Nyrada Inc. has reported positive preclinical results for its lead drug candidate NYR-BI03, demonstrating efficacy in preventing secondary brain injury following traumatic brain injury (TBI), while simultaneously announcing the commencement of Phase 1 clinical trial recruitment for the compound.
The preclinical study, conducted under a Cooperative Research and Development Agreement with the Walter Reed Army Institute of Research (WRAIR) and UNSW Sydney, evaluated NYR-BI03 in a rodent model of penetrating TBI designed to replicate severe head injuries commonly sustained by military personnel.
Promising Preclinical Results in Traumatic Brain Injury
The collaborative study involved 28 test animals that received continuous intravenous infusion of either NYR-BI03 or a vehicle control over a 48-hour period. High-resolution magnetic resonance imaging (MRI) was conducted by UNSW Sydney to assess brain tissue integrity under blinded conditions.
Despite the severe level of penetrating TBI produced using the WRAIR model, NYR-BI03 provided a statistically significant level of neuroprotection (p = 0.043; ANOVA). Fractional anisotropy (FA) MRI analysis identified neuroprotection across six consecutive brain scan levels spanning the region of maximum penetration by the probe.
"The injuries produced using the WRAIR model reflected a severe level of penetrating TBI," noted the company in its announcement. The deviation from uninjured brain tissue integrity was compared between NYR-BI03 treated versus vehicle group at each scan level, with results showing meaningful protection of brain tissue.
Phase 1 Clinical Trial Initiation
Building on these promising preclinical results, Nyrada has begun recruiting for its Phase 1 clinical trial of NYR-BI03. The trial is designed as a double-blind, randomized, placebo-controlled, dose-escalating study comprising five cohorts of eight healthy participants.
Each cohort will receive an intravenous dose of either NYR-BI03 or placebo over three hours, with six active and two placebo participants per cohort. A Safety Review Committee will review accumulated safety and pharmacokinetic data after each completed cohort.
The company expects final trial readouts in the third quarter of 2025.
Mechanism of Action and Broader Applications
NYR-BI03 is a small molecule, first-in-class investigative treatment that works by blocking Transient Receptor Potential Canonical (TRPC) ion channels. This mechanism appears to provide both neuroprotection and cardioprotection.
In February 2024, Nyrada announced preclinical stroke study results showing NYR-BI03 achieved a statistically significant neuroprotective effect, rescuing 42% of brain tissue in the penumbra region of treated animals.
Further expanding its potential applications, in October 2024, the company reported results from a preclinical coronary heart disease study, which demonstrated that NYR-BI03 provided an 86% cardioprotective effect following myocardial ischemic-reperfusion injury.
Potential Clinical Impact
The development of NYR-BI03 addresses significant unmet medical needs in both neurological and cardiovascular conditions. Traumatic brain injury remains a major cause of death and disability worldwide, with limited treatment options available for preventing secondary brain damage after the initial trauma.
Similarly, effective treatments for protecting brain tissue during stroke and heart tissue during myocardial infarction could significantly improve patient outcomes and reduce the burden of these conditions.
The progression to Phase 1 clinical trials represents an important milestone in the development pathway for NYR-BI03, as it transitions from preclinical research to human studies. The trial will provide crucial data on the compound's safety profile and pharmacokinetics in humans, paving the way for potential Phase 2 efficacy studies in specific patient populations.
