A Little Trip, A LOT of Hope!

A few weeks ago, Katelin and I were invited to go to Acadia! On a whim, I googled “Acadia” to see what might popup. First up, beautiful Acadia National Park. Definitely would like to go there! Actually, I can’t believe I had never heard of it before and missed it on our way out West, may have to rectify that one day. Next up, Acadia was a colony up in the Northeast. Eh, been a part of a previous colony for most of my life ;). But, as thrilling as that park looks, I went to an Acadia that’s  EVEN BETTER.

We were invited to meet the new Acadia team that will be about all things Trofinetide. This was a tremendous opportunity for both of us and I want to thank Neuren’s James Shaw and Dr. Nancy Jones for extending the invitation.

Before I get started on our glowingly, awesome day, I want to touch briefly on some smack (I’m definitely dating myself with this slang, lol) going around about Acadia and it’s  NUPLAZID® (pimavanserin) for Patients with Hallucinations and Delusions Associated with Parkinson’s Disease Psychosis. Just to be clear the FDA came out with a reaffirmation of it’s positive affect and positive benefit: risk profile. You can read about it here. Now THAT’s out of the way…

I was SUPER nervous, I really wanted to make a good impression, plus I hadn’t had a reason to put makeup on in awhile, but what a morning. Murphy’s law was in full effect. I did not have shoes to match my outfit. I’m certain there are quite a few parents out there who can relate, lol. The only store open was Target. With 10 minutes left to be on the road, I rush in with Katelin, grab the closest sales associate and say…10 minutes, cream color shoes… let’s go. I managed to try on about 7 different pairs of shoes, including a small boot that got an “All you need is a cowboy hat” from the associate, so of course those were tossed off. But, even though they were renovating the department, we DID IT! And we were off.

Acadia is in this big corporate compound, I found it to be a bit of a maze, parked in the back parking lot, oops, no ramp, walk ALL the way around, up hill mind you, only to met by a big hug and smile from James that made if all worth while.

We were ushered into a nice conference room and Katelin got the head of the table, lol, the honored guest. And then, we got to meet part of the team- So here you go, YOUR Acadia/Neuren team, working to help our children!

Here I thought all this time up to the meeting, “Aw, isn’t that nice they want to meet Katelin and have some lunch.”(Best box lunch by Champagne Bakery I’ve ever had, and the cookies! Yum ) But, that wasn’t all…oh, no. They showed up with NOTEPADS and then BOOM the questions started. So, just so you know, this team is committed! I was surprised at how truly imperative it was for them to tackle this next trial and further production from every possible angle-from how to fundraise, market, make the trial easier on parents, and to understanding obstacles and any information that would make it easier for US, the parents and our children.

Their multi-angle approach is an impressive outreach for a pharmaceutical company, imo. They are extremely cognizant on how difficult life is in Rettland and are willing to make compensations and think outside the box on how they move forward.

Now, I know some parents are like… great, cut to the chase, what does this mean for OUR children. Well, I did get an opportunity to ask some questions as well, some of which I can share with you. First, I found them all very receptive to my questions and James did an excellent job of knowing just who to turn to, to answer them. (From someone who has difficulty remembering names of people I just met, I found this to be a remarkable skill, lol.)

One of their questions, was what is one way to make it easier on parents? My first thought from Katelin’s trial was- you cannot send parents home with a whole bunch of individual doses. So, you all will be happy to know they are working on providing a multi-dose bottle.

Another question/statement that I had was-It is not reasonable to expect parents to travel for a simple blood draw and that perhaps they can come up with a “kit” that can be mailed to parents. It is very common practice at larger labs to draw the blood and either process it for mailing or give it back to parents to mail. Hopefully, something like that can be worked out.

One major question I had-during the pediatric trial, it was discovered that lighter weight girls did not retain as much drug in their systems as heavier girls- Has this been corrected? The answer was yes. James let me know that there is a four tier dosing system-light girls, slightly heavier, a little heavier and then the heaviest-that adjusted for this. I was very pleased to hear this, as it would have been difficult to have a consistent trial without it.

My last question was-Will this be available for boys? James assured me that, if/when (If, hahahaha) it was approved, the intention is that it would simply be indicated in Rett syndrome and not sex specific; so, boys are IN!

What I absolutely do NOT know is what the age cutoff will be for the Phase III trial. I’m not exactly sure it’s been definitely decided and if it has been, I wasn’t told what that will be.

Now, I know the question that is screaming out of all of you… WHEN??????? Neuren and Acadia have announced they are looking at late 2019. Take a look at Neuren’s September Investor Report for a more in-depth look at the Neuren/Acadia partnership.

I also know that this is going to bring some frustration, anxiety, anger. “What is taking SO long!” Just from the short list of concerns/questions above, you can tell that they want to get this RIGHT! Better to dot all your i’s and cross your t’s before you start, than play catch up and have the FDA be unhappy about that.

Just as Katie is the center of attention here….

I left with a sense of relief and comfort, after our little roundtable conversation, that ALL of our children are the focus of their effort. And, I believe beyond a shadow of doubt, that in these two sets of hands-Neuren and Acadia- that our children are in better hands than AllState.

Thank you to the Acadia team on both coasts and James Shaw and Dr. Nancy Jones again, for this amazing opportunity. Hoping for more pictures like this one, James and Nancy. (I won’t mind having quite a few 😉 )