SCYX
Live News • Jul 01
SCYNEXIS Begins Phase 1 Trial of SCY-770 for Polycystic Kidney Disease After FDA Orphan Status SCYNEXIS has started a Phase 1 clinical study of SCY-770, a novel AMPK activator for Autosomal Dominant Polycystic Kidney Disease, with the program already granted FDA Orphan Drug Designation. The company aims to use Phase 1 data to support a Phase 2 proof-of-concept trial planned for Q4 2026.
This new program moves SCYNEXIS into a different therapeutic area beyond its existing focus and could broaden its pipeline, with Orphan Drug Designation potentially offering regulatory benefits if SCY-770 progresses.
SCYNEXIS shares trade at US$4.07, with the stock down 42.7% over the past 90 days, which indicates recent market caution around the company.
The key takeaway is that SCY-770 adds early-stage, high-risk development optionality to SCYNEXIS, and outcomes from this ADPKD program could influence future valuation and funding needs. Announcement • Jun 30
SCYNEXIS, Inc. Initiates Phase 1 Study of SCY-770 for Treatment of Autosomal Dominant Polycystic Kidney Disease SCYNEXIS, Inc. announced initiation of a Phase 1 study of SCY-770, a first-in-class, potent and direct AMP-activated protein kinase (AMPK) activator. The study will characterize the food-effect and define the pharmacokinetics of two dosing regimens for SCY-770 to support dose selection for the Phase 2 study in patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD), on track for Fourth Quarter 2026. The Phase 1 study will enroll healthy participants in a sequential design. In the initial cohort, participants will receive a single 500 mg dose of SCY-770 under fed and fasted conditions. Based on these results, subsequent cohorts will evaluate SCY-770 at 750 mg once daily or 500 mg twice daily or placebo, for seven days to inform dose selection for the planned Phase 2 ADPKD study. Topline data are expected in the Third Quarter of 2026. SCY-770 has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of ADPKD. SCY-770, a novel and highly selective, direct Adenosine Monophosphate (AMP)-activated protein kinase (AMPK) activator, is being developed as a disease-modifying therapy for ADPKD, a progressive genetic kidney disorder with significant unmet medical need. AMPK activation suppresses the mTOR and cAMP signaling pathways that drive cyst cell proliferation and fluid secretion in ADPKD, providing a mechanistically differentiated approach to slowing disease progression. SCY-770 has been evaluated in several Phase 1 trials and one Phase 2a trial in patients with nonalcoholic fatty liver disease (NAFLD). Compelling preclinical pharmacology data supports its potential utility in ADPKD. The Company aims to develop SCY-770 with the goal of reducing cyst growth and disease progression and improving patient quality of life. 
