Setara Raises $90M to Advance STAR-0871 for CMT2C and Overactive Bladder

Setara Biosciences, a clinical-stage biotechnology company focused on genetics-driven precision medicines, has raised $90 million in Series A financing to develop therapies for severe rare diseases and potentially expand their applications into broader patient populations.

New Company Emerges Following Jazz Pharmaceuticals’ Acquisition of Actio

The company was established following Jazz Pharmaceuticals’ acquisition of Actio Biosciences, which included ABS-1230, an investigational treatment for KCNT1-related epilepsy. Setara will advance Actio’s remaining development pipeline, led by STAR-0871 (previously known as ABS-0871).

The financing included participation from existing investors Deerfield Management, Canaan, Droia Ventures, EcoR1, Euclidean Capital, and Regeneron Ventures, alongside Jazz Pharmaceuticals as a new shareholder.

David Goldstein, PhD, Actio’s founder and a human genetics expert, will lead Setara as President and Executive Chair. He will retain his position at Jazz until at least the acceptance of the ABS-1230 New Drug Application (NDA) submission.

STAR-0871 Targets the Genetic Basis of CMT2C

STAR-0871 is a potential first-in-class oral small-molecule inhibitor of transient receptor potential vanilloid 4 (TRPV4), an ion channel involved in several physiological processes.

The programme’s initial focus is Charcot-Marie-Tooth disease type 2C (CMT2C), a rare inherited neuromuscular disorder affecting approximately 2,500 people in the United States. The disease results from gain-of-function mutations in the TRPV4 gene, which cause excessive activation of the ion channel and disrupt motor and sensory functions.

By inhibiting TRPV4, STAR-0871 targets the underlying molecular abnormality rather than treating symptoms alone. This genetic rationale provides the basis for its clinical development in patients with CMT2C.

In addition to CMT2C, the company is evaluating the potential of TRPV4 inhibition in overactive bladder, a substantially larger patient population affecting tens of millions of people in the United States. Existing treatments provide limited efficacy for many patients, creating a significant opportunity for alternative therapeutic approaches.

Phase 1 Completed; Patient Studies Planned for 2027

Setara has completed the healthy volunteer portion of a Phase 1 clinical trial evaluating STAR-0871. The company now plans to initiate two patient-focused studies:

1. THERON Study (CMT2C): A planned Phase 1b study in patients with CMT2C expected to begin in 2027. The U.S. FDA has granted STAR-0871 Rare Pediatric Disease, Orphan Drug, and Fast Track designations for this indication.
2. Phase 2a Study (Overactive Bladder): A parallel study evaluating the compound in patients with overactive bladder.

These two development programmes will assess the therapeutic potential of the same molecular target across distinct clinical settings. While CMT2C offers a genetically defined rare disease population, overactive bladder represents a broad-market opportunity.

Genetics-Driven Pipeline Extends Beyond STAR-0871

Beyond its lead programme, Setara plans to advance early-stage discovery programmes targeting genetic epilepsies and additional indications, including migraine.

Dr. Goldstein described the company’s strategy as an extension of the genetics-driven approach developed at Actio: starting with rare diseases where genetic evidence provides a clear understanding of disease biology before exploring applications in broader patient populations.

The $90 million financing provides sufficient capital to advance STAR-0871 into patient trials while supporting the expansion of additional pipeline programmes. The central clinical question moving forward will be whether TRPV4 inhibition can successfully translate its strong genetic rationale into meaningful therapeutic benefits in both CMT2C and overactive bladder.

Reference

Setara Biosciences Announces $90 Million Series A Financing to Advance Precision Medicines for Rare Genetic Diseases and Related Expansion Indications, Setara Bioscience, 30 October 2026