Aqneursa Approved for Niemann Pick Disease Type C Treatment in EU
IntraBio Announces Promising Results for IB1001 in Niemann-Pick Disease Type C
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New York, NY – July 29, 2025 – IntraBio Inc., a pioneering biopharmaceutical company dedicated to developing targeted therapies for rare neurological adn neurodevelopmental disorders, today announced compelling results from its Phase 2/3 clinical trial evaluating IB1001 (a formulation of N-acetyl-L-leucine) in pediatric and adult patients diagnosed with Niemann-Pick Disease type C (NPC). The study, which enrolled patients aged four years and older across Australia, europe, the United Kingdom, and the United States, demonstrated meaningful potential for IB1001 to modify the disease’s progression and offer neuroprotection.
Understanding Niemann-Pick Disease Type C (NPC)
Niemann-Pick Disease Type C is a rare,inherited metabolic disorder characterized by the body’s inability to properly process and transport cholesterol and other lipids. This leads to a buildup of these substances in various organs, notably the liver, spleen, and brain, causing progressive and severe neurological damage. Symptoms can vary widely but often include ataxia, dysarthria, cognitive impairment, and liver disease, considerably impacting quality of life and lifespan.The rarity of NPC, with an estimated prevalence of 1 in 100,000 to 1 in 150,000 live births, presents significant challenges in diagnosis and treatment growth.
The IB1001 Clinical trial: A Rigorous Approach
The clinical trial was meticulously designed to assess the safety and efficacy of IB1001. Patients underwent a Baseline phase to establish their initial health status.Following this, they were randomized in a 1:1 ratio to receive either orally administered IB1001 or a placebo for a 12-week treatment period.This blinded crossover design allowed for a direct comparison of the effects of IB1001 against placebo within the same patient.
Upon completion of the initial 12-week treatment, patients switched their study medication, receiving the choice treatment (either IB1001 if they were on placebo, or placebo if they were on IB1001) for an additional 12 weeks. This crossover element provided further valuable data on the drug’s impact.
Extended treatment and Long-Term Follow-Up
A significant aspect of the study was the provision for an open-label extension phase. Patients who successfully completed the initial randomized treatment periods were invited to continue receiving IB1001.This allowed for the evaluation of long-term safety and efficacy, with some participants being treated for over five years. This extended observation period is crucial for understanding the sustained benefits and potential long-term effects of IB1001 in managing a chronic and progressive condition like NPC.
About IntraBio: Advancing Neurological Therapies
IntraBio Inc. stands at the forefront of developing and marketing innovative, targeted therapies for a spectrum of neurological and neurodevelopmental disorders. The company’s robust platform technologies are the culmination of decades of dedicated research, fostering collaborations with leading universities and institutions globally. IntraBio leverages the profound expertise of its scientific founder, drawing upon extensive knowledge from the University of Oxford and the University of Munich. This deep scientific foundation underpins IntraBio’s commitment to addressing unmet medical needs in rare diseases.
for more details about IntraBio and its ongoing research, please visit the company’s website at IntraBio.com or connect with them on LinkedIn (@intrabio-inc).
References
- Bremova-etl t, et al.J Neurol 2022;269(3):1651-1662
- Patterson, Marc C., et al. “Disease-modifying, neuroprotective effect of N-acetyl-l-leucine in adult and pediatric patients with Niemann-Pick disease type C.” Neurology 105.1 (2025): E213589.
- Geberhiwot T et al. Orphanet J of Rare Dis. 2018;13:50
- Patterson MC, et al.Orphanet J Rare Dis. 2013; 8: 12; 3. North.