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GRIN Therapeutics Marks Significant Milestone in Neurodevelopmental Disorder Treatment

On January 9, 2026, GRIN Therapeutics, Inc., a pioneer in therapies designed specifically for serious neurodevelopmental disorders, proudly announced the dosing of its first patient in the global Phase 3 Beeline trial. This trial, focused on the investigational drug Radiprodil, aims to evaluate its safety and efficacy in individuals suffering from GRIN-related neurodevelopmental disorder (GRIN-NDD) caused by gain-of-function variants.

Radiprodil operates as a selective negative allosteric modulator targeting the NMDA receptor GluN2B subunit. This means it seeks to not only alleviate symptoms but to directly address the biological foundations of GRIN-NDD, offering new hope to patients and their families. Traditional non-targeted anticonvulsant therapies have limitations, and Radiprodil’s specific approach could provide comprehensive treatment benefits.

Dr. Kristen Park, a pediatric epileptologist at Children’s Hospital Colorado and the principal investigator for the Beeline trial, commented on the collaborative nature of this groundbreaking study. She emphasized, “This study reflects a truly collaborative effort between investigators, patient communities, and industry partners. GRIN-NDD is a complex disorder with limited treatment options today. The Beeline trial gives us a chance to explore whether Radiprodil can also address cognitive and behavioral aspects of the disease, paving the way for the treatment options that patients and families desperately seek.”

Michael Panzara, Chief Medical Officer of GRIN Therapeutics, described this moment as a pivotal step, stating that for the first time, patients will have access to a drug that targets the very receptor responsible for their condition. This signifies a potential modification in the disease’s progression.

The Beeline trial is structured to assess Radiprodil’s effectiveness through disease-specific endpoints, including the GRIN-specific Clinical Global Impression (GRIN-CGI) scale developed in conjunction with caregivers. Traditional clinical outcome measures focusing on seizure frequency will also be utilized. The trial builds upon the promising results from the earlier Phase 1b/2a Honeycomb trial, where patients showed a significant median reduction of 86% in countable motor seizures.

During the Honeycomb trial, 71% of patients reported over a 50% reduction in seizures, with many achieving seizure-free days during the study. Despite the occurrence of seizures, healthcare providers and caregivers noted clinical improvement throughout the trial, underscoring the positive impact of Radiprodil. The safety profile indicated that most adverse events were linked to infections or the underlying disease rather than Radiprodil itself.

Conducted across various global locations, the Beeline trial cannot be understated in terms of its significance for GRIN Therapeutics and the GRIN-NDD community. The collaboration with Angelini Pharma for regions outside North America aims to enhance accessibility should Radiprodil receive approval.

About Radiprodil

Radiprodil's profile as a potent negative allosteric modulator highlights its potential in treating GRIN-NDD. It has gained recognition from regulatory agencies such as the U.S. FDA, receiving Breakthrough Therapy, Orphan Drug, and Rare Pediatric Disease designations, alongside a positive opinion for orphan designation from the EMA.

The Phase 3 Beeline trial aims to comprehensively evaluate the impact of Radiprodil on core aspects of GRIN-NDD, encompassing not just seizure management but also behavioral and functional outcomes. It is additionally being explored as a treatment for other conditions linked to NMDA receptor overexpression, including tuberous sclerosis complex and focal cortical dysplasia type II.

About GRIN Therapeutics

GRIN Therapeutics is committed to developing highly refined therapeutics for neurodevelopmental disorders. The company has made remarkable strides since its inception and remains dedicated to delivering solutions that provide hope to patients and their families. Information about the ongoing trials and their outcomes can be found on GRIN Therapeutics’ website, which serves as a resource for updated clinical research in this field.

This groundbreaking advancement encapsulates the dedication and efforts of researchers, medical professionals, and supportive communities, setting a new precedent for the treatment of neurodevelopmental disorders worldwide.