Atalanta Therapeutics, a biotechnology company focused on developing treatments for neurological disorders, has announced the completion of a $97 million Series B financing round. The funding will be used to advance the company's RNA interference (RNAi) therapies for Huntington's disease and a genetic form of childhood epilepsy into Phase 1 clinical trials. The financing was co-led by EQT Life Sciences and Sanofi Ventures.
RNAi-Based Therapies for Neurological Diseases
Atalanta's approach involves using RNAi to silence the activity of genes that contribute to disease progression. Their lead candidates, ATL-101 for Huntington's disease and ATL-201 for KCNT1-related epilepsy, are designed to target and downregulate specific genes involved in these conditions. The company plans to submit Investigational New Drug (IND) applications to the FDA this year to begin clinical trials in the U.S.
Addressing Delivery Challenges with di-siRNA Technology
A significant challenge in using RNAi for neurological diseases has been achieving effective drug delivery to the central nervous system (CNS). Atalanta's proprietary di-siRNA technology aims to overcome this obstacle by linking two separate RNA molecules to enhance persistence and distribution throughout the brain and spinal cord. According to the company, preclinical studies have demonstrated that their candidates show unparalleled distribution throughout the CNS.
Huntington's Disease Program: ATL-101
Huntington's disease is a progressive neurodegenerative disorder caused by mutations in the HTT gene, leading to the production of a toxic huntingtin protein that damages nerve cells. ATL-101, Atalanta's RNAi-based therapeutic candidate, is designed to silence HTT gene activity, thereby reducing the production of the huntingtin protein.
Preclinical studies in nonhuman primates have shown that a single dose of ATL-101, delivered directly into the spinal canal, resulted in a significant reduction in huntingtin levels in deep brain regions implicated in Huntington's disease. The effects lasted for at least six months, and the treatment was well-tolerated with no significant safety signals observed.
KCNT1-Related Epilepsy Program: ATL-201
ATL-201 is Atalanta’s investigational therapy for KCNT1-related epilepsy, a severe form of childhood epilepsy caused by gain-of-function variants in the KCNT1 gene. This condition leads to frequent, uncontrollable seizures, developmental delays, and intellectual disability. ATL-201 aims to reduce KCNT1 levels and normalize neuronal excitability. Preclinical studies have shown significant seizure reduction and behavioral improvements with good tolerability.
Leadership Insights
"This financing validates the truly transformative potential of Atalanta’s best-in-class di-siRNA platform for delivering oligonucleotide therapies to the central nervous system and the exciting promise of our expansive wholly-owned pipeline," said Alicia Secor, Atalanta’s president and chief executive officer. "Importantly, this Series B will support a path to the clinic for two programs for serious neurological diseases that today lack disease-modifying therapies."
Arno de Wilde, MD, PhD, managing director at EQT Life Sciences, added, "Atalanta’s di-siRNA technology has shown promising ability to durably silence disease-promoting genes throughout previously inaccessible regions of the brain and spinal cord – opening a wide range of treatment possibilities for devastating neurological diseases."
