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Marked and rapid reductions in both pericarditis pain and inflammation
maintained throughout the 26-week study
Episodes of pericarditis per year substantially reduced
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Cardiol Therapeutics' Phase II MAvERIC-Pilot Clinical Results in Recurrent Pericarditis Presented at the American Heart Association Scientific Sessions 2024
MAvERIC-Pilot results support advancing CardiolRx™ into the Phase II/III MAVERIC-2
and the Phase III MAVERIC-3 clinical trials
Cardiol Therapeutics Inc. (NASDAQ: CRDL) (TSX: CRDL) ("Cardiol" or the "Company"), a clinical-stage life sciences company focused on the research and clinical development of anti-inflammatory and anti-fibrotic therapies for the treatment of heart disease, today reported clinical results from its Phase II open-label MAvERIC-Pilot study investigating the impact of CardiolRx™ administered to patients with symptomatic recurrent pericarditis. The data showed that the marked improvements in both pericarditis pain and inflammation, previously reported at the 8-week primary endpoint, were maintained throughout the extension period of the 26-week study. The data were included in an oral presentation as part of the Laennec Clinician-Educator Award & Lecture at the American Heart Association Scientific Sessions 2024. Dr. S. Allen Luis, Co-Director of the Pericardial Diseases Clinic and Associate Professor of Medicine in the Department of Cardiovascular Medicine at the Mayo Clinic, presented on behalf of the MAvERIC-Pilot investigators. These findings support the initiation of a Phase III trial (MAVERIC-3), designed to assess CardiolRx™ for the treatment of pericarditis patients to prevent recurrence. The MAVERIC-3 trial is expected to run in parallel with the recently announced MAVERIC-2 Phase IIIII trial designed to evaluate the impact of CardiolRx™ in recurrent pericarditis patients following cessation of interleukin-1 blocker therapy.
"The data reported today show that patients enrolled in MAvERIC-Pilot, despite the severity of their disease, experienced clinically relevant and rapid reductions in both their pericarditis pain and C-reactive protein levels that were maintained throughout the study. In addition, results demonstrated a substantial reduction in pericarditis episodes per year as compared to the patients' historical event rate prior to the study. Importantly, treatment was shown to be safe and well tolerated in a population who presented with significant disease burden," said Dr. S. Allen Luis, Co-Director of the Pericardial Diseases Clinic and Associate Professor of Medicine in the Department of Cardiovascular Medicine at the Mayo Clinic. "I look forward to further investigation in the upcoming Phase II/III and Phase III clinical trials."
The MAvERIC-Pilot study enrolled 27 participants (average age 53 years; 67% female) at eight clinical sites across the United States. Average disease duration and the number of pericarditis episodes per year prior to trial entry were 2.7 years and 5.8 events per year, respectively. Baseline pericarditis pain score averaged 5.8 out of 10 and the C-reactive protein ("CRP") level averaged 2.0 mg/dL. In addition to pericarditis chest pain, other manifestations of pericarditis-confirmed diagnosis were pericardial effusion in 21 patients (78%), pericardial rub in 4 (15%), and ST-segment elevation or PR depression in 5 (19%). Stable doses of baseline medications for recurrent pericarditis, in any combination, included colchicine (85% of patients), non-steroidal anti-inflammatory drugs (78%), and corticosteroids (41%). The 26-week study consisted of an 8-week treatment period ("TP") followed by an 18-week extension period ("EP"). In the first 10 days of the TP, CardiolRx™ was added to baseline medications for recurrent pericarditis and up-titrated to 10 mg/kg twice daily, or the maximum tolerated dose. Throughout the TP, patients continued receiving this concomitant therapy but were weaned off baseline medications during the EP to assess pericarditis recurrence while on CardiolRx™ monotherapy.
