Roche, a leader in research-focused healthcare with combined strengths in pharmaceuticals and diagnostics, has agreed to acquire Trophos, a privately held biotechnology company based in Marseille, France.

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. These data were first presented in April 2014 at the annual meeting of the American Academy of Neurology (AAN).

“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.”

Under the terms of the agreement, Trophos’s shareholders will receive an upfront cash payment of EUR 120 million, plus additional contingent payments of up to EUR 350 million based on achievement of certain predetermined milestones.

“SMA is a grievous disease with a huge impact on the daily life of patients and their families, who are currently left only with supportive care. We are proud to see the development of this medicine evolving, with the ultimate goal of a potential first medicine for SMA,” said Christine Placet, chief executive officer of Trophos. “This is a tremendous recognition of the work done by Trophos’s teams and supporters over the past 16 years.”

SMA is a life-limiting and highly disabling genetic disease characterised 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.

Olesoxime (TRO19622) is an investigational medicine designed to protect the health of motor nerve cells. Results of a pivotal phase II study of olesoxime in Type II and non-ambulatory Type III SMA patients from the ages of three to 25 years were first presented in April 2014 at the 66th American Academy of Neurology (AAN), Philadelphia, PA, USA. Trophos’s development program was supported by the French Muscular Dystrophy Association. Olesoxime has been granted ‘Orphan Medicinal Product’ designation for the treatment of SMA by the European Medicines Agency and orphan drug designation by the US Food and Drug Administration.

Trophos is a clinical stage pharmaceutical company developing innovative therapeutics. The company has developed a proprietary cholesterol-oxime based chemistry platform.