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Amgen (NASDAQ:AMGN) as we speak introduced the presentation of constructive top-line outcomes from the Part 3 MINT trial evaluating the efficacy and security of UPLIZNA ® (inebilizumab-cdon) for the remedy of adults with generalized myasthenia gravis (gMG), a uncommon autoimmune dysfunction. Outcomes from this randomized, double-blind, placebo-controlled, parallel-group trial can be offered on the Myasthenia Gravis Basis of America (MGFA) Scientific Session throughout the American Affiliation of Neuromuscular & Electrodiagnostic Drugs (AANEM) Annual Assembly, happening Oct. 15-18 in Savannah, Georgia .

The trial met its main endpoint, with a statistically vital change from baseline in Myasthenia Gravis Actions of Every day Dwelling (MG-ADL) rating for UPLIZNA (-4.2) in contrast with placebo (-2.2) (distinction: –1.9, p

“Sufferers dwelling with generalized myasthenia gravis deserve an efficient remedy choice that gives long-term symptom reduction. As soon as permitted, UPLIZNA is predicted to supply a brand new choice for sufferers earlier of their remedy plan,” stated Jay Bradner , M.D., govt vice chairman, Analysis and Improvement, and chief scientific officer at Amgen. “UPLIZNA targets CD19+ pre-B cells, mature B-cells and a few plasmablasts, that are drivers of the illness. The clinically vital outcomes from the MINT trial additional strengthen the rising proof for UPLIZNA in extreme autoimmune ailments and reinforce Amgen’s management in B-cell concentrating on therapeutics.”

Key secondary endpoints had been examined sequentially in a predefined order. UPLIZNA demonstrated a statistically vital and clinically significant change from baseline in comparison with placebo for the primary 4 key secondary endpoints.

UPLIZNA demonstrated a statistically vital change in Quantitative Myasthenia Gravis (QMG) rating for the mixed inhabitants (-4.8) in comparison with placebo (-2.3) at Week 26 (distinction: -2.5, p=0.0002).
Within the AChR+ inhabitants, UPLIZNA demonstrated imply change from baseline (-4.2) in MG-ADL rating at Week 26 in comparison with placebo (-2.4) (distinction: -1.8, p=0.0015).
Within the AChR+ inhabitants, UPLIZNA demonstrated imply change from baseline (-4.4) in QMG rating at Week 26 in comparison with placebo (-2.0) (distinction -2.5, p=0.0011).
Within the MuSK+ inhabitants, UPLIZNA demonstrated imply change from baseline (-3.9) in MG-ADL rating at Week 26 in comparison with placebo (-1.7) (distinction -2.2, p=0.0297).

Moreover, within the MuSK+ inhabitants, the imply change from baseline in QMG rating at Week 26 confirmed a development favoring UPLIZNA however was not statistically vital (-5.2 for UPLIZNA and -3.0 for placebo, distinction -2.3, p=0.1326).

“Myasthenia gravis can have a profound impression on sufferers by inflicting extreme fatigable muscle weak spot that impairs bodily functioning, actions of day by day dwelling and high quality of life,” stated Richard J. Nowak , M.D., M.S., international principal research investigator and director of the Myasthenia Gravis Clinic at Yale College . “This trial demonstrates clinically significant advantages of UPLIZNA, a twice-yearly infused medication with a novel mechanism of motion that selectively targets and depletes CD19+ B cells, that are key upstream drivers of myasthenia gravis. MINT can be the one Part 3 biologic trial that included a protocol-specified steroid taper, an necessary consideration for sufferers as results of extended high-dose steroid use contribute to the general burden of illness.”

The general security outcomes throughout the placebo-controlled interval of the trial had been in step with the recognized security profile of UPLIZNA. The commonest treatment-emergent adversarial occasions had been COVID-19, nasopharyngitis, urinary tract an infection, infusion associated response, headache and cough within the mixed inhabitants.

