For Medical Professionals

Drug Canidates

Mode of Action

IntraBio is a clinical-stage, global biopharmaceutical company that discovers, develops, and commercializes novel therapies for rare and neurodegenerative diseases. IntraBio is currently developing a series of small-molecule drug candidates that have broad applicability to rare and common neurological and inflammatory diseases.

IntraBio’s leading compounds, IB1000s, are a series of orally administered, modified amino-acid analogues that are well tolerated and have an excellent safety profile.

In clinical studies, IB1000 has been observed to have significant efficacy in the treatment of rare lysosomal storage disorders (LSDs) and common neurodegenerative diseases.

Based on the significant non-clinical data and promising findings from Phase 2 observational clinical studies, IntraBio is in the process of applying for multi-national, randomized, placebo-controlled clinical trials for the treatment of patients with Niemann-Pick type C, Tay-Sachs disease, and inherited Cerebellar Ataxias.

IntraBio will provide full details of its clinical programs, including enrollment and inclusion criteria for upcoming clinical trials, as soon as they become available.


Several publications have explored the mechanism of action of IB1000 in different model systems, listed below along with the published observational clinical study outcomes. In addition, there are several peer-reviewed papers that quantify the efficacy of IB1000 in the treatment of neurodegenerative diseases.


Günther L, Beck R, Xiong G, Potschka H, Jahn K, Bartenstein P, Brandt T, Dutia M, Dieterich M, Strupp M, la Fougère C, Zwergal A.

PLoS One. 2015 Mar 24;10(3):e0120891. doi: 10.1371/journal.pone.0120891. eCollection 2015. PMID: 25803613

Lee AJ, Beno DW, Zhang X, Shapiro R, Mason M, Mason-Bright T, Surber B, Edens NK.

Amino Acids. 2015 May;47(5):917-24. doi: 10.1007/s00726-015-1920-6. Epub 2015 Jan 25. PMID: 25618754.

Feil K, Bremova T, Muth C, Schniepp R, Teufel J, Strupp M.

Cerebellum.15(1):38-42. (2016).

Kalla, R., Teufel, J., Feil, K., Strupp, M.

Journal of Neurology 263 (2016).


Bremova T, Malinová V, Amraoui Y, Mengel E, Reinke J, Kolníková M, Strupp M.

Neurology. 2015 Oct 20;85(16):1368-75. doi: 10.1212/WNL.0000000000002041. Epub 2015 Sep 23. PMID: 26400580.

Strupp M, Teufel J, Habs M, Feuerecker R, Muth C, van de Warrenburg BP, Klopstock T, Feil K. J Neurol.

2013 Oct;260(10):2556-61. doi: 10.1007/s00415-013-7016-x. Epub 2013 Jul 9.PMID: 19351170.
Effects of acetyl-DL-leucine on cerebellar ataxia (ALCAT trial): study protocol for a multicenter, multinational, randomized, double-blind, placebo-controlled, crossover phase III trial Feil K, Adrion C, Teufel J, Bösch S, Claassen J, Giordano I, et al. BMC Neurology. 2017;17:7.
An anecdotal report by an Oxford basic neuroscientist: effects of acetyl-DL-leucine on cognitive function and mobility in the elderly. Platt, F., Strupp, M. 2016 June; 263: 1239-40. Doi: 10.1007/s00415-016-8048-9.Epub 2016 Apr 28. PMID: 27126454

Ferber-Viart C, Dubreuil C, Vidal PP. Audiol Neurootol.

2009;14(1):17-25. doi: 10.1159/000148206. Epub 2008 Jul 29. PMID: 18663295
Acetyl-DL-leucine improves gait variability in patients with cerebellar ataxia—a case series Schniepp et al. Cerebellum & Ataxias (2016) 3:8 DOI 10.1186/s40673-016-0046-2
Gait ataxia in humans: vestibular and cerebellar control of dynamic stability J Neurol (2017) 264 (Suppl 1):S87–S92 DOI 10.1007/s00415-017-8482-3

Schniepp, R., Strupp, M. Wuehr, M. Jahn, K. Dieterich, M. Brandt, T. Feil, K.

Cerebellum & Ataxias 3:8 (2016).

Schniepp, R. Mohwald, K. Wuehr, M.

Journal of Neurology. 264 (2017).

Platt, F., Strupp, M.

A case study demonstrating that, in addition to being of benefit in patients with cerebellar ataxia and Niemann-Pick disease type C, acetyl-DL-leucine can increase mobility and quality of life in a healthy elderly subject.Journal of Neurology 263, 1239-40 (2016).