EUROPEAN SOCIETY FOR BLOOD AND MARROW TRANSPLANTATION

MaaT013 Shows Promise in Phase 3 Trial for GI-aGvHD

4 months ago

• MaaT Pharma's MaaT013 met its primary endpoint in the Phase 3 ARES trial, demonstrating a 62% gastrointestinal overall response rate (GI-ORR) in patients with GI-aGvHD. • The study showed a 54% estimated 12-month overall survival rate, with responders at day 28 exhibiting a significantly higher survival rate of 67% compared to non-responders. • MaaT Pharma plans to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in mid-2025 for MaaT013 as a third-line treatment. • The ARES trial represents the first positive pivotal clinical study for a microbiome-based approach in GI-aGvHD, potentially offering a new treatment option for patients.

Narsoplimab Shows Significant Survival Benefit in TA-TMA Patients, BLA Resubmission Planned

5 months ago

• Narsoplimab significantly reduced the risk of mortality in high-risk TA-TMA patients by over 3-fold compared to an external control group. • Sensitivity analyses further confirmed the robustness of narsoplimab's survival benefit, with p-values ranging from 0.00001 to 0.0124. • Omeros plans to resubmit the Biologics License Application (BLA) to the FDA for narsoplimab, seeking approval as the first therapy for TA-TMA. • The European marketing authorisation application (MAA) submission to European regulators is targeted by mid-year 2025.

Orca-T Demonstrates Promising Outcomes in Hematologic Malignancies

5 months ago

• Orca-T, an allogeneic T-cell immunotherapy, shows safety and feasibility in reduced-intensity stem cell transplant for advanced hematologic malignancies. • A retrospective analysis indicates improved overall survival with Orca-T compared to post-transplant cyclophosphamide in high-risk patients. • Phase 1b trial data reveals low rates of graft-vs-host disease and infections, alongside high survival rates with Orca-T treatment. • Ongoing phase 3 Precision-T trial is further evaluating Orca-T versus standard allo-HCT in advanced hematologic malignancies.

Tandem vs. Single Autologous Stem Cell Transplant in Multiple Myeloma: A Comprehensive Analysis

6 months ago

• A recent study in *Nature* investigates the efficacy of single versus tandem autologous stem cell transplantation (ASCT) in newly diagnosed multiple myeloma. • The analysis suggests that tandem ASCT may offer a survival advantage in specific high-risk patient subgroups. • The study highlights the importance of risk stratification in determining the optimal treatment strategy for multiple myeloma patients. • Further research is needed to refine patient selection criteria and optimize the use of tandem ASCT in the context of modern therapies.

Tacrolimus Shows Superiority in Preventing Severe Acute GVHD After Haploidentical Stem Cell Transplant

6 months ago

• A recent study found that tacrolimus is more effective than cyclosporine A in preventing severe acute graft-versus-host disease (GVHD) in haploidentical hematopoietic cell transplantation. • The research, involving 2427 patients with acute myeloid leukemia, showed no significant differences in overall survival or relapse rates between the two drugs. • Tacrolimus was associated with a lower incidence of severe grade 3-4 acute GVHD compared to cyclosporine A in haploidentical transplants, but not in unrelated donor transplants. • The findings suggest tacrolimus may be the preferred option with mycophenolate mofetil for AML patients in first complete remission undergoing haploidentical HCT.

Immix Biopharma's NXC-201 CAR-T Therapy Shows Promise in AL Amyloidosis and Multiple Myeloma

2 years ago

• Immix Biopharma's NXC-201, a BCMA-targeted CAR-T cell therapy, demonstrated a 100% overall response rate in relapsed/refractory AL amyloidosis patients in Phase 1/2a trials. • The FDA has cleared the IND application for NXC-201, enabling U.S. patient dosing and expanding studies of the therapy in relapsed/refractory AL Amyloidosis. • NXC-201 has also shown a 95% overall response rate in heavily pre-treated relapsed/refractory multiple myeloma patients with a median follow-up of 11.9 months. • A virtual KOL event will be hosted to discuss NXC-201's potential as a treatment option for relapsed/refractory AL Amyloidosis, featuring experts in the field.