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Data Highlight Positive Outcomes in Uncontrolled Gout With KRYSTEXXA ® and Progress in Addressing Sjögren's Disease THOUSAND OAKS, Calif., June 12, 2024 /PRNewswire/ -- Amgen (NASDAQ:AMGN) today announced the presentation of data across its diverse portfolio and pipeline at the European Alliance of Associations for Rheumatology's (EULAR) 2024 Congress, June 12-15 in Vienna. The 27 abstracts from Amgen-sponsored and partner-led studies demonstrate Amgen's commitment to improving the lives of patients living with inflammatory and rheumatic disease through the development of novel medicines. Key presentations include the first findings showing dazodalibep improved major biomarkers in Sjögren's disease, as well as data on treatment outcomes for uncontrolled gout based on a post-hoc analysis of the MIRROR trial for KRYSTEXXA® (pegloticase) with methotrexate. "We are deeply committed to advancing patient care through continuous innovation and rigorous science. The data presented at EULAR 2024 further demonstrate our efforts to address complex diseases," said Jay Bradner, M.D., executive vice president, Research and Development, and chief scientific officer at Amgen. "By advancing scientific research, we are not only enhancing our understanding of these conditions, but also paving the way for more effective treatments that have the potential to significantly improve patient outcomes." Additional presentations include new long-term data from the FOREMOST study of Otezla® (apremilast) in early oligoarticular psoriatic arthritis, as well as a post-hoc analysis of TAVNEOS® (avacopan) versus prednisone taper in patients with severe active MPA/GPA and active ear, nose or throat manifestations. For more information on the Amgen abstracts, see below. Abstracts and Presentation Times: Amgen-Sponsored Abstracts Dazodalibep CD40L blockade with dazodalibep significantly improves disease activity, swollen and tender joint counts, while reducing multiple T- and B-cell biomarkers in the MIDORA phase 2 study of patients with moderate-to-severe active rheumatoid arthritis Abstract #AB0746, Rheumatoid Arthritis (Publication only) Dazodalibep (anti-CD40L) effectively reduces multiple proteins associated with B-, T, and dendritic cell biomarkers in Sjögren's disease: Corroboration of immunophenotyping findings from the ALISS phase 2 study Abstract #OP0058, Clinical Abstract Sessions: New Targets and New Treatments in Sjön's Disease (Oral Abstract Presentations), Wednesday, June 12 from 5:00 to 5:10 p.m. CDT Sjögren's Disease Global burden of Sj ö gren's disease (SjD): Findings from a systematic literature review (SLR) of humanistic and clinical outcomes Abstract #AB0815, Sjögren`s syndrome (Publication Only) KRYSTEXXA® (pegloticase) Achievement of "gout remission" during intensive urate-lowering over 52 weeks of pegloticase therapy Abstract #POS0239, Clinical Poster Tours: Gout treatment in 2024 (Poster Tours), Friday, June 14 at 9:30 a.m. CDT Influence of acute gout flare and serum urate lowering on biomarkers of systemic inflammation Abstract #AB0114, Crystal related disorders (Publication Only) Patient-reported quality of life in uncontrolled gout and changes with intensive urate-lowering: Comparison of tophaceous and non-tophaceous patients Abstract #AB0112, Crystal related disorders (Publication Only) Pre-infusion glucocorticoid reduction/elimination in patients with uncontrolled gout treated with pegloticase and methotrexate: Experience of one community rheumatology practice Abstract #POS0936, Crystal related disorders (Poster View), Friday, June 14 at 9:30 a.m. CDT Uncontrolled Gout Cardiovascular disease, chronic kidney disease, pain, and psychological issues in patients with controlled vs. uncontrolled gout: A retrospective claims-based cohort analysis Abstract #POS0569, Crystal related disorders (Poster View), Wednesday, June 12 at 3:30 p.m. CDT The concept of gout remission as viewed by rheumatologists, nephrologists, and primary care physicians Abstract #POS0269, Clinical Poster Tours: Gout treatment in 2024 (Poster Tours), Friday, June 14 at 9:30 a.m. CDT Treatment patterns and quality of life in patients with controlled and uncontrolled gout Abstract #POS0940, Crystal related disorders (Poster View), Friday, June 14 at 9:30 a.m. CDT Otezla® (apremilast) Apremilast reduces axial inflammation in patients with psoriatic arthritis as assessed by CANDEN MRI scoring: Results from the phase 4 MOSAIC study Abstract #POS0982, Psoriatic arthritis (Poster View), Friday, June 14 at 9:30 a.m. CDT Apremilast reduces inflammation as measured by MRI, clinical outcomes, and patient-reported outcomes in patients