Alexion Pharmaceuticals Inc. and New Jersey-based biotech Caelum Biosciences said they’ve begun two late-stage human trials on a drug to treat the rare disease AL amyloidosis.
The disease is caused by an abnormality in plasma cells in the bone marrow. As a result, misfolded amyloid proteins build up in organs throughout the body, impairing their function. When it affects the heart, the median survival is less than one year following diagnosis, officials said.
“While current treatments address the bone marrow disorder that creates the misfolded amyloid proteins, there are no approved therapies for the significant organ damage the disease causes,” Alexion’s research and development head, John Orloff, MD, said in a statement.
Caelum’s drug, a monoclonal antibody called CAEL-101, has the potential to be the first treatment to target and remove the amyloid deposits from the organs, Orloff added.
“Data from Phase 1 studies suggest this treatment approach may improve organ function and long-term survival,” he said.
The companies will conduct two parallel Phase 3 studies at 70 sites around the globe that will collectively include 370 patients — 260 with stage 3a disease and 110 with stage 3b disease.
Enrollment is underway in both studies and the primary objective is to assess overall survival, Alexion said.
The drug will be tested in combination with standard therapy.
Alexion, which is based in Boston but has a large research presence in New Haven, acquired a minority stake in Caelum in January 2019. It has an option to buy the remaining equity in the company based on the outcome of the trials.
Contact Natalie Missakian at news@newhavenbiz.com
