Moxie Study

[Christina Cordaro]

@holmej4 the MOXIe news is amazing! I was at the symposium in Philadelphia with 60+ other FA patients when the press release was announced by the FARA President, Ron Bartek. It was such a powerful moment.

I know when you are a part of a clinical trial, you are not supposed to share your symptoms and reactions of the drug because you don’t want to persuade others who are also participating. So I don’t think we can know any opinions until the FDA approves the drug and it goes to market.

[Jaq]

Christina that’s amazing, I bet it was very emotional being there for the announcement.
It really does give hope!
I am unsure of the whole process, I seen an article last year to say it was recruiting for the Moxie Study and I did enquire for my daughter but had no response, but I thought it was a phase 2. Any idea on what the next steps are? If it’s gone for approval will it still require more trials or have they all completed now? I really hope the approval process is something that is completed in not too distant future as that would be so amazing xx

[Christina Cordaro]

@holmej4 I know it is in the early stages of FDA approval and it could take at least a year. I believe now is the “waiting game”.

[Jaq]

Fingers crossed this goes through quickly. I Am worried of the timeframes for this to be available in the UK which is where we live. Especially with all the Brexit chaos that’s ongoing, as that could really impact on the good work being done for these trials across Europe. But if I have to fly to America and pay for this for my daughter then it’s definitely something I would do.
Let’s hope this is available sooner rather than later and that some of the other good work ongoing in trials also follows on with great results.
It’s nice to have some hope xxx

[Christina Cordaro]

@holmej4 hope is stronger than fear!

[Jayne]

I have not posted here before so apologies if I’m not doing it correctly!

My Son was/is on the Moxie Trial and few people have asked me about it so here’s a brief summary of what our Moxie consultant has explained in layman’s terms!
1. It works! (yay)
2. It not only halts progression, it has actually started to reverse it!
3. MFARS Scores in an FA patient would normally increase 1 to 2 points per year. Moxie has reduced MFARS score by 1.55 points so effectively reversing FA by 1 to 2 years!
(MFARS Scores test swallowing, speech, upper and lower limbs and stability)
4. The drug has to be approved by the FDA before it can be licenced (this is not a given, but is likely).
5. Because FA is an orphan disease, ie currently has no active treatment, it should be fast tracked by the FDA. Historically that is likely to take an absolute minimum of 6 months, but could be longer if there are questions.
6 FDA approval in America won’t automatically give it approval in Europe, UK etc and it will have to go through their approval processes too.
7. Several people have been on the expansion phase (active drug) for coming up to 6 months so new data should be available around December to see if there is a limit to how much Moxie can reverse FA (1.55 points) or whether the positive effect is cumulative and will keep on improving.
8. The medical meeting in December should also give more detailed information on secondary markers like whether Moxie has any impact on cardiac function etc
9. There are no obvious side effects to the drug.
10. Reata now plan to see if Moxie can help Alzheimer’s, Huntington’s Disease, Parkinson’s Epilepsy and Motor Neuron Disease.

Hope that helps 🙂

[Heather]

Does anyone know how a Canadian can join in on the trial or test?

[Jayne]

Hi Heather
Recruitment for the trial finished at the end of 2018, so I don’t think its possible to join it now I’m afraid.
Best Regards
Jayne

[Fenna Paulus]

Hi!
Does anyone have any updates on this?
When will the FDA approve it and will it be available internationally then?
Kind regards, Fenna

[Christina Cordaro]

@fenna last I heard from my neurolgist at CHOP said we should bear more information by this March, but not guaranteed.

[Malcolm Long]

My daughter failed to get onto the trial as she couldn’t pedal a recumbent bike to exhaustion. My concern is the trial is being based on the equivalent of ‘paralympic’ performance and may be skewing results. Hopefully not and the reports and good effects will apply to everyone whatever their FA status.