After being dogged by regulatory setbacks and demands for new trials, MannKind says it gathered clearly positive late-stage data on its inhaled insulin Afrezza from a pair of Phase III studies on Type 1 and Type 2 diabetes.
Investigators reported that the powder formulation inhaled through the company's "Dreamboat" device provided superior reductions in A1c levels, allowed more patients to reach blood glucose target levels and reduced postprandial glucose excursions with no significant difference in the incidence of severe hypoglycemia among Type 2 diabetes patients. In the Type 1 study, the therapy proved noninferior to insulin aspart for decreases in A1c levels, triggered fewer cases of hypoglycemia, significantly improved blood sugar levels and offered an advantage for spurring weight loss.
Cowen's Simos Simeonidis notes that barring any nasty surprises, "we assume that the FDA should approve Afrezza the third time around, sometime in mid-2014. So, assuming it is approved, the question becomes 1) can MannKind find a partner and 2) how much would Afrezza be used?"
MannKind ($MNKD) was forced to go back to the drawing board more than two years ago after a hesitant FDA issued its second complete response letter on Afrezza and the new inhaler. CEO Al Mann had to raise $530 million in debt to back the latest Phase III studies and set up a commercialization group, bringing his total gamble on this product to nearly $1.5 billion.
Mann insisted on sticking it out years after Pfizer ($PFE) and Eli Lilly ($LLY) had thrown in the towel on their own inhaled insulin products, both expensive failures, and a number of analysts remain skeptical about Afrezza's future. Also, no Big Pharma company has been willing to step in and partner on a product that hopes to revolutionize the way insulin is provided to millions of patients, adding to the doubts about its potential.
That cloud of uncertainty may have affected the market response to the results. MannKind's shares--which have been rising steadily over the past 6 months--soared more than 30% in early trading, but by midmorning were up only 14%.
But today, everyone at MannKind was celebrating the results.
"We are pleased that both Phase III studies met their primary efficacy endpoints and that Study 171 also achieved its other main objective of demonstrating comparability between the Gen2 and MedTone inhalers," said Mann in a statement. "The positive results of Study 171 and Study 175 will form the basis of an amendment to our new drug application for Afrezza, which we expect to submit to the Food and Drug Administration early in the fourth quarter of this year."
The FDA has been a tough taskmaster on diabetes treatments, raising the bar several years ago on safety and efficacy reviews. And the agency will take another close look at Afrezza before allowing it on the market.
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