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Biotech Stock Mailbag: Exact Sciences, Galena, Titan, Arena, Breakthrough Therapy

Retail investors own the biggest slice of Galena so I expect the stock's rise and fall will correlate directly with the volume of published stories on sites like Seeking Alpha. [If you're curious, I count 21 stories on Galena published by Seeking Alpha contributors since the beginning of the year -- almost all bullish, naturally.] This trend will end badly for Galena bulls when the NeuVax phase III study fails.

One more note on Galena: The acquisition of U.S. marketing rights to Abstral, a sublingual fentanyl tablet for breakthrough cancer pain, was a dumb move. Abstral is no better than any of the cheap, generic fentanyl products available for use by doctors here. Galena not only went into debt to buy Abstral, but it will now waste even more money trying to sell the product -- unsuccessfully.

Tom B. asks, "What is your take on the FDA's breakthrough therapy designation?

I snarked on Twitter recently that this year's hottest FDA badge of honor -- breakthrough therapy -- was in danger of becoming as irrelevant as the now-derided fast-track status. I hope I'm wrong but the tangible benefits of breakthrough therapy status are unclear because FDA hasn't bothered to tell us what makes it so special. Right now, breakthrough designation is a shiny crown that drug companies want to wear. It elicits oohs and aahs from investors, but that's about it.

Here's how FDA describes breakthrough therapy designation:

Breakthrough therapy designation is intended to expedite the development and review of drugs for serious or life-threatening conditions. The criteria for breakthrough therapy designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy. A breakthrough therapy designation conveys all of the fast track program features (see below for more details on fast track designation), as well as more intensive FDA guidance on an efficient drug development program. The FDA also has an organizational commitment to involve senior management in such guidance. 

"Preliminary clinical evidence" isn't defined, nor is "substantial improvement" but I guess it's like porn, you know it when you see it.

The two primary drug-review branches of FDA have received 50 applications for breakthrough therapy designation from July 2012 through March 31, 2013. Ten drugs were granted the designation, 12 were denied during this period. Presumably, the remaining applications are still under review.

Three breakthrough designations were awarded to the leukemia/lymphoma drug ibrutinib that's being developed by Pharmacyclics (PCYC) and Johnson & Johnson (JNJ). Why the same drug requires three different breakthrough crowns is unclear. Other "winners" disclosed so far include Vertex Pharmaceuticals (VRTX), Pfizer (PFE) and Novartis (NVS).

I don't mean to be totally derisive of breakthrough therapy status. I'm just waiting to see if the new program actually accelerates drug development and approvals. Ibrutinib, for instance, is already in phase III studies, so how much will breakthrough status change timelines, compared to the "old" accelerated approval pathway? And if we rush drugs through shortened clinical trials based on preliminary data, will drug developers and FDA miss important safety findings?
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