GRIN Therapeutics has dosed the first patient in its global Phase III Beeline trial evaluating investigational radiprodil for individuals with GRIN-related neurodevelopmental disorder (GRIN NDD) who have gain-of-function variants.
The Phase III Beeline trial aims to assess the safety and efficacy of radiprodil in this patient group, focusing on the underlying biology of GRIN-NDD.
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Radiprodil acts as a selective negative allosteric modulator of the GluN2B subunit of the NMDA receptor. It is intended to address the dysregulation resulting from receptor overactivation, with the potential to affect various aspects of GRIN-NDD.
This approach could provide benefits compared to currently available non-targeted anticonvulsant treatments.
The Beeline trial utilises a disease-specific endpoint, the GRIN-specific Clinical Global Impression (GRIN-CGI) scale, developed with caregivers’ input. It also includes traditional clinical outcome assessments and seizure reduction measures.
The current study follows results from the Phase Ib/IIa open-label Honeycomb trial, in which patients receiving radiprodil showed a median decrease of 86% in countable motor seizures (CMS) from baseline.
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By GlobalDataIn the previous trial, 71% of participants experienced a reduction in seizures greater than 50%, and six out of seven patients were free of seizures for at least 80% of days during the eight-week maintenance phase.
Clinicians and caregivers reported overall improvements based on established clinical assessment tools. The most frequently observed adverse events were associated with infections or the underlying condition; serious adverse events were also related to these factors and were considered unrelated to radiprodil.
The Phase III Beeline trial is being carried out in several regions worldwide. GRIN Therapeutics’ partnership with Angelini Pharma beyond North America is intended to facilitate potential access to radiprodil in Europe and additional territories if regulatory approval is obtained.
GRIN Therapeutics’ chief medical officer Michael Panzara said: “With the first patient dosed, the Beeline trial marks a pivotal moment for GRIN Therapeutics and the community.
“For the first time, through the Phase III Beeline trial, patients have the opportunity to receive a drug that specifically targets the abnormal receptor leading to their condition – a drug that aims to address the underlying biology of GRIN-NDD, potentially modifying the course of disease.
“In four years, we have advanced from company inception to a global Phase III programme built around deep scientific understanding of the disease and engagement with key stakeholders, including families and global regulatory authorities.”
