LEXINGTON, Mass., Aug. 17, 2016 /PRNewswire/ -- Pulmatrix, Inc. (NASDAQ: PULM) announced today that the U.S. Food & Drug Administration has granted the company's request for orphan drug status for its inhaled drug for treating pulmonary fungal infections in cystic fibrosis patients, named PUR1900.
"Your designation is based on a plausible hypothesis that your drug may be clinically superior to the same drug that is already approved for the same indication," the FDA writes.
Special orphan drug status is reserved for promising drugs to treat rare diseases or conditions, and provides both a quick regulatory path to market and financial incentives for companies developing the drugs.
"This designation is a major boost to our efforts to make this drug available as quickly as possible to cystic fibrosis patients who currently suffer from fungal infections in their lungs, and from the allergic reactions they experience because of the fungal infections," says Pulmatrix CEO Robert Clarke, PhD.
Pulmatrix's PUR1900 combines an existing antifungal drug, itraconazole, with the company's innovative dry powder iSPERSE technology, enabling patients to easily inhale the drug deep into their lungs where it's needed. Recent studies have shown that, taken orally, itraconazole is effective in treating allergic reactions in CF patients who have fungal lung infections. However, high oral doses are needed to get enough of the antifungal drug to the lungs through the bloodstream. That causes severe side effects that must be managed and monitored—including liver toxicity.
"Our technology delivers the drug directly to the lungs," explains Pulmatrix's Chief Scientific Officer, David L. Hava, PhD. "That significantly reduces the risks of side effects and drug-drug interactions, bringing great benefits to patients."
"The estimated addressable market for improved antifungal treatments for CF is in the tens of thousands of patients per year," says Dr. Clarke, "but the inhaled drug could also find much larger markets treating pulmonary fungal infections and other immunocompromised patients that could expand the addressable market to millions of patients per year."
Pulmatrix is a clinical stage biopharmaceutical company developing innovative inhaled therapies to address serious pulmonary disease using its patented iSPERSE™ technology. The Company's proprietary product pipeline is focused on advancing treatments for rare diseases, including PUR1900, an inhaled anti-fungal for patients with cystic fibrosis (CF). In addition, Pulmatrix is pursuing opportunities in major pulmonary diseases through collaborations, including PUR0200, a branded generic in clinical development for chronic obstructive pulmonary disease (COPD). Pulmatrix's product candidates are based on iSPERSE™, its proprietary dry powder delivery platform, which seeks to improve therapeutic delivery to the lungs by maximizing local concentrations and reducing systemic side effects to improve patient outcomes.
Certain statements in this press release that are forward-looking and not statements of historical fact are forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. The Company cautions that such statements involve risks and uncertainties that may materially affect the Company's results of operations. Such forward-looking statements are based on the beliefs of management as well as assumptions made by and information currently available to management. Actual results could differ materially from those contemplated by the forward-looking statements as a result of certain factors, including but not limited to the ability to establish that potential products are efficacious or safe in preclinical or clinical trials; the ability to establish or maintain collaborations on the development of therapeutic candidates; the ability to obtain appropriate or necessary governmental approvals to market potential products; the ability to obtain future funding for developmental products and working capital and to obtain such funding on commercially reasonable terms; the Company's ability to manufacture product candidates on a commercial scale or in collaborations with third parties; changes in the size and nature of competitors; the ability to retain key executives and scientists; and the ability to secure and enforce legal rights related to the Company's products, including patent protection. A discussion of these and other factors, including risks and uncertainties with respect to the Company, is set forth in the Company's annual report on Form 10-K filed by the Company with the Securities and Exchange Commission on March 10, 2016. The Company disclaims any intention or obligation to revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.
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SOURCE Pulmatrix, Inc.