1. - venBio promoted Richard Gaster M.D., Ph.D. to Managing Partner

    venBio today announced the closing of venBio Global Strategic Fund IV, LP ("venBio Fund IV"), its fourth life sciences venture capital fund, exceeding its target and closing on approximately $550 million in capital commitments in an oversubscribed fundraise. The capital was raised from existing and new investors, including a broad range of institutional investors comprising corporate pensions, financial institutions, university endowments and foundations, family offices and funds-of-funds.

    Led by Managing Partners Corey Goodman, Ph.D., Robert Adelman, M.D., Aaron Royston, M.D., and Richard Gaster, M.D., Ph.D., venBio Fund IV will continue to invest primarily in therapeutics…

    - venBio promoted Richard Gaster M.D., Ph.D. to Managing Partner

    venBio today announced the closing of venBio Global Strategic Fund IV, LP ("venBio Fund IV"), its fourth life sciences venture capital fund, exceeding its target and closing on approximately $550 million in capital commitments in an oversubscribed fundraise. The capital was raised from existing and new investors, including a broad range of institutional investors comprising corporate pensions, financial institutions, university endowments and foundations, family offices and funds-of-funds.

    Led by Managing Partners Corey Goodman, Ph.D., Robert Adelman, M.D., Aaron Royston, M.D., and Richard Gaster, M.D., Ph.D., venBio Fund IV will continue to invest primarily in therapeutics companies that are developing biopharmaceuticals for unmet medical needs. The venBio team takes an active role with each of their portfolio companies, providing strategic guidance on a range of business activities including intellectual property, chemistry, manufacturing and controls (CMC), as well as assisting with clinical trials: from trial design to endpoints to regulatory deliberations.

    "We remain committed to our unique approach and strategy and hope the results speak for themselves – our portfolio companies have delivered four drugs to market for six clinical indications, and another seven drug candidates are demonstrating promising late-stage efficacy," said Dr. Adelman.

    "Our portfolio is directly impacting patient lives and we could not have accomplished that without the ongoing commitment from our limited partners, and we are grateful for their continued support for Fund IV," said Dr. Goodman. "With Fund IV we intend to continue our proven approach of helping to build 12-15 companies per fund while doubling down on winners by providing stronger support for our portfolio companies in crossover rounds and at IPO."

    "We are delighted to announce with the closing of Fund IV, the promotion of Dr. Rich Gaster to Managing Partner," said Dr. Royston. "Our core investment team and investment strategy remain the same as we launch our new fund."

    "Our strategy at venBio has always been to turn exceptional science into impactful medicine," said Dr. Gaster. "Every member of our team is involved in every investment that we make, and we believe this collaborative approach is what helps drive our success."

    Sidley Austin LLP served as legal adviser to venBio.

    About venBio

    Established in 2011, venBio is a life science venture capital firm that focuses on novel therapeutics for unmet medical needs. Since inception in 2011, venBio has raised nearly $1.5 billion in capital commitments and led investment rounds in 34 companies, including: venBio-founded Labrys Biologics (acquired by Teva) and ALX Oncology (NASDAQ:ALXO); Aragon Pharmaceuticals (acquired by Johnson & Johnson); Seragon Pharmaceuticals (acquired by Roche); Aurinia Pharmaceuticals (NASDAQ:AUPH); Apellis Pharmaceuticals (NASDAQ:APLS); Turning Point Therapeutics (NASDAQ:TPTX); Precision Biosciences (NASDAQ:DTIL); Akero Therapeutics (NASDAQ:AKRO); Harmony Biosciences (NASDAQ:HRMY); and Pharvaris (NASDAQ:PHVS). For more information, please visit www.venbio.com.

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  2. WALTHAM, Mass., June 03, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company will participate in the following investor conferences in June:

    • Goldman Sachs Healthcare Conference: Fireside chat on Thursday, June 10, 2021 at 8:00 a.m. ET.
    • Bank of America Napa Biopharma Conference: Fireside chat on Wednesday, June 16, 2021 at 4:30 p.m. ET.

    The Conference events will be available via live webcast from the "Events and Presentations" page of the "Investors and Media" section of the company's website at https://investors.apellis.com/events-and-presentations. Replays of the webcasts will be available for 90 days following the event…

    WALTHAM, Mass., June 03, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company will participate in the following investor conferences in June:

    • Goldman Sachs Healthcare Conference: Fireside chat on Thursday, June 10, 2021 at 8:00 a.m. ET.
    • Bank of America Napa Biopharma Conference: Fireside chat on Wednesday, June 16, 2021 at 4:30 p.m. ET.

    The Conference events will be available via live webcast from the "Events and Presentations" page of the "Investors and Media" section of the company's website at https://investors.apellis.com/events-and-presentations. Replays of the webcasts will be available for 90 days following the event.

    About Apellis

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit www.apellis.com.

    Investor Contact:

    Argot Partners



    212.600.1902

     



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  3. NEW YORK, June 1, 2021 /PRNewswire/ -- Acuamark Diagnostics, Inc. ("AcuamarkDx"), a biotechnology company that revolutionizes the global fight against cancer with first-in-kind early-cancer detection technology, today announced that Dr. Cedric Francois has joined its Board of Directors.

    Dr. Cedric Francois, MD, PhD is the visionary Co-Founder and CEO of Apellis Pharmaceuticals (NASDAQ:APLS), which he and his cofounders built from the ground up from novel scientific concept to a life-changing pipeline, and successfully navigated through development, rapid team growth, public listing, and recently, commercialization.

    With his strong leadership skills and experience in growing biotechnology companies, the addition of Dr. Cedric Francois further strengthens AcuamarkDx's Board of Directors team and complements the organization's team of world-class professionals during its next phase of growth. 

    "Early cancer detection – if done right – will make an enormous difference for cancer survival worldwide. I have followed Acuamark Diagnostics over the years and its progress has been nothing less than remarkable.  Its approach for much improved early-cancer detection is fundamentally differentiated, and I cannot wait to see the Company enter the next phase. I look forward working with Bernard and other Directors to bring the Company's much-needed democratized solutions to the broader population and help make AcuamarkDx a tremendous success." - Dr. Cedric Francois,

    Most cancer cases are detected too late, resulting in poor cancer survival of ~50% in the aggregate (globally). AcuamarkDx's unique genetic early-cancer screening technology is designed to produce higher true positive and lower false positive results for early cancer detection, seamlessly and cost-effectively, from a simple tube of blood.

    "We are delighted to welcome to our Board an executive of Cedric's breadth of experience in managing a life science company from an early stage to a successful public company". - William Gedale, Executive Chairman

    "Dr. Francois is first and foremost an incredible human being, deeply compassionate and singularly focused on real solutions which can make real impact to the patient. The addition of such an experienced operator and entrepreneur, someone who successfully navigated both explosive company growth as well as financial markets, is a tremendous value-add to our Board as the company looks towards the future. We are honored to welcome Cedric to our Board of Directors." - Dr. Bernard Peperstraete, MD, Co-Founder and CEO

    For more information, please visit www.acuamarkdx.com, or contact us at .

    About Cedric Francois, MD, PhD

    Dr. Cedric Francois, is Co-Founder and CEO of Apellis Pharmaceuticals (NASDAQ:APLS). Dr Francois and his co-founders were the first group to test complement-inhibiting drug candidates for a range of diseases (e.g. AMD, GA, PNH, etc.). Dr Francois successfully led Apellis through its early development stages, cross-over and IPO, and now leads APLS' rapidly expanding team, therapy portfolio and commercialization efforts.

    Dr. Francois has numerous publications and is inventor on several biotechnology patent applications. He received his MD degree from the Catholic University of Leuven in Belgium and his PhD in physiology from the University of Louisville. Following postgraduate training in pediatric and transplant surgery, Dr. Francois joined the research team that performed the first successful hand transplantation in Louisville in 1999.

    About Acuamark Diagnostics Inc.

    Acuamark Diagnostics is a biotechnology company focused on developing a differentiated approach for  blood-based early-cancer detection, specifically designed to better meet the biological and scale challenges of early-detection. Backed by a highly experienced team of industry developers and scientific experts, it is AcuamarkDx's mission to improve cancer management and survival by intercepting early-cancer more reliably, accessibly and cost-effectively, with first-in-kind, high-precision genetic detection assays.

    Media Contact:

    Bernard Peperstraete, MD

    Chief Executive Officer

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/acuamark-diagnostics-welcomes-dr-cedric-francois-to-its-board-of-directors-301301296.html

    SOURCE AcuamarkDx

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  4. STOCKHOLM, May 25, 2021 /PRNewswire/ -- Swedish Orphan Biovitrum AB (publ) (Sobi™) and Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) (GLOBE NEWSWIRE) today reported positive top-line results from the phase 3 PRINCE study evaluating the efficacy and safety of pegcetacoplan in adults with paroxysmal nocturnal haemoglobinuria (PNH) who are treatment naïve, meaning they had not received a complement inhibitor within three months before entering the study.

    Pegcetacoplan demonstrated statistical superiority on the co-primary endpoints of haemoglobin stabilization and reduction in lactate dehydrogenase (LDH) compared to standard of care, which did not include complement inhibitors, at week 26.

    • 86 per cent of pegcetacoplan treated patients achieved haemoglobin…

    STOCKHOLM, May 25, 2021 /PRNewswire/ -- Swedish Orphan Biovitrum AB (publ) (Sobi™) and Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) (GLOBE NEWSWIRE) today reported positive top-line results from the phase 3 PRINCE study evaluating the efficacy and safety of pegcetacoplan in adults with paroxysmal nocturnal haemoglobinuria (PNH) who are treatment naïve, meaning they had not received a complement inhibitor within three months before entering the study.

    Pegcetacoplan demonstrated statistical superiority on the co-primary endpoints of haemoglobin stabilization and reduction in lactate dehydrogenase (LDH) compared to standard of care, which did not include complement inhibitors, at week 26.

    • 86 per cent of pegcetacoplan treated patients achieved haemoglobin stabilisation compared to 0 per cent of patients on standard of care (p<0.0001). Haemoglobin stabilisation was defined as an avoidance of a >1 g/dL decrease in haemoglobin levels in the absence of transfusions.
    • Mean LDH in the pegcetacoplan group decreased by 90 per cent from a baseline of 2151 U/L [9.5x upper limit of normal (ULN)] to 211 U/L, which is within the normal range, compared to a 14 per cent reduction on standard of care from a baseline of 1946 U/L (8.6x ULN) to 1681 U/L (7.4x ULN) (p<0.0001).

    "The positive PRINCE data showed that pegcetacoplan provided clinically meaningful improvements across multiple measures that are important for patients and build on our recent FDA approval of pegcetacoplan in PNH," said Federico Grossi, M.D., Ph.D., chief medical officer, Apellis. "Combined with previous studies, these results emphasize the potential of pegcetacoplan to provide disease control for all adults with PNH regardless of prior treatment."

    Pegcetacoplan also achieved statistical superiority on several secondary endpoints, including improvements in haemoglobin levels and transfusion avoidance, compared to standard of care, which did not include complement inhibitors.

    • Mean haemoglobin levels in the pegcetacoplan group increased from 9.4 g/dL to 12.1 g/dL compared to an increase from a baseline of 8.7 g/dL to 9.4 g/dL on standard of care (p=0.0019).
    • 91 per cent of patients on pegcetacoplan were transfusion free compared to 22 per cent on standard of care (p<0.0001).

    The safety profile of pegcetacoplan was consistent with previous studies. At week 26, 9 per cent of patients in the pegcetacoplan group experienced a serious adverse event (SAE) compared to 17 per cent on standard of care. One death was reported in each group, and neither were related to treatment. No cases of meningitis or thrombosis were reported in either group. The most common adverse events reported during the study in the pegcetacoplan and standard of care groups, respectively, were injection site reaction (30 per cent vs. 0 per cent), hypokalemia (13 per cent vs. 11 per cent), and fever (9 per cent vs. 0 per cent).

    "The PRINCE study results reinforce the efficacy and safety profile of pegcetacoplan in PNH," said Ravi Rao, Head of Research & Development and Chief Medical Officer at Sobi. "Our hope is to contribute to an improvement of care, and to make a difference in the lives of people with this rare blood disease."

    Detailed results from the PRINCE study will be presented at medical congresses.

    About the PRINCE study

    The PRINCE study (NCT04085601) is a 2:1 (pegcetacoplan: standard of care) randomized, multi-center, open-label, controlled phase 3 study in 53 treatment-naïve adults with paroxysmal nocturnal haemoglobinuria (PNH). The primary objective of this study was to establish the efficacy and safety of pegcetacoplan in patients who have not received treatment with any complement inhibitor within three months prior to screening. During the 26-week randomized, controlled period, patients received either 1080 mg of pegcetacoplan twice weekly or standard of care therapy, which did not include complement inhibitors. Patients in the standard of care group had the option to escape to the pegcetacoplan group if their haemoglobin decreased 2 g/dL or more from their baseline value.

    About pegcetacoplan

    Pegcetacoplan is an investigational therapy targeting C3, the central protein in the complement cascade. It acts proximally in the complement cascade controlling both C3bmediated extravascular haemolysis and terminal complementmediated intravascular haemolysis. Pegcetacoplan is being evaluated in several clinical studies across haematology, ophthalmology, nephrology, and neurology. In May 2021, pegcetacoplan was approved as EMPAVELITM in the US for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH). The marketing authorisation application for pegcetacoplan for paroxysmal nocturnal haemoglobinuria (PNH) is under review by the European Medicines Agency (EMA). Pegcetacoplan was also granted Fast Track designation by the FDA for the treatment of geographic atrophy and received orphan drug designation for the treatment of C3 glomerulopathy by the FDA and EMA. For additional information regarding pegcetacoplan clinical studies, visit apellis.com/our-science/clinical-trials.

    About Paroxysmal Nocturnal Haemoglobinuria (PNH)

    PNH is a rare, chronic, life-threatening blood disorder characterized by the destruction of oxygen-carrying red blood cells through extravascular and intravascular haemolysis. Persistently low haemoglobin can result in frequent transfusions and debilitating symptoms such as severe fatigue, haemoglobinuria and difficulty breathing (dyspnea).

    About Apellis

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within haematology, ophthalmology, nephrology, and neurology. For more information, please visit http://apellis.com.

    About Sobi™

    Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare diseases. Sobi is providing sustainable access to innovative therapies in the areas of haematology, immunology and specialty indications. Today, Sobi employs approximately 1,500 people across Europe, North America, Middle East and Asia. In 2020, Sobi's revenues amounted to SEK 15.3 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. You can find more information about Sobi at sobi.com.

    This information is information that Swedish Orphan Biovitrum AB (publ) is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact persons set out below, at 13:00 CEST on 25 May 2021.

    For more information, please contact

    Paula Treutiger, Head of Communication & Investor Relations

    + 46 733 666 599

    Maria Kruse, Corporate Communication & Investor Relations

    + 46 767 248 830

    Apellis

    Media:

    Lissa Pavluk

     

    617.977.6764

    Investors:

    Argot Partners

    212.600.1902

    This information was brought to you by Cision http://news.cision.com

    https://news.cision.com/swedish-orphan-biovitrum-ab/r/positive-top-line-results-from-the-phase-3-prince-study-of-pegcetacoplan-in-treatment-naive-patients,c3353197

    The following files are available for download:

    https://mb.cision.com/Main/14266/3353197/1422180.pdf

    Positive top-line results from the phase 3 PRINCE study of pegcetacoplan in treatment-naïve patients with PNH

    Cision View original content:http://www.prnewswire.com/news-releases/positive-top-line-results-from-the-phase-3-prince-study-of-pegcetacoplan-in-treatment-naive-patients-with-pnh-301298694.html

    SOURCE Swedish Orphan Biovitrum AB

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    • Mean hemoglobin levels in the EMPAVELI group increased from 9.4 g/dL to 12.1 g/dL compared to an increase from 8.7 g/dL to 9.4 g/dL on standard of care (p=0.0019)



    • The safety profile of EMPAVELI was consistent with previous studies

    WALTHAM, Mass. and STOCKHOLM, Sweden, May 25, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) and Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI…

    • EMPAVELI demonstrated statistical superiority on the co-primary endpoints of hemoglobin stabilization (p<0.0001) and reduction in lactate dehydrogenase (LDH) (p<0.0001) compared to standard of care, which did not include complement inhibitors, at Week 26



    • Mean hemoglobin levels in the EMPAVELI group increased from 9.4 g/dL to 12.1 g/dL compared to an increase from 8.7 g/dL to 9.4 g/dL on standard of care (p=0.0019)



    • 91% of patients on EMPAVELI were transfusion free compared to 22% on standard of care (p<0.0001)



    • The safety profile of EMPAVELI was consistent with previous studies

    WALTHAM, Mass. and STOCKHOLM, Sweden, May 25, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) and Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) today reported positive top-line results from the Phase 3 PRINCE study evaluating the efficacy and safety of EMPAVELI™ (pegcetacoplan) in adults with paroxysmal nocturnal hemoglobinuria (PNH) who are treatment naïve, meaning they had not received a complement inhibitor within three months before entering the study.

    EMPAVELI demonstrated statistical superiority on the co-primary endpoints of hemoglobin stabilization and reduction in lactate dehydrogenase (LDH) compared to standard of care, which did not include complement inhibitors, at Week 26.

    • 86% of EMPAVELI-treated patients achieved hemoglobin stabilization compared to 0% of patients on standard of care (p<0.0001). Hemoglobin stabilization was defined as an avoidance of a >1 g/dL decrease in hemoglobin levels in the absence of transfusions.
    • Mean LDH in the EMPAVELI group decreased by 90% from a baseline of 2151 U/L [9.5x upper limit of normal (ULN)] to 211 U/L, which is within the normal range, compared to a 14% reduction on standard of care from a baseline of 1946 U/L (8.6x ULN) to 1681 U/L (7.4x ULN) (p<0.0001).

    "The positive PRINCE data showed that EMPAVELI provided clinically meaningful improvements across multiple measures that are important for patients and build on our recent FDA approval of EMPAVELI in PNH," said Federico Grossi, M.D., Ph.D., chief medical officer, Apellis. "Combined with previous studies, these results emphasize the potential of EMPAVELI to provide disease control for all adults with PNH regardless of prior treatment."

    EMPAVELI also achieved statistical superiority on several secondary endpoints, including improvements in hemoglobin levels and transfusion avoidance, compared to standard of care, which did not include complement inhibitors.

    • Mean hemoglobin levels in the EMPAVELI group increased from 9.4 g/dL to 12.1 g/dL compared to an increase from a baseline of 8.7 g/dL to 9.4 g/dL on standard of care (p=0.0019).
    • 91% of patients on EMPAVELI were transfusion free compared to 22% on standard of care (p<0.0001).

    The safety profile of EMPAVELI was consistent with previous studies. At Week 26, 9% of patients in the EMPAVELI group experienced a serious adverse event (SAE) compared to 17% on standard of care. One death was reported in each group, and neither were related to treatment. No cases of meningitis or thrombosis were reported in either group. The most common adverse events reported during the study in the EMPAVELI and standard of care groups, respectively, were injection site reaction (30% vs. 0%),

    hypokalemia (13% vs.11%), and fever (9% vs. 0%).

    "The PRINCE study results reinforce the efficacy and safety profile of EMPAVELI in PNH," said Ravi Rao, head of research and development and chief medical officer at Sobi. "Our hope is to contribute to an improvement of care and to make a difference in the lives of people with this rare blood disease."

    Detailed results from the PRINCE study will be presented at medical congresses.

    About the PRINCE Study

    The PRINCE study (NCT04085601) is a 2:1 (EMPAVELI: standard of care) randomized, multi-center, open-label, controlled Phase 3 study in 53 treatment-naïve adults with paroxysmal nocturnal hemoglobinuria (PNH). The primary objective of this study was to establish the efficacy and safety of EMPAVELI™ (pegcetacoplan) in patients who have not received treatment with any complement inhibitor within three months prior to screening. During the 26-week randomized, controlled period, patients received either 1080 mg of EMPAVELI twice weekly or standard of care therapy, which did not include complement inhibitors. Patients in the standard of care group had the option to escape to the EMPAVELI group if their hemoglobin decreased 2 g/dL or more from their baseline value.

    The study was conducted in collaboration with SFJ Pharmaceuticals, who supported the development of EMPAVELI in PNH. SFJ is a global drug development company, which provides a unique and highly customized co-development partnering model for the world's top pharmaceutical and biotechnology companies.

    About EMPAVELI™ (pegcetacoplan)

    EMPAVELI™ (pegcetacoplan) is the first and only approved therapy targeting C3, the central protein in the complement cascade. EMPAVELI acts proximally in the complement cascade controlling both C3b-mediated extravascular hemolysis and terminal complement-mediated intravascular hemolysis. EMPAVELI is approved in the United States for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH).

    U.S. Important Safety Information for EMPAVELI

    BOXED WARNING: SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA

    • Meningococcal infections may occur in patients treated with EMPAVELI and may become rapidly life-threatening or fatal if not recognized and treated early. Use of EMPAVELI may predispose individuals to serious infections, especially those caused by encapsulated bacteria, such as Streptococcus pneumoniae, Neisseria meningitidis types A, C, W, Y, and B, and Haemophilus influenzae type B.
    • Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for vaccinations against encapsulated bacteria.
    • Vaccinate patients at least 2 weeks prior to administering the first dose of EMPAVELI unless the risks of delaying therapy with EMPAVELI outweigh the risk of developing a serious infection.
    • Vaccination reduces, but does not eliminate, the risk of serious infections. Monitor patients for early signs of serious infections and evaluate immediately if infection is suspected.
    • EMPAVELI is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS). Under the EMPAVELI REMS, prescribers must enroll in the program.

    CONTRAINDICATIONS

    • Hypersensitivity to pegcetacoplan or to any of the excipients
    • Not currently vaccinated against certain encapsulated bacteria, unless the risks of delaying EMPAVELI treatment outweigh the risks of developing a bacterial infection with an encapsulated organism
    • Unresolved serious infection caused by encapsulated bacteria including Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae

    WARNINGS AND PRECAUTIONS

    Serious Infections Caused by Encapsulated Bacteria

    The use of EMPAVELI may predispose individuals to serious, life-threatening, or fatal infections caused by encapsulated bacteria, including Streptococcus pneumoniae, Neisseria meningitidis types A, C, W, Y, and B, and Haemophilus influenzae type B (Hib). To reduce the risk of infection, all patients must be vaccinated against these bacteria according to the most current ACIP recommendations for patients with altered immunocompetence associated with complement deficiencies. Revaccinate patients in accordance with ACIP recommendations considering the duration of therapy with EMPAVELI.

    For patients without known history of vaccination, administer required vaccines at least 2 weeks prior to receiving the first dose of EMPAVELI. If immediate therapy with EMPAVELI is indicated, administer required vaccine as soon as possible and provide patients with 2 weeks of antibacterial drug prophylaxis.

    Closely monitor patients for early signs and symptoms of serious infection and evaluate patients immediately if an infection is suspected. Promptly treat known infections. Serious infection may become rapidly life-threatening or fatal if not recognized and treated early. Consider discontinuation of EMPAVELI in patients who are undergoing treatment for serious infections.

    EMPAVELI REMS

    Because of the risk of serious infections, EMPAVELI is available only through a restricted program under a REMS. Under the EMPAVELI REMS, prescribers must enroll in the program and must counsel patients about the risk of serious infection, provide the patients with the REMS educational materials, and ensure patients are vaccinated against encapsulated bacteria. Enrollment and additional information are available by telephone: 1-888-343-7073 or at www.empavelirems.com.

    Infusion-Related Reactions

    Systemic hypersensitivity reactions (e.g., facial swelling, rash, urticaria) have occurred in patients treated with EMPAVELI. One patient (less than 1% in clinical studies) experienced a serious allergic reaction which resolved after treatment with antihistamines. If a severe hypersensitivity reaction (including anaphylaxis) occurs, discontinue EMPAVELI infusion immediately, institute appropriate treatment, per standard of care, and monitor until signs and symptoms are resolved.

    Monitoring PNH Manifestations after Discontinuation of EMPAVELI

    After discontinuing treatment with EMPAVELI, closely monitor for signs and symptoms of hemolysis, identified by elevated LDH levels along with sudden decrease in PNH clone size or hemoglobin, or reappearance of symptoms such as fatigue, hemoglobinuria, abdominal pain, dyspnea, major adverse vascular events (including thrombosis), dysphagia, or erectile dysfunction. Monitor any patient who discontinues EMPAVELI for at least 8 weeks to detect hemolysis and other reactions. If hemolysis, including elevated LDH, occurs after discontinuation of EMPAVELI, consider restarting treatment with EMPAVELI.

    Interference with Laboratory Tests

    There may be interference between silica reagents in coagulation panels and EMPAVELI that results in artificially prolonged activated partial thromboplastin time (aPTT); therefore, avoid the use of silica reagents in coagulation panels.

