Biotechnology company Percheron Therapeutics (ASX: PER) has announced full completion of a nine-month toxicology study in non-human primates for its lead program, avicursen.
Key Points
* The nine-month toxicology study is a key regulatory requirement to enable future clinical trials and commercialization in the United States.
* The study commenced in March 2023 and is now fully completed, with a final study report in hand.
* Results of the study remain broadly consistent with the earlier six-month study, as previously reported in the company’s announcement of 27 May 20241. No new or unexpected toxicities were observed, and no animals died on study. Expected low- grade findings were fully reversible during the recovery period.
* The company expects to discuss these results with the FDA in early CY2025, with the goal of lifting clinical hold in the United States.
“We are very pleased to have this important piece of work completed on schedule,” CEO, Dr James Garner, said.
“The results seem to us, and to our consultants and advisors, to be consistent with earlier observations, which we expect to have positive implications for the future development of avicursen. Given the impending availability of clinical data from the phase IIb clinical trial of avicursen in December 2024, we will likely target our regulatory discussions with the FDA for early CY2025, so as to present the agency with the strongest possible package of data.”
The requirement for a nine-month non-rodent toxicology study prior to any clinical trial involving dosing beyond six months was communicated to the Company by the FDA during previous regulatory discussions. Because avicursen is intended to be administered over an extended period, the study effectively represents a prerequisite for conducting clinical trials in the United States.
Next Steps
The Company expects to discuss the outcomes of the study with the FDA in early CY2025, with a view to enabling the conduct of future clinical trials in the United States and supporting a potential product approval in that market.
Avicursen is the subject of an ongoing international phase IIb randomised controlled trial in non-ambulant boys with Duchenne muscular dystrophy, with initial data expected in December 2024.