On November 16, 2022 Ayala Pharmaceuticals, Inc. (Nasdaq: AYLA), a clinical-stage oncology company focused on developing and commercializing small molecule therapeutics for patients suffering from rare tumors and aggressive cancers, reported that the first patient has been dosed in Part B of RINGSIDE, the Phase 3 randomized segment of the trial evaluating AL102 in desmoid tumors (Press release, Ayala Pharmaceuticals, NOV 16, 2022, View Source [SID1234624152]). AL102 is a potent, selective, oral gamma-secretase inhibitor (GSI).
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"We are pleased to begin enrollment in the Phase 3 segment of RINGSIDE, which is a significant milestone in our AL102 development program," said Roni Mamluk, Ph.D., President and Chief Executive Officer of Ayala. "The emerging data from Phase 2 / Part A have been very promising and we have selected a dose of 1.2mg once-daily for the randomized portion. If approved, AL102 has the potential to improve the lives of people living with desmoid tumors."
The Phase 3 segment of RINGSIDE is a double-blind, multi-center trial enrolling up to 156 patients with progressive disease, randomized between AL102 1.2mg dosed once daily or placebo. The primary endpoint is progression-free survival (PFS) with secondary endpoints including objective response rate (ORR), duration of response (DOR), and patient-reported Quality of Life (QOL) measures.
Positive interim data from Part A, the Phase 2 segment of RINGSIDE were presented at ESMO (Free ESMO Whitepaper) in September 2022. Data showed efficacy across all cohorts, with early responses that deepened over time. The first confirmed partial response (PR) was achieved at week 16 and 3 additional unconfirmed PRs over the follow-up period. AL102 was well tolerated with no dose-limiting toxicities and no Grade 4/5 adverse events. Longer-term data from Phase 2 / Part A of RINGSIDE are expected in mid-2023.
AL102 received Fast Track designation from the U.S. FDA granted in September 2022 for the treatment of progressing desmoid tumors.
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 (RINGSIDE).
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 the RINGSIDE study
The RINGSIDE pivotal Phase 2/3 study is a randomized global multi-center trial. The Phase 2 segment of the study evaluated the efficacy, safety, tolerability, and tumor volume by MRI after 16 weeks of AL102 in patients with desmoid tumors. It enrolled 42 patients and evaluated 3 doses of AL102. Patients who participated in the Phase 2 segment of the study are eligible to enroll into an open-label extension study at the selected dose of 1.2 mg daily, and long-term efficacy and safety will be monitored.
The Phase 3 segment of the study has been initiated. This is a double-blind, placebo-controlled segment enrolling up to 156 patients with progressive disease that compares AL102 at 1.2 mg once daily to placebo. The primary endpoint for the Phase 3 segment is progression-free survival (PFS), with secondary endpoints including objective response rate (ORR), duration of response (DOR), tumor volume reduction, and patient-reported Quality of Life (QOL) measures. 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 (RINGSIDE).