– 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, DE, USA I July 05, 2022 I 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
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 desmoid tumors, adenoid cystic carcinoma and T-cell acute lymphoblastic leukemia (T-ALL). 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. AL102 is currently in a Pivotal Phase 2/3 clinical trials for patients with desmoid tumors (RINGSIDE). For more information, visit www.ayalapharma.com.

SOURCE: Ayala Pharmaceuticals