Rare Daily Staff

Alexion Pharmaceuticals and Caelum Biosciences have entered into a potential $550 million collaboration to develop Caelum’s CAEL-101, a first-in-class amyloid fibril targeted therapy, for light chain amyloidosis, a rare systemic disorder.

AL amyloidosis 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.

CAEL-101 is designed to improve organ function by reducing or eliminating amyloid deposits in patients with AL amyloidosis. Current standard of care includes plasma cell directed chemotherapy and autologous stem cell transplant, but these therapies do not address the organ dysfunction caused by amyloid deposition, and up to 80 percent of patients are ineligible for transplant.

“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,” says John Orloff, executive vice president and head of research and 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.”

The deal provides an opportunity to diversify Alexion’s clinical stage rare hematology portfolio and leverages Alexion’s expertise in rare disease antibody development and commercial franchise in hematology.

As part 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 pay Caelum 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.

Alexion and Caelum will collaborate on the design of the ongoing development program for CAEL-101 with Caelum responsible for conducting the development program through the end of phase 2 and for manufacturing CAEL-101.

“CAEL-101 appears to have a unique capability of binding to both kappa and lambda misfolded proteins,” says Michael Spector, president and CEO of Caelum.

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 had an organ response to standard of care therapy.

CAEL-101 has received Orphan Drug Designation from the U.S. Food and Drug Administration as a therapy for patients with AL amyloidosis and as a radio-imaging agent in AL amyloidosis.

 

February 4, 2019
Photo: Michael Spector, president and CEO of Caelum