+++but I do not think the trials will necessarily be larger than those for Macugen and Lucentis.+++
If you are dealing with a systemic administration for a non-life-threatening indication, the FDA will want extensive safety data, including enough patients to show even rare complications.
I'm thinking that appropriately-sized trials would be similar to ones for diabetes. So, IMHO, you are probably looking for patients in the thousands (3-5?) rather than in the hundreds (Macu ~1200, Lucentis ~700). It is hard for me to put a price to a study of that size, but I would guess that the minimum is probably in the 50-100 mil range. And if a second PIII is required, you can double the amount. That's a big risk, and it will need to be mitigated by a reduction of the up-front money.
If Levitt was thinking that AMD would provide the near-term survival money, while the drug was being developed by the partner, he realized that alternative funds would have to be obtained, hence the financing. That need for cash is compunded by the small milestone from MEDI.
Even with a US blinded PII, if my analysis is correct, the company will not get a large up-front, because the safety issues will only be covered in a PIII.
This is all conjecture, obviously. But if we follow it through, I think that the conclusion we reach is that an in-house PII is unwise, and that he should get the best deal offered. It will be heavily back-ended, but if the costs of the PII are avoided, and the CF foundation carries the bulk of the Lomucin costs, the company is in good financial shape, and can pursue the cancer indications internally.
Thougts?
Bob
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