Results from Orphanzyme inclusion body myositis (IBM) clinical trial

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Research Team
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At the end of March the biotech company Orphanzyme announced negative results from its Phase 2/3 clinical trial testing arimoclomol for the treatment of inclusion body myositis (IBM).

Whilst the exact cause of IBM is not fully understood, some research suggests incorrect folding of certain proteins may play a role. These proteins accumulate as clumps in the muscles, and are thought to result in muscle inflammation and muscle damage. Arimoclomol is a drug that enhances cells’ natural ability to mend incorrectly folded proteins. This study aimed to assess the safety and efficacy of arimoclomol as a potential treatment for people with IBM.

In the trial, arimoclomol did not show a functional benefit on the progression of the condition as measured by the inclusion body myositis functional rating scale (IBM-FRS). The IBM-FRS is a tool to measure the severity of the condition and can be used to see how the condition progresses. It is a set of ten questions to measure ability to do certain physical activities such as climbing stairs or swallowing. The trial also did not meet its secondary endpoints, which were other measures used to test the efficacy of the treatment.

Although this is disappointing news, the learnings from this trial are important. They have increased our knowledge of IBM and will help in designing future clinical trials.

For more information read Orphanzyme’s press release or the study page on

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