Epinephrine Bitartrate

TEANECK, N.J.--(BUSINESS WIRE)--GC Biopharma USA, Inc., a leader in plasma-derived products, announced today that it will present two new analyses and host a patient advocacy symposium at the IgNS 2026 15th National Conference, taking place October 15–18 in Las Vegas, Nevada. The two poster presentations include an expanded multi-lot assessment of aggregation profiles across five commercially available 10% intravenous immunoglobulin (IVIG) products and a post hoc descriptive review of selected safety outcomes from the Phase 3 study of ALYGLO® (immune globulin intravenous, human-stwk, 10% liquid). “These analyses provide additional insight into both the characteristics of commercially available IVIG products and selected safety outcomes from the ALYGLO Phase 3 study,” said Stacey Ness, PharmD, IgCP, CSP, MSCS, AAHIVP, Medical Science Liaison. In the aggregation analysis, all five products demonstrated high purity and low aggregate levels, with statistically significant differences observed among the products. The Phase 3 analysis provides additional characterization of selected events of special interest (ESIs) based on the Clinical Study Report (CSR). No treatment-related thromboembolic events, acute renal failure, deaths, or adverse event-related discontinuations were observed. “These analyses provide additional insight into both the characteristics of commercially available IVIG products and selected safety outcomes from the ALYGLO Phase 3 study,” said Stacey Ness, PharmD, IgCP, CSP, MSCS, AAHIVP, Medical Science Liaison, Medical Affairs, GC Biopharma USA. “Expanding the aggregation study across multiple lots strengthens our understanding of product-to-product differences, while the clinical study report analysis adds detail on events of special interest that were not fully characterized in the original publication.” The expanded aggregation analysis evaluated six lots of ALYGLO and three lots each of four other commercially available 10% IVIG products. Statistically significant differences were observed among the products in monomer and dimer content, fragment content, and particle size. No statistically significant differences were observed in polymer levels or particle size dispersion. Further research is needed to determine the clinical relevance of the observed differences in aggregate attributes. The second poster reports a post hoc, CSR-only descriptive review of selected ESIs and serious adverse events from the Phase 3 ALYGLO clinical trial. Treatment-emergent serious adverse events were reviewed as additional safety context using available subject-level and CSR-reported subgroup characteristics, including age, sex, and infusion schedule. The review found no clinically meaningful thromboembolic, renal, hematologic, blood chemistry, DAT/Coombs, or viral safety signals. However, subjects with a history of thromboembolism and kidney disease were excluded from the clinical trial, which may limit the generalizability of the findings to higher-risk populations. The two posters will be presented during the IgNS poster session on Saturday, October 17, 2026, from 5:00 p.m. to 6:30 p.m. PDT. Expanded Assessment of the Aggregation Profile of Commercially Available Intravenous Immunoglobulin Products Safety in a Phase 3 Study of IVIG-stwk 10%: A Descriptive Review of Events of Special Interest (ESIs) and Serious Adverse Events (SAEs) GC Biopharma will also host the symposium, “Advocacy Across Borders: Global Perspectives, Individual Impact,” on Saturday, October 17, from 7:30 a.m. to 9:00 a.m. PDT in the Summerlin Ballroom. Presenters include: Lynn H. Albizo, JD, Chief Public Policy Officer, Immune Deficiency Foundation Suzanne Strasters, MSN-FNP, IgCN, Head of Clinical Education, GC Biopharma USA Laura Remy-Martin, PhD, MPH, ACRP-CP, Field Medical Science Liaison, GC Biopharma USA Albizo will discuss patient advocacy and public policy and their impact on people with immune deficiencies, drawing on her experience leading legislative and regulatory advocacy at the Immune Deficiency Foundation. As a company with more than 50 years of expertise in plasma protein manufacturing, GC Biopharma is committed to advancing scientific understanding of IVIG therapies and supporting research that may help inform patient care. About ALYGLO® ALYGLO® (immune globulin intravenous, human-stwk) is a glycine-stabilized 10% immunoglobulin G (100 mg/mL) for intravenous infusion, manufactured from pooled human plasma from US donors. The manufacturing process includes multiple steps to reduce the risk of virus transmission. These include solvent/detergent treatment and 20 nm nanofiltration. The ALYGLO manufacturing process also uses G-XI™ Technology, its novel cation exchange (CEX) chromatography, that removes FXIa to undetectable levels.1,2 For more information about ALYGLO and G-XI™ Technology, visit www.alyglo.com. About GC Biopharma GC Biopharma (formerly known as Green Cross Corporation) is a biopharmaceutical company that delivers lifesaving and life-sustaining protein therapeutics and vaccines. Headquartered in Yongin, South Korea, GC Biopharma is a leading global plasma protein and vaccine product manufacturer dedicated to quality healthcare solutions for over half a century. About GC Biopharma USA GC Biopharma USA, headquartered in Teaneck, NJ, is an operations and distribution company of GC Biopharma, that established its sales, marketing, and business operations in 2018 to serve customers and patients throughout the US. Our foundation is built on the expertise of our parent company GC Biopharma, a leading biopharmaceutical company delivering plasma therapies and vaccines worldwide for more than 50 years. With GC Biopharma USA, GC Biopharma further extends its footprint, bringing its expertise and legacy to the US. For more information, visit www.gcbiopharma.us. INDICATION ALYGLO® is indicated for the treatment of primary humoral immunodeficiency (PI) in adults aged 17 years and older. This includes, but is not limited to, congenital agammaglobulinemia, common variable immunodeficiency (CVID), Wiskott-Aldrich syndrome, and severe combined immunodeficiencies. IMPORTANT SAFETY INFORMATION WARNING: THROMBOSIS, RENAL DYSFUNCTION and ACUTE RENAL FAILURE Thrombosis may occur with immune globulin intravenous (IGIV) products, including ALYGLO. Risk factors may include: advanced age, prolonged immobilization, hypercoagulable conditions, history of venous or arterial thrombosis, use of estrogens, indwelling vascular catheters, hyperviscosity, and cardiovascular risk factors. Renal dysfunction, acute renal failure, osmotic nephropathy, and death may occur with the administration of IGIV products in predisposed patients. Renal dysfunction and acute renal failure occur more commonly in patients receiving IGIV products containing sucrose. ALYGLO does not contain sucrose. For patients at risk of thrombosis, renal dysfunction or renal failure, administer ALYGLO at the minimum dose and infusion rate practicable. Ensure adequate hydration in patients before administration. Monitor for signs and symptoms of thrombosis and assess blood viscosity in patients at risk for hyperviscosity. Contraindications: ALYGLO is contraindicated in patients who have a history of anaphylactic or severe systemic reaction to the administration of human immune globulin and in IgA-deficient patients with antibodies to IgA and a history of hypersensitivity. Hypersensitivity: In case of hypersensitivity, discontinue infusion immediately and institute appropriate treatment. Epinephrine should be available for immediate treatment of severe acute hypersensitivity reactions. Hyperproteinemia, Increased Serum Viscosity, and Hyponatremia: Hyperproteinemia, increased serum viscosity, and hyponatremia may occur. Aseptic Meningitis Syndrome (AMS): Aseptic meningitis syndrome (AMS) may occur, especially with high doses or rapid infusion. AMS usually begins within several hours to 2 days following ALYGLO treatment. Discontinuation of treatment has resulted in remission of AMS within several days without sequelae. Hemolysis: Delayed hemolytic anemia due to enhanced red blood cell (RBC) sequestration and acute hemolysis consistent with intravascular hemolysis have been reported. Cases of severe hemolysis-related renal dysfunction/failure or disseminated intravascular coagulation have occurred following infusion of IGIV. Closely monitor patients for clinical signs and symptoms of hemolysis, particularly patients with risk factors. Transfusion-Related Acute Lung Injury: Noncardiogenic pulmonary edema (transfusion-related acute lung injury [TRALI]) may occur. TRALI is characterized by severe respiratory distress, pulmonary edema, hypoxemia, normal left ventricular function, and fever. Patients with TRALI may be managed using oxygen therapy with adequate ventilator support. Monitor patients for pulmonary adverse reactions. Transmissible Infectious Agents: Because ALYGLO is made from human blood, it may carry a risk of transmitting infectious agents (e.g., viruses, the variant Creutzfeldt-Jakob disease [vCJD] agent and, theoretically, the Creutzfeldt-Jakob disease [CJD] agent). Interference with Laboratory Tests: After infusion of immunoglobulin, the transitory rise of the various passively transferred antibodies in the patient’s blood may yield positive serological testing results, with the potential for a misleading interpretation. Adverse reactions (observed in ≥ 5% of study subjects) were headache, nausea/vomiting, fatigue, nasal/sinus congestion, rash, arthralgia, diarrhea, muscle pain/aches, infusion site pain/swelling, abdominal pain/discomfort, cough, and dizziness. It is recommended that ALYGLO be administered separately from other drugs or medications. Please see Important Safety Information for ALYGLO on the preceding pages and refer to the full Prescribing Information (PI) or visit Alyglo.com. If you have an inquiry related to drug safety, or to report adverse events, please contact GC Biopharma USA at 1-833-426-6426 or email medicalinfo@gcbiopharmausa.com. You can also visit FDA.gov/medwatch or call 1-800-FDA-1088. This press release may contain forward-looking statements that express the current beliefs and expectations of the management at GC Biopharma and GC Biopharma USA. Such views do not represent any guarantee by either entity or its government of future performance and involve known and unknown risks, uncertainties, and other factors. GC Biopharma and GC Biopharma USA undertake no obligation to update or revise any forward-looking statement contained in this press release or any other forward-looking statements they may make, except as required by law or stock exchange rule. References: 1. Kang GB, Huber A, Lee J, et al. Cation exchange chromatography removes FXIa from a 10% intravenous immunoglobulin preparation. Front Cardiovasc Med. 2023;10:1253177. 2. ALYGLO Prescribing Information. GC Biopharma; 2025.

Efzimfotase alfa showed improvement across primary and all key secondary endpoints including in fatigue and pain vs placebo at week 25 with continued improvement up to week 49 Efzimfotase alfa recently granted Priority Review in the US Results from the HICKORY Phase III trial showed that efzimfotase alfa (ALXN1850), an investigational enzyme replacement therapy, did not achieve statistical significance but demonstrated a numerical improvement in the primary endpoint of Six-Minute Walk Test (6MWT) in adolescents and adults (≥12 years of age) with hypophosphatasia (HPP) who had not been previously treated with STRENSIQ®(asfotase alfa) at week 25, compared to placebo.1 In the overall population, at week 25, efzimfotase alfa achieved a least squares (LS) mean difference of 17.05 meters (p=0.1675) change from baseline in the primary endpoint of 6MWT, compared to placebo. The lack of statistical significance was largely due to better-than-expected results observed in the adult-onset HPP placebo group.1 In a post hoc analysis, at week 25, 45.2% of patients treated with efzimfotase alfa achieved the minimum clinically important difference (MCID) from baseline in 6MWT (defined as 43 meters in adolescents and 31.1 meters in adults), compared to 30.0% in the placebo group (odds ratio = 2.50 [95% CI: 1.06, 5.91; nominal p=0.0363]), indicating a 2.5-fold higher odds of achieving a clinically meaningful 6MWT response.1 In the overall population, at week 25, efzimfotase alfa demonstrated nominally significant improvement in the key secondary endpoint of FACIT-Fatigue, achieving an LS mean difference of 5.74 (nominal p=0.0016) vs placebo. Other key secondary endpoints numerically favored efzimfotase alfa, including 30-second Sit-to-Stand (30s-STS), Lower Extremity Functional Scale (LEFS) and BPI-SF Pain Severity.1 In the open-label extension, at week 49, patients treated with efzimfotase alfa showed continued improvement across the primary and key secondary endpoints, including 6MWT, 30s-STS, LEFS, BPI-SF Pain Severity and FACIT-Fatigue, and patients switching from placebo to efzimfotase alfa following the randomized evaluation period at week 25 demonstrated similar improvement in these key measures of mobility, physical function, pain and fatigue.1 In a combination of prespecified subgroups of adolescents and adults with pediatric-onset HPP, efzimfotase alfa demonstrated nominally significant and clinically meaningful benefits in the primary endpoint of 6MWT, achieving an LS mean difference of 35.6 meters (nominal p=0.0386) vs placebo at week 25, as well as the key secondary endpoints of 30s-STS (LS mean difference vs placebo: 1.3; nominal p=0.0384) and BPI-SF Pain Severity (LS mean difference vs placebo: -1.0; nominal p=0.0260). In a prespecified subgroup of adults with adult-onset HPP, efzimfotase alfa demonstrated clinically meaningful improvement in the key secondary endpoint of LEFS (LS mean difference vs placebo: 3.5; nominal p=0.2376) and nominally significant and clinically meaningful improvement in FACIT-Fatigue (LS mean difference vs placebo: 7.41; nominal p=0.0021).1 Kathryn Dahir, MD, Director of the Program for Metabolic Bone Disorders at Vanderbilt Health and Associate Director for Clinical Research Translation, Professor in the Department of Internal Medicine, Division of Endocrinology, Diabetes and Metabolism and lead investigator in the HICKORY trial, said: “For adolescents and adults, the chronic and progressive disease burden of HPP can have far-reaching impact on physical function and emotional wellbeing, potentially impairing people’s ability to live independently, form connections and sustain employment. These results from the HICKORY Phase III trial signal benefits across multiple clinically relevant endpoints and represent an important step toward potentially bringing this advanced enzyme replacement therapy to a broad range of people who may benefit.” Gianluca Pirozzi, Senior Vice President, Head of Development, Regulatory and Safety, Alexion, AstraZeneca Rare Disease, said: “The HICKORY Phase III trial builds on the positive results from the MULBERRY and CHESTNUT trials and demonstrates the potential of efzimfotase alfa to address the diverse and complex clinical manifestations of HPP across a broad population of patients. With the recently granted Priority Review in the US, we look forward to bringing this potential new therapy with favorable tolerability and dosing convenience to the HPP community, including those with limited treatment options.” Summary of results at week 25: HICKORY Efzimfotase alfa demonstrated a favorable safety profile and was generally well-tolerated across all three Phase III clinical trials. In a pooled analysis of MULBERRY and HICKORY trials, the annualized Injection Site Reaction (ISR) rate was five times lower with efzimfotase alfa compared to STRENSIQin its registrational trials during the first 24 weeks of treatment. In that analysis, including data from the open-label extension, patients treated with efzimfotase alfa achieved a median of 98.8% ISR-free days while on treatment.1 Results from the HICKORY Phase III trial were presented today in an oral session at the 2026 American Society for Bone and Mineral Research (ASBMR) Annual Meeting in Boston, Massachusetts. Results from the MULBERRY and CHESTNUT trials were recently presented at the 12th International Conference on Children’s Bone Health (ICCBH). STRENSIQ® (asfotase alfa) INDICATION & IMPORTANT SAFETY INFORMATION INCLUDING BOXED WARNING INDICATION STRENSIQ (asfotase alfa) is indicated for the treatment of patients with perinatal/infantile- and juvenile-onset hypophosphatasia (HPP). IMPORTANT SAFETY INFORMATION INCLUDING BOXED WARNING WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Initiate STRENSIQ under the supervision of a healthcare provider with appropriate medical monitoring and support measures. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue STRENSIQ and immediately initiate appropriate medical treatment, including use of epinephrine. Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur [see Warnings and Precautions (5.1)]. WARNINGS AND PRECAUTIONS Life-threatening hypersensitivity reactions, including anaphylaxis, have been reported in STRENSIQ-treated patients. Signs and symptoms consistent with anaphylaxis included difficulty breathing, choking sensation, nausea, periorbital edema, and dizziness. These reactions have occurred within minutes after subcutaneous administration of STRENSIQ and have been observed more than 1 year after treatment initiation. Other hypersensitivity reactions have also been reported in STRENSIQ-treated patients, including vomiting, fever, headache, flushing, irritability, chills, erythema, rash, pruritus, and oral hypoesthesia. Consider the risks and benefits of re-administering STRENSIQ following a severe reaction. If the decision is made to re-administer, monitor patients for a reoccurrence of signs and symptoms of a severe hypersensitivity reaction. Lipodystrophy: Localized lipodystrophy, including lipoatrophy and lipohypertrophy has been reported at injection sites after several months in patients treated with STRENSIQ in clinical trials. Advise patients to follow proper injection technique and to rotate injection sites. Ectopic Calcifications: Patients with HPP are at increased risk for developing ectopic calcifications. Events of ectopic calcification, including ophthalmic (conjunctival and corneal) and renal (nephrocalcinosis, nephrolithiasis), have been reported in the clinical trial experience with STRENSIQ. There was insufficient information to determine whether the reported events were consistent with the disease or due to STRENSIQ. No visual changes or changes in renal function were reported resulting from the occurrence of ectopic calcifications. Ophthalmology examinations and renal ultrasounds are recommended at baseline and periodically during treatment with STRENSIQ to monitor for signs and symptoms of ophthalmic and renal ectopic calcifications and for changes in vision or renal function. Possible Immune-Mediated Clinical Effects: In clinical trials, most STRENSIQ-treated patients developed anti-asfotase alfa antibodies and neutralizing antibodies which resulted in reduced systemic exposure of asfotase alfa. In postmarketing reports, some STRENSIQ-treated patients with initial therapeutic response subsequently developed recurrence and worsening in disease-associated laboratory and radiographic biomarkers (some in association with neutralizing antibodies) suggesting possible immune-mediated effects on STRENSIQ’s pharmacologic action resulting in disease progression. The effect of anti-asfotase alfa antibody formation on the long-term efficacy of STRENSIQ is unknown. There are no marketed anti-asfotase alfa antibody tests. If patients experience progression of HPP symptoms or worsening of disease-associated laboratory and imaging biomarkers after a period of initial therapeutic response to STRENSIQ, consider obtaining anti-asfotase alfa antibody testing by contacting STRENSIQ Medical Information at Alexion at 1-888-765-4747 or by email at medinfo@alexion.com. Close clinical follow up is recommended. ADVERSE REACTIONS In clinical trials, the most common adverse reactions (≥ 10%) reported were injection site reactions (63%), lipodystrophy (28%), ectopic calcifications (14%), and hypersensitivity reactions (12%). Possible immune-mediated clinical effects have been identified during post-approval use of STRENSIQ. DRUG INTERACTIONS Drug Interference with Laboratory Tests: Laboratory tests utilizing alkaline phosphatase (ALP) as a detection reagent could result in erroneous test results for patients receiving treatment due to the presence of asfotase alfa in clinical laboratory samples. Inform laboratory personnel that the patient is being treated with STRENSIQ and discuss use of an alternative testing platform which does not utilize an ALP-conjugated test system. Elevated serum ALP measurements detected through clinical laboratory testing are expected in patients receiving STRENSIQ due to circulating concentrations of asfotase alfa. Do not rely on serum ALP measurements for clinical decision making in patients treated with STRENSIQ. SPECIAL POPULATIONS Pregnancy & Lactation: There are no available data on STRENSIQ use in pregnant women, the presence of STRENSIQ in human milk, or the effects on the breastfed infant or on milk production, to inform a drug associated risk. To report SUSPECTED ADVERSE REACTIONS, contact Alexion Pharmaceuticals, Inc. at 1-844-259-6783 or FDA at 1- 800-FDA-1088 or www.fda.gov/medwatch. Please see full Prescribing Information for STRENSIQ (asfotase alfa), including Boxed WARNING regarding hypersensitivity reactions including anaphylaxis. Notes Hypophosphatasia Hypophosphatasia (HPP) is a rare, chronic, inherited metabolic disease caused by deficient activity of the enzyme alkaline phosphatase (ALP), which is important for building healthy bones and supporting proper muscle function.2 HPP is characterized by defective mineralization (the process that hardens and strengthens bones and teeth), impaired calcium and phosphate regulation and functional impairments, such as muscle weakness, neurologic symptoms, generalized fatigue and pain that can be debilitating.2,3 HPP can be progressive, and clinical manifestations may occur at any age and evolve over time. The addressable HPP patient population is estimated at 13,800 across the US, Germany, France, UK, Italy, Spain, Japan and China.4 HPP affects people of all ages, with approximately 80% of people living with HPP being adults based on a US prevalence study.2,3,5 MULBERRY MULBERRY is a global Phase III randomized, double-blind, placebo-controlled, multicenter trial evaluating the efficacy and safety of efzimfotase alfa (ALXN1850) in pediatric patients (2 to <12 years of age) with hypophosphatasia (HPP) who have not been previously treated with STRENSIQ(asfotase alfa). The trial enrolled 29 patients from 14 countries across North America, South America, Europe and Asia.6 Patients were required to have an HPP diagnosis and the presence of HPP-related rickets on skeletal X-rays and low serum alkaline phosphatase (ALP) activity. Eligible patients also needed to demonstrate either a variant in ALPL, the gene encoding ALP, or elevated levels of plasma pyridoxal 5'-phosphate (PLP), a biomarker of HPP.6 Patients were randomized 2:1 to receive efzimfotase alfa at one of three doses based on predefined weight ranges or placebo, once every two weeks via subcutaneous injection for 24 weeks. The primary endpoint Radiographic Global Impression of Change (RGI-C) Score was assessed at the end of the randomized evaluation period (Day 169), along with multiple secondary endpoints measuring skeletal health and physical function, including change from baseline in the Rickets Severity Score (RSS), Six-Minute Walk Test (6MWT), Bruininks-Oseretsky Test of Motor Proficiency Score (BOT-2) and Peabody Developmental Motor Scales Score (PDMS-3).6 Patients who completed the randomized evaluation period were eligible to continue into an open-label extension period evaluating the safety and efficacy of efzimfotase alfa, which is ongoing.6 CHESTNUT CHESTNUT is a global Phase III randomized, open-label, active-controlled, multicenter trial evaluating the safety and tolerability of efzimfotase alfa in pediatric patients (2 to <12 years of age) with hypophosphatasia (HPP) who have been treated with 6 mg/kg per week of STRENSIQ(asfotase alfa) for at least 6 months prior to study initiation. The trial enrolled 43 patients from seven countries globally.7 Patients were required to have an HPP diagnosis and have been treated with STRENSIQfor at least 6 months before the start of the trial with open growth plates confirmed by X-ray.7 Patients were randomized 1:1 to receive efzimfotase alfa at one of three doses based on predefined weight ranges once every two weeks or 6 mg/kg/week of STRENSIQ via 3x or 6x subcutaneous injections per week for 24 weeks. The primary endpoint is the incidence of treatment-emergent adverse events (TEAEs) at the end of the randomized evaluation period. Key secondary endpoints include change from baseline in the Rickets Severity Score (RSS) and Radiographic Global Impression of Change (RGI-C).7 Patients who completed the randomized evaluation period were eligible to continue into an open-label extension period evaluating the safety and efficacy of efzimfotase alfa, which is ongoing.7 HICKORY HICKORY is a global Phase III randomized, double-blind, placebo-controlled, multicenter trial evaluating the efficacy and safety of efzimfotase alfa (ALXN1850) in adolescents (12 to <18 years of age) and adults with hypophosphatasia (HPP) who have not been previously treated with STRENSIQ(asfotase alfa).The trial enrolled 124 patients from 17 countries across North America, South America, Europe, Asia and Australia.8 Patients were required to have an HPP diagnosis and either a variant in ALPL, the geneencoding alkaline phosphatase (ALP), or elevated levels of plasma pyridoxal 5'-phosphate (PLP), a biomarker of HPP. Eligible patients needed to demonstrate low ALP levels and two separate Six-Minute Walk Tests (6MWTs) below 85% of the predicted distance adjusted for age, sex, weight and height, without a probable cause other than HPP.8 Patients were randomized 2:1 to receive efzimfotase alfa at one of three doses based on predefined weight ranges or placebo, once every two weeks via subcutaneous injection for 24 weeks. The primary endpoint of change from baseline in 6MWT was assessed at the end of the randomized evaluation period (Day 169), along with multiple key secondary endpoints measuring physical function, pain, fatigue, quality of life and safety, including change from baseline in 30-second Sit to Stand (STS) Test Score, Lower Extremity Functional Scale (LEFS) Score, Brief Pain Inventory Short Form (BPI-SF) Score and Functional Assessment of Chronic Illness Therapy – Fatigue (FACIT-Fatigue) Score.8 Patients who completed the randomized evaluation period were eligible to continue into an open-label extension period evaluating the safety and efficacy of efzimfotase alfa, which is ongoing.8 Efzimfotase Alfa (ALXN1850) Efzimfotase alfa (ALXN1850) is an investigational enzyme replacement therapy (ERT) designed to demonstrate efficacy and safety in a broad range of patients with hypophosphatasia (HPP) aged ≥ 2 years, including patients without overt bone manifestations. Efzimfotase alfa is being developed as a subcutaneous treatment administered every two weeks to replace the deficient alkaline phosphatase (ALP) enzyme activity that is the underlying cause of HPP. Efzimfotase alfa has been granted Priority Review by the US FDA for the treatment of patients with HPP aged 2 years and older. STRENSIQ®(asfotase alfa) STRENSIQ® is an enzyme replacement therapy designed to address the underlying cause of hypophosphatasia (HPP) – deficient alkaline phosphatase (ALP). By replacing deficient ALP, treatment with STRENSIQ aims to improve the elevated enzyme substrate levels and improve the body's ability to mineralize bone, thereby preventing serious skeletal and systemic patient morbidity and premature death. STRENSIQ is approved in the US, EU, Japan and other countries for the treatment of certain patients with HPP. STRENSIQ has been granted orphan drug designation by the US Food and Drug Administration (FDA), the European Medicines Agency (EMA) and the Japanese Ministry of Health, Labour and Welfare (MHLW). Alexion Alexion, AstraZeneca Rare Disease, is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and delivery of life-changing medicines. A pioneering leader in rare disease for more than three decades, Alexion was the first to translate the complex biology of the complement system into transformative medicines, and today it continues to build a diversified pipeline across disease areas with significant unmet need, using an array of innovative modalities. As part of AstraZeneca, Alexion is continually expanding its global geographic footprint to serve more rare disease patients around the world. It is headquartered in Boston, US. For more information, please visit www.alexion.us. AstraZeneca AstraZeneca (LSE/STO/NYSE: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialization of prescription medicines in Oncology, Rare Disease, and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca’s innovative medicines are sold in more than 125 countries and used by millions of patients worldwide. Please visit astrazeneca-us.com and follow the Company on social media @AstraZeneca. Media Inquiries US Media Mailbox: usmediateam@astrazeneca.com   References 1. Efficacy and Safety of Alkaline Phosphatase (ALP) Enzyme Replacement Therapy (ERT) Efzimfotase Alfa in Adolescents and Adults with Hypophosphatasia (HPP): Results of HICKORY as Part of a Three-Trial Phase 3 Clinical Program. Presented at the American Society for Bone and Mineral Research (ASBMR) Annual Meeting; 2026 October 11; Boston, Massachusetts. 2. Rockman-Greenberg C. Hypophosphatasia. Pediatr Endocrinol Rev. 2013;10(2):380-388. 3. Dahir KM, et al. Clinical profiles of treated and untreated adults with hypophosphatasia in the Global HPP Registry. Orphanet J Rare Dis. 2022;17(1):277. 4. AstraZeneca Data on File - Epidemiology estimates are composed of a triangulation of different data sources including Data Monitor, Decision Resources Group, Kantar Health, and internal input. Available here. Accessed October 2026. 5. Fang S, et al. Diagnosed prevalence of hypophosphatasia: a retrospective analysis of electronic health records in the United States. Poster presented at ASBMR 2025 Annual Meeting; September 5-8, 2025; Seattle, WA. 6. ClinicalTrials.gov. Phase 3 study of ALXN1850 in treatment-naïve pediatric participants with HPP (MULBERRY). NCT Identifier: NCT06079359. Available here. Accessed October 2026. 7. ClinicalTrials.gov. Phase 3 study of ALXN1850 in pediatric participants with HPP previously treated with asfotase alfa (CHESTNUT). NCT Identifier: NCT06079372. Available here. Accessed October 2026. 8. ClinicalTrials.gov. Phase 3 study of ALXN1850 versus placebo in adolescent and adult participants with HPP who have not previously been treated with asfotase alfa (HICKORY). NCT Identifier: NCT06079281. Available here. Accessed October 2026. You are now leaving AstraZeneca-us.com You have selected a link that will take you to a site maintained by a third party who is solely responsible for its contents. AstraZeneca provides this link as a service to website visitors. AstraZeneca is not responsible for the privacy policy of any third party websites. We encourage you to read the privacy policy of every website you visit. Click ‘cancel’ to return to AstraZeneca’s site or ‘continue’ to proceed. Important notice for users You are about to access AstraZeneca historic archive material. 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