Replies to post #455016 on Anavex Life Sciences Corp (AVXL)

[Investor2014]

Reading this Q&A from the Q1 2024 cc, it seems more than likely a new Rett trial will be required. Although of course Missling does, as he should, leave open the possibility of approval based on completed Rett trials and OLE data. Hard to assign chances of approval in rare indications, but I would say perhaps 25% chance of some kind of approval - perhaps not from the FDA first, as there was no EXCELLENCE patients enrolled in the U.S.

Soumit Roy: I’ll go switching to the Rett program. Two questions. One is, if you can provide us some kind of detail on there was a difference between your trial and Acadia’s trial. The reduction in the – even the placebo arm or the treatment arm is much pronounced in your trial. Does that mean the baseline characteristic was very different in the enrolled patient? And the second is you saw a quick early four week, possibly a clinical benefit, but then it kind of waned away at the 12-week time point. You see as a pharmacokinetics or a dosing issue.

Christopher Missling: No. I think if I can start with the last part, there’s not such a thing actually the trial was really showing an extremely nice improvement score in the active arm with over minus 12.9 in our analysis, which is extremely strong. And trofinetide showed only an improvement in the active arm of minus 5.1. So we showed a stronger signal in the active arm. What happened was in our trial, the placebo also improved and in the trofinetide the placebo did not improve. So that is basically really the difference though the standard error. So the variability of the scores was much higher in our trial than in a trofinetide trial. And we laid out several factors why that is possible and we now understand exactly what happened and we can factor that in, in the explanation very, very nicely.

So it’s really like the variability of the trial, the noise, if you like was much higher in our trial. And let’s not forget, we had a smaller study. We also had a Phase 2/3 was not a pivotal study, Phase 3. And also we had just a two to one randomization with 60 patient or 62 in the active arm and 30 in the placebo arm. And these 30 patients, if a few of them are just basically very noisy, that could derail and increase the noise in the trial and increase the standard error, which we saw in our trial.

Soumit Roy: So should we expect that rate [Rett] still being kind of one of the primary focuses for the company? And maybe one path forward would be to start a fresh pivotal trial with 180 patient, one-to-one randomized, kind of the Acadia size trial.

Christopher Missling: That is a very good point, and it’s exactly a possibility. And again, we would not do that, however, without further get regulatory input, because we might get some feedback, which could be very favorable. But indeed, that is something which could be done very easily. It’s a 12-week study, it’s not too long. And we have, again, a strong interest from the community, given the strong interest, and to continue to stay on study drug and patients in Rett syndrome from our program have been now on this drug for over four years – up to four years. That really shows a very high sticky interest to keep our drug and not to switch to anything else in the meantime.

[crescentmotor]

First that $121 million is supposed to be enough to last 4 years assuming no additional income.

AVXL certainly hasn't made that statement. Here is the most current guidance: "Management believes that the current working capital position will be sufficient to meet the Company’s working capital requirements beyond the next 12 months after the date that these condensed consolidated interim financial statements are issued." If the EMA or the FDA were to require additional trials involving Rett Syndrome or 2-73 for AD, the $121 million would become vastly inadequate in short order.

Second Anavex has been able to sell shares via the ATM and there is no reason to think that won't continue as a way to raise money as needed.

You're maintaining that significant dilution selling shares at $4.50 constitutes a viable/justifiable way to raise money? I don't think so.

Forth Anavex is not in a shaky position.

Until and unless regulatory approval from either the EMA or the FDA within a year or so for AD appears likely, I'll stick with my opinion that AVXL is in a shaky position.

Then there is FTD and PD trials to get started.

Sure--expecting that soon? I think not.

[LakeshoreLeo1953]

$150M was deemed sufficient when Quarterly trips to Market
replenished that balance at a SP in excess of $10.

In light of future trials being a reality and Q dilution an idiotic alternative
other than meeting payroll, I'm not sure there is any of your argument I can support.

I am not conversant in emoji speak so Bas et al you are entertaining yourselves.

[WilliamMunny]

Steady, mentioning as you did at the end of your post the long awaited PD trial, my last communication with an executive at the Shake It Up Australia Foundation about a month ago confirmed that they are still fully expecting that trial to commence this year. While the person said they were encouraging patients interested in this trial (or others, I'm sure) to register on their website so they will be available for recruitment when the trial is about to begin, I don't really take that statement to mean the trial is imminent.