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** UCB announces positive data in myasthenia gravis with zilucoplan phase 3=
study results
------------------------------------------------------------
=C2=B7 Positive topline results show the Phase 3 RAISE (NCT04115293) ziluco=
plan trial met primary and all key secondary endpoints in adults with gener=
alized myasthenia gravis
=C2=B7 The results show a favorable safety profile and good tolerability =
=C2=A0
=C2=B7 UCB plans to proceed with zilucoplan regulatory submissions later th=
is year=C2=A0
=C2=B7 Results follow recent positive topline data from the Phase 3 Mycarin=
G study investigating rozanolixizumab, a monoclonal antibody also being dev=
eloped by UCB in the same indication
=C2=B7 These results are the latest in a series of positive phase 3 data an=
nouncements by the company across its product pipeline
Brussels, Belgium and Atlanta, Ga. =E2=80=93 February 04, 2022 =E2=80=93 07=
:00 CET: Regulated Information =E2=80=93 Inside Information =E2=80=93 UCB, =
a global biopharmaceutical company, today announced positive topline result=
s from the RAISE (NCT04115293) trial^1=C2=A0evaluating its investigational =
treatment zilucoplan, a self-administered, subcutaneous (SC) peptide inhibi=
tor of complement component 5 (C5 inhibitor), versus placebo in adults with=
generalized myasthenia gravis (gMG).
The primary endpoint of the trial was met; a clinically meaningful and stat=
istically significant improvement from baseline in Myasthenia Gravis-Activi=
ties of Daily Living Profile (MG-ADL) total score at Week 12 was observed f=
or the zilucoplan treatment group vs placebo.
All key secondary endpoints were also met, including statistically signific=
ant improvements from baseline in Quantitative Myasthenia Gravis (QMG) scor=
e, Myasthenia Gravis Composite (MGC) score and MG-QoL15r score at Week 12 f=
or the zilucoplan treatment group vs placebo.=C2=A0
The results show zilucoplan was well-tolerated and no major unexpected safe=
ty findings were identified compared to earlier zilucoplan studies. The inc=
idence of serious treatment emergent adverse events (TEAEs) in the zilucopl=
an and placebo treatment arms was similar.
The safety and efficacy of zilucoplan have not been established, and it is =
not approved for use in any indication by any regulatory authority worldwid=
e.=C2=A0
Based on these results, UCB plans to progress with regulatory filings for z=
ilucoplan in gMG in the United States (US), European Union (EU) and Japan, =
beginning later this year. =C2=A0
=E2=80=9CgMG patients can experience varying and debilitating symptoms that=
impact their everyday lives in unique ways,=E2=80=9D said James F. Howard,=
MD, Distinguished Professor of Neuromuscular Disease, Chief, Neuromuscular=
Disorders Section, University of North Carolina School of Medicine and lea=
d investigator in the RAISE trial. =E2=80=9CThese exciting results give us =
additional reason to believe that zilucoplan can offer an important step fo=
rward in addressing the unmet needs of people living with gMG. As we strive=
to improve the management of this complex and unpredictable disease, any n=
ew medicines will be welcomed by physicians to help us realize our goal of =
offering effective and flexible treatment approaches in gMG which are tailo=
red to the needs of individual patients.=E2=80=9D
These findings from RAISE build on the positive results from the Phase 3 My=
carinG study evaluating UCB=E2=80=99s investigational treatment rozanolixiz=
umab, an SC-infused monoclonal antibody targeting the neonatal Fc receptor =
(FcRn) which also met its primary and secondary endpoints with statistical =
significance in gMG.^2
UCB is currently the only company investigating two potential treatments wi=
th different mechanisms of action in gMG. Detailed results from both Phase =
3 trials will be presented at forthcoming medical meetings in 2022.
