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The MDS Foundation
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Yardville, NJ 08620
A global non-profit advocacy organization, supporting patients, families and healthcare providers in the fields of MDS and its related diseases for over 30 years
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Latest News
Explore the forefront of MDS research, treatments, and community happenings to stay informed on the ever-evolving landscape of MDS care.
February 2, 2021
Medexus Pharmaceuticals and medac GmbH enter into a License Agreement for First-in-Class Conditioning Agent for Hematopoietic Stem Cell Transplantation, Treosulfan, in the United States
Medexus Pharmaceuticals has secured exclusive U.S. commercialization rights to treosulfan, an orphan-designated conditioning agent for allogeneic hematopoietic stem cell transplantation (allo-HSCT), through a deal with medac GmbH. Treosulfan, expected to reduce toxicity and improve survival outcomes, has an FDA review date set for August 2021. A Phase III study showed it to be non-inferior to busulfan, the current standard. Medexus plans to launch treosulfan soon after FDA approval, leveraging its U.S. infrastructure. The agreement includes an upfront payment of $5 million, milestone payments, and royalties on net sales.
December 7, 2020
Geron Reports Ten Imetelstat Presentations at American Society of Hematology Annual Meeting
Geron Corporation presented ten studies on its telomerase inhibitor, imetelstat, at the 62nd American Society of Hematology (ASH) Annual Meeting. Highlights include data from Phase 2 IMerge and IMbark trials showing clinical benefits, such as durable transfusion independence in myelodysplastic syndromes (MDS) and improved survival in myelofibrosis (MF). The studies suggest imetelstat’s disease-modifying activity through depletion of abnormal clones and disease mutations. Biomarker data indicate on-target activity by reducing telomerase activity and hTERT expression. These findings support ongoing and upcoming Phase 3 trials. All abstracts submitted were accepted for presentation.
December 5, 2020
Syros Presents New Data from Phase 2 Clinical Trial of SY-1425 and Announces Plans to Initiate Registration-Enabling Trial in MDS and Randomized Phase 2 Trial in AML
Syros Pharmaceuticals announced promising data from its Phase 2 clinical trial of SY-1425, a selective retinoic acid receptor alpha (RARα) agonist, combined with azacitidine for acute myeloid leukemia (AML) patients. Highlights include a 61% composite complete response rate and 18 months median overall survival among responders in RARA-positive newly diagnosed unfit AML patients. The company plans a Phase 3 trial in higher-risk myelodysplastic syndrome (MDS) patients and a Phase 2 trial with a triplet regimen in AML. These trials aim to address unmet needs and potentially establish SY-1425 as a foundational therapy for RARA-positive patients.
December 5, 2020
Improving Understanding of MDS Using an Animated Patient’s Guide to Benefit Patient Health Outcomes
The MDS Foundation introduced an Animated Patient’s Guide to MDS (APG) in 2018 to enhance understanding of Myelodysplastic Syndromes (MDS). Over 24 months, 151,231 views were recorded, with most users intending to implement newfound knowledge in self-management and treatment discussions with their doctors, indicating potential for improved patient outcomes.
October 28, 2020
Geron Announces Publication of IMerge Phase 2 Data in Journal of Clinical Oncology
Geron Corporation announced the publication of data from the IMerge Phase 2 clinical trial in the Journal of Clinical Oncology. The study demonstrated that imetelstat, a telomerase inhibitor, achieved meaningful and durable transfusion independence in patients with lower-risk myelodysplastic syndromes (MDS) who were refractory to or ineligible for erythropoiesis-stimulating agents (ESAs). The trial's results showed a median transfusion independence duration of 21 months and suggested potential disease-modifying activity by reducing malignant clones. No new safety signals were identified, with the most common adverse events being reversible cytopenias. These findings support the ongoing IMerge Phase 3 clinical trial, which is currently enrolling patients globally and aims to provide top-line results by the second half of 2022.
October 16, 2020
VENCLEXTA® (venetoclax) Receives FDA Full Approval for Acute Myeloid Leukemia (AML)
The FDA has granted full approval for VENCLEXTA® (venetoclax) for the treatment of newly diagnosed acute myeloid leukemia (AML) in patients 75 years or older, or those unable to undergo intensive chemotherapy. This decision is backed by data from multiple clinical trials, including the Phase 3 VIALE-A and VIALE-C trials. The VIALE-A trial demonstrated that the combination of VENCLEXTA and azacitidine significantly increased complete remission rates and overall survival compared to azacitidine alone. AML, known for its aggressiveness and low survival rates, now has a new treatment option recommended by the National Comprehensive Cancer Network (NCCN) for patients ineligible for intensive chemotherapy. AbbVie, in collaboration with Roche, is responsible for the development and commercialization of VENCLEXTA.
October 6, 2020
Gamida Cell Announces Positive Topline Data on Secondary Endpoints from Phase 3 Clinical Study of Omidubicel in Patients with Hematologic Malignancies
Gamida Cell Ltd. announced that its Phase 3 clinical study of omidubicel, an investigational cell therapy for patients needing bone marrow transplants, met its secondary endpoints. These endpoints included platelet engraftment by day 42, reduction in bacterial and fungal infections, and fewer days hospitalized within the first 100 days post-transplant. Omidubicel, which has Breakthrough Therapy Designation from the FDA, demonstrated significant improvements over standard umbilical cord blood transplants. The company plans to submit a Biologics License Application (BLA) in the fourth quarter of 2020, aiming to establish omidubicel as a transformative treatment option for patients without matched donors.
September 1, 2020
U.S. Food and Drug Administration Approves Onureg® (azacitidine tablets), a New Oral Therapy, as Continued Treatment for Adults in First Remission with Acute Myeloid Leukemia
The U.S. FDA has approved Onureg® (azacitidine tablets) for adults with acute myeloid leukemia (AML) in their first remission following chemotherapy who cannot undergo intensive curative therapy. This approval is based on the Phase 3 QUAZAR® AML-001 study, which showed that Onureg improved overall survival by nearly 10 months compared to placebo (24.7 months vs. 14.8 months). Onureg is an oral hypomethylating agent and is now the first FDA-approved continued therapy for AML patients in remission. The study noted significant adverse reactions, including myelosuppression and risks during pregnancy. The approval reflects Bristol Myers Squibb’s commitment to advancing cancer treatment.