Windtree Therapeutics Preclinical Study Results Show Potential for KL4 Surfactant as Possible Medical Countermeasure to Radiation Exposure

WARRINGTON, Pa., Nov. 3, 2016 /PRNewswire/ -- Windtree Therapeutics, Inc. (Nasdaq:WINT), a biotechnology company focused on developing aerosolized KL4 surfactant therapies for respiratory diseases, announced today that recently presented preclinical data at the 62^nd Radiation Research Society Annual Meeting suggest that KL4 surfactant could potentially be an effective medical countermeasure to mitigate acute and chronic/late-phase radiation-induced lung injury (pneumonopathy) due to exposure from a nuclear accident or act of terrorism. The Company believes that, as a possible additional application, KL4 surfactant may also mitigate radiation pneumonopathy associated with cancer radiation therapy.

"While we remain primarily focused on the rigorous and timely execution of the AEROSURF® phase 2 clinical development program for premature infants with respiratory distress syndrome (RDS), a Phase I SBIR grant of $600,000 from the National Institute of Allergy and Infectious Diseases (NIAID) of the National Institutes of Health (NIH) provided us the opportunity to explore the use of KL4 surfactant to mitigate damage to lungs after radiation exposure," commented Craig Fraser, President and Chief Executive Officer. "We are very encouraged by the results of this early preclinical work and are grateful for the support provided by the NIH. Based on the results of this initial study, Windtree was awarded $3 million in additional funding from the NIAID to support the further exploration of aerosolized KL4 surfactant as a potential medical countermeasure for radiation exposure."

In this initial study, KL4 surfactant was administered via an intranasal route into the lungs of C57/BL6 mice 24-hours following exposure to a single fraction of high-dose (13.5 Gy) thoracic-targeted X-ray irradiation (XRT). Mice were evaluated for evidence of reduced blood oxygenation and lung inflammation between two to four weeks post-XRT, and lung fibrosis, chronic pneumonitis, oxidative stress and local and systemic inflammation at 18-weeks post-XRT, by assessing lung function, and analyzing bronchoalveolar fluid (BALF), serum and lung tissue.

The data from this study indicate that KL4 surfactant treatment significantly preserved blood oxygenation in irradiated mice two and four weeks post-XRT suggesting reduced acute lung injury, coupled with significantly reduced lung inflammation in irradiated mice three and 18 weeks post-XRT.  KL4 surfactant-treated irradiated mice also showed a decrease in lung fibrosis and pneumonitis at 18 weeks post-XRT, evidence of reduced chronic/late-phase radiation-induced lung injury.

"Though an early study, these data are especially encouraging as the U.S. Department of Health and Human Services has indicated that there is an urgent and unmet need to develop medical countermeasures to prevent radiation pneumonopathy that can be efficiently administered within days, if not hours post-exposure. These data combined with our promising work to deliver aerosolized KL4 surfactant represent progress toward potentially providing an effective and efficient means to counter exposure to potential radiation lung damage under various circumstances, including potentially as a result of cancer radiation therapy," said Robert Segal, M.D., Senior Vice President, Clinical Development & Academic Affairs, Windtree Therapeutics, the study principle investigator, who conducted this research in collaboration with Melpo Christofidou-Solomidou, PhD, Research Professor of Medicine at the University of Pennsylvania.

Acute and chronic/late-phase radiation induced lung injury

Exposure to ionizing radiation (IR) from an unpredictable nuclear reactor accident, a nuclear attack, deliberate terrorist actions, including the detonation of a radiological dispersal device (RDD), and, represents a significant public health concern. Radiological and nuclear threats are complex, as the radiation source, duration and extent of exposure all contribute to the nature and effects of IR exposure. The lung is particularly susceptible to injury from IR-exposure from external radiation sources, as well as from inhaled radioactive particles from nuclear radioactive fallout as an example. Radiation pneumonopathy can manifest with acute radiation pneumonitis (ARS) and/or delayed effects of acute radiation exposure (DEARE), which may lead to progressive, often fatal, pulmonary fibrosis many months or years later, with a median survival of 2 to 4 years. This is also seen with pulmonary radiation exposure as a result of radiation therapy for certain types of cancer.

This preclinical study of KL4 surfactant as a potential medical countermeasure to mitigate acute and chronic/late-phase radiation-induced lung injury was supported, in part, by a $0.6 million Phase I Small Business Innovation Research (SBIR) grant from the National Institute of Allergy and Infectious Diseases (NIAID) of the National Institutes of Health (NIH) under award number R43AI102308.  The follow-on Phase II SBIR $3.0 M grant supporting ongoing research of aerosolized KL4 surfactant as a radioprotectant is being funded under award number R44AI102308.   The content of this press release is solely the responsibility of Windtree Therapeutics, Inc. and does not necessarily represent the official views of the National Institutes of Health or the University of Pennsylvania.

About Windtree Therapeutics

Windtree Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing novel surfactant therapies for respiratory diseases and other potential applications. Windtree's proprietary technology platform includes a synthetic, peptide-containing surfactant (KL4 surfactant) that is structurally similar to endogenous pulmonary surfactant and novel drug-delivery technologies being developed to enable noninvasive administration of aerosolized KL4 surfactant. Windtree is focused initially on improving the management of respiratory distress syndrome (RDS) in premature infants and believes that its proprietary technology may make it possible, over time, to develop a pipeline of KL4 surfactant product candidates to address a variety of respiratory diseases for which there are few or no approved therapies.

Windtree's lead product candidate is AEROSURF®, a novel, investigational drug/device product that combines the Company's proprietary KL4 surfactant and aerosolization technologies.  AEROSURF is being developed to potentially reduce or eliminate the need for endotracheal intubation and mechanical ventilation in the treatment of premature infants with respiratory distress syndrome (RDS).  Enrollment is ongoing in a  phase 2b clinical trial in up to 240 premature infants to study AEROSURF in premature infants 29 to 32-week gestational age receiving nasal continuous positive airway pressure (nCPAP) for RDS, compared to infants receiving nCPAP alone.  The phase 2b trial is a global trial with clinical sites in North America, Europe and Latin America.

For more information, please visit the Company's website at www.windtreetx.com.

Forward-Looking Statements
To the extent that statements in this press release are not strictly historical, all such statements are forward-looking, and are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.  These forward-looking statements are subject to certain risks and uncertainties that could cause actual results to differ materially from the statements made.  Examples of such risks and uncertainties include those risks related to Windtree's aerosolized KL4 surfactant development programs,  including AEROSURF, which may involve time-consuming and expensive clinical trials that may be subject to potentially significant delays or regulatory holds, or fail; risks related to the development of aerosol delivery systems (ADS) and related components; risks related to the manufacture by contract manufacturers or suppliers of drug products, drug substances, ADS and other materials on a timely basis and in sufficient amounts; risks relating to rigorous regulatory requirements, including those of the U.S. Food and Drug Administration or other regulatory authorities that may require significant additional activities, or may not accept or may withhold or delay consideration of applications, or may not approve or may limit approval of Windtree's products; and other risks and uncertainties described in Windtree's filings with the Securities and Exchange Commission including the most recent reports on Forms 10-K, 10-Q and 8-K, and any amendments thereto.

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SOURCE Windtree Therapeutics, Inc.

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