Benzinga | Jan 4, 2021 08:03AM EST

Ayala Pharmaceuticals To Accelerate Development Of AL102 For Treatment Of Desmoid Tumors In Pivotal Phase 2/3 Study

- FDA Agreement from End-of-Phase 1 Meeting to Advance to Potential Registrational Study -

- Pivotal Trial Expected to Initiate in 1H21 -

REHOVOT, Israel & WILMINGTON, Del., Jan. 04, 2021 (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 and aggressive cancers, today announced the completion of its end-of-Phase 1 meeting with the U.S. Food and Drug Administration (FDA) on AL102, a potent, selective, oral gamma secretase inhibitor (GSI), for the treatment of desmoid tumors. The FDA has agreed, based on data from AL101 and AL102 studies including durable responses observed in patients with Desmoid tumors, to proceed with a Phase 2/3 pivotal study, which can potentially be used as a registrational study. Ayala expects to initiate the pivotal Phase 2/3 RINGSIDE study in adult and adolescent patients with desmoid tumors in the first half of 2021.

"This exciting news of entering into a potentially registration-enabling pivotal trial, earlier than expected, represents an important step for Ayala as we are able to accelerate the development of AL102 for the treatment of desmoid tumors based on positive and encouraging feedback from the FDA following our end-of-Phase 1 meeting," said Roni Mamluk, Ph.D., Chief Executive Officer of Ayala. "Desmoid tumors are a rare, debilitating and often disfiguring class of soft-tissue tumors for which there are currently no approved therapies. We believe AL102 is well positioned to potentially provide effective systemic treatment based on the body of data conducted by BMS in patients with desmoid tumors implicating the role of Notch pathway in activating aberrant growth pathways contributing to desmoid tumor growth."

The pivotal Phase 2/3 RINGSIDE trial is designed to evaluate the efficacy, safety and tolerability of AL102 in adult and adolescent patients with desmoid tumors. Part 1 of the study will be open label and will enroll up to 36 patients with progressive desmoid tumors in three study arms across three doses of AL102: 1.2 mg daily (QD), 2 mg twice weekly (QIW), and 4mg twice weekly (QIW) with initial follow up of safety, tolerability and tumor volume by MRI after 16 weeks in order to determine the optimal dose. At the end of part 1, all patients will be eligible to enroll into an open label extension study at the selected dose where long-term efficacy and safety will be monitored.

Part 2 of the study will start immediately after dose selection from part 1 and will be a double-blind placebo-controlled study enrolling up to 156 patients with progressive disease, randomized 2:1 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.

The study is expected to commence in the first half of 2021 with an initial interim data read-out from part 1 and dose selection expected by mid-2022 with part 2 of the study to commence immediately thereafter.