APLS Apellis Pharmaceuticals Inc

Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced that the abstract of the Phase 3 VALIANT study of pegcetacoplan in C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) was accepted as an oral presentation during the High Impact Clinical Trial session at the American Society of Nephrology (ASN) Kidney Week. Two additional abstracts on pegcetacoplan in C3G and primary IC-MPGN were accepted as poster presentations. Kidney Week is being held October 23-27 in San Diego, CA.

Following the presentation at ASN, the Company will host a webinar to discuss the detailed VALIANT results.

High Impact Clinical Trials Oral Presentation Details:

• VALIANT: A Randomized, Multicenter, Double-Blind, Placebo (PBO)-Controlled, Phase 3 Trial of Pegcetacoplan for Patients with Native or Post-transplant Recurrent Glomerulopathy (C3G) or Primary Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN) – Carla Nester, MD, MSA, FASN – High-Impact Clinical Trials 2 – Hall D, Convention Center – Saturday, October 26 – 11:00 a.m. PT

Webinar Details:

• Phase 3 VALIANT Study Results – Carla Nester, MD, MSA, FASN, Jean E. Robillard, M.D., professor of pediatrics, director, division of pediatric nephrology, Stead Family Children’s Hospital, and principal investigator of VALIANT; and Cedric Francois, MD, PhD, chief executive officer at Apellis – Saturday, October 26 – 12:30 p.m. PT
• The live audio webcast and subsequent replay can be accessed through the “Events and Presentations” page of the “Investors and Media” section of the company’s website.

Additional Poster Presentations:

• Pegcetacoplan for Post-transplant Recurrent C3 Glomerulopathy (C3G) or Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN) in NOBLE: 52-Week Patient Evolution – Anuja Java, MD – C3G, TMA, MGRS, Amyloidosis, and More – Exhibition Hall, Convention Center – Saturday, October 26 – 10:00 a.m. PT
• Long-Term Safety and Efficacy of Pegcetacoplan in Patients with C3 Glomerulopathy or Primary Immune-Complex Membranoproliferative Glomerulonephritis: The Long-Term VALE Extension Study – Carla Nester, MD, MSA, FASN – C3G, TMA, MGRS, Amyloidosis, and More – Exhibition Hall, Convention Center – Saturday, October 26 – 10:00 a.m. PT

About the VALIANT StudyThe VALIANT Phase 3 study (NCT05067127) is a randomized, placebo-controlled, double-blinded, multi-center study designed to evaluate pegcetacoplan efficacy and safety in 124 patients who are 12 years of age and older with C3G or primary IC-MPGN. It is the largest single trial conducted in these populations and the only study to include adolescent and adult patients, with native and post-transplant kidneys. Study participants were randomized to receive 1080 mg of pegcetacoplan or placebo twice weekly for 26 weeks. Following this 26-week randomized controlled period, patients are able to proceed to a 26-week open-label phase in which all patients receive pegcetacoplan. The primary endpoint of the study was the log transformed ratio of urine protein-to-creatinine ratio (uPCR) at Week 26 compared to baseline.

About C3 Glomerulopathy (C3G) and Primary Immune-Complex Membranoproliferative Glomerulonephritis (IC-MPGN)C3G and primary IC-MPGN are rare and debilitating kidney diseases that can lead to kidney failure. Excessive C3c deposits are a key marker of disease activity, which can lead to kidney inflammation, damage, and failure. There are no treatments that target the underlying cause of these diseases. Approximately 50% of people living with C3G and IC-MPGN suffer from kidney failure within five to 10 years of diagnosis, requiring a burdensome kidney transplant or lifelong dialysis.1 Additionally, 90% of patients who previously received a kidney transplant will experience disease recurrence.2 The diseases are estimated to affect 5,000 people in the United States and up to 8,000 in Europe.3 

About Pegcetacoplan in Rare DiseasesPegcetacoplan is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, a part of the body’s immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is under investigation for rare diseases across hematology and nephrology. Pegcetacoplan is approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) as EMPAVELI®/Aspaveli® in the United States, European Union, and other countries globally.

About ApellisApellis Pharmaceuticals, Inc. is a global biopharmaceutical company that combines courageous science and compassion to develop life-changing therapies for some of the most challenging diseases patients face. We ushered in the first new class of complement medicine in 15 years and now have two approved medicines targeting C3. These include the first-ever therapy for geographic atrophy, a leading cause of blindness around the world. We believe we have only begun to unlock the potential of targeting C3 across serious retinal, rare, and neurological diseases. For more information, please visit http://apellis.com or follow us on Twitter and LinkedIn.

Apellis Forward-Looking StatementStatements 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 regarding timing of anticipated regulatory submissions. 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, as discussed in the “Risk Factors” section of Apellis’ Annual Report on Form 10-K filed with the Securities and Exchange Commission on February 21, 2023 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 media@apellis.com617.420.4839

Investor Contact: Meredith Kaya meredith.kaya@apellis.com617.599.8178

References 1. C3 glomerulopathy. National Institute of Health, Genetics Home Reference. https://ghr.nlm.nih.gov/condition/c3-glomerulopathy#resources. Accessed November 21, 2019.  2. Tarragón, B, et al. C3 Glomerulopathy Recurs Early after Kidney Transplantation in Serial Biopsies Performed within the First 2 Years after Transplantation. Clinical Journal of the American Society of Nephrology. August 2024; 19(8)1005-1015. doi: 10.2215/CJN.0000000000000474. 3. Data on file using literature consensus.