Big Pharma-backed Atalanta Therapeutics is ramping up for the clinical debut of the company’s lead epilepsy asset in the wake of new data showing the RNA therapy suppressed seizures in mice.
Mice with a rare form of genetic epilepsy that were given a single injection of the drug candidate, dubbed ATL-201, into their cerebrospinal fluid had an average of 84% fewer seizures than control mice three days after treatment, Atalanta scientists said in a recent publication in the journal Epilepsia.
Treated mice also built nests more like those seen in mice without the disease, while untreated mice were unable to effectively turn provided bedding into nests.
The results “underscore the exciting therapeutic potential” of the company’s small interfering RNA (siRNA) platform, Aimee Jackson, Ph.D., Atalanta’s chief scientific officer, said in an April 8 release announcing the results. “We are working diligently to prepare ATL-201 to enter the clinic in the hopes of bringing the first disease-modifying therapy for KCNT1-related epilepsy to the families who urgently need it.”
Atalanta plans to submit an investigational new drug (IND) application for ATL-201 to the FDA this year, according to the release.
ATL-201 is intended to treat KCNT1-related epilepsy, a rare form of the disease caused by a mutated copy of the KCNT1 gene, which codes for a protein that transports potassium. Children with KCNT1-related epilepsy can have frequent seizures that may start within just a few days of being born.
Depending on the exact kind of KCNT1 mutation, these children can also develop complications including poor muscle tone, movement disorders and cognitive impairment.
The mutations in KCNT1 that cause epilepsy all lead to potassium channels that open more than they should; this hyperactivity is thought to disrupt neuron activity and cause the disease’s characteristic seizures.
ATL-201 is an siRNA molecule designed to bind to the mRNA that is transcribed from the KCNT1 gene, preventing it from being turned into protein. Reducing KCNT1 should, in turn, calm neuronal activity and prevent seizures, Atalanta said.
The Boston-based biotech emerged in 2021 with a $110 million series A and deals with both Roche subsidiary Genentech and Biogen. That Biogen pact is no longer active, however, and Atalanta has reacquired full rights to two preclinical programs that were part of the collaboration.
In January, Atalanta bagged a $97 million series B to help bring ATL-201 and another siRNA asset for Huntington’s disease into the clinic. The fundraising round included backing from Sanofi and Novartis, through their respective venture funds.