Percheron Therapeutics Ltd (ASX:PER, OTC:PERCF) has vowed to regroup and diversify its portfolio after some negative topline results from its Phase 2b trial of avicursen (ATL1102) in Duchenne muscular dystrophy (DMD), announced on December 18 last year.
Plans to rebuild
In a letter to shareholders, members of the Percheron board acknowledged the shareholder and patient disappointment familiar to so many of the biotechs that take on the risk of clinical trials, and laid out the company’s plans to rebuild following the setback.
The letter cited a potential portfolio expansion, the exploration of conditions beyond DMD for which the company’s current assets could work, and a strong cash runway, which it has taken steps to conserve.
“By virtue of our recent capital raising activities we are in the fortunate position of having choices in respect of what Percheron may become.
“The board is approaching this question with an entirely open mind and our only guiding principles are to continue to seek impactful new therapies for diseases with high unmet need, and to strive to reconstitute the value of the company on behalf of our shareholders.”
For now, the company said, its first priority is to understand the results of the avicursen trial and to interrogate if there are convincing reasons to continue development in DMD.
Percheron is expecting further data from the trial at various points throughout January and will plan to complete its analyses and share its conclusions by the end of the first quarter of this calendar year.
“To manage expectations, we should be clear that not all trial results are entirely explicable – sometimes drugs simply don’t work – but we think it is important to learn everything we can so that we are able to make rational and informed decisions about the program’s future, and to benefit future research in DMD,” the letter said.
Review of pipeline
Percheron also plans to conduct a broad strategic review of the company’s pipeline to more comprehensively evaluate alternative sources of value within the current assets.
“Avicursen has shown evidence of activity in a range of other conditions, and we have always been clear that, while DMD was the lead indication for the drug, it was not the only use case,” the company said.
Percheron also acknowledged that, regardless of the outcomes of its analyses, it would likely need to diversify its pipeline and it would be looking to add one or more new programs to its portfolio.
“We have already begun exploring opportunities that may provide a faster and more secure path to restoring shareholder value and we have initiated discussions with several potential partners,” the letter said.
Highly optimistic
“Some very successful companies have been built with lesser ingredients, and we remain highly optimistic that Percheron can yet become the business that each of us joined the board to create, and that our shareholders have so resolutely supported in recent years.”
In closing, the company had these words for the DMD community: “It has been our privilege to work with the clinicians, researchers, patient advocates and families who collaborate every day to seek new therapies for patients with this devastating illness, and we will never cease to be inspired by their devotion and perseverance.
“We greatly regret that avicursen has not yet rewarded the hopes that these many stakeholders have invested in it, but we do not regret that we tried.”