Company confirms FDA agreement on key elements of the Phase 3 segment of ongoing RINGSIDE study, including dosing regimen of 1.2mg once daily
Enrollment in Phase 3 continuing as planned
REHOVOT, Israel & MONMOUTH JUNCTION, N.J., July 05, 2023 (GLOBE NEWSWIRE) — Ayala Pharmaceuticals, Inc. (OTCQX: ADXS), a clinical-stage oncology company, today announced that it has concluded an instructive and successful End-of-Phase-2 (EOP2) meeting with the U.S. Food and Drug Administration (FDA). As a result of the meeting, the company confirms that it is in agreement with the FDA on key elements of the randomized Phase 3 segment of RINGSIDE. The Agency accepted the Company’s selection of the 1.2 mg once daily dose being evaluated in the currently-enrolling Phase 3 and the completed and proposed clinical pharmacology plan. As previously agreed with the FDA on a seamless Phase 2/3 design, enrollment in the Phase 3 segment of RINGSIDE commenced in November 2022, and is continuing globally as planned, with target enrollment of 156 patients.
“We are grateful for the FDA’s continued support and guidance, and very pleased to have reached agreement with the Agency on these items,” said Ken Berlin, President and Chief Executive Officer of Ayala. “Supported by the clinical data generated from studies to date, we believe AL102 has the potential to be a best-in-class gamma secretase inhibitor and may offer a promising new treatment option to patients with unresectable, recurrent or progressive desmoid tumors. There are currently no approved therapies for these rare tumors. Current standards of care, which include off-label chemotherapy, radiation and tyrosine kinase inhibitors, are often poorly tolerated and offer inconsistent efficacy. We look forward to completing enrollment of the Phase 3 segment of this important study.”
The Phase 3 segment of the RINGSIDE study 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.
AL102 received Fast Track designation from the U.S. FDA for the treatment of progressing desmoid tumors, a rare disease with no currently approved treatments.
The RINGSIDE pivotal Phase 2/3 study is a randomized global multi-center trial, with a seamless design, allowing Ayala to continue to Phase 3 without concluding the Phase 2 segment. The Phase 2 segment of the study (Part A) 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. Phase 3 of RINGSIDE (Part B) is a double-blind, placebo-controlled, clinical trial enrolling up to 156 patients with progressive disease, comparing AL102 at 1.2 mg once-daily to placebo. For more information on the RINGSIDE Phase 2/3 study of 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, or other 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 surgical morbidity and a high rate of recurrence post-surgery. There are currently no FDA-approved systemic therapies for the treatment of unresectable, recurrent or progressive desmoid tumors.
AL102 is an investigational small molecule gamma secretase inhibitor (GSI) that is designed to potently and selectively inhibit Notch 1, 2, 3 and 4, and is currently being evaluated in the Phase 2/3 RINGSIDE clinical studies in patients with progressing desmoid tumors. AL102 is designed to inhibit the expression of Notch gene targets by blocking the final cleavage step by the gamma secretase required for Notch activation. Ayala obtained an exclusive, worldwide license to develop and commercialize AL102 from Bristol-Myers Squibb Company in November 2017. AL102 was granted U.S. FDA Fast Track Designation for the treatment of DT.
About Ayala Pharmaceuticals, Inc.
Ayala Pharmaceuticals, Inc. is a clinical-stage oncology company primarily focused on developing and commercializing small molecule therapeutics for people living with rare tumors and aggressive cancers and is also developing proprietary Lm-based antigen delivery products for patients suffering from more common cancers. The Company’s lead candidates under development are the oral gamma secretase inhibitor, AL102, for desmoid tumors; ADXS-504, a Lm-based therapy for early-stage prostate cancer; and the intravenous gamma secretase inhibitor, AL101, for adenoid cystic carcinoma. AL102 has received Fast Track Designation from the U.S. FDA and is currently in the Phase 3 segment of a pivotal study for patients with desmoid tumors (RINGSIDE). For more information, visit www.ayalapharma.com.
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