AUSTIN, Texas–(BUSINESS WIRE)–IntraBio Inc. today announced positive topline results from its pivotal Phase III IB1001-303 clinical trial, “Effects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T): A Randomized, Placebo-Controlled, Double-Blind, Crossover Study” (NCT06673056) evaluating N-acetyl-L-leucine (levacetylleucine) in pediatric and adult patients with Ataxia-Telangiectasia (A-T).
The primary endpoint of the trial evaluated the effect of treatment with levacetylleucine on the Scale for the Assessment and Rating of Ataxia (SARA) compared to placebo after 12 weeks of treatment. Treatment with levacetylleucine demonstrated a statistically significant and clinically meaningful -1.88 point improvement of the SARA score compared to placebo (-1.92 on levacetylleucine vs -0.14 on placebo, p<0.001).
The trial also met secondary endpoints, demonstrating statistically significant and clinically meaningful improvement on the International Cooperative Ataxia Rating Scale (ICARS) (-4.22 on levacetylleucine vs. -1.69 on placebo; p=0.003) and the Investigator’s Clinical Global Impression of Improvement (CGI-I) (-0.6 on levacetylleucine vs. -0.2 on placebo; p=0.02). Levacetylleucine was observed to be safe and well-tolerated, with no drug-related serious adverse events, consistent with its established safety profile.
Based on these results, IntraBio plans to immediately advance regulatory submissions to the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), and additional global regulatory authorities.
“This is a breakthrough for patients and families affected by Ataxia-Telangiectasia,” said Dr. Franziska Hoche, Massachusetts General, Investigator of the IB1001-303 study. “A-T is a rare and devastating disorder with no approved treatments. The results from the IB1001-303 trial, demonstrating levacetylleucine significantly improved patients’ neurological symptoms and everyday function, represent a major scientific and clinical milestone, and provide compelling evidence that levacetylleucine has a meaningful impact on A-T patients’ lives.”
“These results mark a major turning point for the Ataxia-Telangiectasia community,” said Brad Margus, Founder of the A-T Children’s Project. “This offers real hope that families will soon have access to their first effective and safe treatment approved for A-T. We look forward to continuing to collaborate with IntraBio to help ensure levacetylleucine is rapidly approved by the FDA and made available for patients in our community, given their urgent need for effective, approved treatments.”
The positive results from IB1001-303 build on prior clinical experience with levacetylleucine across multiple neurological, neurodevelopmental, and mitochondrial disorders. Levacetylleucine is also in late-stage development in the United States and Europe for CACNA1A-related disorders, a group of rare neurological conditions affecting approximately 1 to 2 in 10,000 people.
About IB1001-303
IB1001-303 is a Phase III clinical study evaluating levacetylleucine for the treatment of Ataxia-Telangiectasia (A-T), using the Scale for the Assessment and Rating of Ataxia (SARA) as the primary endpoint. The study includes a randomized, placebo-controlled, double-blind crossover phase, followed by a long-term open-label extension. It was designed in partnership with key opinion leaders and A-T patient organizations to evaluate both symptomatic effects and longer-term clinical outcomes. Levacetylleucine is an investigational drug for A-T and has not yet been approved by the FDA or any other regulatory authority for this indication.
About Ataxia-Telangiectasia
Ataxia-Telangiectasia (A-T) is a rare, inherited, progressive neurodegenerative disorder that typically begins in early childhood. A-T is characterized by degeneration of the cerebellum, leading to worsening loss of coordination, impaired speech and eye movements, and wheelchair dependence. Many patients also develop visible blood vessel changes (telangiectasia), immune system deficiencies with recurrent, life-threatening infections, lung disease and a dramatically increased risk of cancer. There are currently no approved treatments for the treatment of A-T.
About AQNEURSA®
AQNEURSA® (levacetylleucine) is approved in the United States, indicated for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg.
Today, IntraBio also separately announced the approval of AQNEURSA® 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. The most commonly reported adverse reaction with AQNEURSA® was flatulence.
About IntraBio
IntraBio Inc. is an Austin, Texas-based global biopharmaceutical company that develops and commercializes targeted therapies for rare and common neurological, neurodevelopmental, and mitochondrial diseases. IntraBio’s platform technologies result from decades of research and collaboration with universities and institutions worldwide, and leverage the expertise of its scientific founders from the University of Oxford and the University of Munich.
For further information, please contact:
Cass Fields
Vice-President of External Affairs
ccfields@intrabio.com
www.intrabio.com
AUSTIN, Texas–(BUSINESS WIRE)–IntraBio Inc. today announced that the European Commission granted marketing authorization to AQNEURSA® (levacetylleucine) for the treatment of neurological manifestations of Niemann-Pick Type C (NPC) disease, following a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA).
AQNEURSA® is approved in the European Union for use in adults and children aged 6 years and older weighing at least 20 kg. The approved indication includes use in combination with miglustat, or as monotherapy in patients where miglustat is not tolerated.
“This approval represents a significant milestone for the NPC community in Europe,” said Mallory Factor, Chief Executive Officer of IntraBio. “We are grateful to the EMA and European Commission for their thorough review and for recognizing the clinical value of AQNEURSA®. This decision reflects years of breakthrough scientific work and collaboration with clinicians and patient organizations, and it marks an important step toward expanding access to this therapy for people living with NPC.”
The approval is based on results from a Phase III randomized, double-blind, placebo-controlled, crossover study in patients with NPC. In the study, treatment with AQNEURSA® demonstrated a statistically significant and clinically meaningful improvement in neurological signs, symptoms, and functioning after 12 weeks of treatment, as measured by the Scale for the Assessment and Rating of Ataxia (SARA), compared with placebo.1
In the ongoing open-label extension phase of the trial, improvements observed during the initial 12-week study have been sustained,2,3 consistent with a neuroprotective, disease modifying effect over time. Observational comparisons with a natural history control cohort show that treatment with AQNEURSA® was associated with a 118% reduction in annual disease progression after 1 year, as measured by the 5-domain NPC Clinical Severity Scale (NPC-CSS), and a similar reduction after two-years.2,3
Professor Kyriakos Martakis, Associate Professor in Pediatrics at Justus Liebig University Giessen in Germany, a Principal Investigator for the trial, commented, “Niemann-Pick disease Type C is a rare and relentlessly progressive neurological disorder associated with substantial burden for patients and their families. The availability of an approved treatment addressing the neurological manifestations of NPC represents an important development for clinicians, but most of all, for individuals affected by this disease across Europe.”
AQNEURSA® is available as 1 g granules for oral suspension. The active substance, levacetylleucine, is a first-in-class modified amino acid delivered to all tissues, including the central nervous system, designed to correct metabolic dysfunction, improve cellular energy production, and target the underlying processes of neurological dysfunction.1,4
Detailed recommendations for the use of AQNEURSA® are described in the Summary of Product Characteristics (SmPC), which is available on the EMA website in all official European Union languages.
AQNEURSA® was designated as an orphan medicinal product during its development.
About AQNEURSA®
AQNEURSA® (levacetylleucine) is approved in the United States, indicated for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg, and in the European Union for the treatment of neurological manifestations of Niemann-Pick disease Type C, in combination with miglustat or as monotherapy in patients where miglustat is not tolerated, in adults and children aged 6 years and older weighing at least 20 kg.5,6 The most commonly reported adverse reaction with AQNEURSA® was flatulence.
About Niemann-Pick Disease Type C
Niemann-Pick disease Type C (NPC) is a rare (1:100,000 live births), prematurely fatal, autosomal recessive, lysosomal storage disorder.7 The disease presents with systemic, psychiatric, and neurological symptoms, including cerebellar ataxia. NPC is chronic and progressive in nature and is characterized by rapid degeneration of the cerebellum and major organ systems which severely impacts the quality of life.8-10
About IntraBio
IntraBio Inc. is a global biopharmaceutical company that develops and commercializes targeted therapies for rare and common neurological, neurodevelopmental, and mitochondrial diseases. IntraBio’s platform technologies result from decades of research and collaboration with universities and institutions worldwide, and leverage the expertise of its scientific founders from the University of Oxford and the University of Munich.
Today, IntraBio also separately announced the results of a Phase III randomized, double-blind, placebo-controlled, crossover study in patients with Ataxia-Telangiectasia (A-T), a neurodegenerative disease without any approved treatment and impacting approximately 1 in 70,000 people. Based on these results, IntraBio plans to immediately advance regulatory submissions to the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), and additional global regulatory authorities.
For more information about IntraBio, please visit the company’s website at intrabio.com and follow on LinkedIn (@IntraBio-Inc).
U.S. IMPORTANT SAFETY INFORMATION
Embryo-Fetal Toxicity
Pregnancy and Lactation
Adverse Reactions
Drug Interactions
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.
Please click here for Full US Prescribing Information for AQNEURSA: https://www.aqneursahcp.com/wp-content/prescribing-information.pdf
References
1. Bremova-Ertl T, et al. N Engl J Med. 2024;390:421-431
2. Patterson, Marc C., et al. “Disease-modifying, neuroprotective effect of N-acetyl-l-leucine in adult and pediatric patients with Niemann-Pick disease type C.” Neurology 105.1 (2025): e213589.
3. Strupp M, Patterson M, Raymond J, et al. Long-term 24-month findings of N-acetyl-L-leucine for Niemann Pick disease type C. Presented at the 11th European Academy of Neurology Annual Congress 2025; June 21-24, 2025; Helsinki, Finland.
4. Churchill GC, et al. Nature. 2021;11:15812;2
5. AQNEURSA. Prescribing Information. IntraBio Inc
6. AQNEURSA. EU Summary of Product Characteristics (SmPC). IntraBio Ireland Limited.
7. Burton BK, Ellis AG, Orr B, et al. Estimating the prevalence of Niemann-Pick disease type C (NPC) in the United States. Mol Genet Metab. 2021;134:182-187. doi:10.1016/j.ymgme.2021.06.011
8. Geberhiwot T, et al. Orphanet J of Rare Dis. 2018;13:50
9. Patterson MC, et al. Orphanet J Rare Dis. 2013;8:12; 3. NORD. NPC Signs & Symptoms. Published Dec 12, 2023. Accessed May 19, 2024.
10. Vanier MT. Orphanet J Rare Dis. 2010;5:16.
For further information please contact:
Cass Fields
Vice-President of External Affairs
ccfields@intrabio.com
www.intrabio.com
AUSTIN, TX, September 25, 2024– IntraBio Inc., a leader in the discovery and development of innovative drugs for rare neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved AQNEURSA (levacetylleucine) for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg. AQNEURSA is the only FDA-approved stand-alone therapy indicated for the treatment of NPC.
“IntraBio has been dedicated to bringing novel treatments to patients with extremely high unmet medical needs like NPC, and today we celebrate a major milestone in this tremendous effort,” said Mallory Factor, President and Chief Executive Officer of IntraBio. “Patients and families in the NPC community have long awaited an effective, FDA-approved treatment, and we are proud to bring hope to those affected by this devastating disease. We remain committed to ensuring that all patients who can benefit from this novel treatment will have the opportunity to do so. Based on our clinical research, we believe that AQNEURSA may hold potential for treating other rare and common neurodegenerative and neurodevelopmental disorders, and we will continue to rapidly develop AQNEURSA for these additional indications.”
NPC is a rare, inherited lysosomal disease that occurs in about 1 in 100,000 live births. Patients with NPC typically experience systemic, neurological and psychiatric symptoms that can be debilitating and significantly impact functional abilities. Until now, current treatment approaches have not addressed the debilitating effects of NPC on patients’ daily lives.
“The FDA approval of AQNEURSA marks a significant breakthrough for those living with Niemann-Pick disease type C,” commented Laurie Turner, Family Services Manager at the National Niemann-Pick Disease Foundation. “For too long, our community has been without an approved therapy for the treatment of NPC. Today we celebrate this tremendous milestone for individuals and families living with NPC. We are immensely thankful for the dedication to innovative research that has led to this approval, and we are ready to help families embark on this new chapter of treatment.”
The FDA approval is based on data from the IB1001-301 multinational, randomized, double-blind, placebo-controlled, pivotal clinical trial (NCT05163288), which evaluated the impact of AQNEURSA on neurological symptoms and functioning in pediatric (aged 4 years and older) and adult patients (n=60) with a confirmed diagnosis of NPC.
The trial met the primary efficacy endpoint and all secondary endpoints across all cohorts receiving AQNEURSA. Results from the study showed AQNEURSA significantly improved neurological signs and symptoms and demonstrated functional benefits important to everyday life that were evident within 12 weeks. These findings were published in the February 1, 2024 issue of the New England Journal of Medicine.
The primary outcome assessed by the FDA was a modified version of the Scale for the Assessment and Rating of Ataxia (SARA), referred to as the functional SARA (fSARA). SARA is a clinical assessment tool that assesses gait, stability, speech, and upper and lower limb coordination across eight individual domains. fSARA consists only of gait, sitting, stance, and speech disturbance domains of the original SARA with modifications to the scoring responses. The results in patients who received AQNEURSA compared to placebo showed a greater improvement in fSARA score with a mean treatment difference of -0.4 (95% CI: -0.7, -0.2) with a two-sided p-value of <0.001. Results on the fSARA were supported by consistent results demonstrated on the original SARA.
AQNEURSA was well tolerated in the trial with the most common adverse reactions (incidence ≥5% and greater than placebo in Period I of the trial) being abdominal pain, dysphagia, upper respiratory tract infections, and vomiting.
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.
IMPORTANT SAFETY INFORMATION
Embryo-Fetal Toxicity
Pregnancy and Lactation
Adverse Reactions
Drug Interactions
U.S. full Prescribing Information for AQNEURSA is available at https://intrabio.com/wp-content/aqneursa-prescribing-information.pdf.
AQNEURSA CaresTM Support for Patients
IntraBio offers support programs to eligible patients through their Patient Support Service, AQNEURSA CaresTM. 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.
Forward-Looking Statement
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, which are subject to risks, uncertainties, and other factors. These statements include, but are not limited to, IntraBio’s clinical development programs, regulatory submissions and approvals, and potential market opportunities. These forward-looking statements are based on current expectations and assumptions and involve risks and uncertainties that could cause actual results to differ materially.
Factors that may cause such differences include, among others, uncertainties related to the clinical trial process, regulatory approval scope, limitations and timelines, manufacturing, market acceptance, and the impact of competitive products.
For more information about IntraBio, please visit the company’s website at intrabio.com and follow IntraBio on X (@IntraBio) and LinkedIn (@IntraBio-Inc).