Summary of results:
- Primary endpoint of patient-reported pericardial pain on an 11-point numerical rating scale from 0-10 showed a mean reduction of 3.7, from 5.8 at baseline (range of 4 to 10) to 2.1 (range of 0 to 6) at week 8.
- Median time to resolution or near resolution of pain (defined as a score of ≤ 2) was rapid and was observed just 5 days following initiation of CardiolRx™ treatment.
- Reduction in pain was maintained throughout the duration of the trial with a mean reduction of 4.3, from 5.8 at baseline to 1.5 at week 26.
- At week 8, 93% (25/27) of patients reported a pain score reduction.
- CRP normalized (≤0.5 mg/dL) at week 8 in 80% (8/10) of the patients with a baseline CRP of ≥1 mg/dL, with a substantial mean reduction of 5.4 mg/dL being observed (5.7mg/dL to 0.3 mg/dL).
- CRP levels for the entire group of patients were reduced from 2.0 mg/dL at baseline to 0.74 and 0.55 at weeks 8 and 26 respectively, with a median time to CRP normalization of 21 days.
- Freedom from recurrence was maintained in 71% (17/24) of patients during the EP when CardiolRx™ was continued and patients were weaned off baseline medications. For those patients experiencing a recurrence the median time to an episode was 7.7 weeks during the EP.
- Number of pericarditis episodes per year was markedly reduced from 5.8 prior to study to 0.9 during the study.
- CardiolRx™ was shown to be safe and well tolerated with eighty-nine percent of patients (24/27) progressing to the EP and overall study drug compliance reported at 95%.
"The compelling results from MAvERIC-Pilot showed that CardiolRx™ resulted in marked and rapid reductions in pericarditis pain and inflammation in patients with a high degree of disease burden as well as a striking decrease in pericarditis episodes per year. The notable impact of CardiolRx™ on these important clinical endpoints demonstrates its potential to offer a more accessible and non-immunosuppressive therapeutic option for tens of thousands of pericarditis patients," said David Elsley, President and CEO of Cardiol Therapeutics. "These results further support advancing our late-stage MAVERIC clinical development program comprising our recently announced Phase II/III MAVERIC-2 trial as well as our planned MAVERIC-3 Phase III trial. Undertaking both trials in parallel provides the exciting opportunity for CardiolRx™ to address the unmet needs of patients in multiple segments that encompass a broad proportion of the pericarditis population."
MAvERIC-PILOT Phase II Study
To be eligible for enrollment in MAvERIC-Pilot, adult patients (≥18 years) were required to present with at least their third pericarditis episode, which included symptomatic pericarditis chest pain with a numerical rating scale ("NRS") pain score ≥4 (on an 11-point numerical rating scale ("NRS") of 0-10), together with either an elevated level of CRP ≥1 mg/dL, a clinical marker of inflammation, or evidence of pericardial inflammation assessed by cardiac imaging with or without elevated CRP. NRS is a validated instrument used to assess patient-reported pericarditis pain. Zero represents 'no pain at all', whereas the upper limit of 10 represents 'the worst pain ever possible'. At baseline eligible patients were permitted to be receiving stable doses of concomitant medications for recurrent pericarditis (non-steroidal anti-inflammatory drugs and/or colchicine and/or oral corticosteroid therapy in any combination).
Pericarditis
Pericarditis refers to inflammation of the pericardium (the membrane or sac that surrounds the heart) frequently resulting from a viral infection. Following that initial episode patients may have multiple recurrences, and the primary goal of treatment is recurrence prevention. Symptoms include debilitating chest pain, shortness of breath and fatigue, resulting in physical limitations, reduced quality of life, emergency department visits, and hospitalizations. Significant accumulation of pericardial fluid and scarring can progress to life-threatening constriction of the heart. The only FDA-approved therapy for recurrent pericarditis, launched in 2021, is costly and is primarily used as a third-line intervention. On an annual basis, the number of patients in the United States having experienced at least one recurrence is estimated at 38,000. Approximately 60% of patients with multiple recurrences (>1) still suffer for longer than two years, and one third are still impacted at five years. Hospitalization due to recurrent pericarditis is often associated with a 6-8-day length of stay and cost per stay is estimated to range between $20,000 and $30,000 in the United States.
