Setara Launches with $90 Million to Develop Drugs for Rare Diseases and Broader Uses

Rare Daily Staff

Setara Biosciences said it has raised $90 million to develop medicines for rare genetic diseases, launching with a drug candidate it plans to test in both an inherited nerve disorder and the more common condition overactive bladder.

The San Diego company was formed following Jazz Pharmaceuticals’ acquisition of Actio Biosciences. Setara takes over Actio’s remaining drug-development programs, while Jazz retains ABS-1230, an experimental treatment for KCNT1-related epilepsy.

The financing brings together Actio investors Deerfield Management, Canaan, Droia Ventures, EcoR1, Euclidean Capital and Regeneron Ventures, along with Jazz.

Setara’s launch follows a separation outlined when Jazz announced its agreement to buy Actio in August for $820 million upfront and up to $500 million in additional payments tied to regulatory approvals and sales. That agreement called for certain Actio employees, executives and drug programs to move into a new independent company, with Jazz receiving a minority stake.

Setara’s lead experimental drug, STAR-0871, is an oral treatment designed to block TRPV4, a protein that forms a channel through which electrically charged particles move into cells. Certain mutations in the gene that makes this protein cause the channel to become overactive, leading to Charcot-Marie-Tooth disease type 2C, or CMT2C.

The rare inherited disorder affects movement and sensation. Setara estimates that about 2,500 people in the United States have the condition.

By blocking the overactive channel, STAR-0871 is intended to address the biological defect behind the disease. The drug was previously known as ABS-0871.

Setara said it has completed the portion of an early-stage clinical trial involving healthy volunteers. It plans to begin a Phase 1b study, called THERON, in people with CMT2C in 2027. The company also plans to start a separate Phase 2a study in people with overactive bladder that year.

The two planned studies illustrate Setara’s broader strategy: start with rare diseases whose genetic causes offer a clear biological target, then explore whether the same target could be useful in more common conditions. Setara believes TRPV4’s role in other biological processes could make STAR-0871 useful for overactive bladder, which it says affects tens of millions of Americans.

“With Setara, we have the opportunity to build on a model that has already demonstrated its potential and apply it to severe, rare diseases with significant unmet need today,” said David Goldstein, Setara’s co-founder, president and executive chair.

Beyond STAR-0871, the company is developing early-stage programs for genetic epilepsies, with potential applications in more common conditions, including migraine.

Photo: David Goldstein, Setara’s co-founder, president and executive chair.