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

Orphazyme A/S
Company announcement                                                                                       
No. 12/2021
Inside information
Company Registration No. 32266355

Copenhagen – March 29, 2021 – Orphazyme A/S [ORPHA.CO (DK); ORPH (US)], a late-stage biopharmaceutical company pioneering the heat shock protein response for the treatment of neurodegenerative rare diseases, today announced its phase 2/3 trial evaluating arimoclomol for the treatment of inclusion body myositis (IBM), a progressively debilitating muscle-wasting disease, did not meet its primary and secondary endpoints. The primary goal was to evaluate the treatment effect on disease progression as measured by the inclusion body myositis functional rating scale (IBMFRS).

The randomized, placebo-controlled trial was conducted among 150 IBM patients at 12 sites in North America and Europe, in partnership with University College of London and the University of Kansas. Participants were randomized (1:1 ratio) to receive either arimoclomol citrate (400 mg three times daily) or placebo for up to 20 months.
No important safety concerns were detected in the trial. The analysis of the data is continuing and complete findings from the study will be shared in a future scientific forum.

“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,” said Thomas Blaettler, MD, Chief Medical Officer, Orphazyme. “We are grateful to the investigators and their sites, and the many patients and families who graciously participated in the trial.”

“We continue to believe in the promise of arimoclomol and heat shock protein science and are fully committed to our mission to deliver new therapies to patients,” said Anders Vadsholt, interim Chief Executive Officer and Chief Financial Officer, Orphazyme.

Orphazyme expects data from a pivotal Phase 3 trial of arimoclomol in Amyotrophic Lateral Sclerosis (ALS), a neurodegenerative disease, this spring. The company’s applications for arimoclomol (to be branded MIPLYFFA^TM1) for Niemann-Pick disease type C (NPC) are under priority review with the U.S. Food and Drug Administration, with an expected action date in June 2021, as well as with the European Medicines Agency, with an opinion from the Committee for Medicinal Products for Human Use (CHMP) expected later this year.