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** UCB Presents Latest Data from Generalized Myasthenia Gravis Portfolio at=
9th Congress of the European Academy of Neurology (EAN) Meeting
------------------------------------------------------------
=C2=B7 Results presented across UCB=E2=80=99s generalized myasthenia gravis=
(gMG) portfolio
=C2=B7 Expanded data from the MycarinG and RAISE studies showcase further i=
nsights on potential of rozanolixizumab and zilucoplan in generalized myast=
henia gravis (gMG)
=C2=B7 Presentations follow recent U.S. FDA approval of rozanolixizumab for=
treatment of generalized myasthenia gravis (gMG) in adult patients who are=
anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase=
(MuSK) antibody positive and expand evidence base in gMG
Brussels, Belgium, 30th June 2023 =E2=80=93 18:30 PM CEST=E2=80=93 UCB, a g=
lobal biopharmaceutical company, today announced it will present results fr=
om across its portfolio in generalized myasthenia gravis (gMG) at the Europ=
ean Academy of Neurology (EAN) Meeting July 1-4, 2023.=C2=A0
Additional results from the MycarinG and RAISE studies^1,2, and their open-=
label extension studies will be presented, investigating UCB=E2=80=99s roza=
nolixizumab, an SC-infused monoclonal antibody targeting the neonatal Fc re=
ceptor (FcRn)^3,4 and zilucoplan, a self-administered, subcutaneous (SC) pe=
ptide inhibitor of complement component 5 (C5 inhibitor) in adults with gMG=
.^5 These pivotal Phase 3 trials supported U.S., European, and Japanese reg=
ulatory filings of both rozanolixizumab and zilucoplan. Presentations will =
also focus on the importance of real-world data and its use in better under=
standing current patient experiences. In total, nine abstracts will be pres=
ented, including three oral presentations.
In June, rozanolixizumab was approved by the U.S. Food and Drug Administrat=
ion (FDA), for the treatment of generalized myasthenia gravis (gMG) in adul=
t patients who are anti-acetylcholine receptor (AchR) or anti-muscle-specif=
ic tyrosine kinase (MuSK) antibody positive,^6 having been granted Priority=
Review for its Biologic License Application (BLA). In the U.S., under the =
FDAs non-proprietary naming of biological products guidance, the nonproprie=
tary name for this medicine is rozanolixizumab-noli. It is an FDA-approved =
treatment for both anti-AChR and anti-MuSK antibody-positive gMG, the two m=
ost common subtypes of gMG. Rozanolixizumab is currently only approved in t=
he U.S., and is under review by the European Medicines Agency (EMA) and the=
Japanese Pharmaceuticals and Medical Devices Agency (PMDA) for the treatme=
nt of adults with gMG. Responses from regulatory agencies to these submissi=
ons are expected by H1 2024.=C2=A0
Zilucoplan is currently under review by the U.S. FDA, EMA and the Japanese =
PMDA for the treatment of adult gMG patients who are anti-acetylcholine rec=
eptor (AChR) antibody positive. Responses from these regulatory agencies ar=
e expected from H2 2023 onwards. The safety and efficacy of zilucoplan have=
not been established and it is not currently approved for use in any indic=
ation by any regulatory authority worldwide.
These data further inform UCB=E2=80=99s innovative approach to evolving sci=
ence into meaningful solutions that help improve outcomes and address unmet=
needs of people living with gMG.
Donatello Crocetta, Head of Global Rare Disease & Rare Medical, UCB, explai=
ned: =E2=80=9CThe additional results from the Phase 3 MycarinG and RAISE st=
udies demonstrate UCB=E2=80=99s dedication to finding treatment options for=
patients with gMG and reinforce the depth and strength of our expanding ra=
re disease pipeline and portfolio. Following on from our recent approval fo=
r rozanolixizumab in the U.S. for the treatment of adult patients who are a=
nti-acetylcholine receptor or anti-muscle-specific tyrosine kinase antibody=
positive gMG, we=E2=80=99re very excited to be collaborating with the gMG =
community to extend knowledge about new medicines which could, in the futur=
e, play an important role in the treatment of this rare neuromuscular disea=
se.=E2=80=9D
Data from UCB's extensive work to better understand the burden of gMG in di=
fferent European countries will also be presented in 4 posters. Additionall=
y, through an immersive VR experience co-created with the members of the gM=
G community, UCB will enable HCPs to gain a deeper understanding of the dai=
ly challenges experienced by people living with gMG.
