inhibitor mechanism has a differentiated binding site absent in human cells.

• BARDA funding could total up to $214M if all options are exercised, including ~$18.5M in base-period funding; the cost-share contract supports development through NDA submission without equity dilution.