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Chiesi Global Rare Diseases and Protalix BioTherapeutics Receive Positive CHMP Opinion for Pegunigalsidase Alfa for Treatment of Fabry Disease

https://finance.yahoo.com/news/chiesi-global-rare-diseases-protalix-141500734.html

- European Commission decision anticipated in beginning of May 2023 -

PARMA, Italy and BOSTON and CARMIEL, Israel, Feb. 24, 2023 /PRNewswire/ -- Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people affected by rare diseases, and Protalix BioTherapeutics, Inc. (NYSE American:PLX) (TASE:PLX), a biopharmaceutical company, announced today that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending marketing authorization for PRX–102 (pegunigalsidase alfa), the first and only pegylated enzyme for the treatment of adult patients with Fabry disease.

"Chiesi and our partners at Protalix are deeply committed to people living with Fabry disease and their families, many of whom experience unmet medical needs," said Giacomo Chiesi, Head of Chiesi Global Rare Diseases. "Our deepest gratitude to all the individuals with Fabry disease who have participated in clinical trials. Thanks to them, PRX–102 has been extensively studied during the clinical development program, providing the data for the CHMP's evaluation and positive opinion regarding a positive benefit-risk profile for PRX–102. We look forward to advancing towards approval and launch in Europe and will continue our mission to deliver this potential new treatment option to people living with Fabry disease around the world."

PRX–102 is a novel recombinant human a–Galactosidase–A (a–Gal–A) enzyme being investigated as an enzyme replacement therapy (ERT) for the treatment of Fabry disease. The positive CHMP opinion was based on a marketing authorization application (MAA) that includes positive data from a comprehensive set of preclinical, clinical and manufacturing studies evaluating PRX–102. The clinical development program includes the completed Phase 3 BALANCE, BRIDGE, and BRIGHT clinical trials, the Phase 1/2 clinical trial, and ongoing related extension studies that combined represent more than 400 years of exposure to PRX–102. PRX–102 has been studied in more than 140 patients, consisting of both ERT–naïve and ERT–experienced patients, and includes a head–to–head trial versus agalsidase beta.

"We are pleased to be another step closer to approval in Europe with the CHMP's positive opinion recommending marketing authorization for PRX–102 for adult patients with Fabry Disease," said Dror Bashan, Protalix's President and Chief Executive Officer. "We believe that this recommendation further recognizes the strength of the positive dataset from our robust clinical trial program and underscores the potential for PRX–102 to provide a new treatment option for patients with Fabry disease. Data from our clinical program indicates that PRX–102 has the potential to be a long lasting therapy with a favorable tolerability and immunogenicity profile. Together with Chiesi, we remain committed to bringing PRX–102 to market and working to potentially improve the quality of life of patients with Fabry disease. We thank the study personnel for their dedication and look forward to the final European Commission decision on the MAA."

The CHMP opinion is now referred for final action to the European Commission (EC). A final EC decision on the MAA is expected in the beginning of May 2023.

About Fabry Disease
Fabry disease is an X–linked inherited disease that results from deficient activity of the lysosomal a–Galactosidase–A enzyme resulting in progressive accumulation of abnormal deposits of a fatty substance called globotriaosylceramide (Gb3) in the lysosomes throughout a person's body. Fabry disease occurs in one person per 40,000 to 60,000. Fabry patients inherit a deficiency of the a–Galactosidase–A enzyme, which is normally responsible for the breakdown of Gb3. The abnormal storage of Gb3 increases with time and, accordingly, Gb3 accumulates, primarily in the blood vessel and tissues. The ultimate consequences of Gb3 deposition range from episodes of pain and impaired peripheral sensation to end-organ failure – particularly of the kidneys, but also of the heart and the cerebrovascular system.

About PRX–102
PRX–102 (pegunigalsidase alfa) is an investigational, novel, PEGylated enzyme replacement therapy (ERT) under development to treat unmet medical needs for Fabry patients, such as progressive kidney decline. PRX-102 is a plant cell culture-expressed, and chemically modified stabilized version of the recombinant a–Galactosidase–A enzyme. Protein sub-units are covalently bound via chemical cross-linking using short PEG moieties, resulting in a molecule with unique pharmacokinetic parameters. In clinical studies, PRX–102 has been observed to have a circulatory half-life of approximately 80 hours.

About Chiesi Global Rare Diseases
Chiesi Global Rare Diseases is a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people affected by rare diseases. As a family business, Chiesi Group strives to create a world where it is common to have a therapy for all diseases and acts as a force for good, for society and the planet. The goal of the Global Rare Diseases unit is to ensure equal access so as many people as possible can experience their most fulfilling life. The unit collaborates with the rare disease community around the globe to bring voice to underserved people in the health care system. For more information visit www.chiesirarediseases.com.