Protalix BioTherapeutics and Chiesi Global Rare Diseases Present Key Clinical Data of Pegunigalsidase Alfa for the Treatment of Fabry Disease at the 17th Annual WORLDSymposium™ 2021

Phase III BRIDGE open-label, switch-over clinical trial met key objectives for safety and efficacy

Mean overall annualized change in estimated Glomerular Filtration Rate (eGFR slope) improved from -5.9 to -1.2 mL/min/1.73 m2/year

12-months on-treatment Phase III BRIDGE study final results suggest a potential benefit of pegunigalsidase alfa on renal function for Fabry patients who were previously treated with agalsidase alfa

The number of patients with moderately progressing or fast progressing kidney disease decreased and the majority of patients achieved a stable kidney disease status post-switch

Compared with baseline, substantial improvements in plasma lyso-Gb3 levels were observed after 12 months of treatment in male patients and levels improved or remained stable throughout the study in female patients

CARMIEL, Israel, and BOSTON, MA, USA I February 10, 2021 I Protalix BioTherapeutics, Inc. (NYSE American: PLX) (TASE: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx® plant cell-based protein expression system, and Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., an international research-focused healthcare Group (Chiesi Group), today announced that final results from the Phase III BRIDGE clinical trial (NCT03018730) of pegunigalsidase alfa (PRX–102), an investigational therapy in development for the potential treatment of Fabry disease, will be presented at the 17th Annual WORLDSymposium 2021, a research conference dedicated to lysosomal diseases being held virtually February 8–12, 2021.

The final results, which were first announced in December 2020, will be presented in both an oral and a poster presentation by Prof. Ales Linhart, MD, Charles University, Praha, Czech Republic, a principal investigator in the Phase III clinical trials of PRX–102 for the potential treatment of Fabry disease. The Company has already announced the schedule of the presentations.

The BRIDGE clinical trial was a Phase III 12 month open label, single arm switch over study evaluating the safety and efficacy of pegunigalsidase alfa, 1 mg/kg infused every two weeks, in up to 22 Fabry patients previously treated with agalsidase alfa, marketed by Takeda Pharmaceutical Company Limited (formerly Shire Plc) as Replagal®, for at least two years and on a stable dose for at least six months. Replagal is not approved in the United States.

The data to be presented by Prof. Linhart showed substantial improvement in renal function as measured by mean annualized estimated Glomerular Filtration Rate (eGFR slope) in both male and female patients who were switched from agalsidase alfa to pegunigalsidase alfa.

Twenty of twenty-two patients completed the 12-month treatment duration, 18 of which opted to roll over to a long-term extension study and continue to be treated with pegunigalsidase alfa.

In the BRIDGE study, the mean annualized eGFR slope of the study participants improved from -5.90 mL/min/1.73m2/year while on agalsidase alfa to -1.19 mL/min/1.73m2/year on PRX-102 in all patients. Male patients improved from -6.36 mL/min/1.73m2/year to –1.73 mL/min/1.73m2/year and female patients improved from -5.03 mL/min/1.73m2/year to –0.21 mL/min/1.73m2/year. Following the switch to pegunigalsidase alfa there was a decrease in patients with progressing or fast progressing kidney disease, and the majority of patients achieved a stable status post-switch.

An immunogenicity assessment indicated that four out of 20 patients (20%) developed persistent antidrug antibodies over the course of the study, of which two had neutralizing activity.

Baseline characteristics of the 20 patients that completed the study, ranging from ages 28 to 60 years, were as follows: mean eGFR 75.87 mL/min/1.73m2 in males, and 86.14 mL/min/1.73m2 in females and plasma lyso-Gb3 mean levels were 51.81 nM and 13.81 nM in males and females, respectively. While lyso-Gb3 levels remain slightly high, particularly within the male cohort, continuous reduction in lyso-Gb3 levels was observed of 19.55 nM (32.35%) in males and 4.57 nM (29.81%) in females.

"The final analysis of the BRIDGE Study in Fabry patients previously treated with agalsidase alfa demonstrates a potential benefit of pegunigalsidase alfa on renal function," said Prof. Linhart.

Pegunigalsidase alfa was well tolerated in the study, with all adverse events being transient in nature without sequelae. Among the 22 patients enrolled in the BRIDGE study, the majority of treatment emergent adverse events were mild or moderate in severity (96.9%), with two patients (9.1%) withdrawing from the therapy due to hypersensitivity reaction that was resolved. The most common moderate treatment emergent adverse events were nasopharyngitis, headache and dyspnea.

Additional details can be found on the WORLDSymposium website at Copies of the presentation materials will be made available on Protalix's website under the Presentations tab in the Investors section at the time of the poster session.

About Fabry Disease

Fabry disease is an X-linked inherited disease that results from deficient activity of the lysosomal α-Galactosidase-A enzyme resulting in progressive accumulation of abnormal deposits of a fatty substance called globotriaosylceramide (Gb3) in blood vessel walls throughout a person's body.

Fabry disease occurs in one person per 40,000 to 60,000. Fabry patients inherit a deficiency of the α-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 and in the blood vessel walls. 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 Pegunigalsidase Alfa

Pegunigalsidase alfa (PRX-102) is an investigational, plant cell culture-expressed, and chemically modified stabilized version of the recombinant α-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. PRX–102 has been designed to potentially address the continued unmet clinical need in Fabry patients.

About Chiesi Global Rare Diseases

Chiesi Global Rare Diseases is a business unit of the Chiesi Group established in February 2020 and focused on research and development of treatments for rare and ultra-rare disorders. The Global Rare Diseases unit works in collaboration with Chiesi Group to harness the full resources and capabilities of our global network to bring innovative new treatment options to people living with rare diseases, many of whom have limited or no treatments available. The unit is also a dedicated partner with global leaders in patient advocacy, research and patient care. For more information visit

About Chiesi Group

Based in Parma, Italy, Chiesi Farmaceutici is an international research-focused healthcare group with 85 years of experience in the pharmaceutical industry and a global presence in 29 countries. Chiesi researches, develops, and markets innovative drugs in the respiratory therapeutics, specialist medicine, and rare disease areas. Its R&D organization is headquartered in Parma (Italy), and is integrated with R&D groups in France, the USA, the UK, and Sweden to advance Chiesi's pre-clinical, clinical, and registration programs. Chiesi employs nearly 6,000 people.

Chiesi Group is a certified Benefit corporation. For more information, please visit

About Protalix BioTherapeutics, Inc.

Protalix is a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx®. Protalix was the first company to gain FDA approval of a protein produced through plant cell-based in suspension expression system. Protalix's unique expression system represents a new method for developing recombinant proteins in an industrial-scale manner.

Protalix's first product manufactured by ProCellEx, taliglucerase alfa, was approved for marketing by the U.S. Food and Drug Administration in May 2012 and, subsequently, by the regulatory authorities of other countries. Protalix has licensed to Pfizer Inc. the worldwide development and commercialization rights for taliglucerase alfa, excluding Brazil, where Protalix retains full rights.

Protalix's development pipeline consists of proprietary versions of recombinant therapeutic proteins that target established pharmaceutical markets, including the following product candidates: pegunigalsidase alfa, a modified version of the recombinant human α–Galactosidase–A protein for the proposed treatment of Fabry disease; OPRX–106, an orally- delivered anti-inflammatory treatment; alidornase alfa for the treatment of Cystic Fibrosis; and others. Protalix has partnered with Chiesi Global Rare Diseases, both in the United States and outside the United States, for the development and commercialization of pegunigalsidase alfa.

SOURCE: Protalix BioTherapeutics

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