CAEL-101 is a first-in-class amyloid fibril targeted therapy designed to improve organ function by reducing or eliminating amyloid deposits in patients with AL amyloidosis. AL amyloidosis is a rare systemic disorder that causes misfolded immunoglobulin light chain protein to build up in and around tissues, resulting in progressive and widespread organ damage, most commonly the heart and kidneys. Founded by Fortress, Caelum is a company focused on developing treatments for rare and life-threatening diseases.

Under the terms of the agreement, Alexion will acquire a minority equity interest in Caelum and an exclusive option to acquire the remaining equity in the company based on Phase 2 data for pre-negotiated economics.  Alexion will make payments to Caelum totaling up to $60 million, including the purchase price for the equity and milestone-dependent development funding payments. The collaboration also provides for potential additional payments of up to $500 million, including the upfront and regulatory and commercial milestone payments, in the event Alexion exercises the acquisition option. 

 

The collaboration will leverage Alexion’s expertise in rare disease antibody development and commercial franchise in hematology. Alexion and Caelum will collaborate on the design of the ongoing development program for CAEL-101. Caelum will be responsible for conducting the development program through the end of Phase 2 and for manufacturing CAEL-101.
 

Lindsay A. Rosenwald, M.D., Fortress’ chairman, president and chief executive officer, said, “This collaboration with Alexion, a global leader in the rare disease field, is a testament to the Fortress team’s expertise in identifying and developing promising treatments to their full potential. Fortress founded Caelum in January 2017 on the potential of CAEL-101, with the goal of improving treatment options for patients with AL amyloidosis. This partnership represents an exciting opportunity for CAEL-101 to help patients, as well as create value for Fortress investors. We look forward to seeing what Caelum and Alexion will achieve through this collaboration while we continue to develop a robust pipeline of compelling product candidates at Fortress.”