Atalanta Therapeutics, a biotechnology company pioneering RNA interference (RNAi) for the treatment of neurological diseases, today announced the publication of preclinical data in the journal Epilepsia demonstrating proof-of-mechanism of its investigational di-siRNA therapy, ATL-201, in a mouse model of KCNT1-related epilepsy. Durable seizure suppression and behavioral improvements were observed over 6 months, with favorable tolerability. The company plans to submit an Investigational New Drug (IND) application to the U.S. Food and Drug Administration for ATL-201 in 2025.

“We believe these preclinical results underscore the exciting therapeutic potential unlocked by our di-siRNA platform’s ability to achieve distribution broadly throughout the central nervous system,” said Aimee Jackson, Ph.D., Chief Scientific Officer of Atalanta and an author of the study. “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.”

KCNT1-related epilepsy is a rare, severe seizure disorder that affects infants and children. It is driven by gain-of-function variants in the KCNT1 gene, which encodes a potassium channel in neurons. Children with KCNT1-related epilepsy have frequent seizures that are unable to be controlled with anti-seizure medications, and as a result they often experience developmental delays and intellectual disability. In patients with early infantile onset, seizures can become nearly continuous by age six to nine months.

ATL-201 is an investigational RNAi therapy designed to reduce KCNT1 levels and to normalize neuronal excitability.

Key findings of the study included:

• ATL-201 potently reduced KCNT1 transcript levels and blocked KCNT1 potassium currents in vitro.
• When administered to wild-type mice in a single dose via the cerebrospinal fluid (CSF), ATL-201 displayed broad distribution across the brain, reaching over 90 percent of cortical neurons and even penetrating deep brain structures that are challenging to access with other oligonucleotides.
• In mice with mutations that mimic those found in people with KCNT1-related epilepsy, a single dose of ATL-201 delivered via the CSF showed complete seizure suppression up to four months, with therapeutic effects persisting out to six months. Seizure suppression was observed to be dose-dependent.
• ATL-201 demonstrated a favorable tolerability profile in this mouse model, and key serum biomarkers for liver and kidney function remained unchanged.

The research article, “Durable suppression of seizures in a preclinical model of KCNT1 genetic epilepsy with divalent small interfering RNA” by Andreone, et al. appears in Epilepsia and is available online at https://doi.org/10.1111/epi.18278.

About Atalanta Therapeutics

Atalanta Therapeutics is a biotechnology company developing treatments for intractable diseases of the central nervous system using RNA interference. Atalanta’s unique platform of divalent small interfering RNA (di-siRNA) is designed to enable durable, selective gene silencing throughout the brain and spinal cord. Atalanta is advancing a wholly owned pipeline of disease-modifying programs for Huntington’s disease, genetic epilepsy, severe chronic pain, and other neurological diseases in addition to partnered programs as part of a strategic collaboration with Genentech. Atalanta is headquartered in Boston, Mass. For more information, visit www.atalantatx.com.

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