Diffusion Pharmaceuticals LLC today announced positive results of its Phase I/II proof-of-concept study of Trans Sodium Crocetinate (TSC) in patients suffering from walking impairment due to peripheral artery disease (PAD). The study results, presented orally today to an international audience at the American Heart Association Scientific Sessions, demonstrate that TSC could improve walking ability, delay onset of leg pain and enhance overall quality of life for people with PAD. TSC is a novel small molecule drug that treats hypoxia, the lack of oxygen in tissues that underlies PAD.
“Our goal is to bring to market a safe and effective therapy as quickly as possible, providing new hope for patients with PAD, cardiovascular disease, cancer and other conditions related to hypoxia.”
"Based on these encouraging results, we are exploring opportunities with several companies to advance the development of TSC," said David Kalergis, Chief Executive Officer of Diffusion Pharmaceuticals. "Our goal is to bring to market a safe and effective therapy as quickly as possible, providing new hope for patients with PAD, cardiovascular disease, cancer and other conditions related to hypoxia."
In this 48 patient study, a notable dose-response was observed for peak walking time on a graded treadmill test at several TSC dosing levels after five days of treatment. There was a clinically meaningful improvement for the patients who received 1.50 mg/kg of TSC that was nearly three times the improvement observed with the placebo group.
"PAD remains a challenging condition to treat, with an inadequate number of approved therapies. Given the dearth of successful clinical research options in PAD, these study results are exciting," said study presenter William Hiatt, MD, professor of cardiovascular research in the Department of Medicine, University of Colorado School of Medicine; President of CPC Clinical Research and chairman of the American Heart Association's Peripheral Vascular Disease Council. "The magnitude of improvement that we saw with TSC after 5 days of treatment is in the range of that seen after months of therapy with a marketed product for PAD, but with a potentially superior safety profile for TSC."
The Phase I/II trial was a randomized, double-blinded, placebo-controlled study of TSC in patients with intermittent claudication symptoms from PAD. The study enrolled 48 patients, with 5 patients each receiving TSC at doses of 0.25, 0.50, 0.75, 1.00, 1.25, 1.50, 1.75 and 2.0 mg/kg and a placebo arm. Study medication was dosed intravenously as a bolus injection once daily for five consecutive days. Forty patients received TSC and 8 received placebo. TSC was safe and well-tolerated at all doses in this elderly PAD population. Adverse events were not predominant on any drug dose relative to placebo.
The trial was sponsored and funded by Diffusion Pharmaceuticals and conducted in collaboration with CPC Clinical Research, a not-for-profit academic research organization affiliated with the University of Colorado and specializing in the clinical testing of new approaches to the treatment of PAD. CPC is headed by Dr. Hiatt. Investigator study sites included the University of Pennsylvania, Stanford University, the University of Oklahoma and four private research clinics.
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