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NEWS

IntraBio Reports Topline P-III (IB1001-303) Trial Data on Aqneursa for Ataxia-Telangiectasia

Shots: IntraBio has reported the topline P-III (IB1001-303) trial data on Aqneursa (levacetylleucine) vs PBO in pediatric & adult pts with Ataxia-Telangiectasia (A-T) Trial met the 1EP with a significant –1.88 SARA improvement vs PBO (–1.92 vs –0.14) after 12wks.& achieved 2EPs, showing meaningful gains on ICARS (–4.22 vs –1.69) & CGI-I (–0.6 vs –0.2)…
4 days ago

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NEWS

IntraBio’s Aqneursa Receives the EC Approval for Treating Niemann-Pick Disease Type C (NPC)

Shots: The EC has approved Aqneursa (levacetylleucine), available as 1g granules for oral suspension, with or without miglustat for treating adults & pediatric pts (≥6yrs.; ≥20kg) with NPC Approval was backed by P-III (IB1001-301) crossover study assessing Aqneursa vs PBO in NPC pts, which showed improved neurological symptoms & function after 12wks. as measured by…
5 days ago

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NEWS

IntraBio’s Aqneursa Receives the CHMP’s Positive Opinion for Treating Niemann-Pick Disease Type C (NPC)

Shots: The CHMP has recommended Aqneursa (levacetylleucine) for treating adults & pediatric pts with NPC; additional global submissions are planned for 2025 & beyond Approval was backed by P-III (IB1001-301) study assessing Aqneursa vs PBO in pts (≥4yrs.; n=60) with NPC, which showed improved neurological symptoms & function across all 1 & 2EPs within 12wks.,…
July 30, 2025

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