Replies to post #9027 on Biotech Values

[rstor1]

+++. But, I try to put myself in the shoes of an EMEA commissioner facing this decision regarding the world's first approval of a transgenic therapeutic protein. Around the table are some arguments pro and against immediate approval. Then information is made public that GTCB plans to do a very similar study this year to suit the FDA. Wouldn't it be understandable for this commissioner to at least ask for details on that study and perhaps take the expedient position that this decision should be postponed until the US study is completed?+++

Terrific insight, and sounds quite plausible.

I know of an analogous situation. ONCY's drug candidate is a replication-competent oncovirus - also a touchy PR situation, especially with the bad rap that gene therapy has received. They applied for an IND with both the FDA and Health Canada about three years ago. HC approved the start of PI clinicals in glioma relatively soon. The FDA said nothing for three years. When I asked the CEO about the delay with the US trial, he said that the FDA wanted to wait for definitive safety results from the HC trial before giving the go-ahead (which came a couple of weeks ago.)

So it may be that a conservative CYA approach is to wait to see what the other guys do.

What's the most optimistic timeline on the US trial, Dew?

[DewDiligence]

Urche re GTCB:

I give you credit for deep thinking on this matter, but I reject your “let’s wait a couple of years to see what the FDA says” scenario as far-fetched.

Let’s bear in mind that the risks of plasma-derived antithrombin are very real and a far more serious concern in Europe than in the U.S. Why is this? Because plasma-derived antithrombin is widely used in Europe (to the tune of about $200M annually), while it is rarely used in the U.S. due to a lack of supply for the one FDA-approved product (Bayer’s Thrombate).

Now, let’s move on to the probability that the EMEA asks for a new clinical trial. You stated that this would be “unprofessional,” and I think any reasonable person would concur. However, I think we do need to allow for the possibility that the reviewers simply changed their minds about the two cases of suspected, non-clinical DVT. Although GTCB submitted the ATryn application using protocol assistance from the EMEA, this framework is not as rigidly defined as the FDA’s Special Protocol Assessment.

I, too, think this “more clinical data” scenario is unlikely, but not as unlikely as your 10% figure. Hence my 25% number, which may be high, but I like to be conservative for valuation purposes.

My remaining 75% probability comes from EMEA concerns relating to product purification/characterization. Although it may take some non-trivial effort, these kinds of issues ought to be resolvable by GTCB with a little time. JMHO, FWIW