What's New?

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.

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.

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.

Karyopharm Therapeutics Inc. has initiated the Phase 2 expansion of a study investigating eltanexor, a novel oral compound, for the treatment of hypomethylating agent (HMA) refractory myelodysplastic syndrome (MDS). The Phase 2 expansion aims to evaluate eltanexor monotherapy's efficacy, with the primary endpoint being overall response rate (ORR). The decision to expand the study follows promising results from the Phase 1 portion, where eltanexor demonstrated a 53% ORR and a median overall survival of 9.9 months in patients with high-risk, relapsed MDS refractory to HMAs. The study includes an additional 83 patients, with the first patient recently dosed. Eltanexor functions by inhibiting the nuclear export protein XPO1, leading to the accumulation of tumor suppressor proteins and selective induction of apoptosis in cancer cells. The drug has shown promising results in preclinical models, suggesting its potential as a cancer therapy. However, eltanexor is still investigational and has not been approved by regulatory agencies.

Geron Corporation updated progress on its IMerge Phase 3 trial for lower-risk myelodysplastic syndromes (MDS) and reported second-quarter financial results. With over 90% enrollment achieved, the trial is expected to be fully enrolled by Q4 2021, with top-line results anticipated by Q1 2023, three months earlier than planned due to an extended follow-up period prompted by the COVID-19 pandemic. Geron plans an investor day in November 2021 to discuss imetelstat's potential, expansion plans, and a discovery program for second-generation telomerase inhibitors. Financially, the company reported a net loss of $29.6 million for Q2, with $239.1 million in cash and marketable securities as of June 30, 2021, expected to fund operations through Q1 2023.

AbbVie's VENCLEXTA® in combination with azacitidine received Breakthrough Therapy Designation (BTD) from the FDA for treating adult patients with previously untreated intermediate- to very high-risk myelodysplastic syndromes (MDS) based on IPSS-R. MDS, characterized by insufficient healthy blood cell production, affects around 10,000 US patients annually, with 30% progressing to acute myeloid leukemia (AML). This marks the sixth BTD granted to venetoclax, reflecting AbbVie's commitment to addressing challenging myeloid malignancies. The designation, supported by Phase 1b M15-531 study data, accelerates medication development for serious conditions. Venetoclax, jointly developed by AbbVie and Roche, targets the B-cell lymphoma-2 (BCL-2) protein, potentially restoring apoptosis in certain blood cancers.

For over 30 years, umbilical cord blood (UCB) has been a crucial source of hematopoietic stem cells for transplantation, particularly for non-white patients who are underrepresented in adult donor registries. However, UCB transplants have higher early treatment-related morbidity and mortality due to delayed hematopoietic recovery. Advances such as dual UCB grafts and better pre-transplant conditioning have improved outcomes, but delays in recovery persist. Early studies show that ex vivo expansion of UCB stem cells can accelerate neutrophil recovery. Omidubicel, a product derived from a single UCB unit and expanded ex vivo with nicotinamide, enhances hematopoietic recovery. A trial comparing omidubicel to standard UCB transplants found that omidubicel significantly reduced the median time to neutrophil recovery from 22 to 12 days, improved platelet engraftment, and decreased infection rates, with no significant increase in graft-versus-host disease.