{"content":[{"text":"Safety and Tolerability of CAEL-101 in Patients with AL Amyloidosis in a Phase 2 Study for a Median of 49 \n Weeks (#EP1018)","bbox":{"left":0.081765,"top":0.054194,"width":0.8366410000000001,"height":0.034769}},{"text":"Additional longer-term data presented from the Phase 2 study demonstrated that CAEL-101 in combination with \n CyBorD in patients with AL amyloidosis (N=13) was generally well tolerated up to a median treatment duration \n of 49 weeks (range 12-57 weeks), with most patients having received more than 20 infusions of CAEL-101. The \n most common AEs reported were diarrhea, nausea, fatigue, rash, and anemia. In addition, exploratory clinical \n biomarker evaluations showed early signals suggesting possible cardiac and renal response. Specifically, \n median percent changes for biomarkers of cardiac disease (cTnT and NT-proBNP) were lower at each \n subsequent time point measured, suggesting improvement in cardiac function among eight patients with active \n cardiac disease at baseline. Additionally, seven patients with active renal impairment at baseline demonstrated \n renal response, as defined by a decrease of at least 30 percent in proteinuria (an excess of protein in the urine) \n following treatment.","bbox":{"left":0.081765,"top":0.110886,"width":0.8369970000000001,"height":0.18599300000000002}},{"text":"“We are grateful to clinical trial participants who are essential to advancing our work towards new treatment \n options for AL amyloidosis,” said John Orloff, M.D., Executive Vice President and Head of Research and \n Development at Alexion. “We remain committed to working together with the AL amyloidosis community and \n Caelum to evaluate the potential of CAEL-101 as a potentially first-in-class treatment option for patients who are \n living with this devastating disease.”","bbox":{"left":0.081765,"top":0.318803,"width":0.836832,"height":0.09140999999999999}},{"text":"As was previously announced, the Cardiac Amyloid Reaching for Extended Survival (CARES) Phase 3 clinical \n program to evaluate CAEL-101 in combination with SoC therapy in AL amyloidosis has begun. Enrollment is \n underway in two parallel Phase 3 studies – one in patients with Mayo stage IIIa disease (ClinicalTrials.gov \n Identifier: NCT04512235) and one in patients with Mayo stage IIIb disease (ClinicalTrials.gov Identifier: \n NCT04504825) – and will collectively enroll approximately 370 patients globally.","bbox":{"left":0.081765,"top":0.432288,"width":0.8367330000000001,"height":0.09143500000000004}},{"text":"About the CAEL-101 Phase 2 Study \n The Phase 2 multicenter, open-label, dose-selection study (ClinicalTrials.gov Identifier: NCT04304144) is \n designed to evaluate the safety and tolerability of CAEL-101 in combination with standard of care (SoC) therapy \n for patients with AL amyloidosis and determine the recommended dose for Phase 3 studies. The study is divided \n into two parts: Part A examined CAEL-101 in combination with cyclophosphamide-bortezomib-dexamethasone \n (CyBorD) and employed a 3+3 dose escalation design (cohort 1 – 500 mg/m2; cohort 2 – 750 mg/m2 ; cohort \n 3 1000 mg/m2); Part A patients were subsequently up titrated to 1000mg/m2, once this was identified as the \n Phase 3 dose. Part B is examining CAEL-101 at the 1000 mg/m2 dose in combination with CyBorD plus \n daratumumab. Patients from Parts A and B receive CAEL-101 therapy weekly for the four-week observation \n period followed by CAEL-101 doses every other week thereafter, all while continuing to receive SoC therapy. \n Patients continue to receive CAEL-101 per protocol until the end of the study or discontinuation.","bbox":{"left":0.081765,"top":0.544131,"width":0.836994,"height":0.207812}},{"text":"About CAEL-101 \n CAEL-101 is a first-in-class monoclonal antibody (mAb) designed to improve organ function by reducing or \n eliminating amyloid deposits in the tissues and organs of patients with AL amyloidosis. The antibody is designed \n to bind to misfolded light chain proteins and amyloid and shows binding to both kappa and lambda subtypes. In \n a Phase 1a/1b study, CAEL-101 demonstrated improved organ function, including cardiac and renal function, in \n 27 patients with relapsed and refractory AL amyloidosis who had previously not had an organ response to \n standard of care therapy. CAEL-101 has received Orphan Drug Designation from both the U.S. Food and Drug \n Administration and European Medicine Agency as a potential therapy for patients with AL amyloidosis.","bbox":{"left":0.081765,"top":0.774017,"width":0.836856,"height":0.14810200000000007}},{"text":"2","bbox":{"left":0.494788,"top":0.948437,"width":0.010579000000000005,"height":0.01580400000000004}}],"images_bbox":[],"images_bbox_not_text_overlap":[]}