allogeneic umbilical cord-derived mesenchymal stem cells (EN001) / ENCell - LARVOL DELTA

Cell and gene therapy company ENCell said on the 6th that its allogeneic umbilical cord-derived mesenchymal stem cell therapy candidate 'EN001' received orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA) for Duchenne muscular dystrophy (DMD)… (Chosun Biz)

Orphan drug • Duchenne Muscular Dystrophy

ENCell said on the 27th that it applied to the European Medicines Agency (EMA) for orphan drug designation (ODD) for EN001, a candidate to treat Charcot-Marie-Tooth disease (CMT). (Chosun Biz) - "ENCell is currently conducting a domestic clinical trial in patients with CMT1A."

Orphan drug • CNS Disorders

Evaluate the Safety and Potential Efficacy of Human Wharton's Jelly-derived Mesenchymal Stem Cells With Charcot-Marie-Tooth Disease Type 1E (clinicaltrials.gov) - P=N/A | N=3 | Completed | Sponsor: Samsung Medical Center | Not yet recruiting ➔ Completed

Trial completion • Genetic Disorders

Evaluate the Safety and Efficacy of EN001 in Patients With Charcot-Marie-Tooth Disease Type 1A (clinicaltrials.gov) - P1 | N=6 | Completed | Sponsor: ENCell | Active, not recruiting ➔ Completed | N=12 ➔ 6

Enrollment change • Trial completion • Genetic Disorders

Evaluate the Safety and Efficacy of EN001 in Patients With Charcot-Marie-Tooth Disease Type 1A (clinicaltrials.gov) - P1 | N=12 | Active, not recruiting | Sponsor: ENCell | Recruiting ➔ Active, not recruiting

Enrollment closed • Genetic Disorders

ENCell’s EN001 Receives Orphan Drug Designation from the U.S. FDA for Charcot-Marie-Tooth Disease (Businesswire) - "ENCell...announced today that its investigational drug EN001 has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) for the treatment of Charcot-Marie-Tooth disease (CMT)...In October 2024, ENCell reported the results of a Phase 1 clinical trial investigating the safety and exploratory efficacy of repeated low-dose administration of EN001 in CMT type 1A patients...The results showed no occurrence of DLT, no serious adverse events, and no injection-related reactions. Following this successful outcome, ENCell initiated a Phase 1b trial with a high-dose cohort in December 2024 and aims to complete the study within 2025."

Orphan drug • Trial completion date • Genetic Disorders

ENCELL, DMD Stem Cell Treatment 'EN001' Phase 1 "Safety and Tolerance Secured" [Google translation] (HIT News) - P1 | N=7 | NCT05338099 | Sponsor: ENCell | "ENCELL announced on the 13th that the results of the phase 1 single-dose intravenous clinical trial of the next-generation mesenchymal stem cell treatment 'EN001' for patients with Duchenne Muscular Dystrophy (DMD) were published in the international academic journal 'Journal of Clinical Neurology'...The clinical trial was conducted from January to December 2022 on six DMD patients, and the main goal was to confirm the safety and tolerability of EN001. The company announced that the results of the study confirmed safety and tolerability in all patients who received EN001 at low and high doses....The company also explained that in the exploratory efficacy evaluation, lung capacity, lower extremity muscle strength, and creatine kinase (CK) levels tended to be maintained or improved to some extent. Encell plans to verify the efficacy and prove the therapeutic effect through additional clinical trials."

P1 data • Duchenne Muscular Dystrophy

Safety and Tolerability of Wharton's Jelly-Derived Mesenchymal Stem Cells for Patients With Duchenne Muscular Dystrophy: A Phase 1 Clinical Study. (PubMed, J Clin Neurol) - "These results demonstrated that EN001 are safe and well tolerated for patients with DMD, and did not cause serious adverse events. The efficacy of EN001 could be confirmed through larger-scale future studies that incorporate repeated dosing and have a randomized controlled trial design."

Journal • P1 data • Dermatology • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Pain • Pediatrics

Evaluate the Efficacy and Safety of EN001 in Patients With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P1/2 | N=88 | Not yet recruiting | Sponsor: ENCell

New P1/2 trial • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Evaluate the Safety and Efficacy of EN001 in Patients With Charcot-Marie-Tooth Disease Type 1A (clinicaltrials.gov) - P1 | N=12 | Recruiting | Sponsor: ENCell

New P1 trial • Genetic Disorders

Evaluate the Safety and Potential Efficacy of Human Wharton's Jelly-derived Mesenchymal Stem Cells With Charcot-Marie-Tooth Disease Type 1E (clinicaltrials.gov) - P=N/A | N=3 | Not yet recruiting | Sponsor: Samsung Medical Center

New trial • Genetic Disorders

An Open-Label, Dose-Escalation, Phase 1a Study to Evaluate Safety, Tolerability, and Exploratory Efficacy of Intravenous Injection of Allogenic Early-Passage Mesenchymal Stem Cells (MSCs), Derived from Wharton's Jelly in the Umbilical Cord, in Patients with Duchenne Muscular Dystrophy (ASGCT 2023) - "Conclusions EN001 was safe and well-tolerated at both low-dose (5.0x105 cells/kg) and high-dose (2.5x106 cells/kg) for DMD patients. In addition, the data on treatment efficacy indicates that EN001 improved ambulatory capacity, pulmonary function, and muscle strength in some patients.This study suggests that allogeneic early-passage MSCs derived from Wharton's Jelly might have a potent therapeutic effect for DMD treatment and can be used in all subtype-specific DMD patients."

Clinical • P1 data • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Oncology

Determine the Safety and Dose of EN001 in Patients With Charcot-Marie-Tooth Disease (CMT) Type 1A (clinicaltrials.gov) - P1 | N=9 | Completed | Sponsor: ENCell | Recruiting ➔ Completed | Trial completion date: Dec 2027 ➔ Dec 2022

Trial completion • Trial completion date • Genetic Disorders

Determine the Safety and Dose of EN001 in Patients With Duchenne Muscular Dystrophy(DMD) (clinicaltrials.gov) - P1 | N=7 | Completed | Sponsor: ENCell | Recruiting ➔ Completed | N=12 ➔ 7 | Trial completion date: Aug 2028 ➔ Dec 2022

Enrollment change • Trial completion • Trial completion date • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Determine the Safety and Dose of EN001 in Patients With Duchenne Muscular Dystrophy(DMD) (clinicaltrials.gov) - P1 | N=12 | Recruiting | Sponsor: ENCell | Trial completion date: Aug 2027 ➔ Aug 2028 | Trial primary completion date: May 2022 ➔ Jan 2023

Trial completion date • Trial primary completion date • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Determine the Safety and Dose of EN001 in Patients With Charcot-Marie-Tooth Disease (CMT) Type 1A (clinicaltrials.gov) - P1 | N=12 | Recruiting | Sponsor: ENCell | Trial completion date: Aug 2027 ➔ Dec 2027 | Trial primary completion date: May 2022 ➔ Dec 2022

Trial completion date • Trial primary completion date • Genetic Disorders