All is Not Quiet on the Trofinetide Front #2; A Balancing Act

* Warning* Looooong post! If you aren’t interested in financial aspects and just want an update on logistics that have affected the timeline, skip to the second half- there’s also a link to a new update from Acadia, the pharmaceutical company that will be handling the trial. 🙂

*Disclaimer* Everything in this post is my opinion, my way of making sense of the steps being taken and my interpretation [in my limited scope of understanding] of some of the financial/logistical aspects necessary to heed in getting Trofinetide to our children. Everyone should do their own research when considering stock options etc. and the ONLY purpose of this post is to try and explain aspects of getting Trofinetide to market that impacts the timeline.

I will be the first to tell you all, finances and stocks, stock prices, the balance companies need to strike to meet their shareholders’ expectations vs getting new products approved, whatever they may be, just goes right over my head. I want to interject here… Without stockholders there would NEVER have been this blog in the first place because there would be no Trofinetide, so I hope they make a killing. I do know, however, that these expectations are an integral part of how Trofinetide is making its way to market. No company could just plow ahead, throwing money into trials with nary a thought to the long term.

I liken this to…say, a small independent publisher with limited resources- right out of the gate they burn up all their funds publishing a multitude of books, paying illustrators and  printing costs and then find out they have no money left for marketing. The end result? Limited sales and small return on their investment, leading to a failure to thrive in the world of mega-publishers. Even I can see that’s bad business.

So, what we’ve got here is Neuren all the way over in Australia and they know that North America, specifically the United States, is the target to achieve the best chance at Trofinetide getting to market. It began nearly 15 years ago with Intrepid-2566, a program funded by the US Army in conjunction with Walter Reed Army Hospital to address traumatic brain injuries (TBI) in soldiers. This is an interesting story all on its own and I encourage anyone interested to look it up. I’m emotionally invested in finding a way to improve mortality due to TBI as my best friend CPO Theodore Moreland, USN SEAL, SEAL Team 2, died from a TBI sustained in a training accident. If my child did not have Rett syndrome, this whole blog would still probably have started with Trofinetide, just Intrepid-2566 instead of the Texas trial. But, I digress.

Let’s just jump to the present because that’s where all our focus is. I read every announcement Neuren puts out (the stock ones I try to make sense of, but so far I can’t make hide nor hair of any of those, lol). It is mind boggling all the steps it takes to keep this balance because if the scale tips it most certainly will tip to the wrong side. Like it or not, funding a trial takes a LOT, lots and lots, of money, staggering amounts. There was no way Neuren could do it on their own; if we had to wait until they had enough funding on their own we wouldn’t be looking at a pivotal trial starting this year, probably not even in this decade. To this end, Neuren partnered with a US company-Acadia Pharmaceuticals.

This partnership came with all kinds of incentives for both companies. There are a few I understand.

  • Acadia funds the trial (upwards of 50-60 millions of dollars) and gets the North American rights to Trofinetide and first dibs on the rights in Europe and rest of the world.
  • Neuren gets this trial funded, keeps all rights outside of North America AND gets access to all the data from the trial so they don’t have to repeat it. They also got a significant down-payment of $10,000,000
  • Acadia gets the North American rights to Trofinetide in Fragile X, another devastating genetic disease which occurs mostly in males. There has been one Phase II trial so far. In the case of Fragile X, there are too many dendrites on the nerve cells causing sensory overload among other issues. Trofinetide takes the extra ones away, normalizing the number and length just as it does in Rett syndrome. (Isn’t science grand!?!)
  • Neuren keeps the rights for Fragile X outside of North America with similar conditions as they have with Rett syndrome and Acadia.
  • Neuren gets 1/3 of revenue from any “Pediatric Voucher” granted by the FDA. This is a program that grants an expedited marketing review (from 10 months to 6 months)-speeding up the time to market. Because Trofinetide already has Fast Track and Orphan Drug status, it automatically has an expedited review, so this can be sold to another company. It can go for $100,000,000!!!

