When Jazz Pharmaceuticals acquired Actio Biosciences for its epilepsy drug candidate, the companies said Actio’s rare disease assets would be spun off into a separate business. That startup, Setara Biosciences, emerged Friday backed by $90 million to support a lead program in early clinical development.
Actio took a precision medicine approach to drug discovery, using a human genetics platform technology to identify targets and learn about their roles in disease. This approach yielded drug candidates for a range of conditions. The lead program of San Diego-based Setara could address a rare indication and a common one.
STAR-0871 (formerly ABS-0871), is in development for the rare neuromuscular disorder Charcot-Marie-Tooth disease type 2C, a rare neuromuscular disorder that currently has no FDA-approved therapies. This Setara drug is a small molecule inhibitor of TRPV4, an ion channel protein that is widely expressed throughout the body, playing a key role in various physiological functions.
In Charcot-Marie-Tooth, mutations to the TRPV4 gene lead to abnormally active ion channels that lead to the peripheral nerve damage that develops in this rare disease. Setara’s drug is intended to selectively inhibit its target, addressing the underlying cause of the disorder. TRPV4 also plays a role in other physiological processes. Its role in detecting bladder fullness is leading the company to pursue development of the drug in overactive bladder. While overactive bladder affects millions, the few available treatment options have limited efficacy.
A Phase 1 test of the TRPV4 inhibitor was already underway when Jazz’s Actio acquisition was announced in August. Setara now says the healthy volunteer portion of this trial is complete. Next year, the study will expand to a Phase 1b study enrolling patients with Charcot-Marie-Tooth. Parallel to this study, Setara plans a separate Phase 2a trial in overactive bladder. Setara also has other assets from Actio including discovery-stage programs for epilepsies and migraine addressing targets that remain undisclosed.
The most advanced program to come out of Actio’s research was a small molecule inhibitor of KCNT1 ion channels. This drug, ABS-1230, was developed to treat KCNT1-related epilepsy, a rare type of epilepsy that does not typically respond to antiseizure medicines. There are no FDA-approved drugs for KCNT1-related epilepsy. Under Actio, ABS-1230 reached the Phase 2 portion of a potentially pivotal Phase 1b/2a trial. Per terms of the deal, which closed in September, Actio paid $820 million up front; another $500 million is tied to the achievement of milestones.
The investors in Satera’s new financing, a Series A round, include financial backers of Actio: Deerfield Management, Canaan, Droia Ventures, EcoR1, Euclidean Capital, and Regeneron Ventures. Jazz also joined as a new investor in the startup. David Goldstein, the former CEO of Actio, is now president and executive chair of Setara.
“Setara allows us to take the next step in our journey, to carry forward a pipeline of promising programs for rare diseases, with the potential to ultimately reach much broader patient populations,” Goldstein said in a prepared statement.
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