George Tachas, PhD, on Evaluating Antisense Oligonucleotides in Non-Ambulant Boys With DMD

“The most important thing for us is, in the non-ambulant boys at the moment, which is in a phase 2b trial, that we reproduce these results of stabilizing upper limb function by the PUL 2.0 and also strength.The other things are almost like secondary beneficial effects, like maintaining muscle mass and maintaining bone, there will be additional benefits that we can hopefully bring to the treatment.”

Perhceron Therapeutics’ ATL1102 modulated genetic modifiers of disease progression and fibrosis in non-ambulant boys with Duchenne muscular dystrophy (DMD) closer to control levels (P <.0005), including sVCAM-1, LTBP4, BMP5, BMP6, IGF-I, and Thrombospondin-1. ATL1102 is an antisense oligonucleotide designed to target inflammation as a secondary cause of muscle damage in patients with DMD.

These data, from a proteomic analysis of plasma from participants in a phase 2 trial (NCT05938023) of ATL1102, were presented by George Tachas, PhD, lead scientist, Percheron Therapeutics at the 2024 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, held March 3-6, in Orlando, Florida.

CGTLive® spoke with Tachas to learn more about the proteomics analysis and findings with ATL110 in boys with DMD. He shared plans for an upcoming blinded study of ATL1102 which is currently recruiting to around 45 participants.Topline data from the study is expected in the second half of 2024, and Tachas shared his hope that a clinical benefit will be seen corresponding with the proteomic findings.

Click here to view more coverage of the 2024 MDA Conference.

REFERENCE

Tachas G, DeLisle R, Mueller C, et al. ATL1102 treatment of non-ambulant boys with DMD stabilizes function modifying plasma proteins with roles in immune, fibrosis, bone & growth physiology. Presented at: 2024 MDA Clinical and Scientific Conference; March 3-6; Orlando, FL. Poster #V401