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

The trial met its main endpoint, with a statistically important 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 therapy possibility that gives long-term symptom aid. As soon as permitted, UPLIZNA is predicted to supply a brand new possibility for sufferers earlier of their therapy plan,” stated Jay Bradner , M.D., govt vp, 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 important outcomes from the MINT trial additional strengthen the rising proof for UPLIZNA in extreme autoimmune illnesses and reinforce Amgen’s management in B-cell focusing on therapeutics.”

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

UPLIZNA demonstrated a statistically important 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 important (-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 point that impairs bodily functioning, actions of each 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 drugs 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 Section 3 biologic trial that included a protocol-specified steroid taper, an essential consideration for sufferers as results of extended high-dose steroid use contribute to the general burden of illness.”

The general security outcomes through the placebo-controlled interval of the trial had been in keeping with the identified security profile of UPLIZNA. The most typical treatment-emergent hostile occasions had been COVID-19, nasopharyngitis, urinary tract an infection, infusion associated response, headache and cough within the mixed inhabitants.

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

On the 2024 AANEM Annual Assembly, Amgen can 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 total well being care useful resource utilization and prices.

UPLIZNA is presently permitted for the therapy 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 Ailments by FDA following Section 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.

Concerning 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 better than 50% of this rating attributed to non-ocular objects, or an MG-ADL rating of not less than 11, QMG rating of not 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 right down to prednisone 5 mg a day, beginning at Week 4 to Week 24. The MINT trial additionally contains an elective three-year open-label therapy interval.

About Generalized Myasthenia Gravis (gMG)
Generalized myasthenia gravis (gMG) is a uncommon, power, B-cell-mediated autoimmune dysfunction that impairs neuromuscular communication and might trigger muscle weak point, hassle respiratory, problem 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 growing 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 towards AChR, and roughly 7% have detectable antibodies towards MuSK. ⁸ International prevalence is estimated at 2-36 circumstances per 100,000. ⁹ The illness is extra incessantly 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 focus on crucial proteins within the neuromuscular junction. ^1-3

About UPLIZNA® (inebilizumab-cdon) in NMOSD

INDICATION

UPLIZNA® (inebilizumab-cdon) is indicated for the therapy 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
Energetic hepatitis B an infection
Energetic or untreated latent tuberculosis

WARNINGS AND PRECAUTIONS

Infusion Reactions: UPLIZNA may cause infusion reactions, which may embrace headache, nausea, somnolence, dyspnea, fever, myalgia, rash, or different signs. Infusion reactions had been most typical 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 most typical 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 potential if combining UPLIZNA with one other immunosuppressive remedy.

The chance 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 therapy with UPLIZNA. Don’t administer to sufferers with lively hepatitis.

Though no confirmed circumstances 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 ought to be evaluated for tuberculosis threat components and examined for latent an infection previous to initiating UPLIZNA.

Vaccination with live-attenuated or stay vaccines shouldn’t be really helpful throughout therapy 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 equivalent to immunoglobulins G and M (IgG and IgM) with continued UPLIZNA therapy. Monitor the extent of immunoglobulins initially, throughout, and after discontinuation of therapy with UPLIZNA till B-cell repletion particularly in sufferers with opportunistic or recurrent infections.

Fetal Danger: Could trigger fetal hurt primarily based on animal knowledge. Advise females of reproductive potential of the potential threat to a fetus and to make use of an efficient methodology of contraception throughout therapy and for six months after stopping UPLIZNA.

Opposed Reactions: The most typical hostile reactions (not less than 10% of sufferers handled with UPLIZNA and better than placebo) had been urinary tract an infection and arthralgia.

For extra info on UPLIZNA, please see the Full Prescribing Data at www.UPLIZNA.com .

About Amgen
Amgen discovers, develops, manufactures, and delivers progressive medicines to assist tens of millions of sufferers of their struggle towards among the world’s hardest illnesses. Greater than 40 years in the past, Amgen helped to ascertain the biotechnology business and stays on the cutting-edge of innovation, utilizing know-how and human genetic knowledge to push past what’s identified at this time. Amgen is advancing a broad and deep pipeline that builds on its current portfolio of medicines to deal with most cancers, coronary heart illness, osteoporosis, inflammatory illnesses and uncommon illnesses.

In 2024, Amgen was named one of many “World’s Most Progressive Firms” by Quick Firm and certainly one of “America’s Finest Giant Employers” by Forbes, amongst different exterior recognitions . Amgen is among the 30 corporations that comprise the Dow Jones Industrial Common ^® , and additionally it is a part of the Nasdaq-100 Index ^® , which incorporates the most important 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 observe Amgen on X , LinkedIn , Instagram , TikTok , YouTube and Threads .

