KAI Pharmaceuticals
Based: South San Francisco, CA Why It's Fierce: Much of KAI's focus right now is on KAI-9803. The company recently inked a $320 million global licensing deal with Daiichi Sankyo ($20 million up front) for two initial indications. The compound is designed to inhibit the specific PKC enzyme responsible for reperfusion injury. The drug is currently in a 150-patient Phase I/II and was fast-tracked by the FDA. Heart attack patients headed for emergency angioplasty face two critical waves of damage: the first when the heart muscle is starved for oxygen and the second when doctors reestablish blood flow. At that point, reperfusion injury triggers damage to the myocardium. "What's been shown through a variety of preclinical models," said CEO Steve James, "is that when you have reperfusion injury, delta PKC is activated and translocates. Delta PKC sets up a cascade of cell death and necrosis. If you inhibit it, you prevent reperfusion injury at its start." KAI believes that an intracoronary injection of KAI-9803 just prior to deflating the balloon used in angioplasty may prove to be an effective way to protect patients. The technology at KAI was first developed by Dr. Daria Mochly-Rosen at Stanford. She designed small peptides that inhibited pre-receptor interaction and preclinical studies have garnered excellent results. At the urging of the FDA, the clinical trial is aimed at six or seven surrogate endpoints. The ongoing clinical trial should deliver results in the late summer or early fall. Depending on its outcome, KAI has the opportunity of setting up a larger Phase II or perhaps go right into a pivotal trial. When KAI set up its licensing deal, it retained co-promotion rights for acute care and hospital settings. If the therapy progresses as planned, KAI would set up a specialty marketing arm with a small, targeted sales force that could pinpoint acute and specialty audiences. A second therapy–KAI-1455–is being studied as a therapy for protecting tissue against ischemia. There's a "high degree of evidence that activating epsilon PKC is protective. You can't predict your own heart attack, but there are a number of surgical procedures where you block blood flow and cause ischemic damage." What to look for: A Series B for $30 million to $40 million later this year with the chance of going public in the next 18 months to 24 months. |
