ENCell will adjust the clinical strategy for its sarcopenia drug candidate and expand the scope of efficacy verification.
ENCell said on the 3rd that it filed a notice that it had submitted an application for approval to amend the investigational new drug (IND) for the sarcopenia indication of its allogeneic umbilical cord-derived mesenchymal stem cell therapy candidate EN001. The move is seen as a plan to expand from phase 1/2a to phase 1/2 to explore efficacy in more patients.
According to the filing, the trial will be conducted as a multicenter, randomized, double-blind, placebo-controlled study. With approval from the Ministery of Food and Drug Safety, it will be carried out in Korea. Phase 1 will be conducted at a single site at Samsung Medical Center, and phase 2 will be conducted at Samsung Medical Center and additional medical institutions.
In phase 1, safety and tolerability with repeated dosing of EN001 will be evaluated to determine the maximum tolerated dose (MTD) and the recommended phase 2 dose (RP2D). In phase 2, EN001's efficacy versus placebo will be assessed by the change from baseline at week 24 in the total score of the Short Physical Performance Battery (SPPB).
Phase 1 will use an open-label dose-escalation (3+3) design, enrolling 3–6 participants per cohort, administering three doses at 4-week intervals, and then evaluating dose-limiting toxicity (DLT). In phase 2, low-dose, high-dose, and placebo groups will be randomized in a 1:1:1 ratio to compare efficacy and safety.
Primary endpoints are DLT and treatment discontinuation-related adverse drug reactions in phase 1, and the change in SPPB score at week 24 in phase 2. For statistical analysis, analysis of covariance (ANCOVA) will be applied to compare mean changes between each treatment arm and the control arm.
The clinical trial period is expected to be about 30 months from the date of institutional review board (IRB) approval. The target enrollment is 9–18 participants for phase 1 and 114 for phase 2. Phase 2 plans to enroll 38 participants per arm, assuming a 20% dropout rate.
The company said the amendment is intended to more clearly explore the efficacy of EN001 in the low-dose and high-dose groups.