This is the third orphan drug designation received by Sapience for ST101, a first-in-class peptide antagonist of C/EBPβ.
The FDA granted orphan designations to ST101 for AML treatment in April 2018 and for glioma in June 2020.
Sapience Therapeutics CEO and president Dr Barry Kappel said: “Patients with advanced stage melanoma have very poor prognosis with a median survival of less than two years from diagnosis.
“The available treatments are limited, and many patients are refractory to targeted approaches or immunotherapy agents.
“With its unique mechanism of action targeting a key transcription factor C/EBPβ, we have a significant opportunity to deliver a novel therapeutic option with ST101.”
ST101 is currently being assessed in the Phase II portion of an ongoing Phase I-II clinical trial in advanced unresectable and metastatic solid tumor patients.
The trial includes expansion arms that are currently dosing and enrolling patients.
They include patients with GBM, cutaneous melanoma, castration-resistant prostate cancer and locally advanced or metastatic hormone-receptor positive breast cancer.
The two-part, open-label, Phase I-II dose-finding trial will determine ST101’s safety, tolerability, PK, PD and proof-of-concept efficacy in advanced solid tumor patients.
Sapience Therapeutics Regulatory Affairs vice-president Dr Gina Capiaux said: “Receiving our third Orphan Drug Designation is another important regulatory achievement that reinforces the FDA’s recognition of the potential of ST101 to improve clinical outcomes in patients with advanced melanoma.”