Protalix Biotherapeutics Inc (PLX) Receives Clearance of IND Application from the FDA for PRX-102


Protalix Biotherapeutics Inc (NYSEMKT:PLX) 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.  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).

Pegunigalsidase alfa with a 2 mg/kg was found to be safe and well tolerated with no formation of antibodies in the Company’s phase I/II clinical trial of pegunigalsidase alfa for the treatment of Fabry disease.  Additionally, in the phase I/II clinical trial, 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 (PK) 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.

Shares of Protalix are up nearly 4% to $0.96 in pre-market trading Tuesday. PLX has a 1-year high of $1.51 and a 1-year low of $0.26. The stock’s 50-day moving average is $1.10 and its 200-day moving average is $0.76.

Protalix Biotherapeutics, Inc. engages in the development and commercialization of recombinant therapeutic proteins based on ProCellEx plant cell based expression system. It offers Taliglucerase alfa injections under the brand name Elelyso, an enzyme replacement therapy for Gaucher disease. Its drug candidates also include Oral Glucocerebrosidase for potential treatment of Gaucher disease; therapeutic proteins for Fabry disease, and immune diseases such as rheumatoid, psoriatic and juvenile idiopathic arthritis, ankylosing, spondylitis, and plaque psoriasis; and protein for biodefense and other indications.