Pegunigalsidase alfa (PRX-102)

Pegunigalsidase alfa (PRX-102)
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Pegunigalsidase alfa (PRX-102) for the treatment of Fabry Disease

About Fabry Disease

Fabry disease is an X-linked inherited disease that results from abnormal deposits of a fatty substance called globotriaosylceramide in blood vessel walls throughout the body. It occurs in one person per 40,000. Patients with Fabry disease inherit a deficiency of the enzyme alpha-galactosidase-A, which is normally responsible for the breakdown of globotriaosylceramide. The abnormal storage of globotriaosylceramide increases with time and so it accumulates, primarily in the blood and in the blood vessel walls. The channels of blood vessels narrow, leading to decreased blood flow and decreased tissue nourishment. The ultimate consequences of globotriaosylceramide deposition range from episodes of pain and impaired peripheral sensation to end-organ failure—particularly of the kidneys, but also of the heart and cerebrovascular system.
Fabry Disease is generally treated by enzyme replacement therapy (ERT), meaning replacing the missing alpha-Galactosidase-A enzyme with a recombinant protein via intravenous infusion once every two weeks.

About Pegunigalsidase alfa

Pegunigalsidase alfa is a plant cell culture expressed and a chemically modified version of the recombinant alpha-Galactosidase-A protein. Protein sub-units are covalently bound via chemical cross-linking using PEG chains, resulting in a more active and stable molecule than the current available versions. PRX 102 demonstrated enhanced circulatory half-life, with higher enzyme activity in target organs affected by Fabry disease.

Protalix believes that this may potentially lead to a better clinical efficacy and an improved safety profile compared to currently available treatments.

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Clinical data from Phase I/II Study

 

 

14 days of active enzyme

In clinical trials, pegunigalsidase alfa has been shown to be active in the plasma for the entire two weeks between infusions

This level of enzyme activity in the blood is substantially higher than that of currently available enzyme replacement therapies.

Circulatory Half-Life

Pegunigalsidase alfa has demonstrated a half-life of approximately 80 hours. It is a more stable molecule with a significantly longer circulatory half-life than the currently marketed enzyme replacement therapies.

Positive Impact on Kidney Function

Results of the Phase I/II study  with pegunigalsidase alfa showed a stability in eGFR levels of the patients from baseline (BL) through 6 months and 12 months of treatment with the drug. eGFR (estimated glomerular filtration rate) measures level of kidney function and determines stage of kidney disease and function. Consistent eGFR levels means that the kidney function does not deteriorate.

*Germaine et al; 2015

Favorable safety and tolerability

Throughout the Phase I/II trial 417 infusions of pegunigalsidase alfa were administered. This  amounts to approximately 16 patient years.

Pegunigalsidase alfa was found to be  well tolerated, with 98% of events being mild and moderate

Pegunigalsidase alfa demonstrated lower immunogenicity: Only three patients (19%) tested positive for treatment-induced anti-drug antibodies.

Lower immunogenicity means that the drug provokes a lower immune response. As such, patients may potentially experience less side effects

*Fabrazyme prescribing information

POSITIVE IMPACT ON Cardiac FUNCTION

Results of the Phase I/II study with pegunigalsidase alfa showed stability in key parameters relating to cardiac function.

MEANINGFUL REDUCTION IN Pain

Results of the Phase I/II study showed that patients experienced less pain while being treated with pegunigalsidase alfa.

Pivotal Studies

In 2016, we have commenced two pivotal trials to support registration of pegunigalsidase alfa.

The BALANCE Study is a randomized, double blind, active control study of pegunigalsidase alfa (PRX-102) compared to Fabrazyme® in Fabry patients previously treated with Fabrazyme®. The objective of the study is to demonstrate superiority of pegunigalsidase alfa (PRX-102) in renal function by comparison of the eGFR slope (mean annualized change) between treatment groups.

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The BRIDGE study is an open label, single arm switch-over study to assess the efficacy and safety of pegunigalsidase alfa (PRX-102) in Fabry patients currently treated with Replagal®.

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BALANCE Study

You may be eligible to enter our clinical study with our new enzyme replacement therapy for Fabry disease.

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