This past week has been a roller coaster of emotions. Last Tuesday November 12,2014, two days after Maggie’s 4th birthday. Neuren Pharmaceuticals released the information regarding the NNZ trial for Rett Syndrome. This is a big deal. There are several trials going on right now, which is great, but the process of drug trials is long and arduous.  There is a lot of waiting. So when we heard on Sunday that there should be an announcement by Tuesday, we were excited, anxious, nervous, hopeful and everything else.  Remember, there aren’t any treatments or a cure for Rett Syndrome.  When we were diagnosed with Rett and met with Dr. Neul at Baylor (he plays a huge part in the NNZ trial), we were told about the trials going on, but also knew it would be some time before treatments would be available. But here we are less than a year after Maggie’s diagnosis and there has been a milestone in Rett treatment development.  This is huge. I also thought it would be good to explain to family and friends what this press release means and what needs to happen for Maggie to get this drug.

Highlights I took from the Neuren press release….

Neuren’s NNZ-2566 successful in demonstrating clinical benefit in Rett syndrome Phase 2 trial

• Achieved primary endpoint – both dose levels of NNZ-2566 were well tolerated after 28 days of treatment and no safety concerns were identified

• Higher dose (70mg/kg twice daily) exceeded the pre-specified criteria for improvement in core efficacy measures compared with placebo

• The clinical benefit in the trial encompassed core symptoms of Rett syndrome and was observed in both clinician and caregiver assessments

• Meeting with FDA expected in Q1 2015 to discuss further development in Rett syndrome

• Results will enable applications for both Orphan Drug and Breakthrough Therapy designation

What does this mean?  This all means that the drug is safe and what they were wanting it to accomplish it did and then some.  Which is exactly what we needed to happen in the trial.

What does this mean for us for Maggie? Well there are still some obstacles to overcome before the drug is put on the market.  First the FDA has to approve the drug, which is no easy task.  Then the NNZ trial will do another phase.  This phase will be another placebo controlled study, but it will be for pediatrics.  We are not sure if Maggie will qualify for this phase but are hopeful.

After that, there may be a phase 3 trial which would be an open label trial, meaning everyone knows they are on the drug, just more people are on it.  So if the FDA approves the drug and Phase 3 does well, I’m guessing the drug will be on the market within the next 2-3 years (my estimate).  I can’t tell you how many times I have broken down crying because of this awesome news.  The hope for treatment is real. There is a possibility that Maggie will have a drug that can help her…within her childhood.  So now we wait.

Will this drug treat all of Rett Symptoms?  We just don’t know, each girl is different, so it most likely will take a cocktail of drugs to help. The way a treatment was explained to me is, some girls might be fine with one drug, some will need several drugs to help.  Similar to AIDS patients, we need to find the right cocktail of drugs for each girl. This drug is possibly the first of that cocktail.

With all that said.  This is exciting news. Our work isn’t done yet.  It is still a long road but there is palatable hope.

Here is a link to the press release by, who is funding the trial…click here

And here is a link to a blog by the chief science officer at, who explains in better detail what the drug does, etc…click here

Disclaimer: this post was written by Jenny, which means all tears were shed by Jenny too.

4 thoughts on “NNZ trial press report released”

  1. This is so exciting and hopeful. We will wish for continued success of this new drug and perhaps it can get to market on a faster track. Progress for Maggie is in sight! Score one for science!
    Gloria and Mel

  2. Wow, this is tremendous news. You guys are so amazingly involved with every step. This must be hard while taking care of Maggie. But, let’s all hope this is a huge movement! And successful.
    Love, Gloria and Mel

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