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Tarperprumig (also known by its development code ALXN1820) is a protein-based substance developed by Alexion Pharmaceuticals, Inc.[1] It is classified as a bispecific antibody and belongs to a group of medicines called anti-inflammatories and properdin inhibitors.[2] The substance is still investigational: the product records show no marketing authorisation, meaning it has not yet been approved as a medicine for routine use.[1] It has been given orphan drug status, which is a designation used to encourage the development of medicines for rare diseases.[2]
Tarperprumig works by blocking a protein called properdin, which is part of the body's natural defence system known as the complement system.[3] The complement system normally helps protect the body against harmful invaders, but in people with ANCA-associated vasculitis it becomes activated in an unhelpful way and contributes to the disease.[3] By inhibiting properdin, tarperprumig aims to reduce this harmful immune activity.[3] The mechanism of action is recorded as properdin inhibition.[2]
Tarperprumig is not yet authorised for any condition. It is being studied for one condition:
An earlier Phase 1 study evaluated tarperprumig in healthy adults to understand its safety and how the body processes it.[4]
Tarperprumig is prepared as a solution for injection and is given by subcutaneous injection, meaning it is injected under the skin.[1] In the Phase 2 trial, participants receive tarperprumig or a placebo by subcutaneous injection.[1] The trial includes three study arms: tarperprumig every week, tarperprumig alternating weeks, or placebo.[3]
The clinical research on tarperprumig includes a completed Phase 1 study in healthy adults and an ongoing Phase 2 trial in people with ANCA-associated vasculitis.[4][1] The Phase 2 trial is randomised, double-blind, and placebo-controlled, meaning participants are assigned by chance to receive either tarperprumig or a placebo, and neither they nor the researchers know which one they are getting.[3] The trial is currently authorised and plans to enrol 75 participants.[1]
The main measures in the Phase 2 trial are the number of side effects (called treatment-emergent adverse events, or TEAEs, and serious treatment-emergent adverse events, or TESAEs) and changes in safety assessments such as vital signs, physical examinations, laboratory tests, and electrocardiogram (ECG) results.[1] The study also looks at pharmacokinetics (what the body does to the drug) and pharmacodynamics (what the drug does to the body).[3] The trial has a screening period of up to 2 weeks, a treatment period of 52 weeks, and a safety follow-up period of 18 weeks.[3]
The Phase 1 study in healthy adults assessed safety, tolerability, pharmacokinetics, pharmacodynamics, and immunogenicity (whether the body develops an immune response to the medicine).[4]
The Phase 2 trial is specifically designed to measure the side effects of tarperprumig and how severe they are compared to placebo.[3] The trial records list the incidence of treatment-emergent adverse events and serious treatment-emergent adverse events as primary outcome measures, but detailed side effect data from this trial are not yet available.[1] Results from the earlier Phase 1 study in healthy adults are published, but the available source does not provide a specific list of side effects observed.[4]
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