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Protalix Submits BLA for Fabry Disease Candidate PRX-102

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Protalix BioTherapeutics, Inc.  and its partner Chiesi Global Rare Diseases, a unit of Chiesi, announced that they have submitted a biologics license application ("BLA") to the FDA for pegunigalsidase alfa (PRX-102). The BLA seeks approval for their lead candidate under accelerated approval pathway as a treatment for Fabry disease, a rare inherited genetic lysosomal disorder.

The BLA includes data from a completed phase I/II study on pegunigalsidase alfa, including the related extension study, as well as interim data from the Phase III BRIDGE switch-over study and safety data from ongoing clinical studies of the candidate.

Shares of Protalix increased 3.8% on May 28, following the announcement. In fact, the company’s shares have gained 8.2% so far this year compared with the industry’s growth of 2.4%.

Protalix is a small biotech, focusing on the development of recombinant therapeutic proteins developed using its proprietary ProCellEx plant cell-based protein expression system for treating rare diseases.

The company finalized accelerated approval pathway for pegunigalsidase alfa in December 2019, following its discussion with the FDA.

The BRIDGE study is evaluating pegunigalsidase alfa in patients with Fabry disease who received treatment with Takeda’s (TAK - Free Report) Fabry disease medicine, Replagal (agalsidase alfa) for at least two years and were on a stable dose for at least six months. The company announced positive top-line data from the study earlier this month. Data showed substantial improvement in renal function in patients treated with the candidate compared to patients receiving Replagal.

Moreover, Protalix is also conducting a confirmatory phase III study — BALANCE — evaluating pegunigalsidase alfa in Fabry disease. Data from the study will support continued approval for the candidate, if approved under accelerated approval pathway.

The BALANCE study is designed to evaluate the safety and efficacy of pegunigalsidase alfa compared to Sanofi’s (SNY - Free Report) Fabry disease drug, Fabrazyme (agalsidase beta), on renal function in Fabry patients with progressing kidney disease, who have been previously treated with Replagal.

Zacks Rank & Stock to Consider

Protalix currently carries a Zacks Rank #3 (Hold).

Agenus Inc. (AGEN - Free Report) is a stock to consider from the biotech sector carrying a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (String Buy) stocks here.

Agenus’ loss per share estimates have narrowed from $1.23 to $1.14 for 2020 and from $1.26 to $1.13 for 2021 in the past 30 days. The company delivered a positive earnings surprise in three of the trailing four quarters by 13.95%, on average.

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