What's New?

Jazz Pharmaceuticals announced that the FDA approved Vyxeos® (daunorubicin and cytarabine) for treating newly diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC) in pediatric patients aged one year and older. This approval is based on safety data from two single-arm trials (AAML1421 and CPX-MA-1201) and effectiveness data from an adult study. Vyxeos is now indicated for both adults and children with these forms of AML. Jazz Pharmaceuticals emphasizes the critical need for effective pediatric AML therapies and its commitment to broadening cancer research. The studies demonstrated no age-based safety differences, and Vyxeos' approval marks an important advancement for pediatric leukemia treatment.

AbFero Pharmaceuticals, Inc., a clinical-stage company focusing on iron overload diseases, has begun a Phase 1 study of its lead iron chelator, SP-420, in patients with myelodysplastic syndromes (MDS) and myelofibrosis (MF) suffering from transfusional iron overload (TIO). This trial, conducted by Dr. Elizabeth Bowhay-Carnes at UT Health San Antonio, aims to evaluate SP-420's safety and will lay the groundwork for future efficacy studies. AbFero's CEO, Thomas X. Neenan, emphasized the importance of this trial in advancing SP-420's development. Dr. Ruben Mesa of the Mays Cancer Center highlighted the significance of addressing TIO in patients with chronic leukemia and bone marrow disorders. AbFero's technology, based on Professor Raymond Bergeron's discoveries, targets TIO and iron-related aging diseases like AMD and Parkinson's. The study will provide essential safety data in 2021.

Gamida Cell announced positive results from a Phase 3 study of omidubicel, a potential cell therapy for patients with hematologic malignancies needing a bone marrow transplant. The data, presented at the 2021 TCT Meetings, showed that omidubicel led to faster hematopoietic recovery, fewer infections, and reduced hospitalization compared to standard umbilical cord blood transplants. The study met its primary and secondary endpoints, demonstrating significant improvements in neutrophil and platelet engraftment and lower infection rates. These results will support a Biologics License Application (BLA) submission to the FDA in the second half of 2021.

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.

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.

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.

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.

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.