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Since Our Last Update
Since our third quarter update, Protalix Biotherapeutics, Inc. (NYSE:PLX) issued a 2021 Letter to Stockholders, presented two posters at 18th Annual WORLD Symposium 2022, and submitted an MAA for PRX-102.
Highlights since our last update include:
➢ 2021 Letter to Stockholders - December 2022
➢ Two poster presentations at 18th Annual WORLD Symposium 2022 - February 2022
➢ MAA submitted - February 2022
MAA Submission
On February 24, 2022, Protalix and partner Chiesi Global Rare Disease (Chiesi) announced the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for PRX-102 in treatment of adults with Fabry disease. The application was validated, confirming that all essential elements required for scientific assessment were included in the application. The MAA included data from both PRX-102's completed and ongoing trials and trial extensions: 12-month interim data from BALANCE, data from BRIDGE and BRIGHT, Phase I/II trial data in naïve/untreated patients, and from extension studies using bi-weekly 1 mg/kg dosing. If the MAA is approved, PRX-102 will be marketable in all member nations of the European Union. Scientific evaluation of the application will be conducted by the Committee for Medicinal Products for Human Use (CHMP). Upon review completion, the CHMP will issue an opinion on whether PRX-102 may be authorized. This opinion is then expected to be adopted, and the application approved, by the European Commission. Altogether, the scientific evaluation and EC decision are expected to take up to 210 and 67 days, respectively. This timeline does not include any clock stops required to respond to questions which could extend the decision by a few months and the practical duration for approval can range anywhere from 12 – 15 months. Assuming no unforeseen delays, a decision is estimated to occur in 1H:23.
2021 Letter to Stockholders
Protalix CEO, Dror Bashan, issued a 2021 Letter to Stockholders on December 22, 2021. In the Letter, Bashan highlighted Protalix' 2021 milestones. On the regulatory front, Protalix and partner Chiesi completed a Type A meeting in October with the FDA for the BLA for PRX-102. With FDA guidance, the BLA resubmission is now planned for 2H:22. Progress was made toward a Marketing Authorization Application with the EMA. On the clinical front, the last patient in the Phase III BALANCE trial received their final dose of PRX-102 in October 2021. Data for BALANCE is slated to be released in 2Q:22. Topline results were also announced for Phase III BRIGHT, supporting a less-frequent dosing scheme of 2mg/kg IV infusion every four weeks, relieving patient burden. Finally, Protalix' 7.50% Senior Secured Convertible Notes due 2021 were exchanged for a combination of cash and new notes. All 2021 Notes are now extinguished, and Protalix raised almost $50 million in new equity. Protalix also has an ATM offering program in place. The CEO concluded with optimistic remarks about Protalix' 2022 outlook.
Below is our estimate of the timeline for the FDA resubmission and related events:
➢ Type A Meeting - September 2021
➢ Type A Meeting Notes - October 2021
➢ Last patient completion of BALANCE trial - October 2021
➢ Availability of full data package for BALANCE - 1H:22
➢ Resubmission of BLA for PRX-102 - 2H:22
➢ On-site inspection of drug substance and/or fill and finish facilities - 2H:22
➢ Completion of 6-month resubmission review - late 2022/early 2023
➢ PRX-102 commercialization - 2023
Recent Milestones
➢ Request Type A meeting regarding PRX-102 - August 2021
➢ Attend Type A meeting regarding PRX-102 - September 2021
➢ Final dosing of last patient in BALANCE trial - October 2021
➢ EMA submission of PRX-102 - February 2022
➢ BALANCE final unblinded data release - 2Q:22
➢ Resubmission of BLA for PRX-102 - 2H:22
➢ On-site inspection of drug substance and/or fill and finish facilities - 2H:22
➢ Completion of FDA 6-month resubmission review - year-end 2022
➢ EMA approval and EU commercialization of PRX-102 - 1H:23
➢ PRX-102 commercialization - 2023
MAA Submission Timeline
Below we provide a summary of the key steps in the MAA process. Protalix will be pursuing approval via the Centralized Procedure, which is one of four routes available for drug approval in the EU.
➢ Submission of application on day 0
➢ Application assessment – multiple committees & provide scientific opinion
➢ Questions for application developed and returned to sponsor
➢ Clock stop on day 120 - applicant prepares responses
➢ Applicant submits responses
➢ Joint assessment report
➢ Clock stop on day 180 – applicant addresses additional questions/outstanding issues (if needed)
➢ Applicant submits responses
➢ Adoption of opinion on day 210
➢ Commission Decision on day 277
Summary
Since our third quarter update, Protalix has issued a 2021 Letter to Stockholders, presented two posters at 18th Annual WORLD Symposium 2022 and submitted an MAA for PRX-102. We anticipate final data from BALANCE in the second quarter, and resubmission of PRX-102's BLA in 2H:22. After the setback of the FDA's CRL in April 2021, Protalix returns to the regulatory application process with lead candidate PRX-102. Compared with market leader Fabrazyme, PRX-102 represents enhanced longevity in the body through its unique enzyme surface pegylation, which suggests greater efficacy and decreased burden for Fabry patients. With the MAA submission and BLA resubmission expected this year, Protalix could begin commercializing in both EU and US markets in 2023.
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1. Source: Protalix Third Quarter 2021 Form 10-Q
2. Centralised Procedure Process Timetable. Asphalion.
