Replies to post #169067 on Anavex Life Sciences Corp (AVXL)

[Investor2014]

Depends how it will be handled in practice. It is either quite clever or an additional delay over the hope of some investors.

The original 12 week study had a 4 week observation period in front for each of the 50 - 80 girls to be enrolled.

Now it could be that the 7 week P1 like study in only 15 girls eliminates that 4 week observation period for the full P2 placebo controlled trial. This would as far as I can work out reduce the total elapsed time of the trial.

Further more as the P1 like study includes efficacy endpoints, we get a much early peak into the potential outcome of the full placebo controlled P2 trial.

I say that is a win, especially if we take into account that, not least, you suspected all along that a tolerability study would be needed since we are dealing with adolescents patients.

So Anavex cleverly managed to swap a 4 week observation period for the full 50 - 80 cohort for a 7 week dose escalation study in 15 patients - the work of Fadiran perhaps shining through here?

[nidan7500]

Flash:

Became:

“FDA has allowed Anavex to proceed with the Phase 2 study protocol, ANAVEX2-73-RS-001, A Double-Blind, Randomized, Placebo-Controlled, Dose Titration Study of ANAVEX2-73 in Patients with Rett Syndrome using experimental drug ANAVEX®2-73 for the treatment of patients with Rett syndrome. The Phase 2 study is a randomized double-blind, placebo-controlled safety, tolerability, pharmacokinetic and efficacy study of oral liquid ANAVEX®2-73 formulation to treat Rett syndrome. !Pharmacokinetic and dose finding will be investigated in a total of 15 patients over a 7-week treatment period including ANAVEX®2-73-specific genomic precision medicine biomarkers. All patients who participate in the study will be eligible to receive ANAVEX®2-73 under a voluntary open label extension protocol. This study will be followed by a planned placebo-controlled safety and efficacy evaluation of ANAVEX®2-73 over a 3 month treatment period.

https://www.anavex.com/anavex-life-sciences-announces-fda-approval-of-ind-for-phase-2-trial-of-anavex2-73-in-patients-with-rett-syndrome/


To me it seems like a convoluted way of saying the FDA has required Anavex to do a smaller (probably open label) dose titration study before they will allow them to do the Rett trial they had asked for in the original IND they filed.

It’s not clearly spelled out if this is actually a phase 1 or phase 2 trial but the way the PR is written, it certainly leaves the matter open to debate imo.

Flash, thanks for this. I am still stuck back at the Friday announcement (on option day) wondering why AVXL chose to do PR of this magnitude on Friday the week before multiple other CNS big events get covered.

I did predict here there would be a PR before the conference but never expected anything on Friday (w/low confidence of any Monday PR also)...but, this gets tricky...what if an unexpected compression of events (positive) has developed and DR.M and crew decided to go w/Friday's PR b/c there is a lot already planned for next week? I would add to that the possibility that initial trial does have already been administered and RS kids have responded well. Take it from there....

The PR Friday was a surprise (I expected to hear from BIIB re some kind of collaboration). If BIIB objected to any RS PR stepping on their show then they may have influenced the Friday release, it's complicated. I do still see some good news soon w/events escalating quickly. AND, it's about time.

[Steady_T]

What label is applied to the trial is not really important.

What is important is that a trial is approved.

It is very short term. It requires a quite small number of subjects. It leads directly to the larger placebo controlled trial.

Looks like a large portion of the trial costs will be borne by the Rett Foundation. That is good for two reasons. One because it lessens the impact on company resources. Two because it demonstrates the Rett foundation's opinion of 2-73 is high enough for them to put money behind getting it developed.

[XenaLives]

Repost of replied to post:

F1ash Sunday, 10/21/18 04:17:19 AM
Re: avxl_going_long post# 169066 0
Post # 169067 of 203939

”The 12-week trial — which will cost about $1 million total and enroll between 50 and 80 patients — will measure the drug’s effect on seizure reduction, cognitive impairment, mood disorder, autistic behavior and anxiety, Christopher Missling, president and chief executive officer of Anavex, told MarketWatch.”

https://www.marketwatch.com/story/anavex-awarded-grant-to-fund-clinical-trial-of-rett-syndrome-drug-2017-01-19


Became:

“FDA has allowed Anavex to proceed with the Phase 2 study protocol, ANAVEX2-73-RS-001, A Double-Blind, Randomized, Placebo-Controlled, Dose Titration Study of ANAVEX2-73 in Patients with Rett Syndrome using experimental drug ANAVEX®2-73 for the treatment of patients with Rett syndrome. The Phase 2 study is a randomized double-blind, placebo-controlled safety, tolerability, pharmacokinetic and efficacy study of oral liquid ANAVEX®2-73 formulation to treat Rett syndrome. !Pharmacokinetic and dose finding will be investigated in a total of 15 patients over a 7-week treatment period including ANAVEX®2-73-specific genomic precision medicine biomarkers. All patients who participate in the study will be eligible to receive ANAVEX®2-73 under a voluntary open label extension protocol. This study will be followed by a planned placebo-controlled safety and efficacy evaluation of ANAVEX®2-73 over a 3 month treatment period.

https://www.anavex.com/anavex-life-sciences-announces-fda-approval-of-ind-for-phase-2-trial-of-anavex2-73-in-patients-with-rett-syndrome/


To me it seems like a convoluted way of saying the FDA has required Anavex to do a smaller (probably open label) dose titration study before they will allow them to do the Rett trial they had asked for in the original IND they filed.

It’s not clearly spelled out if this is actually a phase 1 or phase 2 trial but the way the PR is written, it certainly leaves the matter open to debate imo.