Percheron Therapeutics is an international biotech company focused on developing novel therapeutics for rare diseases.

The company’s lead asset is ATL1102, an antisense oligonucleotide to VLA4, which is currently the subject of an ongoing international phase IIb randomized controlled trial in Duchenne muscular dystrophy. Data is expected in 2H CY2024. ATL1102 previously showed very promising signals in an exploratory phase IIa study in the same population. The drug has orphan designation from FDA and EMA, and rare pediatric disease designation from FDA.

The company is also developing ATL1103, an antisense oligonucleotide therapy which targets human growth hormone receptor, and which is in phase II for acromegaly.

Both assets were developed utilizing platform technologies licensed from Ionis Pharmaceuticals, Inc (NASDAQ: IONS).

Percheron is listed on the Australian stock exchange (PER) and the Frankfurt stock exchange (AWY) and is quoted via OTC markets in the US (ATHJY).