Percheron Therapeutics completes recruitment in Phase 2B study of ATL1102 in DMD

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Percheron Therapeutics (ASX:PER) has announced the successful completion of recruitment in its ongoing Phase 2b study of ATL1102 in treating Duchenne Muscular Dystrophy.

In June 2023, Percheron commenced recruitment to the randomised controlled trial of ATL1102 in the treatment of non-ambulant boys with Duchenne muscular dystrophy (DMD).

The study is now fully enrolled.

In total, 48 boys were randomised to the study across sixteen hospitals in five countries. The primary endpoint of the study is the change in PUL2.0 score at six months.

“We are delighted to have this study fully recruited,” said Percheron CEO Dr James Garner.

“The need for new therapies in Duchenne muscular dystrophy has never been more acute, and we very much hope that ATL1102 will be able to make an important contribution to the treatment of this devastating disease. We are profoundly grateful to the participating investigators and their teams, and to the families who have agreed to participate. With the study now fully enrolled, we expect to have initial topline data in December 2024.”

ATL1102 is an antisense oligonucleotide that targets CD49d. The company said the administration of ATL1102 has been shown to reduce CD49d-positive lymphocytes, thereby exerting an immunomodulatory effect, which may be therapeutic in various inflammatory diseases.

It said a completed phase 2a study of the drug in nine non-ambulant boys with DMD showed very promising signals of activity, and this work was recently published in a peer-reviewed journal.

The new study's design comprises two doses of ATL1102 compared against placebo and enrolled approximately 15 subjects in each arm. The primary endpoint is the change in the performance of the upper limb module (PUL2.0) at six months. Subjects thereafter transition to an open-label extension phase where those treated with ATL1102 receive a further six months of treatment, and those originally randomised to placebo are reallocated to one of the two active arms.