IntraBio Inc announced today that it has completed recruitment for its Pivotal Trial, Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study (IB1001-301 - NCT05163288 ).
- IntraBio’s pivotal trial for Niemann-Pick disease type C has completed recruitment
- IntraBio’s pivotal trial data readout expected Q2 2023
OXFORD, UK / ACCESSWIRE / December 8, 2022 / IntraBio Inc announced today that it has completed recruitment for its Pivotal Trial, Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study (IB1001-301 - NCT05163288).
Enrollment for IB1001-301 commenced in September 2022. Over 70 NPC patients were screened across 13 multinational trial sites in Australia, Europe, the United Kingdom, and the United States. Given the NPC community’s extremely high interest in IB1001-301 and their high unmet medical need, IntraBio enrolled 130% of target patients for the trial, substantially increasing the power of the study.
“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 quicky. We remain determined to get IB1001 approved as quickly as possible to help meet NPC patients’ urgent unmet medical need.”
Data readout is anticipated before the end of the second quarter 2023.
About IB1001-301
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 / ApprovalPathway
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.
About IntraBio
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’smanagement 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.
For further information, please contact:
Cass Fields
Vice-President of External Affairs
Email: ccfields@intrabio.com
SOURCE: IntraBio Inc.
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