• SCY-770 was well tolerated with a favorable safety profile in healthy adult participants at higher doses than previously evaluated
• Phase 2 proof-of-concept study in patients with ADPKD remains on-track to initiate in the fourth quarter of 2026
• SCY-770 is a potential first-in-class oral direct AMPK activator designed to address multiple underlying drivers of cyst growth and disease progression in ADPKD
JERSEY CITY, N.J., Oct. 06, 2026 (GLOBE NEWSWIRE) -- SCYNEXIS, Inc. (NASDAQ: SCYX), a clinical-stage biotechnology company advancing novel therapies for severe rare diseases, today announced completion of a Phase 1 study of SCY-770, a first-in-class, selective and direct AMP-activated protein kinase (AMPK) activator. The study was conducted to evaluate the safety and tolerability, as well as to characterize the pharmacokinetics of SCY-770 to support dose selection for the Phase 2 study in patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD).
"We continue to make strong progress toward initiating our Phase 2 study of SCY-770 in patients with ADPKD, and the additional pharmacokinetic and safety data generated in this Phase 1 study provide important insights to support dose selection," said Todd Minga, M.D., SVP of Research and Development at SCYNEXIS. "We are encouraged by the favorable safety and tolerability profile observed across the doses studied, which supports continued clinical development of SCY-770. Patients living with ADPKD face a significant unmet medical need, as treatment options remain limited. We look forward to advancing this novel, highly selective oral direct AMPK activator, which has the potential to offer a differentiated therapeutic approach and provide meaningful clinical benefit for patients with this progressive disease."
The Phase 1 study enrolled 25 healthy participants across three cohorts. In the initial cohort, participants received a single 500 mg dose of SCY-770 under fed and fasted conditions. Participants in the subsequent cohorts received SCY-770 at either 750 mg once daily or 500 mg twice daily, or placebo, for seven days. SCY-770 was well tolerated across all cohorts and demonstrated a safety profile consistent with prior clinical experience. Pharmacokinetic data from this and prior studies will inform dose selection for the Phase 2 trial in patients with ADPKD, which remains on track to initiate in the fourth quarter of 2026.
The safety and tolerability of SCY-770 has been well-characterized across multiple clinical trials in approximately 300 participants to date. SCY-770 has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of ADPKD.
About ADPKD
Autosomal Dominant Polycystic Kidney Disease (ADPKD) is a genetic disease caused by mutations of the PKD1 or PKD2 genes which encode polycystin complex 1 (PC1) or polycystin complex 2 (PC2) proteins, critical for normal tubular epithelial cell function. Patients develop fluid-filled cysts in their kidneys that progressively impair kidney function with more than 50% reaching end-stage renal failure by the age of 60 requiring renal replacement therapies (e.g., dialysis or transplant). The U.S. prevalence of ADPKD is estimated to be 140,000 patients, with approximately 6,000 new cases diagnosed each year. ADPKD currently has only one approved therapy, Jynarque (tolvaptan), which achieved approximately $1.5 billion in U.S. sales in 2024 despite limited patient uptake due to safety, tolerability, and monitoring requirements.
About SCY-770
SCY-770 is a novel, highly selective, oral direct activator of adenosine monophosphate-activated protein kinase (AMPK) being developed as a potential disease-modifying therapy for autosomal dominant polycystic kidney disease (ADPKD), a progressive genetic disorder characterized by significant unmet medical need. By activating AMPK, SCY-770 targets multiple pathways implicated in disease progression, including mTOR and cAMP signaling, which are known to drive cyst growth and fluid secretion. In addition, AMPK activation is associated with reduction in inflammation and fibrosis while improving cellular metabolism, offering a differentiated, multi-mechanistic approach to address the underlying drivers of ADPKD. SCY-770 has been evaluated across multiple clinical trials in approximately 300 participants to date. SCY-770 has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of ADPKD.
About SCYNEXIS
SCYNEXIS, Inc. (NASDAQ: SCYX) is a clinical-stage biotechnology company dedicated to advancing innovative solutions for severe rare diseases. SCY-770 is being developed for the treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD) and has been granted Orphan Drug designation. SCYNEXIS's proprietary antifungal platform "fungerps" includes BREXAFEMME® (ibrexafungerp tablets), the first approved representative of this novel class, which has been licensed to GSK, and SCY-247, currently in clinical stages of development for the treatment and prevention of invasive fungal diseases. For more information, visit www.scynexis.com
Forward-Looking Statements
Statements contained in this press release regarding expected future events or results are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including but not limited to statements regarding: the anticipated initiation of the Phase 2 proof-of-concept study of SCY-770 in ADPKD patients in the fourth quarter of 2026; the use of population pharmacokinetic modeling to support Phase 2 dose selection; and the potential for SCY-770 to serve as a disease-modifying therapy for ADPKD. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, risks inherent in regulatory and other costs in developing products, including the risk that SCYNEXIS may not reach alignment with the FDA on the planned Phase 2 study design on the anticipated timeline or at all. These and other risks are described more fully in SCYNEXIS' filings with the Securities and Exchange Commission, including without limitation, the section titled "Risk Factors" in its most recent Annual Report on Form 10-K, and in other filings made with the SEC from time to time. All forward-looking statements contained in this press release speak only as of the date on which they were made. SCYNEXIS undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.
CONTACT:
Investor Relations
John Fraunces
LifeSci Advisors
Tel: 917-355-2395
jfraunces@lifesciadvisors.com