About Cardiol Therapeutics
Cardiol Therapeutics Inc. (NASDAQ: CRDL) (TSX: CRDL) is a clinical-stage life sciences company focused on the research and clinical development of anti-inflammatory and anti-fibrotic therapies for the treatment of heart disease. The Company's lead small molecule drug candidate, CardiolRx™ (cannabidiol) oral solution, is pharmaceutically manufactured and in clinical development for use in the treatment of heart disease. It is recognized that cannabidiol inhibits activation of the inflammasome pathway, an intracellular process known to play an important role in the development and progression of inflammation and fibrosis associated with myocarditis, pericarditis, and heart failure.
Cardiol has received Investigational New Drug Application authorization from the United States Food and Drug Administration ("US FDA") to conduct clinical studies to evaluate the efficacy and safety of CardiolRx™ in two diseases affecting the heart: recurrent pericarditis and acute myocarditis. The MAVERIC Program in recurrent pericarditis, an inflammatory disease of the pericardium which is associated with symptoms including debilitating chest pain, shortness of breath, and fatigue, and results in physical limitations, reduced quality of life, emergency department visits, and hospitalizations, comprises the Phase II MAvERIC-Pilot study (NCT05494788), the Phase II/III MAVERIC-2 trial, and the planned Phase III MAVERIC-3 trial. The ARCHER trial (NCT05180240) is a Phase II study in acute myocarditis, an important cause of acute and fulminant heart failure in young adults and a leading cause of sudden cardiac death in people less than 35 years of age. The US FDA has granted Orphan Drug Designation to CardiolRx™ for the treatment of pericarditis, which includes recurrent pericarditis.
Cardiol is also developing CRD-38, a novel subcutaneously administered drug formulation intended for use in heart failure – a leading cause of death and hospitalization in the developed world, with associated healthcare costs in the United States exceeding $30 billion annually.
For more information about Cardiol Therapeutics, please visit cardiolrx.com.
Cautionary statement regarding forward-looking information:
This news release contains "forward-looking information" within the meaning of applicable securities laws. All statements, other than statements of historical fact, that address activities, events, or developments that Cardiol believes, expects, or anticipates will, may, could, or might occur in the future are "forward-looking information". Forward looking information contained herein may include, but is not limited to statements regarding the Company's plans to expand the MAVERIC clinical development program and advance CardiolRx™ into the Phase II/III MAVERIC-2 and the Phase III MAVERIC-3 clinical trials, the Company's plans to conduct the MAVERIC-2 and MAVERIC-3 studies in parallel, the MAVERIC-3 Phase III study being designed to assess CardiolRx™ for the treatment of the broader population of pericarditis patients to prevent recurrence, the Company's focus on developing anti-inflammatory and anti-fibrotic therapies for the treatment of heart disease, the molecular targets and mechanism of action of the Company's product candidates, the Company's intended clinical studies and trial activities and timelines associated with such activities, including the Company's plan to complete the Phase III study in recurrent pericarditis with CardiolRx, and the Company's plan to advance the development of CRD-38, a novel subcutaneous formulation of cannabidiol intended for use in heart failure. Forward-looking information contained herein reflects the current expectations or beliefs of Cardiol based on information currently available to it and is based on certain assumptions and is also subject to a variety of known and unknown risks and uncertainties and other factors that could cause the actual events or results to differ materially from any future results, performance or achievements expressed or implied by the forward looking information, and are not (and should not be considered to be) guarantees of future performance. These risks and uncertainties and other factors include the risks and uncertainties referred to in the Company's Annual Report on Form 20-F filed with the U.S. Securities and Exchange Commission and Canadian securities regulators on April 1, 2024, as well as the risks and uncertainties associated with product commercialization and clinical studies. These assumptions, risks, uncertainties, and other factors should be considered carefully, and investors should not place undue reliance on the forward-looking information, and such information may not be appropriate for other purposes. Any forward-looking information speaks only as of the date of this press release and, except as may be required by applicable securities laws, Cardiol disclaims any intent or obligation to update or revise such forward-looking information, whether as a result of new information, future events, or results, or otherwise. Investors are cautioned not to rely on these forward-looking statements and are encouraged to read the Supplement, the accompanying Base Prospectus and the documents incorporated by reference therein.
