Posted on August 4th, 2014
by Gerald Commissiong
The month of July turned out to be an incredible fundamental value-building month for Amarantus. We completed our 3rd #C4CT Alzheimer’s-focused Concussion Awareness Summit at the United Nations in addition to presenting data at the Alzheimer’s Association International Conference (AAIC) earlier in the month.
The attendance for #C4CT was nothing short of tremendous, with international participation from Key Opinion Leading (KOLs) scientists, policy-makers, caregivers, family offices and others who were on-hand to participate in what is turning into a powerful forum to share ideas, promote policy changes and foster cross-border collaborations among stakeholders who are all committed to helping create better outcomes for those affected by injuries and diseases of the brain.
On this last point, we were extremely pleased to enter into an exclusive option to license the IP related to this concept, as it represents a major step forward in the Company’s Alzheimer’s program by adding a therapeutic component to our Alzheimer’s blood diagnostic LymPro Test®. Taken together, the ability to diagnose Alzheimer’s early on in the disease process using a simple blood test coupled with a therapeutic intervention centered on a fundamental aspect of disease biology uniquely positions Amarantus in the space. #C4CT was the first public forum Dr. Arendt had ever shared his concepts on Alzheimer’s disease, and certainly stirred significant discussion among the KOLs present as to new ways to further evaluate the hypothesis and means of collaborating with Amarantus on the concepts.
Immediately following Dr. Arendt’s presentation, Dr. Louis Kirby shared some positive top-line interim data from the Company’s ongoing LP-002 clinical study for LymPro where we demonstrated that with a single marker, CD19, we were able to distinguish Alzheimer’s disease from healthy controls in a population of 44 subjects. The Company had been planning on sharing top-line data from the full 72 subject study, and was disappointed this was not possible at #C4CT. When the Company became aware that 3 of the 4 sites had not completed their full enrollment, in part due to stringency in the Alzheimer’s subject selection criteria, we also became aware that one of the sites had exceeded its enrollment criteria as it was able to get through Institutional Review Board (IRB) approval well ahead of the other sites. We decided to un-blind the single site and perform an interim analysis for the purpose of being able to keep our previously outlined timeline on LymPro commercialization, and because #C4CT was a unique opportunity to recruit collaborators for the LymPro project. We will continue to focus our efforts on extracting the best univariate markers from LymPro through the completion of the LP-002 bridging study ahead of endeavoring to establish the multivariate predictive modeling that will become the basis of the ultimate clinical diagnostic that will be used commercially.
It is important to note that a diagnostic cannot be a better predictor of disease than the reference against which it is judged, i.e. if Alzheimer’s can now only be definitively diagnosed post-mortem, for Amarantus to be successful we must find some proxy of that diagnosis against which to be judged other than autopsy, and for now that gold-standard is PET imaging. PET imaging is quite expensive, and the ability of Amarantus to ‘piggy-back’ LymPro on an already defined population of PET-confirmed Alzheimer’s patients is as good a reference as we can possibly expect to get at this stage in LymPro’s development. For Amarantus the purpose of the presentation at #C4CT was to convince the KOL community, who have trials currently enrolling using PET imaging to confirm diagnoses, that LymPro should be added to their ongoing trials. The reason for this is that as Amarantus continues the development of LymPro, we will need to confirm the diagnoses of LymPro-positive subjects in biomarker-identified cohorts in order to initially demonstrate concordance with current FDA-approved gold-standard in-life diagnosis paradigm for Alzheimer’s disease. To that end, we achieved our objective, as the KOL community was extremely impressed that with a single marker we were able to show a highly statistically significant differentiation between Alzheimer’s and a healthy control, and believe that collaborators in the fall will be available for the CLIA-enabling study we will be running at our prospective partner’s laboratory. We will now focus on completing the enrollment for LP-002 protocol and continuing preparations for commercial launch under CLIA. #C4CT presentation was focused on the scientific community, not the investor community.
Our rationale for only showing the KOL community Version 1 data is because Version 1 is the only peer-reviewed evidence published in the literature, and all of the Version 2 data was produced in sub-standard lab conditions at Memory Dx (f/k/a Provista Life Sciences). While we continue to believe that there are other stimulation conditions of the assay under which we may see improved performance of LymPro (and we have already filed IP to cover these concepts), we could not reference anything other than the published literature when convincing the KOL community that LymPro is worthy of inclusion in leading edge diagnostic trials. Our strategy was successful and we expect significant collaborations to emerge as a result.
We are currently reviewing the new guidelines regarding diagnostics in clinical laboratory settings that was recently released by the FDA. The Company had been projecting for some time that the FDA would be updating guidelines vis-à-vis clinical diagnostics and we have been proceeding with the expectation that the stringency on the diagnostic paradigm would be increased over time. We do not foresee this delaying our plans in commercializing LymPro.
In summary, we are extremely pleased with the data we presented to the KOL community last week at #C4CT, and we achieved our strategic objective of convincing those KOLs that LymPro is worthy of being included in already-enrolling PET Alzheimer’s clinical studies. We further positioned Amarantus in the midst of the overall brain research discussion and in-licensed a potential disease-modifying therapeutic program in Alzheimer’s disease based on the fundamental disease biology LymPro is based upon. We believe we are one of the only companies in the world with a drug/early-diagnostic combination in Alzheimer’s disease. We are well positioned to continue forward with our LymPro program and remain on track to achieve our corporate objectives by the end of 2014.
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