Roche buys rare disease firm Trophos in €470m deal

Roche has agreed a €470m deal to buy French pharma company Trophos and its lead drug for the rare neuromuscular disease spinal muscular atrophy (SMA).

Trophos' olesoxime (TRO19622) - which is also in trials for multiple sclerosis - showed promising results in a phase II trial reported last year in SMA, a genetic disease that affects the motor neurons of the voluntary muscles used for activities such as crawling, walking, head and neck control and swallowing.

The orphan disease is the most common genetic cause of infant mortality under the age of two, and is caused by loss of function of the SMN 1 gene, leading to progressive degeneration of nerves in the spinal column and loss of motor neurons.

It affects around 20,000 people worldwide and is emerging as a target for the pharma industry, with Astellas agreeing a $675 licensing deal last month giving it rights to an SMA therapy in development at Cytokinetics, while Biogen Idec and Isis Pharmaceuticals are also developing ISIS-SMNRx for the disease, a drug due to enter phase III soon. Roche also has its own compound for SMA called RG7800 in phase I testing.

Roche is paying €120m upfront for Marseilles-based Trophos, with another €350m promised in the form of milestone payments. The French company formerly had an alliance with Actelion, but the latter withdrew from the collaboration after olesoxime failed a trial in amyotrophic lateral sclerosis in 2011.

The results of Trophos' phase II trial of olesoxime showed a beneficial effect on the maintenance of neuromuscular function in individuals with two slightly less severe forms (type II and type III) of SMA, as well as a reduction in medical complications associated with the disease.

While the most severe form of SMA - type I - is typically fatal by the age of two, type II and III patients can live to adulthood although they generally require high levels of care.

Olesoxime is thought to work by preserving the integrity of mitochondria - organelles that generate the energy used by cells - in stressed neurons. Preclinical studies have shown it can promote the function and survival of neurons subjected to various stress conditions.

The acquisition comes amid a busy period for Roche, which so far this month has also announced a $750m antibiotic deal with Meiji Seika and Fedora, a $1bn agreement to take control of genomics firm Foundation Medicine and a collaboration with genetic testing specialist 23andMe in Parkinson's disease.