Protalix BioTherapeutics Inc

Previous wording

You should read the following discussion and analysis of our financial condition and results of operations together with our financial statements and the consolidated financial statements and the related notes included elsewhere in this Form 10-Q and in our Annual Report on Form 10-K for the year ended December 31, 2024. Some of the information contained in this discussion and analysis, particularly with respect to our plans and strategy for our business and related financing, includes forward-looking statements within the meanings of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, or the Exchange Act, including statements regarding expectations, beliefs, intentions or strategies for the future. When used in this report, the terms “anticipate,” “believe,” “estimate,” “expect,” “can,” “continue,” “could,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” and words or phrases of similar import, as they relate to our company, our subsidiary or our management, are intended to identify forward-looking statements. We intend that all forward-looking statements be subject to the safe-harbor provisions of the Private Securities Litigation Reform Act of 1995. These forward-looking statements are only predictions and reflect our views as of the date they are made with respect to future events and financial performance, and we undertake no obligation to update or revise, nor do we have a policy of updating or revising, any forward-looking statement to reflect events or circumstances after the date on which the statement is made or to reflect the occurrence of unanticipated events, except as may be required under applicable law. Forward-looking statements are subject to many risks and uncertainties that could cause our actual results to differ materially from any future results expressed or implied by the forward-looking statements as a result of several factors, including those set forth in this Quarterly Report on Form 10-Q.

Current wording

You should read the following discussion and analysis of our financial condition and results of operations together with our financial statements and the consolidated financial statements and the related notes included elsewhere in this Form 10-Q and in our Annual Report on Form 10-K for the year ended December 31, 2024. Some of the information contained in this discussion and analysis, particularly with respect to our plans and strategy for our business and related financing, includes forward-looking statements within the meanings of Section 27A of the Securities Act of 1933, as amended, or the Securities Act, and Section 21E of the Securities Exchange Act of 1934, as amended, or the Exchange Act, including statements regarding expectations, beliefs, intentions or strategies for the future. When used in this report, the terms “anticipate,” “believe,” “estimate,” “expect,” “can,” “continue,” “could,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” and words or phrases of similar import, as they relate to our company, our subsidiary or our management, are intended to identify forward-looking statements. We intend that all forward-looking statements be subject to the safe-harbor provisions of the Private Securities Litigation Reform Act of 1995. These forward-looking statements are only predictions and reflect our views as of the date they are made with respect to future events and financial performance, and we undertake no obligation to update or revise, nor do we have a policy of updating or revising, any forward-looking statement to reflect events or circumstances after the date on which the statement is made or to reflect the occurrence of unanticipated events, except as may be required under applicable law. Forward-looking statements are subject to many risks and uncertainties that could cause our actual results to differ materially from any future results expressed or implied by the forward-looking statements as a result of several factors, including those set forth in this Quarterly Report on Form 10-Q.

Previous wording

●risks relating to Elfabrio’s market acceptance, competition, reimbursement and regulatory actions, including as a result of the boxed warning contained in the U.S. Food and Drug Administration, or FDA, approval received for the product;

Current wording

●risks relating to Elfabrio’s market acceptance, competition, reimbursement and regulatory actions, including as a result of the boxed warning contained in the FDA approval received for the product;

Previous wording

●risks relating to our ability to manage our relationship with our collaborators, distributors or partners, including, but not limited to, Pfizer Inc., or Pfizer, and Chiesi Farmaceutici S.p.A., or Chiesi;

Current wording

●risks relating to our ability to manage our relationship with our collaborators, distributors or partners, including, but not limited to, Pfizer and Chiesi;

Previous wording

●risks relating to the compliance by Fundação Oswaldo Cruz, or Fiocruz, an arm of the Brazilian Ministry of Health, or the Brazilian MoH, with its purchase obligations under our supply and technology transfer agreement, which may have a material adverse effect on us and may also result in the termination of such agreement;

Current wording

●risks relating to the compliance Fiocruz with its purchase obligations under the Brazil Agreement, which may have a material adverse effect on us and may also result in the termination of such agreement;

Previous wording

To date, we have successfully developed two commercial products, both of which are enzyme replacement therapies (ERTs): Elfabrio® (pegunigalsidase alfa) for the treatment of adult patients with a confirmed diagnosis of Fabry disease and Elelyso® (taliglucerase alfa) for the treatment of adult patients with Gaucher disease. Elelyso was first approved by the FDA in May 2012 and is now approved for marketing in 23 markets including Brazil, Israel and others. Elfabrio, which we referred to as PRX-102 during its development stage, has been approved for marketing in the United States, the European Union, Great Britain, Switzerland, Peru, Israel, Russia and Singapore. We have licensed the rights to commercialize Elelyso worldwide (other than Brazil) to Pfizer, and in Brazil to Fiocruz. Elelyso is marketed as BioManguinhos alfataliglicerase in Brazil. We have partnered with Chiesi for the development and commercialization of Elfabrio.

