An impressive group of biopharma investors put $66 million into a San Diego startup called Actio Biosciences.
The clinical-stage company said Wednesday morning that existing investor Deerfield Management and new backer Regeneron Ventures co-led Actio’s Series B round. The new capital adds to the
$55 million Series A
that the rare disease company unveiled in September 2023. Some of the same investors in the Series A also participated in the Series B, including Canaan, Droia Ventures and Euclidean Capital.
Actio was founded by CEO David Goldstein and former Gilead chief scientific officer John McHutchison, who recently became
CEO of Tune Therapeutics
.
Goldstein spent many years as an academic human geneticist, leading up to his role as founding director of Columbia University’s Institute for Genomic Medicine. But what he really wanted to do was directly apply those learnings to drug development.
“I tried to convince a few pharma companies to create rare disease units,” Goldstein said in an interview. “That was a complete failure. I spent so many years trying to do that. I just got absolutely nowhere.
“So I eventually decided if I can’t get someone else to do it, I’ll do it myself,” Goldstein continued.
He devised a blueprint to use informatics tools to “scour the entire world of Mendelian disease genes and pluck out exceptional opportunities where you really could have almost every program you work on work.”
That mission led to Actio’s founding in 2021. The company now has nearly 40 employees, with plans to use the new funding for some hiring, though Goldstein wants to maintain its headcount around that size since it’s “trying to be very disciplined.”
Actio is already in Phase 1 with a once-daily oral TRPV4 inhibitor called ABS-0871. The goal is to put ABS-0871 to the test for treating Charcot-Marie-Tooth disease type 2C, or CMT2C, which is a rare disease that impairs both motor and sensory functions, including disrupted vocal cords and breathing, Goldstein said.
The funds will also help get a once-daily KCNT1 inhibitor into the clinic before year’s end for KCNT1-related epilepsy, the company said. The FDA has already attached rare pediatric and orphan drug tags to the experimental medicine, codenamed ABS-1230. The condition can lead to 20 seizures a day.
CMT2C and KCNT1 each impact about 2,500 people in the US, Actio said.
Once both drugs complete healthy volunteer trials, Actio plans to move them into Phase 1b studies next year.
Actio also plans to use the Series B to get to Phase 1b proof-of-concept data in KCNT1-related epilepsy in 2026, Goldstein said; at that point, the biotech would use the data as an anchor for further financing and to get closer to pivotal trials.
The company hasn’t yet determined its registrational study design in KCNT1 since it still needs to see Phase 1b data. However, Goldstein said Actio has done some initial calculations for what a pivotal trial could entail. “Because of the high seizure burden, 30 to 40 patients treated for six months would represent a very powered study,” he said.
For CMT2C, the timelines are a little less clear, according to Goldstein, who noted the Phase 1b in that patient population won’t be as quick. In KCNT1, they’ll be able to tell relatively soon how well ABS-1230 does at reducing seizures.
Actio is not alone in the KCNT1 space. Another biotech that Goldstein helped co-found as an academic called
Praxis Precision Medicines
licensed
its small molecule to UCB in December.
Elsewhere in the KCNT1 field, Atalanta Therapeutics expects to enter the clinic in the near term with an siRNA approach that attracted a
$97 million Series B
at the beginning of this year.
Actio plans to handle all clinical development for its rare disease programs, but it’s open to partnering on broader indications. Goldstein said there’s potential for seeing if its TRPV4 inhibitor could treat overactive bladder.
