Alder BioPharmaceuticals Investor Presentation — May 2019

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Zilucoplan, a Subcutaneously Self -Administered Peptide

Inhibitor of Complement Component 5 (C5), for the

Treatment of Generalized Myasthenia Gravis: Results of a

Phase 2 Randomized, Double -Blind, Placebo -Controlled

Trial and Open -Label Long -Term Extension

James F. Howard, Jr, MD, et al, on behalf of the Zilucoplan MG Study Group

ConfidentialAAN 2019 Emerging Science Session

May 7, 2019, Philadelphia, PARandomized, Double -Blind, Placebo -Controlled Phase 2 Study of

Zilucoplan in a Broad Generalized Myasthenia Gravis Population

Broad Patient Population :

•Generalized myasthenia gravis (MGFA class II -IVa)

•Acetylcholine receptor ( AChR ) antibody positive

•Stable doses of corticosteroids and/or immunosuppressants

•No requirement to be “refractory” or to have failed multiple

•Patients must be vaccinated against meningococcusEndpoints :

•Primary: Change in QMG score from baseline to week 12

•Secondary: Change in MG- ADL, MG Composite, and

MGQoL15r scores from baseline to week 12

•Pre-specified significance testing at a 1 -sided alpha of 0.1

Enrollment : 44 patients (vs. target of 36)

QMG, Quantitative Myasthenia Gravis; MG -ADL, Myasthenia Gravis Activities of Daily Living; MGFA, Myasthenia Gravis Foundation of America; MGQoL15r, 15 -item Myasthenia Gravis Quality -of-Life revised scale; SC, subcutaneous;

SOC, standard of care.2Screening 1:1:1 Randomization 0.1 mg/kg SC + SOC (n=15) Open -Label Extension (n=42)0.3 mg/kg SC + SOC (n=14)

Placebo + SOC (n=15)

Main Study Period (12 w eeks ) Long -Term Extension (12 weeks)

ConfidentialPlacebo arm randomized 1:1 to receive

0.3 mg/kg (n=8) or 0.1 mg/kg (n=6)Zilucoplan Achieved Rapid, Clinically Meaningful, Statistically Significant,

and Sustained Reductions in QMG and MG -ADL

*1-sided analysis of covariance for LS mean change from baseline for 0.3 mg/kg arm vs. placebo; placebo patients re -baselined to zero upon completion of 12 -week main study.

†2-sided t test for LS mean change from week 12 to week 24 for placebo patients crossing over to 0.3 mg/kg (n=8).

‡2-sided t test for LS mean change from week 0 to week 24 for 0.3 mg/kg arm.

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