Statement from GRIN Therapeutics on Radiprodil to our Community

Earlier this month, at the 2021 GRIN Virtual Conference, GRIN Therapeutics announced plans to initiate the first clinical study on Radiprodil. 

See the statement below from GRIN Therapeutics:

The establishment of GRIN Therapeutics this month is not only the result of two years of work by our CEO
Dr. Pierandrea Muglia. It is also the result of the tremendous efforts of the GRIN community: The idea of this company was inspired by meeting the GRIN community two years ago at the 2019 CFERV Conference on
GRIN Variants held at Emory University, in Atlanta.

Dr. Muglia came to this conference knowing that Radiprodil had a mechanism that suggested it might help
patients with gain of function genetic variants in GRIN2B. What he didn’t expect was the maturity all the work produced by the GRIN community—from clinicians, researchers, advocates, and families working together.

This community inspired him to form GRIN Therapeutics to focus entirely on developing potential treatments for the GRIN community—beginning with Radiprodil. This passion has fed and inspired our small, patient-focused team, who we look forward to introducing to you in the coming weeks. Earlier this month, at the 2021 GRIN Virtual Conference, we announced our plans to initiate the first clinical study on Radiprodil in children living with GRIN2B gain of function, with a goal to establish appropriate dosing based on safety and initial effect.

We also shared the following details.

Radioprodil:
• Is a pediatric phase 2 ready, selective NR2B negative allosteric modulator
• Has safety database from more than 400 adults
• Was used safely in three treated infants with an effect on seizures/spasms in these treatment-resistant patients
• Rectifies in-vitro overactive –NR2B gain of function of specific variants (N615I; V618G)
• Shows anticonvulsant activity in several preclinical seizure models
• Has potential to treat seizures and other symptoms in children with GRIN2B gain of function variants

In the next six to nine months we will develop a pediatric formulation and complete a submission to
regulatory bodies. Most importantly, we will also engage in dialogue with key leaders from the GRIN
community. The path to clinical trials is complex, however, we see you as our most important partners in the process to get to the first clinical trial in the GRIN2B.

Additionally, we’d like to introduce and share a statement from Hillary Savoie, Head of Community Engagement and Communications for GRIN Therapeutics:

On behalf of GRIN Therapeutics and Dr. Pierandrea Muglia, we would like to thank the GRIN community for inviting us to present at the 2021 GRIN Virtual Conference, where Dr. Muglia spoke about the recently formed biotech, GRIN Therapeutics. The company will be primarily focused on developing Radiprodil for children with gain of function genetic variants in GRIN2B.

I would also like to introduce myself: I am the Head of Community Engagement and Communications. I have also
spent the last decade as rare disease advocate and the mother of a medically complex child. I joined GRIN Therapeutics to help facilitate the collaborative work with the GRIN community that is required to develop a potential treatment in an ultra-rare disorder. I took on this role so enthusiastically because your community is at the center of the story how this company was formed.

We look forward to sharing the next steps on this path with all of you.
-Hillary Savoie