ETX101 / Encoded Therap - LARVOL DELTA

ETX-DS-005: A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome (clinicaltrials.gov) - P1/2 | N=5 | Active, not recruiting | Sponsor: Encoded Therapeutics | Recruiting ➔ Active, not recruiting

Enrollment closed • CNS Disorders • Epilepsy • Gene Therapies • CSF1

POLARIS Phase 1/2 Program Interim Safety and Preliminary Efficacy Results of ETX101, a One-Time Gene Regulation Therapy, in Young Children with Dravet Syndrome (AES 2025) - P1/2 | "Preliminary results from the POLARIS program demonstrate that ascending dose levels of ETX101 have been well-tolerated with no safety concerns related to study drug. Dose-dependent anti-seizure effects highlight the promising therapeutic potential of ETX101 and support continued development to establish its efficacy and long-term impact. Notably, this research represents the first clinical data for an investigational one-time, cell-type selective, gene regulation therapy, and the first potential one-time, disease-modifying treatment for DS."

Clinical • P1/2 data • CNS Disorders • Epilepsy • CSF1 • JUN • NAV1

A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome (Australia Only) (clinicaltrials.gov) - P1/2 | N=4 | Active, not recruiting | Sponsor: Encoded Therapeutics | Recruiting ➔ Active, not recruiting | Trial completion date: Jun 2030 ➔ Sep 2030 | Trial primary completion date: Jun 2030 ➔ Sep 2030

Enrollment closed • Trial completion date • Trial primary completion date • CNS Disorders • Epilepsy • Gene Therapies • CSF1

Encoded Therapeutics Announces Regenerative Medicine Advanced Therapy (RMAT) Designation Granted by U.S. FDA for ETX101 in SCN1A+ Dravet Syndrome (Businesswire) - "Designation based on preliminary clinical evidence indicating the potential for ETX101 to address unmet medical needs associated with SCN1A+ Dravet syndrome."

FDA event • CNS Disorders

ETX-DS-005: A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome (clinicaltrials.gov) - P1/2 | N=4 | Recruiting | Sponsor: Encoded Therapeutics | Trial completion date: Dec 2029 ➔ Sep 2030 | Trial primary completion date: Dec 2029 ➔ Sep 2030

Trial completion date • Trial primary completion date • CNS Disorders • Epilepsy • Gene Therapies • CSF1

A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children with SCN1A-positive Dravet Syndrome (Australia Only) (clinicaltrials.gov) - P1/2 | N=4 | Recruiting | Sponsor: Encoded Therapeutics | Trial completion date: Dec 2029 ➔ Jun 2030 | Trial primary completion date: Dec 2029 ➔ Jun 2030

Trial completion date • Trial primary completion date • CNS Disorders • Epilepsy • Gene Therapies • CSF1

Encoded Therapeutics Reports Clinical Progress of ETX101 Gene Therapy for Dravet Syndrome, Recaps 2024 Corporate Achievements and Provides 2025 Outlook (Businesswire) - "POLARIS program, launched in 2024 to evaluate ETX101 for SCN1A+ Dravet syndrome, is ongoing. Expect to complete dosing and share preliminary safety and efficacy data in 2H25...Following guidance from FDA, IND-enabling studies have been initiated to support a potential ETX201 IND filing in 2026...Data for both the pain and Alzheimer’s disease programs will be presented at scientific meetings in mid-2025."

IND • P1/2 data • Preclinical • Alzheimer's Disease • CNS Disorders • Pain

ETX-DS-005: A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome (clinicaltrials.gov) - P1/2 | N=4 | Recruiting | Sponsor: Encoded Therapeutics | Not yet recruiting ➔ Recruiting

Enrollment open • Gene therapy • CNS Disorders • Epilepsy • Gene Therapies

ENDEAVOR: A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome (clinicaltrials.gov) - P1/2 | N=22 | Recruiting | Sponsor: Encoded Therapeutics | Not yet recruiting ➔ Recruiting

Enrollment open • Gene therapy • CNS Disorders • Epilepsy • Gene Therapies • CSF1

UK Only: A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome (clinicaltrials.gov) - P1/2 | N=4 | Not yet recruiting | Sponsor: Encoded Therapeutics

New P1/2 trial • CNS Disorders • Epilepsy • Gene Therapies

ENDEAVOR: A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome (clinicaltrials.gov) - P1/2 | N=22 | Not yet recruiting | Sponsor: Encoded Therapeutics | Trial completion date: Oct 2029 ➔ May 2031 | Trial primary completion date: Feb 2026 ➔ May 2027

Gene therapy • Trial completion date • Trial primary completion date • CNS Disorders • Epilepsy • Gene Therapies • CSF1

A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome (Australia Only) (clinicaltrials.gov) - P1/2 | N=4 | Recruiting | Sponsor: Encoded Therapeutics | Not yet recruiting ➔ Recruiting

Enrollment open • CNS Disorders • Epilepsy • Gene Therapies • CSF1

A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome (Australia Only) (clinicaltrials.gov) - P1/2 | N=4 | Not yet recruiting | Sponsor: Encoded Therapeutics

Gene therapy • New P1/2 trial • CNS Disorders • Epilepsy • Gene Therapies • CSF1

ENDEAVOR: A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome (clinicaltrials.gov) - P1/2 | N=22 | Not yet recruiting | Sponsor: Encoded Therapeutics

New P1/2 trial • CNS Disorders • Epilepsy • Gene Therapies • CSF1

Cell-selective AAV-mediated SCN1A Gene Regulation Therapy Rescues Mortality and Seizure Phenotypes in a Dravet Syndrome Mouse Model and is Well Tolerated in Non-human Primates. (PubMed, Hum Gene Ther) - "AAV9-RE-eTF was well tolerated, with no adverse events during administration, no detectable changes in clinical observations, no adverse findings in histopathology, and no dorsal root ganglion-related toxicity. Our results support the clinical development of AAV9-RE-eTF (ETX101) as an effective and targeted disease-modifying approach to SCN1A+ DS."

Journal • Preclinical • CNS Disorders • Epilepsy

Encoded Therapeutics to Present Initial ENVISION Natural History Study Data of Children With SCN1A+ Dravet Syndrome at the American Epilepsy Society 2021 Annual Meeting (Yahoo Finance) - "Encoded Therapeutics today announced the upcoming presentation of initial data from the ENVISION prospective natural history study of children with SCN1A+ Dravet syndrome, a rare developmental and epileptic encephalopathy, at the American Epilepsy Society 2021 Annual Meeting, taking place in Chicago from December 3-7, 2021. Additionally, Encoded will present on its ongoing DRAVET ENGAGE initiative, which aims to capture the Dravet syndrome patient community’s experiences and perspectives, and a summary of molecular diagnostic and phenotypic findings in people with SCN1A+ Dravet syndrome identified through genetic testing."

Observational data • CNS Disorders • Epilepsy