On May 23, 2025 Autolus Therapeutics plc (Nasdaq: AUTL), an early commercial-stage biopharmaceutical company developing, manufacturing and delivering next-generation programmed T cell therapies and candidates, reported that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended European Commission (EC) approval of obecabtagene autoleucel (obe-cel) for the treatment of adult patients, 26+, with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (r/r B-ALL) (Press release, Autolus, MAY 23, 2025, View Source [SID1234653363]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"This positive CHMP opinion is a welcome advancement for physicians and patients in Europe, faced with treating r/r adult B-ALL patients with a poor prognosis," said Dr. Claire Roddie, MD, PhD, FRCPath, Lead Investigator of the FELIX study and Associate Professor of Haematology at the University College London (UCL) Cancer Institute. "Obe-cel’s combination of favorable tolerability and potential long-term outcomes could offer an important new treatment option for patients in the EU."

The CHMP recommendation was based on the results of the FELIX study, an open-label, multi centre, single arm study in adult patients with relapsed or refractory B-cell acute lymphoblastic leukaemia. The results were published in the New England Journal of Medicine in November 20241. In the pivotal cohort of patients, (cohort IIA (n=94)), the Complete Response/Complete Response with Incomplete Haematological Recovery (CR/CRi) for patients who received at least one infusion of obecabtagene autoleucel was 76.6%. Median response duration for all infused patients was 21.2 months. Median event-free survival (EFS) was 11.9 months and the estimated 6- and 12-month event-free survival rates were 65.4% and 49.5%, respectively.

The most common non-laboratory Grade 3 or higher adverse reactions were infections-pathogen unspecified (32%), febrile neutropenia (24%) and bacterial infectious disorders (11%). Cytokine release syndrome developed in 87 of the 127 patients (68.5%), with events of grade 3 or higher in three patients (2.4%). Immune effector cell-associated neurotoxicity syndrome developed in 29 of the 127 patients (22.8%), with grade 3 or higher occurring in nine patients (7%).

"This positive opinion from the CHMP highlights the significant unmet need and importance of effective treatment options for adult r/r B-ALL," said Dr. Christian Itin, Chief Executive Officer of Autolus. "With FDA approval received in November 2024 and an MHRA conditional marketing authorization received in April 2025, we are on our way to bringing this therapy to patients in need globally."

Obe-cel is an autologous CD19 CAR T cell therapy with a proprietary CD19 CAR, invented by a team led by Dr. Martin Pule, at University College London, along with collaborators at Great Ormond Street Hospital and University College London Hospital. The CAR is designed to have a fast "off-rate" which mimics physiological T-cell receptor interactions2.

ALL is an aggressive type of blood cancer that can also involve the lymph nodes, spleen, liver, central nervous system and other organs. In Europe, there are approximately 6,0002 new cases of ALL diagnosed every year. In frontline treatment for adult B-ALL, up to 50% of patients will ultimately relapse3. Survival rates remain very poor in adult patients with r/r ALL, with median overall survival of eight months with conventional treatments4, and the standard-of-care treatment can trigger severe toxicities5.

The positive CHMP opinion is a scientific recommendation for marketing authorization, serving as a basis for the EC’s final decision on Autolus’ MAA for obe-cel for adult r/r B-ALL patients. The EC is expected to make a decision following CHMP recommendation, and the decision will apply to all 27 European Union Member States, Iceland, Norway and Liechtenstein. Obe-cel is currently approved by the U.S. Food and Drug Administration (FDA) (Nov 8, 2024), and authorized by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) (April 25, 2025).

On May 23, 2025 Mabwell (688062.SH), an innovation-driven biopharmaceutical company with entire industry chain, reported multiple clinical research results to be presented at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, including phase Ib/II clinical study data of 9MW2821 (Bulumtatug Fuvedotin, BFv), a novel Nectin-4 targeting ADC, in combination with Toripalimab in locally advanced or metastatic uroepithelial carcinoma to be presented as oral presentation, and clinical study data of 7MW3711 and 9MW2921, a novel B7-H3 targeting ADC and a novel Trop-2 targeting ADC respectively, to be presented as poster presentations (Press release, Mabwell Biotech, MAY 23, 2025, View Source [SID1234653365]). The study of 9MW2821 was the only selected Chinese oral presentation in the field of urothelial carcinoma at this meeting.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Nectin-4 ADC (9MW2821):

In the phase Ib/II clinical study of 9MW2821 in combination with Toripalimab for the treatment of patients with locally advanced or metastatic urothelial carcinoma (la/mUc), a total of 40 patients with previously untreated for la/mUC were enrolled in the study and were treated with 9MW2821 (1.25 mg/kg) and Toripalimab (240 mg).

