Biotech Values

[DewDiligence]

CC highlights:

First of all, there were many questions on the call from both sell-side and buy-side people –so many that they ran out of time with people still in the queue. This is a much higher level of interest than I have seen before for this company.

Regarding the trial-design details, there are several notable points:

1.The variable dosing of 25 and 50mg/m2 used in the phase-1/2 trial has been converted to fixed dosing at 20 and 40mg, irrespective of body size. Since an average-sized patient measures about 1.7m2, the new fixed dose of 40mg is comparable to the old variable dose of 25mg/m2, and the new fixed dose of 20mg is comparable to only half of the 25mg/m2 dose used in the phase-1/2 trials.

Being able to lower the dose so substantially in the new trials is a good sign; it shows that Squalamine is expected to have strong efficacy at much lower doses than previously thought.

2. Patient enrollment is expected to occur rapidly because the trials will not be unduly restricted by disease subgroups (predominantly-classic, minimally-classic, or occult) or by lesion size. The 100-patient trial will enroll up to 10 patients per site at “a few dozen sites,” all of them in the U.S. (Additional details are being withheld for competitive reasons.)

3. Plans are in place to manufacture sufficient amounts of Squalamine for all clinical trials, and the process is scalable for commercial production. (The identity of the contract manufacturer(s) is not being disclosed for competitive reasons.) In other words, manufacturing should not be a limiting factor in the development timeline.

4. One unanswered question is whether the so-called “209” trial (the phase-2 trial with 100 patients) can serve as one of the two “well controlled” trials the FDA requires for marketing approval. This is the approach that Alcon is using with Retaane, so I think there’s a decent possibility that GENR could benefit similarly. If the FDA agrees, then only one rather than two phase-3 trials will be needed to submit an NDA.

--
In terms of the costs, Dr. Levitt stated that GENR has the funds already in hand to finish all of the phase-2 trials and to get part way into phase-3. In other words, it’s unlikely that there will be any financings anytime soon (see #msg-2909211).

Overall, this was a very satisfying conference call. GENR has finally graduated to the big leagues in the AMD sector.