Kadmon Corporation Announces National Institutes of Health (NIH) Agreement To Evaluate Gene Therapy Program

NEW YORK, NY--(Marketwired - June 16, 2014) - Kadmon Corporation, LLC, today announced that it has established a Cooperative Research and Development Agreement (CRADA) with the National Institute of Dental and Craniofacial Research (NIDCR), an institute of the National Institutes of Health, to develop an Adeno-associated virus vector containing the human aquaporin-1 gene (AAV2hAQP1) for the treatment of xerostomia resulting from the use of ionizing radiation (IR) in the treatment of head and neck cancer. Kadmon and NIDCR will also explore the use of the salivary gland as a delivery mechanism for therapeutic proteins into the blood.

A significant consequence of IR treatment is the irreversible damage to salivary glands in the IR field resulting in dramatic and permanent reduction in salivary output. Because saliva plays such a critical role in the protection of the tissues of the upper gastrointestinal tract, xerostomia patients suffer considerable morbidity, including oral infections, mucositis, dysphagia, as well as extreme discomfort. There is no adequate therapy currently available to treat this severe condition, which affects approximately 168,000 patients in the US.

Under terms of the agreement, Kadmon and NIDCR will conduct a Phase 1 study to test the safety of single escalating doses of AAV2hAQP1. Effectiveness, measured by increase in parotid gland salivary output post-IR, will also be tested for up to three years.

As salivary glands are easily and non-invasively accessible, self-contained and able to secrete proteins into the bloodstream, the potential exists to use them to express and secrete specific transgene-encoded proteins into the blood. Based on data previously obtained at NIDCR, and using Kadmon’s technology for transgene expression and regulation, the parties will seek to optimize conditions for the delivery of therapeutic proteins in patients via salivary glands.

“Xerostomia is a prevalent and debilitating condition which we are addressing in a unique and durable way through gene therapy,” said Olivier Danos, Ph.D., Senior Vice President, Molecular Medicine, Synthetic Biology and Gene Regulation, of Kadmon. “In addition, thanks to the physiology of the human salivary gland, this project allows us to study an area which is of great interest, the transgene-mediated production of therapeutic proteins in patients’ salivary glands and their secretion into the blood. We look forward to exploring this potential with NIDCR and its extensive know-how relating to gene transfer into salivary glands.”

Dr. Danos will discuss the CRADA project and Kadmon’s gene therapy program in a presentation at the Jefferies Gene Therapy Summit, which is taking place Monday, June 16, 2014, at 8:50 am ET, at the W Hotel in Boston.

About Kadmon Corporation

Kadmon Corporation, LLC, is a global company built on a 21st-century paradigm for the translation of innovative science into treatment. The company currently offers products and services for the treatment and management of liver diseases, and is pioneering novel medicines in areas of serious disease, including oncology, immunology and infectious, neurodegenerative and ophthalmic diseases. Emphasizing emerging concepts in molecular biology and genomics, Kadmon is developing treatments and treatment combinations that target the metabolomics and signaling pathways associated with disease, with the goal of addressing some of today’s most pressing areas of unmet medical need. For more information, visit www.kadmon.com.

This press release contains forward-looking statements. These forward-looking statements are based on management’s expectations and are subject to certain factors, risks and uncertainties that may cause actual results, outcome of events, timing and performance to differ materially from those expressed or implied by such statements. The information contained in this press release is believed to be current as of the date of original issue. Kadmon expressly disclaims any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations with regard thereto or any change in events, conditions or circumstances on which any such statements are based.