RARE Daily

Orphazyme’s Treatment for Rare Muscle Wasting Disease Fails in Late Stage Trial

March 29, 2021

Rare Daily Staff

Danish biotech Orphazyme reported that its phase 2/3 trial evaluating arimoclomol for the treatment of inclusion body myositis failed to meet its primary and secondary endpoints.

Orphazyme’s shares fell 31 percent on the news.

Inclusion Body Myositis (IBM) is a progressively debilitating muscle-wasting disease characterized by a build-up of protein aggregates and atrophy of muscle cells, which leads to weakness and severe disability over time. There are no approved treatments for IBM.

The primary goal of the trial 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, chief medical officer, Orphazyme.

Orphazyme believes arimoclomol can have a therapeutic effect in several neurodegenerative disorders. The experimental drug amplifies the production of heat-shock proteins (HSPs), which can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes. Arimoclomol is administered orally, crosses the blood brain barrier, and has been studied in 10 phase 1, four phase 2 and two pivotal phase 2/3 trials.

The company expects data from a pivotal phase 3 trial of arimoclomol in amyotrophic lateral sclerosis, a neurodegenerative disease, in the second quarter of 2021. The company’s applications for arimoclomol for the treatment of Niemann-Pick disease type C 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 expected later this year.

Arimoclomol has received orphan drug designation for NPC, IBM, and ALS in the United States and European Union. Arimoclomol has also received fast-track designation from the FDA for NPC, IBM and ALS. In addition, arimoclomol has received breakthrough therapy designation and rare-pediatric disease designation from the FDA for NPC.

“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 CEO and chief financial officer, Orphazyme.

Photo: Anders Vadsholt, interim CEO and chief financial officer, Orphazyme

Stay Connected

Sign up for updates straight to your inbox.