Uniqure readies share stock offering

Following up on its well-received lead programme switch, gene therapy biotech Uniqure announced in late October that it has commenced an underwritten public offering of 5,000,000 of its ordinary shares. In addition, Nasdaq-listed Uniqure intends to grant the underwriters a 30-day option to purchase up to 750,000 additional ordinary shares at the public offering price. or when the offering may be completed, or as to the actual size or terms of the offering. Price and terms have yet to be released.

A couple of days earlier, the Amsterdam-based company had announced that following multi-disciplinary meetings with the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), it plans to "expeditiously advance AMT-061 into a pivotal study in 2018 for patients with severe and moderately severe hemophilia B". AMT-061 combines an AAV5 vector with the FIX-Padua mutant. It replaces its predecessor AMT-060 which has been tested in 10 patients in an ongoing Phase I/II clinical trial. Both molecules are identical in structure apart from two nucleotide substitutions in the coding sequence for FIX. The gene variant, referred to as FIX-Padua, expresses a protein with a single amino acid substitution that has been reported in multiple preclinical and nonclinical studies to provide an approximate 8 to 9-fold increase in FIX activity compared to the wild-type FIX protein. All other critical quality attributes of AMT-061 are expected to be comparable to those of AMT-060. "Our mission in hemophilia B has always been to develop the safest and most effective gene therapy with the broadest application to patients. We believe AMT-061 moves us closer to this goal, as it has the potential to provide optimized clinical and tolerability benefits to nearly all severe and moderately severe patients with hemophilia B," stated Matthew Kapusta, Chief Executive Officer of Uniqure.

Update 25.10.2017: Uniqure has revealed the public offering price. At US$18.25 per share the gross proceeds are expected to be more than US$91m.

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