Date: 2017-04-18
Type of
information: Results
phase: preclinical
Announcement: results
Company: Protalix BioTherapeutics (Israel)
Product: pegunigalsidase alfa (PRX-102 - alpha-galactosidase-A)
Action
mechanism: enzyme replacement therapy. PRX-102 is the Company's proprietary plant cell-expressed, chemically modified recombinant alpha-galactosidase-A enzyme in development as a long-term enzyme replacement therapy (ERT) for the treatment of Fabry disease.
Disease: Fabry disease
Therapeutic
area: Rare diseases - Genetic diseases
Country:
Trial
details:
Latest
news:
- • On April 18, 2017, Protalix BioTherapeutics announced new results from a preclinical trial conducted in collaboration with Prof. Raphael Schiffmann, Director, Institute of Metabolic Disease at the Baylor Research Institute, Dallas, Texas. It was demonstrated that treatment of Fabry mice with pegunigalsidase alfa (PRX-102) slows the progression of small fiber neuropathy when compared to treatment of Fabry mice with currently approved enzyme replacement therapies, and when compared to untreated Fabry mice.
- The preclinical study evaluated 1mg/kg pegunigalsidase alfa compared to Fabrazyme® and Replagal® at the approved clinical doses (1, 0.2 mg/kg, respectively) in Fabry mice, with untreated Fabry mice and wild type (healthy) mice as controls. Bi-weekly intravenous infusions were administered for 3 months.
- Fabry mice treated with pegunigalsidase alfa showed a 53% reduction (p<0.05) in the number of Iba1 spots, a marker for inflammation of the peripheral sensory nerves system, compared to untreated Fabry controls. Levels of Iba1 in the pegunigalsidase alfa-treated mice reached the levels of the healthy mice. In contrast, there was no difference in the Iba1 marker in Fabry mice treated with Replagal® or Fabrazyme®, compared to untreated Fabry mice.
- Additionally, the sensitivity of the nervous system was assessed using the well-established hot plate test, which measures the effect of the enzyme treatment on response time. Pegunigalsidase alfa demonstrated effectiveness in attenuation of small fiber neuropathy progression. “
Is
general: Yes
