News February 7 2020

Orphazyme raises $109m for arimoclomol filings in USA and Europe

Danish biopharma Orphazyme (CPH: ORPHA) has raised 745 million Danish kroner ($109 million) in a directed issue and private placement.

Orphazyme is focused on bringing new treatments to patients living with life-threatening or debilitating rare neurodegenerative diseases.

‘Significant cash runway’

Kim Stratton, chief executive, said: “I would like to thank our investors, both existing and new, for their confidence in Orphazyme as the company enters a very exciting phase in its development.

“This funding provides a significant cash runway for Orphazyme as we progress our lead product, arimoclomol, towards market, and is expected to cover our ambitious clinical development plans well into 2021. In particular, the financing will support our imminent US and European filings of arimoclomol for Niemann-Pick disease Type C and preparations for commercial launch.”

FDA designations

These filings are expected to be submitted this year. Orphazyme has been granted Fast Track and Breakthrough Therapy designation from the US Food and Drug Administration for the development of arimoclomol in Niemann-Pick disease Type C.

Orphazyme also intends to use the proceeds to cover completion of arimoclomol’s clinical development in sporadic inclusion body myositis and amyotrophic lateral sclerosis, and to prepare regulatory filings in Europe and potentially the USA in these indications.