RegulationFast Track (United States), Orphan Drug (United States), Orphan Drug (European Union), Overseas New Drugs Urgently Needed in Clinical Settings (China)

Structure/Sequence

Sequence Code 9157657

Source: *****

Clinical Trials associated with Taliglucerase Alfa

CTRI/2021/02/031283

/ RecruitingPhase 3IIT

A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects with Type 3 Gaucher Disease

Start Date22 Feb 2021

Sponsor / Collaborator-

NCT04002830

/ CompletedPhase 4IIT

A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease

This is a multicenter study to assess the safety and efficacy of taliglucerase alfa (60 units/kg) in previously untreated subjects of any age with Type 3 GD. Subjects will receive an infusion of taliglucerase alfa every 2 weeks for 12 months. Subjects who tolerate the infusions well, and who are treated in centers where home therapy is the SOC will be allowed to switch from site to home treatment at the discretion of the PI but after no less than 3 uneventful infusions at the site.

Start Date20 Nov 2020

Sponsor / Collaborator

Shaare Zedek Medical Center

[+1]

NCT03021941

/ WithdrawnPhase 4

A MULTICENTER, OPEN LABEL, PHARMACOKINETICS, PHARMACODYNAMICS AND SAFETY STUDY OF ELELYSO(TM) (TALIGLUCERASE ALFA) IN PEDIATRIC SUBJECTS WITH TYPE 1 GAUCHER DISEASE

In August of 2014, the FDA approved ELELYSO for long-term enzyme replacement therapy (ERT) for pediatric subjects with a confirmed diagnosis of Type 1 Gaucher disease. The recommended dosage for treatment-naïve adult and pediatric subjects 4 years of age and older is 60 units per kg of body weight administered every other week as a 60 to 120 minute intravenous infusion. As a postmarketing commitment, the Sponsor agreed to evaluate the pharmacokinetics (PK), pharmacodynamics (PD), and safety of Elelyso (taliglucerase alfa) in pediatric subjects with Type 1 Gaucher Disease. in at least 5 subjects with body weight less than 15 kg; at least 5 subjects with body weight 15 to less than 20 kg; and at least 5 subjects with body weight of 20-25 kg with Type 1 Gaucher disease dosed at 60 units/kg every other week.
When applicable, PD measurements for children enrolled in the PK study may be obtained through the taliglucerase alfa registry (PMR 1895-5) and will include organ volumes (spleen and liver), hematological values (hemoglobin and platelets) as well as growth (height and weight) data. Safety data, including any serious hypersensitivity reactions, such as anaphylaxis, as well as changes in antibody status (ie, detection and titers of binding and neutralizing antibodies, and detection of IgE antibodies), will also be collected through the taliglucerase alfa registry.

Start Date31 Jul 2019

Sponsor / Collaborator

Pfizer Inc.

100 Clinical Results associated with Taliglucerase Alfa

100 Translational Medicine associated with Taliglucerase Alfa

100 Patents (Medical) associated with Taliglucerase Alfa

141

Literatures (Medical) associated with Taliglucerase Alfa

01 Apr 2025·Neural Regeneration Research

New glucocerebrosidase antibodies can advance research in the field of neurodegenerative disorders

Article

Author: Ma, Charis ; Sidransky, Ellen ; Rytel, Krystyna ; Chen, Yu

A review.In medical research, there are times when the introduction of a new tool can launch scientific discovery in new directions.While antibody development may be considered mundane, in the field of glucocerebrosidase (GCase) research, the dearth of validated antibodies for different applications has impeded progress in studies of disease pathogenesis and therapeutic development.The recent introduction of new, rigorously evaluated antibodies can now propel research into the link between glucocerebrosidase and Parkinson′s disease (PD) as well as aspects of the pathobiol. of Gaucher disease (Jong et al., 2024).The perspective summarized recommended applications of hGCase-1/17 and hGCase-1/23 antibodies including co-immunoprecipitation, immunofluorescence, and AlphaLISA assays.

01 Sep 2024·GENE THERAPY

Efficacy of an AAV vector encoding a thermostable form of glucocerebrosidase in alleviating symptoms in a Gaucher disease mouse model

Article

Author: Futerman, Anthony H ; Oren, Roni ; Joseph, Tammar ; Tsoory, Michael ; Azulay, Aviya ; Blumenreich, Shani ; Fleishman, Sarel J ; Hochfelder, Ariel ; Milenkovic, Ivan ; Zerbib, Mirie ; Biton, Inbal E

Abstract:

Almost all attempts to date at gene therapy approaches for monogenetic disease have used the amino acid sequences of the natural protein. In the current study, we use a designed, thermostable form of glucocerebrosidase (GCase), the enzyme defective in Gaucher disease (GD), to attempt to alleviate neurological symptoms in a GD mouse that models type 3 disease, i.e. the chronic neuronopathic juvenile subtype. Upon injection of an AAVrh10 (adeno-associated virus, serotype rh10) vector containing the designed GCase (dGCase) into the left lateral ventricle of Gba−/−;Gbatg mice, a significant improvement in body weight and life-span was observed, compared to injection of the same mouse with the wild type enzyme (wtGCase). Moreover, a reduction in levels of glucosylceramide (GlcCer), and an increase in levels of GCase activity were seen in the right hemisphere of Gba−/−;Gbatg mice, concomitantly with a significant improvement in motor function, reduction of neuroinflammation and a reduction in mRNA levels of various genes shown previously to be elevated in the brain of mouse models of neurological forms of GD. Together, these data pave the way for the possible use of modified proteins in gene therapy for lysosomal storage diseases and other monogenetic disorders.

