Clinical Trials

Date: 2017-05-09

Type of information: Clinical trial authorisation

phase: 3

Announcement: clinical trial authorization

Company: Protalix BioTherapeutics (Israel)

Product: pegunigalsidase alfa (PRX-102 - alpha-galactosidase-A)

Action mechanism: enzyme replacement therapy. Pegunigalsidase alfa is a PEGylated, chemically-modified version of the recombinant alpha-galactosidase-A enzyme, in which the protein sub-units are covalently bound via chemical cross-linking using PEG chains, resulting in a longer active and stable molecule compared to currently available enzyme replacement therapies (ERTs).

Disease: Fabry disease

Therapeutic area: Rare diseases - Genetic diseases


Trial details:

Latest news:

  • • On May 9, 2017, Protalix BioTherapeutics announced that the FDA cleared an Investigational New Drug application (IND) for a clinical trial evaluating the safety and efficacy of administering 2 mg/kg of pegunigalsidase alfa (PRX-102) once monthly in Fabry patients. The current dosing regimen for approved enzyme replacement therapies for Fabry disease is once every two weeks.
  • Protalix plans to enroll up to 30 Fabry patients currently treated with an approved enzyme replacement therapy. Participating patients will be switched to 2 mg/kg of pegunigalsidase alfa once-monthly. A safety and efficacy evaluation will occur at twelve months with additional long term follow-up. The company expects to commence this study in the third quarter of 2017.
  • Pegunigalsidase alfa with a 2 mg/kg was found to be safe and well tolerated with no formation of antibodies in a phase I/II clinical trial of pegunigalsidase alfa for the treatment of Fabry disease. Additionally, 2 mg/kg of pegunigalsidase alfa demonstrated approximately a 40 times higher circulatory half-life compared with other enzyme replacement therapies, and, as demonstrated in a Fabry mice model, with materially higher active enzyme reaching target organs affected by Fabry disease. Pharmacokinetic analysis and modeling from the phase I/II clinical trial indicate that pegunigalsidase alfa levels at the second week after infusion remain 10 times higher than published Fabrazyme® levels at the day of infusion. Moreover, the amount of pegunigalsidase alfa in the circulation at weeks three and four, are higher than those of Fabrazyme® during the two-week treatments. These results provide strong rationale for the clinical evaluation of a once-monthly dosing.
Area Under the Curve (AUC) derived from PK data modeling of Fabry patients:
Time (week) pegunigalsidase alfa,* 2 mg/kg IV every 4 weeks (ug•min/mL) Fabrazyme®,** 1 mg/kg IV every 2 weeks (ug•min/mL)
1 1st infusion 59,364 1st infusion ~700
2 7,144 Nil
3 878 2nd infusion ~700
4 108 Nil
* PK modeling based on Phase I/II data    ** Fabrazyme® USPI

Is general: Yes