Protalix: Leveraging Tech In Developing Therapies For Orphan Diseases

Protalix Biotherapeutics (NYSE:PLX) is a drug discovery company that develops therapies for orphan diseases. Protalix has been leveraging its proprietary ProCellEx® platform, which is the key to the company’s production of complex and biologically similar proteins with a major focus on treating rare human diseases.

Orphan diseases are disease which affect a relatively small number of people and as such drug therapies aren't developed because the small size of the market would lend the research and drug unprofitable.

The global orphan drug disease market was valued at $151 billion in 2019 and is expected to reach $340.84 billion by 2027. The industry is dominated by a few players such as Celgene (NASDAQ:CELG), Roche (OTC:RHHBY), Novartis (NYSE:NVS), Sanofi (NASDAQ:SNY), and Amgen (NASDAQ:AMGN). PLX collaborates with various pharma companies to market its drug, for which it earns royalties and milestone payments.

PLX has successfully developed and launched a biological drug to treat Gaucher disease, making Protalix the first and only company to gain regulatory approval for a protein produced through a plant cell-based expression system.

Protalix has four biological drugs in its pipeline with a focus on treating complex diseases like Fabry disease, Refractory Gout, and NETs (Neutrophil Extracellular Traps) related diseases.

Elelyso - Treatment For Gaucher Disease

The Company’s first commercial drug approved by the FDA and EMA is Elelyso (taliglucerase alfa) to treat Type 1 Gaucher Disease (an Orphan Disease). Protalix, in collaboration with Pfizer (NYSE:PFE), developed the drug, and upon its approval, Protalix sold its 40% share in 2015 to Pfizer for $46 million.

The transaction included a $10M stock purchase. Pfizer has global rights to sell and market the drug except in Brazil, where Protalix maintains the distribution rights to the drug. In Brazil, the drug is marketed as BioManguinhos alfataliglicerase in partnership with Fundação Oswaldo Cruz. As a result, Protalix generates revenue from the sale of drug substances to Pfizer, and from its Brazil operations.

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Disclosure: None.

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