Analyst Conference Summary

Biotechnology

conference date: February 27, 2023 @ 5:30 AM Pacific Time
for quarter ending: December 31, 2023 (Q4, fourth quarter 2022)

Forward-looking statements

See Also: Protalix Undervalued on CHMP Recommendation of PRX-102 [Feb. 26, 2023 @ Seeking Alpha]

Overview: Preparing for commercial launches by Chiesi of PRX-102 in Europe and the United States, with approvals possible in May 2023.

Basic data (GAAP):

Revenue was $ million, sequentially from $14.2 million, and up % from $ million year-earlier.

Net income was negative $ million, up sequentially from negative $3.6 million, and up from negative $ million year-earlier.

EPS was negative $, up sequentially from negative $0.07, and up from negative $ year-earlier.

Only 2022 Annual data was released; there is no Q4 specific results at present.

2022 revenue was $62.9 million. Net loss was $6.5 million. EPS loss was $0.22. Ending diluted shares were 29.15 million.

Guidance:

Has a cash runway to the first quarter of 2024.

Conference Highlights:

Dror Bashan, Protalix's CEO, said "We are proud to have had a productive 2022 and start to 2023, culminating in the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) positive opinion recommending marketing authorization for PRX–102 for adult patients with Fabry disease. We completed the pivotal parts of our PRX–102 phase III clinical program this year with the closeout of the BRIGHT and BALANCE trials, forming the basis for regulatory submissions to the EMA and U.S. Food and Drug Administration, both of which were accepted for review. Patients from the various trials remain under treatment with PRX–102 in two different open-label extension studies; 1 mg/kg every two weeks or 2 mg/kg every four weeks. As we move into 2023, we anticipate a significant year for the company, with potential regulatory approvals in both Europe and the United States anticipated in May 2023, as well as continued advancement of our early-stage pipeline." The filing this morning is in relation to an ATM program, is not fundraising (yet).

In February 2023, with partner Chiesi Global Rare Diseases, the CHMP made a possitive recommendation for PRX-102 (pegunigalsidase alfa) for the treatment of adult patients with Fabry disease.The Company and Chiesi submitted an MAA to the EMA for PRX-102 on February 24, 2022. The submission was validated by the EMA. The MAA included final data from the Company's phase III BRIDGE and BRIGHT clinical trials; 12–month interim data from the Company's phase III BALANCE clinical trial; and final data from the Company's phase I/II clinical trial data from naïve/untreated patients, including the extension study related thereto, using 1 mg/kg every other week dosing.

In December 2022 the FDA accepted the BLA for pegunigalsidase alfa and set a PDUFA date of May 9, 2023.

It included the final two year analyses of our phase III BALANCE clinical trial and long-term data from our open-label extension study of in adult patients treated with a 2 mg/kg every four weeks dosage. The initial BLA included a comprehensive set of preclinical, clinical and manufacturing data compiled from our completed phase I/II clinical trial of PRX 102, including the related extension study, interim clinical data from our phase III BRIDGE clinical trial and safety data from our on-going clinical studies of PRX 102 in adult patients receiving 1 mg/kg every two weeks. On April 4, 2022, Protalix, together with Chiesi, had announced positive top-line results from its phase III BALANCE clinical trial, a 24-month, randomized, double-blind, active control study of 1 mg/kg of PRX–102 administered every other week for Fabry disease compared to agalsidase beta in patients previously treated with agalsidase beta for at least one year. CSR is complete as of August 2022. The study met its primary endpoint and demonstrated that PRX-102 was statistically non-inferior to agalsidase beta, as measured by estimated glomerular filtration rate (eGFR) slope. Results showed a favorable tolerability profile for PRX-102, consistent with results from the Company's prior trials. On March 18, 2022, together with Chiesi, we announced positive final results from its phase III BRIGHT clinical trial, multinational open-label, switch-over study designed to evaluate the safety, efficacy and pharmacokinetics of treatment with 2 mg/kg of PRX-102 administered every four weeks for 52 weeks in adult patients previously treated with a commercially available enzyme replacement therapy (Fabrazyme or Replagal). Results of the BRIGHT study indicate that 2 mg/kg of PRX–102 administered by intravenous infusion every four weeks was well tolerated, and Fabry disease assessed by eGFR slope and plasma lyso-Gb3 was stable throughout treatment.

The Fabry market is about $2 billion.

In December 2022 Protalix voluntarily delisted from the Tel Aviv Stock Exchange.

Revenue from sales of taliglucerase alfa for 2022 $16.2 million; from license and R&D services was $47 million.

Has other potential therapies in preclinical development. PRX-115 for refractory gout could enter a Phase 1 clinical trial in Q1 2023. PRX119 still in preclinical work on identifying a potential target.

Cash and equivalents balance ended at $39 million, up sequentially from $21 million. Debt is $28 million in convertible notes due in 2024.

2022 Cost of goods sold was $11 million. R&D (net of grants) expense $38 million. SG&A $11 million. Leaving an operating income of $3 million. Financial expenses $10 million.

Q&A selective summary:

How early could the drug be launched? That is up to Chiesi. It will take a couple of months to finalize reimbursement in Europe and in the U.S.

Physician awareness of PRX-102 in EU? We think there is a high awareness. We have been in the clinic for 9 years.

EU Fabry market size? Chiesi can be more specific, we know it is a high priority for them. $2.1 billion global opportunity.

Ready to launch Phase 1 in PRX-115, will start trials in New Zealand. We will publish the trial protocol. Phase 1 is expected to run until the end of 2023.

Milestones expected? Just the May approvals and 115 launch.

Cash flows? Approval milestone payments should be received within 60 days of the events.

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