Acceleron Pharma's most advanced drug in development may be aiming at the same disease as its Cambridge neighbor, bluebird bio, but CEO John Knopf says there's room in the market for the two very different approaches to the same rare disease.
In today's Boston Business Journal, I wrote a profile of Knopf where I focused on how he came to head the Central Square biotech that went public last year. The next few weeks will be important for Knopf and Acceleron (Nasdaq: XLRN) as both it and bluebird (Nasdaq: BLUE) present head-to-head data at the American Society of Hematology conference Dec. 6-9 in San Francisco. Both companies are targeting beta-thalassemia, an inherited blood disorder in which patients don't have enough red blood cells, albeit their treatments are very different.
Bluebird has been focusing on a gene therapy treatment, which involves transplanting red blood cells with new DNA. Acceleron's approach is a biologic agent called luspatercept, which increases red blood cell count by blocking a type of protein. There are no treatments for the disease, which is present in "thousands" of infants born every year around the world, according to the National Institutes of Health. Knopf said that while some patients may not respond to luspatercept, others will not be eligible for blood transfusions, meaning there's room for both Acceleron and bluebird to succeed.
In Acceleron's case, the data to be given next month will be initial results from a midstage trial of up to 50 patients being conducted in partnership with Celgene. The reason it's important is this drug will begin a Phase 3 trial next year in patients with the same disease — the first late-stage trial for the 10-year-old company, and one which will form the basis of an application for approval.
Luspatercept is also being tested in patients with myelodysplastic syndromes, a bone-marrow disorder. Acceleron has three other drugs in midstage trials aimed at a variety of diseases, including cancer, which are based on the same approach. That approach is based on drugs that block proteins in a group known as transforming growth factor beta protein superfamily. That's the initial focus Knopf had when he began the company in 2004, a few years after after the biotech bubble had risen and waned and a time when he said biotech firms had to be "a little more rigorous" with their science.
Acceleron was very much a company which first came up with a drug, and only later found a disease which it could be used against, said Knopf.
"It was really emerging around that time that there was this family of proteins emerging that had these pretty remarkable activities," he said in a recent interview.
Animal tests confirmed that another of Acceleron's early drugs, sotatercept, increased red blood cell count. Then the company found two kinds of anemia which correspond to the way that drug makes cells. Since Celgene (Nasdaq: CELG) was already a leader in developing drugs for MDS, the New Jersey company ended up being an ideal partner for both sotatercept and luspatercept. Of the two, luspatercept is the only one which the company has slated for a Phase 3 trial.
"It turned out by doing the science ... we appreciated that that was a disease ideally suited for our particular product," Knopf said.
Link:
http://www.bizjournals.com/boston/blog/bioflash/2014/11/acceleron-drug-for-rare-blood-disorder-slated-for.html?page=all