Slide 1: ATL1102 for DMD: Revised Clinical Study Plans
(ASX: ANP) ATL1102 for DMD: Revised Clinical Study Plans September 2022
Slide 2: Antisense Therapeutics Limited – Presentation Disclaimer
Antisense Therapeutics Limited – Presentation Disclaimer This presentation contains certain market estimates and forward-looking statements regarding the Company’s business & the therapeutic & commercial potential of its technologies & products in development. Any statement provided by the Company o
Slide 3: Phase IIb Study Program
Phase IIb Study Program Title: A multicentre, randomised, double-blind, placebo-controlled and open label extension study to assess the efficacy, safety, and pharmacokinetic profile of two dose levels of ATL1102 administered by subcutaneous (sc) injection in non-ambulatory participants with Duchene
Slide 4: Phase IIb Study Program (Continued….)
Phase IIb Study Program (Continued….) Sample size: • Approximately 45 participants to be enrolled (15 per treatment arm) with 42 participants to complete the study Target population: • Participants with DMD (confirmed by genetic testing) and are non-ambulatory, defined as unable to walk 10 meters wi
Slide 5: Phase IIb Study Program (Continued….) Blinded Phase - Study Objectives
Phase IIb Study Program (Continued….) Blinded Phase - Study Objectives Primary objective: To evaluate the effect of ATL1102 on upper limb muscle function in non-ambulant participants with DMD as assessed by change in the Performance of Upper Limb Module for DMD 2.0 (PUL 2.0) score compared to placeb
Slide 6: Randomisation
Phase IIb Study Program (Continued….) A multicentre, randomised, double-blind, placebo-controlled and open label extension study to assess the efficacy, safety, and pharmacokinetic profile of two dose levels of ATL1102 administered by subcutaneous (sc) injection in non-ambulatory participants with D