What's New?

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. 

Rigel Pharmaceuticals (Nasdaq: RIGL) announced that the FDA has granted Orphan Drug designation to its investigational therapy R289 for the treatment of myelodysplastic syndromes (MDS). R289, a dual inhibitor of IRAK1 and IRAK4, is currently under investigation in a Phase 1b study assessing its safety, tolerability, and early effectiveness in patients with lower-risk MDS who have not responded to prior treatments. The Orphan Drug designation underscores the need for new therapies in rare diseases like MDS and provides Rigel with potential benefits such as tax credits, reduced FDA fees, and possible market exclusivity once approved. R289 had previously received Fast Track designation from the FDA for treating transfusion-dependent lower-risk MDS.

Syndax Pharmaceuticals (Nasdaq: SNDX) announced that the FDA has approved Revuforj^® (revumenib) for treating relapsed or refractory acute leukemia with KMT2A translocation in adults and pediatric patients aged one and older. Approved based on positive results from the AUGMENT-101 trial, Revuforj is the first and only menin inhibitor for this indication. Syndax plans to launch the drug this month and will host a conference call today at 6:00 p.m. ET to discuss the approval.

Geron Corporation (Nasdaq: GERN) announced that the European Medicines Agency’s CHMP has recommended approval of RYTELO (imetelstat) for adults with transfusion-dependent anemia from low to intermediate-risk myelodysplastic syndromes without del 5q. The European Commission is expected to finalize the decision in the coming months.

Taiho Oncology, Inc. presented findings from two studies at the 66th American Society of Hematology (ASH) Annual Meeting, focusing on oral therapies for myelodysplastic syndromes (MDS) and MDS/myeloproliferative neoplasm (MPN). The first study, a Phase 1 trial, evaluated ASTX030—a combination of azacitidine and cedazuridine—and established a recommended dose for Phase 2 trials, showing pharmacokinetic profiles comparable to subcutaneous azacitidine. The second study analyzed real-world data comparing INQOVI® (decitabine and cedazuridine) tablets with intravenous or subcutaneous hypomethylating agents, providing insights into clinical outcomes and treatment patterns. These studies underscore Taiho Oncology's commitment to developing oral hypomethylating agents that may reduce treatment burdens for patients with MDS and related conditions.