1.“Missling is reporting endpoints related to the primary endpoint rather than the primary endpoint themselves is high…”
2. “AA was always a pipeline on this board, A fantasy I never bought into given the size of the 2b/"3" and low chance of smashing success…’’
#1. Number one above sounds like AA approval wherein surrogate endpoints are substituted for primary endpoints (and efficacy may be proven later in a confirmatory trial - Aduhelm, et al).
#2. As to the size of the trial (Number two above). The Anavex Alzheimer’s trials were designed as precision medicine trials. See A and B below.
A. "...Precision medicines can benefit patients by increasing the probability of a successful treatment response in selected patient populations. The potential for more immediate signals of efficacy during clinical trials suggests such medicines will reach the market more rapidly than traditional drugs will....
...We found that in the period January 2013–June 2017, precision medicines were developed and reviewed almost two years faster than nonprecision medicines. In addition, approximately 48 percent of the precision medicines in our study qualified for the FDA’s breakthrough therapy designation. Precision medicines were also approved based on fewer pivotal trials with fewer patients, and the trials were less likely to be randomized, blinded, or controlled with either an active or placebo comparator....
...This approach makes possible the use of fewer clinical trials that tend to be smaller and less often feature traditional characteristics such as blinding and randomization, leading to shorter total development and review times. But these trends associated with precision medicines also raise concerns regarding the sufficiency and quality of the evidence on which FDA approval is based and public understanding of evolving evidence standards. The trends necessitate increased reliance on collecting postmarketing data to confirm the drugs’ anticipated risks and benefits...."
"Recent advancements in cancer biomarkers and biomedical technology have begun to transform fundamentals of cancer therapeutics and clinical trials through innovative adaptive trial designs. The goal of these studies is to learn not only if a drug is safe and effective but also how it is best delivered and who will derive the most benefit....
'''Precision medicine is an approach to deliver optimal patient outcomes by integrating clinical and molecular patient data to understand the biological basis of the disease.1 This approach guides selection of the most appropriate targeted therapy based on distinctive patient characteristics and unique molecular features of a malignancy. The aim of this strategy is to optimize patient outcomes, while providing more favorable safety profiles than traditional population-based cancer chemotherapy. ....
B. ...Implementation of clinical trials in the cancer biomarker era requires knowledge, skills, and expertise related to the use of biomarkers and molecularly defined processes underlying a malignancy, as well as an understanding of associated ethical, legal, and social issues to provide competent, safe, and effective health care and patient communication."
Implementation of clinical trials in the cancer biomarker era requires knowledge, skills, and expertise related to the use of biomarkers and molecularly defined processes underlying a malignancy, as well as an understanding of associated ethical, legal, and social issues to provide competent, safe, and effective health care and patient communication.
****** It seems to me that the point of accelerated approval and precision medicine is to make promising drugs for serious diseases available to patients earlier - promising (in case of AA) meaning meeting the reasonable likelihood of success of efficacy being proven later in a confirmatory trial if it can be established that there is a strong connection between the drug in question and and what’s happening within the body of the patient (markers). Precision medicine’s goal is the make drugs available faster based on fewer and smaller trials. Of course, there are many that criticize and do not approve of precision medicine. The same goes for accelerated approval.
Another problem, I think, is that there is a lot of confusion resulting from comparing clinical trial results of precision medicine trials using standards, etc., that apply to traditional clinical trials.
However, I am not drawing any conclusions about the chances of accelerated approval of Blarcamesine pending reporting of all data and information from the Anavex Phase 2b/3 AD trial. However, I am also one of those people that think that there is always more than one side of any situation or story. My way of thinking has been shaped by what I have experienced over many years, including a doctor telling me that I was going to die many years ago. The other side of that story is that I am still here, which I was tempted to say to that doctor when I saw him in a hospital elevator. However, I didn’t do that because I think that doctor was telling me what he honestly thought was the truth based on what he knew. But, the unexpected can always occur.
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