Cantor Healthcare Conference 2021
(Few days late for those who have been unable to tune in.)
- Efficacy with strong biomarker response = key factor of success
- Cantor sees Anavex as a neuro-innovator
- “We don’t micromanage CNS indications by targeting a limited downstream pathway. We allow the body to address - and all individual patients have probably just variations of certain compositions of pathology, some have more abeta, some have more tau, some have more inflammation, and some have more genetic influences - but all of this is really addressed by activation of a more upstream target which is the bodies own defense mechanism, to push back on these manifestations of cellular stress, which is the job of the S1R. And by activating it, we allow the body to do what evolution has enabled, but we allow it to fix it stronger and faster - since it has been depleted due to the chronic element of the disease.”
- All of our pipeline diseases share a ‘chronic nature’ which brings in inflammation and snowballs negative effects over time. Much like negative compound interest.
- We have seen strong reduction or reversal in cognitive effects for Animal models, in Rett syndrome (developmental disease), Parkinson’s (degenerative disease), and in Fragile X (developmental disease).
- Results in Rett, Parkinson’s (to include dementia), and Alzheimer’s has all been exceedingly positive and while each disease is unique, Blarcamesine has provided therapeutic effects as a singular agent which has been correlated very nicely with S1R biomarker response.
- S1R activation can enhance RNA activation. It is believed that as we age we begin to breakdown at the cellular structure. S1R compensates for this breakdown. However, in the AD and PDD patients, there is very low S1R expression. Thus, S1R has been unable to keep up with the breakdown. Blarcamesine boosts this expression to fix these low levels of S1R.
- The population overall has one of two S1R variants: WT S1R or the RS1800866. Overall 80-90% carry the WT S1R (fully functional) gene. The WT S1R patients regularly have a (slightly) higher response over RS. Patients with the RS variant still do better than placebo.
- For Rett syndrome, we hope Blarcamesine can aid immobility, speaking, facial expression, cognition, seizures, and anxiety. The RSBQ measures these items.
- Younger patients tend to have higher plasticity (ability to respond). It is very positive that older Rett patients responded to treatment so well. This bodes nicely for Adult Fragile X trial to come which has seen failure in other trials recently. This failure is surmised to be due to patient age as they did respond somewhat in younger patients [talking about a different company here].
- Excellence study is slightly longer which will provide slight differentiation in design and additional data points.
- An ultra rare disease study is currently being designed. It is of a developmental nature. It will be disclosed soon.
- 3-71 phase 1 data will be released shortly.
- 3-71 will be advanced in Fragile X and cognition.
- 3-71 is S1R and M-receptor, same as 2-73, but with a completely different molecule. Hard to say if 3-71 will act exactly the same/different to 2-73 until comparative trials are ran.
- S1R and M-receptor combination may be key.
- Value creation: Rett syndrome will be first to market. Additionally, Alzheimer’s study readout will be available by this time in 2022.
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