What's New?

Taiho Oncology will showcase findings from five studies at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting, including two oral presentations. Highlights include data on novel therapies such as an all-oral decitabine-cedazuridine combination for AML, zipalertinib for NSCLC with EGFR exon 20 insertion mutations, and real-world studies on metastatic colorectal cancer and cholangiocarcinoma treatments.

Faron Pharmaceuticals has announced positive topline results from its Phase II BEXMAB trial in treatment-resistant higher-risk myelodysplastic syndrome (HR MDS). The combination of bexmarilimab and azacitidine showed high overall response rates and continued strong tolerability. Full data has been submitted to the 2025 ASCO Annual Meeting, and preparations for a Phase III trial are underway.

Orca Bio reports groundbreaking Phase 3 results from the Precision-T study, showing that its investigational therapy Orca-T significantly improves survival free of chronic graft-versus-host disease compared to traditional allogeneic stem cell transplants in patients with AML, ALL, MDS, and MPAL. The therapy demonstrated a 78% cGvHD-free survival rate at one year, with 94% overall survival. Findings will be presented at the 51^st EBMT Annual Meeting and support Orca Bio’s upcoming FDA submission.

A plain-language summary of the ASCERTAIN Phase 3 trial reveals that oral decitabine and cedazuridine (DEC-C) is as effective as intravenous decitabine for patients with myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemia (CMML). The study showed near-identical drug levels in the bloodstream, similar survival outcomes, and comparable side effects. With over half of transfusion-dependent patients achieving independence and the convenience of oral dosing, the findings support oral DEC-C as a practical alternative to IV treatment.

The MDS Foundation is excited to announce the transition of its MDS Message Board to a new and enhanced platform - MDS Exchange.

MDS Exchange is a safe space for patients and carers to connect with other members of the MDS community, share their story, and find disease-specific resources and support. This enhanced community provides a more user-friendly interface, new features for deeper engagement, and ensures that the MDS community can continue to connect, share, and support each other with even greater ease.

Geron’s RYTELO^® (imetelstat) has become the first telomerase inhibitor approved in both the U.S. and Europe. The EC authorization covers adult patients with lower-risk myelodysplastic syndromes (LR-MDS) and transfusion-dependent anemia, offering a new treatment option for those who are ineligible for or have not responded to ESA therapy.

Vor Bio (Nasdaq: VOR), a clinical-stage cell and genome engineering company, today presented novel research evaluating the patient experience and barriers to enrollment and participation in cell and gene therapy (CGT) trials. The data, which was presented at the TANDEM Meetings of ASTCT and CIBMTR, demonstrated the need to improve the process and experience of patients considering and enrolling in CGT trials.

Orca Bio to present efficacy and safety data with Orca-T compared to conventional hematopoietic stem cell transplant (alloHSCT) at the 51st Annual Meeting of the EBMT

MENLO PARK, CA, February 10, 2025 – Orca Bio, a late-stage biotechnology company committed to transforming the lives of patients through high-precision cell therapy, today announced it will present positive results from the pivotal Phase 3 Precision-T study (NCT05316701) of its investigational allogeneic T-cell immunotherapy, Orca-T, in people with hematologic malignancies at the 51st Annual Meeting of the EBMT held March 30-April 2, 2025 in Florence, Italy.