Ayala Pharmaceuticals Announces Interim Data from Part A of the Phase 2/3 RINGSIDE Trial of
AL102 in Desmoid Tumors
July 5, 2022
– Tumor shrinking observed in substantially all patients who were evaluable at 16 weeks –
– Favorable safety results observed: AL102 was well tolerated –
– Additional data to be submitted for presentation at a medical conference in 2H 2022 –
REHOVOT, Israel & WILMINGTON, Del., July 05, 2022 (GLOBE NEWSWIRE) — 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 today announced positive interim results from Part A of the ongoing RINGSIDE Pivotal Phase 2/3 clinical trial evaluating AL102 in desmoid
tumors. AL102 is a potent, selective, oral gamma-secretase inhibitor (GSI).
“We are very excited with the interim data from Part A of the RINGSIDE study, although early, demonstrating initial substantial anti-tumor activity for
AL102 as a single agent as measured by MRI scans,” said Roni Mamluk, Ph.D., Chief Executive Officer of Ayala. “We are also encouraged with the
safety data showing that AL102 appears to be well tolerated. We look forward to presenting a more advanced and comprehensive data set at a
medical meeting later in the year. The results from Part A will be used to determine the dose of AL102 to be evaluated in Part B of RINGSIDE, the
randomized portion of the study, which Ayala is on track to initiate in the third quarter of 2022.”
Interim Results as of the Cut-Off Date of May 1, 2022
Patient enrollment in Part A of RINGSIDE was completed in February 2022. Patients were dosed in AL102 monotherapy
cohorts of 1.2 mg (daily), 2mg (2 days on, 5 days off), or 4mg (2 days on, 5 days off).
The activity of AL102 is being evaluated by change in tumor volume (central MRI readings) and response (per RECIST
1.1) determined by the investigator.
MRI scans showed decreases in tumor size in most of the 13 patients who had reached the 16-week time point.
One patient has reached a unconfirmed partial response at week 16 per RECIST.
AL102 was well tolerated at all dose levels with no dose-limiting toxicities and no Grade 4/5 adverse events were
observed.
The most common treatment-related adverse events were Grade 1-2, including diarrhea, fatigue, skin rash, and nausea.
Gary Gordon, M.D., Ph.D., Chief Medical Officer of Ayala commented: “RINGSIDE is the first study to investigate AL102 exclusively in desmoid tumor
patients and it has been designed to evaluate a range of different doses and dose schedules. Successful management of this disease will likely require
chronic treatment and one of the key goals of our development program is to understand the optimal balance between efficacy, safety, and patient
acceptability. We are encouraged by the early but very promising efficacy data and emerging favorable side effect profile for AL102 reported in these
interim results.”
About the RINGSIDE study
The RINGSIDE pivotal Phase 2/3 study is a randomized multi-center trial. Part A of the study is evaluating the efficacy, safety, tolerability, and tumor
volume by MRI after 16 weeks of AL102 in adult and adolescent patients with desmoid tumors. It enrolled 42 patients and is evaluating 3 doses of
AL102. Patients who participated in Part A will be eligible to enroll into an open-label extension study at the selected dose, and long-term efficacy and
safety will be monitored. 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 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.
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 Ayala Pharmaceuticals