=E2=80=9CWhile there has been recent progress in the treatment of MG, there=
is still a significant unmet need for new, effective treatment options tha=
t address the unpredictable, fluctuating symptoms of MG =E2=80=93 some of w=
hich require urgent treatment or hospitalization =E2=80=93 to improve patie=
nt outcomes and quality of life,=E2=80=9D said Raquel Pardo, Spanish Myasth=
enia Association (AMES), Spain. =E2=80=9CNew research into additional treat=
ment options will be welcomed by the global MG community.=E2=80=9D
Iris Loew-Friedrich, Executive Vice-President and Chief Medical Officer add=
ed: =E2=80=9CThis is the latest in a series of positive Phase 3 data announ=
cements across UCB=E2=80=99s product pipeline validating our patient value =
strategy and laying foundations for future sustainable growth. Today=E2=80=
=99s results represent another significant milestone in UCB=E2=80=99s effor=
ts to bring transformational outcomes to those living with myasthenia gravi=
s. Positive results for zilucoplan and rozanolixizumab =E2=80=93 each with =
a different mechanism of action =E2=80=93 bring us one step closer to achie=
ving our ambition of delivering choice and flexibility for a broad range of=
patients and physicians at each step of their treatment journey, addressin=
g significant unmet needs and offering unique patient value. We thank the M=
G community for their continued insights, partnership and participation in =
this study.=E2=80=9D
About Generalized Myasthenia Gravis (gMG)
Myasthenia gravis is a rare disease impacting almost 200,000 patients in th=
e U.S., EU and Japan. , =C2=A0People living with gMG can experience a varie=
ty of symptoms, including drooping eyelids, double vision and difficulty sw=
allowing, chewing and talking, as well as severe life- threatening weakness=
of the muscles of respiration.^5-8
gMG is a chronic and unpredictable auto-immune disease in which pathogenic =
autoantibodies can inhibit synaptic transmission at the neuro-muscular junc=
tion by targeting specific proteins on the post-synaptic membrane. This dis=
rupts the ability of the nerves to stimulate the muscle and results in a we=
aker contraction. gMG can occur at any age and in any race, although previo=
us studies have shown that women are more often impacted than men.^9=C2=A0C=
omplement activation, a key mediator of antibody function, is recognized as=
an important driver of pathology in gMG.=C2=A0
About the zilucoplan RAISE study^10,11=C2=A0
The RAISE study (Safety, Tolerability, and Efficacy of Zilucoplan in Subjec=
ts With Generalized Myasthenia Gravis (NCT04115293)) is a multi-center, Pha=
se 3, randomized, double-blind, placebo-controlled study to evaluate the ef=
ficacy, safety, and tolerability of zilucoplan in adult patients with gMG.=
=C2=A0
Patients were randomized in a 1:1 ratio to receive daily subcutaneous (SC) =
doses of zilucoplan or placebo for 12 weeks. The study was designed to dete=
rmine if complete complement inhibition can bring clinical benefit to peopl=
e with gMG and if complement inhibition was effective across a broad spectr=
um of patients with acetylcholine receptor antibody positive (AChR Ab+) MG =
regardless of disease duration, prior treatment or response to previous the=
rapies.=C2=A0
The primary endpoint for RAISE study is change from baseline at Week 12 in =
the Myasthenia Gravis-Activities of Daily Living (MG-ADL) score, an eight-i=
tem patient-reported scale developed to assess MG symptoms and their effect=
s on daily activities. Secondary endpoints include change in the Quantitati=
ve Myasthenia Gravis (QMG) score, the Myasthenia Gravis Composite (MGC) and=
the Myasthenia Gravis Quality of Life 15 revised (MG-QoL15r) from baseline=
to Week 12; time to rescue therapy; the percentage with minimum symptom ex=
pression (MSE) (defined as MG-ADL of 0 or 1), the percentage with a =E2=89=
=A53-point reduction in MG-ADL and the percentage with a =E2=89=A55-point r=
eduction in QMG, all measured at Week 12.
