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Deal-happy Roche acquires Trophos

This article was originally published in Scrip

In yet another deal, Roche is acquiring rare disease drug developer Trophos at a price of €120m upfront, and a further €350m in potential milestone payments.

Early this week Roche paid $1.2bn for a stake in molecular information and genomic analysis firm Foundation Medicine; signed a licensing deal for a Phase I antibiotic boosting drug from Meiji Seika Pharma and Fedora Pharmaceuticals for up to $750m; and partnered with Genome pioneer J Craig Venter.

In purchasing the privately held Trophos, Roche will gain the French firm's lead compound olesoxime (TRO19622), which is in Phase II development for spinal muscular atrophy (SMA) – a rare genetic neuromuscular disease most commonly diagnosed in children. Under terms of the €120m deal Trophos shareholders will also be eligible for up to €350m in milestone payments related to the products progress.

"This is a tremendous recognition of the work done by Trophos's teams and supporters over the past 16 years," Trophos CEO Christine Placet said in a statement.

Trophos has seen success for olesoxime in SMA. It reported positive data from its pivotal Phase II/III study in SMA patients in April 2014. The cholesterol-like compound that targets and preserves mitochondrial integrity and function in stressed cells, was shown to have 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. Olesoxime has been granted orphan drug designation by both the FDA and the European Medicines Agency.

However olesoxime has struggled previously in trials for other indications. In December 2011, the drug failed in a Phase III study in amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease). As a result of this failure, partnering company at the time, Actelion, did not exercise its option to acquire Trophos (scripintelligence.com, 13 December 2011).

Development of the compound has also been suspended in neuropathic pain, non-alcohol steatohepatitis (NASH), and diabetic peripheral neuropathy. But the drug is still in Phase I/II trials as a treatment for multiple sclerosis (MS), an area where Roche is already present. The big pharma has its own drug, ocrelizumab, in Phase III trials for primary progressive MS and relapsing MS.

Other developers of SMA therapies include Isis Pharmaceuticals with ISIS-SMNRx, currently in Phase II; and Novartis with BVS-857, in Phase I. Roche also has a Phase I product, RD7800, in development for SMA – partnered with PTC Therapeutics and the SMA Foundation.

Related stories:

13 January 2015: JP MORGAN 2015: Roche in dealmaking mode for antibiotics, diagnostics

12 January 2015: Pharma trial needs drive Roche's $1.2bn Foundation Medicine deal

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