OrPro to advance development of ORP-100 for treatment of cystic fibrosis

OrPro Therapeutics, Inc. today announced that the company has been awarded a National Heart, Lung and Blood Institute Small Business Innovation Research (SBIR) grant from the National Institutes of Health (NIH). The grant enables OrPro to advance development of its lead product, ORP-100, for the treatment of cystic fibrosis (CF), an inherited genetic disease that affects approximately 80,000 people worldwide.

"This award focuses on an inhaled formulation of ORP-100 for topical delivery to the lung surface, a key milestone in our preclinical development program," said Peter B. Heifetz, Ph.D., OrPro president and chief technology officer. "NIH grants are highly competitive, and our proposal received excellent reviews and scores acknowledging OrPro's strong team and innovative approach to CF therapy."

ORP-100 is a recombinant engineered variant of thioredoxin, a human lung protein that has demonstrated in laboratory studies a potent ability to increase the fluidity of mucus. Clearance of the thickened mucus that is characteristic of CF remains a central and poorly met treatment objective. ORP-100 will be administered through an advanced aerosol delivery system, and in contrast to DNA-degrading mucolytics, targets the adhesive protein gel network that is common to all patients with obstructive mucus.

"We are excited about the potential for ORP-100 to be a next-generation mucus-thinning (mucolytic) drug that will offer a convenient, safe and effective treatment alternative to the people who suffer from CF," said Jeff Raser, chief operating officer.  "New therapeutic strategies to correct underlying CF defects have promise, but there still remains a critical need for superior mucolytic therapies in CF and other obstructive pulmonary diseases such as bronchiectasis."

The mucolytic properties of thioredoxin were discovered at leading respiratory hospital National Jewish Health (NJH) in Denver, CO by pediatric pulmonologist Dr. Carl White, M.D.  In 2011, OrPro licensed from NJH an intellectual property portfolio claiming compositions and uses of the thioredoxin active site for the treatment of pulmonary disease.  Dr. White (now at University of Colorado Denver/Children's Hospital Colorado), and his NJH colleague Dr. David Nichols are medical advisors to the company, and will collaborate on the NIH-funded project. 

"We have assembled a world-class team of experts in research, preclinical/clinical development, biologics manufacturing, and finance to help guide OrPro in the development of this exciting new product candidate," said Heifetz, who is also principal investigator on the SBIR grant. "We believe the support from NIH will act as a catalyst for the company and we look forward to completing an initial round of financing to accelerate our development efforts."