US-based biotechnology firm Arcellx has received a clinical hold notification from the US Food and Drug Administration (FDA) for its CART-ddBCMA investigational new drug (IND), which is in the iMMagine-1 Phase 2 clinical programme.
CART-ddBCMA is a BCMA-specific CAR-modified T-cell therapy for the treatment of patients with relapsed or refractory multiple myeloma (rrMM). It uses the company’s BCMA-targeting binding domain.
Arcellx received the FDA clinical hold following a recent patient death.
The biotechnology company is working with FDA to amend the protocol to offer patients more options that are compatible with current clinical practice.
Arcellx believes that constraints on bridging therapy are a contributing cause and is now authorised by the FDA to continue dosing patients who have undergone lymphodepletion.
Arcellx chairman and CEO Rami Elghandour said: “The safety and well-being of patients enrolled in our studies is our top priority.
“In coordination with our investigators, data safety monitoring board (DSMB), and our partners at Kite Pharma, we are working with FDA to address the clinical hold. The expansion of bridging therapy regimens is consistent with what’s currently available in clinical practice and is in the best interest of patients.
“Additionally, we continue to evaluate other potential improvements to the study. We remain confident that CART-ddBCMA is a potential best-in-class therapy for the treatment of patients with rrMM based on the clinical profile observed in the patients dosed to date across our studies.
“We look forward to resolving this matter expeditiously and to continue to advance our therapy to the benefit of patients suffering from rrMM.”
Arcellx’s CART-ddBCMA has secured the Fast Track, Orphan Drug, and Regenerative Medicine Advanced Therapy Designations from the US health regulator.
The investigational therapy is currently in iMMagine-1 Phase 2 study. It is an open-label, multicenter clinical trial designed to evaluate CART-ddBCMA for the treatment of adult patients with rrMM.
The study’s primary objective is to assess the total response rate over a 24-month span. The depth of the disease response, the duration of the response, and overall survival over a 24-month period are secondary endpoints in addition to safety.
