What's New?

A new Molecular International Prognostic Scoring System (IPSS-M) for Myelodysplastic Syndromes (MDS) was developed, enhancing risk stratification and treatment planning based on genetic profiles. Developed through international collaboration, IPSS-M offers personalized risk scores and a web-based calculator for clinical use.

Servier Pharmaceuticals has announced the FDA approval of TIBSOVO® (ivosidenib tablets) in combination with azacitidine for treating newly diagnosed IDH1-mutated acute myeloid leukemia (AML) in adults aged 75 years or older, or those with comorbidities preventing intensive chemotherapy use. This marks the first approval of a therapy targeting cancer metabolism with azacitidine for this patient group. The approval is based on positive data from the Phase 3 AGILE trial, showing significant improvements in event-free survival and overall survival compared to standard treatments. TIBSOVO plus azacitidine demonstrated a threefold improvement in median overall survival (24 months) compared to placebo plus azacitidine (7.9 months). This approval expands the use of TIBSOVO, which is already approved for other IDH1-mutated cancer types. TIBSOVO in combination with azacitidine has shown a favorable safety profile in newly diagnosed AML patients.

Genomic Testing Cooperative (GTC) introduces a program offering free comprehensive molecular profiling to underserved cancer patients, aiming to bridge healthcare disparities. GTC commits to donating 5% of its testing volume and encourages further contributions. Physicians nominate eligible patients, fostering equitable access to precision medicine. GTC urges pharmaceutical involvement to improve access to targeted therapies and clinical trials.

Syros Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) granted orphan drug designation (ODD) to tamibarotene for the treatment of myelodysplastic syndrome (MDS). Tamibarotene, an oral selective retinoic acid receptor alpha (RARα) agonist, is being evaluated in combination with azacitidine in the SELECT-MDS-1 Phase 3 trial for RARA-positive patients with newly diagnosed higher-risk MDS. The designation provides benefits including market exclusivity and tax credits for qualified clinical trials. The ongoing trial is expected to provide data in late 2023 or early 2024, with a potential new drug application filing in 2024. Syros is also evaluating tamibarotene for RARA-positive patients with newly diagnosed unfit acute myeloid leukemia.

Karyopharm Therapeutics has received orphan drug designation from the FDA for eltanexor, a novel oral compound, for the treatment of myelodysplastic syndromes (MDS). The designation acknowledges eltanexor's potential to improve clinical outcomes for patients with HMA-refractory MDS. Positive data from an ongoing Phase 1/2 study showed promising results, with a 53% overall response rate and a median overall survival of 9.9 months. Orphan drug designation qualifies Karyopharm for various incentives, including market exclusivity and tax credits. Eltanexor functions by inhibiting the nuclear export protein XPO1, leading to the accumulation of tumor suppressor proteins in cancer cells. Karyopharm is committed to advancing its clinical trials and bringing this new treatment option to patients.

Curis, Inc. announced positive clinical data for CA-4948, an IRAK-4 inhibitor, in relapsed or refractory AML and MDS patients, showing promising efficacy and a favorable safety profile. The study also revealed encouraging initial data for CI-8993, a monoclonal antibody targeting VISTA in solid tumors, indicating a promising safety profile and potential immune activation. Curis plans to advance both programs, with potential regulatory discussions in 2022 and additional data expected later in the year, marking significant progress in cancer therapeutics.

The global pivotal QuANTUM-First phase 3 trial has delivered positive topline results, marking a significant advancement in the treatment of newly diagnosed FLT3-ITD positive acute myeloid leukemia (AML). The trial demonstrated that adding quizartinib, a highly potent and selective FLT3 inhibitor, to standard induction and consolidation chemotherapy, followed by continued single-agent quizartinib, significantly improved overall survival (OS) compared to standard treatment alone. AML, one of the most common forms of leukemia in adults, has a low five-year survival rate, particularly among patients with FLT3-ITD positive AML, indicating a critical unmet medical need. The phase 3 trial's success represents a promising step forward in improving survival outcomes for this patient population. Daiichi Sankyo plans to share the QuANTUM-First trial data with regulatory authorities globally, aiming to bring this potentially life-saving treatment to patients in need.

Pharmacosmos Group, a leader in developing treatments for iron deficiency and anemia, has acquired AbFero Pharmaceuticals, Inc., a Boston-based clinical stage company specializing in iron overload diseases. Through a subsidiary, Pharmacosmos will acquire all assets of AbFero, including their lead compound SP-420, aimed at advancing iron chelation therapies. This acquisition aligns with Pharmacosmos's expertise and global presence in iron metabolism. SP-420, an orally active iron chelator, offers potential as a safer alternative with better efficacy for patients with conditions like transfusional iron overload. This strategic move underscores the commitment to addressing unmet medical needs in hematological diseases requiring chronic blood transfusions.