Protalix BioTherapeutics Presents Fabry Disease Research Findings

Presented for 16th Annual WORLD Symposium

In a February 10, 2020 press release about clinical data of pegunigalsidase alfa for the treatment of Fabry disease, Protalix BioTherapeutics, Inc, an American biopharmaceutical company, announced that it would present its Phase III BRIDGE clinical trial findings at the 16th Annual WORLD Symposium™ 2020 in Florida. 

The clinical trial study of pegunigalsidase alfa studied its benefits, risks, and safety on 22 patients who had been on agalsidase alfa (Replagal®) for a minimum of two years with a steady dose for a minimum of six months. The participants in the study received 1 mg of pegunigalsidase alfa for every 1 kg of body weight every two weeks. 

How Pegunigalsidase Alfa Treats Fabry Disease 

Fabry disease, also called Anderson-Fabry disease, is an inherited lysosomal storage disease based on an X-linked genetic pattern.

Since it shows up in childhood as acroparesthesias, a condition characterized by numbness, prickling, tingling, or burning sensations in hands and feet, the symptoms are nonspecific. Consequently, many physicians fail to recognize this incipient symptomatology as a precursor to the more serious symptoms that will later appear to suggest Fabry disease.

Only later in adulthood, when the disease affects vital organs, such as the heart and kidney, are doctors likely to correctly diagnose the disease. 

All childhood and late adulthood symptoms arise because of the deficient function of the lysosomal α‑Galactosidase‑A enzyme. Since this enzyme does not work, it does not break down globotriaosylceramide (Gb3).

This, in turn, results in the progressive accumulation of Gb3 fatty substance deposits throughout the body. The abnormal storage of Gb3 in the valvular fibroblasts, conduction system cells, and cardiomyocyte cells of the heart, the endothelial cells in blood vessels, and vascular smooth muscle cells, causes a wild array of symptoms. 

Over time, Gb3 deposits occlude the interior of blood vessels, decrease blood flow, and cause pain and organ failure, particularly in vital organs like the heart and kidneys. 

In this chaotic scene, pegunigalsidase alfa (PRX-102) is analogous to a hero on a white horse riding into a kingdom in disarray and rescuing its hapless denizens. Protalix BioTherapeutics scientists have chemically modified a specific plant cell to mimic a stable version of α‑Galactosidase‑A enzyme to break down GB3 accumulations throughout the body. In clinical studies, this artificial enzyme has a half-life of about 80 hours in the blood circulation. 

Protalix BioTherapeutics’ Developmental Pipeline 

The U.S. Food and Drug Administration (FDA) approved Protalix BioTherapeutics as the first biopharmaceutical company to create recombinant therapeutic proteins through a proprietary plant-cell-based expression system. ProCellEx® is an entirely novel method for the development of recombinant proteins on an industrial scale. 

Pegunigalsidase alfa (PRX-102) has the honor of being the first product manufactured through ProCellEx®. The FDA approved PRX-102 for marketing in May 2012. Since then many other regulatory authorities around the world have also approved it. 

Protalix BioTherapeutics has licensed the worldwide development, production and commercial rights for PRX-102 to Pfizer (except for Brazil where Protalix has established full rights). It has also partnered with Chiesi Farmaceutici S.p.A for the development, production and commercial rights for PRX-102 within and outside the United States. 

Protalix BioTherapeutics has several other clinically superior versions of proprietary recombinant therapeutic proteins for a range of established pharmaceutical markets. 

Besides pegunigalsidase alfa, also known as PRX-102, which is a modified version of the recombinant α‑Galactosidase‑A protein that is used to treat Fabry disease, the company also has OPRX-106, an anti-inflammatory treatment for oral delivery, and alidornase alfa, a cystic fibrosis treatment, in its developmental pipeline. 

Summary

The American biopharmaceutical company, Protalix BioTherapeutics, Inc., has developed several recombinant therapeutic proteins made by its proprietary technology, ProCellEx®, which is a plant cell-based expression system. 

One of its most promising product candidates has been the production of pegunigalsidase alfa (PRX-102). After FDA approval, the company successfully ran a clinical study on patients who had been on a drug called Replagal®, which provided them with the benefits of agalsidase alfa, recombinant formulation of human hydrolase a-galactosidase A (AGAL). 

PRX-102 replaces the function of the lysosomal α‑Galactosidase‑A enzyme, which is not active in people with Fabry disease. As a result, globotriaosylceramide (Gb3) progressively accumulates throughout a person’s body, and the abnormal deposits of Gb3 in the valvular fibroblasts, conduction system cells, and cardiomyocytes cells of the heart, the endothelial cells in blood vessels, and vascular smooth muscle cells manifest as a wide range of symptoms that lead to a variety of health issues.

Some issues compromise organs like the eyes, which leads to cloudy vision, and the ears, which leads to ringing in the ears as well hearing loss. Other issues are life-threatening, such as heart or heart or kidney failure.

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