- IntraBio's pivotal trial for Niemann-Pick disease type C enrolls 100% of target patients
- IntraBio plans to "over enroll" trial by over 125% and complete recruitment December 2022
- IntraBio's pivotal trial data readout expected Q2 2023
OXFORD, UK / ACCESSWIRE / November 22, 2022 / IntraBio Inc announced today that it has enrolled 100% of the target number of patients for its multinational pivotal trial, IB1001-301, "Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study".
Enrollment for IB1001-301 commenced in September 2022, and patients were screened at trial sites in Australia, the Czech Republic, Germany, the Netherlands, Slovakia, Switzerland, the United Kingdom, and the United States. To increase the study's power, IntraBio plans to "over enroll" the trial by over 125% and expects this additional recruitment to be completed by year-end. Data readout is anticipated before the end of the second quarter 2023.
IB1001-301 investigates N-Acetyl-L-Leucine (IB1001) for both the symptomatic and long-term neuroprotective effect of treatment of Niemann-Pick disease Type C (NPC), 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 the treatment of NPC and NPC patient organizations. The study has been designed to support a label for the chronic treatment of NPC symptoms.
IB1001 Development Program / Approval Pathway
This pivotal trial was also 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 of IB1001, IntraBio would be eligible for a "priority review voucher" by the FDA, which can be used or sold, with all sales of these vouchers in the past 2 years over $100 million each. IntraBio is also Phase III ready for three other disease indications.
IntraBio Inc is a biopharmaceutical company with a late-stage drug pipeline, including novel treatments for common and rare neurodegenerative diseases. IntraBio's platform technologies result from decades of research and investment at premier universities and institutions worldwide. Its clinical programs leverage the expertise in lysosomal function and intracellular calcium signalling of its scientific founders from the University of Oxford and the University of Munich.
IntraBio's management team and consultants have a successful track record of drug development in the USA and Europe. This team translates innovative scientific research in the fields of lysosomal biology, autophagy, and neurology into novel drugs for a broad spectrum of genetic and neurodegenerative diseases so to significantly improve the lives of patients.
IntraBio Inc is a US corporation with its principal operations in Oxford, United Kingdom.
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SOURCE: IntraBio Inc.