(Note: This is not an IPO, although the company says this is its IPO of ADS in the United States. This is a follow-on offering: It’s an uplisting from the Nasdaq Copenhagen, where, according to the prospectus, the company’s ordinary shares trade under the symbol “ORPHA” and closed on Sept. 17, 2020, at DKK 82.70 per ordinary share, which equals a price of $13.13 per ADS, based on an exchange rate of DKK 6.2967 per $1.00 as of Sept. 17, 2020, and an ADS-to-ordinary share ratio of 1 to 1.)

We are a late-stage biopharmaceutical company harnessing the amplification of Heat Shock Proteins, or HSPs, in order to develop and commercialize novel therapeutics for the treatment of neurodegenerative orphan diseases. In July 2020, we completed a rolling submission of a new drug application, or NDA, for our product candidate, arimoclomol, with the U.S. Food and Drug Administration, or FDA, for Niemann-Pick disease Type C, or NPC, and intend to submit a marketing authorization application, or MAA, to the European Medicines Agency, or EMA, in the second half of 2020. Arimoclomol is also in registrational clinical trials for the treatment of Amyotrophic Lateral Sclerosis, or ALS, and Sporadic Inclusion Body Myositis, or sIBM, and we intend to advance into pivotal-stage clinical development in neurological Gaucher disease.

Arimoclomol is an orally- or naso/gastrically-administered small molecule that crosses the blood-brain barrier and is designed to selectively amplify the natural role of endogenous HSPs, which protect against cellular toxicity caused by protein misfolding, aggregation and lysosomal dysfunction.