    ADVERSE REACTIONS

    The most common adverse reactions (incidence ≥10% of patients) with EMPAVELI vs. eculizumab were injection-site reactions (39% v. 5%), infections (29% v. 26%), diarrhea (22% v. 3%), abdominal pain (20% v. 10%), respiratory tract infection (15% v. 13%), viral infection (12% v. 8%), and fatigue (12% v. 23%).

    USE IN SPECIFIC POPULATIONS

    Females of Reproductive Potential

    EMPAVELI may cause embryo-fetal harm when administered to pregnant women. Pregnancy testing is recommended for females of reproductive potential prior to treatment with EMPAVELI. Advise female patients of reproductive potential to use effective contraception during treatment with EMPAVELI and for 40 days after the last dose.

    Please see full Prescribing Information, including Boxed WARNING regarding serious infections caused by encapsulated bacteria, and Medication Guide.

    About Paroxysmal Nocturnal Hemoglobinuria (PNH)

    PNH is a rare, chronic, life-threatening blood disorder characterized by the destruction of oxygen-carrying red blood cells through extravascular and intravascular hemolysis. Persistently low hemoglobin can result in frequent transfusions and debilitating symptoms such as severe fatigue, hemoglobinuria and difficulty breathing (dyspnea).

    About Apellis 

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit http://apellis.com.

    About Sobi™

    Sobi is a specialized international biopharmaceutical company transforming the lives of people with rare diseases. Sobi is providing sustainable access to innovative therapies in the areas of haematology, immunology and specialty indications. Today, Sobi employs approximately 1,500 people across Europe, North America, Middle East, and Asia. In 2020, Sobi's revenues amounted to SEK 15.3 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. You can find more information about Sobi at www.sobi.com.

    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 in respect of the expected closing of the exchanges. 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 the conditions for the closing of the exchanges will be satisfied and other factors discussed in the "Risk Factors" section of Apellis' Quarterly Report on Form 10-Q with the Securities and Exchange Commission on April 28, 2021 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.

    Contacts:



    Apellis

    Media:

    Lissa Pavluk 

     

    617.977.6764

    Investors: 

    Argot Partners



    212.600.1902

    Sobi

    Paula Treutiger, Head of Communication & Investor Relations

    + 46 733 666 599

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    + 46 767 248 830

     



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    • EMPAVELI, the first targeted C3 therapy, is approved for use in adults with PNH who are:
      • Treatment naïve
      • Switching from C5 inhibitor Soliris® (eculizumab)
      • Switching from C5 inhibitor Ultomiris® (ravulizumab)
    • EMPAVELI was superior to Soliris for the change from baseline in hemoglobin level at Week 16 in the Phase 3 PEGASUS study

    • 85% of patients treated with EMPAVELI were transfusion free compared to 15% on Soliris at Week 16; EMPAVELI met non-inferiority compared to Soliris on transfusion avoidance

    • Apellis to host investor conference call on Monday, May 17 at 8:00 a.m. ET

    WALTHAM, Mass., May 14, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today…

    • EMPAVELI, the first targeted C3 therapy, is approved for use in adults with PNH who are:

      • Treatment naïve
      • Switching from C5 inhibitor Soliris® (eculizumab)
      • Switching from C5 inhibitor Ultomiris® (ravulizumab)
    • EMPAVELI was superior to Soliris for the change from baseline in hemoglobin level at Week 16 in the Phase 3 PEGASUS study



    • 85% of patients treated with EMPAVELI were transfusion free compared to 15% on Soliris at Week 16; EMPAVELI met non-inferiority compared to Soliris on transfusion avoidance



    • Apellis to host investor conference call on Monday, May 17 at 8:00 a.m. ET

    WALTHAM, Mass., May 14, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the U.S. Food and Drug Administration (FDA) has approved EMPAVELI™ (pegcetacoplan), the first and only targeted C3 therapy for treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH). EMPAVELI is approved for use in adults with PNH who are treatment naïve as well as patients switching from the C5 inhibitors Soliris® (eculizumab) and Ultomiris® (ravulizumab).

    "EMPAVELI has the potential to improve the lives of patients with PNH by increasing hemoglobin and reducing blood transfusion requirements," said Olga Frankfurt, M.D., PEGASUS study investigator and associate professor in the department of medicine, division of hematology and oncology, Robert H. Lurie Comprehensive Cancer Center of Northwestern University. "Through my work as an investigator on the PEGASUS study, I was excited to see EMPAVELI providing broad control of PNH."

    "As the first, FDA-approved targeted C3 therapy, EMPAVELI has the potential to redefine treatment for adults with PNH, including patients switching from any C5 inhibitor and treatment-naïve patients. Thank you to the clinical trial participants, PNH community, investigators, healthcare professionals, SFJ Pharmaceuticals, and more who helped contribute to this significant milestone," said Cedric Francois, M.D., Ph.D., co-founder and chief executive officer, Apellis. "This approval represents a major scientific advancement as EMPAVELI ushers in the first new class of complement medicine in almost 15 years. We look forward to exploring the full potential of targeting C3 and continue to advance registrational programs of this therapy across multiple complement-driven diseases with high unmet need."

    The approval of EMPAVELI is based on results from the head-to-head Phase 3 PEGASUS study, which were recently published in the New England Journal of Medicine. In the PEGASUS study, EMPAVELI met the primary endpoint, demonstrating superiority to Soliris for the change from baseline in hemoglobin level at Week 16 with an adjusted mean increase of 3.84 g/dL of hemoglobin (p<0.0001). Additionally, EMPAVELI met non-inferiority compared to Soliris on the endpoint of transfusion avoidance. Eighty five percent of EMPAVELI-treated patients were transfusion free over 16 weeks versus 15% of Soliris-treated patients.

    "We are pleased to hear of the FDA's decision to approve EMPAVELI, which is an important milestone for patients," said Janice Frey-Angel, chief executive officer and executive director, Aplastic Anemia and MDS International Foundation (AAMDSIF). "Many PNH patients are seeking choices in their treatment, so the approval brings new promise for the PNH community."

    The prescribing information for EMPAVELI contains a boxed warning. EMPAVELI may increase the risk of meningococcal and other serious infections caused by encapsulated bacteria that may become rapidly life threatening or fatal if not recognized and treated early. A Risk Evaluation and Mitigation Strategy (REMS) has been approved by the FDA for EMPAVELI. Prescribers must counsel patients about the risk of serious infection, provide patients with the REMS educational materials, and ensure patients are vaccinated against encapsulated bacteria.

    The most common serious adverse reaction in patients treated with EMPAVELI was infections (5%). The most common adverse reactions (≥10%) with EMPAVELI were injection site reactions (39%), infections (29%), diarrhea (22%), abdominal pain (20%), respiratory tract infection (15%), viral infection (12%), and fatigue (12%). No cases of meningitis and no deaths were reported in patients treated with EMPAVELI.

    PNH is a rare, chronic, life-threatening blood disorder caused by an acquired mutation, which leads to uncontrolled complement activation and the destruction of red blood cells through intravascular and extravascular hemolysis. According to a retrospective and a cross-sectional study of patients treated with C5 inhibitors, at least 72% had persistently low hemoglobin1,2 and at least 36% required one or more transfusions a year.1

    Apellis is committed to helping patients with treatment access and support. ApellisAssist™ is a program designed to provide comprehensive product support to EMPAVELI patients throughout their treatment journey. This program provides services and product resources including insurance support, education, training, as well as financial assistance for eligible patients. Patients and healthcare providers can contact 1-866-692-7527 for more information.

    A Marketing Authorization Application for pegcetacoplan for the treatment of PNH is under review by the European Medicines Agency with the potential for a European Commission decision in the second half of 2021.

    Conference Call and Webcast

    Apellis will host a conference call and webcast to discuss the U.S. Food and Drug Administration (FDA) approval of EMPAVELI™ (pegcetacoplan) on Monday, May 17 at 8:00 a.m. ET. To access the live call by phone, please dial 866-774-0323 (domestic) or 602-563-8683 (international); the conference ID is 3239157. A live audio webcast of the event and accompanying slides may also be accessed through the "Events and Presentations" page of the "Investors and Media" section of the company's website at http://investors.apellis.com/events-and-presentations. A replay of the webcast will be available for 30 days following the event.

    About the PEGASUS Study

    The PEGASUS study (APL2-302; NCT03500549) is a multi-center, randomized, head-to-head Phase 3 study in 80 adults with paroxysmal nocturnal hemoglobinuria (PNH). The primary objective of this study was to establish the efficacy and safety of EMPAVELI™ (pegcetacoplan) compared to Soliris® (eculizumab). Participants must have been on Soliris (stable for at least three months) with a hemoglobin level of <10.5 g/dL at the screening visit. During the four-week run-in, patients were dosed with 1080 mg of EMPAVELI twice weekly (n=41) in addition to their current dose of Soliris. During the 16-week randomized, controlled period, patients were randomized to receive either 1080 mg of EMPAVELI twice weekly or their current dose of Soliris (n=39). All participants completing the randomized controlled period (n=77) opted to enter the open-label EMPAVELI treatment period.

    The study was conducted in collaboration with SFJ Pharmaceuticals, who supported the development of EMPAVELI in PNH. SFJ is a global drug development company, which provides a unique and highly customized co-development partnering model for the world's top pharmaceutical and biotechnology companies.

    About EMPAVELI™ (pegcetacoplan)

    EMPAVELI™ (pegcetacoplan) is the first and only approved therapy targeting C3, the central protein in the complement cascade. EMPAVELI acts proximally in the complement cascade controlling both C3b-mediated extravascular hemolysis and terminal complement-mediated intravascular hemolysis. EMPAVELI is approved in the United States for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH).

    U.S. Important Safety Information for EMPAVELI

    BOXED WARNING: SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA

    • Meningococcal infections may occur in patients treated with EMPAVELI and may become rapidly life-threatening or fatal if not recognized and treated early. Use of EMPAVELI may predispose individuals to serious infections, especially those caused by encapsulated bacteria, such as Streptococcus pneumoniae, Neisseria meningitidis types A, C, W, Y, and B, and Haemophilus influenzae type B.
    • Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for vaccinations against encapsulated bacteria.
    • Vaccinate patients at least 2 weeks prior to administering the first dose of EMPAVELI unless the risks of delaying therapy with EMPAVELI outweigh the risk of developing a serious infection.
    • Vaccination reduces, but does not eliminate, the risk of serious infections. Monitor patients for early signs of serious infections and evaluate immediately if infection is suspected.
    • EMPAVELI is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS). Under the EMPAVELI REMS, prescribers must enroll in the program.

    CONTRAINDICATIONS

    • Hypersensitivity to pegcetacoplan or to any of the excipients
    • Not currently vaccinated against certain encapsulated bacteria, unless the risks of delaying EMPAVELI treatment outweigh the risks of developing a bacterial infection with an encapsulated organism
    • Unresolved serious infection caused by encapsulated bacteria including Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae

    WARNINGS AND PRECAUTIONS

    Serious Infections Caused by Encapsulated Bacteria

    The use of EMPAVELI may predispose individuals to serious, life-threatening, or fatal infections caused by encapsulated bacteria, including Streptococcus pneumoniae, Neisseria meningitidis types A, C, W, Y, and B, and Haemophilus influenzae type B (Hib). To reduce the risk of infection, all patients must be vaccinated against these bacteria according to the most current ACIP recommendations for patients with altered immunocompetence associated with complement deficiencies. Revaccinate patients in accordance with ACIP recommendations considering the duration of therapy with EMPAVELI.

    For patients without known history of vaccination, administer required vaccines at least 2 weeks prior to receiving the first dose of EMPAVELI. If immediate therapy with EMPAVELI is indicated, administer required vaccine as soon as possible and provide patients with 2 weeks of antibacterial drug prophylaxis.

    Closely monitor patients for early signs and symptoms of serious infection and evaluate patients immediately if an infection is suspected. Promptly treat known infections. Serious infection may become rapidly life-threatening or fatal if not recognized and treated early. Consider discontinuation of EMPAVELI in patients who are undergoing treatment for serious infections.

    EMPAVELI REMS

    Because of the risk of serious infections, EMPAVELI is available only through a restricted program under a REMS. Under the EMPAVELI REMS, prescribers must enroll in the program and must counsel patients about the risk of serious infection, provide the patients with the REMS educational materials, and ensure patients are vaccinated against encapsulated bacteria. Enrollment and additional information are available by telephone: 1-888-343-7073 or at www.empavelirems.com.

    Infusion-Related Reactions

    Systemic hypersensitivity reactions (e.g., facial swelling, rash, urticaria) have occurred in patients treated with EMPAVELI. One patient (less than 1% in clinical studies) experienced a serious allergic reaction which resolved after treatment with antihistamines. If a severe hypersensitivity reaction (including anaphylaxis) occurs, discontinue EMPAVELI infusion immediately, institute appropriate treatment, per standard of care, and monitor until signs and symptoms are resolved.

    Monitoring PNH Manifestations after Discontinuation of EMPAVELI

    After discontinuing treatment with EMPAVELI, closely monitor for signs and symptoms of hemolysis, identified by elevated LDH levels along with sudden decrease in PNH clone size or hemoglobin, or reappearance of symptoms such as fatigue, hemoglobinuria, abdominal pain, dyspnea, major adverse vascular events (including thrombosis), dysphagia, or erectile dysfunction. Monitor any patient who discontinues EMPAVELI for at least 8 weeks to detect hemolysis and other reactions. If hemolysis, including elevated LDH, occurs after discontinuation of EMPAVELI, consider restarting treatment with EMPAVELI.

    Interference with Laboratory Tests

    There may be interference between silica reagents in coagulation panels and EMPAVELI that results in artificially prolonged activated partial thromboplastin time (aPTT); therefore, avoid the use of silica reagents in coagulation panels.

    ADVERSE REACTIONS

    The most common adverse reactions (incidence ≥10% of patients) with EMPAVELI vs. eculizumab were injection-site reactions (39% v. 5%), infections (29% v. 26%), diarrhea (22% v. 3%), abdominal pain (20% v. 10%), respiratory tract infection (15% v. 13%), viral infection (12% v. 8%), and fatigue (12% v. 23%).

    USE IN SPECIFIC POPULATIONS

    Females of Reproductive Potential

    EMPAVELI may cause embryo-fetal harm when administered to pregnant women. Pregnancy testing is recommended for females of reproductive potential prior to treatment with EMPAVELI. Advise female patients of reproductive potential to use effective contraception during treatment with EMPAVELI and for 40 days after the last dose.

    Please see full Prescribing Information, including Boxed WARNING regarding serious infections caused by encapsulated bacteria, and Medication Guide.

    About the Apellis and Sobi Collaboration

    Apellis and Sobi entered a collaboration to develop and commercialize systemic pegcetacoplan in October 2020. The companies have global co-development rights for systemic pegcetacoplan. Apellis has exclusive U.S. commercialization rights for systemic pegcetacoplan and retains worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy (GA). Sobi has exclusive ex-U.S. commercialization rights for systemic pegcetacoplan.

    About Apellis 

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit http://apellis.com.

    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 in respect of the expected closing of the exchanges. 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 the conditions for the closing of the exchanges will be satisfied and other factors discussed in the "Risk Factors" section of Apellis' Quarterly Report on Form 10-Q with the Securities and Exchange Commission on April 28, 2021 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.

    Contacts:



    Media:

    Lissa Pavluk 



    617.977.6764

    Investors: 

    Argot Partners 



    212.600.1902



    1.  McKinley C. Extravascular Hemolysis Due to C3-Loading in Patients with PNH Treated with Eculizumab: Defining the Clinical Syndrome. Blood. 2017;130:3471.

    2. Dingli ASH 2020 Abstract/ p.1/ Methods/ ln.1-2; p.2/ Results/ln.7-9; ln.14-15.



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    • Seven accepted abstracts, including an oral presentation, reinforce the potential of pegcetacoplan, an investigational, targeted C3 therapy, to redefine treatment for patients with paroxysmal nocturnal hemoglobinuria (PNH)
    • PDUFA action date of May 14, 2021 set by the U.S. Food and Drug Administration (FDA)

    WALTHAM, Mass., May 12, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that seven abstracts were accepted for presentation and publication at the European Hematology Association (EHA) Virtual Congress to be held June 9-17, 2021. Data support the long-term durability and consistent safety profile of pegcetacoplan, an investigational…

    • Seven accepted abstracts, including an oral presentation, reinforce the potential of pegcetacoplan, an investigational, targeted C3 therapy, to redefine treatment for patients with paroxysmal nocturnal hemoglobinuria (PNH)
    • PDUFA action date of May 14, 2021 set by the U.S. Food and Drug Administration (FDA)

    WALTHAM, Mass., May 12, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that seven abstracts were accepted for presentation and publication at the European Hematology Association (EHA) Virtual Congress to be held June 9-17, 2021. Data support the long-term durability and consistent safety profile of pegcetacoplan, an investigational, targeted C3 therapy, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).

    "The breadth of data presented at EHA add to a growing body of evidence that reinforces the potential of pegcetacoplan to redefine treatment for the PNH community," said Federico Grossi, M.D., Ph.D., chief medical officer of Apellis.

    Marketing applications for pegcetacoplan for the treatment of PNH are under review by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). The FDA granted the application Priority Review designation and set a target action date of May 14, 2021. The application has the potential for a European Commission decision in the second half of 2021.

    Pegcetacoplan Oral Presentation:

    • Forty-eight week efficacy and safety of pegcetacoplan in adult patients with paroxysmal nocturnal hemoglobinuria and suboptimal response to prior eculizumab treatment – S174 – Friday, June 11 at 9:00 CEST

    Pegcetacoplan E-Poster Presentation:

    • Effect of pegcetacoplan on quality of life in patients with paroxysmal nocturnal hemoglobinuria: week 48 of PEGASUS phase 3 trial comparing pegcetacoplan to eculizumab – EP595 – Friday, June 11 at 9:00 CEST
    • Paroxysmal nocturnal hemoglobinuria's humanistic and economic burden in patients receiving C5 inhibitors in Europe – EP1191 – Friday, June 11 at 9:00 CEST

    Published Abstracts:

    • Categorized hematologic response to pegcetacoplan versus eculizumab in patients with paroxysmal nocturnal hemoglobinuria: post hoc analysis of PEGASUS phase 3 randomized trial data
    • Comparative effectiveness of pegcetacoplan versus ravulizumab in patients with paroxysmal nocturnal hemoglobinuria: a matching-adjusted indirect comparison using 26 week PEGASUS phase 3 trial data
    • Injection-site reactions at week 48 in the randomized phase 3 PEGASUS trial of pegcetacoplan compared with eculizumab for individuals with paroxysmal nocturnal hemoglobinuria
    • Long-term effects in subgroups of patients with paroxysmal nocturnal hemoglobinuria treated with pegcetacoplan versus eculizumab: 48-week analysis of PEGASUS phase 3 trial

    About the PEGASUS Study

    The PEGASUS study (APL2-302; NCT03500549) was a multi-center, randomized, active-comparator controlled Phase 3 study in 80 adults with paroxysmal nocturnal hemoglobinuria (PNH). The primary objective of this study was to establish the efficacy and safety of pegcetacoplan compared to eculizumab. Participants must have been on eculizumab (stable for at least three months) with a hemoglobin level of <10.5 g/dL at the screening visit. During the four-week run-in, patients were dosed with 1080 mg of pegcetacoplan twice weekly (n=41) in addition to their current dose of eculizumab. During the 16-week randomized, controlled period, patients were randomized to receive either 1080 mg of pegcetacoplan twice weekly or their current dose of eculizumab (n=39). All participants completing the randomized controlled period entered the open-label pegcetacoplan treatment period.

    About Pegcetacoplan (APL-2) 

    Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Pegcetacoplan is being evaluated in several clinical studies across hematology, ophthalmology, nephrology, and neurology. Marketing applications for pegcetacoplan for paroxysmal nocturnal hemoglobinuria (PNH) are under review by the U.S. Food and Drug Administration (FDA), which has granted the application Priority Review designation, and the European Medicines Agency (EMA). Pegcetacoplan was granted Fast Track designation by the FDA for the treatment of geographic atrophy and received orphan drug designation for the treatment of C3G by the FDA and EMA. For additional information regarding pegcetacoplan clinical studies, visit https://apellis.com/our-science/clinical-trials.

    About Paroxysmal Nocturnal Hemoglobinuria (PNH) 

    Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic, life-threatening blood disorder caused by an acquired mutation which leads to uncontrolled complement activation and the destruction of red blood cells through hemolysis. A retrospective analysis shows that, even on eculizumab, approximately 72% of people with PNH have anemia, a key indicator of ongoing hemolysis.1 The analysis also finds that 36% of patients require one or more transfusions a year and 16% require three or more.1

    About the Apellis and Sobi Collaboration

    Apellis and Sobi entered a collaboration to develop and commercialize systemic pegcetacoplan in October 2020. The companies have global co-development rights for systemic pegcetacoplan. Apellis has exclusive U.S. commercialization rights for systemic pegcetacoplan and retains worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy (GA). Sobi has exclusive ex-U.S. commercialization rights for systemic pegcetacoplan.

    About Apellis 

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit http://apellis.com.

    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 in respect of the expected closing of the exchanges. 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 the conditions for the closing of the exchanges will be satisfied and other factors discussed in the "Risk Factors" section of Apellis' Quarterly Report on Form 10-Q with the Securities and Exchange Commission on April 28, 2021 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: 

    Lissa Pavluk 

      

    617.420.4839

    Investors: 

    Argot Partners



    212.600.1902



    1.  McKinley C. Extravascular Hemolysis Due to C3-Loading in Patients with PNH Treated with Eculizumab: Defining the Clinical Syndrome. Blood. 2017;130:3471.

     



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  5. WALTHAM, Mass., May 07, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company approved the grant of equity awards to two new employees with a grant date of May 3, 2021, as equity inducement awards outside of the company's 2017 Stock Incentive Plan (but under the terms of the 2020 Inducement Stock Incentive Plan) and material to the employees' acceptance of employment with the company. The equity awards were approved in accordance with Nasdaq Listing Rule 5635(c)(4).

    The employees received options to purchase 13,200 shares of Apellis common stock and 7,100 restricted stock units (RSUs). The options have an exercise price…

    WALTHAM, Mass., May 07, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company approved the grant of equity awards to two new employees with a grant date of May 3, 2021, as equity inducement awards outside of the company's 2017 Stock Incentive Plan (but under the terms of the 2020 Inducement Stock Incentive Plan) and material to the employees' acceptance of employment with the company. The equity awards were approved in accordance with Nasdaq Listing Rule 5635(c)(4).

    The employees received options to purchase 13,200 shares of Apellis common stock and 7,100 restricted stock units (RSUs). The options have an exercise price of $50.20, which is equal to the closing price of Apellis common stock on May 3, 2021, the grant date of the options. One-fourth of the shares underlying the employee options will vest on the one year anniversary of the grant date and thereafter 1/48th of the shares underlying the employee options will vest monthly, such that the shares underlying the options granted to the employees will be fully vested on the fourth anniversary of the grant date, subject to the employees' continued employment with Apellis on such vesting dates. Each RSU will vest as to 25% of the shares underlying the RSU award on the first anniversary of the grant date and as to an additional 25% of the shares underlying the RSU award annually thereafter, subject to each such employee's continued employment on each vesting date.

    About Apellis

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit www.apellis.com.

    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 the company's clinical trials will be fully enrolled and completed when anticipated; 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 will be indicative of results that will be generated in future clinical trials; whether pegcetacoplan will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the company's clinical trials will warrant regulatory submissions and whether pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, C3G, IC-MPGN, ALS or any other indication when expected or at all; 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 2, 2020 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.

    Investor Contact:

    Argot Partners



    +1 212.600.1902



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    • Marketing application for pegcetacoplan for paroxysmal nocturnal hemoglobinuria (PNH) is under review by the U.S. Food and Drug Administration (FDA) with a PDUFA target action date of May 14, 2021
    • Top-line results from the Phase 3 PRINCE study in treatment-naïve PNH patients expected in the second quarter of 2021
    • Top-line results from Phase 3 geographic atrophy (GA) studies expected in the third quarter of 2021
    • Three new product candidates advancing into clinical development by the end of 2022
    • Cash and investments of $723.7 million as of March 31, 2021 support cash runway into the second half of 2022
    • Conference call scheduled today at 4:30 p.m. ET

    WALTHAM, Mass., April 28, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global…

    • Marketing application for pegcetacoplan for paroxysmal nocturnal hemoglobinuria (PNH) is under review by the U.S. Food and Drug Administration (FDA) with a PDUFA target action date of May 14, 2021
    • Top-line results from the Phase 3 PRINCE study in treatment-naïve PNH patients expected in the second quarter of 2021
    • Top-line results from Phase 3 geographic atrophy (GA) studies expected in the third quarter of 2021
    • Three new product candidates advancing into clinical development by the end of 2022
    • Cash and investments of $723.7 million as of March 31, 2021 support cash runway into the second half of 2022
    • Conference call scheduled today at 4:30 p.m. ET

    WALTHAM, Mass., April 28, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced its first quarter 2021 financial results and business highlights.

