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IntraBio Completes Over-Enrollment for Pivotal Niemann-Pick Disease Type C Trial, Data Expected Q2 2023

3 years ago3 min read

Key Insights

  • IntraBio successfully completed recruitment for its Phase III pivotal trial of IB1001 (N-Acetyl-L-Leucine) for Niemann-Pick disease type C, enrolling 130% of target patients across 13 international sites.

  • The company over-enrolled by 125% due to high community interest and unmet medical need, substantially increasing the statistical power of the randomized, placebo-controlled, double-blind crossover study.

  • Data readout is anticipated before the end of Q2 2023, with the trial designed to support accelerated approval pathways with both FDA and EMA for chronic NPC symptom treatment.

IntraBio Inc has successfully completed recruitment for its pivotal Phase III trial evaluating IB1001 (N-Acetyl-L-Leucine) in patients with Niemann-Pick disease type C (NPC), enrolling 130% of the target patient population due to exceptional community interest and high unmet medical need.
The multinational trial, designated IB1001-301, is a randomized, placebo-controlled, double-blind, crossover study that commenced enrollment in September 2022. Over 70 NPC patients were screened across 13 trial sites spanning Australia, Europe, the United Kingdom, and the United States, including locations in the Czech Republic, Germany, the Netherlands, Slovakia, and Switzerland.

Accelerated Enrollment Reflects Community Need

"We are thrilled to have completed recruitment and thankful to the dedicated Principal Investigators and Study Teams, as well as NPC Patient Organizations worldwide, who helped us reach this milestone," said Taylor Fields, IntraBio's Chief Product Development Officer. "We are also very appreciative for the NPC community's tremendous enthusiasm and support of our development of IB1001, which enabled us to complete recruitment so quickly."
The company initially reached 100% of target enrollment in November 2022 and subsequently decided to over-enroll by more than 125% to increase the study's statistical power. This over-enrollment was completed by December 2022, substantially enhancing the trial's ability to detect treatment effects.

Trial Design and Regulatory Strategy

IB1001-301 investigates N-Acetyl-L-Leucine for both symptomatic and long-term neuroprotective effects in NPC treatment, with the primary endpoint based on the Scale for the Assessment and Rating of Ataxia. The study was designed in consultation with neurologists engaged in NPC treatment and NPC patient organizations to support a label for chronic treatment of NPC symptoms.
The pivotal trial was designed in consultation with both the US Food and Drug Administration (FDA) and the European Medicine Agency (EMA) to support an accelerated approval pathway for IB1001. Upon approval, IntraBio would be eligible for a priority review voucher from the FDA, which can be used or sold, with all sales of these vouchers in the past two years exceeding $100 million each.

Company Pipeline and Platform

IntraBio is a biopharmaceutical company with a late-stage drug pipeline focused on novel treatments for common and rare neurodegenerative diseases. The company's platform technologies result from decades of research and investment at premier universities and institutions worldwide, with clinical programs leveraging expertise in lysosomal function and intracellular calcium signaling from scientific founders at the University of Oxford and the University of Munich.
Beyond the NPC indication, IntraBio reports being Phase III ready for three other disease indications, though specific details were not disclosed.
Data readout from the IB1001-301 trial is anticipated before the end of the second quarter 2023, representing a critical milestone for both the company and the NPC patient community seeking new treatment options for this rare neurodegenerative disorder.
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