Curocell announced on the 27th that it has filed an application with the Ministry of Food and Drug Safety (MFDS) for approval to amend its Phase 1/2 investigational new drug (IND) plan for CRC01 (anbacaptagene autoleucel, product name Anbalcel) in adult B-cell acute lymphoblastic leukemia (B-ALL).

■ Why Separate the Clinical Trial Plan

The purpose of this amendment application is to separate the clinical trial protocol in order to independently and efficiently operate the acute lymphoblastic leukemia (ALL) trial, following the product approval for relapsed or refractory diffuse large B-cell lymphoma (DLBCL).

The trial will evaluate the tolerability, safety, and efficacy of CRC01 in patients with relapsed or refractory adult B-cell acute lymphoblastic leukemia, and will be conducted as an open-label, multi-center, single-arm study. Trial sites include Seoul St. Mary's Hospital.

■ Phase 1 and Phase 2 Evaluation Design

In Phase 1, the tolerability of CRC01 will be evaluated and the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D) will be determined. In Phase 2, efficacy and safety after CRC01 administration will be confirmed through independent assessment.

The primary endpoint of Phase 2 is the overall complete remission (OCR) rate. The primary analysis will be conducted when patients complete the 3-month post-administration visit or discontinue earlier, and the trial is designed so that efficacy is considered demonstrated if the lower bound of the one-sided 97.5% confidence interval for the overall complete remission rate exceeds 40%.

■ Trial Scale and Timeline

The target number of subjects is up to 18 in three dose cohorts for Phase 1, and approximately 43 patients who complete CRC01 dosing for Phase 2. The trial period is approximately 86 months from the approval date by the MFDS and the Institutional Review Board (IRB), and may vary depending on subject enrollment.

Curocell stated that it submitted the initial application for approval of this clinical trial plan on January 7, 2022, and received approval on March 10 of the same year. If this amendment is approved, the company plans to immediately disclose the relevant details.

However, the company explained that results during the clinical trial and product approval process may fall short of expectations, and that it may therefore modify or abandon its commercialization plans.