MINT is the biggest placebo-controlled gMG medical trial for a biologic remedy (238 adults) and enrolled the biggest variety of sufferers (48 adults) who’re MuSK+. The trial additionally included 190 adults who’re AChR+. Additional information will characterize placebo-controlled efficacy and security of UPLIZNA over 12 months in AChR+ sufferers with gMG.

On the 2024 AANEM Annual Assembly, Amgen may even be presenting a poster titled, “The Burden of Glucocorticoid Use Amongst Sufferers with Generalized Myasthenia Gravis in america ,” highlighting how increased use of glucocorticoids was related to extra toxicities and general well being care useful resource utilization and prices.

UPLIZNA is presently permitted for the remedy of neuromyelitis optica spectrum dysfunction (NMOSD) in grownup sufferers who’re anti-aquaporin-4 (AQP4) antibody constructive in america , European Union, Brazil and Canada , amongst different international locations. UPLIZNA was additionally not too long ago granted Breakthrough Remedy Designation for IgG4-Associated Illnesses by FDA following Part 3 outcomes introduced in June.

Primarily based on the MINT main outcomes, Amgen is planning to file for approval within the U.S., adopted by different key markets.

In regards to the MINT Trial
The MINT trial is a randomized, double-blind, placebo-controlled, parallel-group trial ( NCT04524273 ) evaluating the efficacy and security of UPLIZNA in adults with gMG. The trial enrolled 238 adults with gMG, together with 190 sufferers who’re acetylcholine receptor autoantibody-positive (AChR+) and 48 sufferers who’re muscle-specific kinase autoantibody-positive (MuSK+).

Eligibility standards at screening and randomization included a Myasthenia Gravis Basis of America (MGFA) classification of II, III, or IV illness, MG-ADL rating between 6 and 10 with higher than 50% of this rating attributed to non-ocular objects, or an MG-ADL rating of no less than 11, QMG rating of no less than 11, and use of a corticosteroid and/or non-steroidal immunosuppressant.

The first endpoint was change from baseline in MG-ADL rating at Week 26 within the mixed inhabitants. Key secondary endpoints included change from baseline in QMG scores within the mixed research inhabitants; change from baseline in MG-ADL rating at Week 26 for the AChR+ cohort and individually the MuSK+ cohort; and alter from baseline in QMG rating at Week 26 for the AChR+ cohort and individually the MuSK+ cohort. Sufferers who entered the research taking a corticosteroid had been tapered all the way down to prednisone 5 mg a day, beginning at Week 4 to Week 24. The MINT trial additionally consists of an elective three-year open-label remedy interval.

About Generalized Myasthenia Gravis (gMG)
Generalized myasthenia gravis (gMG) is a uncommon, continual, B-cell-mediated autoimmune dysfunction that impairs neuromuscular communication and might trigger muscle weak spot, hassle respiratory, issue swallowing and impaired speech and imaginative and prescient. ^1-4 Roughly 85% of sufferers with myasthenia gravis have the generalized type, or gMG. ^4,5

The prevalence and incidence of gMG are rising worldwide. ⁵ There are between 80,000 and 100,000 sufferers with myasthenia gravis within the U.S. ^6,7 Roughly 85% of sufferers with myasthenia gravis have detectable antibodies in opposition to AChR, and roughly 7% have detectable antibodies in opposition to MuSK. ⁸ World prevalence is estimated at 2-36 instances per 100,000. ⁹ The illness is extra regularly seen in younger ladies (age 20-30) and males aged 50 years and older. ^5,9

B cells are central to the pathogenesis of gMG. The illness is regarded as primarily pushed by pathogenic CD19+ plasmablasts and plasma cells that concentrate on vital proteins within the neuromuscular junction. ^1-3

About UPLIZNA® (inebilizumab-cdon) in NMOSD

INDICATION

UPLIZNA® (inebilizumab-cdon) is indicated for the remedy of neuromyelitis optica spectrum dysfunction (NMOSD) in grownup sufferers who’re anti-aquaporin-4 (AQP4) antibody constructive.

