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Apellis Presents First Data on APL-2 in C3 Glomerulopathy at ASN Kidney Week

November 8, 2019
APL-2 demonstrated positive preliminary results across key renal measures including a nearly 50% reduction in proteinuria

CRESTWOOD, Ky., and WALTHAM Mass., Nov. 08, 2019 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a clinical-stage biopharmaceutical company focused on the development of novel therapeutic compounds to treat disease through the inhibition of the complement system, today announced positive preliminary results in the C3 glomerulopathy (C3G) cohort of the Phase 2 DISCOVERY study. The data showed that APL-2 (pegcetacoplan), an investigational C3-targeted inhibitor, may have the potential to target the underlying disease process of C3G. Results through study day 84 (12 weeks) were presented at the American Society of Nephrology (ASN) Kidney Week 2019 in Washington D.C., and based on these results, Apellis plans to advance development of APL-2 in C3G.

In the data presented on six C3G patients treated with APL-2, there was a downward trend in mean proteinuria as measured by the urine protein-to-creatinine ratio (uPCR). Mean (SE) uPCR decreased from 2.03 (0.46) mg/mg to 1.05 (0.23) mg/mg at study day 84 (normal range <0.200 mg/mg), and a corresponding normalization of mean serum albumin was observed. Serum C3 levels also increased in all six of these patients. No serious adverse events and no discontinuations due to adverse events were reported in individuals with C3G.

“There is a substantial unmet need for medicines that can prevent the kidney failure that often results from C3G,” said independent expert Matthew Pickering, MB BS, PhD, Professor of Rheumatology and Wellcome Trust Senior Fellow in Clinical Science at Imperial College London. “This novel investigational therapy showed a reduction in mean proteinuria of nearly 50% at day 84. The study data provide early evidence that APL-2 may have the potential to impact disease progression in people with C3G.”

There are no medicines currently approved for C3G, a rare disease that leads to kidney failure within five to 10 years of diagnosis in approximately 50% of people. In C3G, a part of the immune system known as the complement cascade is overactive, which results in the excessive breakdown of a protein called C3. These C3 breakdown products become trapped in the kidney, causing inflammation and damage to the organ. Proteinuria, or loss of blood proteins (e.g. albumin) in the urine, is a common finding in people with C3G, and an indication that the disease is damaging the kidneys.

“We’re encouraged by these early results, which show that APL-2 may have the potential to address the underlying disease process of C3G,” said Federico Grossi, MD, PhD, Chief Medical Officer of Apellis. “We look forward to speaking with regulators in the coming months about advancing clinical development of APL-2 in C3G.”

Detailed Preliminary Results in C3G Cohort of DISCOVERY Trial

The DISCOVERY trial is a Phase 2, open-label study investigating the safety and biological activity of APL-2 in four renal diseases: C3G, immunoglobulin A (IgA) nephropathy, primary membranous nephropathy (PMN), and lupus nephritis (LN). The primary endpoint is change from baseline in proteinuria at week 48 as quantified by uPCR. Secondary endpoints include analysis of serum C3 and estimated glomerular filtration rate (eGFR).

Preliminary data presented at Kidney Week 2019 included safety and demographic data from all eight patients in the C3G cohort through study day 84, and data on renal parameters and complement levels in the six C3G patients who received consistent APL-2 administration from study day 1 to study day 84. In those six patients, the percentage reduction in mean uPCR was 48.23% (11.22), and mean serum albumin normalized from baseline, 3.30 (0.32) g/dL, (normal range 3.50-5.50 g/dL) to study day 84, 3.98 (0.20) g/dL. All six of these patients also had an increase in serum C3 levels following APL-2 administration, and mean plasma C5b-9 levels had decreased by day 28 of APL-2 treatment. Mean serum creatinine and eGFR, markers of kidney function, were stable through study day 84.

About APL-2 (pegcetacoplan)
APL-2, an investigational drug, is designed to inhibit the complement cascade at C3 and may have the potential to treat a wide range of complement-mediated diseases. APL-2 is a synthetic cyclic peptide conjugated to a polyethylene glycol (PEG) polymer that binds specifically to C3 and C3b. Apellis is evaluating APL-2 in clinical studies in several serious diseases, including paroxysmal nocturnal hemoglobinuria (PNH), geographic atrophy (GA), cold agglutinin disease (CAD) and C3G. For additional information regarding our clinical trials, visit

About Apellis

Apellis Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development of novel therapeutic compounds for the treatment of a broad range of life-threatening or debilitating autoimmune diseases based upon complement immunotherapy through the inhibition of the complement system at the level of C3. Apellis is the first company to advance chronic therapy with a C3 inhibitor into clinical trials. For additional information about Apellis and APL-2, please visit

Dr. Pickering is providing scientific advice to Apellis in his capacity as an international expert on C3 glomerulopathy, independently, via Imperial Consultants.

Apellis Forward-Looking Statement

Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements” within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the implications of preliminary clinical data. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: whether preliminary or interim results from a clinical trial will be predictive of the final results of the trial; whether results obtained in preclinical studies and clinical trials such as the results reported in this release will be indicative of results that will be generated in future clinical trials; whether APL-2 will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of such clinical trials will warrant regulatory submissions and whether APL-2 will receive approval from the United States Food and Drug Administration or equivalent foreign regulatory agencies for GA, PNH, C3G or any other indication; whether, if Apellis’ products receive approval, they will be successfully distributed and marketed; and other factors discussed in the “Risk Factors” section of Apellis’ Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 5, 2019 and the risks described in other filings that Apellis may make with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Apellis specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

Media Contact:
Tracy Vineis

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Source: Apellis Pharmaceuticals, Inc.