Anavex Life Sciences Corp (AVXL)

[sokol]

Anavex Biomarker and Adapative Trial Design

The adaptive clinical trial design and bio marker- guided design (mostly used in cancer trials) has been adopted by Anavex. So, I am trying to learn about that. Here is a bit of what I have read:

1.

“..... It has been hypothesized and demonstrated that using molecular markers to stratify patients into appropriate clinical trials can improve success rates of trials. While a study analyzing clinical trial drug development in breast cancer from 1998 to 2012 found that only 14% of drugs in clinical development for advanced disease were approved, when patients were selected based on HER2 status, the success rate increased to 23%. Using HER2 status to stratify patients also led to a 27% decrease in costs (Parker, 2012).

Similarly, clinical trial success rates in NSCLC during the same time period were six times higher for biomarker-targeted therapies and three times higher for receptor-targeted therapies than for trials that were not molecularly guided. The risk-adjusted cost for NSCLC clinical drug development was estimated to be nearly $2 billion (Parker, 2014).

Over the past few years, two new trial methodologies have emerged in order to address this growing need for a new clinical trial approach:

The adaptive design and
The biomarker-guided design.
The goals of these two methods are three-fold:

More effectively match drugs to the populations most likely to benefit based on an individual patient’s molecular profile
Decrease trial costs
Decrease the time to drug approval.
The strategies involved within these new designs include interim trial analyses of individual patient data, as well as pre-trial biomarker screening combined with a deeper analysis of the molecular variability within individual patient tumors. The FDA has drafted industry guidance outlining the use and importance of adaptive trials, and various biomarker status initiatives are underway or are being developed.”

....

“The Adaptive Design Clinical Trial
The purpose of the adaptive trial design is both to increase the likelihood of a study’s success as well as to deliver more in-depth data concerning the treatment’s effects. The FDA defines an adaptive trial as “a study that includes a prospectively planned opportunity for modification of one or more specified aspects of the study design and hypotheses based on analysis of data (usually interim data) from subjects in the study.”

A key feature of this trial design is the use of interim analyses, which permit researchers to customize the trial, for example, by adjusting the randomization process or treatment dose or schedule, based on results from earlier participants in the study. This allows for potentially ineffective parameters within the trial to be minimized, instead of being locked into the study’s original design (FDA, 2010).”

https://obroncology.com/article/new-clinical-trials-designed-to-better-leverage-precision-medicine-2/



2.

“Precision medicine is positioned to revolutionize the healthcare landscape, and the utilization of advanced clinical development technologies is key to supporting precision medicine trials. These technologies can harness a variety of disparate data sources at richer and greater volumes to build more accurate predictive models for different patient subsets. We discuss briefly the nature of precision medicine, the FDA’s initiatives encouraging the development of targeted therapies and the importance of the revolution in genomics for precision medicine. We describe the five Vs of big data and the pivotal role of big data in precision medicine. We present four clinical trial technologies that provide essential capabilities for precision medicine:

(1) Continuous collection, aggregation, and integration of myriad data sources; (2) Adaptive and iterative study design and execution as new information becomes available; (3) Maintenance and management of data throughout the study life cycle; and (4) Advanced analytics for research discovery. Precision medicine shows great promise for developing exploratory hypotheses from its broad variety of data and iterative approach, evaluating these hypotheses, and producing safer, more effective treatments for targeted patient subgroups with advantageous benefit-risk profiles. The complexities of precision medicine study designs require substantial planning, investment, and commitment from all stakeholders.”

https://www.thejournalofprecisionmedicine.com/wp-content/uploads/2017/12/DAVI.pdf

Comment: Anavex is collecting its own data, and it is accessing and using the data from the Alzheimer’s Initiatives, etc.