Percheron Therapeutics (ASX:PER) has announced the completion of a nine-month toxicology study in non-human primates for its lead drug candidate, avicursen, marking a significant step towards potential clinical trials and commercialization in the United States. The study, a critical requirement by the FDA, showed consistent and reversible findings, paving the way for future discussions with the regulatory body.
Toxicology Study Details
The toxicology study, which commenced in March 2023, involved dosing subjects followed by a six-month recovery phase to assess the reversibility of any observed effects. The results remained broadly consistent with an earlier six-month study, with no deaths or unexpected toxicities reported. Low-grade findings were fully reversible during the recovery period, indicating a favorable safety profile for avicursen.
Regulatory Pathway and FDA Discussions
Percheron intends to engage with the FDA in early 2025 to discuss the study results and the potential lifting of the clinical hold on avicursen. The nine-month toxicology study was a prerequisite for conducting clinical trials involving dosing beyond six months, as communicated by the FDA in previous regulatory discussions. According to Percheron, because avicursen is intended to be administered over an extended period, the study effectively represents a prerequisite for conducting clinical trials in the US.
Avicursen's Potential and Ongoing Clinical Trial
Avicursen, an antisense oligonucleotide, targets the CD49d biomarker and has demonstrated immunomodulatory effects by reducing CD49d-positive lymphocytes. It is currently being evaluated in an ongoing international Phase IIb randomized controlled trial in non-ambulant boys with Duchenne muscular dystrophy (DMD). This trial builds upon earlier studies that showed promising signals of activity. Initial data from the Phase IIb trial is expected in December 2024 and will be included in the data package for the FDA.
CEO's Perspective
"We are very pleased to have this important piece of work completed on schedule," said Percheron CEO Dr. James Garner. "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."
