GTC Biotherapeutics (fka GTCB)

[DewDiligence]

Possible / Probable News Flow in 2008-2009

[Updated for disclosures on 2Q08 CC, of
which the most important is the slippage
in the timeline of Leo’s DIC trial.]


1. FDA acceptance of the ATryn BLA for review, expected in late September or early October (45-60 days from the BLA submission date on 8/7/08). The relevance of the acceptance-for-review date is this is when the FDA decides whether to grant a Priority Review (which shortens the review time from 10 months to 6 months).

2. One or more FoB partnerships. There are about half a dozen monoclonal antibodies with FoB potential in which GTC has already developed founder animals. GTC claims to have spoken to prospective FoB partners and hence a deal could come at any time.

3. Results from the phase-2 ATryn study in DIC. The latest guidance from GTC’s partner, Leo Pharma, is for reporting of data in 2H09. (This is a slip relative to the prior guidance of 1H09.) This study will be closely watched by Ovation Pharma and the investment community because DIC addresses a much larger market than antithrombin hereditary deficiency.

4. A partnership for ATryn in Japan. Japan has substantial sales of plasma-derived antithrombin, including an approved indication for DIC/sepsis. These plasma-derived sales represent low-hanging fruit for eventual conversion to ATryn. At least one Japanese clinical study will be needed to obtain marketing approval. Dr. Cox has said that a deal would be more likely in 2009 than in 2008.

5. A development partner for the CD137 oncology program. This would be significant because CD137 is the most advanced internal program that is not based on a plasma protein. However, GTC has given no guidance about when a CD137 partnership deal might be struck.


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