IntraBio Inc. has achieved a remarkable recruitment milestone for its Phase III pivotal trial evaluating N-Acetyl-L-Leucine (IB1001) as a potential treatment for Ataxia-Telangiectasia (A-T), completing enrollment in under two months and over-enrolling by 167%. The rapid recruitment underscores the urgent unmet medical need in the A-T community and positions the company to potentially deliver the first approved therapy for this rare neurological disorder.
Unprecedented Enrollment Success
The Phase III trial, designated IB1001-303, commenced enrollment in May 2025 across ten multinational sites spanning Germany, Slovakia, Spain, Switzerland, the United Kingdom, and the United States. Within just four weeks, the study achieved 100% of its target enrollment. The overwhelming interest from the A-T community ultimately led to over-enrollment of 167% within seven weeks, substantially increasing the statistical power of the trial.
"We are very pleased to have so rapidly completed recruitment for IB1001-303, and are grateful to the dedicated Principal Investigators, Study Teams, A-T Patient Organizations and families who helped us reach this milestone," said Taylor Fields, IntraBio's Chief Development Officer. "The A-T community's enthusiasm for N-Acetyl-L-Leucine is clear, and we remain determined to advance the development program as quickly as possible to help meet A-T patients' urgent unmet medical needs."
Study Design and Clinical Significance
The IB1001-303 study represents a randomized, placebo-controlled, double-blind crossover trial (NCT06673056) designed to evaluate the effects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia. The primary endpoint utilizes the Scale for the Assessment and Rating of Ataxia (SARA), a validated measure for assessing ataxia severity. The study design includes both a controlled crossover phase and a long-term open-label extension to explore symptomatic effects and longer-term clinical outcomes.
The trial was developed in partnership with key opinion leaders and A-T patient organizations, reflecting a collaborative approach to addressing this rare disease. Data readout is anticipated in the first quarter of 2026, marking a critical milestone in the potential development of the first approved treatment for A-T.
Community Impact and Collaboration
The rapid enrollment success reflects strong collaboration between IntraBio and the A-T patient community. Jennifer Thornton, Executive Director of the A-T Children's Project, emphasized the significance of this milestone: "We are thrilled with the news of this milestone, and feel one step closer to finally having the first approved treatment for A-T. We were very pleased to collaborate with IntraBio throughout the planning and enrollment of this trial, and the rapid recruitment is a testament to our community's excitement for, and collective hope about this therapy."
The A-T Children's Project's involvement highlights the importance of patient advocacy organizations in advancing rare disease research and ensuring that clinical trials meet the needs of affected families.
Regulatory and Development Context
N-Acetyl-L-Leucine remains an investigational drug for A-T and has not received approval from the FDA or any other regulatory authority for this indication. The successful completion of recruitment for this pivotal Phase III trial represents a significant step forward in the regulatory pathway for this potential therapy.
IntraBio Inc. operates as a global biopharmaceutical company focused on developing and commercializing targeted therapies for rare and common neurological and neurodevelopmental diseases. The company's platform technologies stem from decades of research and collaboration with universities and institutions worldwide, leveraging expertise from scientific founders at the University of Oxford and the University of Munich.