04 Dec 2023·Journal of osteoporosis

Skeletal Manifestations, Bone Pain, and BMD Changes in Albanian Type 1 Gaucher Patients Treated with Taliglucerase Alfa

Article

Author: Bali, Donjeta ; Velmishi, Virtut ; Dervishi, Ermira ; Tanka, Marjeta ; Tako, Afërdita ; Cullufi, Paskal ; Troja, Erjon ; Kollcaku, Laurant

Gaucher disease is a rare, genetic lysosomal disorder leading to lipid accumulation and dysfunctions in multiple organs. Bone involvement is one of the most prevalent aspects of Gaucher disease. Pain, disability, and reduced quality of life remain the most frequent characteristics of bone involvement in Gaucher patients. Patients and Method. In this study, we will take into consideration data from 24 patients diagnosed with type 1 Gaucher disease. We followed them closely for six years in progress. At baseline, all patients started therapy with taliglucerase alfa at a mean dosage of 45 UI/kg; later, during the study, two of them switched their cure toward velaglucerase alfa. Before baseline evaluations, 12 patients had been treated with imiglucerase at variable duration times. At baseline, we performed an X-ray of long bones and the spine, and each year, different standard assessments were performed, such as bone pain, MRI of the vertebral spine and pelvis, and DEXA for bone density. Four patients left the study for various reasons, two of them at baseline and two others during year 3 (FU3). Results. At baseline, we had 8 children and 16 adults. The average age was 28.7 ± 16.5 SD years. The most frequent skeletal manifestations in our patients were reduction of tibial femoral space (40%), osteonecrosis (36%), and body vertebral reduction (32%). At baseline, 15 patients presented with bone pain to different degrees. Over the years, bone pain in our patients had a gradual improvement. The most dramatic bone pain improvement was seen in a patient who presented bone crises. Another impressive finding was a significant BMD improvement during six years of treatment. Our study showed a significant improvement in BMD comparing FU5 and baseline values (

p

= 0.0007). Especially children demonstrated a significant improvement in BMD (

p

= 0.00061) compared to adults (

p

= 0.3673). Mean BMD change was more indicative in switched patients (

p

= 0.0142) compared to naïve patients (

p

= 0.147). Conclusions. Skeletal manifestations are very different in Gaucher type 1 patients. In our study, as a result of long-term evaluations, it was noticed that the most frequent skeletal manifestation was a reduction of tibiofemoral space. Bone pain has gradually improved in all patients. Also, BMD values have been enhanced over six years of treatment, especially in children.

News (Medical) associated with Taliglucerase Alfa

09 May 2025

·www.prnewswire.com

Protalix BioTherapeutics Reports First Quarter 2025 Financial and Business Results

