What's New?

Syros Pharmaceuticals reported financial results for Q1 2024, highlighting key updates on its clinical programs. The SELECT-MDS-1 Phase 3 trial of tamibarotene passed an interim futility analysis, with pivotal complete response data expected by mid-Q4 2024. Additionally, tamibarotene received FDA Fast Track Designation for treating unfit AML. Financially, the company reported a net loss of $3.7 million, a decrease from Q1 2023's $23.8 million loss. Syros’ cash, cash equivalents, and marketable securities totaled $108.3 million as of March 31, 2024. Further data from ongoing trials is anticipated later in the year​.

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​​.

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.