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** UCB presents efficacy and safety results for zilucoplan and rozanolixizu=
mab in generalized myasthenia gravis
------------------------------------------------------------
=C2=B7 Phase 3 RAISE and MycarinG studies in generalized myasthenia gravis =
(gMG) show zilucoplan and rozanolixizumab improve gMG-specific outcomes wit=
h consistent statistical significance and clinical relevance=C2=A0
=C2=B7 Regulatory submissions for zilucoplan and rozanolixizumab are planne=
d for later this year
=C2=B7 Findings from a real-world smartphone data collection study reaffirm=
UCB=E2=80=99s commitment to delivering pioneering, tailored care solutions=
for individuals living with gMG=C2=A0
Brussels (Belgium), 10 May 2022 =E2=80=93 7:00 (CEST) =E2=80=93 UCB, a glob=
al biopharmaceutical company, today announced results from two Phase 3 stud=
ies evaluating its investigational treatments, zilucoplan, a self-administe=
red, subcutaneous (SC) peptide inhibitor of complement component 5 (C5 inhi=
bitor) and rozanolixizumab, an SC-infused monoclonal antibody targeting the=
neonatal Fc receptor (FcRn) in adults with generalized myasthenia gravis (=
gMG).=C2=A0
These results will be presented as posters at the 14th Myasthenia Gravis Fo=
undation of America (MGFA) International Conference on Myasthenia and Relat=
ed Disorders, taking place from May 10-12, 2022.
Phase 3 RAISE results=C2=A0=C2=A0 =C2=A0
Data from the Phase 3 RAISE trial (NCT04115293) (poster 26)^1=C2=A0demonstr=
ated treatment with zilucoplan (0.3 mg/kg daily) resulted in clinically mea=
ningful and statistically significant improvements in key gMG-specific outc=
omes compared with placebo in patients with acetylcholine receptor autoanti=
body positive (AChR+) gMG.=C2=A0
The study met its primary endpoint with zilucoplan showing a placebo-correc=
ted mean improvement of 2.12 points in the Myasthenia Gravis Activities of =
Daily Living (MG-ADL) score at week 12 (p<0.001). Significant improvement i=
n the MG-ADL was observed from Week 1.=C2=A0
All key secondary endpoints were also met, including statistically signific=
ant improvements in the Quantitative Myasthenia Gravis (QMG) score, Myasthe=
nia Gravis Composite (MGC) and Myasthenia Gravis Quality of Life 15-Item re=
vised (MG-QoL15r), again with significant improvement observed from Week 1.=
The proportion of patients on zilucoplan who responded to treatment by at =
least a 3-point reduction on MG-ADL and at least a 5-point reduction in QMG=
were also significantly higher than placebo. =C2=A0
A favorable safety profile and good tolerability was observed, consistent w=
ith prior data, showing a similar rate of treatment-emergent adverse events=
(TEAEs) between zilucoplan (76.7%) and placebo (70.5%). The most common TE=
AEs were injection site bruising, headache, diarrhea, and MG worsening.
In the RAISE study, 174 patients were randomised to Placebo (N=3D88) and Zi=
lucoplan 0.3 mg/kg (N=3D86). Patient demographics and baseline disease char=
acteristics were generally balanced between treatment arms.
=E2=80=9CThe results from the RAISE study are an exciting development in th=
e gMG treatment paradigm and reinforce the critical role that complement in=
hibition could play for physicians treating patients with this debilitating=
illness. By targeting the underlying mechanisms of gMG at the neuromuscula=
r junction, complement inhibitors like zilucoplan have the potential to pro=
vide rapid, consistent disease control earlier in the disease course. These=
findings are an encouraging sign that we may be able to meet patients=E2=
=80=99 needs effectively, with treatments that are minimally invasive and w=
ell tolerated,=E2=80=9D said James F. Howard, MD, Distinguished Professor o=
f Neuromuscular Disease, Professor of Neurology, Medicine and Allied Health=
, The University of North Carolina at Chapel Hill School of Medicine and le=
ad investigator in the RAISE trial.
Phase 3 MycarinG results
Results from the Phase 3 MycarinG study (NCT03971422) (poster 25)^2 demonst=
rated that rozanolixizumab significantly reduced MG-ADL from baseline to Da=
y 43 at ~7 mg/kg and ~10 mg/kg doses compared with placebo in patients with=
AChR or muscle-specific tyrosine kinase (MuSK) autoantibody positive gMG. =
Rozanolixizumab demonstrated a placebo-corrected mean improvement of 2.586 =
points at the ~7 mg/kg dose and 2.619 points in the MG-ADL at the ~10 mg/kg=
dose compared with placebo (both doses at p<0.001).=C2=A0
Rozanolixizumab treatment reduced mean maximum total IgG levels by more tha=
n 70% (71 % for 7 mg/kg and 78% for 10 mg/kg) and anti-AChR autoantibody le=
vels decreased over the treatment period in line with the total IgG reducti=
on. Rozanolixizumab was generally well tolerated, with the majority of TEAE=
s being mild to moderate in intensity. A higher proportion of TEAEs occurre=
d in the active treatment arms versus placebo (81.3% for ~7 mg/kg, 82.6% fo=
r ~10 mg/kg and 67.2% for placebo). The most frequently reported TEAE was h=
eadache, most of which were mild to moderate, with severe headaches managed=
with over-the-counter analgesic medications. The other most common TEAEs w=
ere diarrhea, pyrexia and nausea.
Due to the fluctuating and unpredictable nature of gMG and the subjectivity=
of symptoms, patient reported outcomes (PROs) help provide greater insight=
into disease impact and more granular detail on the effect of treatments t=
han traditional endpoints. Within the MycarinG study, the MG Symptoms PRO m=
easure, which was developed by UCB with patients, was used to assess a seri=
es of quality-of-life measures, including muscle weakness fatigability, phy=
sical fatigue and bulbar muscle weakness, throughout the treatment and obse=
rvation periods (poster 64)^3. All three MG Symptoms PRO scales or subdomai=
ns showed significant improvement from baseline with rozanolixizumab ~7 mg/=
kg and ~10 mg/kg doses compared with placebo at Day 43, indicating treatmen=
t with rozanolixizumab improves patients=E2=80=99 symptoms and their abilit=
y to undertake daily activities. Further evaluation of the MG Symptoms PRO =
measure is ongoing.
In the MycarinG study, a total of 66 patients were randomized to rozanolixi=
zumab 7 mg/kg, 67 to rozanolixizumab 10 mg/kg and 67 to placebo. Baseline c=
haracteristics were generally balanced between treatment groups.
=E2=80=9CThe one constant in gMG is unpredictability. People living with th=
is disease experience symptoms that are nebulous, fluctuating, and which va=
ry from one day to the next. For this reason, there is an urgent need for m=
ore effective, targeted, and convenient treatments that reduce the burden o=
f disease on patients=E2=80=99 daily lives. The results from the MycarinG s=
tudy are extremely encouraging, and demonstrate the potential of rozanolixi=
zumab as a new, effective and flexible treatment option to help ease the da=
y-to-day burden of this challenging disease and improve treatment outcomes =
for patients,=E2=80=9D said Professor Vera Bril, Professor of Medicine (Neu=
rology), University of Toronto, Director of the Neuromuscular Section, Divi=
sion of Neurology, University of Toronto and University Health Network, Tor=
onto, and lead investigator of the MycarinG study.
=E2=80=9CEvery person living with gMG is unique, so a one-size-fits-all tre=
atment approach will never be appropriate. This is why at UCB we are invest=
igating two medicines with different MOAs. This unique approach means we ma=
y be able to offer physicians and patients a range of treatment options to =
meet the individual needs of many different patients, and therefore leave n=
o one behind in our ambition to improve outcomes in gMG,=E2=80=9D said Iris=
Loew-Friedrich, Executive Vice-President and Chief Medical Officer at UCB.
UCB anticipates filing regulatory submissions for both zilucoplan and rozan=
olixizumab later this year.
Digital innovation for patients living with myasthenia gravis
Findings from a three-month observational study carried out by UCB in colla=
boration with digital health company Sharecare, to collect real-world data =
from gMG patients using smartphones, were also presented at the conference =
(poster 84)^4. Beyond demonstrating that decentralized, smartphone-based me=
thods to collect real-world data from gMG patients are feasible and may pro=
vide enhanced visibility into the burden of the disease, the study also ide=
ntified unique clusters of exacerbation subtypes with specific symptom repr=
esentation. This requires further validation but suggests that digital phen=
otyping holds promise for furthering understanding of exacerbations. This a=
nalysis forms part of UCB=E2=80=99s holistic approach to delivering differe=
ntiated patient experiences, combining patient insights, pioneering researc=
h and artificial intelligence to deliver tailored support for individuals l=
iving with gMG.=C2=A0
=E2=80=9COur gMG pipeline is supported by a holistic and integrated platfor=
m of support services and digital innovation that aims to deliver broad acc=
ess to seamless and flexible services across the gMG care continuum,=E2=80=
=9D continued Charl van Zyl, Executive Vice President Neurology & Head of E=
urope/International Markets at UCB =E2=80=9COur ultimate goal is to transfo=
rm the lives of people living with this challenging disease by putting pati=
ents at the center of our world and advancing scientific solutions that mee=
t their complex and varying needs.=E2=80=9D=C2=A0
For further information, contact UCB:=C2=A0
Brand Communications, Rare Diseases
Jim Baxter
T+32.2.473.78.85.01=C2=A0
jim.baxter@ucb.com=C2=A0
Corporate Communications, Media Relations
Laurent Schots=C2=A0
T+32.2.559.92.64 =C2=A0
Laurent.schots@ucb.com=C2=A0
Investor Relations
Antje Witte =C2=A0 =C2=A0 =C2=A0 =C2=A0=C2=A0
T +32.2.559.94.14=C2=A0
antje.witte@ucb.com
About Generalized Myasthenia Gravis (gMG)
Myasthenia gravis is a rare disease impacting almost 200,000 patients in th=
e U.S., EU and Japan.^5,6=C2=A0 People living with gMG can experience a var=
iety of symptoms, including drooping eyelids, double vision and difficulty =
swallowing, chewing and talking, as well as severe muscular weakness that c=
an result in life threatening weakness of the muscles of respiration.^7
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^8. gMG can occur at any age and in any race, although prev=
ious studies have shown that women are more often impacted than men.^8 Most=
patients with gMG have pathogenic IgG antibodies that disrupt the transmis=
sion of nerve impulses to muscles in the NMJ and some activate the compleme=
nt cascade. =C2=A0Complement-mediated destruction via MAC formation is a ke=
y mechanism causing damage at the NMJ and is the key driver of disease in A=
ChR Ab+ gMG.
About the zilucoplan RAISE study^9
The RAISE study (NCT04115293) is a multi-center, Phase 3, randomized, doubl=
e-blind, placebo-controlled study to confirm the efficacy, safety, and tole=
rability of zilucoplan in patients with gMG. Patients will be randomized in=
a 1:1 ratio to receive daily subcutaneous (SC) doses of zilucoplan or plac=
ebo for 12 weeks.
The primary endpoint for RAISE study is change from baseline to Week 12 in =
the Myasthenia Gravis-Activities of Daily Living (MG-ADL) score. Secondary =
endpoints include change in the Quantitative 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, the proportion requi=
ring rescue therapy, the proportion with minimum symptom expression (MSE) (=
defined as MG-ADL of 0 or 1), the proportion with a =E2=89=A53-point reduct=
ion in MG-ADL and the proportion with a =E2=89=A55-point reduction in QMG, =
all measured at Week 12. The majority of patients taking part in the RAISE =
trial opted to enroll in any future extensions to this clinical trial. As a=
result, UCB is exploring the potential for further extension studies into =
this treatment.
For more information about the trial visit https://clinicaltrials.gov/ct2/s=
how/NCT04115293.=C2=A0
About the rozanolixizumab MycarinG study^10=C2=A0
The MycarinG study (NCT03971422) is a multi-center, Phase 3, randomized, do=
uble-blind, placebo-controlled study evaluating the efficacy and safety of =
rozanolixizumab in adult patients with gMG, with an open-label extension.=
=C2=A0
The primary endpoint for the MycarinG study is change in the Myasthenia Gra=
vis-Activities of Daily Living Profile (MG-ADL) score, an eight-item patien=
t-reported scale developed to assess MG symptoms and their effects on daily=
activities. Additional endpoints include response rates, changes in the My=
asthenia Gravis composite (MGC) score, the Quantitative MG (QMG) score, pat=
ient-reported outcomes and adverse events (AEs). The majority of patients t=
aking part in the MycarinG study opted to enroll in any future extensions t=
o this clinical trial. As a result, UCB is exploring the potential for furt=
her extension studies into this treatment.
For more information about the trial, visit https://clinicaltrials.gov/ct2/=
show/NCT03971422.=C2=A0
About Zilucoplan=C2=A0
Zilucoplan is a once-daily self-administered SC peptide inhibitor of comple=
ment component 5 (C5 inhibitor) under clinical development by UCB in gMG. R=
esults from the RAISE study, a multi-center, Phase 3, randomized, double-bl=
ind, placebo-controlled study demonstrate the efficacy, safety, and tolerab=
ility of zilucoplan in patients with gMG, and regulatory submissions are pl=
anned in 2022.=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 SC administered, humanized monoclonal antibody that sp=
ecifically binds, with high affinity, to human neonatal Fc receptor (FcRn).=
It has been designed to block the interaction of FcRn and Immunoglobulin G=
(IgG), accelerating the catabolism of antibodies and reducing the concentr=
ation of pathogenic IgG autoantibodies.^11,12
Rozanolixizumab is under clinical development with the aim of improving the=
lives of people with pathogenic IgG-autoantibody-driven autoimmune disease=
s.
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.
About UCB=C2=A0
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 8,500 people in=
approximately 40 countries, UCB generated revenue of =E2=82=AC5.8 billion =
in 2021. UCB is listed on Euronext Brussels (symbol: UCB). Follow us on Twi=
tter: @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=
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ates=E2=80=9D, =E2=80=9Cmay=E2=80=9D, =E2=80=9Cwill=E2=80=9D, =E2=80=9Ccont=
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e actual results, financial condition, performance or achievements of UCB, =
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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=
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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=
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References
1. Outcomes from RAISE: A randomized, placebo-controlled, double-blind, Pha=
se 3 trial of zilucoplan in generalized myasthenia gravis. Howard JF et al.=
Data presented at MGFA International Conference 2022. Poster 26.
2. Efficacy and safety of rozanolixizumab in patients with generalized myas=
thenia gravis: A randomized, multicenter, double-blind, placebo-controlled,=
Phase 3 study (MycarinG). Bril V et al. Data presented at MGFA Internation=
al Conference 2022. Poster 25
3. Patient-reported and quality-of-life outcomes from MycarinG, a randomize=
d, placebo-controlled, double-blind, Phase 3 trial of rozanolixizumab in ge=
neralized myasthenia gravis. Habib A et al. Data presented at MGFA Internat=
ional Conference 2022. Poster 64
4. A decentralized, prospective, observational study to collect real-world =
data from patients with myasthenia gravis using smartphones. Steyaert S et =
al. Data presented at MGFA International Conference 2022. Poster 84.
5. 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.
6. Gilhus N. Myasthenia Gravis. N Engl J Med. 2016;375:2570-2581.
7. Hansen JS, et al. Mortality in myasthenia gravis: A nationwide populatio=
n-based follow-up study in Denmark. Muscle Nerve. 2016;53:73-77.
8. Myasthenia Gravis Foundation of America. Clinical Overview of MG. https:=
//myasthenia.org/Professionals/Clinical-Overview-of-MG. Accessed May 2022.
9. Clinical Trials.gov =E2=80=98Safety, Tolerability, and Efficacy of Ziluc=
oplan in Subjects With Generalized Myasthenia Gravis (RAISE)=E2=80=99: http=
s://clinicaltrials.gov/ct2/show/NCT04115293. Accessed May 2022.
10. 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 May 2022.
11. 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).
12. 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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