=E2=80=9CBecause of the high disease and treatment burden that gMG elicits =
along with the impact on a person=E2=80=99s daily life, it is important for=
us to contribute and improve our wealth of knowledge of this debilitating =
rare disease - especially relevant following Myasthenia Gravis Awareness Mo=
nth by focusing - on patients and digital innovations, with the aim to addr=
ess the needs of people living with gMG.=E2=80=9D said Manuela Maronati, He=
ad of Europe, Rare Disease, UCB. =E2=80=9CAs well as increasing our underst=
anding of gMG, we hope to continue to work towards bringing new treatment o=
ptions to patients. We are looking forward to hearing from the European Med=
icines Agency on their review of the Marketing Authorization Application fo=
r both rozanolixizumab and zilucoplan.=E2=80=9D
UCB presentations during EAN 2023
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About Generalized Myasthenia Gravis (gMG)
gMG is a rare disease with a global prevalence of 100=E2=80=93350 cases per=
every 1 million people.^7 People living with gMG can experience a variety =
of symptoms, including severe muscular weakness that can result in life-thr=
eatening weakness of the muscles of respiration, double vision, drooping ey=
elids, and difficulty swallowing, chewing and talking.^8,9=C2=A0
In gMG, pathogenic autoantibodies can impair synaptic transmission at the n=
euromuscular junction (NMJ) by targeting specific proteins on the post-syna=
ptic membrane.^3,10 This disrupts the ability of the nerves to stimulate th=
e muscle and results in a weaker contraction. gMG can occur in any race, ge=
nder or age.^11=C2=A0
About rozanolixizumab
Rozanolixizumab is a subcutaneous administered, humanized monoclonal antibo=
dy that specifically binds, with high affinity, to human neonatal Fc recept=
or (FcRn). It has been designed to block the interaction of FcRn and Immuno=
globulin G (IgG), accelerating the catabolism of antibodies and reducing th=
e concentration of pathogenic IgG autoantibodies.^12=C2=A0
Outside of the U.S. rozanolixizumab is not approved for use in any indicati=
on by any other regulatory authority worldwide.
Important U.S. Safety Information for RYSTIGGO^=C2=AE=C2=A0
WARNINGS AND PRECAUTIONS=C2=A0
Infections: RYSTIGGO may increase the risk of infection. Delay RYSTIGGO adm=
inistration in patients with an active infection until the infection is res=
olved. During treatment with RYSTIGGO, monitor for clinical signs and sympt=
oms of infection. If serious infection occurs, administer appropriate treat=
ment and consider withholding RYSTIGGO until the infection has resolved.
Immunization
Immunization with vaccines during RYSTIGGO treatment has not been studied. =
The safety of immunization with live or live-attenuated vaccines and the re=
sponse to immunization with any vaccine are unknown. Because RYSTIGGO cause=
s a reduction in IgG levels, vaccination with live-attenuated or live vacci=
nes is not recommended during treatment with RYSTIGGO. Evaluate the need to=
administer age-appropriate vaccines according to immunization guidelines b=
efore initiation of a new treatment cycle with RYSTIGGO.
Aseptic Meningitis: Serious adverse reactions of aseptic meningitis (also c=
alled drug-induced aseptic meningitis) have been reported in patients treat=
ed with RYSTIGGO. If symptoms consistent with aseptic meningitis develop, d=
iagnostic workup and treatment should be initiated according to the standar=
d of care.
