TRANSLATIONAL SCIENCES INC. — Department of Health and Human Services SBIR Phase II: NHLBI

Description

DESCRIPTION provided by applicant Each year venous thromboembolism VTE affects up to million Americans and million people worldwide VTE patients have blood clots in the legs venous thrombosis that may travel to the lungs pulmonary embolism Up to of VTE patients die and the annual direct U S healthcare costs are $ billion For more than years anticoagulation has been the standard therapy for VTE Anticoagulation has many drawbacks it does not dissolve existing thrombi up to of patients develop post thrombotic symptoms pain swelling chronic sores up to of patients develop chronic thromboembolic pulmonary hypertension a severe cardiopulmonary disease it is linked to VTE recurrence in up to of patients it has significant bleeding risk and it has not been proven to save lives in a randomized clinical trial High doses of tissue plasminogen activator TPA like agents may prevent post thrombotic complications by dissolving clots but they are only partially successful cause bleeding and do not reduce mortality There is a need for a safer more effective therapy for VTE that saves lives reduces disability and lowers health care costs We Translational Sciences Inc TSI successfully completed a Phase I II STTR in which we discovered and developed a therapeutic antibody Lysimab that dissolves blood clots through a unique mechanism Through synergism Lysimab increases the potency safety and specificity of TPA and it avoids TPA related hemorrhage and neurotoxicity Following FDA recommendations in Phase I studies we successfully developed Lysimab into a stable clot dissolving therapeutic suitable for clinical trials Our Phase II STTR advanced Lysimab through preclinical studies toward human trials by demonstrating safe effective synergistic therapeutic dose combinations of Lysimab and TPA in vivo in a humanized pulmonary embolism model producing and purifying Lysimab under GLP conditions characterizing Lysimabandapos s human tissue binding char acteristics demonstrating Lysimabandapos s remarkable safety profile and pharmacokinetics in pivotal safety toxicology studies in a pharmacologically relevant species completing a successful FDA pre IND meeting raising strategic investment funds for clinical development and securing competitive selection by the NIH SMARTT regulatory team for FDA IND submission Building on this progress this Phase IIb proposal aims to complete a first in human Phase I study of the safety pharmacokinetics pharmacodynamics and biomarker efficacy of Lysimab Then we will leverage our pre clinical clinical data to form a strategic alliance with a Pharmaceutical partner to conduct later phase clinical trials for FDA approval of Lysimab We project that combination TPA Lysimab therapy could lead to the survival of an additional patients per year and a andgt reduction in post thrombotic symptoms and their associated costs Upon completion of this Phase IIb project and transfer of commercialization responsibilities to our strategic partner TSI will investigate the potential benefits of this platform technology to other patients with thrombotc diseases PUBLIC HEALTH RELEVANCE Venous thromboembolism affects about million people per year worldwide costing as much as $ billion per year in the U S alone Anticoagulation therapy is the standard of care for patients with venous thromboembolism but anticoagulation has serious risks and in more than of patients anticoagulation fails to prevent complications that lead to recurrent venous thromboembolism chronic pain and disability This Phase IIb proposal seeks to develop a novel therapy for venous thromboembolism that could markedly reduce death disability and healthcare costs