Orphazyme announces topline results from pivotal trial of arimoclomol for Inclusion Body Myositis (IBM)

Orphazyme has just released the results of the Phase 2/3 trial of Arimoclomol in inclusion body myositis (IBM). Unfortunately, the trial did not meet its primary and secondary endpoints.

Remember, we learn so much from failed trials. As participants in this trial, you have made a difference in medical research no matter if the trial failed or succeeded. So we thank all of the patients and their caregivers who participated in this trial for your dedication and desire to help further the cause of research. You are all warriors.

We understand this is devastating news, and it’s okay to have feelings, whether anger, sadness, confusion. We are often referred to as the heart of the myositis community, so please know we are here for you. You can find online and video support at https://Understandingmyositis.org/support.

MSU is committed to listening and including you so we can be the voice of the community. This only strengthens our resolve to collaborate across the myositis community in advocating for a more patient-centric approach to research. There are already other clinical trials for IBM being launched, so we are hopeful a cure can be found.

There will be additional analyses on the findings, and full study results will be available later this year. If you have questions or comments or feedback, we want to hear from you. Email us at info@understandingmyositis.org.

Below is the statement from Thomas Blaettler, MD, Chief Medical Officer, Orphazyme:

“We recognize these data are disappointing for patients and families who continue to eagerly await a promising option for IBM. We believe the data collected will be useful to the community, since this trial represents one of the largest, long-term studies ever conducted in this disease and will help inform future research in the category. We are grateful to the investigators and their sites, and the many patients and families who graciously participated in the trial.”


We also encourage you to read the full press release



Myositis Support and Understanding Association (MSU) is a patient-centered, all-volunteer 501(c)(3) nonprofit organization Empowering the Myositis Community. Founded by Myositis patients, for Myositis patients, MSU provides education, support, advocacy, access to research and clinical trial matching, and need-based financial assistance.

View more information: Myositis Support


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