Amylyx Educational Summit in Advance of Releasing Data

Kristina1

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Hi guys! Amylyx is hosting a virtual educational summit for PALS and CALS tomorrow 8/20 from 6-8pm. I will be speaking about my experience as a participant in the study and an ongoing partcipant in the open label extension phase. If you'd like to join us you can register here: Online Event
 

Nikki J

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Awesome job Kristina! Thank you!
 

EricInLA

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Yeah, great job Kristina. That was actually very informative. I didn't quite understand the parallel pathways with the different meds but I now I have a decent understanding of it. I'm really hoping the results of the trial are strong.
 

Kristina1

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Thank you! And thanks for tuning in!
 

Samkl

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Oh goodness, I missed this! 🙁
 

CamilleQ

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Oh I missed it :(
 

Nikki J

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Kristina did an great job speaking about her experience with ALS and with the trial.

The results will be published in the next couple of weeks and they are working with the FDA on next steps. It sounded hopeful. remember TUDCA is currently available in the US as a supplement and I think many other places. The other component is prescription and in the US terribly expensive and generally subject to strict PAs
 

CamilleQ

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Hi everybody,

I have a few questions here.

First can we see the conference in replay somewhere?

I have questions about Amylyx but also about drugs for als in general.

Amylyx if I m rigt is sodium phenylbutyrate and tauroursodeoxycholic acid (tudca? the same that is sold as a dietary supplement?) does it significantly slow down the disease? Can that stop it? Does it work for all types of als? Because Riluzol and Edavarone are supposed to slow down the disease but their effects are very questionable. Are Amylyx's results more promising?

Have a nice day or night 😘
 

Nikki J

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I have not seen it anywhere for replay and there was no mention that I heard of it being recorded. Kristina would know for sure but it is not on the company website or twitter account.

yes the same tudca. Preliminary reports were of slowing and so are the anecdotal accounts. As I noted the fuller results will be published within a couple of weeks they expect. Until then we don’t know how well it does

the trial included various phenotypes and had SALS and at least one FALS that I know of.
it has a different target than existing drugs . Most of the doctors I know think the answer especially for SALS will be a combination of different drugs
 

CamilleQ

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Thank you @Nikki J for the answer. Looks like we haven't gotten out of the troubles yet :(
 

EricInLA

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Not yet Camille, but there are promising things on the horizon. :) In light of the point made by Nikki that the answers will probably come from a multitude of different drugs. . . is there any harm in trying them all? I realize the scientists may not like that for purposes of clinical trials because it's hard to prove the affect of a particular drug if you're taking multiple drugs, but one thinks differently when one has this disease. I'm on Riluzole now, with no side effects currently. I'm not doing Edaravon (Radicava) at the moment because my doctor didn't think the evidence was strong on that one, but I might change my mind if I can take it in pill form (should be soon?). Also, I have a big bottle of TUDCA in the pantry that I'd like to start taking soon. If the Amylyx formula becomes affordable, as opposed to off label use, I'd switch to that. And, I'm hoping to participate in the Healey platform trial which is supposed to start soon at my local ALS clinic. I'm not the type to chase miracle cures, but these all seem legitimate and worthwhile, provided there's no major side effects. I'm learning that they attack this disease from different fronts. So, why shouldn't we try them all? I realize this is sort of a rhetorical question, but it's been on my mind.
 

Samkl

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Hi Camille, I asked these questions of Kristina a while back, so you should be able to use the search function to find some answers.
 

lgelb

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The oral edaravone study estimated completion date is November of next year. Then, the company has to clean and analyze the data, write it up, and submit an NDA to the FDA, which usually takes a year or so unless they are under a program that allows for a rolling submission. Then, even under a priority review, the FDA has six months to make a decision (reject, approve, or send back for more work). So realistically even if all the stars align, we're talking mid-2023 approval.

As for trying everything, ALS is at least in part a disease of metabolism and everything you take has to be metabolized, which can literally compete with food for the energy to process it. I would also consider the safety and efficacy of combining multiple, incompletely tested rx and supplements. You can cancel out effects, make effects stronger, cause side effects, etc, and basically negate the "different mechanisms of action" notion. At a minimum, I would check them all against each other somewhere like drugs.com and check the exclusion criteria used in the trials to illuminate other possibly adverse combinations.
 
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ilanarachel

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hi -
so sorry I missed this as I was in the clinical trial. Is there a video/recording of the event?

thanks
 
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