Hypersensitivity Reactions: Hypersensitivity reactions, including angioedem=
a and rash, were observed in patients treated with RYSTIGGO. Management of =
hypersensitivity reactions depends on the type and severity of the reaction=
. Monitor patients during treatment with RYSTIGGO and for 15 minutes after =
for clinical signs and symptoms of hypersensitivity reactions. If a reactio=
n occurs, institute appropriate measures if needed.
ADVERSE REACTIONS
In a placebo-controlled study, the most common adverse reactions (reported =
in at least 10% of RYSTIGGO-treated patients) were headache, infections, di=
arrhea, pyrexia, hypersensitivity reactions, and nausea. Serious infections=
were reported in 4% of patients treated with RYSTIGGO. Three fatal cases o=
f pneumonia were identified, caused by COVID-19 infection in two patients a=
nd an unknown pathogen in one patient. Six cases of infections led to disco=
ntinuation of RYSTIGGO.
Full U.S. Prescribing Information is available at https://www.ucb-usa.com/R=
YSTIGGO-prescribing-information.pdf (https://eur02.safelinks.protection.out=
look.com/?url=3Dhttps%3A%2F%2Fwww.ucb-usa.com%2FRYSTIGGO-prescribing-inform=
ation.pdf&data=3D05%7C01%7CJim.Baxter%40ucb.com%7C12f054d6d36246b0dcf708db7=
69a8ff7%7C237582ad3eab4d44868806ca9f2e613b%7C0%7C0%7C638234178432556862%7CU=
nknown%7CTWFpbGZsb3d8eyJWIjoiMC4wLjAwMDAiLCJQIjoiV2luMzIiLCJBTiI6Ik1haWwiLC=
JXVCI6Mn0%3D%7C3000%7C%7C%7C&sdata=3DE3TjXnLiS9quwi9gMvqi%2Ffn0y5PMh77eaJzZ=
oH%2FRaos%3D&reserved=3D0)
About zilucoplan
Zilucoplan is a once-daily subcutaneous (SC), self-administered peptide inh=
ibitor of complement component 5 (C5 inhibitor) under clinical development =
by UCB in gMG. As a C5 inhibitor, zilucoplan inhibits complement-mediated d=
amage to the neuromuscular junction through its targeted dual mechanism of =
action.^13 In 2019, the U.S. FDA granted orphan drug designation to zilucop=
lan for the treatment of myasthenia gravis.^14 Orphan designation was grant=
ed in July 2022 by the European Commission to zilucoplan for the treatment =
of myasthenia gravis.^15 The safety and efficacy of zilucoplan have not bee=
n established and it is not currently approved for use in any indication by=
any regulatory authority worldwide.
For further information, contact UCB:=C2=A0
Global Communications
Jim Baxter
T +44 7900 605 652
email jim.baxter@ucb.com =C2=A0
Corporate Communications
Laurent Schots=C2=A0
T +32.2.559.92.64=C2=A0
email laurent.schots@ucb.com
Investor Relations
Antje Witte
T +32.2.559.94.14=C2=A0
email antje.witte@ucb.com=C2=A0
About UCB
UCB, Brussels, Belgium (www.ucb.com) is a global biopharmaceutical company =
focused on the discovery and development of innovative medicines and soluti=
ons to transform the lives of people living with severe diseases of the imm=
une system or of the central nervous system. With approximately 8 600 peopl=
e in approximately 40 countries, the company generated revenue of =E2=82=AC=
5.8 billion in 2021. UCB is listed on Euronext Brussels (symbol: UCB). Fol=
low us on Twitter: @UCB_news
Forward-looking statements=C2=A0
This press release may contain forward-looking statements including, withou=
t limitation, statements containing the words =E2=80=9Cbelieves=E2=80=9D, =
=E2=80=9Canticipates=E2=80=9D, =E2=80=9Cexpects=E2=80=9D, =E2=80=9Cintends=
=E2=80=9D, =E2=80=9Cplans=E2=80=9D, =E2=80=9Cseeks=E2=80=9D, =E2=80=9Cestim=
ates=E2=80=9D, =E2=80=9Cmay=E2=80=9D, =E2=80=9Cwill=E2=80=9D, =E2=80=9Ccont=
inue=E2=80=9D and similar expressions. These forward-looking statements are=
based on current plans, estimates and beliefs of management. All statement=
s, other than statements of historical facts, are statements that could be =
deemed forward-looking statements, including estimates of revenues, operati=
ng margins, capital expenditures, cash, other financial information, expect=
ed legal, arbitration, political, regulatory or clinical results or practic=
es and other such estimates and results. By their nature, such forward-look=
ing statements are not guarantees of future performance and are subject to =
known and unknown risks, uncertainties and assumptions which might cause th=
e actual results, financial condition, performance or achievements of UCB, =
or industry results, to differ materially from those that may be expressed =
or implied by such forward-looking statements contained in this press relea=
se. Important factors that could result in such differences include: the gl=
obal spread and impact of COVID-19, changes in general economic, business a=
