Fortress Biotech Announces CAEL-101 Strategic Partnership with Alexion Pharmaceuticals

On Jan 31, 2019

Fortress Biotech announced that Caelum Biosciences has signed a strategic agreement with Alexion Pharmaceuticals to advance the development of CAEL-101 for light chain (AL) amyloidosis. 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.”

Fortress has more than 25 programs in development, including seven late-stage and one mid-stage product candidates, at the Fortress level, at its majority-owned and majority-controlled subsidiaries and at entities in which it holds minority ownership positions. In addition to CAEL-101, late-stage and mid-stage programs include:

Intravenous (IV) tramadol for post-surgical acute pain
MB-107 for X-linked severe combined immunodeficiency (X-SCID)
CUTX-101 (Copper Histidinate) for Menkes disease
CK-101, a third-generation irreversible mutant selective EGFR, for frontline small cell lung cancer with EGFR mutations
CK-301, an anti-PD-L1 mAb, for selected recurrent or metastatic cancers
Triplex for cytomegalovirus (CMV)
CEVA101 for pediatric and adult severe traumatic brain injury

Fortress also has seven marketed products, which include Targadox, Exelderm, Cercade, Luxamend, Ala-Quin, Ala-Scalp and Triderm™.