For more information about the trial visit https://clinicaltrials.gov/ct2/s=
how/NCT04115293.=C2=A0
About the rozanolixizumab MycarinG study^12=C2=A0
The MycarinG study (NCT03971422) is a completed multi-center, Phase 3, rand=
omized, double-blind, placebo-controlled study evaluating the efficacy and =
safety of rozanolixizumab in adult patients with gMG, with an open-label ex=
tension.=C2=A0
The primary endpoint for the MycarinG study is change in the Myasthenia Gra=
vis-Activities of Daily Living Profile (MG-ADL) score. Secondary endpoints =
include response rates, changes in the Myasthenia Gravis Composite (MGC) sc=
ore, the Quantitative MG (QMG) score, patient-reported outcomes and adverse=
events (AEs).=C2=A0
A preliminary analysis of results shows the trial met its primary endpoint,=
demonstrating a statistically significant and clinically meaningful change=
from baseline in the MG-ADL total score at Day 43. All secondary endpoints=
were also met with statistical significance. Overall, rozanolixizumab was =
well tolerated and no new safety signals were identified.^13=C2=A0=C2=A0
For more information about the trial, visit https://clinicaltrials.gov/ct2/=
show/NCT03971422.=C2=A0
About Zilucoplan^11,14
Zilucoplan targets complement component 5 (C5), a component of the terminal=
complement activation pathway, and binds to C5 with high affinity and spec=
ificity. This prevents its cleavage by C5 convertases into the complement c=
omponents C5a and C5b. In addition, zilucoplan is understood to bind to the=
domain of C5 that corresponds to C5b and thereby block binding of C5b to c=
omplement component C6.=C2=A0
Inhibition of C5 cleavage prevents the downstream assembly and activity of =
membrane attack complex (MAC). This dual mechanism of action of zilucoplan =
has the potential to prevent activation of the terminal complement pathway =
and downstream assembly and activity of MAC that can damage and destroy the=
postsynaptic membrane, disrupt ionic channel conductance and impair neurom=
uscular transmission.=C2=A0
Zilucoplan is being investigated in the RAISE study, a multi-center, Phase =
3, randomized, double-blind, placebo-controlled study to evaluate the effic=
acy, safety, and tolerability of zilucoplan in subjects with gMG.
Further indications that are potentially addressable by zilucoplan include =
amyotrophic lateral sclerosis (ALS) and other tissue-based complement-media=
ted disorders with high unmet medical need.=C2=A0
Zilucoplan was selected as one of the first drugs to be tested in a multi-c=
enter ALS platform study sponsored by the Sean M. Healey & AMG Center for A=
LS at Massachusetts General Hospital, Boston, MA.=C2=A0
The safety and efficacy of zilucoplan have not been established and it is n=
ot currently approved for use in any indication by any regulatory authority=
worldwide.
About Rozanolixizumab
Rozanolixizumab is a subcutaneously infused humanized monoclonal antibody t=
hat specifically binds, with high affinity, to human neonatal Fc receptor (=
FcRn). It has been designed to block the interaction of FcRn and Immunoglob=
ulin G (IgG), accelerating the catabolism of antibodies and reducing the co=
ncentration of pathogenic IgG autoantibodies.^15,16=C2=A0
Rozanolixizumab is under clinical development with the aim of improving the=
lives of people with pathogenic IgG-autoantibody-driven autoimmune disease=
s, including gMG, primary immune thrombocytopenia (ITP), myelin oligodendro=
cyte glycoprotein antibody-associated disease (MOG-AD) and autoimmune encep=
halitis (AIE) by driving removal of pathogenic IgG autoantibodies.
The safety and efficacy of rozanolixizumab have not been established and it=
is not approved for use in any indication by any regulatory authority worl=
dwide.
About UCB in Rare Diseases=C2=A0
At UCB, we don=E2=80=99t just see patients or population sizes, we see peop=
le in need. Through decades of serving the neurology and immunology communi=
ties, we have improved lives with impactful medicines and by enhancing the =
social and emotional well-being of patients. As a continuation of our herit=
age, we are now expanding our efforts to tackle rare neurological and immun=
ological diseases where current options offer little hope.=C2=A0
Leading with gMG, UCB are progressing development programmes in a number of=
Rare Diseases, including =C2=A0ITP, MOG-AD and AIE. Alongside cutting-edge=
medicines, UCB are also exploring innovative non-drug solutions with the a=
im of delivering a holistic approach to care.=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 more than 7,600 people in=
approximately 40 countries, UCB generated revenue of =E2=82=AC5.3 billion =
in 2020. UCB is listed on Euronext Brussels (symbol: UCB). Follow us on Twi=
tter: @UCB_news
For further information, contact UCB:
Brand Communications
Jim Baxter
Rare Disease Communications, UCB
T+44.0.7900.605.652 jim.baxter@ucb.com
Corporate Communications
Laurent Schots=C2=A0
Media Relations, UCB =C2=A0
T+32.2.559.92.64 =C2=A0Laurent.schots@ucb.com =C2=A0
Investor Relations
Antje Witte =C2=A0 =C2=A0 =C2=A0 =C2=A0=C2=A0
Investor Relations, UCB
T +32.2.559.94.14 antje.witte@ucb.com=C2=A0
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References
1. Data on file. UCB. February 2022.
=C2=A0
2. UCB Press Release, December 10. 2021 https://www.ucb.com/stories-media/P=
ress-Releases/article/UCB-announces-positive-Phase-3-results-for-rozanolixi=
zumab-in-generalized-myasthenia-gravis Accessed February 2022
3. Chen J, et al. Incidence, mortality, and economic burden of myasthenia g=
ravis in China: A nationwide population-based study. Lancet Reg Health West=
Pac.2020;5:10063.
4. Gilhus N. Myasthenia Gravis. N Engl J Med. 2016;375:2570-2581.
5. Lisak, RP. Best Practice Myasthenia gravis. BMJ Best Practice. 2021. Las=
t accessed December 2021
6. Robertson NP, et al. Myasthenia gravis: a population based epidemiologic=
al study in Cambridgeshire, England. J Neurol Neurosurg Psychiatry. 1998;65=
:492-496.
7. Kupersmith MJ et al. Development of generalized disease at 2 years in pa=
tients with ocular myasthenia gravis. Arch Neurol. 2003;60(2):243-248.=C2=
=A0
8. Hansen JS, et al. Mortality in myasthenia gravis: A nationwide populatio=
n-based follow-up study in Denmark. Muscle Nerve. 2016;53:73-77.
9. Myasthenia Gravis Foundation of America. Clinical Overview of MG. https:=
//myasthenia.org/Professionals/Clinical-Overview-of-MG. Accessed November 2=
021.
10. Clinical Trials.gov =E2=80=98Safety, Tolerability, and Efficacy of Zilu=
coplan in Subjects With Generalized Myasthenia Gravis (RAISE)=E2=80=99: htt=
ps://clinicaltrials.gov/ct2/show/NCT04115293. Accessed November 2021.
11. Howard JF, Jr., et al. JAMA Neurol 2020;77:582=E2=80=9392.
12. Clinical Trials.gov =E2=80=98A Study to Test Efficacy and Safety of Roz=
anolixizumab in Adult Patients With Generalized Myasthenia Gravis=E2=80=99:=
=C2=A0https://clinicaltrials.gov/ct2/show/NCT03971422. Accessed February 2=
022.
13. Data on file. UCB. December 2021
14. Ricardo A, et al. Blood 2015;126:939.
15. Kiessling P, et al. The FcRn inhibitor rozanolixizumab reduces human se=
rum IgG concentration: A randomized phase 1 study. Sci Transl Med. 2017;9(4=
14).eaan1208
16. Smith B, et al. Generation and characterization of a high affinity anti=
-human FcRn antibody, rozanolixizumab, and the effects of different molecul=
ar formats on the reduction of plasma IgG concentration. MAbs.2018;10:1111-=
30.
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