SCYNEXIS Acquires Novel AMPK Activator for Polycystic Kidney Disease

SCY-770 to advance into Phase 2 proof-of-concept study for ADPKD in Q4 2026

Mar. 31, 2026 at 12:23pm

Got story updates? Submit your updates here. ›

A highly detailed, translucent X-ray image showing the internal structures of a human kidney, with glowing cysts and tubules visible against a dark background, conceptually representing the complex biology of polycystic kidney disease.A new therapeutic candidate aims to address the underlying drivers of polycystic kidney disease, a progressive genetic disorder marked by the growth of fluid-filled cysts that damage kidney function.Jersey City Today

SCYNEXIS, Inc. has acquired PXL-770 (now SCY-770), a clinical-stage, highly selective AMPK activator, to develop as a potential disease-modifying therapy for autosomal dominant polycystic kidney disease (ADPKD). SCY-770 is expected to begin a Phase 2 proof-of-concept study in ADPKD patients in Q4 2026, with an anticipated early efficacy readout in the second half of 2027.

Why it matters

ADPKD is the leading genetic cause of end-stage renal failure, affecting an estimated 140,000 patients in the U.S. Despite the significant unmet need, treatment options remain limited, with only one approved therapy that has safety concerns and tolerability issues. SCY-770's novel mechanism of action targeting multiple drivers of ADPKD progression positions it as a promising candidate to potentially deliver meaningful clinical benefits to a broad patient population.

The details

SCY-770 is an oral therapy that has been evaluated in eight clinical trials and has a favorable safety profile. The acquisition strengthens SCYNEXIS's pipeline and development expertise in tackling severe and rare diseases. The company plans to efficiently advance SCY-770 into a Phase 2 proof-of-concept study later this year.

  • SCYNEXIS expects to begin a Phase 2 proof-of-concept study of SCY-770 in ADPKD patients in Q4 2026.
  • An early efficacy readout for the Phase 2 study is anticipated in the second half of 2027.

The players

SCYNEXIS, Inc.

A biotechnology company focused on developing innovative new therapies to address severe rare diseases.

Poxel S.A.

The company that previously owned the rights to PXL-770, now known as SCY-770.

Kenneth Hallows, MD, PhD

A nephrologist and System Division Chief, Nephrology Professor at the Larner College of Medicine, University of Vermont Health.

David Angulo, M.D.

The President and Chief Executive Officer of SCYNEXIS.

Got photos? Submit your photos here. ›

What they’re saying

“We are excited about this transformative asset acquisition, strengthening our pipeline, and dedicating our development expertise and resources to tackle severe and rare diseases.”

— David Angulo, M.D., President and Chief Executive Officer of SCYNEXIS

“ADPKD is a progressive disease characterized by the growth of kidney cysts that ultimately leads to end-stage kidney disease. Patients face a substantial lifelong burden, often requiring renal replacement therapy. Despite the significant unmet need, treatment options remain limited with only one approved therapy, which is associated with safety concerns and suboptimal tolerability. It is encouraging to see a new therapeutic candidate advancing in development, particularly one with a promising MOA that has the potential to deliver a meaningful clinical benefit to a broad population of patients in need.”

— Kenneth Hallows, MD, PhD, Nephrologist, System Division Chief, Nephrology Professor, Larner College of Medicine, University of Vermont Health

What’s next

SCYNEXIS will host a conference call on March 31, 2026 at 8:30 a.m. ET to provide a corporate update on the acquisition and development plans for SCY-770.

The takeaway

This acquisition strengthens SCYNEXIS's pipeline and commitment to developing innovative solutions for severe and rare diseases, with the potential for SCY-770 to address the significant unmet need and treatment limitations for patients living with autosomal dominant polycystic kidney disease.