What's New?

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.

The article discusses new interim Phase 2 data on selinexor, a first-in-class oral Selective Inhibitor of Nuclear Export (SINE) compound, in patients with myelofibrosis (MF) who were previously treated with JAK inhibition. The data, which will be presented at the American Society of Hematology (ASH) 2021 Annual Meeting, shows promising results for selinexor as a potential treatment option for MF patients who have failed treatment with ruxolitinib, the current standard of care. Notably, 33% of patients who received at least 24 weeks of selinexor treatment achieved a response, defined as ≥35% spleen volume reduction (SVR). Additionally, the study demonstrated improvements in anemia status and symptom scores in these patients. The results highlight selinexor's potential in patients with MF who have either progressed following ruxolitinib treatment or cannot tolerate JAK inhibition. The study also paved the way for a new Phase 2 study evaluating single-agent selinexor versus physician's choice in previously treated myelofibrosis patients. The article provides details about the upcoming ASH 2021 abstract presentation, along with information about other presentations at the meeting.

First Phase 3 results from the AGILE trial in patients with newly diagnosed acute myeloid leukemia with an IDH1 mutation show improved event-free survival and various secondary outcomes, including complete remission, overall survival and objective response rate, with ivosidenib tablets (TIBSOVO®) in combination with azacitidine compared to azacitidine plus placebo.