IntraBio Inc. Completes Recruitment for IB1001 A-T Pivotal Clinical Trial

July 01, 2025 at 08:00 AM EDT

IntraBio’s Phase III Pivotal trial for Ataxia-Telangiectasia has completed recruitment in under two months
Pivotal Trial over-enrolled by 167% in under 2 months
Data readout is expected Q1 2026

IntraBio Inc. announced today that it has completed recruitment for its Phase III Pivotal Trial “IB1001-303”, Effects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T): A randomized, placebo-controlled, double-blind, crossover study (NCT06673056).

Enrollment for the Phase III trial commenced in May 2025 and was conducted across ten multinational trial sites in Germany, Slovakia, Spain, Switzerland, the United Kingdom, and the United States. After four weeks, 100% of the target population was enrolled. Due to the high level of interest from the A-T community, IntraBio ultimately over-enrolled the trial by over 167% in 7 weeks, substantially increasing the 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.”

Jennifer Thornton, Executive Director of the A-T Children’s Project, commented, “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. We look forward to continuing to work with and support the A-T community and IntraBio to help as many families as possible potentially benefit from this therapy.”

Data readout for the IB1001-303 trial is anticipated in the first quarter of 2026.

About IB1001-303

IB1001-303 is a Phase III clinical study evaluating N-Acetyl-L-Leucine (IB1001) for the treatment of Ataxia-Telangiectasia (A-T), using the Scale for the Assessment and Rating of Ataxia (SARA) as the primary endpoint. The study includes a randomised, placebo-controlled, double-blind crossover phase, followed by a long-term open-label extension. It was designed in partnership with key opinion leaders and A-T patient organisations to explore both symptomatic effects and longer-term clinical outcomes.

N-Acetyl-L-Leucine is an investigational drug for A-T and has not been approved by the FDA or any other regulatory authority for this indication.

About IntraBio

IntraBio Inc. is a global biopharmaceutical company that develops and commercializes targeted therapies for rare and common neurological and neurodevelopmental diseases. IntraBio's platform technologies result from decades of research and collaboration with universities and institutions worldwide, and leverages the expertise of its scientific founders from the University of Oxford and the University of Munich.