Orphazyme A/S (ticker: ORPHA.CO), a biopharmaceutical company dedicated to developing treatments for patients living with rare diseases, recently announced the completion of enrollment to its phase 3 trial of arimoclomol in amyotrophic lateral sclerosis (ALS)
For more information, please view Orphazyme’s recent announcement here.
ORARIALS-01 is a multi-center, randomized, double-blind, placebo-controlled, parallel-group trial, which evaluates the efficacy and safety of arimoclomol over a 76-week treatment period. Patients diagnosed with ALS who had first appearance of symptoms (weakness) within the previous 18 months were eligible for screening. Following confirmation of eligibility during the screening/baseline period (Day -28 to Day 1), 245 subjects were randomized in a 2:1 ratio to receive either arimoclomol or placebo orally.
The first patient was enrolled into this trial on August 10, 2018, (see announcement here). Recruitment was completed in 11 months from 30 centers located in North America and Europe.
About Worldwide Clinical Trials
Worldwide Clinical Trials employs more than 1,600 professionals around the world, with offices in North and South America, Eastern and Western Europe, Russia, and Asia. Founded by physicians committed to advancing medical science, Worldwide is out to change how the world experiences CROs – in the best possible way. From early phase and bioanalytical sciences through late phase, post-approval and real-world evidence, we provide world-class, full-service drug development services.
With infrastructure and talent spanning 60 countries, we execute predictable, successful studies with operational excellence across a range of therapeutic areas, including central nervous system, cardiovascular, metabolic, immune-mediated inflammatory disorders (IMID), oncology and rare diseases. We never compromise on science or safety. We’re never satisfied with the status quo. We’re the Cure for the Common CRO. For more information, please visit http://www.worldwide.com.
About Orphazyme A/S
Orphazyme is a biopharmaceutical company focused on bringing novel treatments to patients living with life-threatening or debilitating rare diseases. Our research focuses on developing therapies for diseases caused by misfolding of proteins and lysosomal dysfunction. Arimoclomol, the company’s lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C, Gaucher disease, sporadic inclusion body myositis, and amyotrophic lateral sclerosis. The Denmark-based company is listed on Nasdaq Copenhagen (ORPHA.CO). For more information, please visit www.orphazyme.com.
About Amyotrophic Lateral Sclerosis (ALS).
Amyotrophic lateral sclerosis (ALS) is a rare, rapidly progressive, and always fatal neurodegenerative disease. Protein misfolding and aggregation in motor neurons are important contributors to the disease process, which ultimately leads to paralysis of skeletal muscles as well as the muscles that enable breathing. The patient population in Europe and the United States is estimated to be approximately 50,000 patients. Currently, there are only limited treatment options available.
About arimoclomol
Arimoclomol is an investigational drug candidate that amplifies the production of heat-shock proteins (HSPs). HSPs 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 seven phase 1 and three phase 2 trials. Arimoclomol is in clinical development for NPC, Gaucher disease, sIBM, and ALS. Arimoclomol has been granted Orphan Drug Designation (EU and USA) for the treatment of ALS.
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Source: Worldwide Clinical Trials, Inc.
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