Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Higher Doses of OMS906
Research type
Research Study
Full title
A Phase 1 Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Higher Doses of OMS906 by Single Dose Intravenous Administration in Healthy Subjects
IRAS ID
1008854
Contact name
Samantha Magazu
Contact email
Sponsor organisation
Omeros Corporation
ISRCTN Number
ISRCTN16253842
Research summary
Research Summary
Paroxysmal nocturnal haemoglobinuria (PNH) is a rare blood condition where blood cells are attacked and destroyed by the immune system. This can lead to variety of symptoms including blood clots, serious infections and bone marrow failure.\nThe human immune system is the body’s defence mechanism against infections and diseases. The complement system is part of the immune system that uses a group of specialised proteins to fight infection. There are multiple pathways the complement system uses to protect the body, one of which is called the alternative pathway (AP). The AP is activated by viruses, fungi, bacteria and parasites, and forms an important part of the body’s defence mechanism by tagging invading organisms for destruction. However, sometimes this process becomes poorly regulated and the immune system mistakenly attacks and damages parts of its own body, which is known as an autoimmune disease. Autoimmune diseases can trigger the spread of inflammation and tissue destruction within the body. The AP has been implicated in a broad range of autoimmune responses that can lead to kidney failure, heart failure and other serious health problems, resulting in illnesses like PNH. In PNH, uncontrolled activity of the AP results in the destruction of red blood cells causing anaemia.\nTo date there are only a few options to treat PNH. Therefore, we wish to investigate further if OMS906 may help in treating PNH.\nDuring the study, volunteers will be administered a single dose of OMS906 in a supervised clinical setting. \nWe plan to enrol up to 16 healthy volunteers into this study, who will divided into 2 cohorts (8 participants per cohort). During this study they will be infused with a single dose of OMS906 or placebo.
Summary of Results
"The study was organised and funded by Omeros Corporation (the ‘Sponsor’) and was conducted by MAC Clinical Research at 1 study site in the United Kingdom from March 2024 to April 2025.
Why was this study conducted?
The purpose of this study was to test a drug called zaltenibart also known as OMS906 (the ‘study drug’) that is being developed for treating a range of inflammatory and autoimmune diseases associated with a biological process known as the alternative pathway of
complement (AP). An overactive complement system can lead to inflammation and damage to blood cells or internal organs such as the kidneys. Examples of such diseases include paroxysmal nocturnal haemoglobinuria (PNH), C3 glomerulopathy (C3G), and idiopathic
immune complex-mediated glomerulonephritis (ICGN).
The study drug is a type of antibody (a humanised immunoglobulin G4 monoclonal antibody) that binds to an enzyme in blood known as mannan-binding lectin-associated serine protease-3 (MASP-3). As a result, the activity of another enzyme in blood, complement
factor D (CFD), is inhibited reducing activation of the AP. Evidence suggests that reducing the activity of MASP-3 has the potential for the treatment of a broad range of inflammatory and autoimmune diseases.
The main aims of this study were:
• To assess the safety and tolerability of higher single doses of the study drug.
• To assess the relationship between how the body absorbs and removes the study drug, and how levels of the enzymes MASP-3 and CFD in blood are affected.
• To assess whether treatment with the study drug leads to the development of anti-drug antibodies that may affect the safety or effectiveness of the study drug.
Who took part in the study?
A total of 16 healthy participants took part in the study. The first 8 participants were assigned to Group 1 and were randomly assigned to receive the study drug at a dose of 8 mg/kg (6 participants) or placebo (2 participants). Placebo is a substance that looks like the study
drug but does not contain the active ingredient. After all participants in Group 1 had completed Day 28 of the study and data collected had been reviewed to confirm that treatment with the study drug at that dose was safe, the second 8 participants were recruited
and assigned to Group 2. These participants were randomly assigned to receive the study drug at a dose of 12 mg/kg (6 participants) or placebo (2 participants). All participants completed the study.
What were the overall results of the study?
Side effects are unwanted medical issues during the study that may or may not be related to the study drug. No serious side effects or safety observations of concern were reported. The side effects reported were all considered not related or unlikely related to the study drug.
Medical conditions related to infections were the most frequently reported. Treatment with the higher dose of study drug (OMS906 12 mg/kg) resulted in higher levels of study drug in the blood compared to the lower dose (OMS906 8 mg/kg). Both doses of study
drug led to appreciable reductions in the levels of MASP-3 and CFD in blood. The higher dose led to a greater reduction in the levels of these enzymes for a longer period of time than the lower dose. Although 2 participants did develop anti-drug antibodies, this did not seem to affect the safety or the pharmacological effect of the study drug. The study demonstrated promising results related to further development of the study drug for the treatment of inflammatory and autoimmune diseases associated with the AP.
How has this study helped patients and researchers?
This study was conducted on male and female participants aged 20 to 58 years who were considered healthy. The results from this study are limited to the particular people studied and cannot be assumed true for everybody. This research helps future patients and families by helping us understand more about the medicine being studied. Findings from this study will be used to inform future studies with OMS906.
Where can I find more information about this study?
To learn more about this study, visit https://www.isrctn.com/
This summary was completed on 10 December 2025. Newer information since this summary was written may now exist. This summary includes only results from one single study. Other studies may find different results."REC name
Wales REC 1
REC reference
23/WA/0330
Date of REC Opinion
11 Jan 2024
REC opinion
Further Information Favourable Opinion