SCYNEXIS Announces Phase 1 Study Results for SCY-770, a Novel, Highly Selective, Oral Direct AMPK Activator for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)

What happened

SCYNEXIS announced completion of a Phase 1 study of SCY-770 that evaluated safety, tolerability, and pharmacokinetics to support Phase 2 dose selection in ADPKD. The company reported the program has been evaluated across multiple clinical trials in approximately 300 participants to date and that SCY-770 has Orphan Drug Designation from the U.S. Food and Drug Administration. SCYNEXIS stated the Phase 2 proof-of-concept study remains on-track to initiate in the fourth quarter of 2026.

Sources: S1

Study details reported

The Phase 1 study enrolled 25 healthy participants across three cohorts. Dosing in the initial cohort was a single 500 mg dose under fed and fasted conditions. Subsequent cohorts received either 750 mg once daily or 500 mg twice daily, or placebo, for seven days. SCYNEXIS characterized SCY-770 as well tolerated across all cohorts with a safety profile consistent with prior clinical experience.

Sources: S1

Clinical and market context provided by the issuer

SCYNEXIS described SCY-770 as a novel, highly selective, oral direct activator of AMPK designed to target pathways implicated in ADPKD, including mTOR and cAMP signaling, and to address inflammation, fibrosis, and cellular metabolism. The release includes a prevalence estimate and comparator context: the company stated U.S. prevalence of ADPKD is estimated to be 140,000 patients with approximately 6,000 new cases annually and noted that Jynarque (tolvaptan) is the only approved therapy, achieving approximately $1.5 billion in U.S. sales in 2024.

Sources: S1

Next steps and company position

SCYNEXIS said pharmacokinetic data from this and prior studies will inform Phase 2 dose selection and that the Phase 2 proof-of-concept study in patients with ADPKD is on-track to begin in the fourth quarter of 2026. The release reiterated the company’s view that SCY-770 could offer a differentiated, multi-mechanistic approach to treating ADPKD.

Sources: S1

What remains uncertain

• All clinical and development statements are from the company press release and have not been independently verified by this report.
• The press release does not disclose detailed safety event listings, laboratory results, or specific pharmacokinetic parameters from the Phase 1 study.
• The timing and initiation of the Phase 2 study described as "on-track to initiate in the fourth quarter of 2026" are forward-looking statements that may be affected by regulatory review, operational execution, or other risks noted by the issuer.