PEGASO is an observational study designed to collect prospective data on the effectiveness and safety of pegunigalsidase alfa in adult patients with Fabry disease, being treated or planning to start treatment, under real-world setting.

Start Date01 Jan 2026

Sponsor / Collaborator

Chiesi Ltd.

NCT06328608

/ RecruitingPhase 2/3

Multi-centre, Open-label Trial to Assess the saFety, Pharmacodynamics, Efficacy and Pharmacokinetics of pegunigaLsidase Alfa in Patients From 2 Years to Less Than 18 Years of Age With Confirmed FabrY Disease

A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease.

Start Date29 Jul 2025

Sponsor / Collaborator

CHIESI Farmaceutici SpA

[+1]

NCT06663358

/ RecruitingNot Applicable

A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients

A multi-centre, multi-country, observational, non-interventional, retrospective and prospective (hybrid) study among Fabry disease participants treated with pegunigalsidase alfa (Elfabrio®) in routine clinical care.

Start Date06 Nov 2024

Sponsor / Collaborator

CHIESI Farmaceutici SpA

100 Clinical Results associated with Pegunigalsidase alfa

100 Translational Medicine associated with Pegunigalsidase alfa

100 Patents (Medical) associated with Pegunigalsidase alfa

Literatures (Medical) associated with Pegunigalsidase alfa

01 Mar 2025·ADVANCES IN THERAPY

Health State Utility Values in Fabry Disease: Insights from the Pegunigalsidase Alfa Clinical Trials

Article

Author: Koulinska, Irene ; Kunduri, Swati ; Raad-Faherty, Angie ; Lan, Zhiyi ; Tremblay, Gabriel ; Poritz, Julia ; Dorling, Patricia ; Azimpour, Khashayar

INTRODUCTION:

Fabry disease (FD) is a rare lysosomal storage disorder that is associated with pain and progressive damage to the renal, cardiac, and cerebrovascular systems. Enzyme replacement therapy (ERT) is one of the treatment options for FD and the most recently approved ERT agent, pegunigalsidase alfa, has shown clinical efficacy in three phase 3 clinical trials of adults with FD: BALANCE, BRIDGE, and BRIGHT. Recent published guidelines support the mapping of health utility state data to the EuroQol-5 Dimension-3 Level (EQ-5D-3L) index to align with the preferred methodology used by the National Institute for Health and Care Excellence (NICE). Therefore, the primary objective of this study was to estimate EQ-5D-3L values in clinical trials of pegunigalsidase alfa for FD for future cost-utility analyses.

METHODS:

A mixed effects model was developed to predict values derived from EQ-5D-3L for the following health states used in cost-utility analyses: no Fabry clinical event (FCE)/no pain-related adverse event (AE), pain-related AE, cardiac FCE, cerebrovascular FCE, and renal FCE.

RESULTS:

The baseline EQ-5D-3L utility value had a statistically significant (p < 0.0001) impact on utility values, whereas study, age, sex, disease type, treatment arm, kidney function, and serious AE were not statistically significant. Health state utility values with 95% confidence intervals (CI) for the final model were as follows: no FCE/no pain-related AE, 0.8005 (0.7675, 0.8334); pain-related AE, 0.7737 (0.7262, 0.8211); cardiac FCE, 0.7189 (0.6274, 0.8103); cerebrovascular FCE, 0.7923 (0.6633, 0.9212); and renal FCE, 0.6881 (0.3887, 0.9874).

CONCLUSIONS:

The utility values generated by the present study are generally in line with EQ-5D values in the FD literature and can be used to inform both economic evaluations and our understanding of the impact that FD has on quality of life.

TRIAL REGISTRATION:

NCT03018730, NCT02795676, NCT03180840.

