Advanced Inhalation Therapies Receives Orphan Drug Designation of its Proprietary High Dose Formulation of Nitric Oxide for Adjunctive Treatment of Cystic Fibrosis
Rehovot, Israel and New York, NY – February 09, 2015 – Advanced Inhalation Therapies Ltd (AIT), a leading Nitric Oxide therapeutic company, announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to AIT-CF, the Company’s proprietary high dose formulation of nitric oxide (NO) for adjunctive treatment of cystic fibrosis (CF). In the U.S., Orphan Drug Designation provides a variety of incentives, including seven years of market exclusivity, should AIT-CF receive FDA approval.
AIT-CF is a proprietary nitric oxide (NO) formulation and delivery system designed to deliver a high dose formulation (160 ppm) to the lungs using positive air pressure and integrated monitoring parameters. The Company’s novel system has the potential to eliminate microbial infections including bacteria, fungi and viruses. NO is produced naturally by the body as a highly effective antimicrobial defense mechanism, but to date no delivery system has been able to deliver an effective and non-toxic antimicrobial dosage to the lungs. AIT’s unique and proprietary system continuously monitors safety and efficacy parameters in the patient and is adaptable to treat a wide range of lung infections.
Mr. Amir Avniel, Chief Executive Officer of Advanced Inhalation Therapies, commented, “Receiving Orphan Drug Designation in September 2014 represents an important milestone as we continue to advance the clinical development of AIT-CF. Airway phlegm that contains bacterial colonizations and infections is the primary complication of CF. Inhalation of NO has demonstrated anti-infective, anti-inflammatory and vasodilating properties in CF patients. The results from our completed Phase 2 open label, multi-center study of CF patients over 10 years of age demonstrated the safety and efficacy of our product candidate.”
Cystic Fibrosis (CF) is a lifelong, hereditary disease that causes mucus to form in the lungs and other organs. In 90% of CF cases, this mucus blocks the airways in the lungs, making it hard to breathe and leading to serious lung infections. For hospitalized CF patients, current treatments are supportive through the administration of oxygen through inhalation with bronchiodilators, and include broad spectrum antibiotics and sometimes systemic steroids. Because these treatments do not eliminate all microbes, patients often suffer recurrent infections causing lung damage and ultimately in many cases respiratory failure.
The Orphan Drug designation is granted to development-stage novel therapeutics that offer potential value in the treatment of rare diseases and medical conditions that affect fewer than 200,000 patients in the US. Orphan Drug designation qualifies the sponsor of the drug for various development incentives of the Orphan Drug Act, including tax credits for qualified clinical testing. A marketing application for a prescription drug product that has received Orphan Drug designation is not subject to the Prescription Drug User Fee Act (PDUFA) fees unless the application includes an additional indication other than the rare disease, or condition for which the therapeutic was not designated.
About Nitric Oxide (NO)
Nitric Oxide (NO) is a small, naturally produced gas that acts as an anti-infection agent, making it very effective for the treatment of a wide range of diseases. NO is a naturally occurring mechanism within cells and used as first line of defense against microbials by one’s own immune systems. The discovery of nitric oxide’s signaling role in the cardiovascular and nervous systems has been widely acknowledged as a major breakthrough in biology, and the technology was recognized for its potential to broadly impact the field of medicine with the 1998 Nobel Prize for Physiology or Medicine. At higher doses, NO is a highly effective antimicrobial agent. Since many diseases are caused by the combined activity of bacteria and viruses, the ability to eradicate both agents using NO makes this compound a very powerful weapon in the fight against existing and emerging resistant pathogens.
About Advanced Inhalation Therapies
AIT is a biopharmaceutical company developing novel therapeutics based on Nitric Oxide. The Company is applying its therapeutic expertise in NO to address significant medical needs, many of which cannot effectively be addressed with current standards of care, including small molecules and antibodies. AIT is building a pipeline of Nitric Oxide therapeutics as it has completed a Phase II trial for the treatment of respiratory syncytial virus (RSV) infection and a Phase II trial for the treatment of cystic fibrosis. The Company’s leadership position in fundamental patents, technology, and know-how relating to NO has enabled it to develop a unique NO formulation for the treatment of RSV infection and Cystic Fibrosis. The Company, founded in 2011, maintains global headquarters in Rehovot, Israel and has an additional operating unit in New York, NY. For more information, visit www.AIT-Pharm.com.
Various statements in this release concerning our future expectations, plans, and prospects, including without limitation, statements concerning the use of Nitric Oxide therapeutics for the treatment of RSV and other viral disease and the availability of results of clinical trials and studies, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including risks related to: our approach to discover and develop novel drugs, which is unproven and may never lead to marketable products; our ability to fund and the results of further pre-clinical and clinical trials; obtaining, maintaining and protecting intellectual property utilized by our products; our ability to enforce our patents against infringers and to defend our patent portfolio against challenges from third parties; our ability to obtain additional funding to support our business activities; our dependence on third parties for development, manufacture, marketing, sales, and distribution of products; the successful development of our product candidates, all of which are in early stages of development; obtaining regulatory approval for products; competition from others using technology similar to ours and others developing products for similar uses; our dependence on collaborators; and our short operating history; In addition, any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. We do not assume any obligation to update any forward-looking statements.
Racheli Vizman, COO, AIT
2 Derech Meir Weisgal
Rehovot 9632605, Israel
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