Update April 23, 2019 – Orphazyme A/S announced that its Phase II/III trial of arimoclomol for the treatment of sporadic Inclusion Body Myositis (sIBM) is fully enrolled.

“The study is a 20-month trial comparing placebo with active treatment (arimoclomol) with a planned interim analysis at 12 months. However, it is not anticipated that the study will end at this time. The final study results are expected in the first half of 2021 at the conclusion of the 20-month timepoint. The 20-month primary analysis time-point was established with regulatory authorities (FDA and EMA) to maximize chances of success, potentially allowing for a meaningful difference to be seen between participants receiving active treatment vs placebo.”

Performance of interim analysis and study completion expected in H1 2020 and by end 2020, respectively. Results are expected in H1 2021.

“Our team looks forward to continued conversations with The Myositis Association (TMA), Myositis Support & Understanding (MSU), the Muscular Dystrophy Association (MDA), those participating in the Yale IBM Registry, and other patient organizations to ensure the latest information regarding Orphazyme’s clinical development program in sIBM is available to those who need it most: People living with sIBM and their loved ones.”

The Phase II/III trial of arimoclomol for sIBM is a 150-patient, 20-month, randomized, double-blinded, placebo-controlled trial at 11 centers of excellence in the US and one in the UK. Orphazyme has initiated an open-label extension trial to which patients from the Phase II/III trial may enroll and continue treatment.

Read the Full Press Release

Read Thank you Letter to IBM Community

As part of our mission at Myositis Support and Understanding Association (MSU), we are constantly looking for ways to advocate for you, the Myositis Community, by engaging with research and pharmaceutical companies to better the lives of patients and caregivers. One of our goals is to provide you with access to research.

To this end, MSU is excited to announce a new relationship with Orphazyme, the Danish biopharmaceutical company sponsoring the Arimoclomol Phase II/III clinical trial for sporadic inclusion body myositis (sIBM).

In a series of conference calls with Regan Sherman, Associate Director of Patient Advocacy Relations, we discussed the various ways Orphazyme and MSU can work together to benefit those living with sporadic inclusion body myositis. These talks were very productive and we look forward to realizing many of the ideas and events discussed.

As a part of our relationship, we will bring information and updates directly to the sIBM patient and caregiver communities. We will also work together with Orphazyme to help guide education, awareness, outreach, and other efforts valuable to our membership.

“This is an exciting mutually beneficial relationship with a shared goal of a successful clinical trial and ultimately, a treatment for sIBM patients,” says Jerry Williams, Founder and President of Myositis Support and Understanding Association.

Many patients are interested in the arimoclomol trial and with good reason. Sporadic inclusion body myositis (sIBM) is a rare, disabling inflammatory and muscle-wasting disease, one of several in the group of muscle diseases called the Idiopathic Inflammatory Myopathies. There are currently no available treatment options for patients with sIBM. Rather, patients are usually directed to utilize exercise to help slow disease progression by maintaining muscle strength and to make life adaptations, or life hacks, to better live with this disabling disease.

See full Arimoclomol trial information

Further clinical trial summary information can be found on the Pipeline tab on the Orphazyme website.

Register for your free MSU membership

We thank Regan and the entire Orphazyme team for their willingness to work with and learn from myositis nonprofits and community organizations. We also thank the inclusion body myositis community for their strength, patience, and excitement as we continue to work for them.

As with any relationship MSU enter into with pharmaceutical companies, our policies will not be determined or guided by any contributions, financial or otherwise. Member privacy is always protected and members private information is never shared with any third parties unless expressly consented, including informed and signed consent.

About Arimoclomol

Arimoclomol is a new chemical entity with a very favorable safety and tolerability record in humans: Seven Phase I clinical studies have been conducted in healthy volunteers. Arimoclomol is administered orally, three times daily, and can be easily dissolved in liquids or food for best possible patient comfort and compliance. Orphazyme has obtained orphan drug designation for arimoclomol in NPC, ALS, and sIBM from both the FDA and EMA.

About Orphazyme A/S

Orphazyme is a Danish biotech company listed on Nasdaq Copenhagen with a late-stage drug pipeline, developing new treatment options for orphan protein-misfolding diseases. The company was founded in 2009 based on early scientific discovery in heat-shock proteins. The company is headquartered in Copenhagen and currently has 30 employees. The lead candidate arimoclomol is in development as a potential treatment for four orphan diseases; two neuromuscular diseases, sporadic Inclusion Body Myositis (“sIBM”) and Amyotrophic Lateral Sclerosis (“ALS”), and two lysosomal storage diseases, Niemann Pick Type C (“NPC”) and Gaucher disease. For more information, please visit



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

  1. Jerry Williams 2 years ago

    Hi Theodore, Unfortunately, the arimoclomol was a negative study, and didn’t meet the primary and secondary endpoints. Please check our News section for the announcement. I know this is difficult news, but at MSU we are committed to helping find a treatment for IBM. If you are not a part of our support group, I would encourage you to join us. See the Support section of our site, or visit My best to you.

  2. Theodore Rousseau 2 years ago

    Please keep me updated on the efficacy and availability of this drug Arimoclomol, as I am suffering SIBM and is slowly approaching the stage if the inability to walk unaided, I already cannot climb any stairs for the fear of falling and getting up from sitting is becoming extremely difficult.. It would be critical for me to start if the treatment is successful, as soon as possible.

  3. Jerry Williams 4 years ago

    Hi Jim and thank you for your comment. There is also another form of IBM recognized as Familial Inclusion body myositis. I will email you with that information.

    To receive email updates, please register for your MSU membership using the Sign Up link at the very top of the site. I will include this in the email I send to you.

    Thank you again!

  4. Jim Funderburk 4 years ago

    I could thoroughly relate to all of your above informative articles on sIBM, as I was diagnosed with IBM in the early months of 2011 after extensive tests (Including a leg biopsy which confirmed vacuoles) at UVA Rheumatology in Charlottesville, VA, and later that year also confirmed at Johns Hopkins in Baltimore, MD. At that time I was 66 years old and the muscle weaknesses had not as yet progressed enough to be a problem. Now at age 75, the disease has very much progressed causing much difficulty in many daily activities. I am thinking more in terms of sporadic than hereditary IBM as to my specific age experience with this disease. However, I have a 1st and 2nd cousin on my father’s side of family that have also been diagnosed with IBM. And their diagnosis’ were confirmed at much earlier ages (40’s and 50’s). They are currently in much worse condition than I am, and wheelchair bound.
    If you compare known incidence rate to general population, that in my family would be 3 in 70 people, compared to about 1 in 1.3 million. My father was one of 13 children so I am blessed with many 1st and 2nd cousins.
    I would like to be included in your patient address listings to receive any available new information that comes-up about this disease in the future. I am a member of The Myositis Association. Thanks for posting your very significant and informative information about IBM on social media. You may use any of above information for your research and records.

Leave a reply

Your email address will not be published. Required fields are marked *


This site uses Akismet to reduce spam. Learn how your comment data is processed.

© 2023 Myositis Support and Understanding Association (MSU). All rights reserved. | View our Privacy Policy, Terms, and Non-Discrimination policy. MSU is a charitable organization with 501(c)(3) tax-exempt status. Federal ID #47-4570748.

View COVID-19 resources and updates, and stay tuned to your state, local, and federal health agencies, along with the CDC.

Log in with your credentials


Forgot your details?

Register for Free Membership

Send this to a friend