Current wording

approved by the FDA in May 2012 and is now approved for marketing in 23 markets including Brazil, Israel and others. Elfabrio, which we referred to as PRX-102 during its development stage, has been approved for marketing in the United States, the European Union, Great Britain, Switzerland, Peru, Israel, Russia, Singapore Australia and Taiwan. We have licensed the rights to commercialize Elelyso worldwide (other than Brazil) to Pfizer, and in Brazil to Fiocruz. Elelyso is marketed as BioManguinhos alfataliglicerase in Brazil. We have partnered with Chiesi for the development and commercialization of Elfabrio.

Previous wording

We have entered into two exclusive global licensing and supply agreements for PRX-102 for the treatment of Fabry disease with Chiesi; the Chiesi Ex-US Agreement and the Chiesi US Agreement, collectively, the Chiesi Agreements. Under the agreements,

Current wording

Our partnership with Chiesi is governed by two exclusive global licensing and supply agreements for Elfabrio for the treatment of Fabry disease with Chiesi; the Chiesi Ex-US Agreement and the Chiesi US Agreement, or collectively, the Chiesi Agreements. Under the agreements, we have received certain upfront payments and development cost reimbursements, and remain entitled to potential milestone payments and tiered payments for drug product purchased by Chiesi from Protalix Ltd. See Commercialization of Approved Products--Elfabrio (pegunigalsidase alfa/PRX-102) – Chiesi Farmaceutici.

Previous wording

In addition, we are developing PEGylated uricase, or PRX-115, for the treatment of uncontrolled gout, Long Acting (LA) DNase I, or PRX-119, for the treatment of NETs-related diseases, and a number of other technologies and preclinical assets. We have completed a

Current wording

In addition to Elelyso and Elfabrio, we are developing PEGylated uricase, or PRX-115, for the treatment of uncontrolled gout, Long Acting (LA) DNase I, or PRX-119, for the treatment of NETs-related diseases, and a number of other technologies and preclinical assets. We have completed a phase I First-in-Human clinical trial of PRX-115 and we are currently in the advance stages of preparations for a phase II clinical trial of PRX-115 which we expect to commence in the second half of 2025.

Previous wording

ProCellEx consists of a comprehensive set of proprietary technologies and capabilities, including the use of advanced genetic engineering and plant cell culture technology, enabling us to produce complex, proprietary, and biologically equivalent proteins for a variety of human diseases. Our protein expression system facilitates the creation and selection of high-expressing, genetically-stable cell lines capable of expressing recombinant proteins. The system plays an important role in the execution of our corporate strategy as its allows us to develop and produce tailored complex recombinant therapeutic proteins and to genetically engineer and/or chemically modify such proteins pre- and/or post-production. The engineering and modification of the therapeutic proteins have the potential to provide added clinical benefits by improving the biological characteristics (e.g., glycosylation, half-life, immunogenicity).

Current wording

ProCellEx consists of a comprehensive set of proprietary technologies and capabilities, including the use of advanced genetic engineering and plant cell culture technology, enabling us to produce complex, proprietary, and biologically equivalent proteins for a variety of human diseases. Our protein expression system facilitates the creation and selection of high-expressing, genetically-stable cell lines capable of expressing recombinant proteins. The system plays an important role in the execution of our corporate strategy as it allows us to develop and produce tailored complex recombinant therapeutic proteins and to genetically engineer and/or chemically modify such proteins pre- and/or post-production. The engineering and modification of the therapeutic proteins have the potential to provide added clinical benefits by improving the biological characteristics (e.g., glycosylation, half-life, immunogenicity).

Previous wording

natural capability of agrobacterium to transfer a DNA fragment into the plant chromosome, allowing the genome of the plant cell to code for specific proteins of interest. The agrobacterium-mediated transformed cells are then able to produce specific proteins, which are extracted and purified and can be used as therapies to treat a variety of diseases.

Current wording

We developed ProCellEx based on our plant cell culture technology for the development, expression and manufacture of recombinant proteins that are the essential foundation of modern biotechnology. We develop new, recombinant therapeutic proteins by using the natural capability of agrobacterium to transfer a DNA fragment into the plant chromosome, allowing the genome of the plant cell to code for specific proteins of interest. The agrobacterium-mediated transformed cells are then able to produce specific proteins, which are extracted and purified and can be used as therapies to treat a variety of diseases.

Previous wording

We have two commercial products, each of which is an ERT; Elelyso and Elfabrio. Our pipeline of product candidates includes PEGylated uricase for the treatment of uncontrolled gout, Long Acting (LA) DNase I for the treatment of NETs and other technologies and preclinical assets.

Current wording

We have two commercial products, each of which is an ERT; Elelyso and Elfabrio.

