Positive Top-line Results for Phase 3 Trofinetide Trial

Categories: Blog, Homepage, News

Melissa Kennedy, Chief Executive Officer

On December 6, the Rett syndrome community received some very encouraging news: Acadia Pharmaceuticals reported that top-line results for its Lavender Phase 3 clinical trial for trofinetide showed significant improvement over placebo. With these positive findings, Acadia will request a pre-New Drug Application meeting with the FDA in the first quarter of 2022.

This is Really Big News. We are closer than ever to the first FDA-approved treatment for Rett syndrome.

The trofinetide journey began many years ago when IRSF’s (then) Chief Science Officer, Dr. Steve Kaminsky, determined that trofinetide could have treatment potential in Rett syndrome. The IRSF Board of Directors made major financial investments in testing trofinetide, including supporting early phase clinical trials of the compound. That decision was a game-changer for the Rett community.

Upon hearing the Acadia news, Dr. Kaminsky was quick to say that the main reason trofinetide has made its way to this point is because of families that chose to participate in the trial.

“Many people put a lot of time and effort into getting the drug to this point. However, the real credit goes to all the girls and parents who put so much of their lives into the effort. [They] are the true heroes in the story.” – Dr. Steve Kaminsky

Truly, we are all indebted to the families who participated in Phase 2 and Phase 3 studies. The Phase 3 clinical trial enrolled over 180 participants. And we know many more around the world wished they could have been a part of the trial. These families demonstrated courage and commitment to advancing hope toward a treatment for Rett syndrome.

The road to a cure begins with treatments. For over a decade, IRSF has been working to fill the clinical trial pipeline with compounds that show promise. Because of the complexity of Rett syndrome, we will need as many solutions as possible. Hope has never been brighter. Eight years ago, only one company was working on a treatment for Rett syndrome; today, there are over 20.

We celebrate this milestone but recognize that we are very early in the race. IRSF will continue to invest in ground-breaking basic research. We will continue to invest in existing compounds that could be beneficial treatments. We will ensure that the network of Rett clinics throughout the U.S. is fully prepared to engage in clinical trials. We will not stop until there are numerous options available to families living with Rett.

While we’re encouraged with the top-line results reported by Acadia Pharmaceuticals, we must remember this is only top-line results, and further analysis of the trial data is still underway. We must also keep in mind that the FDA has a rigorous process that ensures the benefits of any potential treatment outweigh the risks or side effects seen in the clinical trial.

We want to congratulate Acadia Pharmaceuticals on their news and thank them for their dedication and commitment to the Rett syndrome community. We wish them all the best in the subsequent steps of the FDA-approval process. We’d also like to thank them for inviting IRSF’s Dr. Dominique Pichard to share about Rett syndrome at their recent investor meeting.

At IRSF, we have been anticipating this moment for over eight years. We are grateful to those donors who put their faith in our scientific investment strategy and our research portfolio. Our continued promise is to use your donations to support the most promising science. The Acadia announcement earlier this month is what happens when IRSF puts your donations to work. Please continue to support our Foundation. We will deliver.

With Hope and Gratitude,

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