What's New?

Syndax Pharmaceuticals (Nasdaq: SNDX) announced that the FDA has approved Revuforj^® (revumenib) for treating relapsed or refractory acute leukemia with KMT2A translocation in adults and pediatric patients aged one and older. Approved based on positive results from the AUGMENT-101 trial, Revuforj is the first and only menin inhibitor for this indication. Syndax plans to launch the drug this month and will host a conference call today at 6:00 p.m. ET to discuss the approval.

Geron Corporation (Nasdaq: GERN) announced that the European Medicines Agency’s CHMP has recommended approval of RYTELO (imetelstat) for adults with transfusion-dependent anemia from low to intermediate-risk myelodysplastic syndromes without del 5q. The European Commission is expected to finalize the decision in the coming months.

Taiho Oncology, Inc. presented findings from two studies at the 66th American Society of Hematology (ASH) Annual Meeting, focusing on oral therapies for myelodysplastic syndromes (MDS) and MDS/myeloproliferative neoplasm (MPN). The first study, a Phase 1 trial, evaluated ASTX030—a combination of azacitidine and cedazuridine—and established a recommended dose for Phase 2 trials, showing pharmacokinetic profiles comparable to subcutaneous azacitidine. The second study analyzed real-world data comparing INQOVI® (decitabine and cedazuridine) tablets with intravenous or subcutaneous hypomethylating agents, providing insights into clinical outcomes and treatment patterns. These studies underscore Taiho Oncology's commitment to developing oral hypomethylating agents that may reduce treatment burdens for patients with MDS and related conditions.

Keros Therapeutics has entered into an exclusive global license agreement with Takeda to develop and commercialize elritercept, a treatment for anemia and thrombocytopenia in patients with myelodysplastic syndrome (MDS) and myelofibrosis (MF).

Elritercept is currently in Phase 2 clinical trials for MDS and MF, with a Phase 3 trial for transfusion-dependent anemia in MDS patients set to begin enrollment soon. The partnership underscores Keros’ expertise in TGF-ß protein signaling and positions Takeda to expand its hematologic oncology portfolio.

Syros Pharmaceuticals announced that its Phase 3 SELECT-MDS-1 trial, evaluating tamibarotene combined with azacitidine in newly diagnosed higher-risk myelodysplastic syndrome (HR-MDS) patients with RARA gene overexpression, did not achieve its primary endpoint of complete response (CR) rate. The trial's intent-to-treat analysis showed a CR rate of 23.8% in the treatment arm versus 18.8% in the control arm, a difference that was not statistically significant (p = 0.2084). The combination therapy was generally well-tolerated, with an adverse event profile consistent with previous studies. As a result of these findings, Syros plans to discontinue the study, conduct a thorough review of the data, and assess future steps.

Rigel Pharmaceuticals announced that their Phase 2 study of REZLIDHIA® (olutasidenib) in patients with mutant IDH1 acute myeloid leukemia (AML) who relapsed or were refractory to prior venetoclax-based regimens showed promising results. Olutasidenib achieved durable composite complete remission in 43.8% of patients, with a safety profile consistent with previous data. This suggests olutasidenib could be a valuable treatment for this challenging patient population​​.

Taiho Oncology announced the Phase 3 ASCERTAIN trial results, showing that oral INQOVI® (decitabine and cedazuridine) is pharmacologically equivalent to IV decitabine for treating MDS and CMML. The study reported a median overall survival of 32 months and a 62% overall response rate. Additionally, 20% of patients moved to transplantation. Safety findings were consistent with IV decitabine, with common adverse events like thrombocytopenia, neutropenia, and anemia. The oral formulation offers a convenient alternative to daily clinic visits for treatment.

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​​.