EPI-743 update from Edison Pharma

I participated in the Edison Pharma update call today and below includes a brief on the updates and some commentary. I didn’t include every minute detail. The call was recorded and you can find it on UMDF’s website.

Did you know Edison has SEVEN clinical trials going on right now? Edison said there are more on the horizon, announcing them summer 2013, including those for Mito and specifically MELAS. This is all the more reason to reach out to groups like UMDF and MitoAction as well as your doctor to be prepared when these clinical trials are released, you may not qualify for the Phase IIB EPI-743 trial, but you might for another one.

• New drug, EPI-589, Edison called it the next generation 743 as they try to build a pipeline of drug for mito. EPI-589 is in late stage preclinical now. They did not elaborate on this.

• I posted about the Japan announcement recently. “Edison is very bullish on the partnership.” The company has a long history of pioneering drugs. I don’t think this means a lot to the US right now, but it sure doesn’t hurt to have an infusion of money into the drug company in general.

Update on EPI-743 Trial in the US

Edison is very pleased with the progress to date. They hope to have enrollment finished by the end of June 2013.

·         Four study sites. Seattle is NOT up and running yet, but they said it would be enrolling soon. There is significant documentation, data and criteria for Phase IIB of this trial. Edison is “very satisfied with the current progress.”

·         They hope to release data on this trial by Q4 2013

·         They have 20 of the 30 required spots full.

"Compassionate use is a yes, the question is now simply when." FDA and Edison understand that compassionate care is a hurdle, but one they are willing to try and overcome. Edison said that once Phase IIB of the trial concludes, then perhaps an expanded access program might make sense in Q4 2013. 

The other problem with compassionate care is drug manufacturing. Edison needs to ensure they have enough supply of the drug and the vast majority of manufactured drug is being used for clinical trials. The drug requires  ½ kilo / patient / year.

Criteria questions:

In general, the FDA is requiring Edison to show the drug’s efficacy (effectiveness) as well as safety on as similar of a testing base as possible. That’s why people with trach’s, clinical diagnosis or non-age appropriate cases are not being included. An ideal testing situation would be identical twins and one twin in the placebo and one twin in the real drug to test. That’s not possible and we know that Leigh’s Syndrome manifests in every way under the sun – even if the genetic material is similar or identical as in the case of families. So, they must carefully screen applicants to ensure the group is as similiar as possible.

As far as the criteria of disease regression – they again want to prove their drug works to either stop or reverse the effects of a mitochondrial disease. If you’re stable or making good progress, then there could be false positives on the drug which wouldn’t help anyone. This is also why patients with trachs are not allowed. Oftentimes there is a period of improvement after a trach is put in, creating a false positive. Did you know that nearly 70 % of all Leigh’s patients have a trach.

Someone asked so “What does EPI-743 do” – the long answer as you’d expect it from a researcher…  The Rome EPI-743 trial was meant to first remove false positives from the previous trials. Then it:  1)treat individuals in an open trial 2)explore dose 3)way that the drug works / mechanism of action, register a blood test that would be consistent with how they believe EPI-743 works. The investigator team believes they achieved all three goals.

What we now know, mitochondria has another function than to just produce energy, it also balances and regulates our metabolism.

EPI-743 works to balance two major cogs of metabolism – balance and regulation. There is a second “cog” in the mito that does electronic counting and redox. Molecularly, EPI-743 docks or interacts with NQ01 and it works as a redox factor to replenish the glutathione cycle. Here is an article from a fellow mom and mito warrior on glutathione.

(HUH??).

It also works to combat oxidated stress.

I clearly have a lot of research to do now… I don’t even have appropriate links to share for all that. IF anyone smarter than me is reading this, please email or comment with any facts you have about this process.

But suffice to say, something good is going on. I will leave the science to those who are significantly smarter than me and I will happily be our son’s advocate to getting whatever they dream up next.

EPI-743 has had 75,000 dosing days – I’m relieved, happy and nervous that Will met the criteria of the Phase I expanded use and pray for access to this drug for everyone – no matter age, diagnosis or trach.

Please, if you haven’t already contact MitoAction, UMDF or NAMDAC to get on the radar for mitochondrial disease. We spend so much time doing the daily care of our kids that it’s so easy to push this to the bottom of the list. It could be the five minutes that changes your child’s life for the better.

Disclaimer *not a doctor here, nor researcher, just someone who typed as fast as they could on a conference call.