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FDA Grants Orphan Drug Designation to Ayala’s AL101 for Potential Treatment of Adenoid Cystic Carcinoma (ACC)

May 13, 2019

REHOVOT, Israel & WILMINGTON, Del.--(BUSINESS WIRE)--May 13, 2019--Ayala Pharmaceuticals, a clinical-stage company developing medicines for cancers that are genetically defined, announced today that it has been granted Orphan Drug Designation from the U.S. Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) for AL101, a potent and selective inhibitor of gamma secretase-mediated Notch signalling, for the treatment of ACC.

Orphan Drug Designation is granted to drug therapies intended to treat diseases or conditions that affect fewer than 200,000 people in the United States. Orphan Drug Designation by the FDA entitles Ayala to seven years of market exclusivity for the use of AL101 for the treatment of ACC, if approved, plus significant development incentives, including tax credits related to clinical trial expenses, an exemption from the FDA-user fee, and FDA assistance in clinical trial design.

“Receiving Orphan Drug Designation from the FDA signifies our continued progress and commitment to develop AL101 as a potential treatment for people with ACC,” said Roni Mamluk, Ph.D., Chief Executive Officer at Ayala Pharmaceuticals. “Given the proof of concept demonstrating that AL101 has a significant inhibitory effect on tumor growth in a mouse model using implanted ACC patient-derived xenograft tumors coupled with the significant unmet need in ACC, we look forward to accelerating AL101 development for patients in the U.S.”

ACC is a rare form of cancer. In the U.S., there are approximately 566,000 people diagnosed with cancer each year, and only about 1,224 of them are diagnosed with ACC. According to the Adenoid Cystic Carcinoma Organization International, there are approximately 14,873 Americans alive today living with this disease. Current treatment options include surgery, chemotherapy and/or radiation therapy; however, there is no approved drug for the treatment of ACC.

About AL101

AL101 is a gamma secretase inhibitor developed as a Notch inhibitor for oncology indications. Notch signaling pathway plays an important role in tumorigenesis in several solid and hematological malignancies. Upon ligand binding of the Notch receptor, an important step in the activation of Notch receptors is cleavage by gamma secretase, which frees the Notch intracellular signaling domain.

AL101 is currently in Phase 2 for adenoid cystic carcinoma patients with tumor bearing Notch activating mutations (ACCURACY). For additional information about AL101 ACCURACY clinical trial, please go to www.clinicaltrials.gov. Interested patients and physicians can contact Medical Affairs at Ayala for more information at: accuracy@ayalapharma.com or call refer +1-857-444-0553.

About Ayala Pharmaceuticals

Ayala Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company dedicated to developing targeted cancer therapies for people living with genetically defined cancers. Ayala is broadly developing its product candidates, AL101 and AL102, best-in-class gamma secretase inhibitors, with clinical and preclinical studies underway in both solid tumors (AL101) and hematologic malignancies (AL102).

As a precision oncology company, Ayala was founded in November 2017 with an experienced global management team and a strong investor base. For more information, visit www.ayalapharma.com.

View source version on businesswire.com:https://www.businesswire.com/news/home/20190513005236/en/

CONTACT: Investors

Ayala Pharmaceuticals

+1-857-444-0553

info@ayalapharma.comMedia

Katie Dodge

+1 617-657-1304

kdodge@jpa.com

KEYWORD: UNITED STATES NORTH AMERICA DELAWARE MIDDLE EAST ISRAEL

INDUSTRY KEYWORD: HEALTH BIOTECHNOLOGY CLINICAL TRIALS ONCOLOGY PHARMACEUTICAL FDA GENERAL HEALTH

SOURCE: Ayala Pharmaceuticals, Inc.

Copyright Business Wire 2019.

PUB: 05/13/2019 09:30 AM/DISC: 05/13/2019 09:30 AM

http://www.businesswire.com/news/home/20190513005236/en