    "With a potential U.S. approval for pegcetacoplan just a couple of weeks away, we are at the beginning of a transformational year for Apellis. Our commercial team is prepared to successfully execute our first product launch and meet the needs of PNH patients," said Cedric Francois, M.D., Ph.D., co-founder and chief executive officer of Apellis. "At the same time, we are focused on advancing registrational programs of pegcetacoplan across several debilitating rare diseases and delivering on our goal to become number one in the retina. Geographic atrophy is the most significant remaining unmet need in the retina, and we are excited to see top-line results from our Phase 3 studies of pegcetacoplan in GA in the third quarter of this year.

    "For more than a decade, our team has built the foundation for Apellis' leadership in complement, and we look forward to seeing the results of those efforts come together this year for patients living with serious, complement-driven diseases," Dr. Francois continued.

    First Quarter 2021 Business Highlights and Upcoming Milestones:

    Systemic Pegcetacoplan in Rare Disease

    • In March 2021, Apellis and Sobi announced that the New England Journal of Medicine published results from the Phase 3 PEGASUS study. The data at 16 weeks showed that pegcetacoplan, an investigational targeted C3 therapy, demonstrated statistically superior increases in mean hemoglobin levels compared with the C5 inhibitor Soliris® (eculizumab) and provided substantial improvements in other key markers of disease in adults with PNH who had persistent anemia following treatment with Soliris.
    • Marketing applications for pegcetacoplan for the treatment of PNH are under review by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). The New Drug Application was granted Priority Review by the FDA with a Prescription Drug User Fee Act (PDUFA) target action date of May 14, 2021. The Marketing Authorization Application has the potential for a European Commission decision in the second half of 2021.
    • In the second quarter of 2021, Apellis and Sobi expect to report top-line results from the Phase 3 PRINCE study in PNH patients who are treatment naïve.
    • In the second half of 2021, Apellis expects to initiate a Phase 3 study in immune complex membranoproliferative glomerulonephritis (IC-MPGN) and C3 glomerulopathy (C3G), and Sobi plans to initiate a Phase 3 study in cold agglutinin disease (CAD) and a potentially registrational Phase 2 study in hematopoietic stem cell transplantation-associated thrombotic microangiopathy (HSCT-TMA).

    Intravitreal Pegcetacoplan in Ophthalmology

    • In April 2021, Apellis announced 24-month data from the Phase 1b study of intravitreal pegcetacoplan in patients with advanced geographic atrophy (GA) and low vision. Data from a post hoc analysis demonstrated a 46% decrease in the growth rate of GA lesions in the treated eye compared to the untreated eye in eight patients with bilateral GA (disease in both eyes) at 24 months (p=0.007).
    • In March 2021, Apellis announced that two leading journals, Ophthalmology and the American Journal of Ophthalmology, published post hoc analyses from the Phase 2 FILLY study of intravitreal pegcetacoplan for GA secondary to age-related macular degeneration. The published data underscore the potential of pegcetacoplan for GA.
    • The company expects to announce top-line results from the Phase 3 DERBY and OAKS studies in the third quarter of 2021.

    APL-9 in COVID-19

    • In March 2021, Apellis announced that the company will not pursue additional development of APL-9, an investigational targeted C3 therapy designed for acute interventions, for the treatment of severe COVID-19. The decision followed an interim review of mortality data from the Phase 1/2 study by an independent data monitoring committee (DMC), which found no meaningful reduction in the overall mortality rate in patients treated with APL-9 in combination with standard of care therapy compared to standard of care alone. No safety signals were observed by the DMC.

    Pipeline Expansion

    • Apellis plans to advance three new product candidates into clinical development by the end of 2022.

    First Quarter 2021 Financial Results:

    As of March 31, 2021, Apellis had $723.7 million in cash, cash equivalents, and short-term marketable securities, compared to $646.7 million in cash, cash equivalents, and short-term marketable securities as of March 31, 2020. This increase primarily reflects the addition of cash from the company's convertible offering for gross proceeds of $328.9 million in May 2020, the receipt of $250.0 million in the upfront proceeds for the Sobi transaction in October 2020, and an additional $25.0 million receipt from Sobi in January 2021 less cash used in operations.

    Apellis reported a net loss of $183.7 million for the first quarter of 2021, compared to a net loss of $168.8 million for the same period in 2020.

    Research and development expenses were $84.0 million in the first quarter of 2021, compared to $69.3 million for the same period in 2020. The increase in R&D expense for first quarter 2021 was primarily attributable to an increase in clinical trial costs associated with the ongoing Phase 3 trials and the preparation and commencement of our clinical trials in other indications, personnel-related costs primarily due to the hiring of additional personnel, and increased quality and medical affairs expenses. We expect our research and development expenses to continue to increase as the number of patients in our trials increases and the number of ongoing trials increases.

    General and administrative expenses were $40.6 million in the first quarter of 2021, compared to $29.5 million for the same period in 2020. The increase in general and administrative expenses for the first quarter 2021 was primarily attributable to an increase in employee-related costs, professional and consulting fees, general commercial preparation activities, director stock compensation expense, and insurance.

    Conference Call and Webcast

    Apellis will host a conference call and webcast to discuss its first quarter 2021 financial results and business highlights today, April 28, 2021, at 4:30 p.m. ET. To access the conference call, please dial (866) 774-0323 (local) or (602) 563-8683 (international) at least 10 minutes prior to the start time and refer to conference ID 7883183. A live audio webcast of the event and accompanying slides may also be accessed through the "Events and Presentations" page of the "Investors and Media" section of the company's website at http://investors.apellis.com/events-and-presentations. A replay of the webcast will be available for 30 days following the event.

    About Pegcetacoplan

    Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Pegcetacoplan is being evaluated in several clinical studies across hematology, ophthalmology, nephrology, and neurology. Marketing applications for pegcetacoplan for paroxysmal nocturnal hemoglobinuria (PNH) are under review by the U.S. Food and Drug Administration (FDA), which has granted the application Priority Review designation, and the European Medicines Agency (EMA). Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of geographic atrophy and received orphan drug designation for the treatment of C3 glomerulopathy by the FDA and EMA. For additional information regarding pegcetacoplan clinical trials, visit https://apellis.com/our-science/clinical-trials.

    About APL-9

    APL-9 is an investigational, targeted C3 therapy designed to control the complement cascade centrally and may have the potential to treat a range of diseases caused by excessive activation of complement. APL-9 leverages the same mechanism of action as Apellis' lead compound, pegcetacoplan, but has a lower molecular weight and shorter half-life. APL-9 is designed to be intravenously administered for acute use.

    About Apellis

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit www.apellis.com.

    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 in respect of the expected closing of the exchanges. 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 the conditions for the closing of the exchanges will be satisfied and other factors discussed in the "Risk Factors" section of Apellis' Quarterly Report on Form 10-Q with the Securities and Exchange Commission on April 28, 2021 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.

    Investor Contact:

    Argot Partners



    +1 212.600.1902

    Media Contact:

    Tracy Vineis



    617.420.4839

     
    APELLIS PHARMACEUTICALS, INC.

    CONDENSED CONSOLIDATED BALANCE SHEETS

    (Amounts in thousands, except per share amounts)

      March 31, December 31,
      2021 2020
    Assets (Unaudited)  
    Current assets:    
    Cash and cash equivalents $265,435  $565,779 
    Marketable securities  458,237   311,869 
    Prepaid assets  17,203   11,400 
    Restricted cash  1,552   1,266 
    Other current assets  31,198   26,878 
    Total current assets  773,625   917,192 
    Non-current assets:    
    Right-of-use assets  22,518   17,719 
    Property and equipment, net  7,077   6,803 
    Other assets  6,909   18,855 
    Total assets $810,129  $960,569 
    Liabilities and Stockholders' Equity    
    Current liabilities:    
    Accounts payable $4,158  $8,477 
    Accrued expenses  69,398   111,935 
    Current portion of development derivative liability  6,212   4,230 
    Current portion of right-of-use liabilities  3,902   3,685 
    Total current liabilities  83,670   128,327 
    Long-term liabilities:    
    Convertible senior notes  386,152   358,830 
    Development derivative liability  268,740   253,638 
    Right-of-use liabilities  19,909   15,217 
    Total liabilities  758,471   756,012 
    Commitments and contingencies (note 13)  -   - 
    Stockholders' equity:    
    Preferred stock, $0.0001 par value; 10,000 shares authorized, and zero shares issued and outstanding at March 31, 2021 and December 31, 2020  -   - 
    Common stock, $0.0001 par value; 200,000 shares authorized at March 31, 2021 and December 31, 2020; 80,438 shares issued and outstanding at March 31, 2021, and 76,130 shares issued and outstanding at December 31, 2020  8   8 
    Additional paid-in capital  1,147,263   1,131,013 
    Accumulated other comprehensive loss  (1,620)  (117)
    Accumulated deficit  (1,093,993)  (926,347)
    Total stockholders' equity  51,658   204,557 
    Total liabilities and stockholders' equity $810,129  $960,569 
         



     
    APELLIS PHARMACEUTICALS, INC.
    CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
    (Amounts in thousands, except per share amounts)
        
     For the three months ended March 31,
     2021 2020
        
     (Unaudited)

    Operating expenses:   
    Research and development 84,012   69,282 
    General and administrative 40,579   29,504 
    Operating loss (124,591)  (98,786)
    Loss on extinguishment of debt (39,487)   
    Loss from remeasurement of development derivative liability (17,084)  (68,406)
    Interest expense 134   2,275 
    Interest income (4,175)  (3,919)
    Other income, net 1,544   14 
    Net loss (183,659)  (168,822)
    Other comprehensive loss:   
    Unrealized gain on marketable securities 79   1,394 
    Foreign currency loss (1,582)  (230)
    Total other comprehensive loss (1,503)  1,164 
    Comprehensive loss, net of tax$(185,162) $(167,658)
    Net loss per common share, basic and diluted$(2.32) $(2.29)
    Weighted-average number of common shares used in net loss per common share, basic and diluted 79,219   73,720 
        


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  6. WALTHAM, Mass., April 21, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company will host a conference call and webcast to discuss its first quarter 2021 financial results on Wednesday, April 28, 2021 at 4:30 p.m. ET.

    The event will be available live by dialing (866) 774-0323 (domestic) or (602) 563-8683 (international) and entering the conference ID # 7883183 or via webcast from the "Events and Presentations" page of the "Investors and Media" section of the company's website at https://investors.apellis.com/events-and-presentations. A replay of the webcast will be available for 90 days following the event.

    About Apellis

    WALTHAM, Mass., April 21, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company will host a conference call and webcast to discuss its first quarter 2021 financial results on Wednesday, April 28, 2021 at 4:30 p.m. ET.

    The event will be available live by dialing (866) 774-0323 (domestic) or (602) 563-8683 (international) and entering the conference ID # 7883183 or via webcast from the "Events and Presentations" page of the "Investors and Media" section of the company's website at https://investors.apellis.com/events-and-presentations. A replay of the webcast will be available for 90 days following the event.

    About Apellis

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit www.apellis.com.

    Investor Contact:

    Argot Partners



    +1 212.600.1902



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    • 10 accepted abstracts include five oral presentations from pioneering artificial intelligence (AI) collaboration

    • Presentations support the potential of intravitreal pegcetacoplan, an investigational targeted C3 therapy, as the first treatment for geographic atrophy (GA)

    WALTHAM, Mass., April 16, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that 10 abstracts were accepted for presentation at the virtual Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting to be held May 1-7, 2021. These abstracts feature a breadth of data, from presentations that demonstrate the potential of AI to analyze the growth of GA lesions…

    • 10 accepted abstracts include five oral presentations from pioneering artificial intelligence (AI) collaboration



    • Presentations support the potential of intravitreal pegcetacoplan, an investigational targeted C3 therapy, as the first treatment for geographic atrophy (GA)

    WALTHAM, Mass., April 16, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that 10 abstracts were accepted for presentation at the virtual Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting to be held May 1-7, 2021. These abstracts feature a breadth of data, from presentations that demonstrate the potential of AI to analyze the growth of GA lesions, to new safety and efficacy data for pegcetacoplan, an investigational targeted C3 therapy, in GA.

    "New imaging approaches, AI-based technologies, and ultimately the development of new medicines will transform the treatment of geographic atrophy," said Federico Grossi, M.D., Ph.D., Chief Medical Officer of Apellis. "Our data across these disciplines at ARVO exemplify our leadership in the retina and underscore the potential of pegcetacoplan to become the first medicine for people living with GA, a relentless disabling disease."

    The five oral presentations include studies that evaluated the use of AI to identify, monitor, and predict the growth of GA lesions. The analyses were performed on retinal scans from the positive Phase 2 FILLY study, which showed that pegcetacoplan reduced the growth rate of GA lesions, and were conducted in collaboration with the Ophthalmic Image Analysis (OPTIMA) group at the Medical University of Vienna, one of the world's leading data analysis laboratories for retinal diseases.

    "Our collaboration showed that AI can distinctly and reliably measure GA disease progression," said Ursula Schmidt-Erfurth, M.D., professor and chair of the Department of Ophthalmology at the University Eye Hospital, Vienna, Austria. "The results support the use of AI analytics as an ideal tool to evaluate disease activity as well as therapeutic efficacy in GA."



    The oral presentations from the AI collaboration include:

    • AI-Based Quantification of Photoreceptor Maintenance in the Treatment of Geographic Atrophy Secondary to AMD in the FILLY Trial – May 2, 11:15 a.m. - 12:45 p.m. ET

    • SD-OCT Based Analysis of Treatment Effects on Geographic Atrophy Secondary to AMD in the FILLY Trial of Pegcetacoplan – May 2, 11:15 a.m. - 12:45 p.m. ET
    • Monitoring GA Lesion Size on Optical Coherence Tomography (OCT) Using Automated Deep Learning-Based Image Segmentation in the FILLY Phase 2 Clinical Trial – May 2, 11:15 a.m. - 12:45 p.m. ET

    • Topographic Effects on AI Quantified Regional Progression in the FILLY Trial of Pegcetacoplan for Treatment of Geographic Atrophy Secondary to AMD – May 3, 4:30 p.m. - 6:00 p.m. ET
    • Predictive Identification of the Fastest Progressing Geographic Atrophy Lesions Based on Deep Learning in the Phase 2 FILLY Clinical Trial of Pegcetacoplan – May 3, 4:30 p.m - 6:00 p.m. ET

    Apellis abstracts regarding the safety and efficacy of pegcetacoplan and unmet need in GA include:

    • A Phase 1b Multi-Center, Open Label, Single-Arm Safety Study of Intravitreal Pegcetacoplan Supporting the Phase 3 DERBY and OAKS Studies for Geographic Atrophy: A Solution to "Tarmac Delay" – Poster Abstract – May 1, 10:15 a.m. - 12:00 p.m. ET
    • Safety of Intravitreal Pegcetacoplan in Patients with Neovascular Age-Related Macular Degeneration Receiving Anti-VEGF Therapy – Poster Abstract – May 3, 4:30 p.m. - 6:15 p.m. ET
    • Evaluation of Geographic Atrophy Secondary to Age-Related Macular Degeneration in Clinical Practice: Analysis of the AAO IRIS® Registry – Poster Abstract – May 4, 2:15 p.m. - 4:00 p.m. ET
    • Ocular Distribution of Pegcetacoplan in Rabbits Following a Single Intravitreal Injection. – Poster Abstract – May 5, 9:00 - 10:45 a.m. ET
    • Impact of Pegcetacoplan on Progression of Nascent Atrophy in Age-Related Macular Degeneration (AMD) – Paper Abstract – May 7, 4:15 p.m. - 5:45 p.m. ET

    About OPTIMA

    The Ophthalmic Image Analysis (OPTIMA) group at the Medical University of Vienna is a leading data analysis laboratory for retinal diseases. OPTIMA is a multidisciplinary group in retinology, computer science, and medical physics that develops computerized analysis methods for ophthalmic images and aims to individualize and lower treatment needs for patients affected by retinal diseases.

    About Pegcetacoplan

    Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Pegcetacoplan is being evaluated in several clinical studies across hematology, ophthalmology, nephrology, and neurology. Marketing applications for pegcetacoplan for paroxysmal nocturnal hemoglobinuria (PNH) are under review by the U.S. Food and Drug Administration (FDA), which has granted the application Priority Review designation, and the European Medicines Agency (EMA). Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of geographic atrophy and received orphan drug designation for the treatment of C3G by the FDA and European Medicines Agency. For additional information regarding pegcetacoplan clinical trials, visit https://apellis.com/our-science/clinical-trials.

    About Geographic Atrophy (GA) 

    GA is an advanced form of age-related macular degeneration (AMD), a leading cause of blindness. GA lesions affect the central portion of the retina, known as the macula, which is responsible for central vision. Excessive complement activation drives irreversible lesion growth in GA1, and C3 is the only target to precisely control complement overactivation. GA is progressive and irreversible, leading to central visual impairment and permanent loss of vision. Based on published studies, approximately one million people have GA in the United States and 5 million people have GA globally.2.3 There are currently no approved treatments for GA.

    About Apellis 

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit http://apellis.com.

    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 the company's clinical trials will be fully enrolled and completed when anticipated; 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 will be indicative of results that will be generated in future clinical trials; whether pegcetacoplan will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the company's clinical trials will warrant regulatory submissions and whether pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, C3G, IC-MPGN, ALS or any other indication when expected or at all; whether, if Apellis' products receive approval, they will be successfully distributed and marketed; and other factors discussed in the "Risk Factors" section of Apellis' Annual Report on Form 10-K filed with the Securities and Exchange Commission on February 25, 2021 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.

    Contacts:

    Apellis

    Media:

    Mark Dole



    617.997.3484

    Investors:

    Argot Partners



    212.600.1902

    ____________________________________________

    1 Seddon, JM, Rosner, B. Validated prediction models for macular degeneration progression and predictors of visual acuity loss identify high-risk individuals. Am J Ophthalmol 2019;198:223–261.

    2 Rudnicka AR, Jarrar Z, Wormald R, et al. Age and gender variations in age-related macular degeneration prevalence in populations of European ancestry: a meta-analysis. Ophthalmology 2012;119:571–580.

    3 Wong WL, Su X, Li X, et al. Global prevalence of age-related macular degeneration and disease burden projection for 2020 and 2040: a systematic review and meta-analysis. Lancet Glob Health 2014;2:e106–116.



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    • Post hoc analysis showed a 46% decrease in mean lesion growth in eight patients with bilateral GA comparing treated eye vs. untreated fellow eye at 24 months (p=0.007)
    • Top-line data from Phase 3 DERBY and OAKS studies expected in Q3 2021

    WALTHAM, Mass., April 13, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced 24-month data from the Phase 1b APL2-103 study of pegcetacoplan, an investigational targeted C3 therapy, in patients with advanced geographic atrophy (GA) secondary to age-related macular degeneration (AMD) and low vision. GA is a leading cause of blindness that affects approximately five million people worldwide1,2 and has no…

    • Post hoc analysis showed a 46% decrease in mean lesion growth in eight patients with bilateral GA comparing treated eye vs. untreated fellow eye at 24 months (p=0.007)

    • Top-line data from Phase 3 DERBY and OAKS studies expected in Q3 2021

    WALTHAM, Mass., April 13, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced 24-month data from the Phase 1b APL2-103 study of pegcetacoplan, an investigational targeted C3 therapy, in patients with advanced geographic atrophy (GA) secondary to age-related macular degeneration (AMD) and low vision. GA is a leading cause of blindness that affects approximately five million people worldwide1,2 and has no treatment.

    The Phase 1b study, designed to enroll approximately 12 patients with bilateral GA (disease in both eyes), was initiated to assess the safety of the Phase 3 formulation of pegcetacoplan (15mg/0.1mL). Patients were dosed monthly with pegcetacoplan in one eye using the fellow eye as an untreated control.

    The current post hoc analysis includes eight patients for whom data were available for at least 24 months. In this population, the growth rate of GA lesions in the treated eye was 46% (mean square root) slower than the untreated fellow eye (p=0.007). It has been shown that lesions in both eyes tend to grow at the same rate in patients with bilateral GA.3 Of the 13 enrolled patients, there were no reported cases of inflammation and two patients (16%) developed new-onset exudation during the 24-month study duration.

    "I am encouraged to see the continued effect of intravitreal pegcetacoplan in patients with GA." stated Eleonora Lad, M.D., Ph.D., Associate Professor of Ophthalmology at the Duke University Medical Center. "I believe that these findings, which demonstrate a 46% reduction in GA lesion growth at 24 months, are clinically meaningful. I am excited to learn the Phase 3 DERBY and OAKS results later this year."

    Post hoc analysis of Study APL2-103 at 24 months

    A Media Snippet accompanying this announcement is available by clicking on the image or link below:

    The patient population enrolled in the Phase 1b study is similar to patients enrolled in the Phase 3 DERBY and OAKS studies but allowed for more advanced disease with a wider range of baseline lesion size and lower baseline visual acuity. DERBY and OAKS use the same pegcetacoplan formulation tested in this study and top-line data are expected in the third quarter of 2021.

    Patients who participated in the APL2-103 Phase 1b study will be invited to enroll in the APL2-GA-305 GALE trial. The GALE trial is an open-label extension study designed to evaluate the long-term safety and efficacy of pegcetacoplan in patients with GA who participated in the Phase 1b study or who complete DERBY and OAKS.

    About Pegcetacoplan 

    Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Pegcetacoplan is being evaluated in several clinical studies across hematology, ophthalmology, nephrology, and neurology. Marketing applications for pegcetacoplan for paroxysmal nocturnal hemoglobinuria (PNH) are under review by the U.S. Food and Drug Administration (FDA), which has granted the application Priority Review designation, and the European Medicines Agency (EMA). Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of geographic atrophy and received orphan drug designation for the treatment of C3G by the FDA and European Medicines Agency. For additional information regarding pegcetacoplan clinical trials, visit https://apellis.com/our-science/clinical-trials.

    About Geographic Atrophy (GA) 

    GA is an advanced form of age-related macular degeneration (AMD), a leading cause of blindness. GA lesions affect the central portion of the retina, known as the macula, which is responsible for central vision. Excessive complement activation drives irreversible lesion growth in GA4, and C3 is the only target to precisely control complement overactivation. GA is progressive and irreversible, leading to central visual impairment and permanent loss of vision. Based on published studies, approximately one million people have GA in the United States and 5 million people have GA globally.1,2 There are currently no approved treatments for GA.

    About APL2-103

    The APL2-103 study is a Phase 1b, multicenter, open label, single arm, 24-month clinical trial to assess the safety of monthly intravitreal (IVT) injections of pegcetacoplan in patients diagnosed with advanced geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The primary outcome measures include incidence and severity of ocular and systemic treatment-emergent adverse events (TEAEs).



    About DERBY and OAKS

    DERBY (621 patients enrolled) and OAKS (638 patients enrolled) are Phase 3, multicenter, randomized, double-masked, sham-controlled studies comparing the efficacy and safety of intravitreal pegcetacoplan with sham injections in patients with GA secondary to AMD. The primary objective of the studies is to evaluate the efficacy of pegcetacoplan in patients with GA assessed by change in the total area of GA lesions from baseline as measured by fundus autofluorescence.

    About GALE

    GALE is a Phase 3, multicenter, open label, extension study to evaluate the long-term safety and efficacy of intravitreal pegcetacoplan in patients with GA secondary to AMD. The objectives of the study are to evaluate the long-term incidence and severity of ocular and systemic treatment emergent adverse events as well as change in the total area of GA lesions as measured by fundus autofluorescence.

    About Apellis 

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit http://apellis.com.

    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 the company's clinical trials will be fully enrolled and completed when anticipated; 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 will be indicative of results that will be generated in future clinical trials; whether pegcetacoplan will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the company's clinical trials will warrant regulatory submissions and whether pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, C3G, IC-MPGN, ALS or any other indication when expected or at all; whether, if Apellis' products receive approval, they will be successfully distributed and marketed; and other factors discussed in the "Risk Factors" section of Apellis' Annual Report on Form 10-K filed with the Securities and Exchange Commission on February 25, 2021 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:

    Mark Dole

     

    617.997.3484

    Investor Contact:

    Argot Partners

     

    212.600.1902 

    ____________________________________________________________________________

    1 Rudnicka AR, Jarrar Z, Wormald R, et al. Age and gender variations in age-related macular degeneration prevalence in populations of European ancestry: a meta analysis. Ophthalmology 2012;119:571–580.