Ahead-Wanting Statements
This information launch incorporates forward-looking statements which might be primarily based on the present expectations and beliefs of Amgen. All statements, apart from statements of historic reality, are statements that may very well be deemed forward-looking statements, together with any statements on the end 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 progress 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 important dangers and uncertainties, together with these mentioned beneath and extra totally described within the Securities and Trade Fee stories filed by Amgen, together with our most up-to-date annual report on Type 10-Ok and any subsequent periodic stories on Type 10-Q and present stories on Type 8-Ok. Except 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 could be assured and precise outcomes could differ materially from these we undertaking. Discovery or identification of latest product candidates or improvement of latest indications for current merchandise can’t be assured and motion from idea to product is unsure; consequently, there could be no assure that any specific product candidate or improvement of a brand new indication for an current product will likely be profitable and turn into a industrial product. Additional, preclinical outcomes don’t assure secure and efficient efficiency of product candidates in people. The complexity of the human physique can’t be completely, or typically, even adequately modeled by pc or cell tradition programs or animal fashions. The size of time that it takes for us to finish medical trials and acquire regulatory approval for product advertising has up to now different and we anticipate comparable variability sooner or later. Even when medical trials are profitable, regulatory authorities could query the sufficiency for approval of the trial endpoints we’ve chosen. We develop product candidates internally and thru licensing collaborations, partnerships and joint ventures. Product candidates which might be derived from relationships could also be topic to disputes between the events or could show to be not as efficient or as secure as we could have believed on the time of coming into into such relationship. Additionally, we or others may establish security, uncomfortable side 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 capacity to efficiently market each new and current merchandise domestically and internationally, medical and regulatory developments involving present and future merchandise, gross sales progress 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 stress, political and public scrutiny and reimbursement insurance policies imposed by third-party payers, together with governments, personal insurance policy and managed care suppliers and could also be affected by regulatory, medical and guideline developments and home and worldwide traits towards managed care and healthcare value containment. Moreover, our analysis, testing, pricing, advertising and different operations are topic to intensive 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 latest 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 into topic to important sanctions. Additional, whereas we routinely receive patents for our merchandise and know-how, the safety provided by our patents and patent purposes could also be challenged, invalidated or circumvented by our opponents, or we could fail to prevail in current and future mental property litigation. We carry out a considerable quantity of our industrial manufacturing actions at a number of key services, together with in Puerto Rico , and in addition rely upon third events for a portion of our manufacturing actions, and limits on provide could constrain gross sales of sure of our present merchandise and product candidate improvement. An outbreak of illness or comparable public well being risk, equivalent to COVID-19, and the general public and governmental effort to mitigate towards the unfold of such illness, may have a big hostile 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 could have a fabric hostile 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 industrial 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 latest merchandise. Additional, some uncooked supplies, medical units and element elements for our merchandise are provided by sole third-party suppliers. Sure of our distributors, clients and payers have substantial buying leverage of their dealings with us. The invention of great issues with a product just like certainly one of our merchandise that implicate a complete class of merchandise may have a fabric hostile 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 we’ve acquired, is probably not profitable. There could be no assure that we can notice any of the strategic advantages, synergies or alternatives arising from the Horizon acquisition, and such advantages, synergies or alternatives could take longer to appreciate than anticipated. We could not be capable to efficiently combine Horizon, and such integration could take longer, be tougher or value greater than anticipated. A breakdown, cyberattack or info safety breach of our info know-how programs may compromise the confidentiality, integrity and availability of our programs and our knowledge. Our inventory worth is unstable and could also be affected by a lot of occasions. Our enterprise and operations could also be negatively affected by the failure, or perceived failure, of attaining our environmental, social and governance targets. The results of world local weather change and associated pure disasters may negatively have an effect on our enterprise and operations. International financial situations could amplify sure dangers that have an effect on our enterprise. Our enterprise efficiency may have an effect on or restrict the power of our Board of Administrators to declare a dividend or our capacity to pay a dividend or repurchase our frequent inventory. We could not be capable to entry the capital and credit score markets on phrases which might 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 are usually not permitted for the investigational use(s) mentioned on this information launch, and no conclusions can or ought to be drawn concerning the protection 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, Ok. C. (2017). Autoantibody-producing plasmablasts after B cell depletion recognized in muscle-specific kinase myasthenia gravis. JCI Perception, 2(17):e94263.
Lazaridis Ok., & 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, Ok., & Soliven, B. (2021). Myasthenia Gravis: Epidemiology, Pathophysiology and Medical Manifestations. J Clin Med, 10(11):2235.
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Rodrigues E., Umeh, E., Aishwarya, Navaratnarajah, N., Cole, A., & Moy, Ok. (2024). Incidence and prevalence of myasthenia gravis in america : A claims-based evaluation. Muscle Nerve, 69(2):166-171.
Hehir, M. Ok., & 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 Medication and Life, 14(1):7-16.