Caelum Biosciences, a company specializing in rare-disease therapies and a subsidiary of Fortress Biotech, a biopharmaceutical company acquiring, developing, and commercializing pharmaceutical and biotechnology products, has entered a biopharmaceutical manufacturing agreement with Patheon for process development and cGMP production of Caelum’s lead therapy, CAEL‐101. 

The agreement will support Phase II/III studies of CAEL‐101 for the treatment of amyloid light chain (AL) amyloidosis, a rare systemic disorder that leads to the buildup of amyloid proteins in and around tissues, nerves, and organs, resulting in organ damage and high mortality rates. CAEL‐101 is currently being evaluated by study sponsor Columbia University in a Phase Ib study in AL amyloidosis.

CAEL‐101 has received orphan-drug designation from the US Food and Drug Administration as a therapeutic agent for patients with AL amyloidosis, and as a radio‐imaging agent in amyloidosis.

Source: Fortress Biotech