    2 Wong WL, Su X, Li X, et al. Global prevalence of age-related macular degeneration and disease burden projection for 2020 and 2040: a systematic review and meta-analysis. Lancet Glob Health 2014;2:e106–116.

    3 Sunness JS, et al. The long-term natural history of geographic atrophy from age-related macular degeneration: enlargement of atrophy and implications for interventional clinical trials. Ophthalmology. 2007 Feb; 114(2):271-7.

    4 Seddon, JM, Rosner, B. Validated prediction models for macular degeneration progression and predictors of visual acuity loss identify high-risk individuals. Am J Ophthalmol 2019;198:223–261. 



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  7. STOCKHOLM, Sweden and WALTHAM, Massachusetts, March 18, 2021 /PRNewswire/ -- Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) and Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) today announced that The New England Journal of Medicine (NEJM) published results from the phase 3 PEGASUS study, which demonstrated the superiority of pegcetacoplan, an investigational targeted C3 therapy, in improving haemoglobin levels and showed improvements in key clinical outcomes compared to eculizumab, a C5 inhibitor, in adults with paroxysmal nocturnal haemoglobinuria (PNH) at 16 weeks who had persistent anaemia following treatment with eculizumab.

    "The data published in The New England Journal of Medicine underscore the potential of pegcetacoplan to be a significant…

    STOCKHOLM, Sweden and WALTHAM, Massachusetts, March 18, 2021 /PRNewswire/ -- Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) and Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) today announced that The New England Journal of Medicine (NEJM) published results from the phase 3 PEGASUS study, which demonstrated the superiority of pegcetacoplan, an investigational targeted C3 therapy, in improving haemoglobin levels and showed improvements in key clinical outcomes compared to eculizumab, a C5 inhibitor, in adults with paroxysmal nocturnal haemoglobinuria (PNH) at 16 weeks who had persistent anaemia following treatment with eculizumab.

    "The data published in The New England Journal of Medicine underscore the potential of pegcetacoplan to be a significant advancement for people living with PNH," said Peter Hillmen, M.B., Ch.B., Ph.D, professor of experimental haematology at Leeds Teaching Hospitals NHS Trust and PEGASUS study author. "There is still a need for new treatments because many patients with PNH treated with C5 inhibitors today remain anemic, resulting in moderate to severe fatigue, with a proportion continuing to require transfusions."

    "The PEGASUS study results demonstrate that, if approved, pegcetacoplan has the potential to elevate the standard of care for PNH by providing more complete disease control," said Federico Grossi, M.D., Ph.D., Chief Medical Officer of Apellis. "We are working to quickly bring this potential new treatment to PNH patients and to advance development of pegcetacoplan for many other serious, complement-driven diseases."

    "The NEJM publication of the PEGASUS study results reflect the significance of these data for both the medical and PNH patient communities," said Ravi Rao, Head of Research & Development and Chief Medical Officer at Sobi. "The results further advance our understanding of the role of C3 in PNH and our hope is to contribute to the improvement and care for PNH patients around the world".

    The results published in NEJM highlight that pegcetacoplan met the study's primary endpoint for efficacy, demonstrating superiority to eculizumab with a statistically significant improvement in adjusted means of 3.8 g/dL of haemoglobin at week 16 (p<0.001). Additionally, 85% of pegcetacoplan-treated patients were transfusion free over 16 weeks versus 15% of eculizumab-treated patients. There were meaningful improvements across key markers of disease such as absolute reticulocyte count, lactate dehydrogenase (LDH), and fatigue as measured by the Functional Assessment of Chronic Illness Therapy (FACIT)-fatigue score. 

    Safety was comparable between pegcetacoplan and eculizumab in this study. Seven of 41 patients (17%) in the pegcetacoplan group experienced a SAE, and 6 of 39 patients (15%) in the eculizumab group experienced SAEs. No cases of meningitis and no deaths were reported in either treatment group.

    The most common adverse events reported during the 16-week, randomized, controlled treatment period in the pegcetacoplan and eculizumab groups, respectively, were injection site reactions (37% vs. 3%), diarrhoea (22% vs. 3%), breakthrough haemolysis (10% vs. 23%), headache (7% vs. 23%), and fatigue (5% vs. 15%).

    Top-line results from the PEGASUS study at 48 weeks were recently announced, which showed sustained improvements in key markers of disease as well as a consistent safety profile with previously reported data.

    Marketing applications for pegcetacoplan for the treatment of PNH are under review by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). The FDA granted the application Priority Review designation and set a target action date of May 14, 2021. An opinion from the Committee for Medicinal Products for Human Use (CHMP) is expected in 2021.

    About the PEGASUS Study

    The PEGASUS study (APL2-302; NCT03500549) is a multi-center, randomized, head-to-head phase 3 study in 80 adults with paroxysmal nocturnal haemoglobinuria (PNH). The primary objective of this study was to establish the efficacy and safety of pegcetacoplan compared to eculizumab. Participants must have been on eculizumab (stable for at least three months) with a haemoglobin level of <10.5 g/dL at the screening visit. During the four-week run-in, patients were dosed with 1080 mg of pegcetacoplan twice weekly (n=41) in addition to their current dose of eculizumab. During the 16-week randomized, controlled period, patients were randomized to receive either 1080 mg of pegcetacoplan twice weekly or their current dose of eculizumab (n=39). All participants completing the randomized controlled period (n=77) opted to enter the open-label pegcetacoplan treatment period.

    About pegcetacoplan

    Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Pegcetacoplan is being evaluated in several clinical studies across haematology, ophthalmology, nephrology, and neurology. Marketing applications for pegcetacoplan for paroxysmal nocturnal haemoglobinuria (PNH) are under review by the U.S. Food and Drug Administration (FDA), which has granted the application Priority Review designation, and the European Medicines Agency (EMA). Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of geographic atrophy and received orphan drug designation for the treatment of C3G by the FDA and European Medicines Agency. For additional information regarding pegcetacoplan clinical trials, visit https://apellis.com/our-science/clinical-trials.

    About Paroxysmal Nocturnal Haemoglobinuria (PNH) 

    PNH is a rare, chronic, life-threatening blood disorder characterized by the destruction of oxygen-carrying red blood cells through extravascular and intravascular haemolysis. Persistently low haemoglobin can result in frequent transfusions and debilitating symptoms such as severe fatigue, haemoglobinuria and difficulty breathing (dyspnea). A retrospective analysis shows that, even on eculizumab, approximately 72% of people with PNH have anemi, a key indicator of ongoing haemolysis.1 The analysis also finds that 36% of patients require one or more transfusions a year and 16% require three or more.1

    About Apellis 

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within haematology, ophthalmology, nephrology, and neurology. For more information, please visit http://apellis.com.

    About Sobi

    Sobi is a specialized international biopharmaceutical company transforming the lives of people with rare diseases. Sobi is providing sustainable access to innovative therapies in the areas of haematology, immunology and specialty indications. Today, Sobi employs approximately 1,500 people across Europe, North America, the Middle East, Asia and North Africa. In 2020, Sobi's revenue amounted to SEK 15.3 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. You can find more information about Sobi at www.sobi.com.

    Contacts

    Sobi

    Paula Treutiger, Head of Communication & Investor Relations

    + 46 733 666 599

     

    Maria Kruse, Corporate Communication & Investor Relations

    +46 767 248 830

    Apellis

    Media:

    Lissa Pavluk 

     

    +1 617 977 6764

    Investors:

    Argot Partners



    +1 212 600 1902

    1.  McKinley C. Extravascular Hemolysis Due to C3-Loading in Patients with PNH Treated with Eculizumab: Defining the Clinical Syndrome. Blood. 2017;130:3471.

    This information was brought to you by Cision http://news.cision.com

    https://news.cision.com/swedish-orphan-biovitrum-ab/r/the-nejm-publishes-phase-3-pegasus-study-results-comparing-pegcetacoplan-to-eculizumab-for-pnh,c3308729

    The following files are available for download:

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    The New England Journal of Medicine publishes phase 3 PEGASUS study results comparing pegcetacoplan to eculizumab for PNH

     

    Cision View original content:http://www.prnewswire.com/news-releases/the-nejm-publishes-phase-3-pegasus-study-results-comparing-pegcetacoplan-to-eculizumab-for-pnh-301249722.html

    SOURCE Swedish Orphan Biovitrum AB

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    • Pegcetacoplan, an investigational targeted C3 therapy for serious, complement-driven diseases, demonstrated superiority to eculizumab with a statistically significant improvement in hemoglobin levels and showed improvements in key clinical outcomes

    • Marketing applications for pegcetacoplan are under Priority Review with the FDA and under review with the EMA

    WALTHAM, Mass. and STOCKHOLM, March 17, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) and Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) today announced that the New England Journal of Medicine (NEJM) published results from the Phase 3 PEGASUS study, which demonstrated the superiority of pegcetacoplan, an investigational targeted C3 therapy, in improving hemoglobin…

    • Pegcetacoplan, an investigational targeted C3 therapy for serious, complement-driven diseases, demonstrated superiority to eculizumab with a statistically significant improvement in hemoglobin levels and showed improvements in key clinical outcomes



    • Marketing applications for pegcetacoplan are under Priority Review with the FDA and under review with the EMA

    WALTHAM, Mass. and STOCKHOLM, March 17, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) and Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) today announced that the New England Journal of Medicine (NEJM) published results from the Phase 3 PEGASUS study, which demonstrated the superiority of pegcetacoplan, an investigational targeted C3 therapy, in improving hemoglobin levels and showed improvements in key clinical outcomes compared to eculizumab, a C5 inhibitor, in adults with paroxysmal nocturnal hemoglobinuria (PNH) at 16 weeks who had persistent anemia following treatment with eculizumab.

    "The data published in the New England Journal of Medicine underscore the potential of pegcetacoplan to be a significant advancement for people living with PNH," said Peter Hillmen, M.B., Ch.B., Ph.D, professor of experimental haematology at Leeds Teaching Hospitals NHS Trust and PEGASUS study author. "There is still a need for new treatments because many patients with PNH treated with C5 inhibitors today remain anemic, resulting in moderate to severe fatigue, with a proportion continuing to require transfusions."

    "The PEGASUS study results demonstrate that, if approved, pegcetacoplan has the potential to elevate the standard of care for PNH by providing more complete disease control," said Federico Grossi, M.D., Ph.D., chief medical officer of Apellis. "We are working to quickly bring this potential new treatment to PNH patients and to advance development of pegcetacoplan for many other serious, complement-driven diseases."

    "The NEJM publication of the PEGASUS study results reflect the significance of these data for both the medical and PNH patient communities," said Ravi Rao, head of research and development and chief medical officer at Sobi. "The results further advance our understanding of the role of C3 in PNH and our hope is to contribute to the improvement and care for PNH patients around the world."

    The results published in NEJM highlight that pegcetacoplan met the study's primary endpoint for efficacy, demonstrating superiority to eculizumab with a statistically significant improvement in adjusted means of 3.8 g/dL of hemoglobin at week 16 (p<0.001). Additionally, 85% of pegcetacoplan-treated patients were transfusion free over 16 weeks versus 15% of eculizumab-treated patients. There were meaningful improvements across key markers of disease such as absolute reticulocyte count, lactate dehydrogenase (LDH), and fatigue as measured by the Functional Assessment of Chronic Illness Therapy (FACIT)-fatigue score.

    Safety was comparable between pegcetacoplan and eculizumab in this study. Seven of 41 patients (17%) in the pegcetacoplan group experienced a SAE, and 6 of 39 patients (15%) in the eculizumab group experienced SAEs. No cases of meningitis and no deaths were reported in either treatment group.

    The most common adverse events reported during the 16-week, randomized, controlled treatment period in the pegcetacoplan and eculizumab groups, respectively, were injection site reactions (37% vs. 3%), diarrhea (22% vs. 3%), breakthrough hemolysis (10% vs. 23%), headache (7% vs. 23%), and fatigue (5% vs. 15%).

    Top-line results from the PEGASUS study at 48 weeks were recently announced, which showed sustained improvements in key markers of disease as well as a consistent safety profile with previously reported data.

    Marketing applications for pegcetacoplan for the treatment of PNH are under review by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). The FDA granted the application Priority Review designation and set a target action date of May 14, 2021. An opinion from the Committee for Medicinal Products for Human Use (CHMP) is expected in 2021.

    About the PEGASUS Study

    The PEGASUS study (APL2-302; NCT03500549) is a multi-center, randomized, head-to-head Phase 3 study in 80 adults with paroxysmal nocturnal hemoglobinuria (PNH). The primary objective of this study was to establish the efficacy and safety of pegcetacoplan compared to eculizumab. Participants must have been on eculizumab (stable for at least three months) with a hemoglobin level of <10.5 g/dL at the screening visit. During the four-week run-in, patients were dosed with 1080 mg of pegcetacoplan twice weekly (n=41) in addition to their current dose of eculizumab. During the 16-week randomized, controlled period, patients were randomized to receive either 1080 mg of pegcetacoplan twice weekly or their current dose of eculizumab (n=39). All participants completing the randomized controlled period (n=77) opted to enter the open-label pegcetacoplan treatment period.

    About Pegcetacoplan

    Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Pegcetacoplan is being evaluated in several clinical studies across hematology, ophthalmology, nephrology, and neurology. Marketing applications for pegcetacoplan for paroxysmal nocturnal hemoglobinuria (PNH) are under review by the U.S. Food and Drug Administration (FDA), which has granted the application Priority Review designation, and the European Medicines Agency (EMA). Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of geographic atrophy and received orphan drug designation for the treatment of C3G by the FDA and European Medicines Agency. For additional information regarding pegcetacoplan clinical trials, visit https://apellis.com/our-science/clinical-trials.

    About Paroxysmal Nocturnal Hemoglobinuria (PNH) 

    PNH is a rare, chronic, life-threatening blood disorder characterized by the destruction of oxygen-carrying red blood cells through extravascular and intravascular hemolysis. Persistently low hemoglobin can result in frequent transfusions and debilitating symptoms such as severe fatigue, hemoglobinuria and difficulty breathing (dyspnea). A retrospective analysis shows that, even on eculizumab, approximately 72% of people with PNH have anemia, a key indicator of ongoing hemolysis.1 The analysis also finds that 36% of patients require one or more transfusions a year and 16% require three or more.1

    About Apellis 

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit http://apellis.com.

    About Sobi

    Sobi is a specialized international biopharmaceutical company transforming the lives of people with rare diseases. Sobi is providing sustainable access to innovative therapies in the areas of haematology, immunology and specialty indications. Today, Sobi employs approximately 1,500 people across Europe, North America, the Middle East, Asia and North Africa. In 2020, Sobi's revenue amounted to SEK 15.3 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. You can find more information about Sobi at www.sobi.com.

    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 the company's clinical trials will be fully enrolled and completed when anticipated; 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 will be indicative of results that will be generated in future clinical trials; whether pegcetacoplan will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the company's clinical trials will warrant regulatory submissions and whether pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, C3G, IC-MPGN, ALS or any other indication when expected or at all; whether, if Apellis' products receive approval, they will be successfully distributed and marketed; and other factors discussed in the "Risk Factors" section of Apellis' Annual Report on Form 10-K filed with the Securities and Exchange Commission on February 25, 2020 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.

    Contacts:



    Apellis

    Media:

    Lissa Pavluk 

     

    617.977.6764

    Investors: 

    Argot Partners



    212.600.1902

    Sobi

    Paula Treutiger, Head of Communication & Investor Relations

    + 46 733 666 599

    Maria Kruse, Corporate Communication & Investor Relations

    + 46 767 248 830





    1.  McKinley C. Extravascular Hemolysis Due to C3-Loading in Patients with PNH Treated with Eculizumab: Defining the Clinical Syndrome. Blood. 2017;130:3471.



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    • Data published in Ophthalmology and the American Journal of Ophthalmology underscore the potential of pegcetacoplan, an investigational targeted C3 therapy, for geographic atrophy

    WALTHAM, Mass., March 10, 2021 (GLOBE NEWSWIRE) --  Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that two leading journals, Ophthalmology and the American Journal of Ophthalmology, published post hoc analyses from the Phase 2 FILLY study of intravitreal pegcetacoplan for geographic atrophy (GA) secondary to AMD. GA is a leading cause of blindness that affects approximately five million people around the world1,2 and has no approved treatments.

    • The analysis, "Impact of Baseline…
    • Data published in Ophthalmology and the American Journal of Ophthalmology underscore the potential of pegcetacoplan, an investigational targeted C3 therapy, for geographic atrophy

    WALTHAM, Mass., March 10, 2021 (GLOBE NEWSWIRE) --  Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that two leading journals, Ophthalmology and the American Journal of Ophthalmology, published post hoc analyses from the Phase 2 FILLY study of intravitreal pegcetacoplan for geographic atrophy (GA) secondary to AMD. GA is a leading cause of blindness that affects approximately five million people around the world1,2 and has no approved treatments.

    • The analysis, "Impact of Baseline Characteristics on Geographic Atrophy Progression in the FILLY Trial Evaluating the Complement C3 Inhibitor Pegcetacoplan," was published in the American Journal of Ophthalmology, and showed that efficacy results across patient subgroups were consistent with the FILLY results.



    • Published in Ophthalmology, "Characterizing New-Onset Exudation in the Randomized Phase 2 FILLY Trial of Complement Inhibitor Pegcetacoplan for Geographic Atrophy," further clarified characteristics of investigator-determined exudations reported in the study.

    "Geographic atrophy remains the most significant unmet need in the retina, and we are committed to advancing the first medicine for people living with this relentless disease," said Federico Grossi, M.D., Ph.D., chief medical officer of Apellis. "These data from two new analyses of our positive Phase 2 FILLY study reinforce the potential of pegcetacoplan in GA, and we look forward to sharing results from our Phase 3 GA studies in the third quarter of this year."

    Pegcetacoplan is currently being evaluated in two pivotal studies, DERBY and OAKS, which enrolled more than 1,200 patients with GA.

    About Pegcetacoplan (APL-2)

    Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Pegcetacoplan is being evaluated in several clinical studies across hematology, ophthalmology, nephrology, and neurology. Marketing applications for pegcetacoplan for paroxysmal nocturnal hemoglobinuria (PNH) are under review by the U.S. Food and Drug Administration (FDA), which has granted the application Priority Review designation, and the European Medicines Agency (EMA). Pegcetacoplan was granted Fast Track designation by the FDA for the treatment of geographic atrophy and received orphan drug designation for the treatment of C3G by the FDA and EMA. For additional information regarding pegcetacoplan clinical trials, visit https://apellis.com/our-science/clinical-trials.

    About Geographic Atrophy (GA) 

    GA is an advanced form of age-related macular degeneration (AMD), a leading cause of blindness. GA lesions affect the central portion of the retina, known as the macula, which is responsible for central vision. Excessive complement activation drives irreversible lesion growth in GA3, and C3 is the only target to precisely control complement overactivation. GA is progressive and irreversible, leading to central visual impairment and permanent loss of vision. Based on published studies, approximately one million people have GA in the United States and 5 million people have GA globally.1,2 There are currently no approved treatments for GA.

    About FILLY

    The FILLY study was a 246-patient, Phase 2, multicenter, randomized, single-masked, sham-controlled clinical trial evaluating pegcetacoplan in patients with GA secondary to AMD conducted at over 40 clinical sites in the United States, Australia, and New Zealand. Pegcetacoplan was administered as an intravitreal injection monthly or every other month (EOM) for 12 months, followed by six months of monitoring after the end of treatment. The primary efficacy endpoint was the change in GA lesion area from baseline to Month 12 in pegcetacoplan-treated patients compared to sham.

    About DERBY and OAKS

    DERBY (621 patients enrolled) and OAKS (638 patients enrolled) are Phase 3, multicenter, randomized, double-masked, sham-controlled studies comparing the efficacy and safety of intravitreal pegcetacoplan with sham injections in patients with GA secondary to AMD. The primary objective of the studies is to evaluate the efficacy of pegcetacoplan in patients with GA assessed by change in the total area of GA lesions from baseline as measured by fundus autofluorescence.

    About Apellis 

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit www.apellis.com.

    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 in respect of the expected closing of the exchanges. 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 the conditions for the closing of the exchanges will be satisfied and other factors discussed in the "Risk Factors" section of Apellis' Annual Report on Form 10-K filed with the Securities and Exchange Commission on February 25, 2021 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:

    Mark Dole



    617.997.3484

    Investor Contact:

    Argot Partners

     

    212.600.1902 

    1 Rudnicka AR, Jarrar Z, Wormald R, et al. Age and gender variations in age-related macular degeneration prevalence in populations of European ancestry: a meta-analysis. Ophthalmology 2012;119:571–580.

    2 Wong WL, Su X, Li X, et al. Global prevalence of age-related macular degeneration and disease burden projection for 2020 and 2040: a systematic review and meta-analysis. Lancet Glob Health 2014;2:e106–116.

    3 Seddon, JM, Rosner, B. Validated prediction models for macular degeneration progression and predictors of visual acuity loss identify high-risk individuals. Am J Ophthalmol 2019;198:223–261. 



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  8. WALTHAM, Mass., March 05, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company approved the grant of equity awards to two new employees with grant date of March 1, 2021, as equity inducement awards outside of the company's 2017 Stock Incentive Plan (but under the terms of the 2021 Inducement Stock Incentive Plan) and material to the employees' acceptance of employment with the company. The equity awards were approved in accordance with Nasdaq Listing Rule 5635(c)(4).

    The employees received options to purchase 33,650 shares of Apellis common stock and 825 restricted stock units (RSUs). The options have an exercise price…

    WALTHAM, Mass., March 05, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company approved the grant of equity awards to two new employees with grant date of March 1, 2021, as equity inducement awards outside of the company's 2017 Stock Incentive Plan (but under the terms of the 2021 Inducement Stock Incentive Plan) and material to the employees' acceptance of employment with the company. The equity awards were approved in accordance with Nasdaq Listing Rule 5635(c)(4).

    The employees received options to purchase 33,650 shares of Apellis common stock and 825 restricted stock units (RSUs). The options have an exercise price of $47.68, which is equal to the closing price of Apellis common stock on March 1, 2021, the grant date of the options. One-fourth of the shares underlying the employee options will vest on the one year anniversary of the grant date and thereafter 1/48th of the shares underlying the employee options will vest monthly, such that the shares underlying the options granted to the employees will be fully vested on the fourth anniversary of the grant date, subject to the employees' continued employment with Apellis on such vesting dates. Each RSU will vest as to 25% of the shares underlying the RSU award on the first anniversary of the grant date and as to an additional 25% of the shares underlying the RSU award annually thereafter, subject to each such employee's continued employment on each vesting date.

    About Apellis

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit www.apellis.com.

    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 the company's clinical trials will be fully enrolled and completed when anticipated; 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 will be indicative of results that will be generated in future clinical trials; whether pegcetacoplan will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the company's clinical trials will warrant regulatory submissions and whether pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, C3G, IC-MPGN, ALS or any other indication when expected or at all; 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 2, 2020 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.

    Investor Contact:

    Argot Partners



    +1 212.600.1902



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    • Company will not pursue additional development of APL-9 for the treatment of severe COVID-19

    • Interim review by independent data monitoring committee (DMC) found no meaningful reduction in the overall mortality rate in Phase 1/2 study

    • No safety signals were observed by the DMC

    WALTHAM, Mass., March 04, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company will not pursue additional development of APL-9, an investigational targeted C3 therapy designed for acute interventions, for the treatment of severe COVID-19. The decision followed an interim review of mortality data from the Phase 1/2 study by an independent data monitoring…

    • Company will not pursue additional development of APL-9 for the treatment of severe COVID-19



    • Interim review by independent data monitoring committee (DMC) found no meaningful reduction in the overall mortality rate in Phase 1/2 study



    • No safety signals were observed by the DMC

    WALTHAM, Mass., March 04, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company will not pursue additional development of APL-9, an investigational targeted C3 therapy designed for acute interventions, for the treatment of severe COVID-19. The decision followed an interim review of mortality data from the Phase 1/2 study by an independent data monitoring committee (DMC), which found no meaningful reduction in the overall mortality rate in patients treated with APL-9 in combination with standard of care therapy compared to standard of care alone. No safety signals were observed by the DMC.

    "We initiated the clinical development of APL-9 in patients with severe COVID-19 because we believed that complement dysregulation may play a key role in disease mortality. We felt a responsibility to learn if controlling complement could help save lives during this devastating pandemic," said Lukas Scheibler, Ph.D., chief innovation officer at Apellis. "While the mortality results from this study were not what we had hoped, we extend our heartfelt thanks to the patients and their families, healthcare providers, and investigators who joined us in working to address an urgent public health need. We remain confident in the potential of targeting C3 for complement-driven diseases and are committed to bringing transformative treatments to patients."

