Data Roundup: June 2025 Features Updates from EHA, ASCO, and More

Last month, June 2025, the CGTLive® team was diligently tracking the latest data readouts and published literature on cell and gene therapies within oncology, neurology, rare diseases, and more.

As more and more innovative therapies enter the clinical trial field, more data is accrued every month, buoying excitement in the field and sometimes making or breaking the fates of small biotech companies. Last month delivered data updates from several conferences, such as the European Hematology Association (EHA) 2025 Congress, held June 12 to 15, both virtually and in Milan, Italy, and the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting, held May 30 to June 3, in Chicago, Illinois. Our team has highlighted these below.

Click the read more buttons for more details and information about each update.

Immix Biopharma’s CAR-T NXC-201 Effects Deep Hematologic Responses in Light Chain Amyloidosis

June 3, 2025 - Immix Biopharma and its subsidiary Nexcella's NXC-201, an investigational autologous BCMA-directed chimeric antigen receptor T-cell (CAR-T) therapy, has generated deep hematologic responses in patients with relapsed/refractory (r/r) light chain (AL) amyloidosis who were treated in the phase 1b/2 NEXICART-2 clinical trial (NCT06097832), which is taking place in the United States. The data were presented at the 2025 ASCO Annual Meeting.

The results reported in the ASCO abstract included 7 patients treated in the trial, whose follow-up ranged from 7 to 209 days (median, 97). Following treatment with NXC-201, all 7 patients (100%) showed normalized pathological disease markers. Furthermore, 6 of the 7 patients showed normalized free light chains (FLCs) at 7 to 14 days posttreatment (median, 7) and a decrease in their dFLC to less than 1 mg/dL. At Day 25 or Day 26 posttreatment, 5 of the 7 patients showed minimal residual disease (MRD) negativity in the bone marrow at a sensitivity of 10^-6. One patient did not have MRD data available at the data cutoff. First author Heather Landau, MD, the MSKCC Amyloidosis Program director and the NEXICART-2 principal study investigator, and colleagues also noted that at 15 days posttreatment 1 patient achieved a renal organ response along with resolution of the m-spike. It was additionally stated that as of the abstract’s data cut-off, all of the 7 patients were in a state of complete response or very good partial response, and no relapses had occurred. In addition, at 14 days posttreatment 1 patient achieved an improvement in New York Heart Association (NYHA) class from II to I.

With regard to safety, 4 patients experienced grade 1 cases of cytokine release syndrome (CRS) and 1 patient experienced a case of grade 2 CRS. In 3 patients CRS onset began at Day 1 and in 2 patients CRS onset began at Day 3. All cases lasted less than 24 hours after administration of tocilizumab. No neurotoxicity, febrile neutropenia, treatment-related infections, cardiac toxicity, or deaths occurred in the study. Three patients experienced grade 3 cases of neutropenia and 2 patients experienced grade 4 cases of neutropenia. A grade 4 case of acute on chronic kidney injury occurred in a patient who had preexisting stage 4 chronic kidney disease before being enrolled in the study.

Iovance Biotherapeutics’ Lifileucel Produces Durable Responses in 5-Year Analysis of Advanced Melanoma Data

June 3, 2025 - Iovance Biotherapeutics’ lifileucel (marketed as Amtagvi), an FDA-approved autologous tumor infiltrating lymphocyte (TIL) therapy, has produced durable responses in a 5-year analysis of data from patients with advanced melanoma treated the phase 2 C-144-01 clinical trial (NCT02360579). The analysis is being presented at the 2025 ASCO Annual Meeting, held May 30 to June 3, in Chicago, Illinois, and was simultaneously published in The Journal of Clinical Oncology.

The analysis included 153 patients with a median follow-up of 57.8 months. All of these patients completed or discontinued participation in C-144-01 and 28 of the 153 (18.3%) completed the 5-year study follow-up. The complete response rate was 5.9% and the partial response rate was 25.5%, thus yielding an objective response rate of 31.4%. Furthermore, the patients achieved a median duration of response of 36.5 months (95% confidence interval [CI]: 8.3–not reached). A sustained response at completion of the 5-year assessment was seen in 31.3% of the patients whose disease responded. The time to best response ranged from 1.3 to 30.4 months (median, 1.5 months). The median overall survival (OS) was reported as 13.9 months (95% CI: 10.6–17.8) and the 5-year OS rate was determined to be 19.7% (95% CI: 13.3–27.0). Iovance pointed out that for patients who responded, survival outcomes were consistent without regard to the time that their response began.

