[Edaily Reporter Kim Hyung-il ] Aprogen(007460) announced on the 31st that its subsidiary, Geolit Energy(270520), has secured global exclusive rights to “Burixafor (GPC-100)” and will proceed with U.S. Phase 3 clinical trials and the application for marketing approval.

GPC-100 is a mobilizing agent that directs hematopoietic stem cells and immune cells into the peripheral blood for patients with blood cancers such as multiple myeloma. GPC-100 works by binding to a complex of the CXCR4 and ADRB2 cell membrane proteins and has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA).

Based on the results of its U.S. Phase 2 clinical trial, APTN plans to consult with the FDA to finalize the Phase 3 clinical trial protocol and commence late-stage clinical trials. APTN anticipates the possibility of rapid development for GPC-100, citing the example of Motixapotide (brand name Apexda)—another CXCR4-targeted drug—which received FDA approval in 2023 following a single Phase 3 clinical trial involving 122 participants.

There are also benefits associated with the FDA’s Orphan Drug designation. The company can utilize certain expedited review programs during the approval process, and if approved in the U.S., it will be granted seven years of market exclusivity for the drug as an orphan drug.

APTN is initially targeting the hematopoietic stem cell mobilization market for patients with blood cancers as its primary focus. The company explains that as the use of Darzalex (active ingredient: daratumumab), a treatment for multiple myeloma, expands, GPC-100 can be utilized during the hematopoietic stem cell mobilization process. According to APTN, a U.S. Phase 2 clinical trial confirmed that GPC-100 takes effect within one hour of administration.

APTN explained that the scope of GPC-100’s application could potentially extend beyond hematopoietic stem cell mobilization to the fields of cell and gene therapy. In particular, the company is exploring ways to utilize it as a platform to enhance the efficiency of in vivo CAR-T therapy by leveraging its ability to rapidly increase the number of immune cells in the blood.

APTN plans to conduct a Phase 3 clinical trial in the U.S. and expand the scope of application to include not only multiple myeloma but also acute myeloid leukemia (AML) and sickle cell disease (SCD).