GINER INC — Department of Health and Human Services SBIR Phase II: 200

Description

DESCRIPTION provided by applicant Pancreatic islet transplantation ITx is an emerging treatment for a subset of Type diabetics for which standard insulin therapies are insufficient I is a promising alternative to pancreas transplantation since ITx has significantly less surgical risk and a less toxic immunosuppression regimen Enhanced organ preservation via the proposed technology has the potential to increase the number of lifesaving ITx while improving outcomes There have been infusions in recipients performed in North America most of them in Canada and in recipients in Europe ITx is an approved standard of care in Canada the UK and a number of European countries In the US islet centers are preparing to file for FDA biologics licenses based on the favorable outcomes in the North American clinical trials once those are granted the numbers of US ITx are expected to increase substantially The Giner Portable Pancreas Preservation System P S will be the first complete portable commercial system utilizing the persufflation organ preservation technique The patent pending technology produces oxygen by electrochemical generation The system supplies oxygen via gaseous perfusion andquot persufflationandquot in which humid gas containing oxygen is flowed through the vasculature of the cold stored organ to minimize oxygen deprivation time Persufflation enhances preservation of the pancreas as well as other organs In the Phase I II program after development and fabrication portable persufflation units have been in preclinical and clinical use at seven facilities The units have been utilized in over procedures and over hours of operation including during seven airplane flights The P S has been approved for routine use on Air Canada In preclinical testing for hour preservation the human pancreatic islet yield was higher pandlt relative to the current state of the art preservation method at hours this higher yield would theoretically translate to of current ITx recipients being insulin free with a single ITx and increase the fraction of transplantable islet preparations from to There is also evidence of enhanced islet quality based on insulin secretion assays A Canadian clinical trial at University of Alberta Edmonton started in October using the P S Four of the first four pancreatic persufflated in the pilot trial resulted in transplantable islet preparations compared to their historical standard of transplantable preparations also there was a significant increase in viability as measured by OCR DNA at the day of transplant compared to historical control Two of the three transplanted preparations have resulted in insulin independence for patients at months post transplant These early results are extremely encouraging and confirm our preclinical Phase II results The goal of the Phase IIB program is to advance the technology to clinical implementation At the end of Ph IIB we expect to have a medical device that is ready for filing for K clearance in US and US clinical investigational use and to be sold commercially internationally in Canada Europe and Australia with a CE Mark The Ph IIB has aims design and production of pilot devices meeting international medical device standards preclinical testing with evaluation of islets from research grade human pancreatic and support of clinical testing study already approved and pilot study initiated with ITx in Canada PUBLIC HEALTH RELEVANCE Pancreatic islet transplantation ITx is an emerging treatment for a subset of Type diabetics for which standard insulin therapies are insufficient It is a promising alternative to pancreas transplantation since ITx has significantly less surgical risk and a less toxic immunosuppression regiment Enhanced organ preservation via the Giner Portable Pancreas Preservation System has the potential to significantly increase the number of lifesaving ITx while improving outcomes