Previous wording

Elelyso (taliglucerase alfa), our first commercial product, was approved by the FDA in 2012 for injection as an ERT for the long-term treatment of adult patients with a confirmed diagnosis of type 1 Gaucher disease. In August 2014, the FDA approved Elelyso for injection for pediatric patients. Elelyso is the first plant cell derived recombinant protein to be approved by the FDA for the treatment of Gaucher disease and is now approved in 23 markets including the United States, Brazil, Israel and others.

Current wording

Elelyso (taliglucerase alfa), our first commercial product, was approved by the FDA in 2012 for injection as an ERT for the long-term treatment of adult patients with a confirmed diagnosis of type 1 Gaucher disease. In August 2014, the FDA approved Elelyso for injection for children four years of age and greater. Elelyso is the first plant cell derived recombinant protein to be approved by the FDA for the treatment of Gaucher disease and is now approved in 23 markets including the United States, Brazil, Israel and others.

Previous wording

The global market for Gaucher disease, that includes Sanofi’s Cerezyme®, Shire’s (acquired by Takeda Pharmaceutical Company Limited) Vpriv® and Sanofi’s Cerdelga®, among others, was $1.7 billion in 2024, is forecasted to be approximately $1.7 billion in 2025 and is forecasted to grow at a compound annual growth rate (CAGR) of approximately -0.46% from 2024-2030.

Current wording

The global market for Gaucher disease, that includes Sanofi’s Cerezyme®, Shire’s (acquired by Takeda Pharmaceutical Company Limited) Vpriv® and Sanofi’s Cerdelga®, among others, was $1.7 billion in 2024, is forecasted to be approximately $1.7 billion in 2025 and is forecasted to grow at a compound annual growth rate (CAGR) of approximately -1.66% from 2024-2030.

Previous wording

Elfabrio, our second commercial product, was approved by the EC for marketing in the EU and by the FDA for marketing in the United States in May 2023 for adult patients with Fabry disease. Both approvals cover the 1 mg/kg every two weeks dosage. According to the EMA, overall, the benefit/risk balance of Elfabrio is positive in the claimed indication (Fabry disease). Similarly, the FDA review team concluded that in the context of Fabry disease as a rare, serious disease with limited therapeutic options that may not be suitable to all individual patients, the benefit-risk of Elfabrio is favorable for the treatment of adults with confirmed Fabry

Current wording

Elfabrio, our second commercial product, was approved by the EC for marketing in the EU and by the FDA for marketing in the United States in May 2023 for adult patients with Fabry disease. Both approvals cover the 1 mg/kg every two weeks dosage. According to the EMA, overall, the benefit/risk balance of Elfabrio is positive in the claimed indication (Fabry disease). Similarly, the FDA review team concluded that in the context of Fabry disease as a rare, serious disease with limited therapeutic options that may not be suitable to all individual patients, the benefit-risk of Elfabrio is favorable for the treatment of adults with confirmed Fabry disease. The FDA noted its determination that substantial evidence of effectiveness for Elfabrio in Fabry patients was established with one adequate and well-controlled study, our phase I/II clinical trial of Fabry disease, with confirmatory evidence provided by the

Previous wording

disease. The FDA noted its determination that substantial evidence of effectiveness for Elfabrio in Fabry patients was established with one adequate and well-controlled study, our phase I/II clinical trial of Fabry disease, with confirmatory evidence provided by theBALANCE study. The FDA review team also concluded that theBALANCE study met its primary efficacy endpoint, which assessed the annualized rate of change in eGFR (estimated glomerular filtration rate) over 104 weeks. However, the FDA also determined that the results from theBALANCE study did not support a non-inferiority claim to the comparator product due to the lack of data to support the non-inferiority margin.

Current wording

BALANCE study. The FDA review team also concluded that theBALANCE study met its primary efficacy endpoint, which assessed the annualized rate of change in eGFR (estimated glomerular filtration rate) over 104 weeks. However, the FDA also determined that the results from theBALANCE study did not support a non-inferiority claim to the comparator product due to the lack of data to support the non-inferiority margin.

Previous wording

Since the approvals by the FDA and the EMA, Elfabrio has been approved for marketing in Great Britain, Switzerland, Peru, Israel, Russia and Singapore for long-term enzyme replacement therapy in adult patients with a confirmed diagnosis of Fabry disease.

Current wording

Since the approvals by the FDA and the EMA, Elfabrio has been approved for marketing in Great Britain, Switzerland, Peru, Israel, Russia, Singapore Australia and Taiwan for long-term enzyme replacement therapy in adult patients with a confirmed diagnosis of Fabry disease.

Previous wording

The global market for Fabry disease, that includes agalsidase beta, Sanofi’s Fabrazyme®, agalsidase alfa, Shire’s Replagal® and Amicus Therapeutics’ Galafold®, among others, is forecasted to be approximately $2.3 billion in 2025 and is forecasted to grow at a CAGR of 6.6% from 2024-2030 reaching approximately $3.1 billion in annual sales in 2030.

