On March 8, 2022 Ayala Pharmaceuticals, Inc. (Nasdaq: AYLA), together with M.D. Anderson Cancer Center and the Adenoid Cystic Carcinoma (ACC) Research Foundation reported that the first five patients have been enrolled in a "Window of Opportunity" study for individuals with an aggressive, Notch-activated form of Adenoid Cystic Carcinoma (Press release, Ayala Pharmaceuticals, MAR 8, 2022, View Source [SID1234609642]). Under the leadership of Dr. Renata Ferrarotto at M.D. Anderson, this study is focused on determining the effects of AL101, a gamma-secretase inhibitor being developed by Ayala for the treatment of ACC and other cancers, on the biology of ACC tumors.
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
In this study, participants will have a biopsy to confirm the diagnosis of ACC with Notch activation and will then receive AL101 for 6 to 8 weeks prior to surgery to remove the tumor. AL101 has already shown monotherapy activity in the treatment of individuals with advanced, Notch-activated ACC. The study is being funded by the Department of Defense, the ACC Research Foundation, and Ayala.
"It has been established that mutations in Notch are tumorigenic drivers in ACC and correlate with poorer prognosis. AL101, as a γ-secretase inhibitor, can extinguish Notch signaling in these mutant tumors and has the potential to reduce recurrence and improve long-term patient outcomes," said Dr. Renata Ferrarotto, Associate Professor and Director of Head and Neck Oncology Clinical Research at M.D. Anderson Cancer Center. "This study allows us to monitor changes in the tumor caused by AL101 and to better understand how it affects the biology of the disease."
Dr. Roni Mamluk, Chief Executive Officer of Ayala commented, "There is a compelling rationale for the inhibition of the Notch signaling pathway in ACC. We have already demonstrated clinical proof of concept with AL101 in this disease. The goals of this study are to better understand the mechanism of AL101, help us determine the most successful treatment regime, and generate data that will inform the future development strategy."
Jeffrey Kaufman, Executive Director of the ACC Research Foundation stated, "ACC can be treated with surgery and radiation following initial diagnosis, resulting in relatively high 5-year survival rates. However, late disease recurrence is common and can occur many years after initial treatment. Unfortunately, there are no approved therapies for patients with recurrent/metastatic disease, and the 10 to 20-year survival rates for ACC remain very low. We are very encouraged by the excellent work being conducted by Ayala and M.D. Anderson Cancer Center and the promise that AL101 holds in improving outcomes for patients. We are pleased to support this important study."
For more information on the study, refer to Clinicaltrials.gov identifier NCT04973683.
About Adenoid Cystic Carcinoma (ACC)
ACC is a rare malignancy of the secretory glands including salivary glands, accounting for about 10% of all salivary gland tumors with an annual incidence of 3,400 in the U.S. There is currently no approved standard of care for patients with recurrent/metastatic ACC. Patients with locoregional disease undergo surgery and radiation therapy, with recurring disease treated by chemotherapy. ACC is an immunologically "cold" tumor that is refractory to chemotherapy, with a recurrence rate of about 60% after initial surgery. The Notch pathway has been determined to be an oncogenic driver of ACC and its dysregulation plays a key role in tumorigenesis and correlates with a distinct pattern of metastasis and a poor prognosis.