Half-Year 2022 Financial and Clinical Trials Update slide image

Half-Year 2022 Financial and Clinical Trials Update

Tominersen (RG6042, HTT ASO) Antisense oligonucleotide (ASO) targeting human HTT mRNA Indication Huntington's disease Phase/study Phase III Generation HD1 N=791 # of patients ARM A: Tominersen 120mg q2w ARM B: Tominersen 120mg q4m ARM C: Placebo q2w Design ▪ CUHDRS globally Primary endpoint TFC USA only Status CT Identifier Phase III GEN-EXTEND N=1,050 Roche OLE study in patients participating in prior Roche and Genentech sponsored studies ARM A: Tominersen 120mg q2w • ARM B: Tominersen 120mg q4m ■ Long term safety, tolerability FPI Jan 2019 ▪ Q1 2019 protocol modified to allow for bi-monthly vs four-monthly dosing, FPI for new protocol July 2019 ■ Recruitment completed Q2 2020 ■ Dosing stopped in Q1 2021 based on IDMC recommendation regarding the potential benefit/risk profile for study participants. No new safety signals identified. Data presented at EHDN and CHDI 2022 • ⚫ FPI Q2 2019 Dosing stopped in Q1 2021 NCT03761849 NCT03842969 In collaboration with lonis Pharmaceuticals CUHDRS=composite Unified Huntington's Disease Rating Scale; TFC=total function capacity; HTT=Huntingtin; OLE=Open Label Extension; IDMC-Independent Data Monitoring Committee; CHDI-Huntington's Disease Association of Ireland; EHDN-European Huntington's Disease Network 147 Neuroscience
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