Levacetylleucine

IntraBio Inc. today announced that a positive opinion recommending Orphan Medicinal Product Designation for Acetylleucine for the treatment of CACNA1A disorders has been granted by the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA). The European Commission is expected to grant the designation in Q2 2026. CACNA1A-related disorders are rare, genetic neurological conditions characterized by progressive impairment in coordination, balance, speech, and overall neurological function. Currently, no approved therapies exist for these patients. Acetylleucine is a modified amino acid under development for a range of neurodegenerative and neurodevelopmental disorders. Building on its prior Phase III pivotal trials with levacetylleucine for Niemann-Pick disease type C (NPC) and for Ataxia-Telangiectasia, IntraBio is preparing to initiate a multinational Phase III trial evaluating levacetylleucine for CACNA1A-related disorders in 2026 (ClinicalTrials.gov: NCT07221292). “We are moving with urgency to initiate our global Phase III clinical trial and to advance the investigation of levacetylleucine for as many rare disease communities as possible,” said Taylor Fields, Chief Development Officer at IntraBio. “We remain committed to rigorous label expansion in rare and complex neurological diseases with huge unmet medical need.” Levacetylleucine is an investigational product for the treatment of CACNA1A-related disorders. Its safety and efficacy for this specific indication have not been established. Orphan Medicinal Product Designation in the European Union is granted to therapies intended for the treatment of rare, serious, or life-threatening conditions and provides regulatory incentives, including ten years of market exclusivity following approval, which may be extended to twelve years. Levacetylleucine (AQNEURSA®) 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 ≥15 kg. 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. 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. The content above comes from the network. if any infringement, please contact us to modify.

Only successful pivotal Phase III trial in Ataxia-Telangiectasia (A-T), a rare, progressive neurodegenerative disease with no approved treatments Phase III trial successfully achieved its primary endpoint and key secondary endpoints with high statistical significance Subgroup analysis across the endpoints demonstrates consistent clinical benefit across all demographics IntraBio will immediately proceed with filing for marketing approval in the US, Europe, and other jurisdictions for levacetylleucine in Ataxia-Telangiectasia Building on this success, the company is continuing the expansion of its development program for levacetylleucine into additional indications, including CACNA1A-related disorders and other ataxias and lysosomal storage disorders AUSTIN, Texas--(BUSINESS WIRE)--IntraBio Inc. today announced positive results from the full data set for the 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). Key Findings Efficacy and Safety In the randomized, double-blind, placebo-controlled Phase III study, treatment with levacetylleucine resulted in a statistically significant and clinically meaningful -1.92 point improvement on the Scale for the Assessment and Rating of Ataxia (SARA) compared with a -0.14 point change on placebo (p<0.001). The magnitude and consistency of benefit observed across both pediatric and adult patients underscores the clinical relevance of these findings in a disease characterized by relentless neurological decline. The trial also met its 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). Subgroup analysis of the primary and secondary endpoints demonstrated consistent clinical effects across all demographics (age, gender, disease severity, age of symptom onset, etc.). This positive data provides a strong rationale for levacetylleucine to be used in the treatment of A-T. Levacetylleucine was observed to be safe and well-tolerated, with no drug-related serious adverse events, consistent with its established safety profile. “These results in Ataxia-Telangiectasia build directly on our prior successes with levacetylleucine in Niemann-Pick disease Type C,” said Mallory Factor, Chief Executive Officer of IntraBio. “We have now demonstrated across multiple indications through our clinical development strategy that levacetylleucine can deliver meaningful benefit when studied using rigorous, innovative disease-appropriate trial designs. This gives us strong confidence as we expand the program into additional neurodegenerative and neurodevelopmental diseases, including CACNA1A-related disorders.” Based on the robust findings from the study, IntraBio is proceeding immediately with regulatory submissions to the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), and additional global regulatory authorities, to support the label expansion of levacetylleucine for A-T. 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 is currently no approved therapy for the treatment of A-T. Public reports estimate there are approximately 4,600 diagnosed A-T patients in the U.S. and about 5,000 A-T patients in the U.K. and EU4 countries. 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. US Indication and Important Safety Information AQNEURSA ® is approved 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. EMA Indication and Important Safety Information 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. Contacts For further information, please contact: Cass Fields Vice-President of External Affairs ccfields@intrabio.com