IMPORTANT SAFETY INFORMATION

UPLIZNA is contraindicated in sufferers with:

A historical past of life-threatening infusion response to UPLIZNA
Lively hepatitis B an infection
Lively or untreated latent tuberculosis

WARNINGS AND PRECAUTIONS

Infusion Reactions: UPLIZNA may cause infusion reactions, which might embrace headache, nausea, somnolence, dyspnea, fever, myalgia, rash, or different signs. Infusion reactions had been commonest with the primary infusion however had been additionally noticed throughout subsequent infusions. Administer pre-medication with a corticosteroid, an antihistamine, and an anti-pyretic.

Infections: The commonest infections reported by UPLIZNA-treated sufferers within the randomized and open-label durations included urinary tract an infection (20%), nasopharyngitis (13%), higher respiratory tract an infection (8%), and influenza (7%). Delay UPLIZNA administration in sufferers with an lively an infection till the an infection is resolved.

Elevated immunosuppressive results are doable if combining UPLIZNA with one other immunosuppressive remedy.

The danger of Hepatitis B Virus (HBV) reactivation has been noticed with different B-cell-depleting antibodies. Carry out HBV screening in all sufferers earlier than initiation of remedy with UPLIZNA. Don’t administer to sufferers with lively hepatitis.

Though no confirmed instances of Progressive Multifocal Leukoencephalopathy (PML) had been recognized in UPLIZNA medical trials, JC virus an infection leading to PML has been noticed in sufferers handled with different B-cell-depleting antibodies and different therapies that have an effect on immune competence. On the first signal or symptom suggestive of PML, withhold UPLIZNA and carry out an applicable diagnostic analysis.

Sufferers must be evaluated for tuberculosis danger components and examined for latent an infection previous to initiating UPLIZNA.

Vaccination with live-attenuated or reside vaccines will not be really helpful throughout remedy and after discontinuation, till B-cell repletion.

Discount in Immunoglobulins: There could also be a progressive and extended hypogammaglobulinemia or decline within the ranges of whole and particular person immunoglobulins akin to immunoglobulins G and M (IgG and IgM) with continued UPLIZNA remedy. Monitor the extent of immunoglobulins originally, throughout, and after discontinuation of remedy with UPLIZNA till B-cell repletion particularly in sufferers with opportunistic or recurrent infections.

Fetal Threat: Could trigger fetal hurt primarily based on animal information. Advise females of reproductive potential of the potential danger to a fetus and to make use of an efficient technique of contraception throughout remedy and for six months after stopping UPLIZNA.

Adversarial Reactions: The commonest adversarial reactions (no less than 10% of sufferers handled with UPLIZNA and higher than placebo) had been urinary tract an infection and arthralgia.

For added info on UPLIZNA, please see the Full Prescribing Info at www.UPLIZNA.com .

About Amgen
Amgen discovers, develops, manufactures, and delivers progressive medicines to assist tens of millions of sufferers of their battle in opposition to a number of the world’s hardest ailments. Greater than 40 years in the past, Amgen helped to determine the biotechnology trade and stays on the cutting-edge of innovation, utilizing know-how and human genetic information to push past what’s recognized as we speak. Amgen is advancing a broad and deep pipeline that builds on its present portfolio of medicines to deal with most cancers, coronary heart illness, osteoporosis, inflammatory ailments and uncommon ailments.

In 2024, Amgen was named one of many “World’s Most Progressive Corporations” by Quick Firm and one in every of “America’s Finest Giant Employers” by Forbes, amongst different exterior recognitions . Amgen is without doubt one of the 30 corporations that comprise the Dow Jones Industrial Common ^® , and it’s also a part of the Nasdaq-100 Index ^® , which incorporates the biggest and most progressive non-financial corporations listed on the Nasdaq Inventory Market primarily based on market capitalization.

For extra info, go to Amgen.com and comply with Amgen on X , LinkedIn , Instagram , TikTok , YouTube and Threads .

