The business of Protara is biomarker discovery, validation and measurement. The primary focus is on developing a panel of urine biomarkers assays that will be measured in rodent urine to predict renal toxicity as a service to the pharmaceutical industry. The cost of development of a new drug is estimated to be $1.8 billion. Determining the extent of renal toxicity for a new drug is difficult and may not occur until late in the development cycle. Urine biomarker assays are used by the industry to facilitate the identification of nephrotoxicity but they cannot differentiate between different types of injury and could be more effectively run as an outsourced service. Protara will develop a multiplexed panel of assays to measure traditional urine biomarkers. In addition, we have an agreement to license 21 novel urine biomarkers which appear superior to current biomarkers at predicting acute kidney injury which will be included in the panel. The assay panel will be superior to currently available panels because it will identify the region of the kidney injured, the subcellular organelles injured, the magnitude of the injury and correlate the injury with that caused by other known nephrotoxins.
Purpose of Product. Protara will measure a multiplexed panel of urine biomarker proteins in rats and mice that have been administered drugs that are in the preclinical development stage. Data from the measurements and interpretation of the data regarding the ability of the drug to cause renal failure will be returned.
Features and Benefits. The multiplexed assay will be larger than any that are currently available, will contain proteins for which the concentration is known to change during acute kidney injury and for which we have protected IP. The assays will be more accurate than current assays since they use measurement by mass spectrometry with internal standards. Finally, the product will allow the companies to outsource the measurement of these proteins which is more efficient.
Stage of Development. The company is in the very early development stage. The IP for the biomarkers has been filed by the Medical University of South Carolina and a letter ensuring access to the IP by the inventors has been obtained. An SBIR application was submitted for a phase I study in April 2012 and we plan to resubmit this proposal in December 2012. The members of the company have developed assays for some of the proteins of interest as part of academic work at MUSC but no rodent assays have yet been developed.
Product Limitations. Success of the business will depend on development of the assays, proving that they are able to predict AKI and convincing the pharmaceutical industry to utilize them. Development of the assays is not likely to be a major obstacle since we have developed similar assays for humans without difficulty. Measurement of a subgroup of the assays is already currently done during evaluation of some pharmaceuticals and a panel of 7 proteins that will be included in our panel has been advanced by the Predictive Safety Testing Consortium and approved by the FDA for preclinical evaluation of AKI. Therefore, our panel should be at least as good as the current assays. Our data suggests that many of the new markers for which we will have a protected IP will be better than the current markers. A potential hurdle may be to get general acceptance of this panel by the industry.
Liability. There is a low risk of liability resulting from the measurement of biomarkers. The results will be interpreted according to our in-house results and the limitations of those interpretations will be noted in contracts. Action on the results will be at the discretion of the customer. There is however a potential liability associated with interfering with patents for some of the “traditional” biomarkers we propose to measure. For instance the use of KIM-1 and NGAL as urine biomarkers have been patented.. Potential work-arounds for this are licensing the right to measure these markers, not using these markers in the panel, or measuring the proteins but not interpreting them as renal biomarkers.
Production. The measurement of the assays will require a triple quad mass spectrometer, chromatography equipment and appropriate software. The mass spectrometer will be leased when the SBIR grant is received.
Facilities. Wet lab space in the Charleston Innovation Center, a Charleston incubator will be used. The incubator was established by the South Carolina Research Authority in collaboration with the Medical University of South Carolina.