Aqneursa (levacetylleucine) / IntraBio - LARVOL DELTA

A Phase 3, Multinational, Randomized, Placebo-Controlled, Double-Blind, Crossover Trial (IB1001-304) Evaluating Levacetylleucine in CACNA1A Disorders (MDS Congress 2026) - No abstract available

Clinical • P3 data • CNS Disorders • CACNA1A

Positive Results from a Global Phase 3 Trial (IB1001-303) Evaluating Levacetylleucine in Ataxia-Telangiectasia (MDS Congress 2026) - No abstract available

Clinical • P3 data • Ataxia • CNS Disorders • Movement Disorders

IntraBio…announced that the U.S. Food and Drug Administration (FDA) has approved AQNEURSA (levacetylleucine) for the treatment of ataxia in adult and pediatric patients with ataxia-telangiectasia (A-T) weighing ≥15 kg (Businesswire) - "AQNEURSA is the first and only drug approved for the treatment of A-T anywhere in the world....The approval is supported by the pivotal IB1001-303 study (NCT06673056), a randomized, double-blind, placebo-controlled, crossover trial that enrolled 73 patients aged 4 to 50 years with genetically confirmed A-T across 10 clinical trial sites in Germany, Slovakia, Spain, Switzerland, the United Kingdom, and the United States."

FDA approval • Ataxia • CNS Disorders • Genetic Disorders • Rare Diseases

Subdomain Analysis of the NPC Clinical Severity Scale Evaluating Long-Term Efficacy of Levacetylleucine in NPC (SSIEM 2026) - "Background IB1001-301 is a Phase 3, double-blind, randomized, placebo-controlled, crossover trial evaluating levacetylleucine (also known as N-acetyl-L-leucine) for the treatment of neurological signs and symptoms in Niemann-Pick disease type C (NPC) encompassing a Parent Study and an ongoing open-label Extension Phase (EP). These findings are consistent with results from 24-months of treatment and are indicative of a disease-modifying, neuroprotective effect. The uniform improvement across all subdomains demonstrates the robust and positive efficacy of levacetylleucine across a wide spectrum of neurological functions."

Clinical • Frontotemporal Lobar Degeneration • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders

Long-Term Efficacy and Safety of Levacetylleucine in Adults and Children with Niemann-Pick Disease Type C (SSIEM 2026) - "IB1001-301 is a Phase 3, double-blind, randomized, placebo-controlled, crossover trial evaluating levacetylleucine (also known as N-acetyl-L-leucine) for the treatment of neurological signs and symptoms in NPC encompassing a Parent Study and an ongoing open-label Extension Phase (EP). Conclusion Long-term treatment with levacetylleucine was associated with a statistically significant and clinically meaningful reduction in disease progression and consistent with a neuro-protective, disease modifying effect. Levacetylleucine was well-tolerated with no treatment emergent events occurring in more than 15% of participants on treatment."

Clinical • Ataxia • CNS Disorders • Frontotemporal Lobar Degeneration • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Movement Disorders • Rare Diseases

Measuring Functional Abilities and Assistance Level in People with Niemann-Pick Disease Type C Receiving Levacetylleucine (SSIEM 2026) - "These findings are compelling and consistent with previously reported evidence demonstrating disease stabilization or improvement in a smaller cohort of participants with NPC. [1] WeeFIM and WeeFIM II are trademarks of Uniform Data System for Medical Rehabilitation, a division of UB Foundation Activities, Inc."

Frontotemporal Lobar Degeneration • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders

N-acetyl-L-leucine normalizes Transcription Factor EB activity by stereospecific bidirectional modulation in a HeLa cell model of Niemann-Pick disease type C. (PubMed, PLoS One) - "The effects of the drug are stereospecific: while the L-enantiomer is active, the D-enantiomer and racemate show no effect, revealing the antagonistic properties of the D-enantiomer. This bidirectional normalisation of TFEB activity highlights a direct mechanism through which levacetylleucine modulates lysosomal and autophagic pathways in the HeLa cell model, giving mechanistic insight into its therapeutic potential in NPC, and also across diverse neurological and neurodevelopmental disorders."

Journal • CNS Disorders • Developmental Disorders • Frontotemporal Lobar Degeneration • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Psychiatry • TFEB

Neurofilament light chain (NfL) as a surrogate outcome measure for GM2 gangliosidoses. (PubMed, J Neurol) - P2 | "These findings support NfL as a promising surrogate outcome candidate for GM2 and link biochemical improvement with functional benefit, which is reasonably likely to predict both disease activity and treatment response/clinical benefit."

Biomarker • Journal • Ataxia • CNS Disorders • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Movement Disorders • NEFL

Results from the Global Phase 3 Trial (IB1001-303) Evaluating Levacetylleucine in Ataxia-Telangiectasia (EAN 2026) - "Levacetylleucine demonstrated a significant benefit versus placebo, and clinically meaningful improvements in neurological manifestations of A-T, functioning, and quality of life, and a favorable benefit-risk profile for the treatment of A-T."

Clinical • P3 data • Ataxia • Immunology • Metabolic Disorders • Movement Disorders • Primary Immunodeficiency

Long-term (30 Months) Outcomes of N-Acetyl-L-Leucine in Adults and Children with Niemann-Pick Disease Type C (EAN 2026) - "IB1001-301 is a Phase 3, placebo-controlled, crossover trial encompassing a Parent Study and an ongoing Extension Phase (EP) evaluating the long-term effects of N-acetyl-L-leucine (NALL) in people with NPC. Treatment with NALL demonstrated significant and meaningful long-term improvement, i.e, disease-modifying effects. NALL was well tolerated, and no serious adverse events occurred."

Clinical • Ataxia • Frontotemporal Lobar Degeneration • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Movement Disorders • Rare Diseases

Safety and efficacy of levacetylleucine in ataxia-telangiectasia: a phase 3, randomised, double-blind, placebo-controlled crossover trial. (PubMed, Lancet Neurol) - P3 | "Levacetylleucine showed a significant and clinically meaningful improvement in functioning and was safe and well-tolerated, providing a favourable benefit-risk profile for the treatment of ataxia-telangiectasia. An ongoing open-label extension phase of this trial will investigate potential long-term, neuroprotective and disease-modifying effects."

Clinical • Journal • P3 data • Ataxia • CNS Disorders • Frontotemporal Lobar Degeneration • Genetic Disorders • Immunology • Lysosomal Storage Diseases • Metabolic Disorders • Movement Disorders • Pediatrics • Primary Immunodeficiency • Rare Diseases

IntraBio Announces Submission of Variation Application to the European Medicines Agency for AQNEURSA for Ataxia-Telangiectasia (Businesswire) - "In the United States, IntraBio’s supplemental New Drug Application for AQNEURSA for A-T has been accepted for review by the U.S. Food and Drug Administration and granted Priority Review, with a Prescription Drug User Fee Act (PDUFA) target action date of September 19, 2026....The variation application seeks to expand the label of AQNEURSA to include A-T."

EMA filing • FDA filing • PDUFA • Genetic Disorders • Rare Diseases