N-Acetyl-L-Leucine

Phase 3Small molecule

N-Acetyl-L-Leucine is one of 16 small molecule programs targeting Niemann-Pick disease type C. It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

Niemann-Pick Disease Type CActive, not recruiting

Ataxia-TelangiectasiaActive, not recruiting

Niemann-Pick Disease Type CPhase 3

Active, not recruiting

Ataxia-TelangiectasiaPhase 3

Active, not recruiting

Competition

Competitive Landscape

20 competitors in Ataxia-Telangiectasia

#DrugCompanyPhaseTrials

Actos

Takeda

Approved

1 2

Glucophage

EMD Serono

Approved

1 3

Amantadine Sulphate

SSheba Medical Center

Approved

1 4

Lioresal

Novartis

Phase 1

1 5

N-Acetyl-L-Leucine

IIntraBio Inc

Phase 3

1 6

IB1001

MMedexus Pharma, Inc.

Phase 2

1 7

Conjugated Pneumococcal Vaccine

IInstitute of Child Health

Phase 3

1 8

Decadron

QQuince Therapeutics S.p.A.

Prevenar

1 10

Pneumovax

NNational Institute of Allergy and Infectious Diseases (NIAID)

Preclinical

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q2 2028

Phase 3Active, not recruiting

Ataxia-Telangiectasia

NCT06673056

Q4 2028

Phase 3Not yet recruiting

Study completion

NCT07221292

Phase 3Recruiting

Niemann-Pick Disease Type C

NCT05163288

Clinical

Trials by Indication[1]

Niemann-Pick disease type C1 trial

A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C

Recruiting·IIntraBio Inc·Phase 3·NCT05163288·53 enrolled

Ataxia-telangiectasia1 trial

A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T)

Active, not recruiting·IIntraBio Inc·Phase 3·NCT06673056·60 enrolled

Unspecified1 trial

Pivotal Study of N-acetyl-L-leucine for CACNA1A

Not yet recruiting·IIntraBio Inc·Phase 3·NCT07221292·60 enrolled

Sources

References

[1]
ClinicalTrials.gov — 3 trial records for N-Acetyl-L-Leucine. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — N-Acetyl-L-Leucine compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for N-Acetyl-L-Leucine. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — IntraBio Inc company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 3[1]
Company: IIntraBio Inc

N-Acetyl-L-Leucine

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q2 2028

Q4 2028

Trials by Indication[1]

Niemann-Pick disease type C1 trial

Ataxia-telangiectasia1 trial

Unspecified1 trial

References

Drug Info

Quick Facts

Related News