Percheron Therapeutics Completes Recruitment For Phase IIb Study Of ATL1102

Percheron Therapeutics Ltd (ASX: PER) has completed recruitment to its ongoing international phase IIb study of ATL1102 in the treatment of Duchenne muscular dystrophy (DMS).

In total, 48 boys were randomised to the study across sixteen hospitals in five countries.

The last patient into the study was randomised on 28 May 2024.

Watch: Percheron Therapeutics CEO Dr James Garner On JustStocks Video As ATL1102 Study Completes Recruitment

CEO comment

 Percheron CEO Dr James Garner said: “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.

 “With the study now fully enrolled, we expect to have initial topline data in December 2024.”

Promising signals of activity

TL1102 is an antisense oligonucleotide that targets CD49d.

Administration of ATL1102 has been shown to reduce CD49d positive lymphocytes, which may be therapeutic in a range of inflammatory diseases.

A completed phase IIa study of the drug in nine non-ambulant boys with DMD showed very promising signals of activity.

Read: Percheron Therapeutics Welcomes Positive Preliminary Toxicology Results For ATL1102

Primary endpoint

The present study aims to provide a deeper and more robust evaluation of ATL1102’s potential activity in DMD.

Study design comprises two doses of ATL1102 compared against placebo and enrolled ~15 subjects in each arm.

The primary endpoint is the change in the performance of the upper limb module (PUL2.0) at six months.

Duchenne muscular dystrophy

DMD is a genetic condition that is thought to affect around 1 in 10,000 males.

The disease results from mutations in the DMD gene, which codes for dystrophin, a structural protein in muscle.

Symptoms typically manifest in early childhood, and patients are generally wheelchair-bound by their teens.

Next steps

With enrolment now complete, the timelines for the remainder of the study are as follows:

Percheron expects to discuss emerging results from the study with regulatory agencies such as FDA to determine the optimal path to registration for ATL1102.



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