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KAI Pharmaceuticals Inc.

This article was originally published in Start Up

Executive Summary

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.

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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.

Risks and Rewards of Using Surrogates to Accelerate Commercialization

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.

Small Steps Forward in Treating Ischemic Stroke

Stroke remains a devastating disease that isn't adequately addressed, but the pharmaceutical industry remains wary of the field, having been stung in the past by extremely expensive clinical disappointments. Nevertheless, the opportunity is too enticing for the industry to ignore completely. Two pharma-sponsored major clinical trials are underway for treatment of acute ischemic stroke, foro example. The emerging companies with stroke therapies profiled here, however, are cautiously pushing forward on several fronts, of which the stroke market may or not be the first and foremost application of core technology.

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