That’s all about finance I understand. It’s not much, I know, but this kind of deal took a lot of brokering. I do know, having met the team at Acadia in San Diego, that I personally have complete confidence in having them on our side; clearly Neuren feels the same. There are three resources I can recommend to those who want more insight into this deal. The first is Hotcopper, the Australian stock exchange forum; the second is Neuren’s website and all of its announcements; and this podcast with Dr. Richard Treagus, Executive Chairman.

I will also include this link from Pitt Street Research that goes over all things Neuren in regards to stock pricing etc.


Now, on to some logistics of a Phase III Trofinetide trial that have impacted the timeline. Here, again, the FDA has played a major part. The FDA dictates the standards for any trial in America-from length, number of participants, to the amount of stock a pharmaceutical company has to have in order to complete the trial within the guidelines decided upon. Enter Acadia! Here is a nice overview on this partnership-AusBiotechInvest and Partnering 2018 

First and foremost, no matter what anyone may hear from their Rett specialist, the Phase III has not been “held up” because of the “drug company”; No Acadia=no trial, it’s as simple as that.

As discussed in the previous blog post, the manufacturing enough trial medication is the largest hurdle that needs to be jumped over. It’s not just making Trofinetide; it’s making it storable, making a multiple dose compound so parents aren’t given a hundred tiny little bottles; it’s making doses in a variety of strengths based on a four-tiered system governed by the weight-classes of participants. It even had to take into account that some people are allergic to strawberry which is the original flavoring and make it so that no one can be denied Trofinetide because they are allergic to strawberry; how much would that suck?!?

When I met with the San Diego Acadia team, these were the issues foremost in my mind and I was happy that there was a completely satisfactory answer for every one. The other substantial concern for parents is the cost of participating, so I’m happy to report that Acadia and will work together to help in reimbursing for reasonable travel costs associated with the trial. A shout out to our very own Colleen English who rocks Rettland in so many amazing ways. Without her vision, seeing a need and taking up the cause, the cost of participation would put trials out of reach for many.

Lastly, I think we need to take a look at and appreciate the time consuming effort that must go into choosing sites, creating standards of reporting, training personnel and all the thousand little things we, as parents, don’t even know go into this sort of thing.

Just yesterday, I received a message from Acadia and they sent along this update, shared on When you read it, you will see that they are striving to make this trial as easy as possible to participate in, within the need to adhere to strict guidelines set by the FDA. Of particular note is their hope to include even more sites than the Phase IIb (pediatric trial).

If you’ve made it to the end of this post… well done! and thank you. 🙂 Hope it’s answered some questions you may have had.



This entry was posted in Acadia, cure, FDA, Neuren Pharmaceuticals, Orpan Drug Status, orphan drug designation, Rett Research, Rett Syndrome,,, TBI, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, Trofinetide Phase III and tagged , , , , , , , , , , , , , , , , , , , . Bookmark the permalink.

7 Responses to All is Not Quiet on the Trofinetide Front #2; A Balancing Act

  1. Christine Roberts says:

    Very informative.

  2. Stacy says:

    Thanks for update!! I’m curious about whether Canadians will be able to participate in the Phase III trial. Do you have any insight into this?

  3. melelllan says:

    I did address the question of possible international participants who could afford to get to the US to participate. However, I didn’t get a definitive answer. I think we are still too far away to know one way or the other, yet.

  4. Harsh Miglani says:


    Can Indians participate in this test ?

    Harsh Miglani

  5. melelllan says:

    All the sites will be in America. I haven’t heard yet if they are willing to accep international participants. It would be very expensive for you as I don’t think international travel would be reimbursed, There is a trial in India right now for breath-holding. If you are interested go to

Leave a Reply

Fill in your details below or click an icon to log in: Logo

You are commenting using your account. Log Out /  Change )

Google photo

You are commenting using your Google account. Log Out /  Change )

Twitter picture

You are commenting using your Twitter account. Log Out /  Change )

Facebook photo

You are commenting using your Facebook account. Log Out /  Change )

Connecting to %s