Myelodysplastic syndromes (MDS) are marked by ineffective hematopoiesis and dysplasia, which elevate the risk of progression to acute myeloid leukemia (AML). The inflammatory environment in MDS alters the bone marrow niche, facilitating the growth of cancerous cells while inhibiting the healthy formation of blood cells.
Bluesphere Bio Inc. has received FDA clearance of its IND application for BSB-1001 for patients with relapsed or refractory acute myeloid leukemia (AML), acute lymphocytic leukemia and myelodysplastic syndromes, in conjunction with allogeneic hematopoietic stem cell transplantation (alloHSCT).
Wall Street will be watching closely for such adverse effects as anemia that foiled Gilead Sciences Inc.’s CD47-binding magrolimab earlier this year, but so far Shattuck Labs Inc.’s SL-172154 looks strong in combination with azacitidine to treat front-line higher-risk myelodysplastic syndrome (HR-MDS) and TP53-mutant (TP53m) acute myeloid leukemia (AML).
After Keros Therapeutics Inc.’s first-quarter earnings report, the Wall Street spotlight turned its beam toward additional data due soon from the phase II studies with KER-050, an ActRIIA-Fc fusion protein in myelodysplastic syndrome (MDS) and myelofibrosis.
Geron Corp. executives highlighted a potential billion-dollar market opportunity for imetelstat on the back of positive phase III data for the telomerase inhibitor in patients with lower-risk myelodysplastic syndromes (MDS), news that sent shares (NASDAQ:GERN) rising 36% and could portend the first regulatory win for a scientific approach the company has pursued since the 1990s.
While Aprea Therapeutics Inc.’s data disclosed July 21 from the phase II trial with eprenetapopt – also known as APR-246, a reactivator of mutant tumor suppressor protein p53 – plus azacitidine (AZA) whetted interest in taking aim at the “guardian of the genome,” the company’s fortune took an unexpected turn when the FDA smacked a partial clinical hold on work with the duo.
As developers continue their race in the anti-CD47 space, Gilead Sciences Inc. remains high profile with magrolimab, which has reached phase III development for myelodysplastic syndromes. Meanwhile, Alx Oncology Inc., of Burlingame, Calif., is emerging with potentially the first drug targeting the CD47-SIRPa axis to treat solid tumors as well as hematologic malignancies.
Questions of durability came up regarding Curis Inc.’s latest data with CA-4948, but that didn’t stop shares from soaring to $6.55 by day’s end, an increase of $5.11, or 355%. The ride came after Lexington, Mass.-based Curis rolled out positive preliminary data from the firm’s ongoing open-label, single-arm phase I dose-escalation study with the compound.
