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In The Spotlight: Apellis Pharma

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Shares of Apellis Pharmaceuticals Inc. (APLS), which touched a new high of $29.34 on Wednesday, are up nearly 26% year-to-date while the iShares NASDAQ Biotechnology Index (ETF)(IBB) is down 1.3% for the same period.

Apellis is a clinical-stage biopharmaceutical company focused on developing novel therapeutic compounds for autoimmune and inflammatory diseases.

The Company's lead product candidate is APL-2, whose potential is being tested as a treatment in the areas of ophthalmology, hematology, and nephrology.

APL-2 (intravitreal) has been successfully studied in a phase II clinical trial in patients with geographic atrophy associated with age-related macular degeneration, dubbed FILLY.

Geographic atrophy is a devastating complication of age-related macular degeneration (AMD), causing severe vision loss (Source: NCBI).

In the FILLY trial, the results of which were announced last August, at 12 months, APL-2, administered monthly via intravitreal injection, showed a 29% reduction in the rate of geographic atrophy lesion growth compared to sham. After the 12 month dosing period, subjects were followed for a further six months without treatment. During this period of non-treatment, GA growth resumed but treatment effect was maintained through 18 months, according to the Company.

APL-2 in subcutaneous formulation is under two phase Ib trials in paroxysmal nocturnal hemoglobinuria - one as monotherapy, dubbed PADDOCK, and the other as add-on to Soliris, known as PHAROAH.

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic, life-threatening blood disease characterized by severe anemia, severe abdominal pain, severe headaches, back pain, excessive weakness, fatigue, and recurrent infections.

Last December, the Company presented an update on its phase Ib PHAROAH trial involving 4 patients treated with daily doses of APL-2 270mg in combination with Soliris for at least 12 months.

At baseline (prior to the start of the treatment), the patients' average hemoglobin level was 8.9 g/dL and the average number of transfusions in the 12 months preceding initiation of treatment was 5.25. At one year of treatment, all four patients remain transfusion independent with an average hemoglobin level of 11.6 g/dL.

A phase II study of APL-2 in auto-immune hemolytic anemia (AIHA), and phase II studies of APL-2 in four types of complement-dependent nephropathies, including IgA nephropathy, C3 glomerulonephropathy, primary membranous nephropathy, and lupus nephritis are also ongoing.

Near-term Catalysts:

-- The phase III trial design of APL-2 for the treatment of patients with geographic atrophy associated with AMD (GA) has been finalized, and the study is expected to begin in the second half of 2018.
-- The Company expects to report APL-2 phase 1b Soliris add-on and monotherapy expansion trial updates in the first half of 2018.
-- A phase III trial of APL-2 in PNH remains on track to begin in the second half of 2018.
-- The phase II monotherapy data for APL-2 in auto-immune hemolytic anemia remains on track for the first half of 2018.
-- The phase II monotherapy data for APL-2 in four types of complement-dependent nephropathies are expected in the second half of 2018.

Recent event:

On November 9, 2017, Apellis made its debut on the NASDAQ Global Select Market, offering its shares at a price of $14.00 each. The total gross proceeds from the IPO were approximately $150.0 million.

APLS has thus far hit a low of $12.45 and a high of $29.34. The stock closed Wednesday's (Apr.11) trading at $27.27, up 8.04%.

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