ALEXION & CAELUM DEBUT CAEL-101 FOR AL AMYLOIDOSIS.

 
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Alexion Pharmaceuticals, Inc. and Caelum Biosciences have collaborated to develop 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 to the heart and kidneys.

With a median survival time of less than 18 months following diagnosis and no approved therapies to address the organ damage caused by AL amyloidosis, there is a significant need for new treatments for this devastating disease, said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion. We believe CAEL-101 holds significant promise in being able to help these patients and we're excited to add it to our growing clinical-stage rare hematology portfolio.

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.

About CAEL-101

CAEL-101 is a first-in-class monoclonal antibody (mAb) designed to improve organ function by reducing or eliminating amyloid deposits in the tissues and organs of patients with AL amyloidosis. The antibody is designed to bind to insoluble light chain amyloid protein, including both kappa and lambda subtypes. In a Phase 1a/1b study, CAEL-101 demonstrated improved organ function, including cardiac and renal function, in 27 patients with relapsed and refractory AL amyloidosis who had previously not...

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