Ayala Pharmaceuticals Announces Fast Track Designation Granted by US FDA for AL102 in Progressing Desmoid Tumors
09/27/2022 | 08:01am EDT
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REHOVOT, Israel and WILMINGTON, Del., Sept. 27, 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 that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for AL102 for the treatment of progressing desmoid tumors. AL102 is a potent, selective, oral gamma-secretase inhibitor.
“We are pleased to receive FDA Fast Track designation for AL102 in progressing desmoid tumors, which we believe reinforces the large unmet medical need for patients with this serious disease. This designation holds important advantages that may expedite the development and regulatory review of AL102,” said Roni Mamluk, Ph.D., Chief Executive Officer of Ayala. “We are very encouraged by the emerging body of clinical data supporting AL102 and, if approved, believe that this product could have a meaningful impact on patients’ lives.”
The FDA grants Fast Track designation to facilitate development and expedite the review of therapies with the potential to treat a serious condition where there is an unmet medical need. A therapeutic that receives Fast Track designation can benefit from early and frequent communication with the agency, in addition to a rolling submission of the marketing application, with potential pathways for expedited approval that have the objective of getting important new therapies to patients more quickly.
AL102 is being evaluated in the ongoing RINGSIDE pivotal Phase 2/3 clinical trial in desmoid tumors. Positive interim results from Part A, the Phase 2 segment of this study, were presented at ESMO 2022, showing efficacy across all cohorts, with early tumor responses that deepened over time. AL102 was well tolerated. The company has initiated Part B of RINGSIDE (Phase 3), and is enrolling patients in an open label extension study.
About the RINGSIDE study
The RINGSIDE pivotal Phase 2/3 study is a randomized global 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 patients with desmoid tumors. It enrolled 42 patients and is evaluating 3 doses of AL102. Patients who participated in Part A are eligible to enroll into an open-label extension study at the Part B selected dose of 1.2 mg daily, and long-term efficacy and safety will be monitored.
Part B of the study, the Phase 3 segment, has been initiated. This is a double-blind, placebo-controlled segment enrolling up to 156 patients with progressive disease, comparing AL102 at 1.2 mg once daily to placebo. The primary endpoint for Part B is progression-free survival (PFS) with secondary endpoints including objective response rate (ORR), duration of response (DOR), tumor volume reduction, 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 a 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 Adenoid Cystic Carcinoma (ACC), T-cell Acute Lymphoblastic Leukemia (T-ALL), Desmoid Tumors and Multiple Myeloma (MM). 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.
Contacts:
Investors:
Joyce Allaire
LifeSci Advisors LLC
+1-617-435-6602
jallaire@lifesciadvisors.com
Ayala Pharmaceuticals:
+1-857-444-0553
