MYLEOFIBROSIS (MF)

The US FDA has approved GSK's Ojjaara for the treatment of intermediate or highrisk myelofibrosis, a blood cancer affecting around 25,000 patients in the US.Ojjaara is the first medicine approved for both newly diagnosed and previouslytreated myelofibrosis patients with anemia. The approval is supported by datafrom the pivotal MOMENTUM and SIMPLIFY-1 Phase III trial

CHRONIC MYELOGENOUS LEUKEMIA (CML)

Bosulif (bosutinib) is now indicated for pediatricpatients aged 1+ with newly diagnosed (ND) orresistant or intolerant (R/I) chronic phase (CP) Ph+CML. Efficacy was assessed in the BCHILD trial, withfavorable responses in both ND and R/I patients. Forpediatric patients with ND CP Ph+ CML, the major(MCyR) and complete (CCyR) cytogenetic responseswere 76.2% (95% CI: 52.8, 91.8) and 71.4% (95% CI:47.8, 88.7), respectively. Common side effects includeddiarrhea, abdominal pain, and rash. For pediatricpatients with R/I CP Ph+ CML, the MCyR and CCyRresponses were 82.1% (95% CI: 63.1, 93.9) and 78.6%(95% CI: 59, 91.7), respectively.

AML AND MDS

Updated data from the Phase I/II BEXMAB study ofbexmarilimab in combination with standard of care (SoC)in R/R AML HMA-refractory MDS are consistent with thehigh objective response rate (ORR) observed in theprevious results. 8 of the 11 patients are CompleteResponders (CR) or CR with incomplete blood recovery(CRi). The ORR was 80% in the prior HMA-failure MDSgroup, and the combined ORR across all 22 patients was50%. Bexmarilimab continues to be well-tolerated withno dose-limiting toxicities observed.

HEMATOLOGY

Researchers at the University ofPennsylvania Perelman Schoolof Medicine have developed anew approach to CAR T-celltherapy using CRISPR-basedgene editing. They engineeredboth T cells and hematopoieticstem cells to target a protein called CD45, which ispresent on most blood cells, to create a universal CAR Tcell therapy that can eliminate various blood cancerswhile sparing healthy cells. In mouse models, thetreatment effectively eliminated tumors and did notcause serious side effects. This approach couldtheoretically treat all forms and types of blood cancer.and may soon enter clinical trials

AML

Gilead Sciences has halted its ENHANCE-2 study inAML with TP53 mutations after an ad-hoc analysis andreview by an independent data monitoring committeeindicated that magrolimab is unlikely to provide asurvival benefit compared to the standard of care.
Gilead is now working with study investigators todetermine appropriate next steps for patients involvedin the trial. The news follows a recent partial clinicalhold placed on the initiation of new patients in USstudies evaluating magrolimab to treat AML and recentdiscontinuation of the Phase 3 ENHANCE study ofmagrolimab in higher-risk myelodysplastic syndromes.

MULTIPLE MYELOMA

In a subgroup analysis of the Phase III CARTITUDE-4trial presented at the 20th International MyelomaSociety (IMS) Annual Meeting, patients with multiplemyeloma and poor prognostic features, includinghigh-risk cytogenetics, soft-tissue plasmacytoma,ISS stage III disease, and triple-class exposure,experienced significant progression-free survival (PFS) benefit with single-infusionciltacabtagene autoleucel (cilta-cel). The 12-month PFS rates were favorable inpatients with standard or high cytogenetic risk, soft-tissue plasmacytoma, or tripleclass refractoriness. The analysis affirmed the effectiveness of cilta-cel in a widerange of clinically relevant multiple myeloma subgroups, even those with poorprognostic factors.