Pharma Update
Enspryng in neurological disorders gMG, AIE, and MOG-AD
First-in-class anti-IL-6R mAb with best-in-disease potential in AcHR+ gMG
Enspryng (Anti-IL-6 receptor mAb)
gMG: Autoantibodies at the NMJ1
Ph III (LUMINESCE) trial design
Neutral pH
(7.4)
FcRn
Anti-AChR
Anti-MuSK
Nerve terminal
Anti-LRP4
MG patients
(N=186)
MGFA class II-IV, ≥12 yrs
• MG-ADL ≥5
Enspryng
+
SOC
24 weeks
Mechanism
of anti-MuSKs
AChR+, MUSK+, LRP4+
R
Mechanism
autoantibody positive
1:1
Agrin
of anti-AChRs
LRP4
ACh
Blocked ACh-AChR binding
Patients on stable baseline
therapy
Reduced density
Complement
of AChR
No requirement on previous
Acidic
pH
(6.0)
AChE and/or ColQ
Cell
treatment failure/s
AChR-
MuSKO
Rapsyn.
MAC
MAC formation
and damage of
the postsynaptic
membrane
Placebo
+
SOC
Increased AChR
internalization
Primary endpoint:
and degradation
IL-6
receptor
gets
internalized
and
degraded
Roche
•
.
Recycling mAb with high-affinity to soluble
and membrane-bound IL-6R
Engineered to enable maximal inhibition of
IL-6 signaling
• Convenient SC Q4W home administration
Mean change from baseline MG-ADL score at week 24 in
the AChR+ population
• IL-6 blockade has the potential to reduce autoantibody-mediated NMJ damage in gMG
.
High unmet need: ~10-30% of patients fail SOC therapies with only up to 40% of patients on approved
biologics achieve minimal symptom expression²
⚫ Ph III (LUMINESCE) results in gMg expected in 2024
Additional Ph III studies in 2 other autoantibody mediated rare neurological diseases ongoing with first-in-
disease potential: Ph III (CIELO) in AIE and Ph III (METEOROID) in MOG-AD
1.Gilhus N.E. et al., Nat Rev Dis Primers. 2019; 2. Howard JF Jr, et al. Lancet Neurol. 2021 IL-6R-interleukin 6 receptor; MG=myasthenia gravis; gMG= generalized myasthenia gravis; AcHR-acetylcholine receptor;
NMJ-neuromuscular junction; SC=subcutaneous; Q4W=every 4 weeks; MOG-AD=myelin oligodendrocyte glycoprotein antibody-associated disease; LPI=last patient in; AIE-autoimmune encephalitis; SOC=Standard of care;
MGFA-Myasthenia Gravis Foundation of America; MG-ADL-Myasthenia Gravis Activities of Daily Living
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