With regard to safety, first author Theresa Medina, MD, a medical oncologist at the University of Colorado Cancer Center on the Anschutz Medical Campus, and colleagues noted that no new safety signals appeared in the extended follow-up and that the treatment-emergent adverse events (AEs) that occurred were consistent with the known safety profile of nonmyeloablative lymphodepletion and interleukin-2. Iovance added that AE incidence dropped in a rapid manner in the 2 weeks following administration of lifileucel.

Academic CD19-Directed CAR-T Therapy Shows 86.7% ORR in Follicular Lymphoma

June 12, 2025 - An academic CD19-directed CAR-T therapy has produced an 86.7% (13 patients) objective response rate (ORR) in a study of 15 patients with follicular lymphoma. The data were presented in a poster at the EHA 2025 Congress by Katsiaryna Zharkova, of N. N. Alexandrov National Cancer Centre of Belarus.

Out of the 13 patients who responded to the CAR-T product, 11 patients (73.4%) achieved a complete response (CR) and 2 patients (13.3%) achieved a partial response. The 2 patients who did not achieve a response (13.3%) experienced disease progression. Furthermore, the OS for the 15 treated patients was 93% at a median follow-up of 11.7 months. Zharkova and colleagues reported that progression-free survival was 80%. At a median follow-up of 11.7 months, event-free survival was 80%±14% (13 without events).

“In the group of patients who received second-line therapy, 2 patients had a CR, 1 patient had a partial response, which became complete after 6 months,” Zharkova and colleagues wrote in the poster. “All patients who received first-line treatment achieved a CR and are currently alive without disease progression.”

Study Suggests β-Thalassemia Gene Therapy May Help Improve Growth in Pediatric Patients

June 12, 2025 - A study suggested that gene therapy used to treat transfusion-dependent β-thalassemia (TDT) may help improve growth by positively impacting height-for-age in pediatric patients. The data were presented in a poster at the EHA 2025 Congress by Jingyu Zhao, MPH, of State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin.

“Children and adolescents with TDT often experience growth retardation due to anemia and iron overload,” Zhao and colleagues wrote in the poster’s introduction, providing context for the study. “Although gene therapy can effectively alleviate the clinical symptoms, its impact on growth and development remains uncertain.”

The study included 3 boys and 6 girls who were treated with gene therapy for TDT at 5 to 16 years of age (median age at treatment, 12). Follow-up time for the patients ranged from 7 to 35 months (median, 12 months).

Vertex’s Islet Cell Therapy Zimislecel (VX-880) Restores Endogenous Insulin Secretion in Type 1 Diabetes

June 20, 2025 - Vertex Pharmaceuticals’ zimislecel (also known as VX-880), an investigational allogeneic insulin-producing islet cell therapy, has restored endogenous insulin secretion in patients with type 1 diabetes (T1D) treated in the phase 1/2 FORWARD clinical trial (NCT04786262). The data were announced in an oral presentation at the 85th Scientific Sessions of the American Diabetes Association® (ADA) , held June 20 to 23, in Chicago, IL, and simultaneously reported in the New England Journal of Medicine.

Among 12 patients with T1D with impaired hypoglycemic awareness treated in FORWARD, all 12 showed restored endogenous insulin secretion, as assessed via C-peptide. Furthermore, the patients showed cessation of severe hypoglycemia events and hit recommended glycemic control targets, which constituted A1C at less than 7% and time in range of greater than 70%. Use of exogenous insulin was cut down by a mean of 92% across all patients, with 10 of the 12 patients no longer using exogenous insulin at all.

With regard to safety, AEs were noted to be in line with expectations for islet infusion procedures and the immunosuppressive drug regimens. There were no other AEs seen in the treated patients.