Current wording

The global market for Fabry disease, that includes agalsidase beta, Sanofi’s Fabrazyme®, agalsidase alfa, Shire’s Replagal® and Amicus Therapeutics’ Galafold®, among others, is forecasted to be approximately $2.3 billion in 2025 and is forecasted to grow at a CAGR of 7.39% from 2024-2030 reaching approximately $3.2 billion in annual sales in 2030.

Previous wording

We have licensed to Pfizer the global rights to Elelyso in all markets, excluding Brazil, pursuant to the Amended Pfizer Agreement under which Pfizer retains 100% of revenue and reimburses 100% of direct costs. For the first 10-year period after the execution of the Amended Pfizer Agreement, we have agreed to sell drug substance to Pfizer for the production of Elelyso, subject to certain terms and conditions, and Pfizer maintains the right to extend the supply period for up to two additional 30-month periods, subject to certain terms and conditions. In a subsequent amendment, we agreed that after the completion of the first 10-year supply period, the supply term would automatically extend for a five-year period. Any failure to comply with our supply commitments may subject us to substantial financial penalties. The Amended Pfizer Agreement includes customary provisions regarding cooperation for regulatory matters, patent enforcement, termination, indemnification and insurance requirements. We retain distribution rights to taliglucerase alfa in Brazil.

Current wording

We have licensed to Pfizer the global rights to market and sell Elelyso in all markets, excluding Brazil, pursuant to the Amended Pfizer Agreement. For the first 10-year period after the execution of the Amended Pfizer Agreement, we have agreed to sell drug substance to Pfizer for the production of Elelyso for a fixed cost, subject to certain terms and conditions, and Pfizer maintains the right to extend the supply period for up to two additional 30-month periods, subject to certain terms and conditions. In a subsequent amendment, we agreed that after the completion of the first 10-year supply period, the supply term would automatically extend for a five-year period. Any failure to comply with our supply commitments may subject us to substantial financial penalties. The Amended Pfizer Agreement includes customary provisions regarding cooperation for regulatory matters, patent enforcement, termination, indemnification and insurance requirements. We retain distribution rights to taliglucerase alfa in Brazil.

Previous wording

The Brazil Agreement provides for a staged technology transfer which is intended to transfer to Fiocruz the capacity and skills required for the Brazilian government to construct its own manufacturing facility, at its sole expense, and to produce a sustainable, high-quality, and cost-effective supply of BioManguinhos alfataliglicerase. Fiocruz has not satisfied certain purchase commitments under the Brazil Agreement. Accordingly, we and Fiocruz discuss on a continuous basis, potential steps to maximize sales of BioManguinhos alfataliglicerase sales to the Brazilian MoH.

Current wording

The Brazil Agreement provides for a staged technology transfer which is intended to transfer to Fiocruz the capacity and skills required for the Brazilian government to construct its own manufacturing facility, at its sole expense, and to produce a sustainable, high-quality, and cost-effective supply of BioManguinhos alfataliglicerase. Fiocruz has not satisfied certain purchase commitments under the Brazil Agreement. We have continued to sell BioManguinhos alfataliglicerase for a fixed price through purchase orders. We, on a continuous basis, discuss with Fiocruz potential steps to maximize sales of BioManguinhos alfataliglicerase sales to the Brazilian MoH.

Previous wording

Protalix Ltd. has received $50.0 million in upfront payments and development cost reimbursements of $45 million, and is entitled to more than $1.0 billion in potential milestone payments tiered payments for drug product purchased from Protalix Ltd. equal to 15% - 35% (ex-US) and 15% - 40% (US), depending on the amount of annual net sales in the applicable territories.

Current wording

Under the Chiesi Agreements, Protalix Ltd. has received $50.0 million in upfront payments and development cost reimbursements of $45 million, and is entitled to more than $1.0 billion in potential milestone payments and tiered payments for drug product purchased

Previous wording

Under the Chiesi Ex-US Agreement, we granted to Chiesi an exclusive license for all markets outside of the United States to commercialize PRX-102. Chiesi made an upfront payment to Protalix Ltd. of $25.0 million in connection with the execution of the agreement, and Protalix Ltd. was entitled to additional payments of up to $25.0 million in development costs in the aggregate, capped at $10.0 million per year. Protalix Ltd. is also eligible to receive additional payments of up to a maximum of $320.0 million, in the aggregate, in regulatory and commercial milestone payments. Protalix Ltd. agreed to manufacture all of the PRX-102 needed for all purposes under the agreement, subject to certain exceptions, and Chiesi will purchase PRX-102 from Protalix Ltd., subject to certain terms and conditions. Chiesi is required to make tiered payments of 15% to 35% of its net sales, depending on the amount of annual sales, as consideration for the supply of PRX-102.