    No additional endpoints were analyzed as part of the interim review by the DMC. Enrollment is complete, and the study reached the last patient visit. Apellis plans to provide full results in a scientific forum following completion of the full data analysis.

    About APL-9

    APL-9 is an investigational drug designed to control the complement cascade centrally at C3 and may have the potential to treat a range of diseases caused by excessive or uncontrolled activation of complement. APL-9 leverages the same mechanism of action as Apellis' lead compound, pegcetacoplan (APL-2), but has a lower molecular weight and shorter half-life. APL-9 is designed to be intravenously administered for acute use.



    About Apellis 

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit http://apellis.com.

    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 the company's clinical trials will be fully enrolled and completed when anticipated; 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 will be indicative of results that will be generated in future clinical trials; whether pegcetacoplan will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the company's clinical trials will warrant regulatory submissions and whether pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, C3G, IC-MPGN, ALS or any other indication when expected or at all; whether, if Apellis' products receive approval, they will be successfully distributed and marketed; and other factors discussed in the "Risk Factors" section of Apellis' Annual Report on Form 10-K filed with the Securities and Exchange Commission on February 25, 2020 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: 

    Lissa Pavluk 

      

    617.977.6764

    Investor Contact:

    Argot Partners



    +1 212.600.1902



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    • Marketing applications for pegcetacoplan for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) under review by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). PDUFA target action date is May 14, 2021
    • Top-line results from the Phase 3 PRINCE study in treatment-naïve PNH patients expected in the second quarter of 2021
    • Top-line results from the Phase 3 geographic atrophy (GA) studies expected in the third quarter of 2021
    • Three new product candidates advancing into clinical development by the end of 2022
    • Cash and investments of $877.6 million as of December 31, 2020 support cash runway into the second half of 2022
    • Conference call scheduled today at 4:30 p.m. ET

    WALTHAM, Mass., Feb. 25, 2021 (GLOBE NEWSWIRE…

    • Marketing applications for pegcetacoplan for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) under review by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). PDUFA target action date is May 14, 2021
    • Top-line results from the Phase 3 PRINCE study in treatment-naïve PNH patients expected in the second quarter of 2021
    • Top-line results from the Phase 3 geographic atrophy (GA) studies expected in the third quarter of 2021
    • Three new product candidates advancing into clinical development by the end of 2022
    • Cash and investments of $877.6 million as of December 31, 2020 support cash runway into the second half of 2022
    • Conference call scheduled today at 4:30 p.m. ET

    WALTHAM, Mass., Feb. 25, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced its fourth quarter and full year 2020 financial results and business highlights.

    "2020 was a defining year for Apellis, marked by positive Phase 3 PEGASUS data, which highlighted both the potential of pegcetacoplan to elevate the standard of care in PNH and the broad platform potential of targeting C3 for complement-driven diseases," said Cedric Francois M.D., Ph.D., co-founder and chief executive officer of Apellis. "In 2021, we look forward to a transformational year for Apellis as we further build on our global leadership in complement.

    "With a potential U.S. product approval and commercial launch in PNH in the first half of the year, Phase 3 study readouts in geographic atrophy in the third quarter, and multiple clinical development milestones across our systemic pipeline, we are positioned to deliver on the full potential of targeting C3 across multiple diseases with high unmet need," Dr. Francois continued. "Importantly, we have a unique opportunity to advance the first potential medicine for geographic atrophy, a leading cause of blindness that affects approximately five million people around the world."

    Fourth Quarter Business Highlights and Upcoming Milestones:

    Systemic Pegcetacoplan

    • In December 2020, Apellis and Sobi announced positive top-line results at Week 48 from the Phase 3 PEGASUS study, which found that treatment with pegcetacoplan, an investigational targeted C3 therapy, resulted in sustained hematological and clinical improvements in patients with paroxysmal nocturnal hemoglobinuria (PNH). The safety profile of pegcetacoplan was consistent with previously reported data.
    • In December 2020, the company reported additional analyses from the Phase 3 PEGASUS study, demonstrating statistically significant improvements in overall treatment response and substantial quality-of-life improvements with pegcetacoplan for PNH compared to Soliris® (eculizumab) at 16 weeks. Additionally, a matching-adjusted indirect comparison (MAIC) suggested improvements in clinical, hematological, and quality-of-life outcomes in patients treated with pegcetacoplan compared to Ultomiris® (ravulizumab), a longer-acting C5 inhibitor. The data were presented at the virtual American Society of Hematology Annual Meeting (ASH).
    • In November 2020, Apellis and Sobi announced that the first patient had been dosed in the potentially registrational Phase 2 MERIDIAN study of pegcetacoplan, which is expected to enroll more than 200 adults with sporadic amyotrophic lateral sclerosis (ALS). The companies expect to complete study enrollment in the second half of 2021.
    • In November 2020, the company announced that the FDA accepted and granted Priority Review designation for the New Drug Application (NDA) for pegcetacoplan for the treatment of PNH, with a Prescription Drug User Fee Act (PDUFA) target action date of May 14, 2021. A Marketing Authorization Application (MAA) is also under review by the EMA with the potential for a European Commission decision on the MAA in the second half of 2021.
    • Apellis and Sobi expect to report top-line results from the Phase 3 PRINCE study in PNH patients who are treatment-naïve in the second quarter of 2021.
    • In the second half of 2021, Apellis expects to initiate a Phase 3 study in immune complex membranoproliferative glomerulonephritis (IC-MPGN) and C3 glomerulopathy (C3G), and Sobi plans to initiate a Phase 3 study in cold agglutinin disease (CAD) and a potentially registrational Phase 2 study in hematopoietic stem cell transplantation-associated thrombotic microangiopathy (HSCT-TMA).

    Intravitreal Pegcetacoplan

    • In November 2020, Apellis announced findings from the largest retrospective study in GA, which underscored the high unmet need for a treatment in geographic atrophy. Conducted in partnership with Verana Health, the analysis of the American Academy of Ophthalmology (AAO) IRIS® Registry was presented in a late-breaking oral session at AAO 2020. Results showed that visual acuity substantially worsened over a brief time, with a significant percentage of GA patients becoming ineligible to drive and legally blind within one to two years of diagnosis. The data also reiterated that wet AMD is an expected occurrence in people with GA, which happens nearly three times more frequently in people with wet AMD in the fellow eye than in people with bilateral GA (13.3% vs. 4.7%, respectively, over 12 months).
    • The company expects to announce top-line results from the Phase 3 DERBY and OAKS studies in the third quarter of 2021.

    COVID-19 Clinical Program with APL-9

    • Apellis expects to report top-line data from its Phase 1/2 clinical study of APL-9, a targeted C3 therapy designed for acute conditions, in patients with severe COVID-19 in the second quarter of 2021.

    Pipeline Expansion

    • Apellis plans to advance three new product candidates into clinical development by the end of 2022.

    Corporate Highlights

    • In January 2021, Apellis announced that it entered into separate, privately negotiated exchange agreements with certain holders of its 3.500% Convertible Senior Notes due 2026 issued in September 2019. Under the terms of the exchange agreements, the holders agreed to exchange with Apellis approximately $126.1 million in aggregate principal amount of Notes held by them for an aggregate of 3,906,869 shares of its common stock. The exchange transactions closed on January 25, 2021.

    Fourth Quarter and Full Year 2020 Financial Results:

    As of December 31, 2020, Apellis had $877.6 million in cash, cash equivalents, and short-term marketable securities, compared to $352.0 million in cash and cash equivalents as of December 31, 2019. This increase primarily reflects the addition of cash from the company's follow-on offering for gross proceeds of $404.2 million in January 2020, a convertible offering for gross proceeds of $328.9 million in May 2020, and the receipt of $250.0 million in the upfront proceeds for the Sobi transaction in October 2020, less cash used in operations.

    Apellis reported net income of $78.3 million for the fourth quarter of 2020, compared to a net loss of $113.2 million for the fourth quarter of 2019. This change was due to the Sobi upfront payment, which was recognized as revenue in the fourth quarter. For the full year ending December 31, 2020, Apellis reported a net loss of $344.9 million, compared to a net loss of $304.7 million for the full year ending December 31, 2019.

    Research and development expenses were $75.4 million in the fourth quarter of 2020, compared to $78.5 million for the same period in 2019. For the full year ending December 31, 2020, research and development expenses were $325.0 million compared to $221.0 million for the full year ending December 31, 2019. The increase in R&D expense for full year 2020 was primarily attributable to an increase in manufacturing expenses in connection with the supply of pegcetacoplan for the company's Phase 3 clinical trials and potential commercial launch, clinical trial costs associated with the ongoing Phase 3 trials and planned clinical trials in other indications, compensation and related personnel costs primarily due to the hiring of additional personnel, regulatory and quality expenses, licensee fee to Penn related to the Sobi transaction, research and innovation expense, and device development expenses. We expect our research and development expenses to continue to increase as the number of patients in our trials increases and the number of ongoing trials increases.

    General and administrative expenses were $44.5 million in the fourth quarter of 2020, compared to $27.5 million for the same period in 2019. For the full year ending December 31, 2020, general and administrative expenses were $139.4 million, compared to $67.0 million for the full year ending December 31, 2019. The increase in general and administrative expenses for the full year 2020 was primarily attributable to an increase in professional and consulting fees, employee-related costs due to the hiring of additional personnel, directors stock compensation expense, insurance costs, and office, travel and related costs, offset by a decrease in information technology expenses.

    Conference Call and Webcast

    Apellis will host a conference call and webcast to discuss its fourth quarter and full year 2020 financial results and business highlights today, February 25, 2021, at 4:30 p.m. ET. To access the conference call, please dial (866) 774-0323 (local) or (602) 563-8683 (international) at least 10 minutes prior to the start time and refer to conference ID 6956712. A live audio webcast of the event and accompanying slides may also be accessed through the "Events and Presentations" page of the "Investors and Media" section of the company's website at http://investors.apellis.com/events-and-presentations. A replay of the webcast will be available for 30 days following the event.

    About Pegcetacoplan

    Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Pegcetacoplan is being evaluated in several clinical studies across hematology, ophthalmology, nephrology, and neurology. Marketing applications for pegcetacoplan for paroxysmal nocturnal hemoglobinuria (PNH) are under review by the U.S. Food and Drug Administration (FDA), which has granted the application Priority Review designation, and the European Medicines Agency (EMA). Pegcetacoplan was granted Fast Track designation by the FDA for the treatment of geographic atrophy and received orphan drug designation for the treatment of C3G by the FDA and EMA. For additional information regarding pegcetacoplan clinical trials, visit https://apellis.com/our-science/clinical-trials.

    About APL-9

    APL-9 is an investigational, targeted C3 therapy designed to control the complement cascade centrally and may have the potential to treat a range of diseases caused by excessive activation of complement. APL-9 leverages the same mechanism of action as Apellis' lead compound, pegcetacoplan, but has a lower molecular weight and shorter half-life. APL-9 is designed to be intravenously administered for acute use.

    About Apellis

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit www.apellis.com.

    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 in respect of the expected closing of the exchanges. 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 the conditions for the closing of the exchanges will be satisfied and other factors discussed in the "Risk Factors" section of Apellis' Annual Report on Form 10-K filed with the Securities and Exchange Commission on February 25, 2021 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.



    Investor Contact:

    Argot Partners



    +1 212.600.1902

    Media Contact:

    Tracy Vineis

     

    617.420.4839

     
     
    APELLIS PHARMACEUTICALS, INC.
    CONDENSED CONSOLIDATED BALANCE SHEETS
    (Amounts in thousands, except per share amounts)
      December 31,
       2020   2019 
    Assets    
    Current assets:    
    Cash and cash equivalents $565,779  $351,985 
    Marketable securities  311,869   - 
    Prepaid assets  11,400   19,802 
    Restricted cash  1,266   - 
    Other current assets  26,878   1,308 
    Total current assets  917,192   373,095 
    Non-current assets:    
    Right-of-use assets  17,719   14,110 
    Property and equipment, net  6,803   1,655 
    Other assets  18,855   385 
    Total assets $960,569  $389,245 
    Liabilities and Stockholders' Equity    
    Current liabilities:    
    Accounts payable $8,477  $8,361 
    Accrued expenses  111,935   54,783 
    Current portion of development derivative liability  4,230   - 
    Current portion of right-of-use liabilities  3,685   2,609 
    Total current liabilities  128,327   65,753 
    Long-term liabilities:    
    Convertible senior notes  358,830   142,567 
    Development derivative liability  253,638   134,839 
    Right-of-use liabilities  15,217   11,857 
    Total liabilities  756,012   355,016 
    Commitments and contingencies (note 15)  -   - 
    Stockholders' equity:    
    Preferred stock, $0.0001 par value; 10,000 shares authorized and zero shares issued and outstanding at December 31, 2020 and 2019  -   - 
    Common stock, $0.0001 par value; 200,000 shares authorized at December 31, 2020 and 2019; 76,130 and 63,938 shares issued and outstanding at December 31, 2020 and 2019, respectively  8   6 
    Additional paid-in capital  1,131,013   615,850 
    Accumulated other comprehensive loss  (117)  (154)
    Accumulated deficit  (926,347)  (581,473)
    Total stockholders' equity  204,557   34,229 
    Total liabilities and stockholders' equity $960,569  $389,245 
         



        
    APELLIS PHARMACEUTICALS, INC.
    CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
    (Amounts in thousands, except per share amounts)
            
     For the three months ended December 31, Year Ended December 31,
      2020   2019   2020   2019 
    Revenue:       
    Licensing revenue$250,000  $  $250,494  $ 
    Collaboration revenue       152   
    Total Revenue: 250,000      250,646    
    Operating expenses:       
    Research and development 50,337   78,471   299,921   220,969 
    License expense 25,050      25,050   - 
    General and administrative 44,492   27,469   139,401   67,046 
    Operating expenses: 119,879   105,940   464,372   288,015 
    Operating loss 130,121   (105,940)  (213,726)  (288,015)
    Loss on extinguishment of debt          (1,501)
    Loss from remeasurement of development derivative liability (40,090)  (4,736)  (103,029)  (14,839)
    Interest income 194   1,478   4,164   5,108 
    Interest expense (9,610)  (3,930)  (29,937)  (5,285)
    Other income (expense), net (505)  (89)  (501)  (175)
    Net loss before taxes 80,110   (113,217)  (343,029)  (304,707)
    Income tax expense 1,845      1,845   - 
    Net income/(loss) 78,265   (113,217)  (344,874)  (304,707)
    Other comprehensive loss:       
    Unrealized gain loss (130)     (8)   
      Foreign currency gain/(loss) 1,772   51   45   (31)
    Total other comprehensive loss 1,642   51   37   (31)
    Comprehensive income/(loss), net of tax$79,907  $(113,166) $(344,837) $(304,738)
    Net income/(loss) per common share, basic$1.03  $(1.77) $(4.59) $(4.90)
    Net income/(loss) per common share, diluted$0.93  $(1.77) $(4.59) $(4.90)
    Weighted-average number of common shares used in net loss per common share, basic 75,875   63,901   75,163   62,229 
    Weighted-average number of common shares used in net loss per common share, diluted 94,321   63,901   75,163   62,229 
            


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  9. WALTHAM, Mass., Feb. 22, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company will participate in the following investor conferences in March:

    • Raymond James 42nd Annual Institutional Investors Conference: Fireside chat on Monday, March 1, 2021 at 9:10 a.m. ET.
    • Cowen 41st Annual Health Care Conference: New Drug Launches panel on Wednesday, March 3, 2021 at 10:20 a.m. ET.
    • Oppenheimer 31st Annual Healthcare Conference: Fireside chat on Wednesday, March 17, 2021 at 8:40 a.m. ET.

    The Raymond James and Oppenheimer Conference events will be available via live webcast from the "Events and Presentations" page of the "Investors and…

    WALTHAM, Mass., Feb. 22, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company will participate in the following investor conferences in March:

    • Raymond James 42nd Annual Institutional Investors Conference: Fireside chat on Monday, March 1, 2021 at 9:10 a.m. ET.

    • Cowen 41st Annual Health Care Conference: New Drug Launches panel on Wednesday, March 3, 2021 at 10:20 a.m. ET.

    • Oppenheimer 31st Annual Healthcare Conference: Fireside chat on Wednesday, March 17, 2021 at 8:40 a.m. ET.

    The Raymond James and Oppenheimer Conference events will be available via live webcast from the "Events and Presentations" page of the "Investors and Media" section of the company's website at https://investors.apellis.com/events-and-presentations. Replays of the webcasts will be available for 90 days following the event.

    About Apellis

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit www.apellis.com.

    Investor Contact:

    Argot Partners



    212.600.1902



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  10. WALTHAM, Mass., Feb. 18, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company will host a conference call and webcast to discuss its fourth quarter and full year 2020 financial results on Thursday, February 25, 2021 at 4:30 p.m. ET.

    The event will be available live by dialing (866) 774-0323 (Domestic) or (602) 563-8683 (International) and entering the conference ID # 6956712 or via webcast from the "Events and Presentations" page of the "Investors and Media" section of the company's website at https://investors.apellis.com/events-and-presentations. A replay of the webcast will be available for 90 days following the event…

    WALTHAM, Mass., Feb. 18, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company will host a conference call and webcast to discuss its fourth quarter and full year 2020 financial results on Thursday, February 25, 2021 at 4:30 p.m. ET.

    The event will be available live by dialing (866) 774-0323 (Domestic) or (602) 563-8683 (International) and entering the conference ID # 6956712 or via webcast from the "Events and Presentations" page of the "Investors and Media" section of the company's website at https://investors.apellis.com/events-and-presentations. A replay of the webcast will be available for 90 days following the event.

    About Apellis

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit www.apellis.com.

    Investor Contact:

    Argot Partners



    +1 212.600.1902



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  11. WALTHAM, Mass., Feb. 05, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company approved the grant of equity awards to fourteen new employees with grant date of February 1, 2021, as equity inducement awards outside of the company's 2017 Stock Incentive Plan (but under the terms of the 2021 Inducement Stock Incentive Plan) and material to the employees' acceptance of employment with the company. The equity awards were approved in accordance with Nasdaq Listing Rule 5635(c)(4).

    The employees received options to purchase 56,500 shares of Apellis common stock and 13,275 restricted stock units (RSUs). The options have an exercise…

    WALTHAM, Mass., Feb. 05, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company approved the grant of equity awards to fourteen new employees with grant date of February 1, 2021, as equity inducement awards outside of the company's 2017 Stock Incentive Plan (but under the terms of the 2021 Inducement Stock Incentive Plan) and material to the employees' acceptance of employment with the company. The equity awards were approved in accordance with Nasdaq Listing Rule 5635(c)(4).

    The employees received options to purchase 56,500 shares of Apellis common stock and 13,275 restricted stock units (RSUs). The options have an exercise price of $44.20, which is equal to the closing price of Apellis common stock on February 1, 2021, the grant date of the options. One-fourth of the shares underlying the employee options will vest on the one year anniversary of the grant date and thereafter 1/48th of the shares underlying the employee options will vest monthly, such that the shares underlying the options granted to the employees will be fully vested on the fourth anniversary of the grant date, subject to the employees' continued employment with Apellis on such vesting dates. Each RSU will vest as to 25% of the shares underlying the RSU award on the first anniversary of the grant date and as to an additional 25% of the shares underlying the RSU award annually thereafter, subject to each such employee's continued employment on each vesting date.

    About Apellis

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit www.apellis.com.

    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 the company's clinical trials will be fully enrolled and completed when anticipated; 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 will be indicative of results that will be generated in future clinical trials; whether pegcetacoplan will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the company's clinical trials will warrant regulatory submissions and whether pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, C3G, IC-MPGN, ALS or any other indication when expected or at all; 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 2, 2020 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.

    Investor Contact:

    Argot Partners



    +1 212.600.1902



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  12. WALTHAM, Mass., Jan. 26, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced the closing of its previously announced privately negotiated exchange transactions (the "Exchange Transactions") with certain holders of its 3.500% Convertible Senior Notes due 2026 issued in September 2019 (the "Notes"). In the Exchange Transactions, the holders exchanged approximately $126.1 million in aggregate principal amount of Notes and Apellis issued an aggregate of 3,906,869 shares of its common stock.

    The shares of Apellis' common stock issued in the exchanges were not registered under the Securities Act of 1933, as amended, or the securities laws…

    WALTHAM, Mass., Jan. 26, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced the closing of its previously announced privately negotiated exchange transactions (the "Exchange Transactions") with certain holders of its 3.500% Convertible Senior Notes due 2026 issued in September 2019 (the "Notes"). In the Exchange Transactions, the holders exchanged approximately $126.1 million in aggregate principal amount of Notes and Apellis issued an aggregate of 3,906,869 shares of its common stock.

    The shares of Apellis' common stock issued in the exchanges were not registered under the Securities Act of 1933, as amended, or the securities laws of any state or other jurisdiction, and may not be offered or sold in the United States absent registration or an applicable exemption from the registration requirements of the Securities Act and such other jurisdictions.

    This press release does not constitute an offer to sell or a solicitation to buy any of the securities described herein, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful.

    About Apellis

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology.

    Investor Contact:

    Argot Partners



    +1 212.600.1902

    Media:

    Tracy Vineis



    +1 617 420 4839



    Primary Logo

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  13. WALTHAM, Mass., Jan. 21, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company will host a virtual investor event titled Pegcetacoplan: Advancing the First Potential Treatment for Geographic Atrophy (GA) from 10:00 a.m. to noon ET on January 28, 2021. The virtual meeting will focus on the significant unmet need in GA and the company's intravitreal pegcetacoplan program, which is on track for Phase 3 GA results in the third quarter of 2021.

    Apellis' management team will be joined by the following leading retinal physicians:

    • Caroline R. Baumal, M.D., Professor of Ophthalmology, Tufts Medical Center, New England Eye Center…

    WALTHAM, Mass., Jan. 21, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company will host a virtual investor event titled Pegcetacoplan: Advancing the First Potential Treatment for Geographic Atrophy (GA) from 10:00 a.m. to noon ET on January 28, 2021. The virtual meeting will focus on the significant unmet need in GA and the company's intravitreal pegcetacoplan program, which is on track for Phase 3 GA results in the third quarter of 2021.

    Apellis' management team will be joined by the following leading retinal physicians:

    • Caroline R. Baumal, M.D., Professor of Ophthalmology, Tufts Medical Center, New England Eye Center, Retina Department
    • Nancy M. Holekamp, M.D., Director, Retina Services, Pepose Vision Institute
    • Jeffrey S. Heier, M.D., Director, Retina Service, Ophthalmic Consultants of Boston
    • Pearse A. Keane, M.D., Consultant Ophthalmologist, Moorfields Eye Hospital NHS Foundation Trust; Associate Professor, University College London

    The live webcast and slide presentation will be available from the "Events and Presentations" page of the "Investors and Media" section of the company's website at https://investors.apellis.com/events-and-presentations. To access the event via phone, please dial (833) 353-0413 (local) or (720) 405-3208 (international) at least 10 minutes prior to the start time and refer to conference ID 1162057. A replay of the webcast will be available for 90 days following the event.



    About Apellis

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit www.apellis.com.



    Forward-Looking Statements

    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 in respect of the expected closing of the exchanges. 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 the conditions for the closing of the exchanges will be satisfied 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 2, 2020 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.

    Investor Contact:

    Argot Partners



    +1 212.600.1902

    Media:

    Tracy Vineis



    +1 617 420 4839



    Primary Logo

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  14. WALTHAM, Mass., Jan. 07, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that it has entered into separate, privately negotiated exchange agreements with certain holders of its 3.500% Convertible Senior Notes due 2026 issued in September 2019 (the "Notes"). Under the terms of these exchange agreements, the holders have agreed to exchange with Apellis approximately $107.5 million in aggregate principal amount of Notes held by them for (i) 2,232,808 shares of Apellis' common stock, which is equal to 20.7792 shares per $1,000 principal amount of Notes exchanged plus (ii) an additional number of shares of Apellis' common stock per $1,000…

    WALTHAM, Mass., Jan. 07, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that it has entered into separate, privately negotiated exchange agreements with certain holders of its 3.500% Convertible Senior Notes due 2026 issued in September 2019 (the "Notes"). Under the terms of these exchange agreements, the holders have agreed to exchange with Apellis approximately $107.5 million in aggregate principal amount of Notes held by them for (i) 2,232,808 shares of Apellis' common stock, which is equal to 20.7792 shares per $1,000 principal amount of Notes exchanged plus (ii) an additional number of shares of Apellis' common stock per $1,000 principal amount of Notes exchanged equal to the quotient of (a) $544.07 divided by (b) the average of the daily volume-weighted average prices of Apellis' common stock over the ten consecutive trading days commencing on January 7, 2021. The exchange transactions are expected to close on January 25, 2021, subject to the satisfaction of customary closing conditions.