01 Feb 2025·ADVANCES IN THERAPY

Effects of Current Therapies on Disease Progression in Fabry Disease: A Narrative Review for Better Patient Management in Clinical Practice

Review

Author: Cianci, Vittoria ; Pieroni, Maurizio ; Mignani, Renzo ; Pisani, Antonio ; Biagini, Elena ; Pieruzzi, Federico ; Tuttolomondo, Antonino

Fabry disease (FD) is a rare lysosomal storage disorder that is characterized by renal, neurological, and cardiovascular dysfunction. Four treatments are currently available for patients with FD; three enzyme replacement therapies (ERTs; agalsidase alfa, agalsidase beta, and pegunigalsidase alfa) and one pharmacological chaperone (migalastat). This review focuses on the evidence for the benefits of ERTs and migalastat, and provides an overview of their impact on disease manifestations and quality of life (QoL). Agalsidase beta is associated with renal, neurological, and cardiovascular benefits, and may prevent renal disease progression. Agalsidase alfa provides stabilizing effects across all main organ systems, although minor sex-specific differences exist in patients with more advanced baseline disease. The benefits of agalsidase alfa and agalsidase beta are similar but depend on the extent of baseline disease. Some data indicate that agalsidase beta may be preferable over the longer term. Both agalsidase alfa and agalsidase beta are associated with improved gastrointestinal and pain symptoms, as well as improved QoL. Patients with advanced end-organ damage tend not to respond as optimally to ERTs as those who initiate ERTs before irreversible organ fibrosis develops, highlighting the need for early treatment initiation. Migalastat, which is only approved for patients with amenable missense gene variants, generally stabilizes renal parameters and provides cardiovascular benefits. Migalastat also improves diarrhea and pain, and stabilizes QoL (although ERT may be more effective for pain management), but the neurological effects of migalastat have not been studied. Real-world data raise concerns about effective in vivo amenability of some genetic variants. Future studies with direct treatment comparisons in patients with FD are needed.

01 Jan 2025·BIODRUGS

Relevance of Neutralizing Antibodies for the Pharmacokinetics of Pegunigalsidase Alfa in Patients with Fabry Disease

Article

Author: Brand, Eva ; Rudnicki, Michael ; Cybulla, Markus ; Menke, Elise Raphaela ; Lenders, Malte

BACKGROUND:

Pegunigalsidase alfa is a newly approved drug for the treatment of Fabry disease, designed to increase the plasma half-life and reduce immunogenicity of infused α-galactosidase A (AGAL). We provide the first comprehensive pharmacokinetic and immunogenic data apart from industry-initiated studies.

METHODS:

Pharmacokinetics of pegunigalsidase alfa, amino acid, and polyethylene glycol (PEG)-specific antibodies and immune complexes were measured in treated patients (11 switched, two naïve). Measurements were performed in serum samples drawn directly before and after infusions over three to ten consecutive infusions. Only three patients started directly with 1.0 mg/kg body weight.

RESULTS:

No infusion-associated reactions were reported under pegunigalsidase alfa during the observation. Patients without pre-existing neutralizing anti-AGAL antibodies showed high enzymatic AGAL peak activities and sustained AGAL serum concentrations until the next infusion, which was not observed in those with neutralizing anti-AGAL antibodies. Nine (69.2%) patients presented with pre-existing anti-PEG antibodies (IgG or IgM), which seemed to have no impact on pharmacokinetics during the observation. No new anti-PEG or anti-AGAL antibody formation was observed after treatment initiation. Three (75.0%) patients with pre-existing neutralizing anti-AGAL antibodies showed a titer increase and one (25.0%) patient a decrease. In patients with anti-AGAL antibodies (n = 4) immune-complex formation was detected.

CONCLUSION:

The pharmacokinetics of pegunigalsidase alfa show different profiles depending on the presence of pre-existing neutralizing antibodies, with reduced plasma half-life and peak enzyme activity after infusion in patients with antibodies. The clinical significance of a reduced pegunigalsidase alfa half-life and the formation of immune complexes in antibody-positive patients needs to be analyzed in future studies.

