Anavex Life Sciences Corp (AVXL)

[nidan7500]

whippin..thanks for posting this exceptionally well done report. My highlights of points that stuck w/a true representation of AD stage measurement capability status.
http://www.alzforum.org/news/conference-coverage/which-are-right-tests-satisfy-new-fda-guidance27 Nov 2018

In its February 2018 draft guidance for industry, the U.S. Food and Drug Administration signaled a willingness to consider a slowing of cognitive decline alone as the basis for approval. At the 11th Clinical Trials on Alzheimer’s Disease conference, held October 24–27 in Barcelona, Spain, leaders in the field wrestled with how this new guideline might be put into play, and what cognitive tests might fit the agency’s bill.

I suggest RWE-RWD will be a significant value added data set until a better complimentary metric is developed, particularly at stage 2 levels. "Where the money is".

It is not so clear, after all, whether stage 1 disease actually exists,

(in other words , if it cannot be measured it cannot be managed.)

Aisen noted that the difference in decline is slight in preclinical disease, detectable over three years on a sensitive cognitive battery such as the PACC, and over six years on the CDR-SB. In other words, detecting this modest decline would require either long trials or even more sensitive cognitive measures.

FDA says it will accept cognitive measures alone as the basis for approval in stage 2. It also says a drug’s benefit must be robust and consistent across multiple domains, and that the application will be stronger if supported by biomarker evidence. Petersen suggested mining longitudinal data to identify other early impairments, such as mild neurobehavioral symptoms, that could strengthen the case for a clinical benefit. “Perhaps we could marry subtle behavioral features with tau pathology,” Petersen said.

FDA continues to hold onto the Amyloid Thesis, b/c it can be measured by PET, etc...cause vs correlation?
HUH??

So which test is best for preclinical disease? Hendrix said it depends what researchers want to measure. Some treatments, such as cognitive enhancers, might improve progressive and non-progressive aspects of cognition. In that case, the PACC might be good for evaluating efficacy. Interventions such as nutritional or lifestyle interventions might be expected to slow age-related decline as well as Alzheimer’s pathology, and the API-LOAD might be the best test to evaluate efficacy (Langbaum et al., 2014). For a therapy targeting a specific AD pathology, such as amyloid or tau, the APCC is likely to paint a clearer picture of its effects. “It comes down to figuring out what kind of change you have in an untreated population, and what aspects of that change you’re targeting with your treatment,” Hendrix told Alzforum.

Well, IMO the most reliable measure for AD must be how the patient feels, sleeps, acts and functions. IMO, caregivers and the patients observed QOL will have to remain the standard. When science eventually is able to correlate before-after (some test) results, then they will be able to define the proper metric. Until then they are in the dark, obviously.