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Chronic obstructive pulmonary disease (COPD) is a chronic progressive disease characterized by development of airflow limitation, eventually leading to a largely irreversible loss of lung function. Chronic cigarette smoking is the most important risk factor for this global health issue. According to the National Heart, Lung, and Blood Institute (NHLBI) 13.5 million Americans, approximately 1 in 20 or 4.96%, have COPD. In addition 1 in 18 or 5.51% of the general population or 15 million people are undiagnosed COPD sufferers. About 24 million adults have evidence of impaired lung function indicating that indeed COPD is under-diagnosed. By 2020, COPD is expected to rise to the third leading cause of death in the U.S.A. There are only a few treatment options available to the millions of patients who suffer from this killer disease. None provides a cure and all drugs treat only the symptoms.We are developing CTPi-1, another siRNA drug candidate molecule inhibiting the target RTP801, for this indication. This development is also part of our license agreement with Pfizer and if Pfizer elects to proceed with its development, will be fully funded by Pfizer. We have shown that (1) RTP801 is over-expressed in the lungs of human emphysema patients; (2) RTP801 protein is induced in mouse lungs in response to cigarette smoking exposure; (3) RTP801 KO mice have blunted lung inflammatory and apoptotic response to acute cigarette smoking exposure and do not develop emphysema following prolonged, six month exposure; (4) results from mice treated by CTPi-1 delivered intratracheally have shown efficacy trend similar to that obtained in the KO mice. Dosing optimization and pharmacokinetic studies are presently being performed in animals and we aim to conclude them shortly.
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