Roche has agreed to acquire Trophos, a privately held biotechnology company based in Marseille, France. The company has developed a proprietary cholesterol-oxime based chemistry platform. Trophos's mitochondrial targeted compounds enhance the function and survival of stressed cells by preventing mitochondrial permeability transition, a key determinant of cell death or survival.

Trophos's proprietary screening platform generated olesoxime (TRO19622), which is being developed for SMA, a rare and debilitating genetic neuromuscular disease that is most commonly diagnosed in children. Results from a pivotal phase II clinical trial with olesoxime in SMA showed a beneficial effect on the maintenance of neuromuscular function in individuals with type II and non-ambulatory type III SMA, as well as a reduction in medical complications associated with the disease.

"This acquisition highlights Roche's commitment to developing medicines for spinal muscular atrophy, a serious disease with no effective treatment," said Sandra Horning, M.D., chief medical officer and head of global product development at Roche. "We will build on the work done by Trophos and the French Muscular Dystrophy Association to advance the development of olesoxime and to bring it to people who live with this devastating condition as quickly as possible."

Trophos's shareholders will receive an upfront cash payment of $138 million, plus additional contingent payments of up to $404 million based on achievement of certain predetermined milestones.

SMA is a life-limiting and highly disabling genetic disease characterized by progressive muscle weakness and loss of motor function. SMA affects the motor neurons of the voluntary muscles used for activities such as crawling, walking, head and neck control and swallowing. Typically, SMA presents in early childhood and is the most common genetic cause of infant mortality. It is one of the most common rare diseases, with one in 6,000 to one in 10,000 children affected.

SMA is an autosomal recessive genetic disease caused by a loss of function of the Survival Motor Neuron (SMN) 1 gene, which leads to insufficient levels of SMN protein, progressive deterioration of nerve cells in the spinal cord and loss of motor neurons. The mutated SMN1 gene responsible for SMA is carried by up to 20 million potential parents in the U.S. and E.U., most of them unaware that they are carriers.