Current wording

Under the Chiesi Ex-US Agreement, we granted to Chiesi an exclusive license for all markets outside of the United States to commercialize PRX-102. Chiesi made an upfront payment to Protalix Ltd. of $25.0 million in connection with the execution of the agreement, and Protalix Ltd. was entitled to additional payments of up to $25.0 million in development costs in the aggregate, capped at $10.0 million per year. Protalix Ltd. is also eligible to receive additional payments of up to a maximum of $320.0 million, in the aggregate, in regulatory and commercial milestone payments. Protalix Ltd. agreed to manufacture all of the PRX-102 needed for all purposes under the agreement, subject to certain exceptions, and Chiesi will purchase PRX-102 from Protalix Ltd., subject to certain terms and conditions. Chiesi is required to make tiered payments of 15% to 35% of the net sales of the drug product in each applicable territory as consideration for the supply of PRX-102.

Previous wording

The exclusive license to develop and commercialize PRX-102 in the United States were granted to Chiesi under the Chiesi US Agreement. Protalix Ltd. received an upfront, non-refundable, non-creditable payment of $25.0 million from Chiesi and was entitled to additional payments of up to a maximum of $20.0 million to cover development costs for PRX-102, capped at $7.5 million per year. Protalix Ltd. is also eligible to receive additional payments of up to a maximum of $760.0 million, in the aggregate, in regulatory and commercial milestone payments. Chiesi agreed to make tiered payments of 15% to 40% of its net sales under the Chiesi US Agreement to Protalix Ltd., depending on the amount of annual sales, subject to certain terms and conditions, as consideration for product supply.

Current wording

The exclusive license to develop and commercialize PRX-102 in the United States were granted to Chiesi under the Chiesi US Agreement. Protalix Ltd. received an upfront, non-refundable, non-creditable payment of $25.0 million from Chiesi and was entitled to additional payments of up to a maximum of $20.0 million to cover development costs for PRX-102, capped at $7.5 million per year. Protalix Ltd. is also eligible to receive additional payments of up to a maximum of $760.0 million, in the aggregate, in regulatory and commercial milestone payments. Chiesi agreed to make tiered payments of 15% to 40% of the net sales of the drug product in as consideration for product supply.

Previous wording

PRX-115 is our recombinant PEGylated uricase (urate oxidase) – a chemically modified enzyme under development for the potential treatment of patients with uncontrolled gout. The uricase enzyme, which does not exist naturally in humans, converts urate to allantoin, which is easily eliminated through urine. This recombinant enzyme, expressed via our ProCellEx system, is designed to lower urate levels and improve clinical manifestation of the disease while having low immunogenicity and increased half-life of the drug in the blood. Pre-clinical data demonstrates long half-life, reduced immunogenic risk and high specific activity supports the potential of PRX-115 to be a safe and effective treatment for patients with gout. One-month multiple dosing toxicity studies in two animal species and a 6-month multiple dosing toxicity study in one animal species were conducted to support single- and multiple-dose studies in humans.

Current wording

PRX-115 is our recombinant PEGylated uricase (urate oxidase) – a chemically modified enzyme under development for the potential treatment of patients with uncontrolled gout. The uricase enzyme, which does not exist naturally in humans, converts urate to allantoin, which is easily eliminated through urine. This recombinant enzyme, expressed via our ProCellEx system, is designed to lower urate levels and improve clinical manifestation of the disease while having low immunogenicity and increased half-life of the drug in the blood. Pre-clinical data demonstrates long half-life, reduced immunogenic risk and high specific activity supports the potential of PRX-115 to be a safe and effective treatment for patients with gout. One-month multiple dosing toxicity studies in two animal species and a six-month multiple dosing toxicity study in one animal species were conducted to support single- and multiple-dose studies in humans.

Previous wording

In March 2023, we initiated our phase I clinical trial of PRX-115 for the potential treatment of uncontrolled gout entitled “A Double-blind, Placebo-controlled, Single Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Properties of PRX-115 in Adult Volunteers With Elevated Uric Acid Levels” (NCT05745727), or theFIH study. The study was conducted at New Zealand Clinical Research (NZCR) under the New Zealand Medicines and Medical Devices Safety Authority (MedSafe) and the Health and Disability Ethics Committee (HDEC) guidelines. The study was initially designed to include seven sequential dosing cohorts, each composed of eight subjects (six active and two placebo), a 3:1 ratio. Subjects in each cohort, males and females aged 18 through 65, received a single dose of PRX-115 and were analyzed for safety, pharmacokinetics (PK) and pharmacodynamics (PD) (concentrations of plasma urate) for 85 days.

