Post-merger Q32 Bio plans Phase 2 trial of promising therapy ADX-097

Experimental antibody ADX-097 designed to target only diseased tissues

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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A Phase 2 clinical trial testing Q32 Bio’s ADX-097 in people with ANCA-associated vasculitis (AAV) will be the result of a recently announced merger between the company and Homology Medicines.

While further details on the trial were not disclosed, results are expected in the second half of 2025.

After the all-stock merger is complete, the combined company will operate under the name Q32 Bio. To back the merger, Q32 Bio has secured a $42-million private placement from existing and new investors.

“The proposed merger with Homology Medicines and concurrent private placement is expected to provide Q32 Bio with the capital to drive development of our autoimmune and inflammatory pipeline through multiple clinical milestones,” Jodie Morrison, Q32 Bio’s CEO, said in a joint press release.

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Initial data expected by end of 2024

“This funding is expected to enable us to deliver … proof-of-concept data for ADX-097, a tissue-targeted inhibitor of complement activation, by year-end 2024, and topline ADX-097 clinical results in the second half of 2025,” Morrison added.

A front line of defense for the immune system, the complement system is a domino-like chain of proteins that work together in a tightly regulated manner to protect the body against infection and clear debris.

In AAV, however, the complement system becomes overly active, and is thought to contribute to the uncontrolled immune response that triggers small blood vessel inflammation and damage in people with the disease. This damaging inflammation can happen anywhere in the body, causing a range of symptoms.

Designed with Q32 Bio’s discovery platform that allows the generation of first-in-class complement inhibitors that target diseased tissues, ADX-097 is an antibody that works by binding to and blocking both C3d and factor H, two complement components.

The therapy is expected to stop complement activation only in tissues where it is too active, particularly where there is a high density of C3d molecules, while leaving its action on other tissues elsewhere unharmed, which differentiates it from current complement therapeutics.

In preclinical studies with non-human primates, ADX-097, administered through under-the-skin injections, targeted areas of the skin where excessive activation of the complement system had been induced by ultraviolet B rays. The experimental therapy stopped complement activation in the skin without affecting other parts of the body.

Q32 Bio recently completed a Phase 1 trial testing single or multiple ascending doses of ADX-097 versus a placebo in healthy volunteers, where the treatment was found to be safe and well tolerated.

Initiated last year, the study showed the therapy had a good immunogenicity profile, meaning it did not trigger an unwanted immune response. ADX-097, when given every week, reached levels expected to allow for complete complement suppression in targeted tissues, the release stated.

In addition to the Phase 2 trial in AAV patients, Q32 Bio is planning to launch an open-label basket study, which will test how well ADX-097 works in patients from different disease groups, with initial data expected by the end of 2024.

No estimate was provided by the company as to when either of the ADX-097 trials will launch.