Biotech Values

[DewDiligence]

Elaborating on Urche’s post, here is some additional color on the CC:

1. The EMEA’s ATryn questions fall largely in two areas: a) The robustness of GTC’s purification process; and b) GTC’s ability to demonstrate that ATryn does not produce patient antibodies. (The latter concern is very much on regulators minds because of the recent mishap with JNJ’s Eprex brand of erythropoietin.) GTC is highly confident in both of these areas and they are not expecting to run new clinical trials.

2. EU ATryn timeline. The expected EMEA decision date in October takes into account that much of Europe goes on vacation during August. If the EMEA issues a positive opinion by the end of October, the EU itself is expected to approve ATryn for marketing by the end of 2005 (following the normal 2-3 months to adopt the EMEA’s opinion), allowing for a product launch in early 2006.

3. GTC expects to announce a European partnership deal for ATryn before the approval date. A primary goal of such a partnership will be to obtain financial support for expansion of ATryn beyond the HD indication.

4. The U.S. ATryn trial will begin enrolling patients in the current quarter. There will be 15-20 clinical sites and control-arm historical cases will be taken from both the U.S. and Europe. Enrollment is expected to take approximately one year, allowing for a BLA submission in late 2006 and an FDA approval in late 2007.

5. AAT program. GTC sees an eventual market size comparable to that for ATryn (including non-hereditary indications): $500-700M in annual worldwide sales. GTC is investigating both IV and pulmonary delivery.

6. Two sexually mature goats have been produced for GTC’s malaria program.

7. No update on the albumin program -- it sounds like this program has been moved to the back burner. (This was the one disappointing aspect of the CC for me.)

8. Cash is not a near-term problem (#msg-6175078). GTC has deferred the planned $5M for ATryn manufacturing expansion until closer to the launch date.

9. Present on the CC (aside from Urche and Dew): Phil Nadeau/SG Cowen and Jim Kennedy/Marathon Capital.