Kymera Therapeutics' KT-253 Granted FDA Orphan Drug Designation Novel and Selective MDM2 Degrader for AML Treatment
Kymera Therapeutics, a clinical-stage biopharmaceutical company specializing in targeted protein degradation, has announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to their drug candidate, KT-253, for the treatment of Acute Myeloid Leukemia (AML). KT-253 is a highly potent and selective MDM2 degrader that specifically targets MDM2, a crucial regulator of the tumor suppressor protein p53.
Unlike small molecule inhibitors, which can increase MDM2 protein levels and limit their effectiveness, KT-253 has shown promising results in preclinical studies by overcoming the feedback loop associated with MDM2 and inducing cancer cell death rapidly, even with short exposures. Given the implication of MDM2 overexpression and amplification in AML, KT-253 is being explored for its potential in treating this type of cancer, as well as other liquid and solid tumors.
To evaluate the safety, tolerability, pharmacokinetics/pharmacodynamics, and clinical activity of KT-253, a Phase 1 study was initiated in March 2023. The study aims to enroll patients with relapsed or refractory high-grade myeloid malignancies, including AML, acute lymphocytic leukemia (ALL), lymphoma, and solid tumors. The study will administer intravenous doses of KT-253 once every 3 weeks, with two arms consisting of ascending doses of the drug. The first arm will focus on patients with lymphomas and advanced solid tumors, while the second arm will include patients with high-grade myeloid malignancies and ALL.
Orphan drug designation is granted by the FDA to drugs intended for the treatment, diagnosis, or prevention of rare diseases affecting fewer than 200,000 people in the United States. This designation provides certain benefits and incentives to the drug sponsor, including tax credits for qualified clinical testing, exemption from prescription drug user fees, and seven-year marketing exclusivity upon FDA approval.
