Percheron Halts Avicursen Trial, Raises $14.85M to Pivot Pipeline Strategy
Percheron Therapeutics has terminated its phase IIb avicursen trial for Duchenne muscular dystrophy after failing to meet efficacy endpoints, while securing $14.85 million in fresh capital to explore new therapeutic opportunities.
- Phase IIb avicursen trial in Duchenne muscular dystrophy terminated due to lack of efficacy
- Company raised $13 million via institutional placement and $1.85 million through Share Purchase Plan
- Strategic review underway to identify new pipeline opportunities with shareholder value focus
- US OTCQB uplisting completed, ticker changed to PERCF
- Board faces shareholder challenge with proposed director removals and appointments
Trial Termination Marks a Major Setback
Percheron Therapeutics Limited (ASX:PER) announced the immediate termination of its phase IIb clinical trial of avicursen (ATL1102) in Duchenne muscular dystrophy (DMD) after topline data failed to demonstrate a statistically significant efficacy signal. The trial did not meet its primary endpoint, the Performance of the Upper Limb 2.0 (PUL2.0) score at week 25, with p-values well above the threshold for significance. This outcome was both disappointing and unexpected for the company and its stakeholders.
Despite the setback, avicursen was reported to be safe and well-tolerated, with only mild to moderate injection site reactions observed. However, the lack of efficacy prompted Percheron’s board to conclude that continuing the trial would not serve the interests of patients or shareholders.
Capital Raising to Fund Strategic Pivot
In response to the trial’s termination, Percheron successfully completed a $13 million institutional placement alongside a $1.85 million Share Purchase Plan (SPP), raising a total of $14.85 million. Originally intended to fund the remainder of the avicursen study, these proceeds will now be redirected towards identifying and pursuing new therapeutic opportunities that offer a faster and more reliable path to shareholder value creation.
The company’s CEO, Dr James Garner, emphasized that the strategic review currently underway aims to rebuild the pipeline through in-licensing and other avenues. The recent attendance at the JP Morgan Healthcare Conference facilitated over fifty meetings with potential licensors, with several discussions advancing under confidentiality.
Corporate and Market Developments
Percheron also announced the uplisting of its US foreign shares from the Pink Sheets to the OTCQB market, changing its ticker from ATHJF to PERCF. This move is designed to enhance liquidity and visibility among US investors following the discontinuation of its American Depository Receipt (ADR) facility.
Meanwhile, the company faces governance challenges as a group of shareholders holding over 5% of voting rights have requisitioned a general meeting to propose the removal of the current Chair, Dr Charmaine Gittleson, and CEO Dr James Garner, seeking to appoint new directors. This development adds a layer of uncertainty as the company navigates its strategic reset.
Financial Position and Outlook
Percheron closed the quarter ending 31 December 2024 with a robust cash balance of $17.39 million, bolstered by the recent capital raises and a $2.35 million R&D tax incentive rebate. Operating cash outflows were $2.91 million for the quarter, primarily reflecting clinical trial expenditures prior to termination. The company anticipates trial closure costs in the range of $6 to $7 million but has taken measures to conserve cash and halt further spending on the ATL1102 program.
With an estimated six quarters of funding available at current burn rates, Percheron is positioned to execute its strategic review and pursue new pipeline assets into calendar year 2026. The upcoming investor webinar scheduled for 6 February 2025 will provide further insights into the trial data and the company’s forward plans.
Bottom Line?
Percheron’s pivot from avicursen to new opportunities will be critical to restoring investor confidence and defining its next growth chapter.
Questions in the middle?
- What new therapeutic candidates or partnerships will emerge from Percheron’s strategic review?
- How will the proposed board and executive changes impact company direction and investor sentiment?
- What are the detailed implications of the trial closure costs on Percheron’s financial runway?