IB1001 Effective for NPC Treatment, Study Says

According to Newswise, data from a recent clinical trial evaluating IB1001 for patients with Niemann-Pick disease type C (NPC) shows the therapy as effective, safe, and well-tolerated. Sponsored by biopharmaceutical company IntraBio Inc. (“IntraBio”), the clinical trial ultimately showed IB1001 as able to improve quality of life (QOL) and reduce symptoms for both pediatric and adult patients.


Also known as N-acetyl-L-leucine, IB1001 is an orally administered amino acid which has been shown to improve brain function. It can also change the way glucose and antioxidants are metabolized and prevent or reduce neuroinflammation. IntraBio is also testing IB1001 as a potential treatment for patients with GM2 gangliosidosis and Ataxia-Telangiectasia. The drug also received Orphan Drug, Rare Pediatric Disease, and Fast Track designations in the United States, as well as Orphan Medicinal Product designation in Europe.

During the clinical trial, researchers evaluated the safety, efficacy, and tolerability of IB1001 for patients with NPC who were ages 6 or over. Patients came from America, the UK, and the EU. Ultimately, researchers determined that the drug reduced symptom severity and improved quality of life.

Niemann-Pick Disease

There are three types of Niemann-Pick disease, a rare metabolic disorder: type A, B, and C. In Niemann-Pick disease, patients are unable to transport lipids called sphingomyelin into cells. As a result, sphingomyelin accumulates, causing damage. Niemann-Pick types A and B (NPA and NPB) result from SMPD1 gene mutations. In Niemann-Pick type C (NPC), either NPC1 or NPC2 mutations cause the condition. Patients must inherit two copies of the genetic mutation to inherit Niemann-Pick disease.

Symptoms vary based on disease subtype and where sphingomyelin accumulates. However, symptoms may include:

  • Appetite loss
  • Low blood platelet levels (thrombocytopenia)
  • Difficulty swallowing
  • Spleen and liver enlargement
  • Intellectual delays or loss
  • Balance issues
  • Jaundice
  • Seizures
  • Abdominal pain and swelling
  • Slurred speech
  • Sleep difficulties

Learn more about Niemann-Pick disease.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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