Risks and Rewards of Using Surrogates to Accelerate Commercialization
Executive Summary
Surrogate endpoints, including biomarkers, are a great idea, a legitimate way to speed drug development and thus lower cost and risk. But their use is also problematic from both scientific and, especially, regulatory points of view. Still, the investment proposition is too compelling to ignore, and companies such as KAI Pharmaceuticals, HaptoGuard, and Perlegen are using them in various ways to find drugs, speed their development, or make their eventual marketing more effective.
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KAI Pharmaceuticals Inc.
KAI Pharmaceuticals is the result of one researcher's accumulated wealth of knowledge about the protein kinase C (PKC) family, which plays is involved in such activities as regulation of gene expression, response to ischemic insult, and regulation of hormone responses and ion channels. KAI is investigating the role of PKC in heart function, acute ischemic stroke, heart failure, pain, diabetes and other indications. The company's first drug, a delta PKC inhibitor (KAI-9803), is designed to limit injury during reperfusion by protecting vulnerable tissue, with an initial indication for post-myocardial infarction. It is just entering Phase I/IIa clinical trials. A second indication for limiting reperfusion injury in stroke is in active preclinical stages.