Alexion will acquire the rest of the company if phase II data for anti-amyloid antibody CAEL-101 are positive.
Alexion is focused on the development of drugs to treat rare blood disorders. It has two products on the market approved by the U.S. Food and Drug Administration –– Ultomiris and Soliris –– and is now looking to expand its pipeline.
To that end, Alexion has elected to use the buyout option associated with its collaboration deal with Caelum Biosciences. Caelum was launched in 2017 by startup incubator Fortress Biotech after the latter licensed an anti-amyloid candidate from Columbia University. This candidate was developed based on initial research performed by Alan Solomon at the University of Tennessee Graduate School of Medicine.
The candidate is now referred to as CAEL-101 and is designed to break up pre-existing amyloid deposits in tissues that result in damage to organs, and in particular, the heart and kidneys. According to Caelum CEO Michael Spector, CAEL-101 binds to both kappa and lambda misfolded proteins, which is a unique ability. Phase Ia/Ib study results indicate that the drug is well tolerated and leads to a rapid and clinically relevant organ response, particularly in the heart and kidneys.
As part of the buyout deal, Alexion will pay $60 million for a minority stake in Caelum Biosciences and have an exclusive option to purchase the remainder of the company (for a total deal value of up to $500 million), a decision it will make once phase II data for CAEL-101 are available. With its minority stake, Alexion will be able to have a say in the design of the phase II study.
There are currently no treatments for amyloidosis.