ST101 Granted FDA Fast Track Designation for Glioblastoma Multiforme

The FDA has granted fast track designation to ST101, a peptide antagonist of C/EBPβ, for the treatment of patients with glioblastoma multiforme (GBM), according to a press release issued by Sapience Therapeutics, Inc.

ST101 is already showing efficacy signals in this patient population in a phase 1/2 clinical trial of patients with solid tumors (NCT04478279). So far, 1 partial response has been observed in the cutaneous melanoma subgroup, and several patients with other solid tumors have achieved long-lasting stable disease.

"Glioblastoma is the most aggressive, fatal form of brain cancer with limited-to-no therapeutic options. With the efficacy data from solid tumors and its ability to cross the blood-brain barrier, ST101 could meaningfully improve outcomes for patients battling this debilitating disease and we look forward to advancing it into phase 2," said Barry Kappel, PhD, MBA, chief executive officer and president of Sapience Therapeutics.

The study of ST101 has an open-label, 2-part, dose-finding design. An estimated 162 patients with either GBM, hormone receptor-positive breast cancer, melanoma, or castration-resistant prostate cancer will be enrolled in the study. The coprimary end points of the study are the number of patients who experience dose-limiting toxicity, and the number of patients with adverse events (AEs). The secondary end points of the study include area under the curve, Cmax, terminal half-life, overall response, disease control rate, duration of response, and progression-free survival.

Patients in the GBM cohort must have primary GBM that has recurred or progressed after 1 standard treatment regimen. Standard therapies for GBM include maximal surgical resection, radiotherapy, and concomitant temozolomide (Temodar) with radiotherapy, or adjuvant chemotherapy with temozolomide.

To enroll in the study, patients are required to be at least 18 years of age with an ECOG performance status of 0-1, evaluable disease per RECIST v1.1, disease that progressed on or is non-responsive to the previous line of therapy per RECIST v1.1 and resolved adverse events to grade 1 after prior anti-cancer therapy. All patients must have locally advanced or metastatic disease, agree to a biopsy, and be open to using contraception during the study.

The study is actively recruiting patients who meet the study inclusion criteria at 8 centers across the United States and United Kingdom. US states in which the clinical trial is open include Colorado, Illinois, Michigan, New York, and Tennessee.

This is an exciting regulatory milestone for our program. Fast Track designation for ST101 highlights this unmet need and allows us to work closely with the FDA to deliver this novel therapy to people with GBM as soon as possible,” said Gina Capiaux, PhD, head of Regulatory Affairs at Sapience Therapeutics, in the press release.

With a fast track designation from the FDA, the development of ST101 will be expedited considering its potential to address an unmet medical need. ST101 already has an orphan drug designation from the FDA and European Medicines Agency for the treatment of glioma.

_References:

_{1. Sapience Therapeutics receives FDA fast track designation for st101 for the treatment of recurrent glioblastoma multiforme (GBM). News release. December 6, 2021. Accessed December 6, 2021. https://bit.ly/302i3Pg}

_{2. A phase 1-2 study of st101 in patients with advanced solid tumors. Clinicaltrials.gov. Accessed December 6, 2021. https://bit.ly/3DtrPrj}