FDA Clears Thrombolytic Science's IND for Mutant Prourokinase in Thrombotic Diseases

Thrombolytic Science, LLC (TSI), a biotech company specializing in the development of clot-dissolving treatments for thrombotic diseases, has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application concerning mutant prourokinase (mproUK). This approval will allow TSI to conduct a clinical trial in the United States to further assess the safety and tolerability of mproUK in healthy individuals.

Alexis C. Wallace, MSc. Eng., MBA, CEO of TSI, expressed enthusiasm about this milestone, stating that the FDA’s clearance marks a significant advancement for the company. Wallace highlighted the innovative nature of TSI’s approach to fibrinolytic therapy, which has the potential to revolutionize treatment options for life-threatening thrombotic conditions by offering a faster, safer, and more effective means of reperfusion. Following successful Phase II results from the DUMAS trial involving stroke patients in the Netherlands, TSI is now initiating a Phase II trial in the UK for myocardial infarction, having received approval from the MHRA. The recent IND clearance represents a crucial step in making TSI’s low-dose fibrinolytic treatment more accessible to a broader range of patients worldwide.

The human body’s natural mechanism for clot dissolution involves both tissue plasminogen activator (tPA) and prourokinase. While tPA initiates the process of clot lysis, prourokinase completes the breakdown of the fibrin clot. Victor Gurewich, MD, co-founder of TSI and the discoverer of prourokinase, likened this relationship to that of a car starter and its engine. Gurewich explained that physiological fibrinolysis requires a small bolus of r-tPA followed by the infusion of a low dose of mproUK. Recent clinical studies have demonstrated that this treatment approach is free of bleeding risks and rethrombosis, and it may re-establish blood flow more quickly and be more cost-effective compared to in-hospital surgical solutions.

TSI’s mproUK treatment is rooted in the natural biological mechanism of clot dissolution, which involves the sequential action of tPA and prourokinase. The latter has been stabilized through a single site mutation, significantly enhancing its safety profile without compromising its efficacy in breaking down clots. A proof-of-concept clinical trial, known as the PATENT trial, previously demonstrated the effectiveness of native prourokinase in heart attack patients when preceded by a small dose of tPA. More recently, the DUMAS trial yielded promising results for the use of mutant proUK in patients suffering from ischemic stroke.

Thrombolytic Science, LLC, is a private biotech company dedicated to developing the next generation of clot-dissolving therapies for conditions such as heart attack, ischemic stroke, and other thrombotic diseases. This work is based on pioneering research conducted by the company’s scientific co-founders at Beth Israel Deaconess Medical Center (BIDMC) and Harvard Medical School.

This breakthrough in fibrinolytic therapy promises to deliver enhanced safety and efficacy, potentially transforming the treatment landscape for patients suffering from various thrombotic conditions.