IB1000 Series

Development History

Mode of Action

IntraBio’s leading drug candidate series, IB1000s, have been observed to have clinical efficacy in 19 different indications, including of rare lysosomal storage disorders and common neurodegenerative diseases.

IntraBio is initially developing IB1000s for three orphan indications where there are currently no FDA approved therapies: Niemann-Pick Disease Type C (NPC), Tay-Sach’s Disease (TS), and inherited Cerebellar Ataxias (CA).

Clinical Development

In compassionate use studies, IB1000s have demonstrated statistically significant improvement in key, clinically-validated neurological scales in patients with NPC, Tay-Sachs disease, and inherited Cerebellar Ataxias (as well as additional LSDs and neurodegenerative diseases). These findings have been significantly supported in additional in vitro and in vivo studies, and are described in multiple published peer-review papers.

Regulatory History

IntraBio has been granted Orphan Drug Designations (US Food and Drug Administration)/ Orphan Medicinal Drug Designations (European Commission) for IB1000s for the treatment of NPC, GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease), and Spinocerebellar Ataxias (of which there are over 40 known subtypes) as well as Ataxia-Telangiectasia (FDA only).

IntraBio has also been granted a Rare Pediatric Disease Designation for IB1000s by the FDA for the treatment of NPC and GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease). These Rare Pediatric Disease Designation makes IB1000s eligible for, and expedites the request of, a Rare Pediatric Disease Priority Review Voucher (PRV) granted at the time of marketing approval.

IntraBio has met with regulators in the US and Europe to discuss the clinical trial protocols for IB1001, and intends to submit applications for Clinical Trial Authorizations (CTA) with select EU national regulatory authorities, and Investigational New Drug (IND) applications in the US to initiate the studies.  The trials will be conducted at key specialist centers to clearly establish the safety and efficacy of IB1001 for the treatment of these rare, life-threatening conditions.

IntraBio will provide full details of its clinical programs, including enrollment and inclusion criteria for upcoming clinical trials, as soon as they become available. All official announcements or statements will also be highlighted in the News section of our website. 


IB1000s are a series of orally administered, modified amino-acid analogs/esters that are well tolerated and have an excellent safety profile.

IB1000s are believed to have an effect on the normalization of neuronal membrane potential and intracellular ion regulation via calcium channels, and have been observed to have symptomatic and neuroprotective properties and disease modifying potential.

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).

Cortina-Borja,M., te Vruchte,D., Mengel,E., Amraoui,Y., Imrie, J. ,Jones,S., Dali,C., Fineran,P., Kirkegaard,T., Runz,H., Lachmann,R., Bremova-Ertl,T., Strupp,M., Platt,F.

Demonstrates the annual severity increment score (ASIS) has prognostic value in assessing the progression of Niemann-Pick type C, and that long-term treatment with N-Acetyl-DL-leucine is associated with a reduction in ASIS scores. Orphanet Journal of Rare Diseases 13, 1-16 (2018).

Strupp, M., Bayer, O., Feil, K., Straube, A.

A paper demonstrating the significant benefit of N-Acetyl-DL-Leucine for the prophylactic treatment of migraine, as well as migraine with aura. Journal of Neurology.