About Us


Caelum Biosciences, Inc. (“Caelum”) is a clinical-stage biotechnology company developing treatments for rare and life-threatening diseases. Caelum’s lead asset, CAEL-101 (mAb 11-1F4), is a novel antibody licensed from Columbia University in January 2017 for the treatment of amyloid light chain (“AL”) amyloidosis, a rare systemic disease that can lead to vital organ failure and death. Phase 1a/1b data presented at the American Society of Hematology’s 59th Annual Meeting in December 2017, the 16th International Symposium on Amyloidosis in March 2018, the American Society of Echocardiography 29th Annual Scientific Sessions in June 2018 and the American Society of Hematology’s 60th Annual Meeting in December 2018 support CAEL-101’s potential to be a safe and well-tolerated therapy that promotes amyloid resolution. The data also demonstrated a correlation between a sustained decrease in N-terminal pro-brain natriuretic peptide (NT-proBNP) levels and an improvement in global longitudinal strain (“GLS”) following CAEL-101 treatment in patients with cardiac AL amyloidosis. New long-term follow-up data from the Phase 1a/1b study were presented at the virtual International Symposium on Amyloidosis (ISA) in September 2020. These data demonstrate 78 percent survival (15/19) at a median follow-up of more than three years (37 months) in AL amyloidosis patients treated with CAEL-101 as well as durable organ response among evaluable patients, further supporting the advancement of CAEL-101 into Phase 3 development.

CAEL-101 has received Orphan Drug Designation from both the U.S. Food and Drug Administration and European Medicine Agency as a therapy for patients with AL amyloidosis.

In January 2019, Caelum signed a collaboration agreement with Alexion Pharmaceuticals, Inc. (“Alexion”) to advance the development of CAEL-101.  Under the terms of the agreement, Alexion acquired a minority equity interest in Caelum and an exclusive option to acquire the remaining equity in the company based on Phase 3 data for pre-negotiated economics. Alexion will make payments to Caelum totaling $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, regulatory and commercial milestone payments, in the event Alexion exercises the acquisition option.

Caelum and Alexion’s Phase 2 open-label dose escalation study was conducted to investigate higher doses of CAEL-101 than had been evaluated in Phase 1 studies with a primary objective to identify the best dose to advance into Phase 3 development. The study evaluated the safety and tolerability of CAEL-101 in 13 AL amyloidosis patients at three study sites who received up to 1000 mg/m2 of CAEL-101 (two times the Phase 1 dose) administered in combination with SoC treatment. The study met its primary objectives, supporting the safety and tolerability of CAEL-101 and the selection of the 1000 mg/m2 dose for the Phase 3 study.

In September 2020, Caelum and Alexion announced the initiation of the Cardiac Amyloid Reaching for Extended Survival (CARES) Phase 3 clinical program to evaluate CAEL-101, a first-in-class amyloid fibril targeted therapy, in combination with standard-of-care (SoC) therapy in AL amyloidosis. The CARES clinical program includes two parallel Phase 3 studies – one in patients with Mayo stage IIIa disease and one in patients with Mayo stage IIIb disease – and will collectively enroll approximately 370 patients globally. Enrollment is underway in both studies. The primary objective of the clinical program is to assess overall survival.

Have you recently been diagnosed with
AL Amyloidosis?