ABX464: A Potential Candidate With a Novel Mechanism of Action for the Treatment of Rheumatoid Arthritis

EULAR 2022: ABX464 for Rheumatoid Arthritis

1 June, 2022 | DelveInsight

Phase IIa study in patients with moderate to severe rheumatoid arthritis and inadequate response to MTX and/or anti-TNFα therapeutics shows that oral ABX464 50 mg once daily is safe, well-tolerated, and shows promising efficacy results (Abstract # POS0688).

ABX464 upregulates the expression of the anti-inflammatory microRNA miR-124 and is a first-in-class drug candidate as an oral treatment for moderate to severe RA.

The Phase IIa study displayed encouraging results in evaluating the safety and preliminary efficacy of ABX464 in combination with methotrexate (MTX) in patients with moderate-to-severe active RA who have an inadequate response to MTX or/and to an anti-TNFα therapy.

The primary endpoint was the safety of ABX464; efficacy endpoints included the proportion of patients achieving ACR20/50/70 responses and disease activity scores (DAS28, SDAI, CDAI), EULAR response, DAS28 low disease activity or remission. Compared to placebo, ABX464 50 mg showed significantly higher proportions of patients achieving ACR20 (56%) and ACR50 (31%) responses at Week 12 in the per-protocol population. ABX464 50 mg was safe and well-tolerated, and two serious adverse events (SAEs) were reported (one in the placebo group and one on ABX464 100 mg).

KOL insights

“For rheumatoid arthritis patients, there is still a significant unmet medical need for safe and durable medicines. Alternative therapy approaches, such as obefazimod, based on a novel mechanism of action, are thus required.”– Expert Opinion

“Promising data of the Phase IIa induction study presented at EULAR, the high levels of sustained response rates of the Phase IIa maintenance trial with obefazimod in RA patients, looks encouraging, and along with its unique mode of action and clinical profile, obefazimod has the potential to play a significant role in the future management of RA patients” – Expert Opinion


The Phase II study’s primary endpoint was achieved with 50 mg obefazimod once daily, revealing a favorable safety and tolerability profile in the overall patient population during the 12-week induction period.

The efficacy and safety findings of the first-in-class therapeutic candidate ABX464 suggest further investigation as an oral treatment for RA patients at 50 mg q.d. or less. There is undoubtedly a need for novel mechanisms of action in RA, and obefazimod might fill that void. If this drug shows comparable positive outcomes in further clinical trials, it might become a potential therapy option for RA patients.

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