What's New?

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.

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.

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.

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.

The study by Memorial Sloan Kettering researchers confirms that having two mutated TP53 gene copies, versus one, worsens outcomes in myelodysplastic syndrome and acute myeloid leukemia. Global analysis of 4,444 patients identified TP53 mutation status as crucial for prognosis, emphasizing the need for TP53 assessment at diagnosis.

Astex Pharmaceuticals, Taiho Oncology, and Otsuka Pharmaceutical have announced FDA and Health Canada approval of INQOVI® tablets, the first orally administered hypomethylating agent for intermediate and high-risk MDS and CMML. INQOVI combines decitabine with cedazuridine to prevent degradation, enabling oral dosing. Approval is based on the ASCERTAIN phase 3 study and supporting trials, offering patients a convenient alternative to IV infusions with potential benefits. This milestone underscores the commitment to advancing cancer treatment.

A recent study conducted at Stanford University Medical Center and published in Blood Advances highlights the efficacy of Notable's drug sensitivity screening platform in identifying potentially useful drugs for patients with myelodysplastic syndrome (MDS) refractory to standard therapies. The study, involving 21 MDS patients, demonstrated a median turnaround time of 15 days for returning drug sensitivity data to the Tumor Board, meeting the primary endpoint of the study. Notable's platform showed a positive predictive value of 92%, negative predictive value of 82%, and overall accuracy of 85% in predicting clinical responses. The study also identified correlations between genotype and drug sensitivity patterns, providing valuable insights for personalized treatment recommendations. Notable and Stanford are continuing their collaboration to validate the initial data set and further advance precision medicine in oncology.