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Re: dadofmarcmax post# 122574

Sunday, 10/21/2018 9:51:31 AM

Sunday, October 21, 2018 9:51:31 AM

Post# of 518943
Another blast from the past:



dadofmarcmax Friday, 09/29/17 05:32:23 PM
Re: None Post # 122574 of 169082

Epileptic seizures are by far the most concerning problems that many patients with certain genetic syndromes like Rett, Angelman syndrome, fragile x syndrome, Lennox-gastaut syndrome, etc experience. Epilepsy in these cases is often refractory meaning no matter what combination of meds, dosing, etc is given, these patients still have breakthrough seizures.

All it will take is a Rett trial that can show a clinically significant reduction in seizure frequency relative to baseline compared to a placebo control group. A secondary endpoint of 50% responder rate as well as other secondary endpoints can be included.

This is a study that can be done quickly.

IF the Rett trial shows decline in seizures, my oh my will the floodgates open. Then every epileptologist in the country (me included) will demand that it be tried on our refractory epileptic patients, that it be included for treatment of Lennox gastaut syndrome and other epileptic encephalopathies. I will even be willing to try on infantile spasms. Epilepsy indication can then jump way up to advanced phase 3 trials as adjunctive treatment and monotherapy treatment for focal epilepsy and possibly even generalized epilepsy.

I will be attending AES (American Epilepsy Society) this year in DC and hope to see something g Rett related this December. I expect Q4 of 2017 to be positively transformational for Anavex Life Sciences.

As always, my opinion only. Do your own due diligence.





dadofmarcmax Saturday, 09/30/17 10:44:30 AM
Re: LakeshoreLeo1953 post# 122622 Post # 122643 of 169084

My time with my boys is considered the most sacred time for me - and hence, it always suffers since I always want to be around my two sons.

I'm not sure of your question, leo, so here is my best attempt.

What I am looking for only in Retts is - statistically significant reduction in seizures compared to baseline seizure frequency (relative to placebo control). I am also looking for improvement in coordination.

If Rett trial shows a significant reduction in seizure frequency - this will serve, in my opinion, as a gateway to a much broader epilepsy indication and pave the way for breakthrough designation or accelerated approval for the other orphan drug designations of fragile x syndrome and possibly infantile spasms. With these approvals - the race to broader indication of "partial onset seizures" (or partial epilepsy/focal epilepsy/localization-related epilepsy) would then start - with quicker time to market for epilepsy than what we think of it now. I foresee an epilepsy indication leapfrogging over an AD indication for FDA approval IF the REtt trial is successful.

The Rett trial is much more important that people currently think, in my opinion. IT will be the "make or break' trial for this company and hence, why the design and planning of it is taking so long. With success - the possibilities for other epilepsy-related indications become abundant. With failure - all the other possible epilepsy indications will take longer, if at all, to come to fruition and optimism for quick AD and PD trials will be stymied.

Hope this sheds some light onto how I think of the current position long investors like myself are in.

Hope all is well Leo!



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