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)
- Also being evaluated for CACNA1A-related disorders in the US & EU, IntraBio is planning to submit regulatory filings of Aqneursa to the US FDA, EMA & other global health authorities for the treatment of A-T
Ref: Businesswire | Image: IntraBio | Press Release
Related News: IntraBio’s Aqneursa Receives the EC Approval for Treating Niemann-Pick Disease Type C (NPC)
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