Pharma Focus Asia

Alexion and Caelum Biosciences Announce Collaboration to Develop Targeted Therapy for Light Chain (AL) Amyloidosis

Friday, February 01, 2019

Alexion Pharmaceuticals, Inc. and Caelum Biosciences today announced a collaboration 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.”

Michael Spector, President and Chief Executive Officer of Caelum, said, “CAEL-101 appears to have a unique capability of binding to both kappa and lambda misfolded proteins. Data from the Phase 1a/1b study indicate that CAEL-101 is a well-tolerated therapy that leads to a rapid and clinically relevant organ response, particularly in the heart and kidneys. Further, CAEL-101 showed a statistically significant improvement from baseline in global longitudinal strain, an endpoint that has been correlated with survival in patients with AL amyloidosis. We are very pleased to collaborate with Alexion, a global leader in the rare disease field.”

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.

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