FDA Clears IND for CT071 in RRMM and Primary Plasma Cell Leukemia

• An investigational new drug application was cleared by the FDA for CT071 in this indication.
• CT071 is an autologous chimeric antigen receptor (CAR) T-cell therapy candidate being developed for the treatment of multiple myeloma (MM) and primary plasma cell leukemia (PCL).
• A phase 1 trial evaluating CT071 is ongoing in China.

The FDA has cleared an investigational new drug application for CT071 for the treatment of patients with relapsed/refractory (R/R) MM or R/R primary PCL, according to CARsgen Therapeutics Holdings Limited.¹

CT071 is an autologous CAR T-cell therapy candidate which incorporates a fully human single-chain variable fragment. The therapy works by targeting G protein-coupled receptor class C group 5 member D (GPRC5D). Due to the overexpression of GPRC5D on the surface of malignant plasma cells, as well as the limited presence of GPRC5D on normal tissues, this CAR T-cell therapy is an ideal candidate for the treatment of MM and PCL.

Manufactured with CARsgen's proprietary CARcelerate platform, CT071 yields younger, healthier, and possibly more potent CAR T cells vs conventional manufacturing as the manufacturing time is less than 2 days. The supply capacity is also enhanced with this platform, manufacturing costs are reduced, and the availability of the product and time to get it to the patient is expedited.

A phase 1 clinical trial (NCT05838131) in China is ongoing and evaluating the safety and efficacy of CT071 for the treatment of patients with RRMM or PCL.²

Male and female patients aged 18 years and older with MM who were resistant to at least 2 classes of anti-MM drugs (including at least 1 proteasome inhibitor), MM that failed at least 3 lines of therapy, secondary PCL that had received at least 1 line of therapy, and primary PCL that had progressed after treatment with at least 1 regimen will be included in the study. Patients are required to have progressive disease at the time of enrollment, have serum M-protein ≥ 5 g/L; 2. 24-hour urine M-protein ≥ 200 mg, abnormal serum free light chain (FLC) ratio and affected FLC ≥ 100 mg/L in subjects with MM who did not meet evaluable criteria for either serum or urine M-protein levels, or circulating plasma cells ≥ 5%. Patients also need to have an estimated survival of > 12 weeks, an ECOG performance status of 0-2 and adequate organ function to enroll in the study.

A negative serum pregnancy test at screening is required by all female patients of childbearing potential. They must also be willing to use a highly effective method of contraception within 1 year after receiving the trial treatment and absolutely prohibit egg donation during the trial and within 1 year after receiving the trial treatment. If sexually active with a female of childbearing potential, male patients must use a reliable form of contraception for 1 year after receiving trial treatment. Male patients are also prohibited from donating sperm during the trial and for 1 year after receiving the trial treatment.

REFERENCES:

1. CARsgen's CT071 received IND clearance from the FDA for treating relapsed/refractory multiple myeloma or relapsed/refractory primary plasma cell leukemia. December 4, 2023. Accessed December 5, 2023. News release. CARsgen Therapeutics Holdings Limited. https://tinyurl.com/3r3wfefy

2. Study of CT071 injection in RRMM or PPCL. Multiple Myeloma Research Foundation. Accessed December 5, 2023. https://tinyurl.com/df4hpcjb