NERVIANO, IT and BOSTON, Mass, 9 January, 2024 – Nerviano Medical Sciences S.r.l. (NMS), a part of NMS Group S.p.A. (NMS Group) and Nerviano Medical Sciences, Inc. (NMS-US), a wholly owned subsidiary of NMS Group, focused on the discovery and development of oncology drugs and the largest oncological R&D company in Italy, announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for its next-generation FLT3 inhibitor NMS-03592088 for the treatment of Acute Myeloid Leukemia (AML).
NMS-03592088 is a potent inhibitor of FLT3, KIT and CSF1R that is being assessed as new therapeutic option for AML patients who are relapsed or refractory after treatment with prior standard of care drugs including prior FLT3 inhibitors. Preclinical characterization demonstrated that NMS-03592088 has superior features with respect to approved FLT3 inhibitor drugs supporting its positioning as next generation FLT3 inhibitor. Notably, in addition to the higher biochemical and cellular potency and superior in vivo efficacy, NMS-03592088 showed potent activity in the presence of the gatekeeper resistance mutation F691L, reported as cause of relapse after treatment with first generation FLT3 inhibitors opening the potential for treatment of patients who failed prior FLT3 inhibitor treatment.
The clinical efficacy of NMS-03592088 is being evaluated as monotherapy in a Phase I/II study in Europe and the US. Preliminary evidence of activity was observed during Phase I dose escalation with efficacy demonstrated in patients who had failed prior standard of care including patients who failed prior FLT3 treatments (LINK: AACR2023). The trial is currently enrolling patients in Phase II with different FLT3 positive AML settings explored (NCT03922100).
“The orphan drug designation for NMS-03592088 is a significant achievement for NMS. It underscores the potential of NMS-03592088 and our dedication to making a difference in the lives of patients with FLT3 AML. We are proud to be at the forefront of innovation and remain steadfast in our mission to address unmet medical needs” said Lisa Mahnke, MD, PhD, CMO of NMS | CEO and Managing Director of NMS-US.
“Receiving orphan drug designation for NMS-03592088 is a critical milestone in our development journey. This recognition not only highlights the scientific merit of our approach but also reinforces our commitment to bringing novel therapies to those who need them most. We look forward to advancing NMS-03592088 through further clinical development and regulatory milestones” said Elena Ardini, MSc, Asset Leader of NMS.
The FDA’s Office of Orphan Products Development grants ODD status to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States. ODD provides benefits to drug developers designed to support the development of drugs and biologics for small patient populations with unmet medical needs. These benefits include potential for market exclusivity for seven years upon FDA approval eligibility for tax credits for qualified clinical trials, and waiver of Prescription Drug User Fee Act Application fee.
Link PR: 20240109_FLT3 Project_ODD_PR