The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Kymera Therapeutics' (NASDAQ:KYMR) product candidate KT-333 for the treatment of Cutaneous T-cell Lymphoma (CTCL).
KT-333 is being evaluated in an ongoing Phase 1 clinical trial to assess safety, tolerability and PK/PD of escalating doses in adult patients with relapsed/refractory liquid and solid tumors, including aggressive lymphomas.
Nello Mainolfi, Co-Founder, President and CEO, commented: "This second orphan drug designation reinforces the potential of KT-333 to impact the lives of a broad range of patients with hematological and solid tumors by targeting STAT3, a protein that has been considered undruggable. We have a significant opportunity to deliver an important new medicine with this first-in-class heterobifunctional degrader, and we look forward to working with the lymphoma community to rapidly advance KT-333 in CTCL and exploring its potential in other cancers."
KT-333 is a first-in-class degrader of the transcriptional regulator STAT3. Deregulation of STAT3 signaling has been implicated in the pathogenesis of a variety of cancers, including CTCL.
Kymera received orphan drug designation for KT-333 in the treatment of Peripheral T-cell Lymphoma (PTCL) earlier this year.
FDA grants orphan designation to promote the development of a drug that is expected to have significant therapeutic advantage over existing treatments that target a condition affecting 200,000 or fewer U.S. patients annually.
Price Action : Kymera shares closed Wednesday's trading higher 2.17 percent at $27.81.