Current wording

We have completed a phase I clinical trial of PRX-115 for the potential treatment of uncontrolled gout entitled “A Double-blind, Placebo-controlled, Single Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Properties of PRX-115 in Adult Volunteers With Elevated Uric Acid Levels” (NCT05745727), or theFIH study. The study was conducted at New Zealand Clinical Research (NZCR) under the New Zealand Medicines and Medical Devices Safety Authority (MedSafe) and the Health and Disability Ethics Committee (HDEC) guidelines. The completed study included eight sequential dosing cohorts, each composed of eight subjects (six active and two placebo), a 3:1 ratio. Subjects in each cohort, males and females aged 18 through 65, received a single dose of PRX-115 and were analyzed for safety, pharmacokinetics (PK) and pharmacodynamics (PD) (concentrations of plasma urate) for 85 days. Overall, 64 randomized subjects were enrolled across the eight cohorts; 48 subjects were treated with PRX–115 and 16 subjects were treated with placebo.

Previous wording

Uncontrolled gout is when serum urate (sUA) levels are above the maximum medically appropriate level (6.8 mg/dL), as well as tophi formation and/or flares that cannot be treated with available urate lowering therapies. Currently available ULTs can be effective in treating gout. However, factors such as low adherence, under dosing, disease progression that cause high patient burden or patients that are not suitable for available therapy, require new, effective and safe therapies to treat these underserved uncontrolled gout patients.

Current wording

Uncontrolled gout is when serum urate (sUA) levels are above the maximum medically appropriate level (6.8 mg/dL), as well as tophi formation and/or flares that cannot be treated with available urate lowering therapies. Currently available ULTs can be effective in treating gout. However, factors such as low adherence, under dosing, disease progression that causes high patient burden or patients that are not suitable for available therapy, require new, effective and safe therapies to treat these underserved uncontrolled gout patients.

Previous wording

A key element of our overall strategy is to establish a broad portfolio of patents to protect our proprietary technology, proprietary product and product candidates and their methods of use. As of March 31, 2025, we hold a broad portfolio of 15 patent families consisting of approximately 67 patents in Europe, the United States, Israel and additional countries worldwide, as well as approximately 42 pending patent applications.

Current wording

A key element of our overall strategy is to establish a broad portfolio of patents to protect our proprietary technology, proprietary product and product candidates and their methods of use. As of June 30, 2025, we hold a broad portfolio of 16 patent families consisting of approximately 68 patents in Europe, the United States, Israel and additional countries worldwide, as well as approximately 42 pending patent applications.

Previous wording

We recorded revenues from selling goods of $10.0 million during the three months ended March 31, 2025, an increase of $6.3 million, or 170%, compared to revenues of $3.7 million for the three months ended March 31, 2024. The increase resulted primarily from an increase of $5.9 million in sales to Pfizer and an increase of $0.4 million in sales to Fiocruz (Brazil).

Current wording

We recorded revenues from selling goods of $15.4 million during the three months ended June 30, 2025, an increase of $2.1 million, or 16%, compared to revenues of $13.3 million for the three months ended June 30, 2024. The increase resulted primarily from an increase of $8.0 million in sales to Chiesi, partially offset by a decrease of $4.7 million in sales to Fiocruz (Brazil) and of $1.2 million in sales to Pfizer.

Previous wording

We recorded revenues from license and R&D services of $0.1 million for the three months ended March 31, 2025 and the three months ended March 31, 2024. Revenues from license and R&D services are comprised primarily of revenues we recognized in connection with the Chiesi Agreements. We expect to generate minimal revenues from license and R&D services other than potential regulatory milestone payments.

Current wording

We recorded revenues from license and R&D services of $0.2 million for the three months ended June 30, 2025, and June 30, 2024. Revenues from license and R&D services are comprised primarily of revenues we recognized in connection with the Chiesi Agreements. We expect to generate minimal revenues from license and R&D services other than potential regulatory milestone payments.

Previous wording

For the three months ended March 31, 2025, our total research and development expenses were approximately $3.5 million comprised of approximately $1.8 million of salary and related expenses, approximately $0.8 million in subcontractor-related expenses, approximately $0.2 million of materials-related expenses and approximately $0.7 million of other expenses. For the three months ended March 31, 2024, our total research and development expenses were approximately $2.9 million comprised of approximately $1.5 million of salary and related expenses, approximately $0.5 million of subcontractor-related expenses, approximately $0.2 million of materials-related expenses and approximately $0.7 million of other expenses.

Current wording

For the three months ended June 30, 2025, our total research and development expenses were approximately $6.0 million comprised of approximately $3.0 million in subcontractor-related expenses, approximately $2.0 million of salary and related expenses, approximately $0.2 million of materials-related expenses and approximately $0.8 million of other expenses. For the three months ended June 30, 2024, our total research and development expenses were approximately $3.0 million comprised of approximately $1.6 million of salary and related expenses, approximately $0.5 million in subcontractor-related expenses, approximately $0.2 million of materials-related expenses and approximately $0.7 million of other expenses.

Previous wording

Total increase in research and developments expenses for the three months ended March 31, 2025 was $0.6 million, or 21%, compared to the three months ended March 31, 2024. The increase in research and development expenses resulted primarily fromthe advance in our clinical pipeline.

Current wording

Total increase in research and developments expenses for the three months ended June 30, 2025 was $3.0 million, or 100%, compared to research and developments expenses of $3.0 million for the three months ended June 30, 2024. The increase in research and development expenses resulted primarily from preparations for the planned phase II clinical trial of PRX-115.

Previous wording

Selling, general and administrative expenses were $2.6 million for the three months ended March 31, 2025, a decrease of $0.5 million, or 16%, compared to $3.1 million for the three months ended March 31, 2024. The decrease resulted primarily from a decrease of $0.4 million in salary and related expenses and a decrease of $0.1 million in selling expenses.

Current wording

Selling, general and administrative expenses were $2.6 million for the three months ended June 30, 2025, a decrease of $0.9 million, or 26%, compared to $3.5 million for the three months ended June 30, 2024. The decrease resulted primarily from a decrease of $0.6 million in salary and related expenses and a decrease of $0.3 million in selling expenses.

Previous wording

We recorded a tax benefit of approximately $(0.1) million for the three months ended March 31, 2025 and March 31, 2024. The tax benefit resulted primarily from deferred taxes on income mainly derived from GILTI income mainly in respect of Section 174 of the TCJA. Effective in 2022, Section 174 of the TCJA requires all U.S. companies, for tax purposes, to capitalize and subsequently amortize R&D expenses that fall within the scope of Section 174 over five years for research activities conducted in the United States and over 15 years for research activities conducted outside of the United States, rather than deducting such costs in the current year.

Current wording

We recorded tax expenses of approximately $0.5 million for the three months ended June 30, 2025, compared to a tax benefit of approximately $(0.1) million for the three months ended June 30, 2024. The tax expenses or benefit resulted primarily from taxes on income mainly derived from GILTI income mainly in respect of Section 174 of the TCJA. Effective in 2022, Section 174 of the TCJA requires all U.S. companies, for tax purposes, to capitalize and subsequently amortize R&D expenses that fall within the scope of Section 174 over five years for research activities conducted in the United States and over 15 years for research activities conducted outside of the United States, rather than deducting such costs in the current year. On July 4, 2025, tax reform legislation was enacted in the United States through the passage of HR1 which includes significant corporate tax changes, including a restoration of the current deductibility for domestic research expenditures beginning in 2025, with transition options for previously capitalized amounts. We continue to evaluate the impact that the new legislation will have on the consolidated financial statements.

Previous wording

Cost of goods sold was $8.2 million for the three months ended March 31, 2025, an increase of $5.6 million, or 215%, from cost of goods sold of $2.6 million for the three months ended March 31, 2024. The increase in cost of goods sold was primarily the result of an increase in sales to Pfizer and Fiocruz (Brazil).

Current wording

Cost of goods sold was $14.1 million for the six months ended June 30, 2025, an increase of $2.0 million, or 17%, from cost of goods sold of $12.1 million for the six months ended June 30, 2024. The increase in cost of goods sold was primarily the result of an increase in sales to Chiesi and Pfizer partially offset by a decrease in sales to Fiocruz (Brazil).

Previous wording

Financial income, net was $0.4 million for the three months ended March 31, 2025, compared to financial income, net of $0.1 million for the three months ended March 31, 2024. The difference resulted primarily from lower notes interest expenses due to the September 2024 repayment in full of all the outstanding principal and interest payable under the 2024 Notes, partially offset by lower interest income on bank deposits and higher exchange rate costs.

Current wording

Financial expenses, net was $0.1 million for the six months ended June 30, 2025, compared to financial income, net of $0.3 million for the six months ended June 30, 2024. The decrease resulted primarily from exchange rate costs and lower interest income on bank deposits partially offset by lower notes interest expenses due to the September 2024 repayment in full of all the outstanding principal and interest payable under the 2024 Notes.

Previous wording

Our sources of liquidity include our cash balances and bank deposits. At March 31, 2025, we had $34.7 million in cash and cash equivalents and short term bank deposits. In September 2024, we satisfied the outstanding principal and accrued interest under the 2024 Notes with a cash payment of approximately $21.2 million which was available primarily from the withdrawal of short term deposits. We have primarily financed our operations through sales proceeds, equity and debt financings, business collaborations, and grants funding.

Current wording

Our sources of liquidity include our cash balances and short-term bank deposits. At June 30, 2025, we had $33.4 million in cash and cash equivalents and short term bank deposits. In September 2024, we satisfied the outstanding principal and accrued interest under the 2024 Notes with a cash payment of approximately $21.2 million which was available primarily from the withdrawal of short term deposits. We have primarily financed our operations through sales proceeds, equity and debt financings, business collaborations, and grants funding.

Previous wording

During the quarter ended March 31, 2025, we sold, in the aggregate, 1,325,179 shares of Common Stock under the Sales Agreement generating gross proceeds equal to approximately $3.0 million. In March 2025, we amended the program to provide for the offer and sale of up to an additional $20.0 million. In addition, during the quarter ended March 31, 2025, we issued 908,000 shares of our Common Stock in connection with the exercise of warrants issued in 2020 generating proceeds equal to approximately $2.1 million. The warrants expired on March 11, 2025. Accordingly, no warrants remain outstanding.

