• First and only approved treatment indicated for Ataxia-Telangiectasia (A-T) in the United States or anywhere in the world
• Second FDA-approved indication for AQNEURSA, following previous approval for Niemann-Pick disease type C (NPC) 
• AQNEURSA is commercially available now
• AQNEURSA provides a long-awaited treatment option for patients living with A-T 

IntraBio Inc., a leader in the discovery, development, and commercialization of novel therapies for rare and common neurodegenerative diseases, today 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. AQNEURSA is the first and only drug approved for the treatment of A-T anywhere in the world. In the United States, it is the second indication AQNEURSA is approved to treat, following its initial approval in September 2024 for the treatment of the neurological manifestations in Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg.

“Today marks a tremendous milestone for the A-T community and IntraBio. As the first FDA-approved drug for the treatment of Ataxia-Telangiectasia, AQNEURSA now offers the potential to make meaningful differences in the lives of thousands of patients and their families who have lacked options to treat the diverse and debilitating array of symptoms caused by A-T,” said Mallory Factor, Chief Executive Officer of IntraBio. “This approval is the result of years of work by investigators, advocacy organizations, and above all the patients and families who took part in this research. We are proud to bring the first approved treatment to the A-T community, and we are committed to making sure that families who could benefit from AQNEURSA are able to access it.”

A-T is a rare, progressive, and prematurely fatal autosomal-recessive neurodegenerative disorder that affects an estimated 1 in 40,000 people. A-T is characterized by progressive cerebellar degeneration resulting in worsening ataxia that can affect gait, truncal control, eye movements, balance, speech, and hand coordination, alongside other systemic manifestations. Until today, there were no approved treatments for A-T anywhere in the world.

“This is a historic day for the A-T community and the patients and caregivers who have coped for decades without a treatment approved specifically for A-T,” said Brad Margus, Founder of the A-T Children's Project. “With today’s FDA decision, those impacted by A-T have a treatment option that has demonstrated benefit across a range of A-T symptoms, including those that affect daily life for patients and their loved ones. We are grateful to every family who participated in this research and to IntraBio for their commitment to a disease this rare and this complex. We will keep working to make sure every patient who could benefit has access to it."

PIVOTAL PHASE 3 RESULTS

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. 

The trial met the primary efficacy endpoint and key secondary endpoints were supportive of the primary result. Results from the study showed AQNEURSA significantly improved signs and symptoms of ataxia and demonstrated functional benefits important to everyday life that were evident within 12 weeks. Seventy patients (96%) completed the study and received both placebo and AQNEURSA. These findings were published in the July 2026 issue of The Lancet Neurology.

Key findings included:

• AQNEURSA demonstrated statistically significant improvement compared to placebo on the primary efficacy endpoint, the Scale for the Assessment and Rating of Ataxia (SARA) with a treatment difference of -1.9 points (95% CI: –2.7, –1.1; two-sided p<0.001). 
• The primary outcome assessed by the FDA was the functional SARA (fSARA), a modified version comprising the gait, sitting, stance, and speech disturbance domains. Patients who received AQNEURSA showed a greater improvement in fSARA score, with a mean treatment difference of −0.6 (95% CI: −0.9, −0.2; two-sided p<0.001).
• Improvement was consistent across secondary endpoints and prespecified subgroups, including pediatric and adult patients, with no evidence of heterogeneity in treatment effect across the population.
• AQNEURSA was well tolerated. No treatment-related serious adverse events and no deaths occurred, and no patients discontinued because of a treatment-related adverse event. The most common adverse reactions (incidence ≥5% and greater than placebo) were fall and skin laceration.

ABOUT AQNEURSA® (LEVACETYLLEUCINE)

AQNEURSA® (levacetylleucine) is a first-in-class, chemically modified amino acid approved by the FDA for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg and for ataxia in in adults and pediatric patients with ataxia-telangiectasia (A-T) weighing ≥15 kg. It is taken orally as a suspension. 

The distinct molecular target for AQNEURSA in the treatment of NPC or A-T is unknown. The proposed mechanism of action is the normalization of glucose metabolism, which is correlated with enhanced cerebellar activity. AQNEURSA is designed to enter enzyme-controlled pathways to correct metabolic dysfunction, improve lysosomal function, and enhance mitochondrial function and ATP production. In preclinical studies, AQNEURSA normalized lysosomal function, reduced substrate accumulation, improved mitochondrial ATP production, and reduced neuroinflammation - addressing shared downstream metabolic dysfunction rather than a single genetic defect.

Indication

AQNEURSA^® (levacetylleucine) is indicated for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg.

AQNEURSA^® (levacetylleucine) is indicated for the treatment of ataxia in adults and pediatric patients with ataxia-telangiectasia (A-T) weighing ≥15 kg.

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 treatment during pregnancy should consider the female’s need for AQNEURSA, 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, verify that the patient is not pregnant prior to initiating treatment with AQNEURSA. Advise females of reproductive potential to use effective contraception during treatment with AQNEURSA 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 either human or animal milk, the effects on the breastfed infant or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for AQNEURSA and any potential adverse effects on the breastfed infant from levacetylleucine or from the underlying maternal condition.

Adverse Reactions

• In Niemann-Pick disease type C, the most common adverse reactions (incidence ≥5% and greater than placebo) are abdominal pain, dysphagia, upper respiratory tract infections, and vomiting.
• In ataxia-telangiectasia, the most common adverse reactions (incidence ≥5% and greater than placebo) are fall, skin laceration, and urinary tract infection. 

Drug Interactions

• Avoid concomitant use of AQNEURSA with N-acetyl-DL-leucine or N-acetyl-D-leucine. The D-enantiomer, N-acetyl-D-leucine, competes with levacetylleucine for monocarboxylate transporter uptake, which may reduce the levacetylleucine efficacy.
• Monitor more frequently for P-gp substrate related adverse reactions when used concomitantly with AQNEURSA. AQNEURSA inhibits P-gp; however, 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 www.fda.gov/medwatch.

U.S. full Prescribing Information for AQNEURSA is available at https://intrabio.com/wp-content/aqneursa-prescribing-information.pdf.

ABOUT ATAXIA-TELANGIECTASIA

Ataxia-telangiectasia is a rare, progressive, debilitating, and prematurely fatal autosomal-recessive inherited cerebellar ataxia caused by biallelic pathogenic variants in the ATM gene. Symptoms typically appear in early childhood and include progressive loss of balance and coordination, slurred speech, impaired eye movement, difficulty swallowing, and loss of fine and gross motor function. The disease also features immunodeficiency, sinopulmonary infections, and a substantially increased risk of malignancy, particularly of lymphoid origin. A-T presents heterogeneously, and disease burden accumulates over time as abilities are lost.

AQNEURSA Cares^® Support for Patients

IntraBio offers support programs to eligible patients through their Patient Support Service, AQNEURSA Cares^®. This program includes financial support to reduce or eliminate out-of-pocket costs for qualifying patients and also connects patients with third-party resources. AQNEURSA Cares includes access to financial and educational resources and a dedicated team of specialists. The team is available to help with individuals’ unique challenges including starting treatment, questions about taking the medication, and navigating insurance coverage. Contact 866-200-0419 to speak to an AQNEURSA Cares team representative to seek assistance with any questions or concerns about access to AQNEURSA.

About IntraBio

IntraBio Inc., a US biopharmaceutical company, is focused on the development of novel drugs addressing rare and common neurological diseases. IntraBio's platform technologies result from decades of research and collaboration with universities and institutions worldwide. Its clinical programs are based upon the expertise in lysosomal function and intracellular signaling of its scientific founders from the University of Oxford and the University of Munich.

IntraBio is currently conducting a Phase III IB1001-304 trial in CACNA1A-related disorders, a group of rare, inherited neurological conditions with an estimated incidence of approximately one in 11,700, corresponding to approximately 30,000 individuals in the United States and an estimated 690,000 worldwide, for which there are currently no approved therapies anywhere in the world. 

For more information about IntraBio, please visit the company’s website at intrabio.com and follow on LinkedIn (@IntraBio-Inc).

AQNEURSA^®, AQNEURSA Cares^®, and their respective logos are registered trademarks of IntraBio Inc. ©2026 IntraBio Inc. All rights reserved.

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