103

News (Medical) associated with Pegunigalsidase alfa

09 Sep 2025

·www.globenewswire.com

Chiesi Global Rare Diseases Highlights Continued Commitment to Rare Disease Community at the International Congress of Inborn Errors of Metabolism (ICIEM) 2025

Results presented focus on clinical insights on Fabry disease and alpha-mannosidosis Additional presentations showcase patient-reported journeys and outcomes PARMA, Italy, Sept. 09, 2025 (GLOBE NEWSWIRE) -- Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases, today announced the presentation of nine abstracts at the International Congress of Inborn Errors of Metabolism (ICIEM) 2025, held September 2-6, 2025, in Kyoto, Japan. Focused on Fabry disease and alpha-mannosidosis, these presentations showcase patient-reported journeys and outcomes, alongside integrated results from clinical research in Fabry Disease. As part of Chiesi’s commitment to advancing dialogue in the field, the company also hosted a medical symposium titled “Catching the Clues, Changing the Course of LSDs: Illuminating the Complex Pathways of Rare Disease with Fabry Disease and Alpha-Mannosidosis in Focus.” The symposium explored the shared patient journey and challenges across the lysosomal storage disorder (LSD) care continuum, using Fabry disease and alpha-mannosidosis as illustrative examples. “These presentations reflect our ongoing commitment to not only advancing scientific understanding of Fabry disease and alpha-mannosidosis, but also amplifying the voices and lived experiences of the people affected,” said Mitch Goldman, Senior Vice President R&D, Chiesi Global Rare Diseases. “By combining clinical research with insights from patients and caregivers, we’re working to develop more holistic solutions to potentially address the needs of the rare disease community.” Professor Yoshikatsu Eto, Advanced Clinical Research Center, Southern Tohoku Research Center for Neuroscience, Kanagawa, Japan, added “The breadth of research presented, from clinical insights to patient-reported outcomes, reflects a growing commitment to understanding the full impact of these ultra-rare diseases. It’s encouraging to see continued collaboration between industry, clinicians, and patient communities to drive forward both scientific discovery and inform best practices of care.” Details of the presentations are as follows: Title: Exploring Patient-Reported Experiences with Symptom Worsening, Breakthrough, and Disease Monitoring, based on Treatment Status and Type: Results from a Double-Blind, Cross-Sectional SurveyPresenter: Irene Koulinska, M.D., Chiesi Global Rare DiseasesSummary: This study surveyed 238 adults with Fabry disease in the US and Canada, examining experiences with symptom worsening and disease monitoring, highlighting communication gaps between patients and physicians. 62% of treated patients rated their disease monitoring as excellent, compared to lower rates among treatment-naïve patients (20%) and those who discontinued therapy (29%). Common challenges included symptom progression, increased use of co-medications, and infusion-related issues, varying by enzyme replacement therapy (ERT) type. Title: Evaluating the relationship between antidrug antibodies and infusion-related reactions/ safety outcomes in patients with Fabry disease receiving enzyme replacement therapy (ERT): a systematic literature reviewPresenter: Patricio Aguiar, M.D., Ph.D., Lisbon UniversitySummary: A systematic literature review was conducted to evaluate the relationship between antidrug antibodies (ADAs) and safety/tolerability outcomes in patients with Fabry disease receiving ERT. Across six studies, most findings reported a positive association between ADA-positivity and an increased risk of infusion-related reactions (IRRs) and adverse events. Small sample sizes, retrospective designs, inconsistent ADA assessments, and variability in safety and tolerability outcomes limit the interpretation of these findings. The review also highlighted substantial variability in ADA assessment protocols, underscoring the need for future research to adopt standardized, validated methods to enhance the comparability and reliability of findings. Title: Extending the interval between pegunigalsidase alfa infusions in patients with Fabry disease: five-year interim results from the ongoing BRIGHT51 studyPresenter: John A. Bernat, M.D., Ph.D., University of Iowa Health CareSummary: A 5-year interim analysis from the ongoing BRIGHT51 study (NCT03614234) shows that long-term treatment with pegunigalsidase alfa 2 mg/kg every four weeks (E4W) continues to be well tolerated in stable adults with Fabry disease who switched from other enzyme replacement therapies. Among 29 participants, treatment-related adverse events were mild, and no serious events were reported. IRRs affected 31% of patients, mainly within the first year, and were more common among those with baseline ADA. Pain and quality of life remained stable; however, further research is needed to evaluate the long-term effects on kidney function. These findings support the ongoing evaluation of pegunigalsidase alfa E4W for long-term safety and effectiveness. Title: Evaluating the relationship between infusion-related reactions and antidrug antibody status: results from 109 patients with Fabry disease treated with pegunigalsidase alfaPresenter: John A. Bernat, M.D., Ph.D., University of Iowa Health CareSummary: Across the clinical program, an analysis of 109 patients receiving pegunigalsidase alfa 1 mg/kg, both ERT-naïve and ERT-switch, showed that 76% experienced no infusion-related reactions (IRRs), while 24% experienced IRRs, most of which occurred within the first year of treatment. Patients with antidrug antibodies (ADAs), especially those with higher titers, were more likely to experience IRRs. However, 19% of patients with baseline ADAs had no IRRs, suggesting that specific ADA characteristics, rather than ADA