Key Takeaways
- Azer-cel shows strong response rates in relapsed/refractory diffuse large B-cell lymphoma (DLBCL): The allogeneic CAR-T therapy achieved a 75% overall response rate and 55% complete response rate in relapsed/refractory DLBCL patients.
- FDA Fast Track and regulatory momentum: Imugene plans a Type B meeting with the FDA in Q4 2025 to discuss pivotal trial design following Fast Track Designation.
- Off-the-shelf CAR-T offers accessibility advantage: Azer-cel’s allogeneic design aims to overcome limitations of autologous CAR-T, including manufacturing delays and geographic treatment access.
Results from a Phase Ib trial (NCT03666000) showed that Imugene’s azer-cel (azercabtagene zapreleucel), an off-the-shelf, allogeneic CAR-T therapy, demonstrated significant promise in patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL). According to the company, it plans to request a Type B meeting with the FDA in Q4 to discuss registrational trial design, based on the strength of the data and following the therapy’s Fast Track Designation in March.1
Can Azer-cel Transform the CAR-T Landscape in Relapsed/Refractory DLBCL?
"We are very pleased with the continued positive data coming from the azer-cel trial, which further reinforces its potential as a treatment for DLBCL patients who have failed several previous lines of therapy,” said Leslie Chong, managing director, CEO, Imugene, in a press release. “The data also significantly improves our position from both a regulatory and commercial standpoint, and we look forward to expanding on these discussions with the FDA. Additionally, given the positive results, we are opening the trial to other niche blood cancer indications, such as PCNSL and other subtypes of B Cell Lymphoma, for CAR T naïve patients.This is a high unmet need with potential to expedite and expand the scope of azer-cel."
Trial Design and Objectives
- The ongoing, open-label, multicenter, parallel assignment, dose-escalation, and dose-expansion clinical trial is evaluating the safety and clinical activity of azer-cel in approximately 129 patients.
- Patients were randomly assigned to receive one of six different doses of the treatment.
- The dual primary endpoints of the trial are the frequency of patients with treatment-related adverse events (AEs), defined as dose-limiting toxicities, and objective response rate (ORR).
- Key secondary endpoints include complete response (CR), duration of response (DOR), progression-free survival, overall survival, time to next treatment, and the number of participants with AEs.2
Promising Early Results
- Results from July showed a 75% ORR and a 55% CR among evaluable patients treated with azer-cel in combination with interleukin-2, with some responses ongoing beyond 450 days.
- Currently, the DOR is still maturing, with full results expected at a later date.
- At this time, Imugene is still enrolling patients in the trial, with up to ten sites in the United States and six sites in Australia planned, after the first Australian patient was dosed in January.
Market Impact and Disease Burden
According to Imugene, DLBCL is the most common form of non-Hodgkin's lymphoma (NHL) and is also the fastest growing, with an estimated 160,000 cases reported globally and around 30,000 new cases in the United States annually.1
According to the American Cancer Society, NHL is among the most common cancers in the United States, representing 4% of all cases. By the end of 2025, it is estimated there will be approximately 80,350 new cases of NHL in the United States and 19,390 deaths. NHL can occur at any age and is one of the most common cancers in children, adolescents, and young adults.3
Commitment to Advancing Blood Cancer Therapies
"DLBCL remains one of the most aggressive forms of lymphoma, and despite the existing therapies, there are a large number of patients that still face relapse or resistance,” said John Byon, chief medical officer, Imugene, in the press release. “We are seeing significant potential from azer-cel to date in its ability to provide a critical step forward for these patients who have relapsed on multiple therapies, offering deep and durable responses with a one-time treatment. We remain deeply committed to transforming the standard of care in difficult-to-treat blood cancers, where significant unmet medical need still exists."
References
- Imugene Announces Outstanding Response Rates from the Phase 1b Trial of the Azer-cel Allogeneic CAR T in 3L+ DLBCL. PR Newswire. July 14, 2025. Accessed July 16, 2025. https://www.prnewswire.com/news-releases/imugene-announces-outstanding-response-rates-from-the-phase-1b-trial-of-the-azer-cel-allogeneic-car-t-in-3l-dlbcl-302504067.html
- Dose-escalation, Dose-expansion Study of Safety of Azer-cel (PBCAR0191) in Patients With r/r NHL and r/r B-cell ALL. Clinicaltrials.gov. Accessed July 16, 2025. https://clinicaltrials.gov/study/NCT03666000?utm_source=chatgpt.com
- Key Statistics for Non-Hodgkin Lymphoma. American Cancer Society. Accessed July 16, 2025. https://www.cancer.org/cancer/types/non-hodgkin-lymphoma/about/key-statistics.html
