Roche bets $545M on Trophos' once-failed orphan drug

Roche ($RHHBY) has signed a deal to trade up to €470 million ($545 million) for Trophos, a French company at work on a mid-stage treatment for the rare and debilitating spinal muscular atrophy (SMA).

Under the deal, Roche will hand over €120 million ($139 million) up front with the promise of €350 million ($406 million) more tied to the progress of olesoxime, Trophos' in-development treatment for SMA. In a Phase II study, the drug demonstrated a marked effect on patients with the neuromuscular disease, helping restore normal function and reducing related complications, Roche said.

Now the Swiss drugmaker is taking up the reins with hopes of making olesoxime the first approved drug for SMA, which affects between one in 6,000 to one in 10,000 children around the world, the company said.

Roche's Sandra Horning

"This acquisition highlights Roche's commitment to developing medicines for spinal muscular atrophy, a serious disease with no effective treatment," Chief Medical Officer Sandra Horning said in a statement. "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."

In addition to its lead candidate, Roche is acquiring Trophos' TRO40303, a Phase I treatment for cardiac injury caused by lack of oxygen, and an undisclosed number of preclinical assets. Also of particular interest to Roche is the biotech's proprietary screening platform, which generates mitochondrial targeted compounds and bred olesoxime.

The deal is welcome news for Trophos' investors, who endured a costly disappointment back in 2011 when olesoxime failed a Phase III trial in amyotrophic lateral sclerosis, leading Actelion, which had an option to buy the company, to walk away from a $250 million deal. Trophos then rallied around olesoxime's potential in SMA and is also developing the drug for multiple sclerosis.

For Roche, the agreement is its fourth big splash in less than a week, coming on the heels of its $1.2 billion move to acquire a majority stake in cancer diagnostics pioneer Foundation Medicine ($FMI), its $750 million handshake with Meiji Seika Pharma and Fedora to expand work in antibiotics and its partnership with genomics luminary J. Craig Venter.

- read the statement

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