presence alone, may influence IRR risk. Title: Clinical assessment of disease severity in patients with Fabry disease treated with pegunigalsidase alfa: an integrated analysisPresenter: John A. Bernat, M.D., Ph.D., University of Iowa Health CareSummary: Across the clinical program, analysis of 87 patients treated with pegunigalsidase alfa showed that disease severity, as measured by the Mainz Severity Score Index (MSSI), improved or remained stable over 24 months. In ERT-naive patients, the proportion with mild MSSI scores rose from 50% at baseline to 81% at month 12. In ERT-switch patients, scores remained stable, with a slight trend toward improvement by month 24. All MSSI domains showed positive changes as early as month 6, supporting the potential of pegunigalsidase alfa to stabilize or reduce Fabry disease burden over the long term. Title: Navigating the real-world challenges of alpha-mannosidosis patients and caregivers: Understanding their journeys during, and after, diagnosisPresenter: Nato V. Vashakmadze, M.D., Ph.D., Pirogov Russian National Research Medical UniversitySummary: This study, based on surveys from 12 caregivers of 16 patients with alpha-mannosidosis across Russia, Spain, and Turkey, explored the real-world challenges families face, including isolation, limited disease information, scarce support services, and the burden of educating local healthcare providers due to low awareness. The findings underscore a pressing need for enhanced support systems and more comprehensive disease education. Title: The Real-World Journey of Patients with Alpha-Mannosidosis: A Retrospective US Claims Database StudyPresenter: Robert. J. Hopkin, M.D., Cincinnati Children's Hospital Medical CenterSummary: This retrospective open-claims analysis utilized tokenized data from 37 patients to depict the clinical burden and disease trajectory of alpha-mannosidosis. Early-onset cases were marked by elevated rates of hearing loss, developmental delays, and congenital anomalies, while adulthood was characterized by prevalent musculoskeletal complications, pain, and mobility challenges, accompanied by increased healthcare resource utilization as the disease advanced. Title: Changes in clinical manifestations in patients with alpha-mannosidosis treated with velmanase alfa therapy in the French Etoile Alpha registryPresenter: Nathalie Guffon, M.D., Femme Mère Enfant Hospital in LyonSummary: A retrospective analysis of 16 patients with alpha-mannosidosis treated with velmanase alfa (VA) in France, using data from the national Etoile Alpha registry, showed significant improvements in motor, quality of life, and psychological domains (p < 0.0001), with 47.4% of 247 clinical manifestations improving and 52.6% stabilizing after ~4.7 years of treatment. No clinical category experienced decline, with neuromuscular and skeletal/osteoarticular symptoms being the most common and largely stable. These real-world findings support the long-term clinical benefit of VA in managing alpha-mannosidosis. Title: Breaking Barriers in Lysosomal Storage Disorder Diagnosis: Using Machine Learning and No-Cost Genetic Testing to Improve Early Detection and AccessPresenter: Robert. J. Hopkin, M.D., Cincinnati Children's Hospital Medical CenterSummary: This study employed a machine learning–driven platform to analyze 21,500 self- or proxy-reported symptom assessments. 485 individuals identified as at-risk were offered complimentary genetic testing, with 184 providing samples for whole-exome sequencing. 10 tested positive for LSD variants, revealing pathogenic, likely pathogenic, and variants of uncertain significance across 10 genes. These findings illustrate the potential of AI-guided testing to broaden access to rare disease diagnostics and emphasize the importance of physician education and carrier family support to close diagnostic gaps. About Chiesi GroupChiesi is a research-oriented international biopharmaceutical group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The company’s mission is to improve people’s quality of life and act responsibly towards both the community and the environment.By changing its legal status to a Benefit Corporation in Italy, the US, France and Colombia, Chiesi’s commitment to creating shared value for society as a whole is legally binding and central to company-wide decision-making. As a certified B Corp since 2019, Chiesi is part of a global community of businesses that meet high standards of social and environmental impact. The company aims to reach Net-Zero greenhouse gases (GHG) emissions by 2035. With 90 years of experience, Chiesi is headquartered in Parma (Italy), with 31 affiliates worldwide, and counts more than 7,500 employees. The Group’s research and development centre in Parma works alongside 6 other important R&D hubs in France, the US, Canada, China, the UK, and Sweden. About Chiesi Global Rare DiseasesChiesi Global Rare Diseases is a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases. As a family business, Chiesi Group strives to create a world where it is common to have a therapy for all diseases and acts as a force for good, for society and the planet. The goal of the Global Rare Diseases unit is to ensure equal access so as many people as possible can experience their most fulfilling life. The unit collaborates with the rare disease community around the globe to bring voice to underserved people in the health care system. For more information visit www.chiesirarediseases.com About Chiesi in JapanChiesi Group has expanded its global presence with the establishment of Chiesi Japan in 2022. As part of the Group’s commitment to scientific advancement and international collaboration, Chiesi Japan serves as a regional base to support medical and research communities, contributing to ongoing efforts in the field of rare diseases. Chiesi Global Rare Diseases Media ContactSky StriarLifeSci Communications sstriar@lifescicomms.com

