Protalix Biotherapeutics, Inc.

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

TORONTO and HAIFA, Israel, Jan. 21, 2025 (GLOBE NEWSWIRE) -- NurExone Biologic Inc. (TSXV: NRX), (OTCQB: NRXBF), (Germany: J90) (“NurExone” or the “Company”), a developer of exosome-based therapies for regenerative medicine, is pleased to announce that, subject to TSX Venture Exchange (“TSXV”) approval, it has closed a non-brokered private placement of 856,996 units (“Units”) at a price of C$0.56 per Unit for aggregate gross proceeds of C$479,917.76 (the “Offering”). The Company intends to use the proceeds of the Offering for working capital purposes. In addition, the Company is pleased to announce that, further to its press release dated August 28, 2023 (the “August 28, 2023 Release”), the Company has received gross proceeds of C$727,755.04 through the exercise of 2,140,456 Class A Warrants at a price of C$0.34 per Class A Warrant issued in the first tranche of the non-brokered private placement of the Company which closed on August 25, 2023 (the “August 2023 Offering”). Capitalized terms not otherwise defined herein have the meanings attributed to them in the August 28, 2023 Release. Terms of the Offering Each Unit consisted of (i) one common share in the capital of the Company (each, a “Common Share”), and (ii) one Common Share purchase warrant (each, a “Warrant”). Each Warrant entitles the holder thereof to purchase one Common Share at a price of C$0.70 per Common Share for a period of 36 months, subject to acceleration. If the daily volume weighted average trading price of the Common Shares on the TSXV for any period of 20 consecutive trading days equals or exceeds C$1.75, the Company may, upon providing written notice to the holders of the Warrants (the “Acceleration Notice”), accelerate the expiry date of the Warrants to the date that is 45 days following the date of the Acceleration Notice. In addition, following the date of the issuance of the Warrants, if the Company lists the Common Shares to a nationally recognized stock exchange in the United States, the Company may upon providing an Acceleration Notice, accelerate the expiry date of the Warrants to the date that is 45 days following the date of the Acceleration Notice. If the Warrants are not exercised by the applicable accelerated expiry dates, the Warrants will expire and be of no further force or effect. Closing of the Offering is subject to receipt of all necessary regulatory approvals, including TSXV, and all securities issued under the Offering are subject to a statutory hold period of four months and one day from the closing of the Offering. Warrant Exercises Following the Company providing the outstanding Class A Warrant holders an acceleration notice on December 17, 2024 that the Class A Warrant acceleration trigger was met, when the daily volume weighted average trading price of the Common Shares on the TSXV equalled or exceeded C$0.69 for a period of 20 consecutive trading days, 2,140,456 Class A Warrants were exercised at a price of $0.34 per Class A Warrant, providing the Company C$727,755.04 in gross proceeds. The effect of such exercises, along with the prior exercise of 181,818 Class A Warrant back in March 2024, resulted in all Class A Warrants issued in the August 2023 Offering being exercised. Statements from the CEO and CFO Eran Ovadya, NurExone’s CFO, expressed: “we sincerely appreciate the trust our investors have placed in us. The warrant exercises and private placement have generated slightly more than C$1.2 million, providing essential support for our mission.” Dr. Lior Shaltiel, NurExone’s CEO, added: “the successful fundraising efforts demonstrate confidence in NurExone’s vision and strategy. These funds will allow us to accelerate our R&D activities and drive forward key collaborations. Additionally, we are pleased to welcome Dr. Tali Kizhner as our new Director of Research and Development (“R&D”). Her outstanding expertise in biologics and proven leadership in advancing therapeutic programs will be invaluable as we prepare to move to clinical trials and achieve our next set of milestones.” This news release does not constitute an offer to sell or a solicitation of an offer to buy any of the securities described in this news release in the United States. Such securities have not been, and will not be, registered under the United States Securities Act of 1933, as amended (the “U.S. Securities Act”), or any state securities laws, and, accordingly, may not be offered or sold within the United States, or to or for the account or benefit of persons in the United States or “U.S. Persons”, as such term is defined in Regulation S promulgated under the U.S. Securities Act, unless registered under the U.S. Securities Act and applicable state securities laws or pursuant to an exemption from such registration requirements. Director of R&D Appointment The Company has appointed Dr. Tali Kizhner as its new Director of R&D, reinforcing the Company’s leadership as it advances toward clinical trials. With over 15 years of R&D and chemistry, manufacturing and controls expertise, Dr. Kizhner has led groundbreaking initiatives in therapeutic protein development and dietary supplements. She joins NurExone from Biond Biologics, where she specialized in intracellular delivery of biologics, and previously led global R&D efforts at International Flavors & Fragrances. At Protalix Biotherapeutics, she played a pivotal role in developing biologics, including FDA- and EMEA-approved treatments for Fabry disease. Dr. Kizhner, who holds a Ph.D. in Biotechnology and Food Engineering from the Technion – Israel Institute of Technology, brings expertise and leadership to guide NurExone’s promising therapies through the upcoming stages of development and approvals. About NurExone NurExone Biologic Inc. is a TSXV and OTCQB listed pharmaceutical company that is developing a platform for biologically guided exosome-based therapies to be delivered, minimally invasive, to patients who have suffered Central Nervous System injuries. The Company’s first product, ExoPTEN for acute spinal cord injury, was proven to recover motor function in 75% of laboratory rats when administered intranasally. ExoPTEN has been granted Orphan Drug Designation by the FDA and European agency, European Medicines Agency. The NurExone platform technology is expected to offer novel solutions to drug companies interested in minimally invasive targeted drug delivery for other indications. For additional information and a brief interview, please watch Who is NurExone?, visit www.nurexone.com or follow NurExone on LinkedIn, Twitter, Facebook, or YouTube. For more information, please contact: Dr. Lior ShaltielChief Executive Officer and DirectorPhone: +972-52-4803034Email: info@nurexone.com Oak Hill Financial Inc.2 Bloor Street, Suite 2900Toronto, Ontario M4W 3E2Investor Relations - CanadaPhone: +1-647-479-5803Email: info@oakhillfinancial.ca Dr. Eva ReuterInvestor Relations - GermanyPhone: +49-69-1532-5857Email: e.reuter@dr-reuter.eu Allele Capital PartnersInvestor Relations - USPhone: +1 978-857-5075Email: aeriksen@allelecapital.com FORWARD-LOOKING STATEMENTS This press release contains certain “forward-looking statements” that reflect the Company’s current expectations and projections about its future results. Wherever possible, words such as “may”, “will”, “should”, “could”, “expect”, “plan”, “intend”, “anticipate”, “believe”, “estimate”, “predict” or “potential” or the negative or other variations of these words, or similar words or phrases, have been used to identify these forward-looking statements. Forward-looking statements in this press release include, but are not limited to, statements relating to: the Company completing the Offering on the terms indicated herein; the Company receiving all regulatory approvals; the use of proceeds from the Offering; the Company advancing towards clinical trials; Dr. Kizhner’s role within the Company; and the NurExone platform technology offering novel solutions to drug companies interested in minimally invasive targeted drug delivery for other indications. These statements reflect management’s current beliefs and are based on information currently available to management as at the date hereof. In developing the forward-looking statements in this press release, we have applied several material assumptions, including: the Company realizing on the benefits of exosomes; the Company will produce and supply exosomes for a wide range of applications; the ability of the Company’s products to be used for patient treatment; the Company fulfilling its intended future plans and expectations; there being growing clinical demand for innovative treatments in spinal cord, optic nerve, and other therapeutic areas; the Company carrying out its pre-clinical trials and realizing upon the benefits of the pre-clinical trials; the Company’s realizing upon the potential for exosome-loaded drugs in regenerating or repairing damaged nerves; the Company maintaining its ongoing commitment to using its ExoTherapy platform to advance the field of regenerative medicine and cell therapy applications; the Company will complete the Offering on the terms indicated herein; the Company will receive all regulatory approvals; the Company will use the proceeds from the Offering as outlined herein; the Company will advance to the clinical trial stage; Dr. Kizhner will be the director of R&D and provide the services as set out herein; and the NurExone platform technology will offer novel solutions to drug companies interested in minimally invasive targeted drug delivery for other indications. Forward-looking statements involve significant risk, uncertainties and assumptions. Many factors could cause actual results, performance or achievements to differ materially from the results discussed or implied in the forward-looking statements. These risks and uncertainties include, but are not limited to risks related to: the Company’s early stage of development; lack of revenues to date; government regulation; market acceptance for its products; rapid technological change; dependence on key personnel, including Dr. Kizhner; dependence on the Company’s strategic partners; the fact that preclinical drug development is uncertain, and the drug product candidates of the Company may never advance to clinical trials; the fact that results of preclinical studies and early-stage clinical trials may not be predictive of the results of later stage clinical trials; the uncertain outcome, cost, and timing of product development activities, preclinical studies and clinical trials of the Company; the uncertain clinical development process, including the risk that clinical trials may not have an effective design or generate positive results; the inability to obtain or maintain regulatory approval of the drug product candidates of the Company; the introduction of competing drugs that are safer, more effective or less expensive than, or otherwise superior to, the drug product candidates of the Company; the initiation, conduct, and completion of preclinical studies and clinical trials may be delayed, adversely affected or impacted by unforeseen issues; the inability to obtain adequate financing; the inability to obtain or maintain intellectual property protection for the drug product candidates of the Company; risks that the Company’s intellectual property and technology won’t have the intended impact on the Company and/or its business; the Company’s inability to carry out its pre-clinical trials and realize upon the stated benefits of the pre-clinical trials; the inability of the Company to realize on the benefits of exosomes; the inability of the Company to produce and/or supply exosomes for a wide range of applications; the inability of the Company’s products to be used for patient treatment; there not being broader adoption in the field and/or cell therapy applications; the inability of the Company to fulfill its intended future plans and expectations; there not being growing clinical demand for innovative treatments in spinal cord, optic nerve, and/or other therapeutic areas; the inability of the Company to collaborate with pharma companies; the Company’s inability to realize upon the stated potential for exosome-loaded drugs in regenerating or repairing damaged nerves; the Company’s inability to maintain its ongoing commitment to using its ExoTherapy platform to advance the field of regenerative medicine and/or cell therapy applications; the Company’s inability to expand into further studies; the Company’s inability to complete the Offering on the terms indicated herein or at all; the Company will not receive all required regulatory approvals; the Company will not use the proceeds from the Offering as outlined herein; the NurExone platform technology not offering novel solutions to drug companies interested in minimally invasive targeted drug delivery for other indications; and the risks discussed under the heading “Risk Factors” on pages 44 to 51 of the Company’s Annual Information Form dated August 27, 2024, a copy of which is available under the Company’s SEDAR+ profile at www.sedarplus.ca. These factors should be considered carefully, and readers should not place undue reliance on the forward-looking statements. Although the forward-looking statements contained in this press release are based upon what management believes to be reasonable assumptions, the Company cannot assure readers that actual results will be consistent with these forward-looking statements. These forward-looking statements are made as of the date of this press release, and the Company assumes no obligation to update or revise them to reflect new events or circumstances, except as required by law. Neither TSXV nor its Regulation Services Provider (as that term is defined in the policies of the TSXV) accepts responsibility for the adequacy or accuracy of this release.

Throughout 2024, the team at Fabry Disease News brought our readers all the latest updates on research related to Fabry disease and advances in diagnosis and treatment.Here is a list of the 10 most-read articles of last year. We look forward to continuing to be a resource to the Fabry community, and sharing informative stories with you, in 2025.A U.S. clinical trial designed to test a gene therapy for Fabry disease dosed its first participant in August. Fabry is caused by mutations in the GLA gene, and this therapy, called AMT-191, aims to deliver a healthy copy of the GLA gene to cells in the liver. The clinical trial, sponsored by AMT-191’s developer uniQure, is expected to test two doses of the gene therapy in a dozen patients, with a main goal of assessing the treatment’s safety. The trial is still recruiting adults with Fabry disease, ages 18 to 50, at sites in New York and Virginia. Eligible patients will have had a suboptimal response after at least one year of enzyme replacement therapy.Read this story hereData from two clinical trials testing a gene therapy for Fabry disease suggested that the experimental treatment may improve heart function. The trials are testing 4D-310, a gene therapy developed by 4D Molecular Therapeutics, that aims to deliver a healthy copy of the defective GLA gene specifically to cells in the heart — an organ that’s often hit hard by Fabry disease and that typically does not show much benefit with the use of currently available treatments. In addition to showing promising effects on heart function after one to two years, available data suggested that this gene therapy may help improve patients’ quality of life.Read this story hereLooking for damage to small nerve fibers may help doctors identify Fabry disease, according to a retrospective study of more than two dozen patients. Clinical outcomes in Fabry patients are generally best when treatment is started as early as possible, so diagnosing the disease quickly is crucial to get the best outcomes for patients, the researchers noted. Nonetheless, people with Fabry often face long delays in getting a correct diagnosis. This study found that many people with Fabry show evidence of damage to small nerve fibers, implying that looking for this type of damage could help aid in the diagnostic process.Read this story hereTreatment with Elfabrio (pegunigalsidase alfa) slowed the progression of kidney disease in adults with Fabry disease, data from a clinical trial showed. Elfabrio is an enzyme replacement therapy for Fabry that was developed by Protalix Biotherapeutics and Chiesi Global Rare Diseases. The treatment was approved in the U.S. last year based in part on data from a Phase 3 clinical trial. Final published data from the trial showed that treatment with Elfabrio slowed decline in kidney function, a common complication of Fabry disease.Read this story herePeople with Fabry disease have higher than normal levels of certain inflammatory proteins in their blood, a study found. Inflammation has long been thought to play a role in Fabry, but the link between inflammation and disease progression is not fully understood. This study found that Fabry patients with higher levels of inflammatory markers tend to have more severe disease. Data also suggested that approved Fabry treatments may help lower inflammatory markers.Read this story hereInterim data from the Phase 1/2 STAAR clinical trial that’s testing the safety of ST-920 (isaralgagene civaparvovec) — an experimental gene therapy for Fabry being developed by Sangamo Therapeutics — suggest that the treatment is well tolerated. Two serious safety issues, specifically one instance of fever and one of muscle pain, were reported among the trial’s more than 30 participants. Available data also hint that this gene therapy seems to be working as intended, with patients experiencing stabilized kidney function, lessened disease severity, and better life quality. The trial is expected to conclude later this year.Read this story hereAn international team of scientists created a mathematical model to help identify Fabry disease using data routinely collected by insurance providers. The new algorithm essentially works by looking for patterns of insurance claims that are more frequently seen in people with Fabry, with the model showing promise particularly for young and/or female potential patients. The researchers hope that this tool could ultimately help Fabry patients get diagnosed faster and more efficiently.Read this story hereEarly last year, the U.S. Food and Drug Administration signaled that data from one well-designed clinical trial could be enough to support approval of ST-920, Sangamo’s gene therapy for Fabry disease. The agency said that approval could potentially be considered based on data from one further trial, added to available evidence including data from the ongoing Phase 1/2 STAAR clinical study. ST-920 has been awarded orphan drug status in both the U.S. and the European Union — a designation aimed at speeding the development of treatments for rare diseases with various incentives.Read this story hereThe case of a 46-year-old man in Europe who was diagnosed with Fabry after complaining of an unexplained, chronic cough highlighted the symptom as a possible sign of the disease, according to a report by clinicians in Poland. Lung problems like chronic cough can be a symptom of Fabry, but they are often overlooked by clinicians, the researchers noted. As such, these scientists emphasized that doctors should consider the possibility of Fabry in people with unexplained lung symptoms, especially if other potential signs of the disease are present.Read this story hereClinicians caring for people with unexplained chronic pain should be on the lookout for Fabry disease, scientists argued in a study from France. Their report described outcomes from a program in which nearly 1,000 people with chronic pain were systematically evaluated for Fabry, leading to a diagnosis of the disorder in one individual. That result shows that, while Fabry is rare among people with chronic pain, it may be the cause of symptoms for some people.Read this story hereWe at Fabry Disease News hope these stories and all our reporting made us a helpful source of information throughout 2024, and we wish all our readers a bright new year!