Final decision will be based on Phase III ALS trial data, due by the end of 2011.

Swiss biopharma firm Actelion is paying Trophos €10 million (about $12.6 million) for an exclusive option to buy the company and get its hands on the late-stage amyotrophic lateral sclerosis (ALS) drug olesoxime. The Phase III study evaluating the mitochondrial pore modulator is due to report by the end of 2011. At this point Actelion will be able to exercise its option and purchase Trophos outright for between €125 million (roughly $158 million) and €195 million (some $246 million) in cash, dependent on regulatory approvals and the clinical progress of Trophos’ CNS drug pipeline.

The firms also agreed on a research collaboration that will give Actelion access to Trophos’ CNS assay technology and compound library. Trophos says its in vitro technology mimics neuronal degeneration pathways and can be used to screen compounds for their ability to impact on neurodegenerative processes.

Trophos is developing a pipeline of candidates for the treatment of diseases including ALS, spinal muscular atrophy (SMA), chemotherapy-induced peripheral neuropathy, and cardiac ischemia reperfusion injury. The pipeline is based on the firm’s cholesterol-oxime chemistry and comprises drug candidates that Trophos claims enhance the function and survival of stressed cells via modulation of dysfunctional mitochondria through interactions at the permeability transition pore (mPTP).

The development of lead compound olesoxime has been supported by €6 million (about $7.6 million) in funding from the EU’s Seventh Framework program. The drug is in addition poised to start in Phase II development as a treatment for SMA. It is being progressed through development for this indication with funding support from the French Association Française contre les Myopathies. Trophos’ second mitochondrial pore modulator candidate TRO40303 is reportedly poised to start in clinical development as a treatment for cardiac ischemia-reperfusion injury. The company has additional preclinical programs focused on disorders including multiple sclerosis and Parksinon disease.

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