Business Description

We are a commercial stage biopharmaceutical company focused on the discovery, development, production and commercialization of innovative therapeutics for rare diseases with significant unmet needs. We are the first and only company to gain FDA approval of a protein produced through plant cell-based expression in suspension. ProCellEx®, our unique, proprietary plant cell-based protein expression system represents a new method for developing recombinant proteins in an industrial-scale manner. Our corporate strategy includes development of treatments for rare and orphan diseases. To execute on our strategy, we are turning our focus to new, early-stage product candidates that treat indications for which there are high unmet needs in terms of efficacy and safety, including renal diseases. We believe our treatments of interest will address both genetic and non-genetic diseases. We currently intend to use our ProCellEx platform and PEGylation capabilities, as well as other modalities such as small molecules and antibodies, to take advantage of highly innovative opportunities. Consistent with this strategy, we are developing PEGylated uricase, or PRX-115, for the treatment of uncontrolled gout, Long Acting (LA) DNase I, or PRX-119, for the treatment of NETs-related diseases, and a number of other technologies and preclinical assets. We have completed a Phase 1 First-in-Human clinical trial of PRX-115. Currently, we are actively recruiting for, and the first patients have been randomized in a Phase 2 clinical trial of PRX-115 for the treatment of uncontrolled gout which we refer to as the RELEASE study. To date, we have successfully developed two commercial products, both of which are enzyme replacement therapies (ERTs): Elelyso ® (taliglucerase alfa) for the treatment of adult patients and children four years of age and older with Gaucher disease and Elfabrio ® (pegunigalsidase alfa) for the treatment of adult patients with a confirmed diagnosis of Fabry disease. Elelyso, our first commercial product, is our proprietary recombinant form of glucocerebrosidase (GCD), an enzyme naturally found in human cells that is mutated or deficient in patients with Gaucher disease . Elelyso was approved by the FDA in 2012 for injection as an ERT for the long-term treatment of adult patients with a confirmed diagnosis of type 1 Gaucher disease. Elelyso is the first plant cell derived recombinant protein to be approved by the FDA for the treatment of Gaucher disease and is now approved in more than 40 markets including the United States, Brazil, Israel and others. It is not approved in the European Union (EU). In August 2014, the FDA approved Elelyso for injection for children four years of age and greater. We have licensed the rights to commercialize Elelyso worldwide (other than Brazil) to Pfizer pursuant to the terms of an amended and restated exclusive license and supply agreement, or the Amended Pfizer Agreement, which is in effect until 2030. We sell drug substance to Pfizer for the production of Elelyso, subject to certain terms and conditions, and Pfizer retains the revenues generated from its sales of the product. In Brazil, we have licensed rights to commercialize Elelyso to Fiocruz. Elelyso is marketed as BioManguinhos alfataliglicerase in Brazil. We retain distribution rights to Elelyso in Brazil. See Commercialization of Approved Products --Elelyso – Pfizer and --Alfataliglicerase – Fundação Oswaldo Cruz (Fiocruz). ​ Our sales of Elelyso to Pfizer and Fiocruz are made at a fixed price directly to Pfizer and Fiocruz who maintain product in inventory, and we recognize revenue from those sales upon delivery. The timing of such sales do not directly reflect patient demand and, on a period-to-period basis, there may be variations in the orders placed by each of Pfizer and Fiocruz resulting in variability in our period-to-period results. There may be periods during which no orders are placed by either Pfizer or Fiocruz, whether as a result of inventory de-stocking or other factors.…