Percheron Therapeutics Welcomes Positive Preliminary Toxicology Results For ATL1102

Percheron Therapeutics Ltd (ASX: PER) has confirmed that no new or unexpected toxicities were identified in the preclinical toxicology assessment of its investigational new drug, ATL1102.

ATL1102 is an antisense oligonucleotide targeting the CD49d receptor.

The nine-month toxicology study is a key regulatory requirement to enable future clinical trials and commercialisation in the United States.

Percheron accordingly commenced the study in March 2023.

Recovery phase to conclude in June 2024

The study is performed in accordance with Good Laboratory Practice (GLP) by a specialist contract research organisation.

Dosing concluded on schedule in December 2023.

The majority of the animals then underwent pathological examination.

A proportion of the animals continued into a recovery phase, during which ATL1102 is not administered, and the purpose of which is to establish the reversibility of any observations.

This recovery phase is expected to conclude in June 2024, after which the recovery animals will also undergo pathological examination.

“Broadly confirmatory so far”

 Percheron CEO Dr James Garner said: “We are very encouraged by the results we have seen so far.

 “While some analyses remain ongoing, and definitive interpretation awaits completion of the recovery phase, the observations to date seem quite consistent with earlier data, and the study has not identified any new or unexpected toxicities associated with ATL1102.

 “We await data from the recovery phase of the study before we can draw final conclusions, but the results appear to be broadly confirmatory so far.”

Next steps

Final data from the nine-month toxicology study, together with specialist evaluation and interpretation of the results, are expected to be available in 4Q CY2024.

Percheron expects to discuss the outcomes of the study with FDA once the final data is available.

This will enable the conduct of future studies in the US, should they be needed, and as a necessary step toward any future NDA filing.

Duchenne muscular dystrophy

ATL1102 is currently the subject of an ongoing international phase IIb clinical trial in non-ambulant boys with Duchenne muscular dystrophy (DMD).

Data is expected in 2H CY2024.

DMD is a severe type of muscular dystrophy predominantly affecting boys.

The onset of muscle weakness typically begins around age four, with rapid progression.



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