Company Registration No. 32266355
Copenhagen – June 11, 2021 – Orphazyme A/S (ORPHA.CO; ORPH), an advanced-stage biopharmaceutical company pioneering heat shock protein response for the treatment of rare diseases, today announced that American Depositary Shares (ADSs) representing its common shares in Nasdaq dos US have since June 10, 2021 experienced extreme volatility in price and trading volume. The company is not aware of any material change in its clinical development programs, financial condition or results of operations that would explain such price volatility or trading volume that has occurred since June 10, 2021. Investors who purchase ADS or shares of the company they may lose a significant portion of their investments if the price of these bonds later declines.
Orphazyme applications for arimoclomol (branded MIPLYFFATM)1 for Niemann-Pick type C disease (NPC) are under priority review with the US Food and Drug Administration, with a PDUFA action date scheduled for June 17, 2021, as well as with the European Medicines Agency, with an opinion Committee for Medicinal Products for Human Use (CHMP) are expected later this year.
For additional information, please contact:
Copenhagen: Anders Vadsholt, CFO, +45 28989055
Chicago: Molly Carey Poarch, Global Media, +1-773-770-6888
About Orphazyme A/S
Orphazyme is an advanced-stage biopharmaceutical company pioneering the response of heat shock proteins for the treatment of rare diseases. The company is leveraging the amplification of heat shock proteins (or HSPs) to develop and commercialize new therapeutics for diseases caused by protein misfolding, protein aggregation and lysosomal dysfunction, including lysosomal storage diseases. Arimoclomol, the company’s lead candidate, is in clinical development for Niemann-Pick type C (NPC) disease and Gaucher disease. Orphazyme is headquartered in Denmark and has operations in the US and Switzerland. Orphazyme shares are listed on Nasdaq U.S. (ORPH) and Nasdaq Copenhagen (ORPHA.CO).
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 lysosome function. Arimoclomol is given orally and has now been studied in 10 phase 1 trials, four phase 2 trials, and three pivotal phase 2/3 trials. Arimoclomol is in clinical development for NPC and Gaucher disease. Arimoclomol has received an orphan drug (ODD) designation for NPC in the US and EU. Arimoclomol has received fast-track (FTD) designation from the US Food and Drug Administration (FDA) for NPC. In addition, arimoclomol has received FDA’s Innovative Therapy Designation (BTD) and Pediatric Rare Disease Designation (RPDD) for NPC. Arimoclomol is an experimental treatment and has not been approved by the FDA.
This company announcement may contain certain forward-looking statements, including regarding the PDUFA’s expected action date of June 17, 2021 for arimoclomol for the treatment of NPC, the potential approval of arimoclomol in the United States in June, and the Committee’s opinion of Medicinal Products for Human Use (CHMP) later this year. While the Company believes that its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this Company’s announcement of future events are subject to (i) change without notice and (ii) factors beyond its control from the company. Such statements may include, without limitation, any statements preceded by, followed by, or including words such as “target”, “believes”, “expects”, “intends”, “intends”, “may”, “anticipates”, “estimate” , ”“plan”,“project”,“will”,“may have”,“probable”,“should”,“would”,“could”and other words and terms of similar or negative meaning. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company’s control that may cause the Company’s actual results, performance or achievements to be materially different from the expected results, performance or achievements expressed or implied by such forward-looking statements. Except as required by law, the Company does not undertake any obligation to update these forward-looking statements publicly, or to update the reasons why actual results may differ materially from those anticipated in the forward-looking statements, even if new information becomes available. used in the future.