Positive Phase I results for small molecule complement inhibitor

Medical laboratory

Biopharmaceutical company Annexon has reported promising results from its Phase I single ascending dose (SAD) and multiple ascending dose (MAD) healthy volunteer study of ANX1502.

ANX1502 is a first-in-kind oral, selective small molecule inhibitor that targets the active form of C1s responsible for propagating classical pathway activation in association with C1q.

In the study, ANX1502 achieved target serum levels and demonstrated pharmacokinetic (PK) measures that support advancement into a proof-of-concept clinical study to assess pharmacodynamics (PD) and efficacy in patients with cold agglutinin disease (CAD) in 2024.

“After more than a decade of groundbreaking research targeting the early classical complement pathway, we are excited to have reached an important step in the clinical development of ANX1502, our first-in-kind small molecule complement inhibitor that we believe can have meaningful impact on a range of autoimmune conditions,” said Ted Yednock, Chief Innovation Officer of Annexon.

“We’re very encouraged by the results from our Phase I SAD/MAD trial showing that ANX1502 was well tolerated and achieved target drug levels with supportive impact on a key biomarker in healthy volunteers. Based on these data, we look forward to advancing a tablet formulation of ANX1502 into a proof-of-concept study in patients with CAD, which enables us to further explore larger opportunities in serious autoimmune diseases.”

According to the study results, dose-proportional PK and targeted levels of active drug were observed across both SAD and MAD cohorts. Single doses of 525-1025mg ANX1502 suppressed C4d serum levels in healthy volunteers with higher than median baseline C4d.

Across all doses evaluated, ANX1502 was generally well tolerated with mild to moderate treatment-emergent adverse events (TEAEs). No serious adverse events were reported, and there were no significant clinical or lab findings.

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