Percheron Therapeutics is set to broaden its pipeline following the disappointing Phase IIb trial results of avicursen (ATL1102) in Duchenne muscular dystrophy (DMD). The company aims to explore new therapeutic avenues for avicursen and other assets, while also seeking to in-license new programs.
The announcement follows the Phase IIb trial (NCT05938023) of avicursen, which failed to meet its primary endpoint, the Performance of the Upper Limb 2.0 (PUL2.0) score at week 25 compared to placebo, in boys with non-ambulatory DMD. The news led to an 83.3% drop in Percheron’s share price.
Strategic Review and Future Plans
In a letter to shareholders, Percheron's board outlined a three-pronged approach to rebuild value. First, the company will further analyze the avicursen trial data, expected this month, to determine whether to continue its development in DMD. The analysis is slated for completion by the end of Q1 2025.
Simultaneously, Percheron will conduct a strategic review of its pipeline to identify alternative applications for its current assets. According to GlobalData’s drugs database, avicursen is in Phase II testing for relapsing-remitting multiple sclerosis (RRMS) and preclinical stages for limb girdle muscular dystrophy and epilepsy.
The company will also decide on the future of atesidorsen (ATL1103), currently in Phase II development for acromegaly, with conclusions expected within Q1 of this year.
Expanding the Portfolio
Percheron is also looking beyond its current portfolio to add new programs. The board has initiated discussions with potential partners to explore opportunities that may provide a faster and more secure path to restoring shareholder value.
The company has emphasized its commitment to seeking impactful new therapies for diseases with high unmet needs. "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 board stated in its letter to shareholders.
DMD Context
DMD is a genetic disorder characterized by progressive muscle degeneration, primarily affecting boys. It occurs in approximately one in every 3,500 male births worldwide, highlighting the significant unmet need for effective treatments.
