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Kymera Therapeutics Granted FDA Orphan Drug Designation for KT-253: A Novel and Selective MDM2 Degrader in the Treatment of Acute Myeloid Leukemia

Friday, June 23, 2023

Kymera Therapeutics, a clinical-stage biopharmaceutical company specializing in targeted protein degradation, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to KT-253 for the treatment of Acute Myeloid Leukemia (AML).

KT-253 is a novel and potent degrader that specifically targets MDM2, a critical regulator of the tumor suppressor protein p53. In many cancers, p53 remains functional, making it an attractive target for therapeutic intervention. However, conventional small molecule inhibitors that stabilize p53 often face limitations due to a feedback loop that increases MDM2 levels, reducing their effectiveness. Preclinical studies have shown that KT-253 can overcome this feedback loop and induce rapid cancer cell death, even with short exposure periods. Given the role of MDM2 in AML and other cancers, KT-253 is being explored as a potential treatment option.

Nello Mainolfi, the Founder, President, and CEO of Kymera Therapeutics, expressed excitement about the orphan drug designation and its potential to address the challenges associated with targeting MDM2. Mainolfi emphasized the company's dedication to advancing the development of KT-253 in AML and exploring its therapeutic potential in other hematological and solid tumors.

A Phase 1 study of KT-253 was initiated in March 2023 to assess its safety, tolerability, pharmacokinetics/pharmacodynamics, and clinical activity in patients with relapsed or refractory high-grade myeloid malignancies, including AML, acute lymphocytic leukemia (ALL), lymphoma, and solid tumors. The study aims to determine the recommended Phase 2 dose of KT-253 and consists of two arms with escalating doses of the drug.

Orphan drug designation by the FDA is granted to drugs intended for the treatment, diagnosis, or prevention of rare diseases affecting fewer than 200,000 individuals in the United States. This designation provides various development incentives, including tax credits, fee exemptions, and seven years of marketing exclusivity upon FDA approval.

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