Appclon Begins Preparation for Rapid Approval Application of CART Therapy AT101

Appclon announced on the 7th that the Phase 2 clinical trial of the CAR-T therapy AT101 under development is progressing smoothly, and preparations for expedited approval are being accelerated.

AT101 is an advanced biopharmaceutical being developed for patients with B-cell lymphoma (BCL) who have relapsed or are unresponsive to existing treatments. An interim analysis of the treatment responsiveness of Phase 2 patients who have already received AT101 has begun, and based on this, an application for designation as an expedited review advanced biopharmaceutical (hereinafter referred to as expedited review designation) will be submitted.

Currently, CAR-T therapies targeting CD19 in domestic and international markets include Kymriah, Yescarta, and Breyanzi, with Kymriah being the only one commercially available in Korea. Since CAR-T therapy is administered as a single dose to patients with no other treatment options, high response rates and low relapse rates are key factors determining the success of the therapy.

In fact, although Novartis's Kymriah was the first to be commercialized in the global market, the latecomer Yescarta is leading the CD19 CAR-T therapy market. This is because it is considered a favorable therapy due to better efficacy with a single administration.

In the case of Appclon, it is expected to have sufficient competitiveness through differentiated benefits compared to existing global CAR-T therapies, as it possesses a new antibody mechanism based on h1218, developed for the first time in the world. Excellent effects were already confirmed in Phase 1 clinical trials in terms of patient response rate and post-administration patient survival rate.

An Appclon official stated, “Since receiving approval for the expedited testing method from the Ministry of Food and Drug Safety (MFDS) last August, the AT101 administration period has been shortened, enabling faster treatment for patients. Expectations for the interim analysis results are rising, and we plan to apply for the first step of expedited approval, the ‘expedited review designation,’ in the first half of this year.”

He added, “The development of AT101 and the pursuit of expedited approval are expected to provide new treatment opportunities and hope to B-cell lymphoma patients worldwide.”

The expedited review designation is a system where the MFDS forms a dedicated team to rapidly review drugs that treat life-threatening or serious diseases when safety and efficacy are proven. This opens the way to obtain approval faster than the usual process of receiving product approval after clinical trials are completed. It also provides benefits such as customized review and partial exemption of clinical trial data.

Appclon is preparing related documents in cooperation with consulting firms experienced in GMP (Good Manufacturing Practice) evaluation to shorten the AT101 product launch schedule as much as possible. Based on the successful development of AT101 and the establishment of an automated production process system, the company is also focusing on expanding various follow-up pipelines, including the switchable CAR-T AT501 targeting solid tumors.