IntraBio’s Pivotal Phase III Trial in CACNA1A-Related Disorders Is Enrolling and Is Expected to Be Over-Enrolled and Complete Recruitment in October

IB1001-304 is the first pivotal clinical trial conducted specifically for CACNA1A-related disorders, for which there are currently no approved therapies anywhere in the world.

• Recruitment for IB1001-304 has commenced; IntraBio anticipates the study will over-enroll and complete recruitment in October 2026.
• CACNA1A-related disorders have an incidence of approximately 1 in 11,700, corresponding to more than 30,000 individuals in the United States alone and an estimated 690,000 worldwide.
• IB1001-304 uses the same pivotal trial design that supported FDA approval and European Commission authorization of levacetylleucine (AQNEURSA®) in Niemann-Pick disease type C.

AUSTIN, Texas--(BUSINESS WIRE)--IntraBio Inc., a global biopharmaceutical company developing and commercializing therapies for rare and common neurological diseases, today announced that its pivotal Phase III IB1001-304 trial evaluating levacetylleucine in individuals with CACNA1A-related disorders is enrolling. The company anticipates the study will over-enroll and complete recruitment in October 2026, with the trial designed to take 6-months to complete.

IB1001-304 is the first pivotal clinical trial conducted specifically for CACNA1A-related disorders. The study uses the same design as the pivotal trial that supported U.S. FDA approval and European Commission authorization of levacetylleucine (AQNEURSA®) for the neurological manifestations of Niemann-Pick disease type C (NPC), and that supported IntraBio’s positive Phase III study in Ataxia-Telangiectasia (A-T). The supplemental New Drug Application for A-T is currently under FDA Priority Review, with a PDUFA target action date of September 19, 2026.

“This trial marks a pivotal milestone for the CACNA1A community,” said Lisa Manaster, Co-Founder and President of the CACNA1A Foundation. “As the mother of a daughter living with a CACNA1A-related disorder, I know firsthand what it means to have real hope that we are moving closer to having an approved, safe, and effective treatment. Given the urgent unmet need our families face, we look forward to continuing to collaborate with IntraBio to help ensure this novel treatment is rapidly made available to patients in our community.”

CACNA1A-related disorders refer to a group of rare, inherited neurological conditions. Despite the severity and lifelong course of these conditions, there are currently no approved therapies indicated for CACNA1A-related disorders anywhere in the world. The estimated incidence of CACNA1A-related disorders is approximately 1 in 11,700, corresponding to approximately 30,000 individuals in the United States alone and an estimated 690,000 worldwide.

About IB1001-304

IB1001-304 is a multinational, randomized, double-blind, placebo-controlled crossover Phase III clinical trial evaluating levacetylleucine in individuals aged four years and older with genetically confirmed CACNA1A-related disorders.

The trial is being conducted at multinational sites in Austria, Germany, Greece, Italy, Switzerland, the United Kingdom and the United States. Further information about IB1001-304, including participating locations and eligibility requirements, is available at ClinicalTrials.gov under study NCT07221292.

The study is registered on ClinicalTrials.gov as NCT07221292 and under EU CT Number 2025-523828-51. Levacetylleucine is investigational for the treatment of CACNA1A-related disorders and has not been approved by the FDA, MHRA, European Commission, Swissmedic or any other regulatory authority for this indication.

About CACNA1A-Related Disorders

CACNA1A-related disorders are rare, inherited neurological conditions caused by pathogenic variants in the CACNA1A gene, which plays a central role in calcium-channel function within the nervous system. Symptoms can begin in infancy, childhood or adulthood.

The estimated incidence of CACNA1A-related disorders is approximately one in 11,700. There are currently no approved therapies indicated for CACNA1A-related disorders.

About AQNEURSA® (levacetylleucine)

U.S. Indication

AQNEURSA® (levacetylleucine) is indicated 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 combination with miglustat or as a monotherapy in patients where miglustat is not tolerated, in adults and children aged 6 years and older and weighing at least 20 kg. See EMA Indication and Important Safety Information: https://www.ema.europa.eu/en/medicines/human/EPAR/aqneursa

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.

Forward-Looking Statements

This press release contains forward-looking statements, including statements regarding the anticipated pace and completion of enrollment in IB1001-304, the expected timing on which the study will close to further participants, the potential of levacetylleucine in CACNA1A-related disorders and other indications, and the anticipated timing of regulatory action. These statements are based on IntraBio’s current expectations and are subject to risks and uncertainties, including the pace of patient identification and screening, site activation timelines, and regulatory and clinical developments. Actual results may differ materially. IntraBio undertakes no obligation to update any forward-looking statement except as required by law.