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Syros Pharmaceuticals announced initial data from their SELECT-AML-1 Phase 2 trial showing a 100% complete remission (CR) or complete remission with incomplete hematologic recovery (CRi) rate in newly diagnosed unfit AML patients with RARA gene overexpression treated with tamibarotene, venetoclax, and azacitidine, compared to 70% with venetoclax and azacitidine alone. The triplet regimen demonstrated favorable tolerability, with no new safety signals. Additional data from the trial is expected in 2024​​.

Servier Pharmaceuticals announced that the FDA has approved TIBSOVO® (ivosidenib tablets) for the treatment of IDH1-mutated relapsed or refractory (R/R) myelodysplastic syndromes (MDS). TIBSOVO is the first targeted therapy approved for this subset of MDS patients. This approval is based on a Phase 1 study showing a 38.9% complete remission rate and an 83.3% overall response rate. This marks the fifth indication for TIBSOVO, reinforcing Servier’s leadership in mutant IDH inhibition.

The MDS Foundation partners with SparkCures to enhance clinical trial navigation for myelodysplastic syndromes (MDS) patients. SparkCures will develop a custom platform for personalized trial screening, aiming to simplify the complex landscape of trial options. The collaboration aims to empower patients, caregivers, and healthcare providers, with plans for international expansion in 2024.

Geron Corporation announced that the FDA has accepted its New Drug Application (NDA) for imetelstat, a telomerase inhibitor, for treating transfusion-dependent anemia in patients with lower-risk myelodysplastic syndromes (MDS). The acceptance is based on positive results from the IMerge Phase 3 trial, which demonstrated significant efficacy in achieving red blood cell transfusion independence. Geron expects to disclose the timeline for the FDA review soon and plans to submit a Marketing Authorization Application in the EU by Q4 2023. This milestone highlights the potential of imetelstat to become a new standard of care in lower-risk MDS .

In the global phase III COMMANDS trial, luspatercept significantly improved outcomes for patients with low-risk, transfusion-dependent myelodysplastic syndrome (MDS). Compared to epoetin alfa, an erythropoiesis-stimulating agent, luspatercept nearly doubled the likelihood of achieving transfusion independence and increasing hemoglobin levels. The interim analysis showed 58.5% of luspatercept-treated patients met the primary endpoint, versus 31.2% with epoetin alfa (P < .0001). These results suggest a paradigm shift in treating lower-risk MDS, positioning luspatercept as a new first-line therapy.

The article presents updated findings from the QUAZAR AML-001 trial, investigating the long-term survival outcomes of patients with acute myeloid leukemia (AML) in first remission after chemotherapy. The study focused on the efficacy of oral azacitidine (Oral-AZA) versus placebo in older patients ineligible for stem cell transplantation. Results revealed that Oral-AZA significantly prolonged overall survival and relapse-free survival compared to placebo. Patients receiving Oral-AZA demonstrated sustained survival benefits over five years, with higher survival rates at 3 and 5 years compared to placebo. Additionally, the study identified clinical and biological factors associated with long-term survival, highlighting the potential of Oral-AZA maintenance therapy for patients in remission after chemotherapy.

Geron Corporation has announced positive top-line results from its IMerge Phase 3 clinical trial of imetelstat, a telomerase inhibitor, for lower-risk myelodysplastic syndromes (MDS). The trial met its primary endpoint of 8-week transfusion independence (TI) and a key secondary endpoint of 24-week TI, both with highly statistically significant improvements. The median duration of TI approached one year for 8-week responders and 1.5 years for 24-week responders. Efficacy results were significant across key MDS subtypes, including varying transfusion burdens and IPSS risk categories. Safety results were consistent with previous trials, showing no new safety signals. Based on these results, Geron plans to submit regulatory applications in the U.S. and EU in 2023 and prepare for a potential U.S. commercial launch in 2024.

The article discusses the presentation of overall survival data for oral decitabine and cedazuridine (marketed as INQOVI®) in patients with myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML) harboring TP53 mutations. The Phase 3 ASCERTAIN trial revealed that patients with biallelic TP53 mutations achieved a median overall survival of 13 months, while the median overall survival in the overall study population was 32 months. These results suggest the potential utility of this oral hypomethylating agent in patients with MDS and CMML harboring TP53 mutations, which are typically associated with poor outcomes. The study underscores the importance of identifying patients who may benefit from this treatment option and highlights the ongoing efforts to address the unmet needs of these patient populations.