    The shares of Apellis' common stock issuable in the exchanges have not been registered under the Securities Act of 1933, as amended, or the securities laws of any state or other jurisdiction, and may not be offered or sold in the United States absent registration or an applicable exemption from the registration requirements of the Securities Act and such other jurisdictions.

    This press release does not constitute an offer to sell or a solicitation to buy any of the securities described herein, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful.

    About Apellis

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology.

    Forward-Looking Statements

    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 in respect of the expected closing of the exchanges. 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 the conditions for the closing of the exchanges will be satisfied 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 2, 2020 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:

    Tracy Vineis



    +1 617 420 4839

    Investor Contact:

    Argot Partners



    +1 212.600.1902



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  15. WALTHAM, Mass., Jan. 05, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company will present at the 39th Annual J.P. Morgan Healthcare Conference on Tuesday, January 12, 2021 at 10:50 a.m. ET. The conference will be held in a virtual meeting format.

    The presentation will be given by Cedric Francois, M.D., Ph.D., co-founder and chief executive officer of Apellis, and will be available live via webcast from the "Events and Presentations" page of the "Investors and Media" section of the company's website at https://investors.apellis.com/events-and-presentations. A replay of the webcast will be available for 90 days following…

    WALTHAM, Mass., Jan. 05, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company will present at the 39th Annual J.P. Morgan Healthcare Conference on Tuesday, January 12, 2021 at 10:50 a.m. ET. The conference will be held in a virtual meeting format.

    The presentation will be given by Cedric Francois, M.D., Ph.D., co-founder and chief executive officer of Apellis, and will be available live via webcast from the "Events and Presentations" page of the "Investors and Media" section of the company's website at https://investors.apellis.com/events-and-presentations. A replay of the webcast will be available for 90 days following the event.

    About Apellis

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit www.apellis.com.

    Investor Contact:

    Argot Partners



    +1 212.600.1902



    Primary Logo

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  16. STOCKHOLM and WALTHAM, Mass., Dec. 10, 2020 /PRNewswire/ -- Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) and Apellis Pharmaceuticals Inc. (NASDAQ:APLS) today announced positive top-line results at week 48 from the phase 3 PEGASUS study, which demonstrated sustained haematological and clinical improvements in patients with paroxysmal nocturnal haemoglobinuria (PNH) who were treated with pegcetacoplan, an investigational, targeted C3 therapy. The safety profile of pegcetacoplan was consistent with previously reported data and no new safety signals were identified.

    All patients (n=77) who completed the 16-week randomized controlled period of the PEGASUS study, which evaluated pegcetacoplan compared to eculizumab, entered the open-label…

    STOCKHOLM and WALTHAM, Mass., Dec. 10, 2020 /PRNewswire/ -- Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) and Apellis Pharmaceuticals Inc. (NASDAQ:APLS) today announced positive top-line results at week 48 from the phase 3 PEGASUS study, which demonstrated sustained haematological and clinical improvements in patients with paroxysmal nocturnal haemoglobinuria (PNH) who were treated with pegcetacoplan, an investigational, targeted C3 therapy. The safety profile of pegcetacoplan was consistent with previously reported data and no new safety signals were identified.

    All patients (n=77) who completed the 16-week randomized controlled period of the PEGASUS study, which evaluated pegcetacoplan compared to eculizumab, entered the open-label period and received pegcetacoplan from week 17 to week 48.

    At week 48, haemoglobin increases were sustained in pegcetacoplan-treated patients with a mean improvement from baseline of 2.7 g/dL which is equal to the 2.7 g/dL mean increase seen at week 16 with pegcetacoplan-treated patients.

    Additionally, eculizumab-treated patients who switched to pegcetacoplan during the open-label period experienced sustained improvements in haemoglobin and other haematological and clinical measures, similar to patients treated with pegcetacoplan monotherapy during the randomized controlled period.

    "These long-term results show that pegcetacoplan has the potential to help patients with PNH gain and maintain more complete control of the disease," said Federico Grossi, M.D., Ph.D., chief medical officer of Apellis. "The sustained haematologic and quality-of-life improvements and consistent safety profile of pegcetacoplan observed in this study adds to a growing body of evidence that demonstrates the potential of this investigational, targeted C3 therapy to elevate the standard of care and improve the lives of people with PNH."

    In addition to a sustained improvement in haemoglobin, patients treated with pegcetacoplan maintained improvements across key secondary endpoints. Throughout the 48-week study, 73 per cent of patients treated with pegcetacoplan remained transfusion free. For comparison, 25 per cent of patients were transfusion free over the year prior to entering the PEGASUS study while on treatment with eculizumab. Improvements across additional markers of disease, such as reticulocyte count, lactate dehydrogenase (LDH) levels and the Functional Assessment of Chronic Illness Therapy (FACIT)-fatigue scores, were maintained.

    Overall, the safety profile of pegcetacoplan was consistent with previously reported data throughout the 48-week study. 24 of 80 pegcetacoplan monotherapy-treated patients (30 per cent) experienced a serious adverse event (SAE); five of the SAEs (6 per cent) were assessed to be possibly related to study treatment. No cases of meningitis were reported. One death was reported due to COVID-19 and was unrelated to study treatment. The most common adverse events (AEs) reported throughout the study were injection site reactions (36 per cent), haemolysis (24 per cent), and diarrhoea (21 per cent). 12 out of 80 patients (15 per cent) discontinued due to adverse events, with five discontinuations due to haemolysis. 64 of the 67 patients (96 per cent) who completed the open-label period opted to enter the extension study.

    "Despite existing treatments, many patients with PNH continue to suffer from persistently low haemoglobin, which can lead to a need for frequent transfusions and debilitating fatigue," said Ravi Rao, Head of R&D and Chief Medical Officer at Sobi. "The long-term data suggest that pegcetacoplan, if approved has the potential to provide meaningful and durable benefits to these patients with high unmet medical need."

    Marketing applications for pegcetacoplan for the treatment of PNH are under review by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). The FDA granted the application Priority Review designation and set a target action date of May 14, 2021. An opinion from the Committee for Medicinal Products for Human Use (CHMP) is expected in 2021.

    Detailed data will be presented at a future medical congress.

    About the PEGASUS Study

    The PEGASUS study (APL2-302; NCT03500549) is a multi-center, randomized, head-to-head phase 3 study in 80 adults with paroxysmal nocturnal haemoglobinuria (PNH). The primary objective of this study was to establish the efficacy and safety of pegcetacoplan compared to eculizumab. Participants must have been on eculizumab (stable for at least three months) with a haemoglobin level of <10.5 g/dL at the screening visit. During the four-week run-in, patients were dosed with 1080 mg of pegcetacoplan twice weekly (n=41) in addition to their current dose of eculizumab. During the 16-week randomized, controlled period, patients were randomized to receive either 1080 mg of pegcetacoplan twice weekly or their current dose of eculizumab (n=39). All participants completing the randomized controlled period (n=77) opted to enter the open-label pegcetacoplan treatment period. 

    About pegcetacoplan

    Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Pegcetacoplan is being evaluated in several clinical studies across haematology, ophthalmology, nephrology, and neurology. Marketing applications for pegcetacoplan for paroxysmal nocturnal haemoglobinuria (PNH) are under review by the U.S. Food and Drug Administration (FDA), which has granted the application Priority Review designation, and the European Medicines Agency (EMA). Pegcetacoplan was granted Fast Track designation by the FDA for the treatment of geographic atrophy, and received orphan drug designation for the treatment of C3 glomerulopathy by the FDA and EMA. For additional information regarding pegcetacoplan clinical studies, visit apellis.com/our-science/clinical-trials.

    About Paroxysmal Nocturnal Haemoglobinuria (PNH)

    PNH is a rare, chronic, life-threatening blood disorder characterized by the destruction of oxygen-carrying red blood cells through extravascular and intravascular haemolysis. Persistently low haemoglobin can result in frequent transfusions and debilitating symptoms such as severe fatigue, haemoglobinuria and difficulty breathing (dyspnea). A retrospective analysis shows that, even on eculizumab, approximately 72 per cent of people with PNH have anemia, a key indicator of ongoing haemolysis.1 The analysis also finds that 36 per cent of patients require one or more transfusions a year and 16 per cent require three or more.1

    About Apellis

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within haematology, ophthalmology, nephrology, and neurology. For more information, please visit www.apellis.com.

    About Sobi™

    Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare diseases. Sobi is providing sustainable access to innovative therapies in the areas of haematology, immunology and specialty indications. Today, Sobi employs approximately 1,500 people across Europe, North America, the Middle East, Russia and North Africa. In 2019, Sobi's revenues amounted to SEK 14.2 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. You can find more information about Sobi at www.sobi.com.

    This information is information that Swedish Orphan Biovitrum AB (publ) is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out below, at 12:00 CET on 10 December 2020.

    For more information please contact

    Sobi

    Paula Treutiger, Head of Communication & Investor Relations

    + 46 733 666 599

    Apellis

    Media

    Tracy Vineis



    +1 617 420 4839

    Investors

    Sam Martin / Maghan Meyers

    /

    +1 212 600 1902

    1.  McKinley C. Extravascular Hemolysis Due to C3-Loading in Patients with PNH Treated with Eculizumab: Defining the Clinical Syndrome. Blood. 2017;130:3471.

     

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    Sobi and Apellis report positive top-line results at 48 weeks from the phase 3 PEGASUS study of pegcetacoplan in PNH

     

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    • Treatment with pegcetacoplan resulted in a sustained improvement in hemoglobin with a mean increase from baseline of 2.7 g/dL at Week 48, which is equal to the 2.7 g/dL increase seen at Week 16 with pegcetacoplan-treated patients
    • Sustained improvements in transfusion avoidance, reticulocyte count, lactate dehydrogenase (LDH) level, and Functional Assessment of Chronic Illness Therapy (FACIT)-fatigue score were observed in patients treated with pegcetacoplan
    • Safety profile of pegcetacoplan was consistent with previously reported data

    WALTHAM, Mass. and STOCKHOLM, Sweden, Dec. 10, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) and Sobi™ Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) today announced positive top-line…

    • Treatment with pegcetacoplan resulted in a sustained improvement in hemoglobin with a mean increase from baseline of 2.7 g/dL at Week 48, which is equal to the 2.7 g/dL increase seen at Week 16 with pegcetacoplan-treated patients
    • Sustained improvements in transfusion avoidance, reticulocyte count, lactate dehydrogenase (LDH) level, and Functional Assessment of Chronic Illness Therapy (FACIT)-fatigue score were observed in patients treated with pegcetacoplan
    • Safety profile of pegcetacoplan was consistent with previously reported data

    WALTHAM, Mass. and STOCKHOLM, Sweden, Dec. 10, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) and Sobi™ Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) today announced positive top-line results at Week 48 from the Phase 3 PEGASUS study, which demonstrated sustained hematological and clinical improvements in patients with paroxysmal nocturnal hemoglobinuria (PNH) who were treated with pegcetacoplan, an investigational, targeted C3 therapy. The safety profile of pegcetacoplan was consistent with previously reported data, and no new safety signals were identified.

    All patients (n=77) who completed the 16-week randomized controlled period of the PEGASUS study, which evaluated pegcetacoplan compared to eculizumab, entered the open-label period and received pegcetacoplan from Week 17 to Week 48.

    At Week 48, hemoglobin increases were sustained in pegcetacoplan-treated patients with a mean improvement from baseline of 2.7 g/dL, which is equal to the 2.7 g/dL mean increase seen at Week 16 with pegcetacoplan-treated patients. Additionally, eculizumab-treated patients who switched to pegcetacoplan during the open-label period experienced sustained improvements in hemoglobin and other hematological and clinical measures, similar to patients treated with pegcetacoplan monotherapy during the randomized controlled period.

    "These long-term results show that pegcetacoplan has the potential to help patients with PNH gain and maintain more complete control of the disease," said Federico Grossi, M.D., Ph.D., chief medical officer of Apellis. "The sustained hematologic and quality-of-life improvements and consistent safety profile of pegcetacoplan observed in this study adds to a growing body of evidence that demonstrates the potential of this investigational, targeted C3 therapy to elevate the standard of care and improve the lives of people with PNH."

    In addition to a sustained improvement in hemoglobin, patients treated with pegcetacoplan maintained improvements across key secondary endpoints. Throughout the 48-week study, 73% of patients treated with pegcetacoplan remained transfusion free. For comparison, 25% of patients were transfusion free over the year prior to entering the PEGASUS study while on treatment with eculizumab. Improvements across additional markers of disease, such as reticulocyte count, lactate dehydrogenase (LDH) levels, and the Functional Assessment of Chronic Illness Therapy (FACIT)-fatigue scores, were maintained.

    Overall, the safety profile of pegcetacoplan was consistent with previously reported data throughout the 48-week study. Twenty-four of 80 pegcetacoplan monotherapy-treated patients (30%) experienced a serious adverse event (SAE); five of the SAEs (6%) were assessed to be possibly related to study treatment. No cases of meningitis were reported. One death was reported due to COVID-19 and was unrelated to study treatment. The most common adverse events (AEs) reported throughout the study were injection site reactions (36%), hemolysis (24%), and diarrhea (21%). Twelve out of 80 patients (15%) discontinued due to adverse events, with five discontinuations due to hemolysis. Sixty-four of the 67 patients (96%) who completed the open-label period opted to enter the extension study.

    "Despite existing treatments, many patients with PNH continue to suffer from persistently low hemoglobin, which can lead to a need for frequent transfusions and debilitating fatigue," said Ravi Rao, head of R&D and chief medical officer at Sobi. "The long-term data suggest that pegcetacoplan, if approved, has the potential to provide meaningful and durable benefits to these patients with high unmet medical need."

    Marketing applications for pegcetacoplan for the treatment of PNH are under review by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). The FDA granted the application Priority Review designation and set a target action date of May 14, 2021. An opinion from the Committee for Medicinal Products for Human Use (CHMP) is expected in 2021.

    Detailed data will be presented at a future medical congress.

    About the PEGASUS Study

    The PEGASUS study (APL2-302; NCT03500549) is a multi-center, randomized, head-to-head Phase 3 study in 80 adults with paroxysmal nocturnal hemoglobinuria (PNH). The primary objective of this study was to establish the efficacy and safety of pegcetacoplan compared to eculizumab. Participants must have been on eculizumab (stable for at least three months) with a hemoglobin level of <10.5 g/dL at the screening visit. During the four-week run-in, patients were dosed with 1080 mg of pegcetacoplan twice weekly (n=41) in addition to their current dose of eculizumab. During the 16-week randomized, controlled period, patients were randomized to receive either 1080 mg of pegcetacoplan twice weekly or their current dose of eculizumab (n=39). All participants completing the randomized controlled period (n=77) opted to enter the open-label pegcetacoplan treatment period.

    About Pegcetacoplan

    Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Pegcetacoplan is being evaluated in several clinical studies across hematology, ophthalmology, nephrology, and neurology. Marketing applications for pegcetacoplan for paroxysmal nocturnal hemoglobinuria (PNH) are under review by the U.S. Food and Drug Administration (FDA), which has granted the application Priority Review designation, and the European Medicines Agency (EMA). Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of geographic atrophy, and received orphan drug designation for the treatment of C3G by the FDA and European Medicines Agency. For additional information regarding pegcetacoplan clinical trials, visit https://apellis.com/our-science/clinical-trials.

    About Paroxysmal Nocturnal Hemoglobinuria (PNH) 

    PNH is a rare, chronic, life-threatening blood disorder characterized by the destruction of oxygen-carrying red blood cells through extravascular and intravascular hemolysis. Persistently low hemoglobin can result in frequent transfusions and debilitating symptoms such as severe fatigue, hemoglobinuria and difficulty breathing (dyspnea). A retrospective analysis shows that, even on eculizumab, approximately 72% of people with PNH have anemia, a key indicator of ongoing hemolysis.1 The analysis also finds that 36% of patients require one or more transfusions a year and 16% require three or more.1

    About Apellis 

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit http://apellis.com.

    About Sobi

    Sobi is a specialized international biopharmaceutical company transforming the lives of people with rare diseases. Sobi is providing sustainable access to innovative therapies in the areas of hematology, immunology and specialty indications. Today, Sobi employs approximately 1,500 people across Europe, North America, the Middle East, Russia and North Africa. In 2019, Sobi's revenue amounted to SEK 14.2 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. You can find more information about Sobi at www.sobi.com.

    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 the company's clinical trials will be fully enrolled and completed when anticipated; 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 will be indicative of results that will be generated in future clinical trials; whether pegcetacoplan will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the company's clinical trials will warrant regulatory submissions and whether pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, C3G, IC-MPGN, ALS or any other indication when expected or at all; 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 2, 2020 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.

    Contacts:



    Apellis

    Media:

    Lissa Pavluk 

     

    617.977.6764

    Investors: 

    Argot Partners



    +1 212.600.1902

    Sobi

    Paula Treutiger, Head of Communication & Investor Relations

    + 46 733 666 599





    Linda Holmström, Corporate Communication & Investor Relations

    + 46 708 734 095





    1. McKinley C. Extravascular Hemolysis Due to C3-Loading in Patients with PNH Treated with Eculizumab: Defining the Clinical Syndrome. Blood. 2017;130:3471.



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    • Data were presented at the American Society of Hematology (ASH) Annual Meeting

    • In an analysis from the 16-week Phase 3 PEGASUS study, 70.7% of patients treated with pegcetacoplan compared to 5.1% of eculizumab-treated patients achieved a good, major, or complete hematologic response based on published classifications

    • Quality-of-life substantially improved for pegcetacoplan-treated patients, reaching near-normal levels on several measures, and did not change for eculizumab-treated patients in a PEGASUS analysis at 16 weeks

    • A matching-adjusted indirect comparison suggests that 76% more patients on pegcetacoplan achieved hemoglobin stabilization and 71% more patients on pegcetacoplan were transfusion free compared to ravulizumab, a long-acting C5
    • Data were presented at the American Society of Hematology (ASH) Annual Meeting



    • In an analysis from the 16-week Phase 3 PEGASUS study, 70.7% of patients treated with pegcetacoplan compared to 5.1% of eculizumab-treated patients achieved a good, major, or complete hematologic response based on published classifications



    • Quality-of-life substantially improved for pegcetacoplan-treated patients, reaching near-normal levels on several measures, and did not change for eculizumab-treated patients in a PEGASUS analysis at 16 weeks



    • A matching-adjusted indirect comparison suggests that 76% more patients on pegcetacoplan achieved hemoglobin stabilization and 71% more patients on pegcetacoplan were transfusion free compared to ravulizumab, a long-acting C5 inhibitor

    WALTHAM, Mass., Dec. 07, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) today announced new analyses from the Phase 3 PEGASUS study, which demonstrated statistically significant improvements in treatment responses and substantial quality-of-life improvements with pegcetacoplan, an investigational, targeted C3 therapy, versus eculizumab, a C5 inhibitor, for paroxysmal nocturnal hemoglobinuria (PNH) at 16 weeks. Additionally, a matching-adjusted indirect comparison (MAIC) showed improvements in clinical, hematologic, and quality-of-life outcomes in patients treated with pegcetacoplan compared to ravulizumab, a long-acting C5 inhibitor. The data were presented at the virtual American Society of Hematology Annual Meeting (ASH) taking place December 5 - 8, 2020.

    Substantial Improvements in Hematologic Responses and Quality-of-Life Demonstrated in Patients Treated with Pegcetacoplan Compared to Eculizumab

    In an analysis of hematologic responses from the PEGASUS study, using response classifications developed independently by PNH experts that were published in Frontiers in Immunology,1 the findings showed:

    • Approximately 70.7% of pegcetacoplan-treated patients (29/41) compared to 5.1% of eculizumab-treated patients (2/39) achieved a good, major, or complete hematologic response (p<0.0001), which means the individual treated with pegcetacoplan reached a level of mild or no anemia and did not require transfusions.
    • 39% of patients treated with pegcetacoplan (16/41) compared to 0% of patients treated with eculizumab (0/39) achieved a complete response (p<0.0001), meaning they were transfusion free, experienced stable hemoglobin and lactate dehydrogenase (LDH) in the normal range, and showed no evidence of hemolysis based on LDH levels and reticulocyte count.

    In a PEGASUS analysis of quality-of-life:

    • Quality-of-life substantially improved for patients treated with pegcetacoplan, reaching near-normal levels on scales with a population norm, while no change was seen for eculizumab-treated patients. All scores were comparable at baseline across measures.

    "Many PNH patients treated with C5 inhibitors still suffer from a significant disease burden, including frequent transfusions and debilitating fatigue, which reinforces the urgent need for new treatments," said Brian Mulherin, M.D., PEGASUS study investigator and hematologist at St. Vincent Health Services. "The data presented at ASH show that pegcetacoplan, a targeted C3 therapy, demonstrate substantial and clinically meaningful improvements across a range of important hematologic measures compared to C5 inhibition."

    MAIC Found Greater Improvements in Hemoglobin Stabilization, Transfusion Avoidance, and Fatigue with Pegcetacoplan Versus Ravulizumab

    Using an MAIC methodology, individual patient data from the PEGASUS study were compared to aggregate, published results from the ALXN1210-PNH-302 study, which compared ravulizumab and eculizumab among patients with PNH who previously were treated with eculizumab.

    Detailed findings suggest:

    • 76% more patients treated with pegcetacoplan achieved hemoglobin stabilization compared to ravulizumab, a long-acting C5 inhibitor.
    • 71% more patients treated with pegcetacoplan were transfusion free compared to ravulizumab.
    • In mean change from baseline in the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue score, pegcetacoplan showed an adjusted difference of nine points versus ravulizumab, suggesting pegcetacoplan is associated with an improvement that greatly exceeds the three-point threshold generally considered to be clinically meaningful.

    In the absence of a clinical head-to-head study, MAIC is a valid and accepted method for comparative effectiveness research used by health technology assessment bodies across the world.2,3

    "Along with positive changes in hemoglobin, we are seeing major improvements in fatigue and other quality-of-life measures in patients treated with pegcetacoplan compared to C5 inhibition," said Federico Grossi, M.D., chief medical officer at Apellis. "These data show that the hematologic and clinical improvements demonstrated by pegcetacoplan have the potential to translate into meaningful outcomes for patients."

    Marketing applications for pegcetacoplan for the treatment of PNH were accepted by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). The target date for the FDA decision is May 14, 2021, and an opinion from the Committee for Medicinal Products for Human Use (CHMP) in the European Union (EU) is expected in 2021.

    About the PEGASUS Study

    The PEGASUS study (APL2-302; NCT03500549) is a multi-center, randomized, active-comparator controlled Phase 3 study in 80 adults with paroxysmal nocturnal hemoglobinuria (PNH). The primary objective of this study was to establish the efficacy and safety of pegcetacoplan compared to eculizumab. Participants must have been on eculizumab (stable for at least three months) with a hemoglobin level of <10.5 g/dL at the screening visit. During the four-week run-in, patients were dosed with 1080 mg of pegcetacoplan twice weekly (n=41) in addition to their current dose of eculizumab. During the 16-week randomized, controlled period, patients were randomized to receive either 1080 mg of pegcetacoplan twice weekly or their current dose of eculizumab (n=39). All participants completing the randomized controlled period (n=77) opted to enter the open-label pegcetacoplan treatment period.

    About the Matching-Adjusted Indirect Comparison (MAIC) Analysis

    Using a matching-adjusted indirect comparison (MAIC) methodology, individual patient data from the PEGASUS study were compared to aggregate, published results from the ALXN1210-PNH-302 study,4 which compared the C5 inhibitors ravulizumab and eculizumab among patients with PNH who were previously treated with eculizumab. To adjust for cross-study differences in baseline characteristics, propensity score weighting was used to balance demographic and clinical characteristics. Outcomes assessed from the PEGASUS study at week 16 and the ALXN1210-PNH-302 study at week 26 included transfusion avoidance, number of units transfused, hemoglobin stabilization, and change in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue score. As with other MAIC analyses, matching may not adjust for all confounding factors due to differences inherent in study design and entry criteria.

    About Pegcetacoplan

    Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Pegcetacoplan is being evaluated in several clinical studies across hematology, ophthalmology, nephrology, and neurology. Marketing applications for pegcetacoplan for paroxysmal nocturnal hemoglobinuria (PNH) are under review by the U.S. Food and Drug Administration (FDA), which has granted the application Priority Review designation, and the European Medicines Agency (EMA). Pegcetacoplan was also granted Fast Track designation by the FDA for the treatment of geographic atrophy and received orphan drug designation for the treatment of C3 glomerulopathy by the FDA and EMA. For additional information regarding pegcetacoplan clinical trials, visit https://apellis.com/our-science/clinical-trials.