nd competitive conditions, the inability to obtain necessary regulatory app=
rovals or to obtain them on acceptable terms or within expected timing, cos=
ts associated with research and development, changes in the prospects for p=
roducts in the pipeline or under development by UCB, effects of future judi=
cial decisions or governmental investigations, safety, quality, data integr=
ity or manufacturing issues; potential or actual data security and data pri=
vacy breaches, or disruptions of our information technology systems, produc=
t liability claims, challenges to patent protection for products or product=
candidates, competition from other products including biosimilars, changes=
in laws or regulations, exchange rate fluctuations, changes or uncertainti=
es in tax laws or the administration of such laws, and hiring and retention=
of its employees. There is no guarantee that new product candidates will b=
e discovered or identified in the pipeline, will progress to product approv=
al or that new indications for existing products will be developed and appr=
oved. Movement from concept to commercial product is uncertain; preclinical=
results do not guarantee safety and efficacy of product candidates in huma=
ns. So far, the complexity of the human body cannot be reproduced in comput=
er models, cell culture systems or animal models. The length of the timing =
to complete clinical trials and to get regulatory approval for product mark=
eting has varied in the past and UCB expects similar unpredictability going=
forward. Products or potential products, which are the subject of partners=
hips, joint ventures or licensing collaborations may be subject to differen=
ces disputes between the partners or may prove to be not as safe, effective=
or commercially successful as UCB may have believed at the start of such p=
artnership. UCB=E2=80=99s efforts to acquire other products or companies an=
d to integrate the operations of such acquired companies may not be as succ=
essful as UCB may have believed at the moment of acquisition. Also, UCB or =
others could discover safety, side effects or manufacturing problems with i=
ts products and/or devices after they are marketed. The discovery of signif=
icant problems with a product similar to one of UCB=E2=80=99s products that=
implicate an entire class of products may have a material adverse effect o=
n sales of the entire class of affected products. Moreover, sales may be im=
pacted by international and domestic trends toward managed care and health =
care cost containment, including pricing pressure, political and public scr=
utiny, customer and prescriber patterns or practices, and the reimbursement=
policies imposed by third-party payers as well as legislation affecting bi=
opharmaceutical pricing and reimbursement activities and outcomes. Finally,=
a breakdown, cyberattack or information security breach could compromise t=
he confidentiality, integrity and availability of UCB=E2=80=99s data and sy=
stems. Given these uncertainties, you should not place undue reliance on an=
y of such forward-looking statements. There can be no guarantee that the in=
vestigational or approved products described in this press release will be =
submitted or approved for sale or for any additional indications or labelli=
ng in any market, or at any particular time, nor can there be any guarantee=
that such products will be or will continue to be commercially successful =
in the future. UCB is providing this information, including forward-looking=
statements, only as of the date of this press release and it does not refl=
ect any potential impact from the evolving COVID-19 pandemic, unless indica=
ted otherwise. UCB is following the worldwide developments diligently to as=
sess the financial significance of this pandemic to UCB. UCB expressly disc=
laims any duty to update any information contained in this press release, e=
ither to confirm the actual results or to report or reflect any change in i=
ts forward-looking statements with regard thereto or any change in events, =
conditions or circumstances on which any such statement is based, unless su=
ch statement is required pursuant to applicable laws and regulations. Addit=
ionally, information contained in this document shall not constitute an off=
er to sell or the solicitation of an offer to buy any securities, nor shall=
there be any offer, solicitation or sale of securities in any jurisdiction=
in which such offer, solicitation or sale would be unlawful prior to the r=
egistration or qualification under the securities laws of such jurisdiction=
.
___________________=C2=A0
1. Clinical Trials.gov. 'A Study to Test Efficacy and Safety of Rozanolixiz=
umab in Adult Patients With Generalized Myasthenia Gravis'. =C2=A0 https://=
clinicaltrials.gov/ct2/show/NCT03971422 (https://clinicaltrials.gov/ct2/sho=
w/NCT03971422. Accessed June 2023) . Accessed June 2023.
2. Clinical Trials.gov. 'Safety, Tolerability, and Efficacy of Zilucoplan i=
n Subjects With Generalized Myasthenia Gravis (RAISE)' https://clinicaltria=
ls.gov/ct2/show/NCT04115293. Accessed June 2023.
3. Smith B, et al. Generation and characterization of a high affinity anti-=
human FcRn antibody, rozanolixizumab, and the effects of different molecula=
r formats on the reduction of plasma IgG concentration. MAbs. 2018;10:1111-=
30.
4. Kiessling P, et al. The FcRn inhibitor rozanolixizumab reduces human ser=
um IgG concentration: A randomized phase 1 study. Sci Transl Med. 2017;9(41=
4:eaan1208).
5. Howard J, et al. Clinical Effects of the Self-administered Subcutaneous =
Complement Inhibitor Zilucoplan in Patients With Moderate to Severe General=
ized Myasthenia Gravis: Results of a Phase 2 Randomized, Double-Blind, Plac=
ebo-Controlled, Multicenter Clinical Trial. JAMA Neurol. 2020 May 1;77(5):5=
82-92.
6. RYSTIGGO=C2=AE U.S. Prescribing Information
7. Punga AR, et al. Epidemiology, diagnostics, and biomarkers of autoimmune=
neuromuscular junction disorders. Lancet Neurol. 2022;21(2):176-88.
8. Myasthenia Gravis Foundation of America. Clinical Overview of MG. https:=
//myasthenia.org/Professionals/Clinical-Overview-of-MG. Accessed June 2023.
9. Hansen JS, et al. Mortality in myasthenia gravis: A nationwide populatio=
n-based follow-up study in Denmark. Muscle Nerve. 2016;53: 73-7.
10. Juel VC, Massey JM. Myasthenia gravis. Orphanet J Rare Dis. 2007;2:44.
11. Myasthenia Gravis Foundation of America. MG Quick Facts. https://myasth=
enia.org/MG-Education/MG-Quick-Facts. Accessed June 2023.
12. Wolfe G, et al. Myasthenia gravis activities of daily living profile. N=
eurology. 1992;52(7):1487-9.
13. Howard J, et al. Efficacy and safety of zilucoplan in patients with gen=
eralised myasthenia gravis: A randomised, double-blind, placebo-controlled,=
Phase 3 study (RAISE). Lancet Neurol. 2023 May;22(5):395-406.
14. US Food and Drug Administration. https://www.accessdata.fda.gov/scripts=
/opdlisting/oopd/detailedIndex.cfm?cfgridkey=3D699319. Accessed June 2023.
15. European Medicines Agency, EU/3/22/2650: Orphan designation for the tre=
atment of myasthenia gravis https://www.ema.europa.eu/en/medicines/human/or=
phan-designations/eu-3-22-2650. Accessed June 2023=C2=A0
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