Pharma Focus Europe

Encoded Therapeutics Receives UK Clinical Trials Authorization for ETX101, a Gene Therapy Candidate for Dravet Syndrome

Tuesday, February 27, 2024

Encoded Therapeutics Inc., a biotechnology company specializing in genetic medicines for severe central nervous system (CNS) disorders, has received approval from the United Kingdom (UK) Medicines and Healthcare products Regulatory Agency (MHRA) for its Clinical Trial Authorization (CTA) application for the EXPEDITION Study of ETX101. This study aims to evaluate ETX101 as a potential treatment for children with SCN1A+ Dravet syndrome, a prevalent form of developmental and epileptic encephalopathy characterized by treatment-resistant seizures and developmental stagnation.

The UK CTA approval represents a significant step in Encoded's global regulatory strategy for ETX101's clinical development. This strategy encompasses clearance for an investigational new drug (IND) application in the United States and approval under the Clinical Trial Approval (CTA) scheme in Australia.

Encoded, expressed excitement about the approval, marking the third regulatory milestone for ETX101. He underscored the company's dedication to advancing their global clinical development program, known as POLARIS, to provide potential therapy to Dravet syndrome patients worldwide.

Led by Professor Andreas Brunklaus, the EXPEDITION trial aims to address the urgent need for disease modification strategies for Dravet syndrome in the UK. This trial signifies a collaborative effort to develop therapies targeting not only symptom alleviation but also underlying mechanisms, recognizing the severe impact of Dravet syndrome on affected children and their families.

EXPEDITION is part of Encoded’s POLARIS program, a Phase 1/2 dose escalation study evaluating ETX101 in children aged 6 months to <4 years with SCN1A+ Dravet syndrome in the UK. The study aims to assess safety, tolerability, preliminary efficacy, and therapeutic dose selection. It is expected to begin in mid-2024 concurrently with trials in the US (ENDEAVOR) and Australia (WAYFINDER).

Encoded’s ETX101 Clinical Development Program, POLARIS, integrates comprehensive preclinical research, biomarker discovery initiatives (ELUCIDATE), patient-focused drug development efforts (Dravet ENGAGE), and a recent natural history study (ENVISION). This groundwork informs the rigorous clinical design of studies like ENDEAVOR, WAYFINDER, and EXPEDITION, which evaluate the safety and preliminary efficacy of ETX101 in infants and young children with SCN1A+ Dravet syndrome.

ETX101 is a potential one-time, disease-modifying gene regulation therapy designed to target the underlying cause of SCN1A+ Dravet syndrome. By upregulating the expression of the SCN1A gene, ETX101 aims to increase the production of NaV1.1 protein sodium channels in the brain, potentially addressing a wide range of symptoms associated with Dravet syndrome.

Dravet syndrome is a severe, lifelong CNS disorder affecting approximately 1 in 16,000 births worldwide, characterized by loss-of-function variants in the SCN1A gene. Its symptoms include frequent, prolonged, and treatment-resistant seizures, cognitive and developmental stagnation, sleep abnormalities, motor impairment, and behavioral difficulties.



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