    About Paroxysmal Nocturnal Hemoglobinuria (PNH) 

    PNH is a rare, chronic, life-threatening blood disorder characterized by the destruction of oxygen-carrying red blood cells through extravascular and intravascular hemolysis. Persistently low hemoglobin can result in frequent transfusions and debilitating symptoms such as severe fatigue, hemoglobinuria, and difficulty breathing (dyspnea). A retrospective analysis shows that, even on eculizumab, approximately 72% of people with PNH have anemia, a key indicator of ongoing hemolysis.5 The analysis also finds that 36% of patients require one or more transfusions a year and 16% require three or more.5

    About the Apellis and Sobi Collaboration

    Apellis and Sobi entered a collaboration to develop and commercialize systemic pegcetacoplan in October 2020. The companies have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-U.S. commercialization rights for systemic pegcetacoplan, and Apellis has exclusive U.S. commercialization rights for systemic pegcetacoplan and retains worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy (GA).

    About Apellis 

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit http://apellis.com.

    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 the company's clinical trials will be fully enrolled and completed when anticipated; 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 will be indicative of results that will be generated in future clinical trials; whether pegcetacoplan will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the company's clinical trials will warrant regulatory submissions and whether pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, C3G, IC-MPGN, ALS or any other indication when expected or at all; 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 2, 2020 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.

    Contacts:



    Media:

    Lissa Pavluk 

     

    617.977.6764

    Investors: 

    Argot Partners



    +1 212.600.1902



    1. Risitano AM, Marotta S, Ricci P, et al. Anti-complement treatment for paroxysmal nocturnal hemoglobinuria: time for proximal complement inhibition? A position paper from the SAAWP of the EBMT. Front Immunol. 2019;10:1157.

    2. Phillippo DM, Ades AE, Dias S, Palmer S, Abrams KR, and Welton NJ. Methods for Population-Adjusted Indirect Comparisons in Health Technology Appraisal. Medical Decision Making 2018;38(2): 200-211.

    3. Signorovitch JE, Sikirica V, Erder MH, Xie J, Lu M, Hodgkins PS, Betts KA, and Wu EQ. Matching-Adjusted Indirect Comparisons: A New Tool for Timely Comparative Effectiveness Research. Value in Health 2012;15(6):940-947.

    4. Kulasekararaj AG, Hill A, Rottinghaus ST, et al. Ravulizumab (ALXN1210) vs eculizumab in C5-inhibitor-experienced adult patients with PNH: the 302 study. Blood. 2019;133(6):540-549.

    5. McKinley C. Extravascular Hemolysis Due to C3-Loading in Patients with PNH Treated with Eculizumab: Defining the Clinical Syndrome. Blood. 2017;130:3471.



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  17. WALTHAM, Mass., Dec. 04, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company approved the grant of equity awards to two new employees with grant date of December 1, 2020, as equity inducement awards outside of the company's 2017 Stock Incentive Plan (but under the terms of the 2020 Inducement Stock Incentive Plan) and material to the employees' acceptance of employment with the company. The equity awards were approved on October 27, 2020 and November 16, 2020, in accordance with Nasdaq Listing Rule 5635(c)(4).

    The employees received options to purchase 13,400 shares of Apellis common stock and 6,700 restricted stock units…

    WALTHAM, Mass., Dec. 04, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company approved the grant of equity awards to two new employees with grant date of December 1, 2020, as equity inducement awards outside of the company's 2017 Stock Incentive Plan (but under the terms of the 2020 Inducement Stock Incentive Plan) and material to the employees' acceptance of employment with the company. The equity awards were approved on October 27, 2020 and November 16, 2020, in accordance with Nasdaq Listing Rule 5635(c)(4).

    The employees received options to purchase 13,400 shares of Apellis common stock and 6,700 restricted stock units (RSUs). The options have an exercise price of $46.82, which is equal to the closing price of Apellis common stock on December 1, 2020, the grant date of the options. One-fourth of the shares underlying the employee options will vest on the one year anniversary of the grant date and thereafter 1/48th of the shares underlying the employee options will vest monthly, such that the shares underlying the options granted to the employees will be fully vested on the fourth anniversary of the grant date, subject to the employees' continued employment with Apellis on such vesting dates.   Each RSU will vest as to 25% of the shares underlying the RSU award on the first anniversary of the grant date and as to an additional 25% of the shares underlying the RSU award annually thereafter, subject to each such employee's continued employment on each vesting date.

    About Apellis

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit www.apellis.com.

    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 the company's clinical trials will be fully enrolled and completed when anticipated; 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 will be indicative of results that will be generated in future clinical trials; whether pegcetacoplan will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the company's clinical trials will warrant regulatory submissions and whether pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, C3G, IC-MPGN, ALS or any other indication when expected or at all; 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 2, 2020 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.

    Investor Contact:

    Argot Partners



    +1 212.600.1902



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  18. WALTHAM, Mass., Nov. 30, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company will present at the 2020 Evercore ISI HealthCONx Conference on Thursday, December 3, 2020 at 1:25 p.m. ET. The conference will be held in a virtual meeting format.

    Cedric Francois, M.D., Ph.D., co-founder and chief executive officer of Apellis, will participate in a fireside chat. The event will be available live via webcast from the "Events and Presentations" page of the "Investors and Media" section of the company's website at https://investors.apellis.com/events-and-presentations. A replay of the webcast will be available for 90 days following…

    WALTHAM, Mass., Nov. 30, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company will present at the 2020 Evercore ISI HealthCONx Conference on Thursday, December 3, 2020 at 1:25 p.m. ET. The conference will be held in a virtual meeting format.

    Cedric Francois, M.D., Ph.D., co-founder and chief executive officer of Apellis, will participate in a fireside chat. The event will be available live via webcast from the "Events and Presentations" page of the "Investors and Media" section of the company's website at https://investors.apellis.com/events-and-presentations. A replay of the webcast will be available for 90 days following the event.

    About Apellis

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit www.apellis.com.

    Investor Contact:

    Argot Partners



    +1 212.600.1902



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  19. STOCKHOLM and WALTHAM, Mass., Nov. 19, 2020 /PRNewswire/ -- Swedish Orphan Biovitrum AB (publ) (SobiTM) (STO:SOBI) and Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) today announced that the first patient has been dosed in the potentially registrational phase 2 MERIDIAN study of pegcetacoplan, a targeted C3 therapy, in approximately 200 adults with sporadic amyotrophic lateral sclerosis (ALS).

    Studies suggest that elevated levels of C3 may play a role in the progression of ALS, a neurodegenerative disease that leads to progressive muscle weakness and paralysis. The MERIDIAN study will assess whether pegcetacoplan may offer a new treatment approach for people living with ALS by controlling complement activation at the level of C3. There are currently…

    STOCKHOLM and WALTHAM, Mass., Nov. 19, 2020 /PRNewswire/ -- Swedish Orphan Biovitrum AB (publ) (SobiTM) (STO:SOBI) and Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) today announced that the first patient has been dosed in the potentially registrational phase 2 MERIDIAN study of pegcetacoplan, a targeted C3 therapy, in approximately 200 adults with sporadic amyotrophic lateral sclerosis (ALS).

    Studies suggest that elevated levels of C3 may play a role in the progression of ALS, a neurodegenerative disease that leads to progressive muscle weakness and paralysis. The MERIDIAN study will assess whether pegcetacoplan may offer a new treatment approach for people living with ALS by controlling complement activation at the level of C3. There are currently no treatments to slow the advance of ALS.

    "ALS patients have a very high unmet need. They expect more and better treatment options," said Bashar Al-Nakhala, Chief Operations Officer of the ALS Therapeutic Development Institute. "We are pleased that Apellis and Sobi have joined the ALS clinical development community with our shared goal of halting the devastating progression of ALS."

    "We are delighted that the first patient in the phase 2 clinical study has been dosed as there is an urgency for a treatment for patients with ALS" said Ravi Rao, Head of R&D and Chief Medical Officer at Sobi. "In collaboration with Apellis, we look forward to evaluating the potential of pegcetacoplan in patients with ALS."

    "Based on the suspected role of C3 in ALS, we are working urgently to understand whether pegcetacoplan, a targeted C3 therapy, has the potential to slow disease progression and make a difference for the ALS community," said Federico Grossi, M.D., Ph.D, Chief Medical Officer of Apellis. "We designed the MERIDIAN study based on significant feedback from the community, and we are dedicated to continuing our partnership to one day bring a meaningful therapy to families living with ALS."

    The phase 2 MERIDIAN study (APL2-ALS-206) is a potentially registrational, randomized, double-blind, placebo-controlled, multicenter study designed to evaluate the efficacy and safety of pegcetacoplan in approximately 200 adults with sporadic ALS. Study participants will be randomized in a 2:1 ratio to receive pegcetacoplan or placebo while continuing to receive their existing standard of care treatment for ALS. After 52 weeks of blinded treatment, all patients in the study will receive pegcetacoplan. To reduce the burden on people living with ALS and their caregivers, the study has been designed to minimize the number of in-clinic visits, with approximately six clinic visits in the first year and four in the open-label second year.

    The primary endpoint of the study is the Combined Assessment of Function and Survival (CAFS) rank scores at week 52. Key secondary endpoints include measures of lung function, muscle strength, and quality of life. For more information about the phase 2 MERIDIAN study, visit www.clinicaltrials.gov (NCT04579666).

    About amyotrophic lateral sclerosis (ALS)

    ALS is a devastating neurodegenerative disease that results in progressive muscle weakness and paralysis due to the death of nerve cells, called motor neurons, in the brain and spinal cord.1,2 The death of motor neurons leads to the progressive loss of voluntary muscle movement required for speaking, walking, swallowing, and breathing.1,2 In individuals with ALS, high levels of C3 are present at the neuromuscular junction3 where motor neurons communicate directly to muscle cells. Numerous studies suggest that elevated levels of C3 present throughout the motor system of ALS patients are likely to contribute to chronic neuroinflammation and the death of motor neurons.3,4,5 There are currently no approved treatments that stop or reverse the progression of ALS, which impacts ~225,000 patients worldwide.6

    About pegcetacoplan (APL-2)

    Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Pegcetacoplan is being evaluated in several clinical studies across haematology, ophthalmology, nephrology, and neurology. Marketing applications for pegcetacoplan for paroxysmal nocturnal haemoglobinuria (PNH) are under review by the U.S. Food and Drug Administration (FDA), which has granted the application Priority Review designation, and the European Medicines Agency (EMA). Pegcetacoplan was also granted Fast Track designation by the FDA for the treatment of PNH and for the treatment of geographic atrophy and received orphan drug designation for the treatment of C3 glomerulopathy by the FDA and EMA. For additional information regarding pegcetacoplan clinical studies, visit apellis.com/our-science/clinical-trials.

    About Apellis

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within haematology, ophthalmology, nephrology, and neurology. For more information, please visit https://www.apellis.com.

    About Sobi™

    Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare diseases. Sobi is providing sustainable access to innovative therapies in the areas of haematology, immunology and specialty indications. Today, Sobi employs approximately 1,500 people across Europe, North America, the Middle East, Russia and North Africa. In 2019, Sobi's revenues amounted to SEK 14.2 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. You can find more information about Sobi www.sobi.com.

    For more information please contact

    Sobi

    Paula Treutiger, Head of Communication & Investor Relations

    + 46 733 666 599

    Linda Holmström, Corporate Communication & Investor Relations

    + 46 708 734 095

    Apellis

    Media

    Tracy Vineis



    +1 617 420 4839

    Investors

    Sam Martin / Maghan Meyers

    /

    +1 212 600 1902

    1 National Institute of Neurological Disorders and Stroke. (2020). Amyotrophic Lateral Sclerosis Fact Sheet. Retrieved from https://www.ninds.nih.gov/Disorders/Patient-Caregiver-Education/Fact-Sheets/Amyotrophic-lateral-Sclerosis-ALS-Fact-Sheet

    2 ALS Association. What is ALS? Retrieved June 2020 from https://www.als.org/understanding-als/what-is-als

    3 Bahia El Idrissi N, et al. J Neuroinflammation. 2016;13(1):72.4 Sta M, et al. Neurobiol Dis. 2011;42(3):211-220.

    4 Woodruff, et al.,  PNAS January 7, 2014 111 (1) E3-E4

    5 Lee, et al Journal of Neuroinflammation volume 15: 171 (2018)25 Arthur K et al. Nat Commun, 2016, Vol 7, article 12408

    6 Arthur K et al. Nat Commun, 2016, Vol 7, article 12408

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    https://news.cision.com/swedish-orphan-biovitrum-ab/r/sobi-and-apellis-announce-first-patient-dosed-in-potentially-registrational-als-study-of-pegcetacopl,c3240178

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    Sobi and Apellis announce first patient dosed in potentially registrational ALS study of pegcetacoplan

     

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    SOURCE Swedish Orphan Biovitrum AB

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    • 52-week Phase 2 MERIDIAN study to evaluate pegcetacoplan, a targeted C3 therapy, in approximately 200 adults with ALS

    WALTHAM, Mass. and STOCKHOLM, Sweden, Nov. 19, 2020 (GLOBE NEWSWIRE) --  Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) and Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) today announced that the first patient has been dosed in the potentially registrational Phase 2 MERIDIAN study of pegcetacoplan, a targeted C3 therapy, in approximately 200 adults with sporadic amyotrophic lateral sclerosis (ALS).

    Studies suggest that elevated levels of C3 may play a role in the progression of ALS, a neurodegenerative disease that leads to progressive muscle weakness and paralysis. The MERIDIAN study will assess whether pegcetacoplan may…

    • 52-week Phase 2 MERIDIAN study to evaluate pegcetacoplan, a targeted C3 therapy, in approximately 200 adults with ALS

    WALTHAM, Mass. and STOCKHOLM, Sweden, Nov. 19, 2020 (GLOBE NEWSWIRE) --  Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) and Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) today announced that the first patient has been dosed in the potentially registrational Phase 2 MERIDIAN study of pegcetacoplan, a targeted C3 therapy, in approximately 200 adults with sporadic amyotrophic lateral sclerosis (ALS).

    Studies suggest that elevated levels of C3 may play a role in the progression of ALS, a neurodegenerative disease that leads to progressive muscle weakness and paralysis. The MERIDIAN study will assess whether pegcetacoplan may offer a new treatment approach for people living with ALS by controlling complement activation at the level of C3. There are currently no treatments to slow the advance of ALS.

    "ALS patients have a very high unmet need. They expect more and better treatment options," said Bashar Al-Nakhala, chief operations officer of the ALS Therapeutic Development Institute. "We are pleased that Apellis and Sobi have joined the ALS clinical development community with our shared goal of halting the devastating progression of ALS."

    "Based on the suspected role of C3 in ALS, we are working urgently to understand whether pegcetacoplan, a targeted C3 therapy, has the potential to slow disease progression and make a difference for the ALS community," said Federico Grossi, M.D., Ph.D, chief medical officer of Apellis. "We designed the MERIDIAN study based on significant feedback from the community, and we are dedicated to continuing our partnership to one day bring a meaningful therapy to families living with ALS."

    "We are delighted that the first patient in the Phase 2 clinical study has been dosed as there is an urgency for a treatment for patients with ALS," said Ravi Rao, head of R&D and chief medical officer at Sobi. "In collaboration with Apellis, we look forward to evaluating the potential of pegcetacoplan in patients with ALS."

    The Phase 2 MERIDIAN study (APL2-ALS-206) is a potentially registrational, randomized, double-blind, placebo-controlled, multicenter study designed to evaluate the efficacy and safety of pegcetacoplan in approximately 200 adults with sporadic ALS. Study participants will be randomized in a 2:1 ratio to receive pegcetacoplan or placebo while continuing to receive their existing standard of care treatment for ALS. After 52 weeks of blinded treatment, all patients in the study will receive pegcetacoplan. To reduce the burden on people living with ALS and their caregivers, the study has been designed to minimize the number of in-clinic visits, with approximately six clinic visits in the first year and four in the open-label second year.

    The primary endpoint of the study is the Combined Assessment of Function and Survival (CAFS) rank scores at week 52. Key secondary endpoints include measures of lung function, muscle strength, and quality of life. For more information about the Phase 2 MERIDIAN study, visit https://www.clinicaltrials.gov (NCT04579666).

    About Amyotrophic Lateral Sclerosis (ALS)

    ALS is a devastating neurodegenerative disease that results in progressive muscle weakness and paralysis due to the death of nerve cells, called motor neurons, in the brain and spinal cord.1,2 The death of motor neurons leads to the progressive loss of voluntary muscle movement required for speaking, walking, swallowing, and breathing.1,2 In individuals with ALS, high levels of C3 are present at the neuromuscular junction3 where motor neurons communicate directly to muscle cells. Numerous studies suggest that elevated levels of C3 present throughout the motor system of ALS patients are likely to contribute to chronic neuroinflammation and the death of motor neurons.3,4,5 There are currently no approved treatments that stop or reverse the progression of ALS, which impacts ~225,000 patients worldwide.6

    About Pegcetacoplan (APL-2)

    Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Marketing applications for pegcetacoplan for paroxysmal nocturnal hemoglobinuria (PNH) are under review by the U.S. Food and Drug Administration (FDA), which has granted the application Priority Review designation, and the European Medicines Agency (EMA). Pegcetacoplan was also granted Fast Track designation by the FDA for the treatment of PNH and for the treatment of geographic atrophy and received orphan drug designation for the treatment of C3 glomerulopathy by the FDA and EMA. For additional information regarding pegcetacoplan clinical trials, visit https://apellis.com/our-science/clinical-trials.

    About Apellis

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit www.apellis.com.

    About Sobi™

    Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare diseases. Sobi is providing sustainable access to innovative therapies in the areas of hematology, immunology and specialty indications. Today, Sobi employs approximately 1,500 people across Europe, North America, the Middle East, Russia and North Africa. In 2019, Sobi's revenues amounted to SEK 14.2 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. You can find more information about Sobi at www.sobi.com.

    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 the company's clinical trials will be fully enrolled and completed when anticipated; 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 will be indicative of results that will be generated in future clinical trials; whether pegcetacoplan will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the company's clinical trials will warrant regulatory submissions and whether pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, C3G, IC-MPGN, ALS or any other indication when expected or at all; 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 2, 2020 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.

    For more information please contact

    Apellis

    Media

    Tracy Vineis



    +1 617 420 4839

    Investor Contact:

    Argot Partners



    +1 212.600.1902

    Sobi

    Paula Treutiger, Head of Communication & Investor Relations

    + 46 733 666 599

     

    Linda Holmström, Corporate Communication & Investor Relations

    + 46 708 734 095

    1 National Institute of Neurological Disorders and Stroke. (2020). Amyotrophic Lateral Sclerosis Fact Sheet. Retrieved from https://www.ninds.nih.gov/Disorders/Patient-Caregiver-Education/Fact-Sheets/Amyotrophic-lateral-Sclerosis-ALS-Fact-Sheet

    2 ALS Association. What is ALS? Retrieved June 2020 from https://www.als.org/understanding-als/what-is-als

    3 Bahia El Idrissi N, et al. J Neuroinflammation. 2016;13(1):72.4 Sta M, et al. Neurobiol Dis. 2011;42(3):211-220.

    4 Woodruff, et al., PNAS January 7, 2014 111 (1) E3-E4

    5 Lee, et al Journal of Neuroinflammation volume 15: 171 (2018)25 Arthur K et al. Nat Commun, 2016, Vol 7, article 12408

    6 Arthur K et al. Nat Commun, 2016, Vol 7, article 12408

     



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    • PDUFA target action date is May 14, 2021
    • FDA has stated that it is not currently planning to hold an advisory committee meeting to discuss the application
    • Pegcetacoplan demonstrated superiority to eculizumab in improving hemoglobin levels in Phase 3 PEGASUS head-to-head study as well as substantial improvements in other clinical measures
    • Apellis plans to open an early access program in the United States for pegcetacoplan for people living with PNH

    WALTHAM, Mass., Nov. 16, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review designation for the New Drug…

    • PDUFA target action date is May 14, 2021
    • FDA has stated that it is not currently planning to hold an advisory committee meeting to discuss the application
    • Pegcetacoplan demonstrated superiority to eculizumab in improving hemoglobin levels in Phase 3 PEGASUS head-to-head study as well as substantial improvements in other clinical measures
    • Apellis plans to open an early access program in the United States for pegcetacoplan for people living with PNH

    WALTHAM, Mass., Nov. 16, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review designation for the New Drug Application (NDA) for pegcetacoplan for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). The Prescription Drug User Fee Act (PDUFA) target action date is May 14, 2021. The FDA has stated that it is not currently planning to hold an advisory committee meeting to discuss the application.

    "For more than a decade, the only treatment options available for PNH have been C5 inhibitors, and many patients still suffer from persistently low hemoglobin, often resulting in debilitating fatigue and frequent transfusions. The NDA priority review takes us one step closer to bringing pegcetacoplan, a targeted C3 therapy with the potential to redefine PNH treatment, to patients in need," said Federico Grossi, M.D., Ph.D., chief medical officer of Apellis. "The data in the application validate the broad potential of targeting C3, and we continue to advance several registrational studies in serious diseases with few or no treatments." 

    The NDA submission is based on results from the head-to-head Phase 3 PEGASUS study, which met its primary endpoint, demonstrating the superiority of pegcetacoplan to eculizumab with a statistically significant improvement in hemoglobin levels at 16 weeks. The data also demonstrated higher normalization rates across key markers of hemolysis and a clinically meaningful improvement in Functional Assessment of Chronic Illness Therapy (FACIT)-fatigue score. The safety profile of pegcetacoplan was comparable to eculizumab in the study.

    Priority Review designation is granted to marketing applications for medicines that treat a serious condition and if approved, would provide a significant improvement in the safety or effectiveness of the treatment, prevention, or diagnosis of a serious condition. Pegcetacoplan was previously granted Fast Track designation by the FDA for the treatment of PNH.

    Apellis plans to open an early access program (EAP) in the United States for pegcetacoplan for patients with PNH who are experiencing ongoing disease activity despite treatment with C5 inhibition. The EAP will be available for a limited time while the FDA is reviewing the pegcetacoplan NDA. EAPs are potential pathways for patients with life-threatening or serious diseases to access investigational therapies outside of clinical trials when no comparable or satisfactory alternative therapy options are available. More information on the EAP is available at https://apellis.com/for-patients/early-access-program/.

    About the PEGASUS Study

    The PEGASUS study (APL2-302; NCT03500549) is a multi-center, randomized, active-comparator controlled Phase 3 study in 80 adults with paroxysmal nocturnal hemoglobinuria (PNH). The primary objective of this study was to establish the efficacy and safety of pegcetacoplan compared to eculizumab. Participants must have been on eculizumab (stable for at least three months) with a hemoglobin level of <10.5 g/dL at the screening visit. During the four-week run-in, patients were dosed with 1080 mg of pegcetacoplan twice weekly (n=41) in addition to their current dose of eculizumab. During the 16-week randomized, controlled period, patients were randomized to receive either 1080 mg of pegcetacoplan twice weekly or their current dose of eculizumab (n=39). All participants completing the randomized controlled period entered the open-label pegcetacoplan treatment period.

    The study was conducted in collaboration with SFJ Pharmaceuticals, who supported the development of pegcetacoplan in PNH. SFJ is a global drug development company, which provides a unique and highly customized co-development partnering model for the world's top pharmaceutical and biotechnology companies.

    About Pegcetacoplan (APL-2) 

    Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Apellis is evaluating pegcetacoplan in several clinical studies across hematology, ophthalmology, nephrology, and neurology. Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and the treatment of geographic atrophy, and received orphan drug designation for the treatment of C3G by the FDA and European Medicines Agency. For additional information regarding our clinical trials, visit https://apellis.com/our-science/clinical-trials.

    About Paroxysmal Nocturnal Hemoglobinuria (PNH) 

    PNH is a rare, chronic, life-threatening blood disorder characterized by the destruction of oxygen-carrying red blood cells through extravascular and intravascular hemolysis. Persistently low hemoglobin can result in frequent transfusions and debilitating symptoms such as severe fatigue, hemoglobinuria, and difficulty breathing (dyspnea). A retrospective analysis shows that, even on eculizumab, approximately 72% of people with PNH have anemia, a key indicator of ongoing hemolysis.1 The analysis also finds that 36% of patients require one or more transfusions a year and 16% require three or more.1

    About the Apellis and Sobi Collaboration

    Apellis and Sobi entered a collaboration to develop and commercialize systemic pegcetacoplan in October 2020. The companies have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-U.S. commercialization rights for systemic pegcetacoplan, and Apellis has exclusive U.S. commercialization rights for systemic pegcetacoplan and retains worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy (GA).

    About Apellis 

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, and nephrology. For more information, please visit http://apellis.com.

