Servier Enrolls First Patient in Phase Ib/II Trial of ASO Therapy for KCNT1-Related Pediatric Epilepsy
核心洞察
Servier has enrolled the first patient in a first-in-human Phase Ib/II study evaluating an antisense oligonucleotide (ASO) targeting KCNT1-related developmental and epileptic encephalopathy (搜索) (KCNT1 (搜索)-DEE) in children.
The investigational ASO is designed to degrade KCNT1 (搜索) mRNA, addressing the genetic root cause of this devastating early-onset epilepsy syndrome that currently has no curative or disease-modifying treatments.
Global clinical trial sites are now open and actively enrolling patients across the U.S., Europe, and Japan, with the asset developed internally by Servier scientists.
Servier announced on June 25, 2026, that the first patient has been enrolled in the United States in a Phase Ib/II first-in-human clinical trial evaluating an investigational antisense oligonucleotide (ASO) for children with KCNT1-related developmental and epileptic encephalopathy (搜索) (KCNT1 (搜索)-DEE). Global clinical trial sites are now open and enrolling in the U.S., Europe, and Japan.
The study will assess the safety, tolerability, pharmacokinetic, and pharmacodynamic profile of Servier's ASO molecule, which was developed internally by the company's scientists. The asset represents an innovative modality designed to target the genetic cause of KCNT1 (搜索)-DEE by degrading KCNT1 mRNA, with the aim of modifying the disease course in affected patients.
A Devastating Pediatric Epilepsy with No Approved Treatments
Pathogenic variants in the KCNT1 (搜索) gene lead to a severe, early-onset epilepsy syndrome associated with profound developmental impairment. The disease carries a high degree of mortality among children, and there are currently no curative or disease-modifying treatments available. Seizures can begin as early as the first days or months of life.
"In many children with a KCNT1 (搜索) variant, seizures can begin as early as the first days or months of life and some never get to go home," said Justin West, Co-Founder and President of the KCNT1 Epilepsy Foundation. "Our mission is to accelerate the development of effective treatments because time is not on our side. We're grateful that the first patient was enrolled in this clinical trial to evaluate this KCNT1-targeted ASO in children with this disease. It's a meaningful step forward for families who have been waiting far too long, and we thank Servier for their commitment to this critical work."
Servier's Strategic Focus on Rare Neurology
The enrollment marks a significant step in Servier's broader neurology strategy, which concentrates on small molecules targeting messenger RNA (mRNA), including antisense oligonucleotides, as well as pharmacological molecules and monoclonal antibodies. The company's R&D efforts support neurological diseases associated with genetic mechanisms, including refractory epilepsy, rare movement disorders, and neuromuscular diseases.
"Servier's strategic direction in neurology aims to accelerate drug development for patients with rare neurological disorders," said Nitza Thomasson, Global Head, R&D Neurology at Servier. "This asset was developed by Servier scientists and highlights our commitment and capability to translate scientific discoveries into clinical innovations that may deliver meaningful benefits to patients. We look forward to enrolling additional patients in this clinical trial for this drug candidate."
Pre-clinical studies have shown the investigational ASO to be well-tolerated, supporting its advancement into human trials.
Broader Commitment: n-Lorem Foundation Partnership
The KCNT1 (搜索)-DEE trial follows closely on Servier's separate announcement on June 10, 2026, of a multi-target research collaboration with the n-Lorem Foundation, a non-profit organization that charitably provides experimental ASO medicines to nano-rare patients with high unmet needs. Under that agreement, n-Lorem research teams will leverage their ASO technology platform to engineer preclinical candidates, which Servier will advance into clinical development.
"We are proud to partner with n-Lorem, sharing the same commitment to advancing personalized ASOs and bringing meaningful therapies to people living with rare genetic neurological disorders who have few, if any, treatment options available today," said Thomasson. Stanley T. Crooke, Founder, Chairman and CEO of n-Lorem, added: "We welcome Servier to our growing list of supporters and partners and look forward to contributing to Servier's commitment to patients with rare neurological disorders and to advancing ASO technology. This is truly a win, win for patients with rare diseases."
Together, these initiatives underscore Servier's 2030 ambition of developing innovative treatments for people living with rare neurological diseases, spanning refractory epilepsy, genetically-driven autism spectrum disorders, leukodystrophies, peripheral neuropathies, movement disorders, and neuromuscular diseases.