On May 12, 2021 Caelum Biosciences and Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) reported that two abstracts on CAEL-101, a potentially first-in-class amyloid fibril targeted therapy, have been accepted for e-poster presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress 2021, taking place virtually from June 9 to 17, 2021 (Press release, Alexion, MAY 12, 2021, View Source [SID1234579765]). Both presentations will include new data from the Phase 2 open-label dose escalation study evaluating the safety and tolerability of CAEL-101 in combination with standard-of-care (SoC) therapy in AL amyloidosis. One will feature data studying safety and tolerability in AL amyloidosis patients treated with 1000 mg/m2 CAEL-101 in combination with cyclophosphamide-bortezomib-dexamethasone plus daratumumab. The second will include new data further supporting the selection of the 1000 mg/m2 dose for the ongoing Phase 3 study and demonstrating the possible stabilization of cardiac disease as assessed by Mayo staging.

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The abstracts accepted for e-poster presentations are listed below and are now available on the EHA (Free EHA Whitepaper) website. All e-poster presentations will be made available on the virtual congress platform on June 11, 2021 at 09:00 a.m. CEST (3:00 a.m. EDT) and will be available throughout the duration of the Congress.

Safety and Tolerability of CAEL-101 in Combination with Cyclophosphamide-Bortezomib-Dexamethasone and Daratumumab in Patients with AL Amyloidosis. Abstract #EP1017– e-poster presentation; session title: Myeloma and other monoclonal gammopathies – Clinical.

Safety and Tolerability of CAEL-101 in Patients with AL Amyloidosis in a Phase 2 Study for a Median of 38 Weeks. Abstract #EP1018 – e-poster presentation; session title: Myeloma and other monoclonal gammopathies – Clinical.

As was previously announced, the Cardiac Amyloid Reaching for Extended Survival (CARES) Phase 3 clinical program to evaluate CAEL-101 in combination with SoC therapy in AL amyloidosis has begun. Enrollment is underway in two parallel Phase 3 studies – one in patients with Mayo stage IIIa disease (ClinicalTrials.gov Identifier: NCT04512235) and one in patients with Mayo stage IIIb disease (ClinicalTrials.gov Identifier: NCT04504825) – and will collectively enroll approximately 370 patients globally. The Phase 2 program continues with the ongoing evaluation of CAEL-101 in combination with SoC therapy plus daratumumab.

About CAEL-101

CAEL-101 is a first-in-class monoclonal antibody (mAb) designed to improve organ function by reducing or eliminating amyloid deposits in the tissues and organs of patients with AL amyloidosis. The antibody is designed to bind to misfolded light chain protein and amyloid and shows binding to both kappa and lambda subtypes. 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 both the U.S. Food and Drug Administration and European Medicine Agency as a potential therapy for patients with AL amyloidosis.

About AL Amyloidosis

AL amyloidosis is a rare systemic disorder caused by an abnormality of plasma cells in the bone marrow. Misfolded immunoglobulin light chains produced by plasma cells aggregate and form fibrils that deposit in tissues and organs. This deposition can cause widespread and progressive organ damage and high mortality rates, with death most frequently occurring as a result of cardiac failure. 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.

AL amyloidosis is a rare disease but is the most common form of systemic amyloidosis. There are approximately 22,000 patients across the United States, France, Germany, Italy, Spain and the United Kingdom. AL amyloidosis has a one-year mortality rate of 47 percent, 76 percent of which is caused by cardiac amyloidosis.