European Medicine Regulators Recommend Approval of AQNEURSA® (levacetylleucine) for Treatment of Niemann-Pick Disease Type C in EU

IntraBio Inc., a global biopharmaceutical company, continues to make strides in the treatment of rare neurological and neurodevelopmental diseases. The company's flagship product, AQNEURSA (levacetylleucine), has recently received U.S. FDA approval and has been experiencing rapid adoption as a frontline therapy for Niemann-Pick disease (NPC).

AQNEURSA, developed by scientists from the University of Oxford and the University of Munich, has shown rapid symptomatic improvement, long-term benefit, and disease-modifying, neuroprotective effects according to Phase III pivotal trial data and extension phase data. The most common adverse reactions (incidence ≥5% and greater than placebo) for AQNEURSA include abdominal pain, dysphagia, upper respiratory tract infections, and vomiting. It is important to note that AQNEURSA may cause embryo-fetal harm when administered during pregnancy.

Patients aged 4 years and older were screened at trial sites in Australia, Europe, the United Kingdom, and the United States. The European Commission decision for AQNEURSA's approval is expected later this year, following the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) issuing a positive opinion recommending approval.

Takeda GmbH plans to obtain approval for Aqneursa for Niemann-Pick disease in Germany. IntraBio remains on track to proceed with additional global regulatory submissions for AQNEURSA in 2025 and beyond.

IntraBio's platform technologies result from decades of research and collaboration with universities and institutions worldwide. The company is currently conducting a multinational, randomized, placebo-controlled, crossover trial (IB1001-301, NCT05163288) evaluating the safety and efficacy of IB1001 (AQNEURSA, levacetylleucine) in pediatric and adult patients with NPC.

In this trial, patients were assessed during a baseline period and then randomly assigned (1:1) to receive orally administered IB1001 or placebo for 12 weeks. At the end of the 12-week treatment period, patients crossed over and initiated therapy with the alternate study drug (IB1001 or placebo) over the subsequent 12-week period. The trial's primary outcome measures include changes in the modified Rankin Scale (mRS) and the NPC disease-specific Quality of Life Questionnaire (NPC QOL-19).

Some patients who completed the study have been dosed for over 5 years in an open-label Extension Phase. Data readout for IntraBio's Phase III Pivotal trial for Ataxia-Telangiectasia is expected in Q1 of 2026.

IntraBio's commitment to advancing treatments for rare diseases like NPC is evident in their ongoing efforts. With the positive opinion from the CHMP and ongoing trials, the future looks promising for those affected by NPC.