The FDA granted orphan drug designation to CLN-049 for relapsed or refractory acute myeloid leukemia based on data from early-phase studies.
The FDA has granted an orphan drug designation to CLN-049, an investigational bispecific FLT3xCD3 T-cell engager, as a potential therapeutic option for patients with relapsed/refractory acute myeloid leukemia (AML).1
CLN-049 is currently being evaluated in 2 early-phase studies in AML and myelodysplastic syndrome (MDS), including relapsed/refractory AML, including a 3-part, phase 1 trial (NCT05143996) examining CLN-049 in relapsed or refractory AML or myelodysplastic syndromes (MDS).2 The other is a phase 1 trial (EUCT2023-506572-27-00) evaluating CLN-049 in AML with minimal residual disease (MRD).3
“FDA orphan drug designation for CLN-049 emphasizes both the urgent need for new therapies for people living with relapsed or refractory AML—including patients with TP53-mutated AML who currently face a particularly poor prognosis—and the potential of this FLT3-directed T-cell engager to expand treatment options across the broadest population of [patients with] AML,” Jeffrey Jones, MD, MBA, chief medical officer of Cullinan Therapeutics, stated in a news release.1 “Coupled with promising results from our ongoing phase 1 program, this designation by the FDA reinforces a shared goal to rapidly advance novel therapies for patients living with AML.”
The open-label, multicenter, first-in-human, trial (NCT05143996) is enrolling patients with relapsed or refractory AML or MDS who are at least 18 years of age.2 Patients also need to have a white blood cell count less than 20,000/uL at the time of their first dose, an ECOG performance status of 2 or less, a creatinine clearance of 60 mL/min or less per Cockcroft-Gault formula, and a total bilirubin of no more than 1.5 times the upper limit of normal.
If patients have acute promyelocytic leukemia, active central nervous system involvement, isolated extramedullary disease, or have undergone prior organ allografts or allogeneic hematopoietic transplant within 6 months prior to their first dose, they are not included in the trial.
Part A of the trial is utilizing a single ascending dose design while evaluating intravenous (IV) CLN-049. Furthermore, part B is utilizing a multiple ascending dose design while also evaluating IV CLN-049. Finally, Part C is a multiple ascending dose design, except this portion will evaluate subcutaneous injections of CLN-049.
End points for the study include the number of treatment-related adverse effects, the maximum concentration of CLN-049, safety, pharmacokinetics, pharmacodynamics, and preliminary efficacy.
The other phase 1 study (EUCT2023-506572-27-00) is evaluating CLN-049 in patients with AML with MRD who are between 18 and 64 years of age in Spain or Germany.3 The trial currently has the estimated end date of August 14, 2026.
CLN-049 previously received