Company to host conference call and webcast today at 8:30 a.m. EDT CARMIEL, Israel, May 9, 2025 /PRNewswire/ -- Protalix BioTherapeutics, Inc. (NYSE American: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx® plant cell-based protein expression system, today reported financial results for the quarter ended March 31, 2025, and provided a business and clinical update. "We had another solid quarter, with an increase in revenues from selling goods compared to the prior year quarter," said Dror Bashan, Protalix's President and Chief Executive Officer. "Given the promising results obtained in 2024 from our first-in-human study of our gout candidate, PRX-115, we are focused on building on the momentum and working toward initiating a phase II clinical trial in patients with gout later this year. At the same time, we continued to evaluate additional pipeline candidates for potential further development, including PRX-119 as well as various early-stage clinical assets." First Quarter 2025 and Recent Business and Clinical Highlights Pipeline Developments PRX-115 In 2024, we successfully completed the First-in-Human (FIH) phase I clinical trial of PRX-115, our recombinant PEGylated uricase product candidate in development as a potential treatment for uncontrolled gout. The study is designed to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD; reduction of uric acid) following a single dose of PRX-115 in subjects with elevated uric acid levels. We are in the advance stages of preparations for the phase II clinical trial we expect to commence during the second half of 2025. The preliminary results of the FIH study demonstrate that PRX-115 has the potential to offer an effective uric acid-lowering treatment with an added benefit of a potentially wide dosing interval, which may enhance patient compliance and treatment flexibility. Further studies are needed to confirm the long-term safety and efficacy of PRX-115 in the gout patient population. The results were presented in a late-breaking poster at the American College of Rheumatology (ACR) Convergence 2024, being held November 14-19, 2024 at the Walter E. Washington Convention Center in Washington, D.C. A copy of the poster is available on the Protalix website here: . Pegunigalsidase alfa In March 2025, our global development and commercial partner, Chiesi Global Rare Diseases, announced multiple presentations on pegunigalsidase alfa and Fabry Disease at the 21st Annual WORLDSymposium™ Research Meeting. The Poster presentations are available in the publications section of Protalix's website. First Quarter 2025 Financial Highlights 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 Inc. and an increase of $0.4 million in sales to Fundação Oswaldo Cruz, or Fiocruz (Brazil). 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 our license and supply agreements with Chiesi. We expect to generate minimal revenues from license and R&D services other than potential regulatory milestone payments. 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 resulted primarily from an increase in sales to Pfizer and Fiocruz (Brazil). 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. 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 from the advance in our clinical pipeline. 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. 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 our then outstanding 7.50% Senior Secured Convertible Promissory Notes, partially offset by lower interest income on bank deposits and higher exchange rate costs. For the three months ended each of March 31, 2025 and March 31, 2024, we recorded a tax benefit of approximately $(0.1) million. The tax benefit resulted primarily from deferred taxes on income mainly derived from GILTI income mainly in respect of Section 174 of the U.S. Tax Cuts and Jobs Act of 2017, or 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. Cash, cash equivalents and short term bank deposits were approximately $34.7 million at March 31, 2025. Net loss for the quarter ended March 31, 2025 was approximately $3.6 million, or $0.05 per share, basic and diluted, compared to $4.6 million, or $0.06 per share, basic and diluted, for the same period in 2024. Conference Call and Webcast Information We will host a conference call today, May 9, 2025 at 8:30 am EDT, to review the financial results and provide a business update. To participate in the conference call, please dial the following numbers prior to the start of the call: Conference Call Details: Date: Friday, May 9, 2025 Time: 8:30 a.m. Eastern Daylight Time (EDT) Toll Free: 1-877-423-9813 International: 1-201-689-8573 Israeli Toll Free: 1-809-406-247 Conference ID: 13753682 Call me™: The Call me™ feature allows you to avoid the wait for an operator; you enter your phone number on the platform and the system calls you right away. Webcast Details: The conference will be webcast live from the Protalix website and will be available via the following links: Company Link: Webcast Link: Conference ID: 13753682 Participants are requested to access the websites at least 15 minutes ahead of the conference to register, download and install any necessary audio software. A replay of the call will be available for two weeks on the Events Calendar of the Investors section of the Protalix website, at the above link. About Protalix BioTherapeutics, Inc. Protalix is a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx. It is the first company to gain U.S. Food and Drug Administration (FDA) approval of a protein produced through plant cell-based in suspension expression system. This unique expression system represents a new method for developing recombinant proteins in an industrial-scale manner. Protalix has licensed to Pfizer Inc. the worldwide development and commercialization rights to taliglucerase alfa for the treatment of Gaucher disease, Protalix's first product manufactured through ProCellEx, excluding in Brazil, where Protalix retains full rights. Protalix's second product, Elfabrio®, was approved by both the FDA and the European Medicines Agency in May 2023. Protalix has partnered with Chiesi Farmaceutici S.p.A. for the global development and commercialization of Elfabrio. Protalix's development