For further information, please contact:
Trevor Burns, Investor Relations +1-289-910-0855
trevor.burns@cardiolrx.com
To view the source version of this press release, please visit https://www.newsfilecorp.com/release/230401
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Radiopharm Theranostics CEO Shares Company Milestones as NASDAQ Listing Nears
Canevari provided some updates about the joint venture with MD Anderson Cancer Center and the progress with the B7-H3 targeting radio-antibody (BetaBart). BetaBart is the first targeted radiopharmaceutical against the 4Ig subtype of B7-H3, the most common subtype expressed in human tumors.
“Management is initially targeting small cell lung cancer but sees additional opportunities in colon, renal, lung, cervical, prostate and glioma cancers,” Canevari said. The joint venture is expecting to undertake its first-in-human Phase 1/2 therapeutic trial by mid-2025.
Canevari also outlined the progress of RAD’s focus programs including RAD101 for imaging brain metastases; RAD204 in NSCLC; RAD301 imaging in pancreatic cancer; and RAD202 in breast/gastric:
- RAD 101, an F18 radiolabeled Pivalate for brain metastases, received FDA IND approval for a Phase 2b trial aiming to enroll 30 patients, with the first dose expected in Q4 2024 and results anticipated mid-2025.
- RAD 204, a Lu-177-labeled nanobody targeting PD-L1 in NSCLC, recently dosed its first patient in a Phase 1 dose-escalating trial in Australia.
- RAD 301, a 68Ga-labeled αvβ6-integrin for imaging Pancreatic Ductal Adenocarcinoma, also recently dosed its first patient in a Phase 1 trial at Montefiore Medical Center, building on positive compassionate use data from 99 patients.
- RAD 402, targeting advanced prostate cancer with Tb-161, which has shown higher tumor uptake compared to Lu-177.
RAD initiated the process to obtain asecondary listing on the Nasdaq Capital Market in 2023, which the company expects to be completed by the end of 2024.
Highlights of the report
- Clinical Updates -- RAD has advanced several clinical programs which include RAD 101 with the first dose expected in Q4 2024.
- Collaborations with Leading Industry Players -- RAD has increased its ownership in Radiopharm Ventures to 75 percent from 51 percent.
- NASDAQ Listing Expected YE 2024 -- RAD is progressing toward a secondary listing on the NASDAQ Capital Market by the end of 2024.
- Cash Runway Into June 2026 -- In June 2024, RAD announced a capital raise of AU$70 million (USD $47million)
For the full analyst report, click here.
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AMGEN PRESENTS NEW DATA ACROSS RARE INFLAMMATORY DISEASES AT ACR 2024
MITIGATE Phase 3 Study Results Reinforce Promise of UPLIZNA ® as the First Potential Treatment tor IgG4-RD
Phase 4 AGILE Data Support Shortening KRYSTEXXA ® Infusion Time
Amgen (NASDAQ:AMGN) today announced the presentation of new data across its rare disease portfolio and pipeline at the annual American College of Rheumatology (ACR) Convergence 2024 conference in Washington, D.C. Nov. 14-19, 2024 . New data showcase reduction in disease activity by UPLIZNA® (inebilizumab-cdon) in Immunoglobulin G4-Related Disease (IgG4-RD) and support shorter infusion times for KRYSTEXXA® (pegloticase) co-administered with weekly oral methotrexate 15 mg.
"These data add to the growing body of evidence for UPLIZNA and KRYSTEXXA and strengthen our commitment to developing new treatment options for rare diseases like IgG4-RD and uncontrolled gout," said Jay Bradner , M.D., executive vice president of Research and Development and chief scientific officer at Amgen. "Patients living with these debilitating conditions deserve new approaches targeting the underlying causes of disease, potentially improving outcomes and enhancing the overall treatment experience."
Key presentations include:
A Phase 3, Randomized, Double-Blind, Multicenter, Placebo-Controlled Study of Inebilizumab in IgG4-Related Disease (MITIGATE): Primary Efficacy and Safety Findings
Abstract #0775, Abstract Session: Saturday, Nov. 16 from 1:00 p.m. – 1:15 p.m. ET
MITIGATE, the first randomized, double-blind, placebo-controlled study ever conducted in IgG4-RD, evaluated the safety and efficacy of CD19+ B-cell depletion with UPLIZNA.
Key findings include*:
- A clinically meaningful and statistically significant 87% reduction in the risk of IgG4-RD flare compared to placebo (Hazard Ratio 0.13, p
- A reduction in annualized flare rate for treated and adjudication committee-determined flares during the placebo-controlled period; 0.10 for participants receiving UPLIZNA compared to 0.71 for participants receiving placebo (p
- 57.4% (39 of 68) of participants receiving UPLIZNA achieved flare-free, treatment-free, complete remission at Week 52 compared to 22.4% (15 of 67) participants receiving placebo (p
- 58.8% (40 of 68) of participants receiving UPLIZNA achieved flare-free, corticosteroid-free, complete remission at Week 52 compared to 22.4% (15 of 67) participants receiving placebo (p
- Confirmation of the unique mechanism of action of UPLIZNA to deliver rapid and sustained depletion of peripheral B cells leading to lowered levels of disease biomarkers. Flares are indicative of high disease activity.
Notably, 89.7% (61 of 68) of UPLIZNA-treated patients required no glucocorticoid treatment for disease control during the placebo-controlled period, compared to 37.3% (25 of 67) of patients on placebo. After Week 8, UPLIZNA-treated patients experienced a ten-fold reduction in total glucocorticoid use relative to placebo.
The safety results in the placebo-controlled period were consistent with the established safety profile of UPLIZNA. The most common treatment-emergent adverse events included COVID-19, lymphopenia, urinary tract infection, and headache.
The data were simultaneously published in the New England Journal of Medicine . In August, the U.S. Food and Drug Administration granted Breakthrough Therapy Designation for UPLIZNA in IgG4-RD based on data from the MITIGATE study, and regulatory filing activities are currently underway.
*All p-values follow the New England Journal of Medicine reporting guidelines; values smaller than 0.001 are presented as 0.001.
Safety, Tolerability and Efficacy of Pegloticase Administered with a Shorter Infusion Duration in Subjects with Uncontrolled Gout Receiving Methotrexate: Primary Findings of the AGILE Open-label Trial
Abstract #2012, Poster Session C: Monday, Nov. 18 from 10:30 p.m. – 12:30 p.m. ET
The AGILE trial assessed the safety, tolerability and efficacy of KRYSTEXXA administered with a shorter infusion duration in patients with uncontrolled gout receiving methotrexate as co-administration.
Safety and efficacy data from the 60-minute infusion duration cohort of the AGILE trial are similar to the MIRROR randomized clinical trial and current administration of KRYSTEXXA with methotrexate over at least 120 minutes.
Key findings include:
- 67.2% (78 of 116) of participants receiving a 60-minute infusion duration of KRYSTEXXA with methotrexate achieved and maintained a response during Month 6, defined as a urate level of
- 6.0% (7 of 116) of participants receiving a 60-minute infusion duration of KRYSTEXXA with methotrexate experienced an infusion reaction, including anaphylaxis (1.7%; 2 of 116 participants), based on adjudicated results.
Regulatory filings for the AGILE study findings are currently underway.
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.
About KRYSTEXXA ® (pegloticase)
KRYSTEXXA 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.
KRYSTEXXA ® (pegloticase) U.S. Indication
KRYSTEXXA 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.
- Premedicate with antihistamines and corticosteroids and closely monitor for anaphylaxis for an appropriate period after administration of KRYSTEXXA.
- Monitor serum uric acid levels prior to each infusion and discontinue treatment if levels increase to above 6 mg/dL, particularly when 2 consecutive levels above 6 mg/dL are observed.
- Screen patients at risk for glucose-6-phosphate dehydrogenase (G6PD) deficiency 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. Exercise caution in patients who have congestive heart failure and monitor patients closely following infusion.
ADVERSE REACTIONS
The most commonly reported adverse reactions (≥5%) are:
- KRYSTEXXA co-administration with methotrexate: 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 Immunoglobulin G4-related disease (IgG4-RD)
Immunoglobulin G4-related disease (IgG4-RD) is a chronic, systemic, immune-mediated, fibroinflammatory disease which can affect numerous and generally multiple organs of the body. It is a progressive disease affecting new organs over time either consecutively or simultaneously and is characterized by periods of remission and unpredictable disease flares. IgG4-RD can cause irreversible organ damage with or without the presence of symptoms. Awareness of how organ damage manifests is critically important to inform the timely diagnosis of IgG4-RD. B cells are central to the pathogenesis of IgG4-RD. In IgG4-RD, CD19-expressing (CD19+) B cells are thought to drive inflammatory and fibrotic processes and interact with other immune cells that contribute to disease activity.
The incidence is estimated at 1-5 in 100,000 although the number of IgG4-RD patients is difficult to determine based on limited epidemiology data. The typical age of onset of IgG4-RD is between 50 and 70 years old and, unlike many other immune-mediated diseases, IgG4-RD is more likely to occur in men than women.
About UPLIZNA ® (inebilizumab-cdon)
UPLIZNA is a humanized monoclonal antibody (mAb) that causes targeted and sustained depletion of key cells that contribute to underlying disease process (autoantibody-producing CD19+ B cells, including plasmablasts and some plasma cells). After two initial infusions, patients need one dose of UPLIZNA every six months.
UPLIZNA is currently approved for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive in the United States and other countries around the world.
UPLIZNA ® (inebilizumab-cdon) U.S. INDICATION
UPLIZNA is indicated for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive.
IMPORTANT SAFETY INFORMATION
UPLIZNA is contraindicated in patients with:
- A history of life-threatening infusion reaction to UPLIZNA
- Active hepatitis B infection
- Active or untreated latent tuberculosis
WARNINGS AND PRECAUTIONS
Infusion Reactions: UPLIZNA can cause infusion reactions, which can include headache, nausea, somnolence, dyspnea, fever, myalgia, rash, or other symptoms. Infusion reactions were most common with the first infusion but were also observed during subsequent infusions. Administer pre-medication with a corticosteroid, an antihistamine, and an anti-pyretic.
Infections: The most common infections reported by UPLIZNA-treated patients in the randomized and open-label periods included urinary tract infection (20%), nasopharyngitis (13%), upper respiratory tract infection (8%), and influenza (7%). Delay UPLIZNA administration in patients with an active infection until the infection is resolved.
Increased immunosuppressive effects are possible if combining UPLIZNA with another immunosuppressive therapy.
The risk of Hepatitis B Virus (HBV) reactivation has been observed with other B-cell-depleting antibodies. Perform HBV screening in all patients before initiation of treatment with UPLIZNA. Do not administer to patients with active hepatitis.
Although no confirmed cases of Progressive Multifocal Leukoencephalopathy (PML) were identified in UPLIZNA clinical trials, JC virus infection resulting in PML has been observed in patients treated with other B-cell-depleting antibodies and other therapies that affect immune competence. At the first sign or symptom suggestive of PML, withhold UPLIZNA and perform an appropriate diagnostic evaluation.
Patients should be evaluated for tuberculosis risk factors and tested for latent infection prior to initiating UPLIZNA.
Vaccination with live-attenuated or live vaccines is not recommended during treatment and after discontinuation, until B-cell repletion.
Reduction in Immunoglobulins: There may be a progressive and prolonged hypogammaglobulinemia or decline in the levels of total and individual immunoglobulins such as immunoglobulins G and M (IgG and IgM) with continued UPLIZNA treatment. Monitor the level of immunoglobulins at the beginning, during, and after discontinuation of treatment with UPLIZNA until B-cell repletion especially in patients with opportunistic or recurrent infections.
Fetal Risk: May cause fetal harm based on animal data. Advise females of reproductive potential of the potential risk to a fetus and to use an effective method of contraception during treatment and for 6 months after stopping UPLIZNA.
Adverse Reactions: The most common adverse reactions (at least 10% of patients treated with UPLIZNA and greater than placebo) were urinary tract infection and arthralgia.
For additional information on UPLIZNA, please see the Full Prescribing Information at www.UPLIZNA.com .
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 .
Forward-Looking Statements
This news release contains forward-looking statements that are based on the current expectations and beliefs of Amgen. All statements, other than statements of historical fact, are statements that could be deemed forward-looking statements, including any statements on the outcome, benefits and synergies of collaborations, or potential collaborations, with any other company (including BeiGene, Ltd. or Kyowa Kirin Co., Ltd.), the performance of Otezla® (apremilast) (including anticipated Otezla sales growth and the timing of non-GAAP EPS accretion), our acquisitions of Teneobio, Inc., ChemoCentryx, Inc., or Horizon Therapeutics plc (including the prospective performance and outlook of Horizon's business, performance and opportunities, any potential strategic benefits, synergies or opportunities expected as a result of such acquisition, and any projected impacts from the Horizon acquisition on our acquisition-related expenses going forward), as well as estimates of revenues, operating margins, capital expenditures, cash, other financial metrics, expected legal, arbitration, political, regulatory or clinical results or practices, customer and prescriber patterns or practices, reimbursement activities and outcomes, effects of pandemics or other widespread health problems on our business, outcomes, progress, and other such estimates and results. Forward-looking statements involve significant risks and uncertainties, including those discussed below and more fully described in the Securities and Exchange Commission reports filed by Amgen, including our most recent annual report on Form 10-K and any subsequent periodic reports on Form 10-Q and current reports on Form 8-K. Unless otherwise noted, Amgen is providing this information as of the date of this news release and does not undertake any obligation to update any forward-looking statements contained in this document as a result of new information, future events or otherwise.
No forward-looking statement can be guaranteed and actual results may differ materially from those we project. Discovery or identification of new product candidates or development of new indications for existing products cannot be guaranteed and movement from concept to product is uncertain; consequently, there can be no guarantee that any particular product candidate or development of a new indication for an existing product will be successful and become a commercial product. Further, preclinical results do not guarantee safe and effective performance of product candidates in humans. The complexity of the human body cannot be perfectly, or sometimes, even adequately modeled by computer or cell culture systems or animal models. The length of time that it takes for us to complete clinical trials and obtain regulatory approval for product marketing has in the past varied and we expect similar variability in the future.
Even when clinical trials are successful, regulatory authorities may question the sufficiency for approval of the trial endpoints we have selected. We develop product candidates internally and through licensing collaborations, partnerships and joint ventures. Product candidates that are derived from relationships may be subject to disputes between the parties or may prove to be not as effective or as safe as we may have believed at the time of entering into such relationship. Also, we or others could identify safety, side effects or manufacturing problems with our products, including our devices, after they are on the market.
Our results may be affected by our ability to successfully market both new and existing products domestically and internationally, clinical and regulatory developments involving current and future products, sales growth of recently launched products, competition from other products including biosimilars, difficulties or delays in manufacturing our products and global economic conditions. In addition, sales of our products are affected by pricing pressure, political and public scrutiny and reimbursement policies imposed by third-party payers, including governments, private insurance plans and managed care providers and may be affected by regulatory, clinical and guideline developments and domestic and international trends toward managed care and healthcare cost containment. Furthermore, our research, testing, pricing, marketing and other operations are subject to extensive regulation by domestic and foreign government regulatory authorities. Our business may be impacted by government investigations, litigation and product liability claims. In addition, our business may be impacted by the adoption of new tax legislation or exposure to additional tax liabilities. If we fail to meet the compliance obligations in the corporate integrity agreement between us and the U.S. government, we could become subject to significant sanctions. Further, while we routinely obtain patents for our products and technology, the protection offered by our patents and patent applications may be challenged, invalidated or circumvented by our competitors, or we may fail to prevail in present and future intellectual property litigation. We perform a substantial amount of our commercial manufacturing activities at a few key facilities, including in Puerto Rico , and also depend on third parties for a portion of our manufacturing activities, and limits on supply may constrain sales of certain of our current products and product candidate development. An outbreak of disease or similar public health threat, such as COVID-19, and the public and governmental effort to mitigate against the spread of such disease, could have a significant adverse effect on the supply of materials for our manufacturing activities, the distribution of our products, the commercialization of our product candidates, and our clinical trial operations, and any such events may have a material adverse effect on our product development, product sales, business and results of operations. We rely on collaborations with third parties for the development of some of our product candidates and for the commercialization and sales of some of our commercial products. In addition, we compete with other companies with respect to many of our marketed products as well as for the discovery and development of new products. Further, some raw materials, medical devices and component parts for our products are supplied by sole third-party suppliers. Certain of our distributors, customers and payers have substantial purchasing leverage in their dealings with us. The discovery of significant problems with a product similar to one of our products that implicate an entire class of products could have a material adverse effect on sales of the affected products and on our business and results of operations. Our efforts to collaborate with or acquire other companies, products or technology, and to integrate the operations of companies or to support the products or technology we have acquired, may not be successful. There can be no guarantee that we will be able to realize any of the strategic benefits, synergies or opportunities arising from the Horizon acquisition, and such benefits, synergies or opportunities may take longer to realize than expected. We may not be able to successfully integrate Horizon, and such integration may take longer, be more difficult or cost more than expected. A breakdown, cyberattack or information security breach of our information technology systems could compromise the confidentiality, integrity and availability of our systems and our data. Our stock price is volatile and may be affected by a number of events. Our business and operations may be negatively affected by the failure, or perceived failure, of achieving our environmental, social and governance objectives. The effects of global climate change and related natural disasters could negatively affect our business and operations. Global economic conditions may magnify certain risks that affect our business. Our business performance could affect or limit the ability of our Board of Directors to declare a dividend or our ability to pay a dividend or repurchase our common stock. We may not be able to access the capital and credit markets on terms that are favorable to us, or at all.
The scientific information discussed in this news release related to our product candidates is preliminary and investigative. Further, any scientific information discussed in this news release relating to new indications for our products is preliminary and investigative and is not part of the labeling approved by the U.S. Food and Drug Administration for the products. The products are not approved for the investigational use(s) discussed in this news release, and no conclusions can or should be drawn regarding the safety or effectiveness of the products for these uses.
CONTACT: Amgen, Thousand Oaks
Madison Howard , 773-635-4910 (media)
Elissa Snook , 609-251-1407 (media)
Justin Claeys , 805-313-9775 (investors)
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SOURCE Amgen
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