GSK, Angiochem dream of brain delivery for enzyme disorder drugs

Angiochem will work with GlaxoSmithKline ($GSK) on one of the ultimate drug delivery challenges: new treatments that can cross the blood-brain barrier to successfully restore enzyme function in the brain for rare diseases.

The deal could be lucrative. Angiochem stands to make as much as $31.5 million upfront if GSK decides to license multiple drug candidates. With royalties and other expected payments down the line, GSK could fork over more than $300 million, according to Angiochem's announcement touting the deal.

Montreal-based Angiochem, a clinical stage biotech, will help the global pharmaceutical giant develop EPiC-enzyme related treatments that rely on the LRP-1 mediated pathway to penetrate the blood-brain barrier and help enzyme function return to normal in the brain. This kind of treatment would address a number of genetic disorders including Fabry disease, Gaucher's disease and Pompe disease, which can crop up in young children and cause cognitive and physical decline, among many other problems.

A new way to deliver enzyme treatments for these and other lysosomal storage diseases is necessary, because current enzyme replacement therapy--delivered intravenously--only helps manage the disorders and doesn't prevent the worsening neurological and other symptoms. Angiochem and GSK envision a drug that crosses the blood-brain barrier as being able to restore crucial enzyme production in the brain, so it can resume processing of lipids or glycoproteins inside cells.

Rare disease spaces can be immensely profitable (See: Genzyme, and peers.) And as FierceBiotech told you when this story first broke over the weekend, Angiochem has already advanced using its technology to deliver cancer drugs to attack tumors in the brain. It even made $35 million in 2010 after signing over to Geron ($GERN) rights to its Phase II brain cancer drug.

"This collaboration will further validate the wide-ranging potential for our BBB (blood-brain barrier) platform across multiple therapeutic areas," Angiochem President and CEO Jean-Paul Castaigne said in a statement announcing the deal. That's a pretty bold statement, which will take time to prove.

- here's the release