Overview

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.  CAEL-101 has received Orphan Drug Designation from the U.S. Food and Drug Administration as a therapeutic agent for patients with AL amyloidosis, and as a radio-imaging agent in 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 2 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 is planning  a Phase 2 clinical program for CAEL-101.