Q32 Bio pushes back Phase 2 trial of ADX-097 in AAV patients
No serious treatment side effects seen in Phase 1 trial in healthy volunteers
Q32 Bio is delaying the start of a planned Phase 2 clinical trial designed to test its ADX-097 therapy candidate in people with ANCA-associated vasculitis (AAV).
The trial had been expected to begin in 2025.
While details on that planned Phase 2 trial in AAV patients had not been disclosed, its initial results had been anticipated by the second half of next year.
Now, however, recruitment for that trial will be delayed for an unknown period of time while Q32 Bio instead turns its attention to another therapy candidate, bempikibart, now in clinical trials for treating alopecia areata, an autoimmune disease that causes patchy hair loss. Q32 Bio also is advancing a basket study testing ADX-097 for kidney damage in autoimmune and immune-related diseases, according to a company press release announcing updates on both therapies.
“The company will defer enrollment into the planned Phase 2 clinical trial of ADX-097 in ANCA-associated vasculitis … previously expected to begin in 2025, to focus efforts on continued enrollment in the ongoing bempikibart [alopecia areata] and ADX-097 [kidney] basket Phase 2 clinical trials,” the press release stated.
The ongoing basket trial (NCT06419205) is designed to test ADX-097 in adults with autoimmune or immune-related conditions known to cause kidney damage. No timeline was given as to when the ADX-097 trial in AAV now may start.
Phase 2 trial in AAV patients was designed to follow study in healthy people
Most cases of AAV, a group of autoimmune diseases marked by inflammation and damage to small blood vessels, are driven by the production of self-reactive antibodies called ANCAs.
Excessive activation of the complement system, a network of immune proteins that act in a chain reaction to protect the body from potential threats, is also thought to contribute to AAV.
This uncontrolled immune response can cause damage to tissues in various parts of the body, resulting in a wide range of symptoms.
ADX-097 is an antibody designed to target two complement proteins, C3d and factor H. Unlike other complement-targeting treatments, ADX-097 is expected to act only on tissues where C3d levels are high, indicating an overly active complement system, without affecting tissues with normal complement activation.
In a first-in-human Phase 1 trial, different doses of ADX-097 were administered to healthy volunteers intravenously, or directly into the bloodstream, or through under-the-skin, or subcutaneous, injections.
The study’s main goals were to assess how safe ADX-097 was, how it moves into, through, and out of the body — known as a drug’s pharmacokinetics — and its effects on the body, or its pharmacodynamics.
ADX-097 was found to be well tolerated, without serious side effects or immune reactions at any dose. Also, weekly subcutaneous injections at a dose of 450 mg were found to reach levels in the blood that are expected to be enough to suppress the complement system in damaged tissues, but not in other body tissues.
As such, this dosage is expected to efficiently block complement overactivation in target tissues, while avoiding widespread suppression of C3d and factor H.
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