Levacetylleucine

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

Quince Therapeutics’ once-monthly steroid-based treatment failed a Phase 3 trial for a rare genetic condition called ataxia-telangiectasia, prompting the Bay Area company to say it would stop development of the experimental product. Thursday’s announcement walloped the company’s shares $QNCX , which were down more than 90% on Friday morning compared with Wednesday’s close. Quince’s therapy, called eDSP, involves a machine that uses some of a patient’s own blood, encapsulating the steroid dexamethasone into blood cells that are then administered back to the patient. The approach was meant to mitigate the side effects of daily steroid treatment. However, in a Phase 3 study with 105 patients, Quince’s treatment did not significantly improve scores at six months compared to placebo on a measure of disease called the RmICARS. The measure primarily examined a patient’s posture and gait. Quince said that the difference in average change in the two arms on the RmICARS was -1.30, with a p-value of 0.0851. In addition, the study failed to meet a key secondary endpoint — the Clinical Global Impression of Severity. Quince’s pivotal trial failure in ataxia-telangiectasia comes one week after IntraBio said its treatment for the disease succeeded. IntraBio is developing a drug called Aqneursa, which is a modified version of leucine, and plans to submit its application to the FDA for ataxia-telangiectasia in April. Ataxia-telangiectasia is a progressive disease that starts in childhood and whose name describes its key symptoms: ataxia — a loss of muscle control — and telangiectasias — tiny red blood vessels that appear on the skin. But the disease can affect many functions of the body, including the immune and nervous systems. With its decision to stop work on its treatment, Quince said it “intends to preserve cash and explore available options.”