Clinical Result

30 Jun 2025

·www.prnewswire.com

Protalix BioTherapeutics Added to Russell 3000® and 2000 Indexes®

CARMIEL, Israel, June 30, 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 announced the Company has been added to the Russell 3000® and Russell 2000® Indexes, effective as of the U.S. market close on June 27, 2025, as part of the 2025 Russell indexes annual reconstitution. The annual reconstitution of the Russell US indexes captures the 4,000 largest US stocks as of April 30, ranking them by total market capitalization. Membership in the Russell 3000® Index, which remains in place for one year, means automatic inclusion in the large-cap Russell 1000® Index or small-cap Russell 2000® Index as well as the appropriate growth and value style indexes. FTSE Russell determines membership for its Russell indexes primarily by objective, market-capitalization rankings and style attributes. "We are very pleased to be included in the Russell 3000 and Russell 2000 Indexes," said Dror Bashan, Protalix's President and Chief Executive Officer. "We welcome the enhanced visibility provided by our inclusion in these indexes as we continue progressing on our strategic path." The Russell indexes are widely used by investment managers and institutional investors for index funds and as benchmarks for active investment strategies. According to data as of the end of June 2024, about $10.6 trillion in assets are benchmarked against the Russell US indexes, which belong to FTSE Russell, the global index provider. For more information on the Russell 3000® Index and the Russell indexes reconstitution, go to the "Russell Reconstitution" section on the FTSE Russell website. 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 letter 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, including statements regarding the Company's expected inclusion in the Russell 3000® and 2000® Indexes, 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. Factors that might cause material differences include, among others: risks related 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 conflicts; risks related to the regulatory approval and commercial success of our 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; and other factors described in our filings with the U.S. Securities and Exchange Commission. The statements in this letter 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

Drug Approval

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

100 Deals associated with Pegunigalsidase alfa

External Link

KEGG	Wiki	ATC	Drug Bank
D11685	-	-	DB14992

R&D Status

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Fabry Disease	European Union	CHIESI Farmaceutici SpA	04 May 2023
Fabry Disease	Iceland	CHIESI Farmaceutici SpA	04 May 2023
Fabry Disease	Liechtenstein	CHIESI Farmaceutici SpA	04 May 2023
Fabry Disease	Norway	CHIESI Farmaceutici SpA	04 May 2023

