Roche said today it will acquire Trophos for up to €470 million (about $543 million), in a deal that will expand the pharma giant’s portfolio in neuromuscular disease with high medical need, anchored by the Phase II/III-completed spinal muscular atrophy (SMA) compound olesoxime (TRO19622).

Olesoxime showed statistically significant positive results last year in data from a pivotal Phase II/III study presented in April 2014 at the annual meeting of the American Academy of Neurology. The study assessed Olesoxime in Type II and non-ambulatory Type III SMA patients ages 3-25.

Study results showed the drug candidate preserved motor function for two years using the Motor Function Measure scale (MFM) D1+D2 as the primary endpoint. Patients dosed with olesoxime fared better than those taking placebo, who experienced a loss of motor function starting from a mean score of 39% at baseline to 37.1% after two years.

Olesoxime also generated positive results on the pivotal trial’s secondary endpoint of motor function as measured by the Hammersmith Functional Motor Scale (HFMS). The percentage of patients who maintained motor function during the trial was 48% of those taking the experimental drug, compared with 28% taking placebo.

Developed by Trophos in-house through its own screening platform, olesoxime is the lead product in the company’s cholesterol-oxime family of compounds, designed to target and preserve mitochondrial integrity and function in stressed cells. Olesoxime has been granted the FDA’s orphan drug designation, as well as the ‘Orphan Medicinal Product’ designation for the treatment of SMA by the European Medicines Agency.

“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,” Sandra Horning, M.D., Roche’s CMO and head of global product development, said in a statement.

Olesoxime is also in Phase II development as a potential add-on therapy to Interferon beta for multiple sclerosis (MS), funded through a grant from the French National Research Agency (ANR).

The compound was initially developed as a treatment against amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease). A Phase III trial in 512 ALS patients failed in 2011 as olesoxime didn't show a significant increase in survival versus placebo in patients receiving riluzole (Rilutek®). However, that trial also showed improvement in patients' function as measured by the ALSFRS-R functional rating scale.

Following that setback, Actelion said it would not exercise its exclusive option to acquire Trophos for up to €195 million ($225 million). Actelion shelled out €10 million ($11.6 million) for that option in 2010, as part of an agreement that also launched a research collaboration giving Actelion access to Trophos’ CNS assay technology and compound library.

Roche agreed to pay €120 million (about $139 million) to shareholders of privately held Trophos, plus up to €350 million ($404 million) in payments based on achieving unspecified milestones.

In addition to olesoxime, Trophos’ pipeline includes TRO40303, another in-house-discovered compound that is in Phase I/Ib development for cardiac ischemia-reperfusion injury. An unnamed compound is in preclinical development for “neurodegenerative diseases,” Trophos states on its website.

Founded in 1999, Trophos is based in Marseille, France, and has been funded by the French Muscular Dystrophy Association (AFM) and a syndicate of private equity funds including ACG Management, OTC Agregator, Amundi Private Equity Funds, Turenne Capital, Sofipaca, and Vesale Partners.

The planned Trophos acquisition is Roche’s fourth deal announced this week. The pharma giant acquired a majority stake in Foundation Medicine for $1.18 billion, and obtained rights from Meiji Seika Pharma and Fedora Pharmaceuticals to develop OP0595, a Phase I infectious-disease compound, for up-to-$750 million.

In addition, Roche’s Genentech subsidiary agreed to furnish tens of thousands of de-identified samples for whole genome sequencing and analysis to Human Longevity, Inc., the genomics and cell therapy-based diagnostic and therapeutic company whose CEO and co-founder is J. Craig Venter, Ph.D. The value of that multi-year deal was undisclosed.

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