Replies to post #466261 on Anavex Life Sciences Corp (AVXL)

[Hosai]

Don't think this is right Doc, Gemini says those 0.5 CDR G patients are usually seen as stage 1 or 2 AD rather than stage 3 so is indeed relavant for the guidance as early stage. I doubt Anavex would have got that wrong they literally have mentioned the guidance in the PR.
Almost two thirds of their trial were this early stage.
They passed CDR-SB anyway with low p value too either way.

[12x]

Doc, page 1 of the new FDA guideline does seem to cover Stages 1 through 3 (see below). But do we know how to map 5 levels of CDR scores (0,0.5,1,2,3) to the 6 stages in FDA guidelines?

Early Alzheimer’s Disease: Developing Drugs for Treatment Guidance for Industry

I. INTRODUCTION

The purpose of this guidance is to assist sponsors in the clinical development of drugs for the treatment of the stages of sporadic Alzheimer’s disease (AD) that occur before the onset of overt dementia (i.e., Stages 1 through 3; discussed in section III). These stages are collectively referred to as “early AD” in this guidance; however, it is recognized that AD occurs on a continuum and patients in the last stage of early AD (i.e., late Stage 3) and patients with AD in the earliest stages of overt dementia (i.e., early Stage 4) may not differ significantly in clinical presentation.

[frrol]

The drugged patients are 'very mild dementia' on the CDR-SB scale, placing them somewhere in AD stages 2 to 3, in which case your second paragraph taken from the FDA guidance would be the operating one (if based on Sum of Boxes). And we do appear to meet the "positive trends on functional outcome assessments" with ADL.

The bigger issue with our TEAEs is the impact it had on our already low n. The regulators will have to get comfortable with not just the eye-raising number of dropouts but the number of completed participants and the limited endpoint data sets.