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02795676 (BALANCE, Pubmed \| J Med Genet)	Phase 3	Fabry Disease lyso-Gb3 concentrations	-	Pegunigalsidase alfa 1 mg/kg	ihggffajdr(obsyjjagsw) = vwfxqcafkh ubptqhvwdp (xbbtqhxcnj )	Positive	01 Jun 2024
				Agalsidase beta 1 mg/kg	ihggffajdr(obsyjjagsw) = neoyzmczms ubptqhvwdp (xbbtqhxcnj )
NCT02795676 (BALANCE, PRNewswire) Manual	Phase 3	Fabry Disease	77	pegunigalsidase alfa-iwxj	sucrpeussq(qrwyidnnbr) = oeejbslznx xrtmcmhivv (rvrcacnukn ) View more	Positive	10 Jan 2024
				agalsidase beta	sucrpeussq(qrwyidnnbr) = qqoxoyiysn xrtmcmhivv (rvrcacnukn ) View more
NCT03018730 (BRIDGE, Pubmed \| Orphanet J Rare Dis)	Phase 3	Fabry Disease plasma lyso-Gb3 level	-	Pegunigalsidase alfa 1 mg/kg	nlrpxezkfk(zimevexcxe) = 35% out of 20 patients were positive for pegunigalsidase alfa antidrug antibodies (ADAs) at ≥ 1 study timepoint, two of whom had pre-existing ADAs at baseline zrzhliyxvj (gjkbvmtceq ) View more	Positive	21 Oct 2023
SSIEM2023	Not Applicable	-	113	Pegunigalsidase alfa 1 mg/kg every other week	paqgzsieyn(vbwlylgado) = inwfteplub oafketeogj (rslxcxucxx ) View more	-	30 Aug 2023
				Pegunigalsidase alfa 2 mg/kg every 4 weeks	paqgzsieyn(vbwlylgado) = eyfwazkvpa oafketeogj (rslxcxucxx ) View more
SSIEM2023	Not Applicable	-	142	Pegunigalsidase alfa	wkkarqvewy(jmzypttkoa) = 25% of pts had an IRR atmiaieziy (ljuvihvulm ) View more	-	30 Aug 2023
NCT02795676 (BALANCE study \| BALANCE, CTgov)	Phase 3	Fabry Disease	78	PRX-102 (pegunigalsidase alfa) (PRX-102 (Pegunigalsidase Alfa))	gdjxnwkied(egcokrojhf) = ducoozihhg nshawhhmpg (syyujkmbdn, eepjqzmoki - eunpemnwiz) View more	-	02 Aug 2023
				agalsidase beta (Agalsidase Beta)	gdjxnwkied(egcokrojhf) = rgwtfpsujr nshawhhmpg (syyujkmbdn, nlpnsatayo - ijclpwfmrk) View more
NCT01678898 (FDA) Manual	Phase 1/2	Fabry Disease	14	ELFABRIO	cwhqtqebin(jltwbrucvw) = ziaqodwdkl hnilpqzlmd (mjrchwetim ) View more	Positive	09 May 2023
				ELFABRIO (Males)	cwhqtqebin(jltwbrucvw) = xbehsksccr hnilpqzlmd (mjrchwetim ) View more
NCT03566017 (FDA) Manual	Phase 3	Fabry Disease	77	ELFABRIO	wxvtutnlwh(zaopbopznf) = rxxucoiock ypojuugjyj (dyunmgkdka ) View more	Positive	09 May 2023
				agalsidase beta	wxvtutnlwh(zaopbopznf) = fyfhuzxcyj ypojuugjyj (dyunmgkdka )
EMA Manual	Phase 1/2	Fabry Disease	13	Elfabrio	zyvehymbtf(dvfpfojuoy) = mxqahbcgrh aiirrkvxin (wyrkpsljrl ) View more	Positive	08 May 2023
NCT03180840 (BRIGHT, SSIEM2022)	Phase 3	Fabry Disease Maintenance α-galactosidase A	30	Pegunigalsidase alfa 2.0 mg/kg	yssnhfjept(iudqqgudex) = ruzsngxgqs ddgtiippem (fiycmblgez )	Positive	30 Aug 2022

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Biosimilar

Competitive landscape of biosimilars in different countries/locations. Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

AI Agents Built for Biopharma Breakthroughs

Accelerate discovery. Empower decisions. Transform outcomes.

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis