tarperprumig (ALXN1820) / AstraZeneca - LARVOL DELTA

Safety and Efficacy of Tarperprumig in Adult Participants With Anti-Neutrophil Cytoplasmic Antibody (ANCA)-Associated Vasculitis (clinicaltrials.gov) - P2 | N=75 | Recruiting | Sponsor: Alexion Pharmaceuticals, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • ANCA Vasculitis • Vasculitis

Safety and Efficacy of Tarperprumig in Adult Participants With Anti-Neutrophil Cytoplasmic Antibody (ANCA)-Associated Vasculitis (clinicaltrials.gov) - P2 | N=75 | Not yet recruiting | Sponsor: Alexion Pharmaceuticals, Inc.

New P2 trial • ANCA Vasculitis • Vasculitis

Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of ALXN1820 (Tarperprumig) in Healthy Adults: Results of a Phase I Study. (PubMed, Clin Transl Sci) - P1 | "These results support continued investigation of tarperprumig to treat diseases involving complement activation. Trial Registration: EudraCT: 2021-002472-39/NCT04631562."

Clinical • Journal • P1 data • PK/PD data • Infectious Disease

AstraZeneca CEO defends 'fantastic' Alexion buyout in face of $753M hit from dropped drug (FierceBiotech) - "Buying Alexion remains a 'fantastic acquisition' for AstraZeneca, the Big Pharma’s CEO has said, even as the company takes a $753 million hit for abandoning one of the drugs from that acquisition...The drug in question, vemircopan, was once a hot prospect at AstraZeneca...the abandonment of other Alexion drugs in the form of ALXN1840 for Wilson disease and ALXN1820 for sickle cell disease...The drugmaker said....in its fourth-quarter clinical trials update that it stopped the studies because of a lack of efficacy. AstraZeneca used slightly different language when disclosing the broader termination of the program, attributing the decision to phase 2 safety and efficacy data."

Discontinued • Trial status • Genetic Disorders • IgA Nephropathy • Lupus Nephritis • Sickle Cell Disease

Characterization of the bispecific VHH antibody tarperprumig (ALXN1820) specific for properdin and designed for low-volume administration. (PubMed, MAbs) - "Tarperprumig cross-reacts with cynomolgus monkey FP and serum albumin. In summary, tarperprumig exhibits properties tailored for subcutaneous administration and is currently in clinical development for the treatment of complement AP-related disorders."

Journal • Hematological Disorders

PHOENIX: Safety, Efficacy, Pharmacokinetic, and Pharmacodynamic Study of ALXN1820 in Adult Participants With Sickle Cell Disease (clinicaltrials.gov) - P2 | N=2 | Terminated | Sponsor: Alexion Pharmaceuticals, Inc. | Phase classification: P2a ➔ P2 | N=30 ➔ 2 | Trial completion date: Jun 2024 ➔ Jan 2024 | Active, not recruiting ➔ Terminated | Trial primary completion date: Jun 2024 ➔ Jan 2024; Sponsor decision to terminate program.

Enrollment change • Phase classification • Trial completion date • Trial primary completion date • Trial termination • Genetic Disorders • Hematological Disorders • Sickle Cell Disease

PHOENIX: Safety, Efficacy, Pharmacokinetic, and Pharmacodynamic Study of ALXN1820 in Adult Participants With Sickle Cell Disease (clinicaltrials.gov) - P2a | N=30 | Active, not recruiting | Sponsor: Alexion | Recruiting ➔ Active, not recruiting

Enrollment closed • Genetic Disorders • Hematological Disorders • Sickle Cell Disease • HPX

Safety, Efficacy, Pharmacokinetic, and Pharmacodynamic Study of ALXN1820 in Adult Participants With Sickle Cell Disease (clinicaltrials.gov) - P2a | N=30 | Recruiting | Sponsor: Alexion | Not yet recruiting ➔ Recruiting | Trial completion date: Feb 2024 ➔ Jun 2024 | Trial primary completion date: Feb 2024 ➔ Jun 2024

Enrollment open • Trial completion date • Trial primary completion date • Genetic Disorders • Hematological Disorders • Sickle Cell Disease • HPX

A Phase 2a, Randomized, Open-Label Study to Evaluate Multiple Dosing Regimens of Subcutaneous ALXN1820 in Adult Patients with Sickle Cell Disease (ASH 2022) - P1 | "Concomitant treatment with stable dose of hydroxyurea is permitted during the study as applicable. Key exclusion criteria are receiving voxelotor or crizanlizumab within 60 days of providing informed consent and, prior treatment with complement inhibitors within 6 months of first study dose... Complement activation may play an important role in the pathophysiology of SCD. Treatment with an anti-properdin antibody significantly ameliorated signs of hemolysis and VOC in a mouse model of SCD supporting the potential of ALXN1820 to treat SCD. Data from this Phase 2 study are anticipated to confirm the potential of ALXN1820 to treat SCD and guide the design of future studies in patients with SCD and other complement-mediated diseases."

Clinical • P2a data • Anemia • Bone Marrow Transplantation • Genetic Disorders • Hematological Disorders • Infectious Disease • Influenza • Meningococcal Infections • Pneumococcal Infections • Pneumonia • Respiratory Diseases • Sickle Cell Disease • Transplantation • HPX

Study of ALXN1820 in Healthy Adult Participants (clinicaltrials.gov) - P1 | N=60 | Completed | Sponsor: Alexion Pharmaceuticals | Active, not recruiting ➔ Completed | Trial completion date: Jan 2023 ➔ Oct 2022 | Trial primary completion date: Jan 2023 ➔ Sep 2022

Trial completion • Trial completion date • Trial primary completion date

Safety, Efficacy, Pharmacokinetic, and Pharmacodynamic Study of ALXN1820 in Adult Participants With Sickle Cell Disease (clinicaltrials.gov) - P2a | N=30 | Not yet recruiting | Sponsor: Alexion Pharmaceuticals

New P2a trial • Genetic Disorders • Hematological Disorders • Sickle Cell Disease • HPX