Alexion Pharmaceuticals, which has a significant research presence in New Haven, is teaming up with a New Jersey-based drugmaker to do clinical testing on a drug designed to treat a rare disorder involving an abnormality of plasma cells in the bone marrow.
The Phase III testing that researchers from Alexion and Caelum Biosciences will be conducting will examine 370 individuals with varying stages of AL amyloidosis to see how they react to the drug candidate CAEL-101. The rare systemic disorder results in a deposit of misfolded amyloid proteins in tissues and organs and typically kills those suffering from it within a year. The testing will focus on how many of the research subjects are able to survive as a result of taking the drug.
Phase III testing is the final step in the drug development process before the federal Food and Drug Administration considers approving a treatment for sale to the public.
“In AL amyloidosis, misfolded amyloid proteins can build up in many organs throughout the body, including the heart and kidneys, causing significant damage to these organs and impairing their function,” said Dr. John Orloff, executive vice president and head of research and development at Alexion. “CAEL-101 has the potential to be the first treatment to target and remove the amyloid deposits from these organs.”
Michael Spector, president and chief executive officer of Caelum, said long-term survival data from AL amyloidosis patients treated with CAEL-101 in Phase I testing “showed that 78 percent were still alive after a median follow-up time of more than three years.”
Boston-based Alexion specializes in developing drugs that treat rare diseases.