Topigen Pharmaceuticals Inc, an emerging biopharmaceutical company specializing in developing treatments for respiratory diseases has announced preliminary data from its first phase II clinical trial of inhaled TPI-ASM8 as a single agent in patients with allergic asthma.

The results indicate that inhaled TPI-ASM8 demonstrated protection in early and late stage allergic responses in patients with asthma. Paul O'Byrne, MD, chairman of medicine, McMaster University, Canada and the principal investigator of the study commented on the results, said, "These are impressive and encouraging data for a potential new class of therapeutics for treating respiratory diseases. This is the first demonstration of efficacy with a RNA-targeting drug in respiratory patients. In this 'proof-of-principle' trial, inhaled TPI-ASM8 was shown to be safe and well tolerated and it demonstrated significant biological and physiological activity in study subjects, including substantial reductions of eosinophil cell levels and suppression of target gene expression. I am looking forward to working on expanded phase II studies with TOPIGEN to exploit the full potential of this novel drug."

The 17-patient, randomized, placebo-controlled, multi centre, crossover, allergen challenge phase II trial was designed to assess overall safety and tolerability and to demonstrate proof-of-principle that inhaled TPI-ASM8 would show biological activity in the respiratory tract of patients with mild-tomoderate asthma.

The study evaluated repeated administration of once daily doses of inhaled TPI-ASM8, given for four days at delivered doses similar to currently marketed steroids. Paolo Renzi, MD, Topigen's chief scientific officer and founder commented, "Topigen is the first company to show a biological effect in an established model of asthma with lung-specific antisense oligonucleotide therapy. This was our very first attempt to examine the activity of inhaled TPI-ASM8 in patients and these results confirmed earlier findings seen in our pre-clinical studies. Assuming that these data are confirmed in subsequent and larger clinical studies, we are confident that we can develop a meaningful therapeutic alternative to inhaled corticosteroids for patients with asthma. There is a pressing need to develop new drugs with highly specific mechanisms of action that can alter the course of the disease while minimizing systemic side effects." Paul K. Wotton, PhD, president and CEO of Topigen added, "This study provides strong validation of Topigen's novel approach on multiple levels. First and foremost, Topigen's proprietary antisense therapy approach works effectively in the respiratory tract. Second, our specific and multi-targeted approach is safe and effective in this patient population. And third, it provides validation of our receptor targets in patients with asthma. This is a significant milestone towards building Topigen as a leading biopharmaceutical company focused on developing innovative treatments for respiratory diseases."