Percheron Therapeutics Successfully Completes Nine-Month Toxicology Study for Avicursen
PER (PER) Share Update September 2024 Sunday 29th
Percheron Therapeutics Completes Key Toxicology Study for Avicursen
Percheron Therapeutics Limited (ASX: PER) has announced the successful completion of a crucial nine-month toxicology study in non-human primates for its leading drug candidate, avicursen. This milestone is a significant step toward future clinical trials and potential commercialization in the United States.
Instant Summary:
- Completion of a nine-month toxicology study in non-human primates.
- Study results consistent with earlier six-month study.
- No new or unexpected toxicities observed.
- Expected low-grade findings were fully reversible.
- Plans to discuss results with FDA in early 2025.
Study Completion and Results
Percheron Therapeutics Limited has successfully completed a nine-month toxicology study in non-human primates for its lead program, avicursen. This study is a key regulatory requirement for enabling future clinical trials and commercialization in the United States. The study commenced in March 2023 and concluded with a final report in hand.
The results of the study are broadly consistent with the earlier six-month study, as previously reported in May 2024. No new or unexpected toxicities were observed, and no animals died during the study. All expected low-grade findings were fully reversible during the recovery period.
Regulatory Path Forward
The company plans to discuss these results with the FDA in early 2025, aiming to lift the clinical hold in the United States. 'We are very pleased to have this important piece of work completed on schedule,' commented 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, Percheron plans to target regulatory discussions with the FDA for early 2025 to present the strongest possible package of data.
Study Details
The nine-month toxicology study was performed in accordance with Good Laboratory Practice (GLP) by a specialist contract research organization. Dosing concluded on schedule in December 2023. The majority of the animals then underwent pathological examination, while the remaining animals continued into a recovery phase, during which avicursen was not administered. The six-month recovery phase concluded in June 2024, after which the recovery animals also underwent pathological examination.
Next Steps
Percheron expects to discuss the outcomes of the study with the FDA in early 2025, with a view to enabling the conduct of future clinical trials in the United States and supporting a potential product approval in that market. The exact timing of these interactions will be determined in consultation with the company’s regulatory advisors.
Avicursen is the subject of an ongoing international phase IIb randomized controlled trial in non-ambulant boys with Duchenne muscular dystrophy, with initial data expected in December 2024.
The successful completion of the nine-month toxicology study is a significant milestone for Percheron Therapeutics. It not only meets a critical regulatory requirement but also paves the way for future clinical trials in the United States. This achievement is likely to have a positive impact on the company's stock as it demonstrates progress and adherence to regulatory guidelines. Investors may view this as a step closer to potential commercialization, thereby boosting investor confidence.
Investor Reaction:
Analysts are likely to react positively to this announcement, as it indicates substantial progress in the development of avicursen. The consistency of the study results with earlier findings and the absence of new toxicities are encouraging signs. However, the true impact on the stock will depend on the upcoming discussions with the FDA and the results of the phase IIb clinical trial expected in December 2024.
Conclusion:
Investors should keep an eye on Percheron Therapeutics as it moves forward with its regulatory discussions and awaits the results of the phase IIb clinical trial. The successful completion of the nine-month toxicology study is a promising development, and the company's strategic steps in the coming months will be crucial in determining its future trajectory.