Current wording

During the six-month period ended June 30, 2025, we sold, in the aggregate, 2,775,215 shares of Common Stock under the Sales Agreement generating gross proceeds equal to approximately $7.0 million. In March 2025, we amended the Sales Agreement to provide for the offer and sale of up to an additional $20.0 million in shares of Common Stock. As of June 30, 2025, approximately $15.7 million in shares of Common Stock remain available to be sold under the Sales Agreement.

Previous wording

Net cash used in operations was $5.1 million for thethree months ended March 31, 2025. The net loss for thethree months ended March 31, 2025 of $3.6 million was increased by a $1.3 million decrease in accounts payable and accruals, a $2.3 million increase in accounts receivable-trade and other assets and $0.4 million financial income, net and was offset by a $0.5 million in share-based compensation, a $1.7 million decrease in inventories and $0.3 million in depreciation. Net cash used in investing activities for the three months ended March 31, 2025 was $0.3 million and consisted primarily of the purchase of property and equipment. Net cash provided by financing activities for thethree months ended March 31, 2025 was $5.1 million and consisted of $2.9 million proceeds from issuance of Common Stock under the Sales Agreement, net and $2.2 million from the exercise of warrants and options.

Current wording

Net cash used in operations was $10.3 million for thesix months ended June 30, 2025. The net loss for thesix months ended June 30, 2025 of $3.5 million was increased by a $6.9 million increase in accounts receivable-trade and other assets, a $1.9 million decrease in accounts payable and accruals, and was offset by a $1.0 million in share-based compensation and $0.7 million in depreciation. Net cash used in investing activities for the six months ended June 30, 2025 was $0.8 million and consisted primarily of the purchase of property and equipment. Net cash provided by financing activities for thesix months ended June 30, 2025 was $9.2 million and consisted of $6.8 million proceeds from issuance of Common Stock under the Sales Agreement, net and $2.4 million from the exercise of warrants and options.

Previous wording

Net cash provided by operations was $4.2 million for the three months ended March 31, 2024. The net loss for the three months ended March 31, 2024 of $4.6 million was increased by a $3.3 million increase in inventories, a $1.4 million decrease in accounts payable and accruals and was offset by an $11.0 million increase in contracts liability, a $1.7 million decrease in accounts receivable-trade and other assets and $1.0 million in share-based compensation. Net cash used in investing activities for the three months ended March 31, 2024 was $0.6 million and consisted primarily of the purchase of property and equipment.

Current wording

Net cash provided by operations was $0.6 million for the six months ended June 30, 2024. The net loss for the six months ended June 30, 2024 of $6.8 million was increased by a $1.7 million increase in inventories, a $5.3 million increase in accounts receivable-trade and other assets and $1.0 million of financial income, net, and was offset by a $12.7 million increase in contracts liability and $2.0 million in share-based compensation. Net cash used in investing activities for the six months ended June 30, 2024 was $0.8 million and consisted primarily of the purchase of property and equipment.

Previous wording

Since our inception, we have incurred significant research and development expenditures which have not been offset by revenues. We have not generated significant revenues from sales of Elelyso, and commercial sales of Elfabrio only commenced in the middle of

Current wording

Since our inception, we have incurred significant research and development expenditures which have not been offset by revenues. We have not generated significant revenues from sales of Elelyso, and commercial sales of Elfabrio only commenced in the middle of 2023. We have generated operating losses from our continuing operations since our inception although the revenues generated in the years ended December 31, 2023 and 2024 exceeded our expenditures for the same periods.

Previous wording

We expect to finance our future cash needs through sales of Elfabrio and Elelyso, corporate collaborations, licensing or similar arrangements, public or private equity offerings and/or debt financings. As of March 31, 2025, shares of Common Stock for total gross proceeds of approximately $19.7 million remain available to be sold under the Sales Agreement. We currently do not have any commitments for future external funding, except with respect to the milestone payments that may become payable under the Chiesi Agreements.

Current wording

We expect to finance our future cash needs through sales of Elfabrio and Elelyso, corporate collaborations, licensing or similar arrangements, public or private equity offerings and/or debt financings. We currently do not have any commitments for future external funding, except with respect to the milestone payments that may become payable under the Chiesi Agreements.

Previous wording

Currency fluctuations could affect us through increased or decreased acquisition costs for certain goods and services and salaries expenses. We do not believe currency fluctuations have had a material effect on our results of operations during the three months ended March 31, 2025.

Current wording

Currency fluctuations could affect us through increased or decreased acquisition costs for certain goods and services and salaries expenses. We do not believe currency fluctuations have had a material effect on our results of operations during the three and six months ended June 30, 2025.