Human Apyrase Therapy for Acute Ischemic Stroke

Award Information
Agency: Department of Health and Human Services
Branch: National Institutes of Health
Contract: 2SB1NS060175-04
Agency Tracking Number: SB1NS060175
Amount: $2,736,786.00
Phase: Phase I
Program: SBIR
Solicitation Topic Code: 105
Solicitation Number: PAR16-027
Solicitation Year: 2016
Award Year: 2016
Award Start Date (Proposal Award Date): 2016-08-01
Award End Date (Contract End Date): 2019-07-31
Small Business Information
1005 N WARSON RD, Saint Louis, MO, 63132-2900
DUNS: 192266141
HUBZone Owned: N
Woman Owned: N
Socially and Economically Disadvantaged: N
Principal Investigator
 (312) 339-8455
Business Contact
Phone: (312) 339-8455
Research Institution
Principal Investigator Program Director Last First Middle Chen Ridong Abstract There remains a crucial unmet medical need in acute ischemic stroke AIS treatment Approximately of patients die within one year and are permanently disabled making AIS the fourth leading cause of death and the leading cause of adult disability in the USA with an estimated cost range of $ billion annually Recombinant tissue plasminogen activator rt PA is the only drug approved by the FDA for restoring cerebral blood flow and improving patientandapos s functional outcome with no survival improvement Currently only of AIS patients receive rt PA as its use is limited by narrow time window of administration up to hours post symptom and fold increased intracranial hemorrhage Recently endovascular treatment with retrievable stents has been shown to improve the outcomes in small subpopulation of patients with proximal vessel occlusion The failures of numerous neuroprotective therapies in clinical trials suggest that neuroprotection alone without restoration of tissue perfusion may not be adequate for treatment of stroke Thus developing the combinational therapy of rt PA and or retrievable stents with human apyrase as a safe adjunctive antithrombotic that also will attenuate reperfusion and rt PA related hemorrhagic transformation will be of great importance for stroke patients that promises to extend the time window and improve clinical outcome and survival APT is a proprietary and optimized human apyrase which is a homolog of CD Administration of multi functional APT scavenges pro thrombotic ADP and pro inflammatory ATP which are both released at sites of thrombosis and injury Hence co administration of APT with rt PA will prevent thrombotic re occlusion and maintain vascular integrity necessary to prevent hemorrhagic transformation The efficacy and safety of APT in combination with rt PA has been demonstrated in the clinically relevant models of thrombo embolic stroke adult and aged animals in two independent laboratories as recommended by the Stroke Therapy Academic Industry Roundtable funded by a Phase II SBIR grant R NS Therefore this CRP project is well positioned to successfully advance APT to IND Specifically we propose Develop APT production process and validate IND enabling study plan with FDA Establish protocols for manufacturing cGMP Current Good Manufacturing Practice drug product for toxicology and IND filing Evaluate APT for safety in nonclinical toxicology studies and Prepare and file IND application With the successful filing of IND we will perform Phase I clinical trials of APT to obtain safety pharmacokinetic and pharmacodynamic biomarker data in healthy volunteers and then Phase II and III trials for stroke patients The long term goal of this project is to market APT as combination treatment with r tPA to provide a highly effective and safe acute therapy for AIS with at least a h treatment window This treatment regimen could be used by emergency room physicians and even in a mobile stroke unit to achieve faster prehospital treatment with rt PA for over of AIS patients to improve functional outcomes as well as survival a dramatic increase from the of AIS patients currently treated with r tPA which does not provide survival benefit Principal Investigator Program Director Last First Middle Chen Ridong Narrative The efficacy and safety of combining an optimized human apyrase with r tPA has been demonstrated unequivocally in several relevant models of thrombo embolic stroke including in the stroke model of aged rats With a strong interdisciplinary drug development team we propose to complete activities necessary to enable IND filing

* Information listed above is at the time of submission. *

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