Pivotal Phase III Trial Meets All Primary and Key Secondary Endpoints with High Statistical Significance
Levacetylleucine Could Become the First-Ever FDA-Approved Treatment for Ataxia-Telangiectasia
- FDA accepts sNDA for AQNEURSA® (levacetylleucine) for Ataxia-Telangiectasia — a rare, progressive neurodegenerative disease with no currently approved treatments
- Priority Review granted; PDUFA target action date of 19 September 2026
- Pivotal Phase III trial (IB1001-303) met primary and key secondary endpoints with high statistical significance, demonstrating clinically meaningful improvements in neurological signs, symptoms, and function in pediatric and adult patients
- If approved, AQNEURSA would be the first FDA-approved therapy for A-T
AUSTIN, Texas--(BUSINESS WIRE)--IntraBio Inc., a global biopharmaceutical company focused on developing and commercializing therapies for neurological diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review its supplemental New Drug Application (sNDA) for AQNEURSA® (levacetylleucine) for the treatment of Ataxia-Telangiectasia (A-T) in adults and pediatric patients.
The FDA simultaneously granted Priority Review and assigned a PDUFA target action date of 19 September 2026. If approved, AQNEURSA would become the first FDA-approved treatment for A-T.
“Today’s announcement represents a profound step forward — not just for IntraBio, but for the thousands of patients and families living with Ataxia-Telangiectasia who have waited far too long for a treatment option. Our Phase III trial didn’t just meet the bar — it cleared it with high statistical significance across both primary and key secondary endpoints, delivering meaningful improvements in neurological function. The FDA’s Priority Review designation reflects the urgency of this unmet need, and we are fully committed to working with the agency to make AQNEURSA the first approved therapy for A-T as swiftly as possible.”
— Mallory Factor, President and Chief Executive Officer, IntraBio
The submission is supported by data from a pivotal Phase III clinical trial evaluating levacetylleucine in adult and pediatric patients with A-T. In this randomized, double-blind, placebo-controlled crossover study, levacetylleucine met its primary endpoint and all key secondary endpoints with high statistical significance, demonstrating clinically meaningful improvements in neurological signs, symptoms, and functioning. The compound was generally safe and well-tolerated, with no drug-related serious adverse events observed — consistent with its established safety profile.
ABOUT ATAXIA-TELANGIECTASIA (A-T)
Ataxia-Telangiectasia is a rare, inherited, progressive neurodegenerative disorder that typically manifests in early childhood. A-T is estimated to affect 1 in 40,000-100,000 people. It is characterized by cerebellar degeneration leading to worsening loss of coordination, impaired speech, abnormal eye movements, and eventual wheelchair dependence. Many patients also develop telangiectasia, immune deficiency with recurrent and potentially life-threatening infections, pulmonary disease, and a significantly elevated risk of cancer. There are currently no FDA-approved therapies for A-T.
ABOUT AQNEURSA® (LEVACETYLLEUCINE)
U.S. Indication
AQNEURSA® (levacetylleucine) is approved in the United States for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing at least 15 kg.
U.S. IMPORTANT SAFETY INFORMATION
Embryo-Fetal Toxicity
Based on findings from animal reproduction studies, AQNEURSA may cause embryo-fetal harm when administered during pregnancy. The decision to continue or discontinue AQNEURSA during pregnancy should consider the patient’s clinical need, the potential drug-related risks to the fetus, and the potential adverse outcomes from untreated maternal disease.
Pregnancy and Lactation
For females of reproductive potential, confirm the patient is not pregnant prior to initiating treatment. Advise use of effective contraception during treatment and for 7 days after the last dose if AQNEURSA is discontinued. There are no data on the presence of levacetylleucine or its metabolites in human or animal milk; the developmental and health benefits of breastfeeding should be weighed against clinical need and potential risks to the infant.
Adverse Reactions
The most common adverse reactions (incidence ≥5% and greater than placebo) are abdominal pain, dysphagia, upper respiratory tract infections, and vomiting.
Drug Interactions
Avoid concomitant use of AQNEURSA with N-acetyl-DL-leucine or N-acetyl-D-leucine. The D-enantiomer competes with levacetylleucine for monocarboxylate transporter uptake, potentially reducing efficacy. Monitor more frequently for P-gp substrate-related adverse reactions when used concomitantly with AQNEURSA, as AQNEURSA inhibits P-gp; the clinical significance of this finding has not been fully characterized.
To report SUSPECTED ADVERSE REACTIONS, contact IntraBio Inc. at 1-833-306-9677 or FDA at 1-800-FDA-1088 or visit www.fda.gov/medwatch.
Please see Full U.S. Prescribing Information for AQNEURSA at aqneursahcp.com.
EU (EMA) Indication
AQNEURSA® is authorized in the European Union for the treatment of neurological manifestations of Niemann-Pick disease type C in adults and children aged 6 years and older weighing at least 20 kg, either in combination with miglustat or as monotherapy in patients who cannot tolerate miglustat. See EMA Indication and Important Safety Information.
ABOUT INTRABIO
IntraBio Inc. is a global biopharmaceutical company headquartered in Austin, Texas, focused on developing and commercializing targeted therapies for rare and common neurological, neurodevelopmental, and mitochondrial diseases. IntraBio’s platform technologies are built on decades of scientific research and collaboration with leading institutions worldwide, including the University of Oxford and the University of Munich.
Contacts
MEDIA & INVESTOR CONTACT
Cass Fields | Vice President, External Affairs & Marketing
ccfields@intrabio.com | www.intrabio.com