As of December 19, 2024, the objective response rate (ORR) was 87.5% and the confirmed ORR was 80%. The disease control rate (DCR) was 92.5%. Median progression-free survival (PFS) and duration of response (DoR) have not been achieved. No new safety signals of 9MW2821 or Toripalimab were observed in this study.

9MW2821 stands out as the first clinical-stage drug candidate among Chinese companies for this specific target. And it’s the first Nectin-4 targeting ADC to disclose clinical efficacy data in cervical, esophageal, and breast cancers. Currently, 3 Phase III pivotal clinical trials are ongoing. Its monotherapy and combination therapy with Toripalimab for urothelial carcinoma have been granted Breakthrough Therapy Designation (BTD) by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) in China. It has also been granted Fast Track Designations (FTD) for 3 indications and Orphan Drug Designation (ODD) for 1 indication by the U.S. Food and Drug Administration (FDA).

B7-H3 ADC (7MW3711):

7MW3711 has enrolled 43 patients as of January 2, 2025 in a phase I/II clinical study in patients with advanced solid tumors. During the dose-escalation phase, dose-limiting toxicity (DLT) was not observed and the maximum tolerated dose (MTD) has not been reached. In patients evaluated at doses of 4.5 mg/kg or higher, the ORR for esophageal cancer, ovarian cancer, and prostate cancer was 33.3%, 60.0%, and 50.0%, respectively, and the DCR was 100%.

In the phase I/II clinical study in lung cancer patients, 37 lung cancer patients were enrolled as of January 8, 2025, including 16 small cell lung cancer (SCLC) patients and 21 non-small cell lung cancer (NSCLC) patients. Common grade ≥3 adverse reactions were neutrophil count decreased, white blood cell count decreased, anemia, lymphocyte count decreased, and platelet count decreased. Among the 25 patients who received 7MW3711 at a dose of 4.5 mg/kg or greater and completed at least one tumor evaluation, the ORR was 36.0% and the DCR was 96.0%; of these, the ORR and DCR were 62.5% and 100.0%, respectively, for patients with small-cell lung cancer at a dose of 4.5 mg/kg. Among squamous lung cancer (Sq-NSCLC) patients with a B7-H3 H-score >5, the ORR and DCR were 37.5% and 87.5%, respectively.

The study results suggest that 7MW3711 has a tolerable safety profile and good anti-tumor activity in patients with advanced tumors such as lung, esophageal, prostate and ovarian cancers.

7MW3711 was developed with Mabwell’s Interchain-Disulfide Drug Conjugate (IDDC) platform. It consists of an innovative antibody molecule, a novel linker, and a novel payload: Mtoxin, which is a topoisomerase I inhibitor. It has been granted ODD by the FDA for the treatment of SCLC.

Trop-2 ADC (9MW2921):

In the first-in-human clinical study in patients with advanced solid tumors, a total of 39 patients were enrolled as of November 12, 2024. The most common ≥3 grade adverse were stomatitis, anemia, white blood cell count decreased, neutropenia, lymphocyte count decreased, etc. The ORR of 3.0 mg/kg dose group was 42.1% and the DCR was 84.2%. Under this dose level, the ORR and DCR was 75% and 100% for endometrial cancer, 50% and 75% for HR+/HER2- breast cancer, 50% and 100% for gastric cancer, 25% and 100% for non-squamous non-small cell lung cancer.

The study result suggests that 9MW2921 has a tolerable safety profile and good anti-tumor activity in patients with advanced tumors.

9MW2921 was developed with Mabwell’s Interchain-Disulfide Drug Conjugate (IDDC) platform. It consists of an innovative antibody molecule, a novel linker, and a novel payload: Mtoxin, which is a topoisomerase I inhibitor.

On May 23, 2025 BriaCell Therapeutics Corp. (Nasdaq: BCTX, BCTXW, BCTXZ), (TSX: BCT) (" BriaCell " or the " Company "), a clinical-stage biotechnology company developing novel immunotherapies to transform cancer care, reported the presentation of positive survival and clinical benefit data in three clinical poster presentations and one publish-only abstract at the upcoming 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting taking place May 30 – June 3 at McCormick Place, Chicago, IL (Press release, BriaCell Therapeutics, MAY 23, 2025, View Source [SID1234653598]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are highly encouraged by the survival and clinical benefit data from our Phase 2 Bria-IMT study which meets and outperforms outcomes seen with FDA approved therapies – despite our patients being more heavily pre-treated," stated Dr. William V. Williams, BriaCell’s President & CEO. "These results reinforce our belief in Bria-IMT’s potential to address the urgent, unmet needs of patients with metastatic breast cancer and we look forward to confirming results in our ongoing pivotal Phase 3 study."

