CSL889 hemopexin / CSL Behring - LARVOL DELTA

Safety, Efficacy, and Pharmacokinetics of CSL889 in Adults and Adolescents With Sickle Cell Disease During Vaso-Occlusive Crisis (clinicaltrials.gov) - P2/3 | N=260 | Recruiting | Sponsor: CSL Behring | Not yet recruiting ➔ Recruiting

Enrollment open • Genetic Disorders • Hematological Disorders • Sickle Cell Disease

Safety, Efficacy, and Pharmacokinetics of CSL889 in Adults and Adolescents With Sickle Cell Disease During Vaso-Occlusive Crisis (clinicaltrials.gov) - P2/3 | N=260 | Not yet recruiting | Sponsor: CSL Behring

New P2/3 trial • Genetic Disorders • Hematological Disorders • Sickle Cell Disease

PHASE 1 STUDY OF SAFETY AND PHARMACOKINETICS OF CSL889 (HEMOPEXIN) IN ADULTS WITH SCD (EHA 2024) - P1 | "Half of the subjects in Part A and all in Part B reported concomitanthydroxyurea use. In Part B, 3 subjects were receiving voxelotor and/or crizanlizumab... CSL889 had an excellent safety and tolerability profile when administered as a single dose up to 200 mg/kg insubjects with stable SCD and at 60 mg/kg in subjects with VOC. The observed half-life may support dosingonce daily or every other day. These results provide a strong foundation for future trials to evaluate potentialefficacy."

Clinical • P1 data • PK/PD data • Anemia • Genetic Disorders • Hematological Disorders • Infectious Disease • Novel Coronavirus Disease • Pain • Sickle Cell Disease • HPX

Safety of Single Doses of CSL889 in Adult Patients With Sickle Cell Disease (clinicaltrials.gov) - P1 | N=28 | Completed | Sponsor: CSL Behring | Recruiting ➔ Completed

Trial completion • Genetic Disorders • Hematological Disorders • Sickle Cell Disease

Safety of Single Doses of CSL889 in Adult Patients With Sickle Cell Disease (clinicaltrials.gov) - P1 | N=32 | Recruiting | Sponsor: CSL Behring | Trial completion date: Mar 2023 ➔ Jul 2023 | Trial primary completion date: Mar 2023 ➔ Jul 2023

Trial completion date • Trial primary completion date • Genetic Disorders • Hematological Disorders • Sickle Cell Disease

Safety of Single Doses of CSL889 in Adult Patients With Sickle Cell Disease (clinicaltrials.gov) - P1 | N=32 | Recruiting | Sponsor: CSL Behring | Trial completion date: Jul 2022 ➔ Mar 2023 | Trial primary completion date: Jul 2022 ➔ Mar 2023

Trial completion date • Trial primary completion date • Genetic Disorders • Hematological Disorders • Sickle Cell Disease

Safety of Single Doses of CSL889 in Adult Patients With Sickle Cell Disease (clinicaltrials.gov) - P1; N=32; Recruiting; Sponsor: CSL Behring; N=24 ➔ 32

Clinical • Enrollment change • Genetic Disorders • Hematological Disorders • Sickle Cell Disease

[VIRTUAL] PRECLINICAL SAFETY AND TOXICITY ASSESSMENT OF A HUMAN PLASMA DERIVED HEMOPEXIN DRUG PRODUCT (CSL889) FOR THE TREATMENT OF ACUTE VOC IN SICKLE CELL DISEASE (EHA 2021) - "Similar to the reaction observed in wildtype mice immunological reactions were observed in some dosing groups after repeat administration, which are assessed as a result of the application of the heterologous human protein. Conclusion The toxicological program demonstrated that CSL889 has a favorable safety profile and supports the clinical development of hemopexin for the treatment of acute VOC in sickle cell disease."

Preclinical • Anemia • Cardiovascular • Complement-mediated Rare Disorders • Genetic Disorders • Hematological Disorders • Immunology • Ophthalmology • Sickle Cell Disease • HPX

[VIRTUAL] PHARMACOKINETIC PROPERTIES OF A HUMAN PLASMA DERIVED HEMOPEXIN DRUG PRODUCT (CSL889) IN TOWNES HBSS-, WILD-TYPE MICE, RATS AND CYNOMOLGUS MONKEYS FOR THE TREATMENT OF ACUTE VOC IN SICKLE CELL DISEASE (EHA 2021) - "Following repeated dosing in Townes HbSS mice increasing hemopexin concentration levels correlate with decreasing total heme plasma levels. The increase of total heme towards the end is most probably due to the saturation of the clearance mechanism and ongoing hemolysis."

PK/PD data • Preclinical • Anemia • Complement-mediated Rare Disorders • Genetic Disorders • Hematological Disorders • Sickle Cell Disease • HPX

Safety of Single Ascending Doses of CSL889 in Adult Patients With Stable Sickle Cell Disease (clinicaltrials.gov) - P1; N=24; Recruiting; Sponsor: CSL Behring; Initiation date: Dec 2020 ➔ Mar 2021

Clinical • Trial initiation date • Genetic Disorders • Hematological Disorders • Sickle Cell Disease

CSL889 Designated as Orphan Drug in Europe and US for Treating Sickle Cell Disease (Sickle Cell Anemia News) - "The European Commission and the U.S. Food and Drug Administration have granted orphan drug status to CSL889, an investigational therapy for the treatment of acute vaso-occlusive crises (VOCs) in sickle cell disease (SCD), CSL Behring, the medicine’s maker, announced in a press release."

Orphan drug

Safety of Single Ascending Doses of CSL889 in Adult Patients With Stable Sickle Cell Disease (clinicaltrials.gov) - P1; N=24; Recruiting; Sponsor: CSL Behring; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open • Genetic Disorders • Hematological Disorders • Sickle Cell Disease

Safety of Single Ascending Doses of CSL889 in Adult Patients With Stable Sickle Cell Disease (clinicaltrials.gov) - P1; N=24; Not yet recruiting; Sponsor: CSL Behring; Trial completion date: Mar 2021 ➔ May 2022; Trial primary completion date: Mar 2021 ➔ May 2022

Clinical • Trial completion date • Trial primary completion date • Gene Therapies • Genetic Disorders • Hematological Disorders • Sickle Cell Disease

Safety of Single Ascending Doses of CSL889 in Adult Patients With Stable Sickle Cell Disease (clinicaltrials.gov) - P1; N=24; Not yet recruiting; Sponsor: CSL Behring; Initiation date: Mar 2020 ➔ Sep 2020

Clinical • Trial initiation date

Safety of Single Ascending Doses of CSL889 in Adult Patients With Stable Sickle Cell Disease (clinicaltrials.gov) - P1; N=24; Not yet recruiting; Sponsor: CSL Behring

Clinical • New P1 trial