FDA Grants Orphan Drug Designation to LNS8801 for Metastatic Cutaneous Melanoma
Source: Targeted Oncology, January 2023
The FDA has granted orphan drug designation for LNS8801 for the treatment of patients with metastatic cutaneous melanoma, according to Linnaeus Therapeutics, Inc.
LNS8801 is an oral, bioavailable, highly specific, and potent agonist of GPER. GPER activation by LNS8801 works to rapidly and durably deplete c-Myc protein levels. Preclinical models of LNS8801 have shown the agent to elicit potent antitumor activities across multiple tumor types, rapidly shrinking tumors and inducing immune memory.
The basis of this orphan drug designation comes from findings from the phase ongoing 1/2 adaptive-design clinical trial (NCT04130516) of LNS8801. In the trial, LNS8801 has proven to be safe and well-tolerated. LNS8801 also has demonstrated target engagement, c-Myc protein depletion, and clinical benefit in patients with advanced cancers.