Servier Announces First Patient Enrolled in Phase Ib/II Study for the Treatment of a Rare Pediatric Epilepsy Syndrome in the U.S.

• This clinical trial further enhances Servier's neurology franchise focused on targeting rare indications for patients with significant unmet needs
• Global clinical trial sites are open and enrolling in the U.S., Europe and Japan

BOSTON, June 25, 2026 /PRNewswire/ -- Servier today announced the first patient has been enrolled in the U.S. in a Phase Ib/II first-in-human study to evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic profile of its antisense oligonucleotide (ASO) molecule in children with KCNT1-related developmental and epileptic encephalopathy (KCNT1-DEE).

"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."

Pathogenic variants in the KCNT1 gene lead to a devastating early-onset epilepsy syndrome associated with profound developmental impairment. The disease has a high degree of mortality among children and there are currently no curative or disease-modifying treatments available. The asset being tested in this clinical trial is an investigational ASO, which is an innovative modality designed to target the genetic cause of KCNT1-DEE. It acts by degrading KCNT1 mRNA with the aim of modifying the disease in patients and has been shown to be well-tolerated in pre-clinical studies.

"In many children with a KCNT1 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."

The first-in-human clinical trial is enrolling children with KCNT1-DEE. Additional details regarding the clinical trial can be found here.

Press Contact
Ken DiCienzo
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About Servier in Neurology
At Servier, we are committed to focusing our R&D efforts to support neurological diseases associated with genetic mechanisms, this includes but is not limited to: refractory epilepsy, rare movement disorders, and neuromuscular diseases.

Our scientists and researchers are united around one mission, to bring innovative therapies to patients in need, through in-house developed compounds and externally sourced assets, regardless of the size of the patient population.

Servier's neurology research is concentrated on the small molecules targeting messenger RNA (mRNA), including antisense oligonucleotides (ASOs), as well as pharmacological molecules and monoclonal antibodies.

About Servier Pharmaceuticals in the U.S.
As the U.S. affiliate of the Servier Group, Servier Pharmaceuticals is focused on delivering transformative oncology and neurology medicines that fill a critical need for patients who are underserved by current therapies.

Since opening its headquarters in Boston, U.S., in 2018, Servier Pharmaceuticals quickly became a leader in cancer care and is taking bold steps to build a powerful portfolio of precision therapeutics for patients living with cancer and neurological conditions.

Governed by a non-profit foundation, Servier Group's private corporate governance enables the company to combine its resources and global network with cutting-edge science and creativity to develop tailored solutions that drive meaningful progress for patients.

Servier is actively seeking alliances, partnerships and acquisitions across all stages of oncology and neurology research and development to accelerate the delivery of innovative treatments to patients as part of its trailblazing One Innovation Engine R&D strategy.

For more information about Servier, visit Servier.us and follow Servier on LinkedIn.

SOURCE Servier Pharmaceuticals