"These data provide additional validation of the mechanism of action and support the clinical efficacy of Bria-IMT," noted Giuseppe Del Priore, MD, MPH, BriaCell’s Chief Medical Officer. "Importantly, the regimen has been well-tolerated and its favorable safety and efficacy profile positions it as a promising, less toxic therapeutic option for patients battling metastatic breast cancer."

The details of the poster presentation sessions and publish-only abstract are listed below.

Abstract Title: Update on phase III pivotal trial of Bria-IMT + CPI vs physician’s choice in advanced metastatic breast cancer (BRIA-ABC)
Session Date and Time: June 2, 2025 9:00 AM-12:00 PM CDT
Abstract Number for Publication: TPS1138
Poster Board Number: 108a
Session Type and Title: Poster Session – Breast Cancer—Metastatic

Preliminary selected clinical and biomarker data from BriaCell’s ongoing pivotal Phase 3 study of Bria-IMT plus an immune checkpoint inhibitor (CPI) in metastatic breast cancer ( NCT06072612 ).

Abstract Title: Bria-IMT + checkpoint inhibitor: Phase I/II survival results compared to benchmark trials in metastatic breast cancer
Session Date and Time: June 2, 2025 9:00 AM-12:00 PM CDT
Abstract Number for Publication: 1096
Poster Board Number: 75
Session Type and Title: Poster Session – Breast Cancer—Metastatic

Phase 2 study of Bria-IMT in combination immunotherapy with an immune checkpoint inhibitor (CPI) consisted of 54 patients; 11 received pembrolizumab, 44 retifanlimab (1crossover).

Table 1: Median overall survival (OS), progression-free survival (PFS), objective response rate (ORR), and clinical benefit rate (CBR) were evaluated against two pivotal Phase 3 studies of FDA approved drugs, ASCENT (Trodelvy (SG) or Treatment of Physicians Choice (TPC) in triple-negative breast cancer (TNBC)) and TROPiCS-02 (SG or TPC in Hormone Receptor +/HER2- MBC)
Trial (Cohort) Age (median, range) Prior Therapies (median, range) OS (months) PFS (months) ORR
(%) CBR
(%)
Bria-IMT (Overall Cohort) 61 (38-81) 6 (2-13) 9.9 (1.8-30.3) 3.6 10% 55%
Bria-IMT (Phase 3 regimen) 62 (44-80) 6 (2-13) 13.43 (1.8-30.3) 3.6 (1.8-16.5) 14 % 61 %
ASCENT (SG) 54 (27-82) 4 (2-17) 11.8 4.8 31% 40%
ASCENT (TPC) 53 (27-81) 4 (2-14) 6.9 1.7 4% 8%
TROPiCS-02 (SG) 57 (49-65) 3 14.4 5.5 21% 34%
TROPiCS-02 (TPC) 55 (48-63) 3 11.2 4 14% 22%
Bria-IMT was well-tolerated with no treatment-related discontinuations. 22% of patients are still in active survival follow up.

This analysis does not stratify Bria-IMT patients by hormone receptor or HER2 status, and the dataset reflects the information available at the time of abstract submission. Updated data will be shared during the company’s presentation at ASCO (Free ASCO Whitepaper) 2025.

PFS (median) and OS (median): Patients treated with Bria-IMT’s selected Phase 3 formulation (without IFNγ; N = 37) demonstrated significantly improved progression-free survival (3.6 vs. 2.6 months; P = 0.01) and overall survival (13.4 vs. 6.9 months; P = 0.01) compared to those who received the alternate formulation not advanced to Phase 3.

The overall survival observed in the Phase 3 formulation cohort (13.4 months) was comparable to that reported in the ASCENT (11.8 months) and TROPiCS-02 (14.4 months) studies and exceeded survival outcomes in their respective treatment of physician’s choice (TPC) arms (6.9 and 11.2 months).

BriaCell’s Phase 2 study also achieved a Clinical Benefit Rate (CBR) of 61%, outperforming ASCENT (40%) and TROPiCS-02 (34%), and an Objective Response Rate (ORR) of 14%, which matched or exceeded the TPC arms in both studies (4% and 14%).

Importantly, these outcomes were achieved in a more heavily pretreated patient population than those in the comparator trials—highlighting Bria-IMT’s strong anti-cancer activity. The favorable efficacy profile of the Phase 3 formulation supports its continued evaluation in BriaCell’s ongoing pivotal Phase 3 trial comparing Bria-IMT to treatment of physician’s choice ( NCT06072612 ).

Abstract Title: Trial in progress: A study of Bria-OTS cellular immunotherapy in metastatic recurrent breast cancer
Session Date and Time: June 2, 2025 9:00 AM-12:00 PM CDT
Abstract Number for Publication: TPS1136
Poster Board Number: 107a
Session Type and Title: Poster Session – Breast Cancer—Metastatic

Heavily pre-treated MBC patients were treated in a dose escalation Phase 1/2 study of Bria-OTS monotherapy (single agent). The Phase 1 part of the study has enrolled and treated 3 patients. The first patient treated with single agent therapy has confirmed resolution of a breast cancer metastasis in the lung and remains on study.

