Latest news with #KCNT1
Yahoo
15 hours ago
- Business
- Yahoo
A startup banks $66M to pursue ‘inclusive precision medicine'
This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter. Actio Biosciences, a San Diego-based biotechnology startup, announced Wednesday it raised a $66 million Series B financing to support drug research it's initially aiming at rare genetic diseases, but sees having broader potential, too. Actio's most advanced program is in early-stage testing for the degenerative nerve disorder Charcot-Marie-Tooth disease, but may also be useful in treating overactive bladder, the company said. A second program focused on a genetic form of epilepsy is expected to enter the clinic by the end of the year. The startup's Series B round was co-led by new investor Regeneron Ventures and existing backer Deerfield Management. Canaan, Droia Ventures and Euclidean Capital also participated. Actio emerged from stealth in late 2023 with $55 million in Series A funding and, since then, has brought two small molecule drugs either into or near clinical testing. One of the medicines it's developing, ABS-1230, is for epilepsies caused by mutations in a gene called KCNT1. These KCNT1-related epilepsies can strike early and come with severe health complications, such as an impact on brain function or even death. They're also the target of programs Praxis Precision Medicines, Servier and Atalanta Therapeutics, among others, are pursuing through different drugmaking methods. David Goldstein, Actio's CEO and formerly co-founder of Praxis, claimed that small molecules still hold the most promise for targeting epilepsies related to KCNT1, mutations which cause the overactivation of a kind of ion channel expressed in the brain. Some researchers turned to biologics, believing that they may have a better chance dealing with the disease's myriad mutations, he said. However, Actio believes its drug ABS-1230, which blocks this malfunctioning ion channel, should inhibit all repeatedly observed, disease-causing mutations, making it useful to many patients with the condition. "This kind of inclusive precision medicine is a key priority for the company," Goldstein said. Actio's other drug, ABS-0871, blocks a different ion channel protein called TRPV4 and is currently in a Phase 1 trial with healthy volunteers. By the end of the year, though, Actio intends to start a Phase 1b study in people with the Type 2C form of Charcot-Marie-Tooth, which is characterized by severe muscle weakness and respiratory complications. ABS-0871 is hoping it will show promise in overactive bladder, too, as part of the company's strategy to use insights from its rare disease research in more common disorders. Actio began raising its Series B round at the beginning of the year, and was able to complete it despite an accelerating, sector-wide pullback that's making it harder for companies to close funding rounds. Goldstein attributed its success to picking programs that have 'very high biological plausibility.' 'I'm sure that the climate will return back to funding those ideas that might be huge payoffs later, but it's just a little bit hard to predict,' Goldstein said. 'You really need to have programs that have a pretty predictable path.' Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Wire
16 hours ago
- Business
- Business Wire
Actio Biosciences Secures $66 Million Series B Financing to Advance Genetics-Driven Pipeline of Small Molecule Therapeutics for Rare and Common Diseases
SAN DIEGO--(BUSINESS WIRE)--Actio Biosciences, a clinical-stage biotechnology company leveraging a novel approach to genetics and precision medicine to develop new therapeutics that target shared underlying biology in both rare and common diseases, today announced the closing of a $66 million Series B financing. New investor Regeneron Ventures and current investor Deerfield Management co-led the financing, with participation from existing investors Canaan, Droia Ventures and Euclidean Capital. Actio's pipeline is led by ABS-1230, a KCNT1 inhibitor for the treatment of KCNT1-related epilepsy and other genetic epilepsies, and ABS-0871, a TRPV4 inhibitor currently being evaluated in a Phase 1 healthy volunteer clinical trial for the treatment of Charcot-Marie-Tooth disease type 2C (CMT2C) and overactive bladder. 'We have made tremendous progress across our pipeline – executing a precision medicine strategy that targets the root causes of disease through genetically informed drug development,' said David Goldstein, Ph.D., co-founder and CEO of Actio. 'ABS-1230 and ABS-0871 have the potential to be transformative disease-modifying therapies in their respective rare indications and growing evidence supports expansion into broader indications. This new funding from industry-leading investors speaks to the value of our approach and provides us with important resources to continue advancing our programs.' The Series B financing will support the advancement of Actio's lead programs, ABS-1230 and ABS-0871. ABS-1230 is a potential first-in-class oral, selective small molecule KCNT1 inhibitor for the treatment of KCNT1-related epilepsy, a rare and often fatal pediatric epileptic encephalopathy with a United States (U.S.) prevalence of approximately 2,500 individuals. In preclinical studies, ABS-1230 has been shown to inhibit all recurrently observed pathogenic mutations in the KCNT1 gene, making ABS-1230 broadly applicable to patients with KCNT1-positive epilepsy. Actio plans to initiate the healthy volunteer portion of a Phase 1 clinical trial of ABS-1230 in the second half of 2025 with plans to expand into a proof-of-concept Phase 1b study in KCNT1-related epilepsy patients in early 2026. ABS-1230 was recently granted both rare pediatric and orphan drug designations from the U.S. Food and Drug Administration (FDA). ABS-0871 is a potential first-in-class oral, small molecule TRPV4 inhibitor for the treatment of CMT2C, a rare inherited disorder that affects both motor and sensory functions with a U.S. prevalence of approximately 2,500 individuals. Preclinical evaluation of this program in novel construct valid CMT2C rare disease models has demonstrated marked improvements in motor function and mobility, compared to untreated controls. Given its mechanism of action targeting TRPV4, ABS-0871 may also have therapeutic potential for the treatment of overactive bladder. Actio is currently advancing ABS-0871 in the healthy volunteer portion of a Phase 1 clinical trial with plans to expand into a Phase 1b study in patients with TRPV4+ CMT2C in 2026. ABS-0871 was granted rare pediatric drug, orphan drug and fast track designations from the FDA. 'Actio has done an exceptional job of identifying serious unmet needs in rare disease and advancing solutions that may also be applicable to patients with more common diseases,' said Jason Fuller, Ph.D., Partner, Deerfield Management. 'We're pleased to support the company, encouraged by its progress, and excited by the team's potential to make a difference for patients.' About Actio Biosciences Actio Biosciences is a clinical-stage company leveraging advances in precision medicine to develop new therapeutics that target shared genetics in rare and common diseases—bringing meaningful medicines from one to many. Applying its expertise in genetics, drug discovery and data sciences, Actio seeks to identify programs where both biological and technical risk can be minimized to streamline the drug development process and bring forward exceptionally potent and precisely targeted therapeutics. Actio is advancing two lead rare disease programs – ABS-0871 and ABS-1230 – with first-in-class potential for the treatment of Charcot-Marie-Tooth disease, type 2C (CMT2C) and KCNT1-related epilepsy, respectively. Founded in October 2021, the San Diego-based company is guided by leaders in genetics and drug development and backed by top healthcare investors. For more information, please visit and follow the company on LinkedIn and X.
