everyone getting asked to dance but avi: September 9, 2010 08:00 AME-mail| |Comments (0)| Text size – + Globe Staff
Acceleron Pharma Inc. of Cambridge and British drug maker Shire plc announced a joint venture to develop muscle therapeutics for so-called orphan diseases.
If all objectives of the venture are met, the deal could be worth as much as $498 million to Acceleron, the two companies said in a press release; Shire will make an upfront cash payment to Acceleron of $45 million. Shire has a presence in Lexington.
Shire and Acceleron will share development costs in North America and Europe, and Shire will be responsible for development costs in other markets, the release said.
The collaboration will initially focus on ACE-031, an Acceleron drug candidate that is currently in a Phase 2a trial for the treatment of patients with Duchenne Muscular Dystrophy. Duchenne Muscular Dystrophy is a fatal orphan muscle disease with no currently approved treatment, the release noted.
"This collaboration is an excellent strategic fit to the work that Shire is already doing on behalf of patients with rare diseases," Sylvie Gregoire, president of Shire Human Genetic Therapies, said in a statement.
This agreement seemingly extends Shire's interest in drugs for rare diseases. Shire now has a drug for Gaucher disease that competes with one of the most important drugs of Genzyme Corp., the Cambridge biotechnology company.
Acceleron's drug is a muscle builder - so you'd have to have some muscle to build up on, I suppose. Thus it would be a drug that could be given after our drug starts to build up some new muscle in a DMD kid. In other words it's complimentary to our drug. A good choice for Shire. (I mean AVI's drug plus Acceleron's)
Not exactly. ACE-031 is supposed to create new muscles (muscle growth hence increasing strength), inhibits muscle downregulators. In post-menopausal women it increase the lean muscle mass and reduced fat (so hopefully fibrosis), it also increase the bone mass (another problem in DMD). It is now tested in ambulatory DMD boys. They also noticed , in mice, that it upregulates utrophin so the new muscles did not look like "DMD muscles". It remains to be seen how it translates to humans. I do think that the big thing is that dystrophin still does not exist to protect the newly created muscles, so unless the utrophin is upregulated significantly it might not work as well. However, a combination of exon skipping and ACE-031 should provide better results than each drug alone. In theory at least...we don't know if any or both will work in DMD patients.
One very important point here is that ACE-031 can be used for many diseases: muscular dystrophies, ALS, muscles loss due to cancer etc. So the market is much bigger than for exon skipping.