REHOVOT, Israel and WILMINGTON, Del., Sept. 18, 2020 (GLOBE NEWSWIRE) — Ayala Pharmaceuticals, Inc. (Nasdaq: AYLA) (Ayala or the Company), a clinical-stage oncology company focused on developing and commercializing small molecule therapeutics for patients suffering from rare and aggressive cancers, primarily in genetically defined patient populations, today announced positive interim results from the ongoing Phase 2 ACCURACY clinical trial of AL101 for the treatment of recurrent/metastatic (R/M) adenoid cystic carcinoma (ACC) harboring Notch-activating mutations in a mini oral presentation at the European Society for Medical Oncology (ESMO) Virtual Congress 2020.

“The study’s interim results are encouraging as we continue to study AL101 as a potential therapy for ACC patients with Notch-activating mutations. ACC is an orphan disease with no approved therapies and patients with Notch mutations have a more aggressive disease course and poorer survival outcomes as compared to patients with Notch wild-type,” said Renata Ferrarotto, M.D., Associate Professor, Department of Thoracic/Head and Neck Medical Oncology, The University of Texas MD Anderson Cancer Center and principal investigator in the study. “It’s promising to see meaningful clinical activity, including a significant disease control rate with a single agent in this patient population that represents a major unmet clinical need.”

“We are pleased to see promising progress from the Phase 2 ACCURACY clinical trial of AL101 as a potential monotherapy for ACC with a significant disease control rate in the 4mg arm of 68% from 40 evaluable patients, building upon the 61% disease control rate from 27 patients that we reported in 2019,” said Roni Mamluk, Ph.D., Chief Executive Officer of Ayala. “Within the context of this difficult to treat cancer, in which people living with ACC have become accustomed to historically low response rates and failed therapies, it’s exciting to see signs of advancement with both safety and efficacy. We remain on track with enrollment in the 6mg dose cohort arm and we look forward to providing further trial progress updates in the first half of 2021.”

The ongoing Phase 2 ACCURACY clinical trial is an open-label, single-arm, multi-center study to assess the clinical activity of AL101 using radiographic assessments of patients with R/M ACC demonstrating disease progression within 6 months prior to dosing. The Company is evaluating the safety and efficacy of AL101 for the treatment of R/M ACC with Notch-activating mutations in two dose cohorts, 4mg once per week (QW) and 6mg QW. Dosing for the 6mg QW cohort commenced in March 2020 and is currently enrolling up to 42 subjects.

Updated Preliminary Safety and Efficacy Results:
As of July 30, 2020, of the 45 subjects who were enrolled in the 4 mg QW cohort, 40 were evaluable for efficacy for a best response by investigators using RECIST 1.1 criteria.

Partial responses were observed in six subjects (15%) and stable disease was observed in 21 subjects (53%), yielding a 68% disease control rate;
Approximately 40% of evaluable patients remained on drug for at least six months after entering the study with disease progression, including two patients treated beyond progression; and
Pharmacokinetics (PK) results were similar to those of the AL101 Phase 1 study with no major effect by CYP inhibitors or substrates on AL101 exposure.

AL101 was generally observed to be well-tolerated, with most adverse events being mild to moderate in severity:

Most treatment related adverse events (TRAEs) were of grade 1/2 severity; and
The most common TRAEs of any grade included diarrhea (60%) of which 4% was grade 3, fatigue (51%) of which 4% was grade 3, nausea (49%) of which 2% was grade 3 and hypophosphatemia (42%) of which 4% was grade 3.

Virtual Key Opinion Leader (KOL) Conference Call and Webcast Event
Ayala will host a virtual event for investors and analysts to review the interim data from the Phase 2 ACCURACY clinical trial of AL101 for the treatment of R/M ACC. The live webcast event will begin at 8:00am ET and will include a discussion of the data with Alan L. Ho, M.D., Ph.D., Medical Oncologist at Memorial Sloan Kettering and a lead investigator of the ACCURACY clinical trial, as well as presentations from Ayala management.

To access the call, please dial 833-519-1339 (United States & Canada) or 914-800-3901(international) and reference the conference ID3995105. A live webcast of the conference call will be available on the Investors and News section of Ayala’s website at ir.ayalapharma.com. A replay of the webcast and accompanying slides will be available on the Ayala website for 90 days following the call.