"These days Markey looks at every company from that same perspective. He wants to understand, first, the science then the unmet need next.
That’s how he’s arrived on his best idea, MannKind (NASDAQ: MNKD) with its inhalable insulin product called Afrezza (human insulin of recombinant DNA [rDNA] origin). The company has seen its share of controversy and debate by the investment community, and in its existence, the company has been issued two complete response letters (CRLs) by the FDA which amounted to big setbacks along the way. After developing the product with initial inhaling device, the company had to start over, this time with a smaller palm-sized inhaler that can be used discretely at the dinner table with a brief hand-over-mouth maneuver. Patient friendliness and expediency were a major part of the rationale for making the change, but FDA wanted to see this product work, not in a small trial, but in a great big one to see if data would yield the same bioequivalence as the original device. In short, it’s not only about top-line glucose control, but also about knowing that the insulin and the delivery device together will lower hemoglobin A1c (A1c) levels enough to satisfy regulators. A1c measurement is vital because it represents how well glucose levels have been maintained over prior weeks, not just today. Now we’re waiting results of two phase 3 clinical trials, the Affinity 1 for type 1 and Affinity 2 for type 2 diabetes. Both sets of data will be delivered in mid-summer and if the results are what Markey expects, the new drug application will be filed in early October.
MannKind founder and CEO Al Mann is passionate about diabetes. He told me back in 1998 when we were discussing his insulin pump company MiniMed which would later be acquired by Medtronic, “I don’t work for money anymore.” He continued, “I’m giving away my money.” Indeed, he gave $112.5 million to USC, and he was in process of giving a similar amount to UCLA to further diabetes research. Mann is obsessive about glycemic control because he has seen the cause-effect result of uncontrolled blood glucose on cardiovascular disease. The point is, Mann stands ready to fund MannKind’s Afrezza to completion, whatever its needs. He has said recently that the company plans to charge about $2,000 ($2K) per year per patient for Afrezza. There are 19 million (19M) diagnosed diabetics in the U.S. alone. Imagine better blood glucose management and no multiple daily needle sticks. You can do the fourth-grade math yourself to see the market opportunity."
http://beta.fool.com/streetwisereport/2013/04/01/three-outside-the-box-biotechs/28437/