Ahead-Trying Statements
This information launch comprises forward-looking statements which can be primarily based on the present expectations and beliefs of Amgen. All statements, apart from statements of historic truth, are statements that may very well be deemed forward-looking statements, together with any statements on the result, advantages and synergies of collaborations, or potential collaborations, with every other firm (together with BeiGene, Ltd. or Kyowa Kirin Co., Ltd.), the efficiency of Otezla ^® (apremilast) (together with anticipated Otezla gross sales development and the timing of non-GAAP EPS accretion), our acquisitions of Teneobio, Inc., ChemoCentryx, Inc., or Horizon Therapeutics plc (together with the possible efficiency and outlook of Horizon’s enterprise, efficiency and alternatives, any potential strategic advantages, synergies or alternatives anticipated on account of such acquisition, and any projected impacts from the Horizon acquisition on our acquisition-related bills going ahead), in addition to estimates of revenues, working margins, capital expenditures, money, different monetary metrics, anticipated authorized, arbitration, political, regulatory or medical outcomes or practices, buyer and prescriber patterns or practices, reimbursement actions and outcomes, results of pandemics or different widespread well being issues on our enterprise, outcomes, progress, and different such estimates and outcomes. Ahead-looking statements contain vital dangers and uncertainties, together with these mentioned under and extra absolutely described within the Securities and Alternate Fee studies filed by Amgen, together with our most up-to-date annual report on Type 10-Okay and any subsequent periodic studies on Type 10-Q and present studies on Type 8-Okay. Until in any other case famous, Amgen is offering this info as of the date of this information launch and doesn’t undertake any obligation to replace any forward-looking statements contained on this doc on account of new info, future occasions or in any other case.

No forward-looking assertion will be assured and precise outcomes might differ materially from these we mission. Discovery or identification of recent product candidates or improvement of recent indications for present merchandise can’t be assured and motion from idea to product is unsure; consequently, there will be no assure that any specific product candidate or improvement of a brand new indication for an present product can be profitable and turn out to be a business product. Additional, preclinical outcomes don’t assure protected and efficient efficiency of product candidates in people. The complexity of the human physique can’t be completely, or generally, even adequately modeled by laptop or cell tradition techniques or animal fashions. The size of time that it takes for us to finish medical trials and procure regulatory approval for product advertising and marketing has previously assorted and we anticipate comparable variability sooner or later. Even when medical trials are profitable, regulatory authorities might query the sufficiency for approval of the trial endpoints now we have chosen. We develop product candidates internally and thru licensing collaborations, partnerships and joint ventures. Product candidates which can be derived from relationships could also be topic to disputes between the events or might show to be not as efficient or as protected as we might have believed on the time of coming into into such relationship. Additionally, we or others may determine security, negative effects or manufacturing issues with our merchandise, together with our units, after they’re available on the market.

