Percheron Therapeutics has announced that it will investigate avicursen (ATL1102) in other indications besides Duchenne muscular dystrophy (DMD) following disappointing Phase IIb results last month.
In a letter to shareholders published today (6 January), the chair of the board Dr Charmaine Gittleson, alongside non-executive director Dr Gil Price and managing director Dr James Garner, told shareholders that the company “will undertake a broad strategic review of the company’s pipeline to more comprehensively evaluate alternative sources of value within the current assets”.
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The review follows negative topline results from the Phase IIb trial (NCT05938023) of avicursen in boys with non-ambulatory DMD. Announced on 18 December, the results demonstrated that the trial did not meet its primary endpoint, which was the Performance of the Upper Limb 2.0 (PUL2.0) score at week 25 compared to placebo.
The news represented what the cosignatories today called “an enormous disappointment for all our shareholders”, and prompted an 83.3% drop in Percheron’s share price, which is yet to recover.
According to the letter to shareholders, Percheron will now adopt a three-pronged approach: the company will first look to better understand the results of the avicursen trial in order to determine whether to continue development in DMD. It expects to receive further data from the trial this month and plans to complete its analyses by the close of Q1 2025.
Simultaneously, the company will review its pipeline “to more comprehensively evaluate alternative sources of value within the current assets”.
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By GlobalDataAccording to GlobalData’s drugs database, avicursen is currently under development for the treatment of other indications including relapsing-remitting multiple sclerosis (RRMS), for which it is currently in Phase II testing. The drug is also in the preclinical stages for testing in the treatment of limb girdle muscular dystrophy and epilepsy.
Elsewhere in its pipeline, Percheron will also make a “definitive decision” about the future of atesidorsen (ATL1103), which is currently in Phase II development for acromegaly. The company expects to complete discussions within Q1 and will share its conclusions this year.
The third and final prong of the approach looks beyond avicursen and outside of the company’s current portfolio, as Percheron will consider adding new programmes. In today’s letter, the cosignatories said: “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.”
Which potential new avenues the board members are considering are currently unclear, although the letter outlined that upcoming decisions would be “weighty and intricate”, as board members look to establish the direction of the company post-DMD trial.
DMD is a genetic disorder, characterised by progressive muscle degeneration. It is usually diagnosed in children aged two or three and primarily affects boys, occurring in approximately one in every 3,500 male births worldwide.
