Articles from IntraBio Inc.

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.

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).

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).

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).

IntraBio Inc., a biopharmaceutical company focused on developing therapies for rare neurological diseases, today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending approval of AQNEURSA® (levacetylleucine) for the treatment of Niemann-Pick disease type C (NPC).

IntraBio Inc. announced today that it has completed recruitment for its Phase III Pivotal Trial “IB1001-303”, Effects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T): A randomized, placebo-controlled, double-blind, crossover study (NCT06673056).

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.

- First positive clinical trial for GM2 Gangliosidosis - IB1001 demonstrated statistically significant and clinically meaningful change in both the primary and secondary endpoints - Significant improvement in gait, fine motor skills, speech, cognition, overall functioning and quality of life reported - IB1001 rapidly improved both motor and cognitive symptoms in 6-weeks, consistent with its pharmacological action - Subgroup analysis across the

Multinational clinical trial is the first successful clinical trial for GM2 Gangliosidosis; favourable safety and efficacy data consistent with previously announced IB1001 clinical trial results for NPC OXFORD, UK / ACCESSWIRE / August 19, 2021 / IntraBio Inc today announced positive data from its multinational clinical trial of IB1001 for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff disease). IB1001 demonstrated a statistically