pipeline consists of proprietary versions of recombinant therapeutic proteins that target established pharmaceutical markets, including the following product candidates: PRX–115, a plant cell-expressed recombinant PEGylated uricase for the treatment of uncontrolled gout; PRX–119, a plant cell-expressed long acting DNase I for the treatment of NETs-related diseases; and others. Forward-Looking Statements To the extent that statements in this press release are not strictly historical, all such statements are forward-looking, and are made pursuant to the safe-harbor provisions of the Private Securities Litigation Reform Act of 1995. The terms "anticipate," "believe," "estimate," "expect," "can," "continue," "could," "intend," "may," "plan," "potential," "predict," "project," "should," "will," "would" and other words or phrases of similar import are intended to identify forward-looking statements. These forward-looking statements are subject to known and unknown risks and uncertainties that may cause actual future experience and results to differ materially from the statements made. These statements are based on our current beliefs and expectations as to such future outcomes. Drug discovery and development involve a high degree of risk and the final results of a clinical trial may be different than the preliminary findings for the clinical trial. Factors that might cause material differences include, among others: risks related to the commercialization of Elfabrio® (pegunigalsidase alfa-iwxj), our approved product for the treatment of adult patients with Fabry disease; 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; the possible disruption of our operations due to the war declared by Israel's security cabinet against the Hamas terrorist organization located in the Gaza Strip, the military campaign against the Hezbollah and other terrorist activities and armed conflict, including as a result of the disruption of the operations of certain regulatory authorities and of certain of our suppliers, collaborative partners, licensees, clinical trial sites, distributors and customers, and the risk that the current hostilities will result in a greater regional conflict; risks related to the regulatory approval and commercial success of our other product and product candidates, if approved; risks related to our expectations with respect to the projected market of our products and product candidates; failure or delay in the commencement or completion of our preclinical studies and clinical trials, which may be caused by several factors, including: slower than expected rates of patient recruitment; unforeseen safety issues; determination of dosing issues; lack of effectiveness during clinical trials; inability to satisfactorily demonstrate non-inferiority to approved therapies; inability or unwillingness of medical investigators and institutional review boards to follow our clinical protocols; inability to monitor patients adequately during or after treatment; and/or lack of sufficient funding to finance our clinical trials; delays in the approval or potential rejection of any applications we file with the FDA, European Medicines Agency or other health regulatory authorities for our other product candidates, and other risks relating to the review process; risks associated with global conditions and developments such as new or increased tariffs, new trade restrictions, supply chain challenges, the inflationary environment and tight labor market, and instability in the banking industry, which may adversely impact our business, operations and ability to raise additional financing if and as required and on terms acceptable to us; risks related to any transactions we may effect in the public or private equity or debt markets to raise capital to finance future research and development activities, general and administrative expenses and working capital; risks relating to our evaluation and pursuit of strategic partnerships; the risk that the results of our clinical trials will not support the applicable claims of safety or efficacy and that our product candidates will not have the desired effects or will be associated with undesirable side effects or other unexpected characteristics; risks relating to our ability to manage our relationship with our collaborators, distributors or partners, including, but not limited to, Pfizer and Chiesi; risks related to the amount and sufficiency of our cash and cash equivalents and short-term bank deposits; risks relating to changes to interim, top-line or preliminary data from clinical trials that we announce or publish; risks relating to the compliance by Fundação Oswaldo Cruz, an arm of the Brazilian Ministry of Health, 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; risk of significant lawsuits, including stockholder litigation, which is common in the life sciences sector; our dependence on performance by third-party providers of services and supplies, including without limitation, clinical trial services; the inherent risks and uncertainties in developing drug platforms and products of the type we are developing; the impact of development of competing therapies and/or technologies by other companies; risks related to our supply of drug products to Pfizer; potential product liability risks, and risks of securing adequate levels of related insurance coverage; the possibility of infringing a third-party's patents or other intellectual property rights and the uncertainty of obtaining patents covering our products and processes and successfully enforcing our intellectual property rights against third-parties; risks relating to changes in healthcare laws, rules and regulations in the United States or elsewhere; and other factors described in our filings with the U.S. Securities and Exchange Commission. The statements in this press release are valid only as of the date hereof and we disclaim any obligation to update this information, except as may be required by law. Investor Contact Mike Moyer, Managing Director LifeSci Advisors +1-617-308-4306 [email protected] Logo: SOURCE Protalix BioTherapeutics, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 1Financial StatementDrug ApprovalPhase 2Clinical Result

17 Mar 2025

·www.prnewswire.com

Protalix BioTherapeutics Reports Fiscal Year 2024 Financial and Business Results

Company to host conference call and webcast today at 8:30 a.m. EDT CARMIEL, Israel, March 17, 2025 /PRNewswire/ -- Protalix BioTherapeutics, Inc. (NYSE American: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx® plant cell-based protein expression system, today reported financial results for the fiscal year ended December 31, 2024, and provided a business and clinical update. "2024 was a record year in revenues from selling goods for Protalix, as we experienced increases in all three of our revenue streams, Chiesi, Pfizer and Brazil," said Dror Bashan, Protalix's President and Chief Executive Officer. "We are pleased with the promising results from our first-in-human study of our gout candidate, PRX-115, in adult volunteers with elevated uric acid levels, and hope to build on this momentum with the goal of initiating a phase II clinical trial in patients with gout during the second half of 2025. At the same time, we continued to evaluate additional pipeline candidates, including PRX-119, for potential further development. Now that our debt is fully repaid and we no longer have outstanding warrants, our balance sheet is stronger and we are well-positioned to continue executing on our strategy through 2025 and beyond." Fiscal Year 2024 and Recent Business Highlights Pipeline and Clinical Developments In 2024, we successfully completed the First-in-Human (FIH) phase I clinical trial of PRX-115, our recombinant PEGylated uricase product candidate in development as a potential treatment for uncontrolled gout. The study is designed to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD; reduction of uric acid) following a single dose of PRX-115 in subjects with elevated uric acid levels. The results of the FIH study demonstrate that PRX-115 has the potential to offer an effective uric acid-lowering treatment with an added benefit of a potentially wide dosing interval, which may enhance patient compliance and treatment flexibility. Further studies are needed to confirm the long-term safety and efficacy of PRX-115 in the gout patient population. The results were presented in a late-breaking poster at the American College of Rheumatology (ACR) Convergence 2024, being held November 14-19, 2024 at the Walter E. Washington Convention Center in Washington, D.C. A copy of the poster is available on the Protalix website here: . In June 2024, we hosted an Investor Day highlighting current treatment landscapes and clinical results for Fabry disease and uncontrolled gout. The event featured presentations from key opinion leaders (KOLs) Aleš Linhart, D.Sc., FESC (Charles University, Prague) and Naomi Schlesinger, M.D. (University of Utah). Our leadership also provided insight into our strategy and future plans. The KOL presentation slides can be found in the Presentations section of the Protalix website: . Pegunigalsidase alfa In December 2024, we and our global development and commercial partner, Chiesi Global Rare Diseases, announced that the European Medicines Agency (EMA) validated the Variation Submission for pegunigalsidase alfa to label a less frequent dosing regimen at a dose of 2 mg/kg administered every four weeks in adult patients with Fabry disease. The variation submission is supported by a revised Population-PK model and new exposure-response analyses, and by the clinical data on pegunigalsidase alfa 2 mg/kg every four weeks from our completed phase III BRIGHT clinical trial of pegunigalsidase alfa (PB-102-F50) and the ongoing extension study. Corporate Developments In September 2024, we repaid in full all of the outstanding principal and interest payable under our then outstanding 7.50% Senior Secured Convertible Promissory Notes due September 2024. The repayment of the convertible notes at maturity was financed entirely with available cash. Since December 31, 2024, 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 from such exercises. The warrants expired on March 11, 2025. Accordingly, no warrants remain outstanding. Fiscal Year 2024 Financial Highlights We recorded revenues from selling goods of $53.0 million for the year ended December 31, 2024, an increase of $12.6 million, or 31%, compared to revenues of $40.4 million for the year ended December 31, 2023. The increase resulted primarily from an increase of $11.8 million in sales to Chiesi, an increase of $0.6 million in sales to Brazil and an increase of $0.1 million in sales to Pfizer Inc., or Pfizer. We recorded revenues from license and R&D services of $0.4 million for the year ended December 31, 2024, a decrease of $24.7 million, or 98%, compared to revenues of $25.1 million for the year ended December 31, 2023. Revenues from license and R&D services are comprised primarily of revenues we recognized in connection with the Company's license and supply agreements with Chiesi Farmaceutici S.p.A., or Chiesi. The revenues from license and R&D services for the year ended December 31, 2023 included the $20.0 million regulatory milestone payment from Chiesi in connection with the approval by the U.S. Food and Drug Administration (FDA) of Elfabrio® granted during that period. The remaining decrease resulted from the completion of our revenue-generating research and development obligations with respect to Elfabrio and, as Elfabrio was approved in the United States and the European Union in May 2023, from the completion of the regulatory processes related to the review of the Biologics License Application (BLA) and the Marketing Authorization Application (MAA) for Elfabrio by the FDA and EMA, respectively. As a result of the completion of the Fabry clinical program in 2023, we expect to generate minimal revenues from license and R&D services other than potential regulatory and commercial milestone payments. Cost of goods sold was $24.3 million for the year ended December 31, 2024, an increase of $1.3 million, or 6%, compared to cost of goods sold of $23.0 million for the year ended December 31, 2023. The increase in cost of goods sold was primarily the result of the increase in sales to Chiesi. In addition, during the year ended December 31, 2023 a portion of the costs for certain drug substance sold were recognized as research and development expenses, not cost of goods sold, as such drug substance was produced as part of our research and development activities. For the year ended December 31, 2024, our total research and development expenses were approximately $13.0 million comprised of approximately $7.1 million of salary and related expenses, approximately $2.4 million in subcontractor-related expenses, approximately $0.9 million of materials-related expenses and approximately $2.6 million of other expenses. For the year ended December 31, 2023, our total research and development expenses were approximately $17.1 million comprised of approximately $7.8 million of salary and related expenses, approximately $6.3 million in subcontractor-related expenses, approximately $0.6 million of materials-related expenses and approximately $2.4 million of other expenses. Total decrease in research and developments expenses was $4.1 million, or 24%, for the year ended December 31, 2024 compared to the year ended December 31, 2023. The decrease in research and development expenses resulted primarily from the completion of our Fabry clinical program and the regulatory processes related to the BLA and MAA review of Elfabrio by the applicable regulatory agencies. Selling, general and administrative expenses were $12.2 million for the year ended December 31, 2024, a decrease of $2.8 million, or 19%, from $15.0 million for the year ended December 31, 2023. The decrease resulted primarily from a decrease of $1.8 million in professional fees and of $1.0 million in salaries and related expenses. Financial income, net was $0.2 million for the year ended December 31, 2024, compared to financial expenses, net of $1.9 million for the year ended December 31, 2023. The difference resulted primarily from a decrease of approximately $1.4 million in lower interest and related expenses due to the conversion of notes in 2023 and the September 2024 repayment in full of all the outstanding principal and interest payable under the remaining notes, as well as an increase in interest income, net of $0.7 million. For the year ended December 31, 2024, we recorded income taxes of approximately $1.2 million, an increase of $0.9 million, or 300%, compared to income taxes of $0.3 million for the year ended December 31, 2023. The income taxes resulted primarily from the provision for current taxes on income mainly derived from GILTI income mainly in respect of Section 174 of the U.S. Tax Cuts and Jobs Act, or 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. Cash, cash equivalents and short-term bank deposits were approximately $34.8 million at December 31, 2024. Net income for the year ended December 31, 2024 was approximately $2.9 million, or $0.04 per share, basic and diluted, compared to $8.3 million or $0.12 per share, basic, and $0.09 per share, diluted, for the same period in 2023. Conference Call and Webcast Information We will host a conference call today, March 17, 2025, at 8:30 am EDT, to review the financial results and provide a business update. To participate in the conference call, please dial the following numbers prior to the start of the call: Conference Call Details: Date: Monday, March 17, 2025 Time: 8:30 a.m. Eastern Daylight Time (EDT) Toll Free: 1-877-423-9813 International: 1-201-689-8573 Israeli Toll Free: 1-809-406-247 Conference ID: 13752080 Call me™: The Call me™ feature allows you to avoid the wait for an operator; you enter your phone number on the platform and the system calls you right away. Webcast Details: The conference will be webcast live from the Protalix website and will be available via the following links: Company Link: Webcast Link: Conference ID: 13752080 Participants are requested to access the websites at least 15 minutes ahead of the conference to register, download and install any necessary audio software. A replay of the call will be available for two weeks on the Events Calendar of the Investors section of the Protalix website, at the above link. About Protalix BioTherapeutics, Inc. Protalix is a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx. It is the first company to gain U.S. Food and Drug Administration (FDA) approval of a protein produced through plant cell-based in suspension expression system. This unique expression system represents a new method for developing recombinant proteins in an industrial-scale manner. Protalix has licensed to Pfizer Inc. the worldwide development and commercialization rights to taliglucerase alfa for the treatment of Gaucher disease, Protalix's first product manufactured through ProCellEx, excluding in Brazil, where Protalix retains full rights. Protalix's second product, Elfabrio®, was approved by both the FDA and the European Medicines Agency in May 2023. Protalix has partnered with Chiesi Farmaceutici S.p.A. for the global development and commercialization of Elfabrio. Protalix's development pipeline consists of proprietary versions of recombinant therapeutic proteins that target established pharmaceutical markets, including the following product candidates: PRX–115, a plant cell-expressed recombinant PEGylated uricase for the treatment of uncontrolled gout; PRX–119, a plant cell-expressed long acting DNase I for the treatment of NETs-related diseases; and others. Forward-Looking Statements To the extent that statements in this press release are not strictly historical, all such statements are forward-looking, and are made pursuant to the safe-harbor provisions of the Private Securities Litigation Reform Act of 1995. The terms "anticipate," "believe," "estimate," "expect," "can," "continue," "could," "intend," "may," "plan," "potential," "predict," "project," "should," "will," "would" and other words or phrases of similar import are intended to identify forward-looking statements. These forward-looking statements are subject to known and unknown risks and uncertainties that may cause actual future experience and results to differ materially from the statements made. These statements are based on our current beliefs and expectations as to such future outcomes. Drug discovery and development involve a high degree of risk and the final results of a clinical trial may be different than the preliminary findings for the clinical trial. Factors that might cause material differences include, among others: risks related to the commercialization of Elfabrio® (pegunigalsidase alfa-iwxj), our approved product for the treatment of adult patients with Fabry disease; 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; the possible disruption of our operations due to the war declared by Israel's security cabinet against the Hamas terrorist organization located in the Gaza Strip, the military campaign against the Hezbollah and other terrorist activities and armed conflict, including as a result of the disruption of the operations of certain regulatory authorities and of certain of our suppliers, collaborative partners, licensees, clinical trial sites, distributors and customers, and the risk that the current hostilities will result in a greater regional conflict; risks related to the regulatory approval and commercial success of our other product and product candidates, if approved; risks related to our expectations with respect to the projected market of our products and product candidates; failure or delay in the commencement or completion of our preclinical studies and clinical trials, which may be caused by several factors, including: slower than expected rates of patient recruitment; unforeseen safety issues; determination of dosing issues; lack of effectiveness during clinical trials; inability to satisfactorily demonstrate non-inferiority to approved therapies; inability or unwillingness of medical investigators and institutional review boards to follow our clinical protocols; inability to monitor patients adequately during or after treatment; and/or lack of sufficient funding to finance our clinical trials; delays in the approval or potential rejection of any applications we file with the FDA, EMA or other health regulatory authorities for our other product candidates, and other risks relating to the review process; risks associated with global conditions and developments such as new or increased tariffs, new trade restrictions, supply chain challenges, the inflationary environment and tight labor market, and instability in the banking industry, which may adversely impact our business, operations and ability to raise additional financing if and as required and on terms acceptable to us; risks related to any transactions we may effect in the public or private equity or debt markets to raise capital to finance future research and development activities, general and administrative expenses and working capital; risks relating to our evaluation and pursuit of strategic partnerships; the risk that the results of our clinical trials will not support the applicable claims of safety or efficacy and that our product candidates will not have the desired effects or will be associated with undesirable side effects or other unexpected characteristics; risks relating to our ability to manage our relationship with our collaborators, distributors or partners, including, but not limited to, Pfizer and Chiesi; risks related to the amount and sufficiency of our cash and cash equivalents and short-term bank deposits; risks relating to changes to interim, top-line or preliminary data from clinical trials that we announce or publish; risks relating to the compliance by Fundação Oswaldo Cruz, an arm of the Brazilian Ministry of Health, 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; risk of significant lawsuits, including stockholder litigation, which is common in the life sciences sector; our dependence on performance by third-party providers of services and supplies, including without limitation, clinical trial services; the inherent risks and uncertainties in developing drug platforms and products of the type we are developing; the impact of development of competing therapies and/or technologies by other companies; risks related to our supply of drug products to Pfizer; potential product liability risks, and risks of securing adequate levels of related insurance coverage; the possibility of infringing a third-party's patents or other intellectual property rights and the uncertainty of obtaining patents covering our products and processes and successfully enforcing our intellectual property rights against third-parties; risks relating to changes in healthcare laws, rules and regulations in the United States or elsewhere; and other factors described in our filings with the U.S. Securities and Exchange Commission. The statements in this press release are valid only as of the date hereof and we disclaim any obligation to update this information, except as may be required by law. Investor Contact Mike Moyer, Managing Director LifeSci Advisors +1-617-308-4306 [email protected] Logo - SOURCE Protalix BioTherapeutics, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 1Drug ApprovalFinancial StatementClinical ResultPhase 3

