Ayala Pharmaceuticals, Inc. a clinical-stage oncology company focused on developing and commercializing small molecule therapeutics for patients suffering from rare tumors and aggressive cancers, today announced that it has completed patient enrollment in Part A of the Phase 2/3 RINGSIDE clinical trial evaluating AL102 in desmoid tumors. AL102 is a potent, selective, oral gamma-secretase inhibitor (GSI). The Company expects to report interim results from Part A by mid-2022.

“Completion of enrollment in Part A of the RINGSIDE trial is an important milestone in our AL102 clinical program for desmoid tumors,” said Roni Mamluk, Ph.D., Chief Executive Officer of Ayala. “Part A will help us determine the optimal dose for the randomized portion of the study, we will be looking for safety and signs of anti-tumor activity based on MRI. We are extremely grateful to the patients who are participating and we look forward to sharing top line data around mid-year.”

A total of 36 patients have been enrolled in Part A of the RINGSIDE study which is evaluating the safety, tolerability of AL102, as well as tumor volume by MRI at 16 weeks. Three dosing regimens of AL102 are being tested to determine the optimal dose regimen to advance forward.

In Part A, the effect of food on AL102 absorption was tested in a sub-study and PK results indicate that no food restrictions are required.

Part B of the study will be double-blind, placebo-controlled, and will start immediately after dose selection from Part A, enrolling up to 156 patients age 12 and up with progressive disease, randomized between AL102 or placebo. The study’s primary endpoint will be progression-free survival (PFS) with secondary endpoints including objective response rate (ORR), duration of response (DOR,) and patient-reported Quality of Life (QOL) measures. Patients who participated in Part A are eligible to enroll into an open-label extension study at the selected dose, and long-term efficacy and safety will be monitored.

For more information on the RINGSIDE Phase 2/3 study with AL102 for the treatment of desmoid tumors, please visit ClinicalTrials.gov and reference Identifier NCT04871282.

About AL102

AL102 is a potent, selective, oral gamma secretase inhibitor (GSI). AL102 is currently being developed for the treatment of desmoid tumors, as well as in combination with Novartis’ B-cell maturation antigen (BCMA)-targeting agents for the treatment of multiple myeloma (MM).

About Desmoid Tumors

Desmoid tumors, also called aggressive fibromatosis or desmoid-type fibromatosis, are rare connective tissue tumors that typically arise in the upper and lower extremities, abdominal wall, head and neck area, mesenteric root and chest wall with the potential to arise in additional parts of the body. Desmoid tumors do not metastasize, but often aggressively infiltrate neurovascular structures and vital organs. People living with desmoid tumors are often limited in their daily life due to chronic pain, functional deficits, general decrease in their quality of life and organ dysfunction. Desmoid tumors have an annual incidence of approximately 1,700 patients in the United States and typically occur in patients between the ages of 15 and 60 years. They are most commonly diagnosed in young adults between 30-40 years of age and are more prevalent in females. Today, surgery is no longer regarded as the cornerstone treatment of desmoid tumors due to high rate of recurrence post-surgery and there are currently no FDA-approved systemic therapies for the treatment of unresectable, recurrent or progressive desmoid tumors.

About Ayala Pharmaceuticals
Ayala Pharmaceuticals, Inc. is a clinical-stage oncology company focused on developing and commercializing small molecule therapeutics for patients suffering from rare tumors and aggressive cancers. Ayala’s approach is focused on predicating, identifying and addressing tumorigenic drivers of cancer through a combination of its bioinformatics platform and next-generation sequencing to deliver targeted therapies to underserved patient populations. The company has two product candidates under development, AL101 and AL102, targeting the aberrant activation of the Notch pathway with gamma secretase inhibitors to treat a variety of tumors including Adenoid Cystic Carcinoma, Triple Negative Breast Cancer (TNBC), T-cell Acute Lymphoblastic Leukemia (T-ALL), Desmoid Tumors and Multiple Myeloma (MM) (in collaboration with Novartis). AL101, has received Fast Track Designation and Orphan Drug Designation from the U.S. FDA and is currently in a Phase 2 clinical trial for patients with ACC (ACCURACY) bearing Notch activating mutations and in a Phase 2 clinical trial for patients with TNBC (TENACITY) bearing Notch activating mutations and other gene rearrangements. AL102 is currently in a Pivotal Phase 2/3 clinical trials for patients with desmoid tumors (RINGSIDE) and is being evaluated in a Phase 1 clinical trial in combination with Novartis’ BMCA targeting agent, WVT078, in Patients with relapsed/refractory Multiple Myeloma