Benzinga | Dec 22, 2021 06:53AM EST

Protalix BioTherapeutics Issues 2021 Letter To Stockholders

Protalix BioTherapeutics, Inc. (NYSE:PLX) (TASE: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx(r) plant cell-based protein expression system, today announced the following letter from its President and Chief Executive Officer, Dror Bashan, to its stockholders and the investment community.

Dear Protalix Stockholders,

As we look back and reflect on this past year, I am proud of our team and of the progress we have made in a time of continued Covid-19-related uncertainty across all corners of the globe. While the pandemic has added, and continues to add, many challenges that are out of our control, we remain resolute and focused on that which was within our control, including our operational, clinical and regulatory work. We continue to make progress on all fronts and firmly believe that we are on the right path forward towards meaningful, value-adding milestones and transformational catalysts.

I am pleased to briefly highlight some of our more notable milestones achieved in 2021.

Regulatory Advancements

Together with our development and commercialization partner, Chiesi Farmaceutici S.p.A., we completed a Type A meeting in October with the U.S. Food and Drug Administration (FDA) for the biologics license application (BLA) for PRX-102 for the treatment of adult patients with Fabry disease. We gained clarity regarding the FDA's expectations and confirmed our pathway for resubmission of a PRX-102 BLA, planned for the second half of 2022.

In addition, we have made important progress in our pre-submission activities toward an anticipated Marketing Authorization Application (MAA) with the European Medicines Agency (EMA). In our October meeting with the Rapporteur and Co-Rapporteur of the EMA, we and Chiesi discussed the MAA scope and gained general support for the anticipated PRX--102 MAA submission planned for the first quarter of 2022.

Clinical Advancements

The last patient in our Phase III BALANCE clinical trial, a 24-month, randomized, double blind, active control study of PRX-102 in Fabry patients with impaired renal function, received the final dose in October 2021. We anticipate releasing un-blinded final data in the second quarter of 2022 after all remaining patients have completed the 24-month treatment period. The determination to plan the PRX-102 MAA submission for the first quarter of 2022 was based on the interim analysis of the 12-month data generated from the BALANCE study, which were released in June 2021, in combination with previously reported positive data from our Phase III BRIGHT and BRIDGE clinical trials.

Earlier in the year, we announced positive topline results from our Phase III BRIGHT clinical trial, a 12-month, open-label, switch-over study designed to evaluate the safety, efficacy and pharmacokinetics of PRX--102 via intravenous (IV) infusions of 2 mg/kg administered every 4 weeks in Fabry patients previously treated with a commercially available enzyme replacement therapy (ERT). Topline results of the BRIGHT study indicate that 2 mg/kg of PRX-102 administered by IV infusion every 4 weeks are well tolerated among treated patients, and stable clinical presentation was maintained in adult Fabry patients.

Corporate & Financial Developments

Earlier this fall, we strengthened our balance sheet through exchanges of a substantial majority of our outstanding 7.50% Senior Secured Convertible Notes due 2021 for a combination of cash and new notes. In brief, we issued new 7.50% Senior Secured Convertible Notes due 2024 with an aggregate of $54.65 million principal amount and made principal and interest payments of approximately $27.00 million. The remaining 2021 Notes were repaid on the November 2021 maturity date. Over the course of the year, we raised almost $50.00 million in new equity, and we have an At-the-Market offering program in place should we find market conditions favorable for additional financing. These transactions secured our balance sheet, and we are gratified to be in a solid financial position to support our plans and strategies through our important, value-enhancing milestones.

Looking Forward

We have much to be thankful for this year, including our progress towards our BLA resubmission in the United States; important clarity on an MAA submission in Europe; positive data and progress across all of our clinical trials and a strengthened balance sheet. Going into the new year, we look forward with great anticipation to continuing our progress in advancing a potential good alternative for the Fabry community, and thank our employees for their tireless efforts in driving Protalix forward and building on our scientific foundation. We also take this opportunity to express our appreciation for the close collaboration we have with Chiesi, our commercial partner. Finally, we would like to thank you, our stockholders, for your support and look forward to sharing our successes with you.

Sincerely,

Dror Bashan, President & Chief Executive Officer