Haima Therapeutics LLC — Department of Health and Human Services SBIR Phase I: NHLBI

Description

PROJECT SUMMARY ABSTRACT The normal platelet count in human blood isLand these platelets are responsible for efficient hemostatic surveillance and bleeding mitigation in the bodyThrombocytopeniaTCPlow platelet countis a condition caused by a decrease in platelet productionincrease in platelet destruction or consumptionor increase in splenic sequestrationThe age adjusted prevalence of immune thrombocytopenic purpuraITPis estimated to beperpersons in the USAwhile drug induced TCP affectsperpersonsThe majorityandgtof TCP cases result from autoimmune disorders or are induced by chemoand radiotherapies as well as other pharmaceuticalsAt platelet counts andltLsurgical procedures are complicated by high bleeding risks and any minor injury may lead to excessive bleedingAt counts andltLspontaneous bleeding becomes a major concernThereforefrequent therapeutic and prophylactic platelet transfusions are required to increase platelet counts and mitigate bleeding risksHoweverthe availability of natural platelet products is severely limited by a near static pool of donors and a very short shelf lifedaysdue to high risks of bacterial contamination and storage related platelet activation and degranulationThereforethere exists a significant unmet clinical need for a therapeutic agent that can mitigate bleeding complications intravenously in TCP patientswhile allowing reduced contamination and long shelf life without compromising functionTo this endwe have developed a synthetic hemostatic nanotechnologycalled SynthoPlateTMthat mimics platelet s ability to adhere and aggregate specifically at a site of injury in the bodyThis technology has demonstrated platelet mimetic hemostatic functions in vitro as well as in vivo in proof of concept murine and porcine models of bleedingBuilding on this preliminary datathis proposal aims to establish the minimum and maximum effective doses of SynthoPlateTM needed to enhance clotting properties in vitro and mitigate bleeding in vivo in a mouse model of TCPSuccess with the aims of this Phase I SBIR proposal will establish SynthoPlateTM as an innovative intravenously transfusable technology to mitigate bleeding complications in TCPFurthermoresuccessful therapeutic demonstration with SynthoPlateTM in TCP mice will allow subsequent dosingsafetytoxicity and efficacy studies with GMP manufactured SynthoPlate in large animal modelsthus allowing IND enabling studies towards clinical developmentenvisioned in Phase II PROJECT NARRATIVE Thrombocytopenia is a condition characterized by low platelet countspatients with thrombocytopenia are at risk of excessiveuncontrollable bleedingThe current standard of care is transfusion with donor platelet products to mitigate bleeding risksHoweverdonor platelets are expensivecarry significant risk of infectionshave variable hemostatic efficacyand have very short shelf lifedaysOur innovative SynthoPlateTM technology can mimic platelet s hemostatic functions while providing advantages of large scale production and sterilizability to reduce contamination risk and ensure long shelf life