Replies to post #126 on Biotech Values

[DewDiligence]

More on dosing/scheduling:

>> It appears that the 25 mg dose is more than enough, so a dosing regimen would probably not be needed for the study.<<

We have agreement on this key point! I feel more confident in this assertion knowing that we are on the same wavelength.

My hunch is that GENR could even go lower than 25mg/m2 and still get efficacy in AMD. However, there is no need to go lower because the 25mg dose is well-tolerated. Moreover, the animal studies on Squalamine incorporated the 25mg dose, so it is advantageous to preserve the relevance of this animal data for the eventual NDA package.

>> In the upcoming phase 2 it seems that they should try a maintenance dose of one month for some patients, and 2 months for another group, of course the people getting the maintenance every 2 months would get a placebo, at the in -between months. <<

Such a design would produce a useful comparison provided that: 1) The trial is fairly large; 2) The patients are assigned randomly into the two arms; 3) The evaluators are blinded to the treatment assigned to a given patient. These design features (especially the first one) make for a more expensive trial, and that is the reason I remain skeptical that GENR will embark on such a study without a partner.

>> I have the feeling that as you say, they could probably get by, with the every 2 month dosing and still beat the competition, but WHAT IF the monthly maintenance improves vision TREMENDOUSLY. Wouldn't it then be worth doing? <<

If the partner pays the bills, absolutely yes. However, if GENR were to embark on such a trial solo, it could be interpreted as a signal that Squalamine’s much touted long intracellular half-life might not be long enough after all.

I’m going to go out on a limb and say that the probability is at least 80% that GENR inks a partnership deal before the first patient in any new Squalamine trial is enrolled. Feedback welcomed.