Our outcomes could also be affected by our means to efficiently market each new and present merchandise domestically and internationally, medical and regulatory developments involving present and future merchandise, gross sales development of not too long ago launched merchandise, competitors from different merchandise together with biosimilars, difficulties or delays in manufacturing our merchandise and international financial situations. As well as, gross sales of our merchandise are affected by pricing strain, political and public scrutiny and reimbursement insurance policies imposed by third-party payers, together with governments, non-public insurance policy and managed care suppliers and could also be affected by regulatory, medical and guideline developments and home and worldwide tendencies towards managed care and healthcare value containment. Moreover, our analysis, testing, pricing, advertising and marketing and different operations are topic to in depth regulation by home and overseas authorities regulatory authorities. Our enterprise could also be impacted by authorities investigations, litigation and product legal responsibility claims. As well as, our enterprise could also be impacted by the adoption of recent tax laws or publicity to extra tax liabilities. If we fail to fulfill the compliance obligations within the company integrity settlement between us and the U.S. authorities, we may turn out to be topic to vital sanctions. Additional, whereas we routinely get hold of patents for our merchandise and know-how, the safety supplied by our patents and patent purposes could also be challenged, invalidated or circumvented by our rivals, or we might fail to prevail in current and future mental property litigation. We carry out a considerable quantity of our business manufacturing actions at just a few key services, together with in Puerto Rico , and likewise rely upon third events for a portion of our manufacturing actions, and limits on provide might constrain gross sales of sure of our present merchandise and product candidate improvement. An outbreak of illness or comparable public well being risk, akin to COVID-19, and the general public and governmental effort to mitigate in opposition to the unfold of such illness, may have a big adversarial impact on the availability of supplies for our manufacturing actions, the distribution of our merchandise, the commercialization of our product candidates, and our medical trial operations, and any such occasions might have a cloth adversarial impact on our product improvement, product gross sales, enterprise and outcomes of operations. We depend on collaborations with third events for the event of a few of our product candidates and for the commercialization and gross sales of a few of our business merchandise. As well as, we compete with different corporations with respect to lots of our marketed merchandise in addition to for the invention and improvement of recent merchandise. Additional, some uncooked supplies, medical units and element elements for our merchandise are equipped by sole third-party suppliers. Sure of our distributors, clients and payers have substantial buying leverage of their dealings with us. The invention of serious issues with a product just like one in every of our merchandise that implicate a complete class of merchandise may have a cloth adversarial impact on gross sales of the affected merchandise and on our enterprise and outcomes of operations. Our efforts to collaborate with or purchase different corporations, merchandise or know-how, and to combine the operations of corporations or to assist the merchandise or know-how now we have acquired, will not be profitable. There will be no assure that we will understand any of the strategic advantages, synergies or alternatives arising from the Horizon acquisition, and such advantages, synergies or alternatives might take longer to appreciate than anticipated. We might not be capable of efficiently combine Horizon, and such integration might take longer, be tougher or value greater than anticipated. A breakdown, cyberattack or info safety breach of our info know-how techniques may compromise the confidentiality, integrity and availability of our techniques and our information. Our inventory value is unstable and could also be affected by a variety of occasions. Our enterprise and operations could also be negatively affected by the failure, or perceived failure, of attaining our environmental, social and governance aims. The consequences of world local weather change and associated pure disasters may negatively have an effect on our enterprise and operations. World financial situations might amplify sure dangers that have an effect on our enterprise. Our enterprise efficiency may have an effect on or restrict the flexibility of our Board of Administrators to declare a dividend or our means to pay a dividend or repurchase our frequent inventory. We might not be capable of entry the capital and credit score markets on phrases which can be favorable to us, or in any respect.

Any scientific info mentioned on this information launch regarding new indications for our merchandise is preliminary and investigative and isn’t a part of the labeling permitted by the U.S. Meals and Drug Administration for the merchandise. The merchandise should not permitted for the investigational use(s) mentioned on this information launch, and no conclusions can or must be drawn concerning the security or effectiveness of the merchandise for these makes use of.

CONTACT: Amgen, Thousand Oaks
Madison Howard , 773-636-4910 (media)
Elissa Snook , 609-251-1407 (media)
Justin Claeys , 805-313-9775 (traders)

References

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Stathopoulos P., Kumar, A., Nowak, R. J., & O’Connor, Okay. C. (2017). Autoantibody-producing plasmablasts after B cell depletion recognized in muscle-specific kinase myasthenia gravis. JCI Perception, 2(17):e94263.
Lazaridis Okay., & 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, Okay., & Soliven, B. (2021). Myasthenia Gravis: Epidemiology, Pathophysiology and Scientific Manifestations. J Clin Med, 10(11):2235.
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Rodrigues E., Umeh, E., Aishwarya, Navaratnarajah, N., Cole, A., & Moy, Okay. (2024). Incidence and prevalence of myasthenia gravis in america : A claims-based evaluation. Muscle Nerve, 69(2):166-171.
Hehir, M. Okay., & Silvestri, N. J. (2018). Generalized myasthenia gravis: classification, medical presentation, pure historical past, 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 Drugs and Life, 14(1):7-16.