SAN DIEGO, Oct. 9, 2014 /PRNewswire/ — OrPro Therapeutics, Inc., a pre-clinical stage biopharmaceutical company, announced that it will present data on the development of its inhaled treatment for cystic fibrosis (CF) at the 28thAnnual North American Cystic Fibrosis Conference, October 9 – 11 in Atlanta.
CF is an inherited genetic disease that affects approximately 80,000 people worldwide. OrPro’s lead compound, ORP-100, is an enhanced version of a naturally-occurring human enzyme, thioredoxin, which has been engineered to normalize CF secretions in the airway through a unique patent-pending mechanism.
A poster-session presentation titled “A Novel Inhaled Mucus-Normalizing Therapy For Cystic Fibrosis” will include data from initial in vitro and in vivo characterization of ORP-100 demonstrating that the compound significantly reduces the excess viscosity and adhesiveness of CF patient sputum, and does not generate inflammation in animals even when administered at many times the anticipated human dose.
OrPro conducted these preclinical studies – which were funded in part through a grant from the National Institutes of Health – in collaboration with National Jewish Health (Denver, CO). The company is also collaborating on additional non-clinical studies with the Cystic Fibrosis Foundation (Cystic Fibrosis Foundation Therapeutics) and another leading CF-research institution, the University of North Carolina Medical Center (Chapel Hill, NC).
ORP-100 binds to and disrupts the abnormal bonds that form between the mucin protein chains that make up the bulk of the respiratory mucus, with the intended benefit of improving mucus transport. This targeted, site-specific mechanism of action differs significantly from other mucus-modification approaches: the ORP-100 molecule remains bound once it has acted, which facilitates extracellular clearance and may prevent new mucus bonds from forming. The potency and long-lasting mucus-normalizing activity inherent in the ORP-100 enzymatic activity observed in preclinical studies may create efficacy and safety advantages in the clinic that could offer the possibility of disease modification.
OrPro Therapeutics, Inc. is a pre-clinical stage biopharmaceutical company headquartered in San Diego, CA. The company’s goal is to develop a breakthrough class of safe, well-tolerated and more effective inhaled, non-systemic drugs based on the thioredoxin active site for the treatment of patients with serious obstructive pulmonary diseases. OrPro’s lead product, ORP-100, an aerosolized variant of recombinant human thioredoxin, targets pathological, adhesive mucus and has the potential for broader clinical efficacy than existing mucolytic approaches.