Publish-Only Abstract Title : Impact of HLA Matching on Clinical Outcomes in a Phase 2 Trial of Bria-IMT Plus Anti PD1 in Advanced Breast Cancer

Following the presentations, copies of the presentations will be posted on View Source

On May 23, 2025 GV20 Therapeutics (GV20), a clinical-stage AI-powered biotherapeutics company, reported that Dr. Kristopher Wentzel from the Angeles Clinic and Research Institute will present updated clinical and translational data of GV20-0251 monotherapy at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2025 Annual Meeting, taking place in Chicago, IL on May 30- June 3, 2025 (Press release, GV20 Therapeutics, MAY 23, 2025, View Source [SID1234653366]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

This presentation at ASCO (Free ASCO Whitepaper) builds on previously presented clinical data of the novel immune checkpoint IGSF8 inhibitor GV20-0251 (Wentzel et al, ESMO (Free ESMO Whitepaper) 2024) and will report updated clinical and translational findings from the monotherapy dose escalation portion of the ongoing Phase 1/2 trial evaluating GV20-0251 in patients with advanced solid tumors resistant to anti-PD(L)1 and other standard therapies (NCT05669430).

GV20-251 is the first clinical stage, AI-designed antibody therapeutic against an AI-predicted target in the clinic.

Presentation details (Abstract 2531):

Title: Preliminary monotherapy efficacy of novel immune checkpoint blockade GV20-0251 (anti-IGSF8) in advanced melanoma patients with primary resistance to anti-PD1
Session Title: Developmental Therapeutics—Immunotherapy
Session Type: Poster session
Session Date/Time: Monday, June 2, 2025, 1:30 PM – 4:30 PM CDT
Location: Hall A – Posters and Exhibits
Board Number: 178

On May 23, 2025 Verismo Therapeutics, a clinical-stage CAR T company pioneering the KIR-CAR platform, reported additional details on the Trials in Progress poster presentation at the upcoming 2025 American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, being held from May 30 – June 3, 2025 in Chicago, IL (Press release, Verismo Therapeutics, MAY 23, 2025, View Source [SID1234653367]). The trial Principal Investigator, Janos L. Tanyi, MD, PhD, of the Perelman School of Medicine at the University of Pennsylvania, will present an overview of the ongoing STAR-101 Phase 1 clinical study (NCT05568680).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The STAR-101 trial is a first-in-human, multicenter, open-label, Phase 1 dose-escalation study designed to evaluate the safety, feasibility, and preliminary efficacy of SynKIR-110 in patients with advanced mesothelin-expressing tumors, including ovarian cancer, mesothelioma, and cholangiocarcinoma who have received at least 1 prior line of systemic therapy. SynKIR-110 utilizes Verismo’s novel KIR-CAR platform, employing a multi-chain signaling approach derived from natural killer cells in T cells. Preclinically, this unique mechanism has shown the ability to reduce T cell exhaustion and improve anti-tumor activity and functional persistence, thereby potentially overcoming critical hurdles that limit traditional CAR T cell therapies in solid tumors.

"This study represents an important advancement for ovarian cancer, a disease with few available treatment options," Dr. Tanyi said. "This novel therapy could have the potential to impact solid tumors by overcoming key limitations of existing CAR T cell treatments."

Poster Details:

Poster Board Number: 522a

Location: Hall A – Posters and Exhibits

Abstract Number: TPS5630

Abstract Title: SynKIR-CAR T Cell Advanced Research (STAR)-101 Phase 1 clinical trial for patients with advanced mesothelin-expressing ovarian cancer, mesothelioma, or cholangiocarcinoma.

Presenting Author: Janos L. Tanyi, M.D., Ph.D., Perelman School of Medicine at the University of Pennsylvania

Session Title: Gynecologic Cancer

Session Date: June 1, 2025, 9:00 AM-12:00 PM CDT

The Poster will be made available on the company’s website following the presentation.

About the KIR-CAR Platform

The KIR-CAR platform is a multi-chain CAR T cell therapy that has shown highly effective prolonged solid tumor treatment in otherwise CAR-resistant preclinical animal models with challenging tumor microenvironments. Using NK cell derived KIR and DAP12 split signaling provides a novel paired activation and co-stimulation separate from the usual T cell stimulation pathways. KIR-CAR also enables sustained chimeric receptor expression with improved long-term CAR T cell function and decreased T cell exhaustion. This results in CAR T cell resistance to tumor immunosuppression, prolonged functional persistence and improved tumor elimination. Together, this platform provides the potential for improving CAR T treatment in both solid and hematologic tumors.