Caelum Biosciences and Alexion Pharmaceuticals Acceptance of Abstracts in Separates Studies
Collaborating firms, Caelum Biosciences and Alexion Pharmaceuticals, Inc., announced that two abstracts on CAEL-101, a "first-in-class amyloid fibril targeted therapy," has been accepted for presentation at the 62nd American Society of Hematology or ASH Annual Meeting and Exposition, happening virtually from December 5 to 8, 2020.
New Cleveland Clinic data will be presented specifically on the safety, efficacy, and tolerability of CAEL-101, combined with standard-of-care or SoC therapy in AL amyloidosis from Phase 2 open-label dose-escalation study, suggesting organ response's early evidence.
Furthermore, Caelum data, demonstrating further that "safety and tolerability of CAEL-101 and support the selection of 1000 mg/m2 dose" for the study's Phase 3 will also be part of the presentation.
As announced previously, CARES or the Cardiac Amyloid Reaching for Extended Survival Phase 3, a clinical program evaluating CAEL-101, combined with the SoC therapy in AL amyloidosis has started.
Also, according to the companies, enrollment is currently ongoing in two parallel Phase 3 studies. One study is intended for patients with "Mayo stage IIIa disease," and another in Mayo stage IIIb disease, and will collectively sign up an estimate of 370 patients worldwide.
The Phase 2 study continues with the added study arm to investigate CAEL-101 combined with SOc treatment and daratumumab.
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Alexion’s six other abstracts which include new data from Phase 3 clinical trial extension research representing the efficacy and safety of ULTOMIRIS® for PNH in older patients.
Six More Abstracts To Be Presented
Also announced was the acceptance of Alexion's six other abstracts which include new data from Phase 3 clinical trial extension research representing the efficacy and safety of ULTOMIRIS® or
"ravulizumab-cwvz" for PNH or paroxysmal nocturnal hemoglobinuria treatment in older patients whether or not, they have a history of bone marrow disorder, as well as those receiving associated use of immunosuppressive therapy."
Accepted data comprise a new analysis from to two Phase 3 extension studies, demonstrating similar safety and consistent, as well as durable effectiveness of ULTOMIRIS® over 52 weeks when used to treat adults aged over 65 years old and have PNH, compared to adults who are 65 years and below.
In addition, new data is showing the efficacy and safety of ULTOMIRIS® when concomitantly used with IST or immunosuppressive therapy in patients who have PNH.
Essentially, a retroactive assessment from the said Phase 3 extension study will exhibit the majority of adult patients who have PNH and aplastic anemia who were given treatment with ULTOMIRIS®, avoided the need for a transfusion for a maximum of a 52-week period, supporting ULTOMIRIS® use in patients with PNH, regardless of their history of bone marrow disorder, who have not been given prior treatment with a complement inhibitor.
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A Leader in Discovering Life-Changing Treatments
Alexion Pharmaceuticals is a global biopharmaceutical firm with a focus on serving patients and families impacted by rare illnesses and distressing conditions by discovering, developing, and commercializing life-changing treatments.
As a leader in rare conditions for over 25 years now, the pharmaceutical firm has devised and commercialized two approved "complement inhibitors" to treat patients who have PNH and AHUS or atypical hemolytic uremic syndrome.
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Nov 05, 2020 07:00 AM EST
