Anavex Life Sciences Corp (AVXL)

[nidan7500]

Over time many of us have speculated here about PD/PDD and what is going to happen next w/A2-73 trials, our investments and trial participants. Lately I have been trying to better understand the trials process time lines and just what the hell is magical about the periodicity. I ask myself, "what the hell is special about 14 weeks?". Is it as appears just an arbitrary choice having nothing in particular to do w/the science or the disease or good planning or what ever ??.

What if the FDA trial timelines are meaningless from a science, patient or specific disease standpoint? What if (n years) ago some rules committee decided that everything(trials) will just have these time lines b/c we say so and then we all move on while every trial always gets stopped and criteria are applied at these specific points. OK SHAT happens that is a rule then.

Then I start to wonder (cannot help myself as a Dr. Peter Senge former Systems thinking intern-student at MIT) ...just what kind of system is that and what if we have diseases that move faster/slower than the trial plan, will we miss or not see something important? (Asynchronous comes to mind) Well, maybe we would miss something important and also then what does the CRO do/say ??? Not knowing they missed the bus? How do we find out and adjust?

I have no clue how the trial timelines were set. W/S and the rest of the world seem to think it is all meaningful, so that is all we have to know. The fact that TIME is probably the most precious element that any of us have/use should be somehow considered.


SO WHAT, you say? Good point, except that we know that PD/PDD is/are very slow developing (years) CNS diseases which appear to have links to gene and other environmentally conscious matters.

The current PDD trial reporting from/by CRO and subsequently AVXL is late. Late for what I ask? Well, the trial measured timelines , etc, Blah, blah.

What if 14 (or n) weeks is the wrong metric-cycle and the are variation w/treatment is subject specific based on genes and other eco-history for each patient??? What if the MOA for AVXL (upstream) requires a different timeline cycle b/c this is PDD-PD and it moves very slowly and what if we have no good idea what to measure or when or how?

Well, just a few basic questions for which is have no answer but it seems obvious that some one should know and how does our planned timeline fit w/any of the participants Bio-Sequencing-evolution pathologies?

HH has a strong sense for the importance of PM and he is probably right. In the meantime we all stumble along in a slightly advanced path waiting for ??? Waiting for some smart person to have an AHAA moment or what ever it is going to take. The reality is, PDD and PD are very slow moving-developing (read changing) CNS diseases which we are treating with an UPSTREAM MOA expecting/waiting to see Restoration and Regeneration, which may in fact actually be happening as we speak. How would we know? They are probably a poor choice for any demonstration of efficacy based on some arbitrary timeline.


https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5989714/

What Is Precision Medicine?

The National Research Council’s Toward Precision Medicine (6) adopted the definition of precision medicine from the President’s Council of Advisors on Science and Technology in 2008 as: “The tailoring of medical treatment to the individual characteristics of each patient…to classify individuals into subpopulations that differ in their susceptibility to a particular disease or their response to a specific treatment. Preventative or therapeutic interventions can then be concentrated on those who will benefit, sparing expense and side effects for those who will not”. As the definition suggests, the power of precision medicine lies in its ability to guide health care decisions toward the most effective treatment for a given patient, and thus, improve care quality, while reducing the need for unnecessary diagnostic testing and therapies.

The conceptual distinction between personalized and precision medicine
Personalized medicine refers to an approach to patients that considers their genetic make-up but with attention to their preferences, beliefs, attitudes, knowledge and social context, whereas precision medicine describes a model for health care delivery that relies heavily on data, analytics, and information. This model goes beyond genomics and has vast implications for our nation’s research agenda and for its implementation and adoption into health care. Precision medicine – and the ecosystem that supports it -- must embrace patient centered-ness and engagement, digital health, genomics and other molecular technologies, data sharing and data science to be successful.

Components of the Precision Medicine Ecosystem
A precision medicine ecosystem ideally links patients, providers, clinical laboratories and researchers (see figure 1). With the advent of EMRs and robust IT systems supporting both research and health care delivery, patients (and research participants) who agree to provide biospecimens and share their clinical and research data are at the epicenter of contributions to the research enterprise. Researchers generate new findings from the data derived from samples linked to digital phenotypes, family history and environmental exposures all captured as part of clinical care. Clinicians utilize a growing knowledge base curated from clinical laboratories (8–10). This assembly of data from a variety of sources sets the stage for a powerful precision medicine ecosystem which, when coupled to others, leads to a dissemination of knowledge that enables other systems to benefit. The extended precision medicine ecosystem includes government as sponsors of precision medicine research and regulators of precision medicine products, industry as partners in development and commercialization of precision medicine products, professional societies as enablers of the training of the next generation of researchers, providers, and policy analysts, and payers who evaluate the appropriateness of precision medicine interventions and the financing that support their use in health care.

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Exhibit 1
The Precision Medicine Ecosystem

Source: Adapted from reference 7

The Learning health system and precision medicine
The inclusion of genomic data in a knowledge-generating health care system infrastructure is a way to harness the full potential of that information to optimize patient care (11, see figure 2). In such a system, clinical practice and research inform each other with the goal of improving the efficiency and effectiveness of disease prevention, diagnosis, and treatment (12).