Antisense Therapeutics Limited (ASX: ANP, US OTC: ATHJY, FSE: AWY), is an Australian ASX listed (dual-listed in Germany) biotechnology company, developing and commercializing antisense pharmaceuticals for large unmet markets in rare diseases. The products are in-licensed from Ionis Pharmaceuticals Inc. (NASDAQ: IONS), an established leader in antisense drug development.
The Company is developing ATL1102, an antisense inhibitor of the CD49d receptor, for Duchenne muscular dystrophy (DMD). ATL1102 has also demonstrated biologic activity in MS patients.
In 2020 highly promising Phase II trial results on a small study of 9 patients met successful endpoints for safety and efficacy and demonstrated consistent improvements or stabilisation across different measures of motor function and strength.
The company will commence Phase IIb/III Clinical Trials in Europe in 2022. These trials will be a double-bind, pivotal trial with 114 non-ambulant patients. This trial will also be an approvable study positive results should lead to commercialisation and licencing revenue. The global market for DMD therapy is around €3.5 billion.
Early in 2022 the company also announced a world-first study to assess up to 7,000 plasma proteins in Long COVID-19 patients as a collaboration with US based researchers. The study will look for blood disease markers to assess if amenable to treatment with ATL1102.