Ipsen and Pharnext in €91M tie-up in neuromuscular diseases

Licensing

Drug repositioning specialist Pharnext has outlicensed its lead programme to Ipsen SA in a deal with a headline value of at least €91 million. Ipsen is taking up convertible bonds and will pay Pharnext €91 million in milestones plus royalties on sales, if it decides to take the programme, currently in preclinical development, forward at the end of Phase II.

The programme consists of selected combinations of over 200 off-patent, approved compounds thrown up by Pharnext’s systems biology in silico screening technology. These drugs have been shown to have an effect in preclinical models of Charcot-Marie-Tooth, a chronic neuromuscular disorder.

Charcot-Marie-Tooth is classified as an Orphan Disease with 30,000 sufferers in France and three million worldwide. Currently there is no treatment. More common peripheral neuropathies, such as that caused by diabetes, affect over 30 million people worldwide and may be amenable to the compounds uncovered in Pharnext’s screen.

Pharnext is applying advances in systems biology to find new uses for approved drugs. Understanding of the biological networks involved in particular diseases states is used to identify existing drugs that act on one or more proteins that are involved in the disease process.

The company was co-founded in June 2007 by Daniel Cohen and Philippe Pouletty, two leaders of the French biotech sector. Cohen has taken a lead in applying genomics to drug discovery, while Pouletty, France’s leading biotechnology entrepreneur, is General Partner at the venture capital firm Truffle Partners and Chairman of the national industry association, France Biotech.

Truffle invested €3.5 million in the formation of Pharnext. The Paris-based company has also received €3.4 million from the French Innovation Agency, OSEO.

Having identified 200 compounds that act on proteins involved in peripheral neuropathies, and pinpointed some combinations that are active in models of Charcot-Marie-Tooth, Pharnext is widening its search to find drugs that are active in aspects of Alzheimer’s disease, Amyotrophic lateral sclerosis, multiple sclerosis, schizophrenia and inherited disorders including cystic fibrosis, Duchenne muscular dystrophy and Down’s syndrome.

Pharnext first accumulates genetic data relating to a particular disease, using this to identify proteins involved in the disease state, and pick out approved drugs that act on any of these proteins. The drugs are then screened alone, and in combination, to generate preclinical candidates.

Pharnext says this approach to discovery is twice as fast and generates more effective compounds, than traditional methods.