Analyst Conference Summary


Protalix Biotherapeutics

conference date: May 4, 2023 @ 5:30 AM Pacific Time
for quarter ending: March 31, 2023 (Q1, first quarter 2023)

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 FDA PDUFA of May 9, 2023.

Basic data (GAAP):

Revenue was $9.6 million, sequentially from $na million, and down 40% from $16.1 million year-earlier.

Net income was negative $3.1 million, up sequentially from negative $na million, and down from negative $2.3 million year-earlier.

EPS was negative $0.05, up sequentially from negative $na, and flat from negative $0.05 year-earlier.


Has a cash runway to the first quarter of 2024.

Conference Highlights:

Dror Bashan, Protalix's CEO, said "While we are awaiting for the European Commission and the U.S. FDA decisions, we and our partner, Chiesi, remain committed to bringing PRX-102 to market and improving the lives of patients with Fabry disease. In addition, we are making progress in our early stage PRX–115 program in severe gout with the initiation of our first-in-human Phase I clinical trial."

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 Q1 2023 $5.1 million; from license and R&D services was $4.5 million.

On March 27, 2023, Protalix announced that the first patient was dosed in a Phase I clinical trial of PRX–115, a recombinant PEGylated uricase product candidate under development as a potential treatment for severe gout. The FIH trial is a double-blind, placebo-controlled, single ascending dose study designed to evaluate the safety, pharmacokinetics, pharmacodynamics and immunogenicity of PRX–115 in approximately 56 patients with elevated uric acid levels (>6.0 mg/dL) and no previous exposure to PEGylated uricase.

PRX-119 still in preclinical work on identifying a potential target.

Cash and equivalents balance ended at $33 million, down sequentially from $39 million. Debt is $28 million in convertible notes due in 2024.

2022 Cost of goods sold was $3 million. R&D (net of grants) expense $6 million. SG&A $3 million. Leaving an operating income of negative $3 million. Taxes $0.2 million. Financial expenses $0.5 million.

Q&A selective summary:

PRX-115, New Zealand? We do benefit from tax benefits there, but the reason for NZ was site expertise.

Secondary endpoints for PRX-115? After safety, we want to see immunogenicity, uric acid reduction, frequency of dosing. There is nothing to show right now.

Next steps on approval? We expect the EU approval before the FDA approval. Chiesi will plan the launch.

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