with psoriatic arthritis: Results from the phase 4 MOSAIC study Abstract #POS0968, Psoriatic arthritis (Poster View), Friday, June 14 at 9:30 a.m. CDT Apremilast treatment in early oligoarticular psoriatic arthritis (PsA) improves clinical and patient-reported outcomes for up to 48 weeks – Data from the FOREMOST study Abstract #POS0976, Psoriatic arthritis (Poster View), Friday, June 14 at 9:30 a.m. CDT Apremilast for psoriatic arthritis in the Netherlands: Real-world data from the REWARD study Abstract #AB0453, Psoriatic arthritis (Publication Only) The use of disease activity thresholds for the Psoriatic Arthritis Impact of Disease (PsAID-12) questionnaire to assess patient perceptions of disease burden in patients with early oligoarticular psoriatic arthritis treated with apremilast in the FOREMOST study Abstract #AB0473, Psoriatic arthritis (Publication Only) Prolia® (denosumab) Comparative effectiveness of denosumab versus bisphosphonates among treatment-experienced postmenopausal women with osteoporosis in the U.S. Medicare program Abstract #POS0089, Clinical Poster Tours: Treatment Options in Metabolic bone diseases and Osteoporosis (Poster Tours), Thursday, June 13 at 9:54 a.m. CDT Comparative effectiveness of denosumab versus zoledronic acid among postmenopausal women with osteoporosis in the U.S. Medicare program Abstract #POS0583, Other diseases (Poster View), Thursday, June 13 at 9:30 a.m. CDT Risk of fragility fracture after long-term discontinuation of osteoporosis treatment in po st-menopausal osteoporosis women in France: A population-based study conducted on the nationwide claim database (SNDS) Abstract #OP0035, Clinical Abstract Sessions: Risk factors and treatment in osteoporosis (Oral Abstract Presentations), Wednesday, June 12 at 4:50 p.m. CDT TAVNEOS® (avacopan) Avacopan versus prednisone taper in patients with ANCA-associated vasculitis and ear, nose, or throat involvement Abstract #OP0174, Clinical Abstract Sessions: Vasculitis across different vessel sizes (Oral Abstract Presentations), Thursday, June 13 at 11:20 a.m. CDT Data from the ADVOCATE trial on 28 patients with ANCA-associated vasculitis who received avacopan without concomitant glucocorticoid use in the first 29 days Abstract #POS0246, Clinical Poster Tours: Miscellaneous in Vasculitis (Poster Tours), Friday, June 14 at 9:36 a.m. CDT UPLIZNA ® ( inebilizumab-cdon ) Burden of glucocorticoid use and associated toxicities in commercially insured adults with IgG4-related disease Abstract #POS0357, Clinical Poster Tours: IgG4 Related Disease (Poster Tours), Friday, June 14 at 2:45 p.m. CDT Insights into the design and study population of MITIGATE: The first multinational randomized controlled clinical trial in IgG4-related disease (IgG4-RD), evaluating the efficacy and safety of inebilizumab Abstract #POS0347, Clinical Poster Tours: IgG4 Related Disease (Poster Tours), Friday, June 14 at 2:45 p.m. CDT Partner-Led Abstracts Sjögren's Disease The use of natural language processing to characterize disease burden: Sexual distress in Sjögren's disease Abstract #AB0793, Sjön`s syndrome (Publication Only) Using social media listening to characterize the flare lexicon in patients with Sjögren's disease Abstract #AB0794, Sjön`s syndrome (Publication Only) TAVNEOS® (avacopan) Safety and effectiveness of avacopan beyond 52 weeks: Experience to date in the Early Access Program (EAP) Abstract #POS0865, Vasculitis, small and medium size vessels (Poster View), Thursday, June 13 at 2:45 p.m. CDT Design of AVACOSTAR: A real-world study of avacopan in ANCA-associated vasculitis (AAV) Abstract #AB1241, Vasculitis, small and medium size vessels (Publication Only) Uncontrolled Gout Osteoporosis in patients with gout in the United States Abstract #POS0563, Crystal related disorders (Poster View), Wednesday, June 12 at 3:30 p.m. CDT About KRYSTEXXA® (pegloticase) KRYSTEXXA ® (pegloticase) is the first and only biologic approved by the FDA to treat adults living with uncontrolled gout, a painful and debilitating inflammatory condition with which people continue to have abnormally high levels of uric acid and symptoms despite the use of conventional therapies. In 2022, the FDA approved expanding labeling to include co-administration with the immunomodulator methotrexate, based on results from the MIRROR randomized controlled trial, which showed significant improvements in efficacy and safety, including a reduction in infusion reactions. About Uncontrolled Gout Gout is a chronic, progressive inflammatory form of arthritis that is caused by high urate levels in the body. Tiny needle-like crystals can form and build up almost anywhere in the body. Patients with uncontrolled gout continue to have high levels of uric acid and ongoing symptoms of gout despite the use of oral urate-lowering therapies. Uncontrolled gout is a chronic, systemic disease, and if not addressed can have significant clinical consequences. KRYSTEXXA U.S. Indication KRYSTEXXA (pegloticase) is indicated for the treatment of chronic gout in adult patients who have failed to normalize serum uric acid and whose signs and symptoms are inadequately controlled with xanthine oxidase inhibitors at the maximum medically appropriate dose or for whom these drugs are contraindicated. Limitations of Use: KRYSTEXXA is not recommended for the treatment of asymptomatic hyperuricemia. KRYSTEXXA U.S. Important Safety Information WARNING: ANAPHYLAXIS AND INFUSION REACTIONS, G6PD DEFICIENCY ASSOCIATED HEMOLYSIS AND METHEMOGLOBINEMIA Anaphylaxis and infusion reactions have been reported to occur during and after administration of KRYSTEXXA. Anaphylaxis may occur with any infusion, including a first infusion and generally manifests within 2 hours of the infusion. Delayed hypersensitivity reactions have also been reported. KRYSTEXXA should be administered in healthcare settings and by healthcare providers prepared to manage anaphylaxis and infusion reactions. Patients should be premedicated with antihistamines and corticosteroids and closely monitored for anaphylaxis for an appropriate period after administration of KRYSTEXXA. Serum uric acid levels should be monitored prior to each infusion and treatment discontinued if levels increase to above 6 mg/dL, particularly when 2 consecutive levels above 6 mg/dL are observed. Patients at risk for glucose-6-phosphate dehydrogenase (G6PD) deficiency should be screened prior to starting KRYSTEXXA. Hemolysis and methemoglobinemia have been reported with KRYSTEXXA in patients with G6PD deficiency. KRYSTEXXA is contraindicated in patients with G6PD deficiency. CONTRAINDICATIONS In patients with G6PD deficiency. In patients with history of serious hypersensitivity reactions, including anaphylaxis, to KRYSTEXXA or any of its components. WARNINGS AND PRECAUTIONS Gout Flares: An increase in gout flares is frequently observed upon initiation of anti-hyperuricemic therapy, including KRYSTEXXA. Gout flare prophylaxis with a non-steroidal anti-inflammatory drug (NSAID) or colchicine is recommended starting at least 1 week before initiation of KRYSTEXXA therapy and lasting at least 6 months, unless medically contraindicated or not tolerated. Congestive Heart Failure: KRYSTEXXA has not been formally studied in patients with congestive heart failure, but some patients in the pre-marketing placebo-controlled clinical trials experienced exacerbation. Caution should be exercised in patients who have congestive heart failure and patients should be closely monitored following infusion. ADVERSE REACTIONS The most commonly reported adverse reactions (≥5%) are: KRYSTEXXA co-administration with methotrexate trial: gout flares, arthralgia, COVID-19, nausea and fatigue; KRYSTEXXA alone: gout flares, arthralgia, COVID-19, nausea, fatigue, infusion reactions, pain in extremity, hypertension and vomiting. KRYSTEXXA pre-marketing placebo-controlled trials: gout flares, infusion reactions, nausea, contusion or ecchymosis, nasopharyngitis, constipation, chest pain, anaphylaxis and vomiting. Please see Full Prescribing Information, including Boxed Warning. About Otezla® (apremilast) Otezla® (apremilast) is an oral small-molecule inhibitor of phosphodiesterase 4 (PDE4) specific for cyclic adenosine monophosphate (cAMP). PDE4 inhibition results in increased intracellular cAMP levels, which is thought to indirectly modulate the production of inflammatory mediators. The specific mechanism(s) by which Otezla exerts its therapeutic action in patients is not well defined. Since its initial FDA approval in 2014, Otezla has been prescribed to more than 840,000 patients worldwide. About Psoriatic Arthritis Psoriatic arthritis is a chronic, inflammatory form of arthritis, which can cause swelling, stiffness and pain in and around the joints that worsens over time and can decrease physical function. It is estimated that nearly 38 million people worldwide have psoriatic arthritis. Around a third of people living with psoriasis may go on to develop psoriatic arthritis. If left untreated, psoriatic arthritis can cause disability. Otezla U.S. Indications Otezla (apremilast) is indicated for the treatment of adult patients with plaque psoriasis who are candidates for phototherapy or systemic therapy. Otezla is indicated for the treatment of adult patients with active psoriatic arthritis. Otezla is indicated for the treatment of adult patients with oral ulcers associated with Behçet's Disease. Otezla U.S. Important Safety Information Contraindications Otezla is contraindicated in patients with a known hypersensitivity to apremilast or to any of the excipients in the formulation Warnings and Precautions Hypersensitivity: Hypersensitivity reactions, including angioedema and anaphylaxis, have been reported during postmarketing surveillance. If signs or symptoms of serious hypersensitivity reactions occur, discontinue Otezla and institute appropriate therapy Diarrhea, Nausea, and Vomiting: Cases of severe diarrhea, nausea, and vomiting were associated with the use of Otezla. Most events occurred within the first few weeks of treatment. In some cases, patients were hospitalized. Patients 65 years of age or older and patients taking medications that can lead to volume depletion or hypotension may be at a higher risk of complications from severe diarrhea, nausea, or vomiting. Monitor patients who are more susceptible to complications of diarrhea or vomiting; advise patients to contact their healthcare provider. Consider Otezla dose reduction or suspension if patients develop severe diarrhea, nausea, or vomiting Depression: Carefully weigh the risks and benefits of treatment with Otezla for patients with a history of depression and/or suicidal thoughts/behavior, or in patients who develop such symptoms while on Otezla. Patients, caregivers, and families should be advised of the need to be alert for the emergence or worsening of depression, suicidal thoughts or other mood changes, and they should contact their healthcare provider if such changes occur Plaque Psoriasis: Treatment with Otezla is associated with an increase in depression. During clinical trials in patients with moderate to severe plaque psoriasis, 1.3% (12/920) of patients reported depression compared to 0.4% (2/506) on placebo. Depression was reported as serious in 0.1% (1/1308) of patients exposed to Otezla, compared to none in placebo-treated patients (0/506). Suicidal behavior was observed in 0.1% (1/1308) of patients on Otezla, compared to 0.2% (1/506) on placebo. One patient treated with Otezla attempted suicide; one patient on placebo committed suicide Psoriatic Arthritis: Treatment with Otezla is associated with an increase in depression. During clinical trials, 1.0% (10/998) reported depression or depressed mood compared to 0.8% (4/495) treated with placebo. Suicidal ideation and behavior was observed in 0.2% (3/1441) of patients on Otezla, compared to none in placebo-treated patients. Depression was reported as serious in 0.2% (3/1441) of patients exposed to Otezla, compared to none in placebo-treated patients (0/495). Two patients who received placebo committed suicide compared to none on Otezla Behçet's Disease: Treatment with Otezla is associated with an increase in depression. During the clinical trial, 1% (1/104) reported depression or depressed mood compared to 1% (1/103) treated with placebo. No instances of suicidal ideation or behavior were reported in patients treated with Otezla or treated with placebo Weight Decrease: Monitor body weight regularly; evaluate unexplained or clinically significant weight loss, and consider discontinuation of Otezla Plaque Psoriasis: Body weight loss of 5-10% occurred in 12% (96/784) of patients with moderate to severe plaque psoriasis treated with Otezla and in 5% (19/382) of patients treated with placebo. Body weight loss of ≥10% occurred in 2% (16/784) of patients treated with Otezla compared to 1% (3/382) of patients treated with placebo Psoriatic Arthritis: Body weight loss of 5-10% was reported in 10% (49/497) of patients taking Otezla and in 3.3% (16/495) of patients taking placebo Behçet's Disease: Body weight loss of >5% was reported in 4.9% (5/103) of patients taking Otezla and in 3.9% (4/102) of patients taking placebo Drug Interactions: Apremilast exposure was decreased when Otezla was co-administered with rifampin, a strong CYP450 enzyme inducer; loss of Otezla efficacy may occur. Concomitant use of Otezla with CYP450 enzyme inducers (e.g., rifampin, phenobarbital, carbamazepine, phenytoin) is not recommended Adverse Reactions Plaque Psoriasis: The most common adverse reactions (≥ 5%) are diarrhea, nausea, upper respiratory tract infection, and headache, including tension headache. Overall, the safety profile of Otezla in patients with mild to moderate plaque psoriasis was consistent with the safety profile previously established in adult patients with moderate to severe plaque psoriasis Psoriatic Arthritis: The most common adverse reactions (≥ 5%) are diarrhea, nausea, and headache Behçet's Disease: The most common adverse reactions (≥ 10%) are diarrhea, nausea, headache, and upper respiratory tract infection. Use in Specific Populations Otezla has not been studied in pregnant women. Advise pregnant women of the potential risk of fetal loss. Please click here for Otezla® Full Prescribing Information. About TAVNEOS® (avacopan) TAVNEOS® (avacopan), approved by the FDA as an adjunctive treatment of severe active ANCA-associated vasculitis (GPA/MPA), is a first-in-class, orally administered small molecule that employs a novel, highly targeted mode of action. TAVNEOS blocks the complement 5a receptor (C5aR) from binding C5a, the pro-inflammatory complement system fragment. This is presumed to block C5a-mediated neutrophil activation and migration. The precise mechanism of TAVNEOS in ANCA-associated vasculitis (GPA/MPA) has not been definitively established. About ANCA-Associated Vasculitis ANCA-associated vasculitis is an umbrella term for a group of multi-system autoimmune diseases with small vessel inflammation. Inflamed vessels may rupture or become occluded giving rise to a broad array of clinical symptoms and signs related to a systemic inflammatory response which may result in profound impairment in the kidneys, lungs and other organs. Prior to the approval of TAVNEOS in severe active ANCA-associated vasculitis (GPA/MPA), the combination of immunosuppressants most often used for the treatment of ANCA-associated vasculitis (GPA/MPA) include cyclophosphamide or rituximab, combined with daily glucocorticoids (steroids) for prolonged periods, which can be associated with significant clinical consequences. TAVNEOS U.S. Indication TAVNEOS is indicated as an adjunctive treatment of adult patients with severe active anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis (granulomatosis with polyangiitis [GPA] and microscopic polyangiitis [MPA]) in combination with standard therapy including glucocorticoids. TAVNEOS does not eliminate glucocorticoid use. TAVNEOS U.S. Important Safety Information Contraindications Serious hypersensitivity to avacopan or to any of the excipients. Warning and Precautions Hepatotoxicity: Serious cases of hepatic injury have been observed in patients taking TAVNEOS, including life-threatening events. Obtain liver test panel before initiating TAVNEOS, every 4 weeks after start of therapy for six months and as clinically indicated thereafter. Monitor patients closely for hepatic adverse reactions, and consider pausing or discontinuing treatment as clinically indicated (refer to section 5.1 of the Prescribing Information). TAVNEOS is not recommended for patients with active, untreated and/or uncontrolled chronic liver disease (e.g., chronic active hepatitis B, untreated hepatitis C, uncontrolled autoimmune hepatitis) and cirrhosis. Consider the risk and benefit before administering this drug to a patient with liver disease. Serious Hypersensitivity Reactions: Cases of angioedema occurred in a clinical trial, including one serious event requiring hospitalization. Discontinue immediately if angioedema occurs and manage accordingly. TAVNEOS must not be re-administered unless another cause has been established. Hepatitis B Virus (HBV) Reactivation: Hepatitis B reactivation, including life threatening hepatitis B, was observed in the clinical program. Screen patients for HBV. For patients with evidence of prior infection, consult with physicians with expertise in HBV and monitor during TAVNEOS therapy and for six months following. If patients develop HBV reactivation, immediately discontinue TAVNEOS and concomitant therapies associated with HBV reactivation, and consult with experts before resuming. Serious Infections: Serious infections, including fatal infections, have been reported in patients receiving TAVNEOS. The most common serious infections reported in TAVNEOS group were pneumonia and urinary tract infections. Avoid use of TAVNEOS in patients with active, serious infection, including localized infections. Consider the risks and benefits before initiating TAVNEOS in patients with chronic infection, at increased risk of infection or who have been to places where certain infections are common. Adverse Reactions The most common adverse reactions (≥5% of patients and higher in the TAVNEOS group vs. prednisone group) were: nausea, headache, hypertension, diarrhea, vomiting, rash, fatigue, upper abdominal pain, dizziness, blood creatinine increased, and paresthesia. Drug Interactions Avoid coadministration of TAVNEOS with strong and moderate CYP3A4 enzyme inducers. Reduce TAVNEOS dose when co-administered with strong CYP3A4 enzyme inhibitors to 30 mg once daily. Monitor for adverse reactions and consider dose reduction of certain sensitive CYP3A4 substrates. Please see Full Prescribing Information and Medication Guide. About Amgen Amgen discovers, develops, manufactures and delivers innovative medicines to help millions of patients in their fight against some of the world's toughest diseases. More than 40 years ago, Amgen helped to establish the biotechnology industry and remains on the cutting-edge of innovation, using technology and human genetic data to push beyond what's known today. Amgen is advancing a broad and deep pipeline that builds on its existing portfolio of medicines to treat cancer, heart disease, osteoporosis, inflammatory diseases and rare diseases. In 2024, Amgen was named one of the "World's Most Innovative Companies" by Fast Company and one of "America's Best Large Employers" by Forbes, among other external recognitions. Amgen is one of the 30 companies that comprise the Dow Jones Industrial Average®, and it is also part of the Nasdaq-100 Index®, which includes the largest and most innovative non-financial companies listed on the Nasdaq Stock Market based on market capitalization. For more information, visit Amgen.com and follow Amgen on X, LinkedIn, Instagram, TikTok, YouTube and Threads. 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Results from a randomized, placebo-controlled study of TRIKAFTA® in people with cystic fibrosis with rare, non-F508del CFTR mutations showed statistically significant and clinically meaningful improvements in the primary and all secondary endpoints Interim results of largest real-world study of TRIKAFTA® showed sustained improvement in lung function at three years as well as lower rates of lung transplant and death in people with cystic fibrosis, compared to pre-TRIKAFTA® initiation BOSTON--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that data on TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor), also known in the European Union and in the U.K. as KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor, were presented at this year’s European Cystic Fibrosis Society’s (ECFS) 47th European Cystic Fibrosis Conference held June 5-8, 2024, in Glasgow, Scotland. Data from a randomized, double-blind, Phase 3 study (abstract WS06.04) demonstrated that people with CF who have rare, non-F508del mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene responsive to TRIKAFTA® in vitro demonstrated clinical benefit from receiving TRIKAFTA®. Compared to placebo, lung function improved by 9.2 percentage points as measured by ppFEV1, CFTR function improved (as measured by mean sweat chloride concentration reductions of 28.3 mmol/L), and pulmonary exacerbations were reduced by 72% per year. Safety and tolerability were generally consistent with the established safety pro TRIKAFTA®. Vertex also presented the interim analysis (IA) of a registry-based study of real-world data collected from people with CF initiating TRIKAFTA® from 2019-20 in the U.S. and KAFTRIO® plus ivacaftor from 2020-21 in Germany (abstract WS01.04). The ongoing five-year post-authorization study is the largest real-world study of people with CF treated with TRIKAFTA®/KAFTRIO® to date, including more than 16,000 people with CF from the U.S. Cystic Fibrosis Foundation Patient Registry (CFFPR) and approximately 3,000 people with CF from the German CF Registry. The IA showed clinically meaningful, disease-modifying benefits for TRIKAFTA®/KAFTRIO®, including a 76% and 70% reduction in the cumulative annual rate of pulmonary exacerbations in the U.S. and in Germany, respectively, compared to the year prior to TRIKAFTA®/KATRIO® treatment. In addition, there was a 62% lower rate of death in the U.S. and 84% lower in Germany; and an 86% lower rate of lung transplant in the U.S. and 96% lower in Germany compared to the 2019 U.S. CFFPR and German CF Registry populations (pre-TRIKAFTA®/KAFTRIO®). No new safety concerns were identified. “The breadth of TRIKAFTA data presented at ECFS is further evidence of the significant potential of this disease-modifying medicine,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. “These studies demonstrate that TRIKAFTA is changing the course of CF treatment and the lives of those living with CF.” Additional Presentations Other Vertex presentations at the conference this year include: Abstract WS15.02, entitled “Real-World Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in People With Cystic Fibrosis and ELX/TEZ/IVA-Responsive, Non-F508del CFTR Genotypes” Abstract EPS10.08, entitled “LONGITUDE: An Observational Study of the Long-term Effectiveness of ELX/TEZ/IVA in People With CF Using Data From the UK CF Registry – Preliminary Results From the Subgroup Aged 6-11 Years” Abstract P096, entitled “Qualitative Interviews Confirming the Faithful Electronic Migration of the Preschool Pictorial Cystic Fibrosis Questionnaire-Revised (CFQ-R) and Parent Preschool CFQ-R” Abstract EPS6.05, entitled “Clinical Outcomes in Concurrent Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Treated vs. Ineligible Cohorts in the US Cystic Fibrosis Foundation Patient Registry (CFFPR) During COVID-19” Abstract P063, entitled “ELX/TEZ/IVA has beneficial effects on clinical outcomes and quality of life in people with cystic fibrosis in the real-world TRAJECTORY study” Abstract P072, entitled “Real-World Impact of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Italy: A Retrospective Study From a CF Center” About Cystic Fibrosis Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 92,000 people globally. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF, and these mutations can be identified by a genetic test. While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. CFTR mutations lead to CF by causing CFTR protein to be defective or by leading to a shortage or absence of CFTR protein at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic lung infections and progressive lung damage that eventually leads to death for many patients. The median age of death is in the 30s, but with treatment, projected survival is improving. Today Vertex CF medicines are treating over 65,000 people with CF across 60 countries on six continents. This represents 2/3 of the diagnosed people with CF eligible for CFTR modulator therapy. About TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) In people with certain types of mutations in the CFTR gene, the CFTR protein is not processed or folded normally within the cell, and this can prevent the CFTR protein from reaching the cell surface and functioning properly. TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is an oral medicine designed to increase the quantity and function of the CFTR protein at the cell surface. Elexacaftor and tezacaftor work together to increase the amount of mature protein at the cell surface. Ivacaftor, which is known as a CFTR potentiator, is designed to facilitate the ability of CFTR proteins to transport salt and water across the cell membrane. The combined actions of elexacaftor, tezacaftor and ivacaftor help hydrate and clear mucus from the airways. TRIKAFTA U.S. INDICATIONS AND IMPORTANT SAFETY INFORMATION FOR INDICATIONS AND USAGE TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients aged 2 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or another mutation that is responsive to treatment with TRIKAFTA. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if TRIKAFTA is safe and effective in children under 2 years of age. IMPORTANT SAFETY INFORMATION Before taking TRIKAFTA, patients should tell their doctor about all of their medical conditions, including if they: are allergic to TRIKAFTA or any ingredients in TRIKAFTA, have kidney problems, have or have had liver problems, are pregnant or plan to become pregnant because it is not known if TRIKAFTA will harm an unborn baby, or are breastfeeding or planning to breastfeed because it is not known if TRIKAFTA passes into breast milk. Patients should tell their doctor about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. TRIKAFTA may affect the way other medicines work, and other medicines may affect how TRIKAFTA works. The dose of TRIKAFTA may need to be adjusted when taken with certain medicines. Patients should ask their doctor or pharmacist for a list of these medicines if they are not sure. Patients should especially tell their doctor if they take: antibiotics such as rifampin or rifabutin; seizure medicines such as phenobarbital, carbamazepine, or phenytoin; St. John’s wort; antifungal medicines including ketoconazole, itraconazole, posaconazole, voriconazole, or fluconazole; antibiotics including telithromycin, clarithromycin, or erythromycin. Patients should avoid food or drink that contains grapefruit while taking TRIKAFTA. TRIKAFTA can cause serious side effects, including: Liver damage and worsening of liver function in patients with severe liver disease that can be serious and may require transplantation. Liver damage has also happened in patients without liver disease. High liver enzymes in the blood, which is a common side effect in patients treated with TRIKAFTA. These can be serious and may be a sign of liver injury. The patient’s doctor will do blood tests to check their liver before they start TRIKAFTA, every 3 months during the first year of taking TRIKAFTA, and every year while taking TRIKAFTA. Patients should call their doctor right away if they have any of the following symptoms of liver problems: pain or discomfort in the upper right stomach (abdominal) area; yellowing of the skin or the white part of the eyes; loss of appetite; nausea or vomiting; dark, amber-colored urine. Serious allergic reactions have happened to patients who are treated with TRIKAFTA. Call your healthcare provider or go to the emergency room right away if you have any symptoms of an allergic reaction. Symptoms of an allergic reaction may include: rash or hives; tightness of the chest or throat or difficulty breathing; swelling of the face, lips and/or tongue; difficulty swallowing; and light-headedness or dizziness. Abnormality of the eye lens (cataract) has been noted in some children and adolescents treated with TRIKAFTA. If the patient is a child or adolescent, their doctor should perform eye examinations before and during treatment with TRIKAFTA to look for cataracts. The most common side effects of TRIKAFTA include headache, upper respiratory tract infection (common cold) including stuffy and runny nose, stomach (abdominal) pain, diarrhea, rash, increase in liver enzymes, increase in a certain blood enzyme called creatine phosphokinase, flu (influenza), inflamed sinuses, and increase in blood bilirubin. Patients should tell their doctor if they have any side effect that bothers them or that does not go away. These are not all the possible side effects of TRIKAFTA. For more information, patients should ask their doctor or pharmacist. Please click here to see the full U.S. Prescribing Information for TRIKAFTA. About KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor In people with certain types of mutations in the CFTR gene, the CFTR protein is not processed or folded normally within the cell, and this can prevent the CFTR protein from reaching the cell surface and functioning properly. KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor is an oral medicine designed to increase the quantity and function of the CFTR protein at the cell surface. Elexacaftor and tezacaftor work together to increase the amount of mature protein at the cell surface by binding to different sites on the CFTR protein. Ivacaftor, which is known as a CFTR potentiator, is designed to facilitate the ability of CFTR proteins to transport salt and water across the cell membrane. The combined actions of ivacaftor, tezacaftor and elexacaftor help hydrate and clear mucus from the airways. KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor is approved in the European Union and in the U.K. for the treatment of cystic fibrosis (CF) in patients aged 2 years and older who have at least one copy of the F508del mutation in the CFTR gene. For complete product information, please see the Summary of Product Characteristics that can be found on and on . About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has approved medicines that treat the underlying causes of multiple chronic, life-shortening genetic diseases — cystic fibrosis, sickle cell disease and transfusion-dependent beta thalassemia — and continues to advance clinical and research programs in these diseases. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including acute and neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes, myotonic dystrophy type 1 and alpha-1 antitrypsin deficiency. Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 14 consecutive years on Science magazine's Top Employers list and one of Fortune’s 100 Best Companies to Work For. For company updates and to learn more about Vertex's history of innovation, visit or follow us on LinkedIn, Facebook, Instagram, YouTube and Twitter/X. Special Note Regarding Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements by Carmen Bozic, M.D. in this press release, and statements regarding our expectations for the benefits of TRIKAFTA/KAFTRIO. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company’s studies may not be indicative of final clinical trial results, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy, or other reasons, that data may not be available on the anticipated timeline, or at all, that our development programs may experience delays, and other risks listed under the heading “Risk Factors” in Vertex's most recent annual report and subsequent filings filed with the Securities and Exchange Commission at and available through the company's website at . You should not place undue reliance on these statements or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. (VRTX-GEN) View source version on businesswire.com: Contacts Vertex Pharmaceuticals Incorporated Investors: InvestorInfo@vrtx.com Media: mediainfo@vrtx.com or U.S.: 617-341-6992 or International: +44 20 3204 5275 Source: Vertex Pharmaceuticals Incorporated View this news release online at: