Apremilast

Patients Reported Improvement in Ability to Conduct Daily Activities with Twice-Yearly Dosing * Late-Breaking Data to be Presented at AAN 2025 THOUSAND OAKS, Calif., March 13, 2025 /PRNewswire/ -- Amgen (NASDAQ:AMGN) today announced new data from the Phase 3, registrational MINT trial evaluating the efficacy and safety of UPLIZNA® (inebilizumab-cdon) in adults living with generalized myasthenia gravis (gMG). The results demonstrated durable and sustained efficacy of UPLIZNA in patients with acetylcholine receptor autoantibody-positive (AChR+) gMG with two doses a year, following an initial loading dose. Findings will be presented as a late-breaking oral presentation during the American Academy of Neurology (AAN) Annual Meeting on April 8, 2025, in San Diego. The Phase 3 MINT trial, which was a randomized-control trial, evaluated UPLIZNA in muscle-specific kinase autoantibody-positive (MuSK+) and AChR+ gMG patients, with the MuSK+ group followed for 26 weeks and the AChR+ group followed for 52 weeks. The trial demonstrated continued improvement in efficacy of UPLIZNA compared to placebo (adjusted difference, −2.8, 95% CI, −3.9 to −1.7) as measured by the change in baseline of Myasthenia Gravis Activities of Daily Living (MG-ADL) score in the AChR+ subpopulation through week 52. Among the AChR+ patients in the UPLIZNA group, 72.3% had a ≥3 point improvement in the MG-ADL score, compared to 45.2% in placebo.1 As previously disclosed at the 2024 American Association of Neuromuscular & Electrodiagnostic Medicine Annual Meeting, the trial met its primary endpoint, with a statistically significant change from baseline in MG-ADL score for UPLIZNA (-4.2) compared with placebo (-2.2) (difference: –1.9, p<0.0001) at Week 26 for the combined study population. "The 52-week MINT trial results highlight the potential for a new standard of care in gMG, offering durable symptom relief with a simplified treatment regimen," said Jay Bradner, M.D., executive vice president of Research and Development at Amgen. "These findings reinforce UPLIZNA's ability to provide sustained symptom relief with just two doses per year—an important advancement for patients living with generalized myasthenia gravis—while underscoring our commitment to developing transformative therapies for people facing complex autoimmune diseases." Change from baseline in the Quantitative Myasthenia Gravis (QMG) score was also greater for patients in the UPLIZNA group as compared to placebo at Week 52 (adjusted difference, −4.3, 95% CI, −5.9 to −2.8). Among the AChR+ patients in the UPLIZNA group, 69.2% improved by ≥3 points in the QMG score, compared to 41.8% in the placebo group.1 MINT was the first and only Phase 3 trial for a biologic to incorporate a corticosteroid taper into its protocol. Patients who entered the study taking corticosteroids were tapered down starting at Week 4 to prednisone 5 mg per day by Week 24. "I'm looking forward to further examining the 52-week MINT data with my colleagues in the neurology community at AAN," said Richard J. Nowak, M.D., M.S., global principal study investigator and director of the Myasthenia Gravis Clinic at Yale University. "These results showed that UPLIZNA consistently relieved burdensome symptoms and improved activities of daily living for gMG patients." No new safety signals were identified. The overall TEAE profile during the study period is consistent with the known safety profile for the approved indication (NMOSD). The most common adverse events included infusion-related reactions, nasopharyngitis and urinary tract infections. UPLIZNA is currently approved for the treatment of adult patients with anti-aquaporin-4 (AQP4) antibody positive neuromyelitis optica spectrum disorder (NMOSD) and is under priority FDA review for the treatment of Immunoglobulin G4-related disease (IgG4-RD) with a PDUFA date of April 3, 2025. The FDA has granted UPLIZNA Orphan Drug Designation for the treatment of gMG. Regulatory filing activities are underway with submission anticipated to be complete in H1 2025. *After an initial loading dose. About the MINT Trial The MINT trial is a randomized, double-blind, placebo-controlled, parallel-group trial (NCT04524273) evaluating the efficacy and safety of UPLIZNA in adults with gMG. The trial enrolled 238 adults with gMG, including 190 patients who are acetylcholine receptor autoantibody-positive (AChR+) and 48 patients who are muscle-specific kinase autoantibody-positive (MuSK+). Eligibility criteria at screening and randomization included a Myasthenia Gravis Foundation of America (MGFA) classification of II, III, or IV disease, MG-ADL score between 6 and 10 with greater than 50% of this score attributed to non-ocular items, or an MG-ADL score of at least 11, QMG score of at least 11, and use of a corticosteroid and/or non-steroidal immunosuppressant. The primary endpoint was change from baseline in MG-ADL score at Week 26 in the combined population. Key secondary endpoints included change from baseline in QMG scores in the combined study population; change from baseline in MG-ADL score at Week 26 for the AChR+ cohort and separately the MuSK+ cohort; and change from baseline in QMG score at Week 26 for the AChR+ cohort and separately the MuSK+ cohort. Patients who entered the study taking a corticosteroid were tapered down to prednisone 5 mg a day, starting at Week 4 to Week 24. The MINT trial also includes an optional three-year open-label treatment period. About Generalized Myasthenia Gravis (gMG) Generalized myasthenia gravis (gMG) is a rare, chronic, B-cell-mediated autoimmune disorder that impairs neuromuscular communication and can cause muscle weakness, trouble breathing, difficulty swallowing and impaired speech and vision.2-4 Approximately 85% of patients with myasthenia gravis have the generalized form, or gMG.5,6 The prevalence and incidence of gMG are increasing worldwide.6 There are between 80,000 and 100,000 patients with myasthenia gravis in the U.S.7,8 Approximately 85% of patients with myasthenia gravis have detectable antibodies against AChR, and approximately 7% have detectable antibodies against MuSK.9 Global prevalence is estimated at 2-36 cases per 100,000.10 The disease is more frequently seen in young women (age 20-30) and men aged 50 years and older.6,10 B cells are central to the pathogenesis of gMG. The disease is thought to be primarily driven by pathogenic CD19+ plasmablasts and plasma cells that target critical proteins in the neuromuscular junction.2-4 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). The precise mechanism by which UPLIZNA exerts its therapeutic effects is unknown. After two initial infusions, patients need one dose of UPLIZNA every six months. About UPLIZNA in NMOSD INDICATION AND IMPORTANT SAFETY INFORMATION INDICATION UPLIZNA (inebilizumab-cdon) 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. 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The scientific information discussed in this news release relating to new indications for our product is preliminary and investigative and is not part of the labeling approved by the U.S. Food and Drug Administration for the product. The product is 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 product for these uses. CONTACT: Amgen, Thousand Oaks Madison Howard, 773-636-4910 (media) Elissa Snook, 609-251-1407 (media) Justin Claeys, 805-313-9775 (investors) References Nowak R, Phase 3 Myasthenia Gravis Inebilizumab Trial (MINT): Efficacy Data in AChR+ Generalized MG Subpopulation Through Week-52 [Late-breaking oral presentation]. To be presented at American Academy of Neurology Annual Meeting (5-9 April 2025). Available at: Yi, J. S., Guptill, J. T., Stathopoulos, P., Nowak, R. J., & O'Connor, K. C. (2018). B cells in the pathophysiology of myasthenia gravis. Muscle Nerve, 57(2):172-184. Willcox H. N., Newsom-Davis, J., & Calder, L. R. (1984). Cell types required for anti-acetylcholine receptor antibody synthesis by cultured thymocytes and blood lymphocytes in myasthenia gravis. Clinical and Experimental Immunology., 58:97-106. Stathopoulos P., Kumar, A., Nowak, R. J., & O'Connor, K. C. (2017). Autoantibody-producing plasmablasts after B cell depletion identified in muscle-specific kinase myasthenia gravis. JCI Insight, 2(17):e94263. Lazaridis K., & Tzartos, S. J. (2020). Autoantibody Specificities in Myasthenia Gravis; Implications for Improved Diagnostics and Therapeutics. Frontiers in Immunology, 11:212. Dresser L., Wlodarski, R., Rezania, K., & Soliven, B. (2021). Myasthenia Gravis: Epidemiology, Pathophysiology and Clinical Manifestations. J Clin Med, 10(11):2235. Ye et al. Frontiers in Neurology. (2024);15:1339167. Rodrigues E., Umeh, E., Aishwarya, Navaratnarajah, N., Cole, A., & Moy, K. (2024). Incidence and prevalence of myasthenia gravis in the United States: A claims-based analysis. Muscle Nerve, 69(2):166-171. Hehir, M. K., & Silvestri, N. J. (2018). Generalized myasthenia gravis: classification, clinical presentation, natural history, and epidemiology. Neurologic Clinics, 36:253-60. Bubuioc, A. M., Kudebayeva, A., Turuspekova, S., Lisnic, V., & Leone, M. A. (2021). The epidemiology of myasthenia gravis. Journal of Medicine and Life, 14(1):7-16. SOURCE Amgen WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED