Anavex Life Sciences Corp (AVXL)

[MayoMobile]

Anavex Fiscal 2021 4th Quarter Conference Call
For those who haven't had the chance to listen over the holiday.

Question from Cantor: How do you feel about the expected AVATAR/EXCELLENCE affect sizes when you contrast against the RS-001 adult low dose trial?
A: We had large effect sizes for RS-001. The effect sizes were larger than 1 (very large) and we anticipate we may see higher in AVATAR as dosing is higher in AVATAR. EXCELLENCE patient population was increased for additional power at different age brackets at request by regulators.

More info on Anavex effect size: https://investorshub.advfn.com/boards/read_msg.aspx?message_id=165324944

Question from Cantor: Why were the primary and secondary endpoints changed for EXCELLENCE?
A: RSBQ is an in-depth 45 question endpoint which is quite rigorous. CGI-I has lower reliability than RSBQ, but we left it in.

Question from Cantor: Regarding the PDD program, have you met with regulators to discuss?
A: We are planning to do that and are currently doing so with the Parkinson’s foundation.

“We are still including which we have not yet reported, the total gene analysis of the PD study. So that means we’ve not only looked at the S1R gene expression changes in mRNA, but also the gene expression of all participants in the active arm, as well as the placebo arm and believe that additional intelligence can be drawn out of that to make an informed design of a study”

I found Missling’s response to this quite entertaining as it almost seems to have been taken from this speculative post: https://investorshub.advfn.com/boards/read_msg.aspx?message_id=165501593
“However, genomic sequencing probably surprised the company who’s now fashioning this intelligence into a robust package for regulators”.

Question from HC Wainwright: How in early in 2022 do you see filing an NDA?
A: We must wait for the data to be released.

Question from HC Wainwright: Are you cross-analyzing data from Rett syndrome and Fragile-X to create a mega dataset?
A: That is correct. The phenotypes overlap with these two indications. We plan on using the ADAMS endpoint for Fragile-X which had shown good efficacy in the RS-001. Note: Combining clinical data for similar disorders makes a lot of sense as it allows for Anavex to skip phase 2a trials and go straight to phase 2b/3s with greater confidence - which appears to be exactly the route Anavex is taking.

Question from HC Wainwright: Have 2-73 and 3-71 been tested together, or are there plans to do so?
A: There hasn’t been much data to differentiate the two yet. Each drug may have its own merits, but we have not made plans yet to compare the two in similar disorders.

Question from HC Wainwright: Can you comment on the prophylactic trial for dementia prevention? We saw recent advances with unique brain signatures in patients under the age of 39. Are you planning specific tests to help with that trial in the future?
A: We have investigated that as well, and yes, we anticipate using very early markers in our trial.

For more information on past/current preventative trials see: https://investorshub.advfn.com/boards/read_msg.aspx?message_id=165211402

Question from Jones Trading: Can you talk about the AVATAR and EXCELLENCE differences?
A: Target dose for AVATAR is higher than the RS-001 study. We have not disclosed the exact dose yet. Note: In the third quarter conference call Missling mentioned the secrecy of dosing was to prevent unblinding of the trial due to tight knit patient community.

Question from Jones Trading: Are there plans for the AD trial patients going through week 48?
A: There is an extension study ongoing (ATTENTION), some have finished the extension and have requested a further extension which was granted. We will have 3 years’ worth of data by the time the extension has concluded. There is a 94% conversion from the standard trial into the extension.

Question from Jones Trading: What do you think chances are of approval with current Rett trials?
A: We must let the data speak for itself. Current trials are very promising for approval as we have seen good efficacy, there are no drugs approved, and difficulty involved with filling adult trials due to lesser population. We have 3 Rett trials which is more than what is normally required, so we expect to have significant data for regulators and believe chances are high for approval.

Question from BTIG: For Rett syndrome, is the definition of response the same from adult patient to pediatric patient, and is that how clinicians see response?
A: That is correct. Note: Rett data was clinically meaningful, the term ‘clinically meaningful’ is the absolute gold standard for drug approval. A clinically meaningful drug meets a certain established threshold which indicates the drug will have a genuine positive effect on daily life.

Question from BTIG: Is Frontotemporal dementia still expected to be the first indication for 3-71 in 2022?
A: Pending successful phase-1 safety data, yes, we expect to move forward with Frontotemporal dementia or similar indication.

Question from BTIG: Which indications will you prioritize going forward? Which do you see for partnerships?
A: With the rare diseases we see building these into a commercial entity ourselves. For Parkinson’s disease and Alzheimer’s, we see it possibly being more powerful with a large pharma partner as it requires marketing and individual clinician buy-in. If we partner, we will be doing this “at the right time” to maximize upside for investors. Note: It is my opinion that Missling would see "the right time" as post AD 2b/3 publication. Often times big pharma present low-ball offers at earlier stages due to the risk or uncertainty of clinical outcomes. If Anavex can show statistically significant and clinically meaningful data readouts for their AD trial, this eliminates risk for big pharma and prevents only upside from a partner standpoint. This situation would place Anavex in a premier spot to garner a highly favorable commercial deal.