Advancing AX-0810 through ongoing Phase 1 dosing, with target engagement data expected 1H 2026Selected Development Candidates AX-2402 for Rett syndrome (MECP2, R270X) and AX-2911 for MASH (PNPLA3)Ended 2025 with € 92.4 million cash and cash equivalents and achie
Initial pipeline programs with liver delivery to address Cholestatic Diseases targeting NTCP and Cardiovascular Disease targeting B4GALT1; initiation of clinical trials anticipated in late 2024/early 2025
Axiomer activity demonstrated across multiple preclinical in vitro
Initial AX-0810 data show no safety signals after 4 weeks of dosing and pharmacokinetics consistent with non-clinical data; Phase 1 enrollment and dosing in healthy volunteers ongoing with target engagement data expected in H1 2026, followed by inclusion of a patient cohort
Access based on positive interim analysis of clinical data as well as preclinical data to date PRIME designation provides a pathway for frequent and early interactions with the EMA aimed at supporting accelerated evaluation and approval
Reported rapid, significant and durable improvements in vision at twelve months
Concordant improvement in key secondary outcome measures
The target registration dose of sepofarsen was well-tolerated with a favorable benefit/risk profile
Strengthens confidence
Des améliorations rapides, significatives et durables de la vision à douze mois ont été rapportéesAmélioration concordante des mesures des résultats secondaires clésLa dose d'enregistrement cible du sepofarsen a été bien tolérée avec un profil risque/bénéfice fa
