IntraBio Announces Positive Results from IB1001-301 Study of Aqneursa (Levacecarnil) for Niemann-Pick Disease Type C

[City, State] – [Date] – IntraBio Inc., a biopharmaceutical company focused on neurological and neurodevelopmental disorders, today announced positive results from its multinational, randomized, placebo-controlled crossover study, IB1001-301 (NCT05163288), evaluating the safety and effectiveness of Aqneursa (levacecarnil) in pediatric and adult patients with Niemann-Pick disease type C (NPC).

Niemann-Pick disease type C is a rare, autosomal-recessive lysosomal storage disease that is fatal at an early stage. The condition manifests with systemic, psychiatric, and neurological symptoms, including cerebellar ataxia. NPC is a chronic and progressive disease characterized by rapid degeneration of the cerebellum and vital organ systems, substantially impacting patients’ quality of life.

The IB1001-301 study enrolled patients aged 4 years and older at study centers in Australia, Europe, the United Kingdom, and the united States. Following a Baseline phase,participants were randomized (1:1) to receive either orally administered IB1001 (Aqneursa) or a placebo for 12 weeks. After the initial 12-week treatment period, patients switched to the other study medication for a subsequent 12-week period. Patients who completed the study had the prospect to enroll in an open-label extension phase, with some receiving treatment for over five years.

IntraBio is dedicated to developing and marketing targeted therapies for rare and common neurological and neurodevelopmental disorders. The company’s platform technologies are built upon decades of research and collaborations with universities and institutions globally, leveraging the expertise of its scientific founders from the university of Oxford and the University of Munich.

More facts about IntraBio can be found on the company’s website at intrabio.com and on LinkedIn (@intrabio-inc).

References:

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2. 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.
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5. AQNEURSA. Prescribing Information. IntraBio Inc
6. Burton BK, Ellis AG, Orr B, et al. estimating the prevalence of Niemann-Pick disease type C (NPC) in the United States. Mol Genet Metab 2021; 134: 182-187.doi: 10.1016/J.Ymgme.2021.06.011
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About Niemann-Pick Disease Type C (NPC):

Niemann-Pick disease type C (NPC) is a rare, inherited metabolic disorder that affects the body’s ability to process cholesterol and other fatty substances. This leads to a buildup of these substances in various organs, including the brain, liver, and spleen. NPC is a progressive disease that can cause a wide range of symptoms,including neurological problems such as ataxia (loss of coordination),difficulty with speech and swallowing,and cognitive impairment. Psychiatric symptoms can also occur. The disease is typically fatal at an early age, and there is currently no cure. Treatment focuses on managing symptoms and improving quality of life.About Aqneursa (Levacecarnil):

Aqneursa (levacecarnil) is an investigational therapy being developed by IntraBio for the treatment of Niemann-Pick disease type C and other rare neurological disorders. It is a novel, orally administered small molecule designed to address the underlying cellular dysfunction associated with these diseases.

Contact:

Cass Fields
Vice-President of External Affairs
ccfields@intrabio.com
intrabio.com