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Re: plexrec post# 298495

Friday, 02/12/2021 9:15:11 AM

Friday, February 12, 2021 9:15:11 AM

Post# of 462171
plexrec, here you go. From the transcript....towards the end of his response/answer.

Ram Selvaraju -- H.C. Wainwright -- Analyst

OK, great. And then just three other very quick ones. Firstly, assuming you have positive data in Rett syndrome, what implications might that have for future development of blarcamesine in others orphan neurological diseases? And can you elaborate at this juncture, what would potentially be your highest priority choices with regard to future orphan CNS indication development? Secondly, what is the significance from a commercial standpoint of the oral solution, in particular, as this pertains to Rett syndrome? I think, you know, we may have touched upon this in the past, but it probably is worthwhile reiterating that. And then lastly, I was wondering if you could comment at this point about the possibility of co-formulation, co-administration, synergistic combination of blarcamesine with other compounds in the Anavex pipeline? And if that's likely to be the case, if that is something that you think might be something you wind up exploring in the future which other compounds or which other types of compounds from the Anavex earlier stage portfolio might be likely to be most synergistic with blarcamesine? Thanks.

Christopher Missling -- President and Chief Executive Officer

Right. So the first question regarding -- or the second question regarding the solution, I think it's a very strong advantage because many of these kids or girls, they have a hard time swallowing. Some even cannot even eat, they have an artificial pouch to -- a feeding tube and liquid formulation is the only way actually to provide a drug substance to the patients other than by injection which is very overly inconvenient and cumbersome. So that is a very strategic advantage of the therapeutic administration.

The first question regarding what could trigger the approval of Rett syndrome, we have seen that ANAVEX 2-73 has been extremely strong in treatment studies, but to also affect the pathology of Fragile X, which is autism spectrum disorder, as well as, infantile spasm and Angelman syndrome, another rare disease, these all three diseases. But Fragile X is the largest rare disease of this autism spectrum disorder and with a multiple of the patient than Rett syndrome patients. So since we have very strong preclinical data and Rett syndrome happens to be a part of the autism spectrum disorder, given now we have basically supportive data, very strong data preclinically in clinical in Rett syndrome, we have now very strong and supportive data in preclinical in Fragile X. We believe that the chances have increased to also be successful in a clinical study in the Fragile X and that's why we're planning, and we already have developed the study design to move into Fragile X as a next study for ANAVEX 2-73.

Regarding the last questions about synergies, we don't know if the synergy with another sigma-1 agonist might be the best choice. I would rather think that a synergy could be with other compounds targeting other pathways, like if Adu will get approved, we believe it will be synergistic to ANAVEX 2-73 because it is a different pathway. But also we have seen that ANAVEX 2-73 has been shown preclinically to prevent the disease. So it's not only the treatment which is the goal and that's what we're now finding out with our ongoing Phase 3 study in Alzheimer's disease.

But also thinking ahead strategically that one day of ANAVEX 2-73 could be used as a daily mini-aspirin potentially to avoid being -- coming in near to be in a situation where you get affected by or afflicted by such a horrible disease like Alzheimer's disease.


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