23 Dec 2024

·www.prnewswire.com

Protalix BioTherapeutics Issues 2025 Letter to Stockholders

CARMIEL, Israel, Dec. 23, 2024 /PRNewswire/ -- Protalix BioTherapeutics, Inc. (NYSE-American: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx® plant cell based protein expression system, today announced the following letter from its President and Chief Executive Officer, Dror Bashan, to its stockholders. December 23, 2024 Dear Protalix Stockholders, As we conclude 2024, I am filled with a sense of accomplishment as well as excitement for the year ahead. This past year, Protalix has achieved significant milestones that position our company for long-term growth. The success we have achieved this year is a direct result of our team's hard work and dedication. We're proud to share these accomplishments with you. Our commercial partner, Chiesi Global Rare Diseases, is playing a pivotal role in our journey, and we continue to collaborate with Chiesi in its efforts. Most recently, the European Medicine Agency (EMA) validated Chiesi's Variation Submission for pegunigalsidase alfa to label a less frequent dosing regimen (every four weeks) for patients with Fabry disease in the European Union. This submission exemplifies Protalix's and Chiesi's mutual commitment to reducing the treatment burden faced by patients with Fabry disease. We have made important progress on our pipeline programs over the past year. We completed a phase I First-in-Human clinical trial evaluating PRX 115, our recombinant PEGylated uricase (urate oxidase) in development for the potential treatment of uncontrolled gout. Results from the trial demonstrate the potential of PRX-115 to be a safe and effective uric-acid lowering treatment with a potential wide dosing interval. We were proud to present this data to the medical community at the American College of Rheumatology (ACR) Convergence 2024 conference in November and are looking forward to advancing PRX-115 into a phase II clinical trial in patients with uncontrolled gout in the second half of 2025. We are also excited to have fine-tuned our R&D strategy moving forward, leveraging our ProCellEx platform and other capabilities in prioritized renal rare diseases. Our expert R&D team continues to evaluate plant-based drug delivery systems that may allow protective delivery of different modalities. The team is laser focused on these efforts, and we are eager to share more pipeline developments throughout the coming year. We are satisfied with the growth in our sales to Chiesi of Elfabrio®, Elelyso® to Pfizer Inc. and Uplyso® to Brazil which makes up our three revenue streams. We look forward to continuing growth of our sales in the coming years. Finally, by employing financial discipline over the last few years, we were able to repay in full all of our outstanding convertible notes, making us a debt-free company. We are proud of this achievement, and our strong balance sheet enables us to support ongoing operations. Our community continues to face the challenges of military activity in Israel. The suffering experienced by people in all sectors of these conflicts is heart-breaking. Despite the impact of these events on our colleagues and community, on both a personal and professional level, we have successfully maintained full, uninterrupted operations throughout the conflict. The continued dedication of our employees is a source of pride and we look forward to more peaceful times. Looking ahead, the future of Protalix is exciting. I am confident in our ability to deliver innovative solutions to improve the lives of patients in need. I want to express my sincere gratitude to our dedicated employees, our supportive Board of Directors and our valued shareholders. Your collective efforts and belief in our mission are instrumental in driving our progress. We sincerely thank you. Truly yours, Dror Bashan, President & Chief Executive Officer About Protalix BioTherapeutics, Inc. Protalix is a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx. It is the first company to gain U.S. Food and Drug Administration (FDA) approval of a protein produced through plant cell-based in suspension expression system. This unique expression system represents a new method for developing recombinant proteins in an industrial-scale manner. Protalix has licensed to Pfizer Inc. the worldwide development and commercialization rights to taliglucerase alfa for the treatment of Gaucher disease, Protalix's first product manufactured through ProCellEx, excluding in Brazil, where Protalix retains full rights. Protalix's second product, Elfabrio®, was approved by both the FDA and the European Medicines Agency in May 2023. Protalix has partnered with Chiesi Farmaceutici S.p.A. for the global development and commercialization of Elfabrio. Protalix's development pipeline consists of proprietary versions of recombinant therapeutic proteins that target established pharmaceutical markets, including the following product candidates: PRX–115, a plant cell-expressed recombinant PEGylated uricase for the treatment of uncontrolled gout; PRX–119, a plant cell-expressed long action DNase I for the treatment of NETs-related diseases; and others. Forward-Looking Statements To the extent that statements in this press release are not strictly historical, all such statements are forward-looking, and are made pursuant to the safe-harbor provisions of the Private Securities Litigation Reform Act of 1995. The terms "expect," "anticipate," "believe," "estimate," "project," "may," "plan," "will," "would," "should" and "intend," and other words or phrases of similar import are intended to identify forward-looking statements. These forward-looking statements are subject to known and unknown risks and uncertainties that may cause actual future experience and results to differ materially from the statements made. These statements are based on our current beliefs and expectations as to such future outcomes. Drug discovery and development involve a high degree of risk and the final results of a clinical trial may be different than the preliminary findings for the clinical trial. Factors that might cause material differences include, among others: risks related to the commercialization of Elfabrio® (pegunigalsidase alfa-iwxj), our approved product for the treatment of adult patients with Fabry disease; 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; the possible disruption of our operations due to the war declared by Israel's security cabinet against the Hamas terrorist organization located in the Gaza Strip, the military campaign against the Hezbollah and other terrorist activities and armed conflict, including as a result of the disruption of the operations of certain regulatory authorities and of certain of our suppliers, collaborative partners, licensees, clinical trial sites, distributors and customers, and the risk that the current hostilities will result in a greater regional conflict; risks related to the regulatory approval and commercial success of our other product and product candidates, if approved; risks related to our expectations with respect to the potential commercial value of our products and product candidates; failure or delay in the commencement or completion of our preclinical studies and clinical trials, which may be caused by several factors, including: slower than expected rates of patient recruitment; unforeseen safety issues; determination of dosing issues; lack of effectiveness during clinical trials; inability to satisfactorily demonstrate non-inferiority to approved therapies; inability or unwillingness of medical investigators and institutional review boards to follow our clinical protocols; inability to monitor patients adequately during or after treatment; and/or lack of sufficient funding to finance our clinical trials; delays in the approval or potential rejection of any applications we file with the FDA, EMA or other health regulatory authorities for our other product candidates, and other risks relating to the review process; risks associated with global conditions and developments such as supply chain challenges, the inflationary environment and tight labor market, and instability in the banking industry, which may adversely impact our business, operations and ability to raise additional financing if and as required and on terms acceptable to us; risks related to any transactions we may effect in the public or private equity markets to raise capital to finance future research and development activities, general and administrative expenses and working capital; risks relating to our evaluation and pursuit of strategic partnerships; the risk that the results of our clinical trials will not support the applicable claims of safety or efficacy and that our product candidates will not have the desired effects or will be associated with undesirable side effects or other unexpected characteristics; 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.; risks related to the amount and sufficiency of our cash and cash equivalents; risks relating to changes to interim, topline or preliminary data from clinical trials that we announce or publish; risks relating to the compliance by Fundação Oswaldo Cruz, an arm of the Brazilian Ministry of Health, 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; risk of significant lawsuits, including stockholder litigation, which is common in the life sciences sector; our dependence on performance by third-party providers of services and supplies, including without limitation, clinical trial services; the inherent risks and uncertainties in developing drug platforms and products of the type we are developing; the impact of development of competing therapies and/or technologies by other companies; risks related to our supply of drug products to Pfizer; potential product liability risks, and risks of securing adequate levels of related insurance coverage; the possibility of infringing a third-party's patents or other intellectual property rights and the uncertainty of obtaining patents covering our products and processes and successfully enforcing our intellectual property rights against third-parties; and risks relating to changes in healthcare laws, rules and regulations in the United States or elsewhere; and other factors described in our filings with the U.S. Securities and Exchange Commission. The statements in this press release are valid only as of the date hereof and we disclaim any obligation to update this information, except as may be required by law. Investor Contact Mike Moyer, Managing Director LifeSci Advisors +1-617-308-4306 [email protected] Logo - SOURCE Protalix BioTherapeutics, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 1Drug ApprovalPhase 2

100 Deals associated with Taliglucerase Alfa

External Link

KEGG	Wiki	ATC	Drug Bank
D09675	Taliglucerase Alfa	A16AB11	DB08876

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Gaucher Disease, Type 1	United States	Pfizer Inc.	01 May 2012

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Gaucher Disease	NDA/BLA	European Union	Pfizer Inc.	-

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04002830 (CTgov)	Phase 4	Gaucher Disease, Type 3	14	Taliglucerase Alfa	fdodnsuctj(cymqlnurqv) = oiqebtehld gxyyiyiwuw (tguakzrvnw, 8.67) View more	-	17 Dec 2024
LEAP-study (MDS2022)	Not Applicable	Parkinson Disease glucocerebrosidase (GBA) gene missense mutation	445	glucocerebrosidase genotype	tdhluappst(mglwyxicxa) = qcssdvmjrl rmgmhaktqq (fdhzdmuilv, -0.07 to 0.03)	Negative	15 Sep 2022
Pubmed \| Blood Cells Mol Dis	Not Applicable	Gaucher Disease	-	Taliglucerase alfa	twsqjptbqv(nxtmwmycvt) = Most adverse events were mild or moderate; treatment-related adverse events were mild and transient wvefrowakf (bxtvjugkwq )	-	01 May 2020
				Imiglucerase
NCT01422187 (PB-06-001, Pubmed \| Blood Cells Mol Dis)	Phase 3	Gaucher Disease	-	Taliglucerase alfa 30U/kg	dmkxussgek(xhpgsuhdes) = xejwntlgot mllttvrtkv (finplbbqwc ) View more	Positive	01 Sep 2019
				Taliglucerase alfa 60U/kg	dmkxussgek(xhpgsuhdes) = ahsxxiaeyl mllttvrtkv (finplbbqwc ) View more
NCT01411228 (Pubmed \| Blood Cells Mol Dis) Manual	Phase 3	Gaucher Disease	10	taliglucerase alfa (treatment-naïve patients)	afwnxhslyi(vylixhwtbd) = bammggwpwj hznvxmiehr (pgplrvyvfx ) View more	Positive	01 Feb 2018
				taliglucerase alfa (treatment-naïve patients)	afwnxhslyi(vylixhwtbd) = vlcuhxusyv hznvxmiehr (pgplrvyvfx ) View more
NCT00705939 (PB-06-003, Pubmed \| Am J Hematol) Manual	Phase 3	Gaucher Disease	18	taliglucerase alfa	imqhrujgwx(akcyxusanl) = nmoicsgita ocprzbvdmr (opdaptiwbx ) View more	Positive	01 Jul 2016
NCT00705939 (Pubmed \| Am J Hematol)	Phase 3	Gaucher Disease	23	Taliglucerase alfa 30 U/kg	qefullrlib(eothdrlgqr) = esywjiaxny aijuzsfrtr (lzpnctfutj ) View more	-	01 Jul 2016
				Taliglucerase alfa 60 U/kg	qefullrlib(eothdrlgqr) = gznqjliwem aijuzsfrtr (lzpnctfutj ) View more
MDS2016	Not Applicable	Parkinson Disease GBA mutations	-	Glucocerebrosidase	ccafxdgswn(jrirfnqkre) = vwtsksbwcj ltxpbouasc (ftvzmqtbkk )	-	19 Jun 2016
				Glucocerebrosidase	ccafxdgswn(jrirfnqkre) = bxnfrpspgr ltxpbouasc (ftvzmqtbkk )
NCT01411228 (CTgov)	Phase 3	Gaucher Disease	15	Taliglucerase alfa (60 Units/kg)	pfcywgfqqo(tblumgbckx) = iohdwzbpfq wryafykkrj (tllpfxhzjv, xxsweonbma - idlijkmyqm) View more	-	18 Jan 2016
				Taliglucerase alfa (30 Units/kg)	pfcywgfqqo(tblumgbckx) = gftubvkgsc wryafykkrj (tllpfxhzjv, rrugecyovv - tyhpjpyvnc) View more
NCT01422187 (PB-06-001, CTgov)	Phase 3	Gaucher Disease	19	Taliglucerase alfa (Taliglucerase Alfa 30 Units/kg)	yvmbfmpkwp(yedzhhlahj) = hdeojweadt jnsmoifrxv (czbdlkoyvh, 454.9) View more	-	14 Dec 2015
				Taliglucerase alfa (Taliglucerase Alfa 60 Units/kg)	yvmbfmpkwp(yedzhhlahj) = jqigohikxr jnsmoifrxv (czbdlkoyvh, 451.7) View more

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