What's New?

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.

The MDS Foundation introduces the IPSS-Molecular, a groundbreaking prognostic scoring system for Myelodysplastic Syndromes (MDS). Developed through global collaboration, it integrates genetic data to offer personalized risk assessment, revolutionizing treatment strategies. The IPSS-M is set to become the international standard, aiding clinicians with a web-based calculator for tailored patient care.

Geron Corporation presented four sets of data on imetelstat, its telomerase inhibitor, at the Virtual Edition of the European Hematology Association (EHA) Annual Congress.

First, from the IMerge Phase 2 trial, they reported encouraging transfusion independence rates and potential disease-modifying effects, suggesting imetelstat's efficacy in treating lower risk myelodysplastic syndromes (MDS).

Second, data from the IMbark Phase 2 trial indicated improved overall survival with imetelstat treatment in patients with myelofibrosis (MF), along with other clinical benefits such as fibrosis improvement and symptom response.

These findings provide support for Geron's ongoing Phase 3 trials of imetelstat. In summary, imetelstat shows promise in treating hematologic malignancies, potentially altering disease progression, and improving patient outcomes.

Aprea Therapeutics announced the completion of patient enrollment for its Phase 3 clinical trial evaluating eprenetapopt combined with azacitidine for front-line treatment of TP53 mutant myelodysplastic syndromes (MDS). Topline results are expected by the end of 2020, with regulatory submissions planned for 2021 in the US and EU. The trial aims to compare the combination therapy to azacitidine alone, involving 154 patients with a primary endpoint of complete remission rate. Eprenetapopt is designed to reactivate the mutant p53 tumor suppressor protein, which is commonly mutated in various cancers, including MDS.

Astex Pharmaceuticals announced that the U.S. FDA has accepted its New Drug Application (NDA) for Priority Review for the combination oral hypomethylating agent cedazuridine and decitabine (ASTX727 or oral C-DEC), intended for the treatment of myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML). This application is supported by data from the Phase 3 ASCERTAIN study, which demonstrated the equivalence of oral C-DEC to intravenous decitabine over a 5-day treatment cycle. The FDA's Priority Review designation could accelerate the approval process to six months. If approved, oral C-DEC would become the first oral hypomethylating agent for these conditions in the U.S.

Bristol-Myers Squibb and Acceleron Pharma announced the publication of results from the pivotal phase 3 MEDALIST trial in the New England Journal of Medicine. The study evaluated Reblozyl® (luspatercept-aamt) for treating anemia in adults with very low- to intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts who require red blood cell transfusions and are unresponsive to erythropoiesis-stimulating agents. The MEDALIST trial showed that Reblozyl significantly improved red blood cell transfusion independence and met key secondary endpoints, with manageable safety profiles. The U.S. FDA is reviewing Reblozyl for this MDS indication, with a decision expected by April 4, 2020.