    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 the company's clinical trials will be fully enrolled and completed when anticipated; 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 will be indicative of results that will be generated in future clinical trials; whether pegcetacoplan will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the company's clinical trials will warrant regulatory submissions and whether pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, C3G, IC-MPGN, ALS or any other indication when expected or at all; 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 2, 2020 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: 

    Lissa Pavluk 

     

    617.977.6764

    Investor Contact:

    Argot Partners



    +1 212.600.1902



    1.  McKinley C. Extravascular Hemolysis Due to C3-Loading in Patients with PNH Treated with Eculizumab: Defining the Clinical Syndrome. Blood. 2017;130:3471.



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    • Verana Health analysis of American Academy of Ophthalmology IRIS® Registry presented in a late-breaking oral session

    • Real-world clinical data show significant disease progression over a two-year period in more than 69,000 patients with GA, highlighting the urgent need for treatment

    • Patients were nearly three times more likely to develop new onset wet age-related macular degeneration (AMD) in an eye with GA when wet AMD had already been detected in the contralateral eye

    WALTHAM, Mass., Nov. 13, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced findings from the largest retrospective database study in geographic atrophy (GA) secondary to…

    • Verana Health analysis of American Academy of Ophthalmology IRIS® Registry presented in a late-breaking oral session



    • Real-world clinical data show significant disease progression over a two-year period in more than 69,000 patients with GA, highlighting the urgent need for treatment



    • Patients were nearly three times more likely to develop new onset wet age-related macular degeneration (AMD) in an eye with GA when wet AMD had already been detected in the contralateral eye

    WALTHAM, Mass., Nov. 13, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced findings from the largest retrospective database study in geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The analysis of the American Academy of Ophthalmology (AAO) IRIS® (Intelligent Research in Sight) Registry, the nation's first comprehensive clinical registry for eye disease, was conducted in partnership with Verana Health, the world's leading data analysis group in retinal diseases. The study highlights the significant impact of GA progression on vision, underscoring the high unmet need for GA treatment in clinical practice. The data were presented today in a late-breaking oral session as part of the Retina Subspecialty Day at AAO 2020 Virtual.

    The retrospective study included more than 69,000 patients diagnosed with GA and analyzed changes in visual acuity and disease progression for over two years, as well as the occurrence of concurrent wet AMD. GA is a complement-driven eye disease1,2 that can lead to significant vision loss and affects approximately five million people around the world.3,4  

    "There is no approved therapy for GA and with new agents under development, it is essential to have a detailed understanding of disease progression in real-world clinical practice," said Ehsan Rahimy, M.D., lead study author and surgical and medical vitreoretinal specialist at the Palo Alto Medical Foundation. "The data show that GA patients at their first encounter have useful vision that may be preserved if an effective treatment were available. The progressive loss of visual acuity observed in this study over a two-year period underscores the urgent need for a therapy to slow disease progression." 

    Key findings from the real-world clinical data show: 

    • Progression from GA to new onset wet AMD was observed in 4.7% of patients with bilateral GA (GA in both eyes) and 13.3% of patients with wet AMD in the contralateral eye during the first 12 months. The rate at 24 months was 8.2% and 21.6% in bilateral GA and wet AMD in the contralateral eye, respectively.



    • At the first study visit, patients presented with relatively preserved vision, especially in eyes with extrafoveal GA lesions (lesions outside the fovea, which is the central portion of the retina). However, patients with extrafoveal and foveal GA lesions progressively lost vision over time at a rate of approximately five letters per year. 



    • A large proportion of GA patients did not return for a follow-up visit after two years. Of the GA patients potentially eligible for inclusion in the analysis, only 40% had a follow-up visit after two years and were ultimately included in the study.

    "As we work to develop the first potential medicine for people with GA, we are committed to improving understanding of the disease. Our collaboration with Verana Health and the AAO IRIS® Registry shows that wet AMD is not a rare occurrence in GA patients, and there is an opportunity to preserve vision if new treatments become available," said Federico Grossi, M.D., Ph.D., chief medical officer of Apellis. "These results highlight the significant unmet need in GA, and we are working urgently to advance pegcetacoplan, a targeted C3 therapy, in two ongoing Phase 3 GA studies." 

    About the IRIS® Registry

    The American Academy of Ophthalmology IRIS® (Intelligent Research in Sight) Registry is the nation's first electronic health record-based comprehensive eye disease and condition registry. As of September 2020, the registry features over 59.99 million unique patients and includes 16,030 clinicians. It is a centralized data repository and reporting tool that can analyze patient data to produce easy-to-interpret national and inter-practice benchmark reports and provide scientific information to improve public health. The reports can validate the quality of care ophthalmologists provide and pinpoint opportunities for improvement.

    About Verana Health

    Verana Health, Inc. partners with leading medical associations to transform clinical data into actionable real-world evidence. These partnerships enable Verana Health to harness the comprehensive data found in qualified clinical data registries and other specialty data sources to accelerate medical research and enhance patient care. Learn more at veranahealth.com.

    About Geographic Atrophy (GA)  

    GA is an advanced form of age-related macular degeneration (AMD), a leading cause of blindness. Excessive complement activation drives irreversible lesion growth in GA,5 and C3 is the only target to precisely control complement overactivation. Pegcetacoplan, studied in early and late-stage trials comprising a total of approximately 1,500 patients, is the only targeted C3 inhibitor being evaluated in late-stage trials to control lesion growth in GA.6 

    GA lesions affect the central portion of the retina, known as the macula, which is responsible for central vision. GA is progressive and irreversible, leading to central visual impairment and permanent loss of vision. Based on published studies, approximately one million people have GA in the United States and five million people have GA globally.2,7 There are currently no approved treatments for GA. 

    About Pegcetacoplan (APL-2)  

    Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Apellis is evaluating pegcetacoplan in several clinical studies across hematology, ophthalmology, nephrology, and neurology. Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and the treatment of geographic atrophy, and received orphan drug designation for the treatment of C3 glomerulopathy (C3G) by the FDA and European Medicines Agency. For additional information regarding our clinical trials, visit https://apellis.com/our-science/clinical-trials

    About Apellis  

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit http://apellis.com

    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 the company's clinical trials will be fully enrolled and completed when anticipated; 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 will be indicative of results that will be generated in future clinical trials; whether pegcetacoplan will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the company's clinical trials will warrant regulatory submissions and whether pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, C3G, IC-MPGN, ALS or any other indication when expected or at all; 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 2, 2020 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: 

    Mark Dole 

     

    +1 617 997 3484 

    Investor Contact: 

    Argot Partners 



    +1 212 600 1902 

    Weber, BHF, Issa, PC, et al. The Role of the Complement System in Age-Related Macular Degeneration.  

    Dtsch Arztebl Int 2014; 111(8): 133–8.  

    2 Heesterbeek, TJ, Lechanteur YTE, et al. Complement activation levels are related to disease stage in AMD. Invest Ophthalmol Vis Sci. 2020;61(3):18.  

    3 Rudnicka AR, Jarrar Z, Wormald R, et al. Age and gender variations in age-related macular degeneration prevalence in populations of European ancestry: a meta-analysis. Ophthalmology 2012;119:571–580. 

    4 Wong WL, Su X, Li X, et al. Global prevalence of age-related macular degeneration and disease burden projection for 2020 and 2040: a systematic review and meta-analysis. Lancet Glob Health 2014;2:e106–116. 

    5 Seddon, JM, Rosner, B. Validated prediction models for macular degeneration progression and predictors of visual acuity loss identify high-risk individuals. Am J Ophthalmol 2019;198:223–261.  

    6 Yates, JRW, Sepp T, et al. Complement C3 Variant and the Risk of Age-Related Macular Degeneration. N Engl J Med 2007; 357. 

    7 Weber, BHF, Issa, PC, et al. The Role of the Complement System in Age-Related Macular Degeneration. Dtsch Arztebl Int 2014; 111(8): 133–8. 

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  20. WALTHAM, Mass., Nov. 11, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company will participate in the upcoming investor conferences in November:

    • Stifel 2020 Virtual Healthcare Conference on Wednesday, November 18, 2020 at 10:00 a.m. ET
    • Jefferies Virtual London Healthcare Conference on Thursday, November 19, 2020 at 6:45 a.m. ET

    Cedric Francois, M.D., Ph.D., co-founder and chief executive officer of Apellis, will participate in a fireside chat at the Stifel 2020 Virtual Healthcare Conference and a podium presentation at the Jefferies Virtual London Healthcare Conference.

    The events will be available via live webcast from…

    WALTHAM, Mass., Nov. 11, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company will participate in the upcoming investor conferences in November:

    • Stifel 2020 Virtual Healthcare Conference on Wednesday, November 18, 2020 at 10:00 a.m. ET
    • Jefferies Virtual London Healthcare Conference on Thursday, November 19, 2020 at 6:45 a.m. ET

    Cedric Francois, M.D., Ph.D., co-founder and chief executive officer of Apellis, will participate in a fireside chat at the Stifel 2020 Virtual Healthcare Conference and a podium presentation at the Jefferies Virtual London Healthcare Conference.

    The events will be available via live webcast from the "Events and Presentations" page of the "Investors and Media" section of the company's website at https://investors.apellis.com/events-and-presentations. Replays of the webcasts will be available for 90 days following the events.

    About Apellis

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit http://apellis.com.

    Investor Contact:

    Sam Martin / Maghan Meyers

    Argot Partners

    /

    212.600.1902

     

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    • Eight abstracts accepted for presentation emphasize the potential of targeted C3 therapy to elevate the standard of care in paroxysmal nocturnal hemoglobinuria (PNH)
    • New analyses from the Phase 3 PEGASUS head-to-head study demonstrate greater treatment response and quality-of-life improvements with pegcetacoplan versus eculizumab, a C5 inhibitor

    • Using a matching-adjusted indirect comparison (MAIC), improvements in clinical, hematological and quality-of-life outcomes were demonstrated in patients treated with pegcetacoplan compared to ravulizumab, a long-acting C5 inhibitor

    WALTHAM, Mass., Nov. 05, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today…

    • Eight abstracts accepted for presentation emphasize the potential of targeted C3 therapy to elevate the standard of care in paroxysmal nocturnal hemoglobinuria (PNH)
    • New analyses from the Phase 3 PEGASUS head-to-head study demonstrate greater treatment response and quality-of-life improvements with pegcetacoplan versus eculizumab, a C5 inhibitor



    • Using a matching-adjusted indirect comparison (MAIC), improvements in clinical, hematological and quality-of-life outcomes were demonstrated in patients treated with pegcetacoplan compared to ravulizumab, a long-acting C5 inhibitor

    WALTHAM, Mass., Nov. 05, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that eight abstracts were accepted for presentation at the virtual American Society of Hematology (ASH) Annual Meeting to be held December 5-8, 2020. Data support the positive efficacy and safety of pegcetacoplan, a targeted C3 therapy, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).

    Highlights include new analyses from the Phase 3 head-to-head PEGASUS study, which demonstrate a markedly greater proportion of patients achieved better hematological responses as well as quality-of-life improvements with pegcetacoplan versus eculizumab, a C5 inhibitor. Additionally, using a matching-adjusted indirect comparison (MAIC) methodology, patients treated with pegcetacoplan enrolled in the PEGASUS study experienced greater improvements in hemoglobin stabilization, transfusion avoidance and fatigue compared to patients treated with ravulizumab, a long-acting C5 inhibitor, using the data from the ALXN1210-PNH-302 published study. In the absence of a clinical head-to-head study, MAIC is a valid and accepted method for comparative effectiveness research used by health technology assessment bodies across the world.1,2

    "We continue to see in the analyses that pegcetacoplan demonstrated a substantial improvement over C5 inhibition," said Federico Grossi, M.D., Ph.D., Chief Medical Officer of Apellis. "The breadth of data that we are presenting at ASH this year emphasizes the potential of pegcetacoplan to elevate the standard of care in PNH."

    Accepted abstracts regarding pegcetacoplan include:

    • Results of the PEGASUS Phase 3 Randomized Trial Demonstrating Superiority of the C3 Inhibitor, Pegcetacoplan, Compared to Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria – #2579 – December 7, 7:00 a.m. – 3:00 p.m. PT
    • Categorized Hematologic Response to Pegcetacoplan Versus Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria: Post Hoc Analysis of Data from a Phase 3 Randomized Trial (PEGASUS) – #2588 – December 7, 7:00 a.m. – 3:00 p.m. PT
    • Effect of Pegcetacoplan on Quality of Life in Patients with Paroxysmal Nocturnal Hemoglobinuria from the PEGASUS Phase 3 Trial Comparing Pegcetacoplan to Eculizumab – #764 – December 5, 7:00 a.m. – 3:30 p.m. PT
    • Pegcetacoplan is Superior to Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria Regardless of Prior Transfusion Requirement – #1681 – December 6, 7:00 a.m. – 3:30 p.m. PT
    • C3 Inhibition with Pegcetacoplan in Patients with Paroxysmal Nocturnal Hemoglobinuria: Results from the PADDOCK and PALOMINO Trials – #753 – December 5, 7:00 a.m. – 3:30 p.m. PT
    • Comparative Effectiveness of Pegcetacoplan Versus Ravulizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria Previously Treated with Eculizumab: A Matching-Adjusted Indirect Comparison – #2581 – December 7, 7:00 a.m. – 3:00 p.m. PT

    Accepted abstracts regarding additional data in the PNH population include:

    • Real-World Eculizumab Dosing Patterns among Patients with Paroxysmal Nocturnal Hemoglobinuria in a US Population – #3412 – December 7, 7:00 a.m. – 3:00 p.m. PT
    • Real-World Treatment Patterns and Healthcare Resource Utilization (HRU) of Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Receiving Eculizumab in a US Population – #3415 – December 7, 7:00 a.m. – 3:00 p.m. PT

    About the PEGASUS Study

    The PEGASUS study (APL2-302; NCT03500549) is a multi-center, randomized, active-comparator controlled Phase 3 study in 80 adults with paroxysmal nocturnal hemoglobinuria (PNH). The primary objective of this study was to establish the efficacy and safety of pegcetacoplan compared to eculizumab. Participants must have been on eculizumab (stable for at least three months) with a hemoglobin level of <10.5 g/dL at the screening visit. During the four-week run-in, patients were dosed with 1080 mg of pegcetacoplan twice weekly (n=41) in addition to their current dose of eculizumab. During the 16-week randomized, controlled period, patients were randomized to receive either 1080 mg of pegcetacoplan twice weekly or their current dose of eculizumab (n=39). All participants completing the randomized controlled period entered the open-label pegcetacoplan treatment period.

    About the Matching-Adjusted Indirect Comparison Analysis

    Using a matching-adjusted indirect comparison (MAIC) methodology, individual patient data from the PEGASUS study were compared to aggregate, published results from the ALXN1210-PNH-302 study, which compared ravulizumab and eculizumab among patients with PNH who previously were treated with eculizumab. To adjust for cross-study differences in baseline characteristics, propensity score weighting was used to balance demographic and clinical characteristics. Outcomes assessed from the PEGASUS study at week 16 and the ALXN1210-PNH-302 study at week 26 included transfusion avoidance, number of units transfused, hemoglobin stabilization and change in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue score. As with other MAIC analyses, matching may not adjust for all confounding factors due to differences inherent in study design and entry criteria.

    About Pegcetacoplan (APL-2) 

    Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Apellis is evaluating pegcetacoplan in several clinical studies across hematology, ophthalmology, nephrology, and neurology. Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and the treatment of geographic atrophy, and received orphan drug designation for the treatment of C3G by the FDA and European Medicines Agency. For additional information regarding our clinical trials, visit https://apellis.com/our-science/clinical-trials.



    About Paroxysmal Nocturnal Hemoglobinuria (PNH) 

    PNH is a rare, chronic, life-threatening blood disorder characterized by the destruction of oxygen-carrying red blood cells through extravascular and intravascular hemolysis. Persistently low hemoglobin can result in frequent transfusions and debilitating symptoms such as severe fatigue, hemoglobinuria and difficulty breathing (dyspnea). A retrospective analysis shows that, even on eculizumab, approximately 72% of people with PNH have anemia, a key indicator of ongoing hemolysis.3 The analysis also finds that 36% of patients require one or more transfusions a year and 16% require three or more.3

    About Apellis 

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit http://apellis.com.

    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 the company's clinical trials will be fully enrolled and completed when anticipated; 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 will be indicative of results that will be generated in future clinical trials; whether pegcetacoplan will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the company's clinical trials will warrant regulatory submissions and whether pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, C3G, IC-MPGN, ALS or any other indication when expected or at all; 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 2, 2020 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: 

    Lissa Pavluk 

      

    617.420.4839

    Investor Contact:  

    Sam Martin / Maghan Meyers 

    Argot Partners 

     /  

    212.600.1902



    1. Phillippo DM, Ades AE, Dias S, Palmer S, Abrams KR, and Welton NJ. Methods for Population-Adjusted Indirect Comparisons in Health Technology Appraisal. Medical Decision Making 2018;38(2): 200-211.

    2. Signorovitch JE, Sikirica V, Erder MH, Xie J, Lu M, Hodgkins PS, Betts KA, and Wu EQ. Matching-Adjusted Indirect Comparisons: A New Tool for Timely Comparative Effectiveness Research. Value in Health 2012;15(6):940-947.

    3. McKinley C. Extravascular Hemolysis Due to C3-Loading in Patients with PNH Treated with Eculizumab: Defining the Clinical Syndrome. Blood. 2017;130:3471.

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    • Strategic collaboration announced with Sobi for global co-development and exclusive ex-U.S. commercialization rights for systemic pegcetacoplan
    • Marketing authorization application (MAA) validated by EMA for pegcetacoplan in PNH. New drug application (NDA) filing decision by FDA expected in the fourth quarter of 2020
    • Late-breaking oral presentation at EURETINA demonstrated that intravitreal pegcetacoplan slowed progression of early disease in patients with geographic atrophy (GA) in post hoc analysis of Phase 2 FILLY study
    • Data in seven patients with bilateral GA showed a 52% decrease in mean lesion growth comparing intravitreal pegcetacoplan-treated eye versus untreated eye at 18 months (p=0.01) in post hoc analysis of Phase 1b study
    • Promising 48-week
    • Strategic collaboration announced with Sobi for global co-development and exclusive ex-U.S. commercialization rights for systemic pegcetacoplan
    • Marketing authorization application (MAA) validated by EMA for pegcetacoplan in PNH. New drug application (NDA) filing decision by FDA expected in the fourth quarter of 2020

    • Late-breaking oral presentation at EURETINA demonstrated that intravitreal pegcetacoplan slowed progression of early disease in patients with geographic atrophy (GA) in post hoc analysis of Phase 2 FILLY study
    • Data in seven patients with bilateral GA showed a 52% decrease in mean lesion growth comparing intravitreal pegcetacoplan-treated eye versus untreated eye at 18 months (p=0.01) in post hoc analysis of Phase 1b study
    • Promising 48-week Phase 2 DISCOVERY results demonstrated a 73.3% reduction in mean proteinuria in C3G patients treated with pegcetacoplan

    • Cash and investments of $728.2 million as of September 30, 2020 along with $250 million upfront and other payments from Sobi support cash runway into the second half of 2022

    WALTHAM, Mass., Nov. 02, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced its third quarter 2020 financial results and business highlights.

    "This has been another remarkable quarter for Apellis, culminating in our new collaboration with Sobi for global co-development and ex-U.S. commercialization of systemic pegcetacoplan for up to $1.25 billion in payments plus tiered royalties ranging from high teens to high twenties," said Cedric Francois, M.D., Ph.D., co-founder and chief executive officer of Apellis. "This collaboration allows us to more fully explore the pipeline-in-a-product opportunity of systemic pegcetacoplan while also putting us in a position of financial strength for the Phase 3 readout in geographic atrophy next year, a program for which we retain worldwide rights.

    "During the quarter, we also announced new data in several diseases including geographic atrophy, C3G, and severe COVID-19, further reinforcing the broad platform potential of our targeted C3 approach. Our team remains focused on delivering groundbreaking therapies for patients with a wide range of serious diseases, beginning with a potential U.S. launch in PNH."

    Business Highlights and Upcoming Milestones:

    Systemic Pegcetacoplan (APL-2)

    • In October 2020, Apellis announced a strategic collaboration with Swedish Orphan Biovitrum AB (publ) (Sobi) for the global co-development and exclusive ex-U.S. commercialization rights for systemic pegcetacoplan. Apellis retains U.S. commercialization rights for systemic pegcetacoplan and worldwide commercial rights for intravitreal pegcetacoplan. Under the terms of the agreement, Sobi will make an upfront payment to Apellis of $250 million and up to $915 million in other regulatory and commercial milestone payments and will contribute $80 million in committed reimbursement payments over a four-year period for research and development. Apellis will also be eligible for tiered double-digit royalties on sales ranging from high teens to high twenties. As part of the collaboration, the companies plan to start two new registrational programs in cold agglutinin disease (CAD) and hematopoietic stem cell transplantation-associated thrombotic microangiopathy (HSCT-TMA) in 2021.
    • In October 2020, Apellis presented 48-week data from the Phase 2 DISCOVERY study of pegcetacoplan in patients with C3G at the American Society of Nephrology (ASN) Kidney Week 2020. At week 48, pegcetacoplan demonstrated sustained improvements across key clinical measures including a 73.3% reduction in mean proteinuria, an important marker of kidney damage. No serious or severe adverse events were reported, and pegcetacoplan was well tolerated overall.
    • In October 2020, Apellis announced the initiation of registrational programs of pegcetacoplan in patients with immune complex membranoproliferative glomerulonephritis (IC-MPGN) or C3G and in patients with amyotrophic lateral sclerosis (ALS). The IC-MPGN / C3G registrational program consists of the recently initiated Phase 2 NOBLE study focused on the histopathology of the kidneys and a Phase 3 study that is expected to begin in the first half of 2021. The company expects the first patient to be dosed in the potentially registrational Phase 2 MERIDIAN study in ALS, Apellis' first neurological program, by the end of 2020.
    • In September 2020, Apellis announced the submission of an NDA to the U.S. Food and Drug Administration (FDA) and an MAA to the European Medicines Agency (EMA) for pegcetacoplan for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). The EMA validated the MAA in October 2020, and an opinion from the Committee for Medicinal Products for Human Use (CHMP) is expected in 2021. The FDA's decision on acceptance of the NDA and guidance on a potential Prescription Drug User Fee Act (PDUFA) target action date is expected in the fourth quarter of 2020. In October, Apellis submitted a marketing application for pegcetacoplan in PNH to the Australian Therapeutic Goods Administration (TGA). The TGA granted orphan drug designation to pegcetacoplan in PNH in September.
    • The company expects to report 48-week top-line results from the PEGASUS study by the end of the year and top-line results from the Phase 3 PRINCE study in patients with PNH who are treatment-naïve in the first half of 2021.

    Intravitreal Pegcetacoplan

    • In October 2020, Apellis announced 18-month data from the Phase 1b study of intravitreal pegcetacoplan in patients with advanced geographic atrophy (GA) and low vision. Data from a post hoc analysis demonstrated a 52% decrease in the growth rate of GA lesions in the treated eye compared to the untreated eye in seven patients with bilateral GA (disease in both eyes) at 18 months (p=0.01).
    • In October 2020, Apellis presented results of a post hoc analysis of the Phase 2 FILLY study at a late-breaking oral session at the European Society of Retina Specialists (EURETINA) 2020 Virtual Meeting. The data showed a 39% reduction in the rate of progression from nascent GA, an earlier form of disease, to GA in patients treated with pegcetacoplan monthly versus sham injections, demonstrating that intravitreal pegcetacoplan may slow early disease progression.
    • The company expects to announce top-line results from the Phase 3 DERBY and OAKS studies in the third quarter of 2021.

    COVID-19 Clinical Program

    • In October 2020, the company announced preliminary results from a Phase 1/2 clinical study of APL-9, a targeted C3 therapy designed for acute conditions, in patients with severe COVID-19. Preliminary open-label safety data from six patients with respiratory failure, including acute respiratory distress syndrome, supported advancement of the study. Apellis is currently enrolling an additional 60 patients into the randomized, double-blind, controlled phase of the study.
    • The company also announced data from an observational study in 41 patients hospitalized with COVID-19, which showed a correlation between increased complement activation and disease severity, supporting further evaluation of targeted C3 therapies for treating severe COVID-19.

    Third Quarter 2020 Financial Results:

    As of September 30, 2020, Apellis had $728.2 million in cash, cash equivalents, and short-term marketable securities, compared to $352 million in cash and cash equivalents as of December 31, 2019. This does not include the $250 million upfront proceeds from the Sobi transaction that was announced on October 27, 2020.

    Apellis reported a net loss of $135.7 million for the third quarter of 2020, compared to a net loss of $69.8 million for the third quarter of 2019.

    Research and development expenses were $93.2 million in the third quarter of 2020, compared to $51.3 million for the same period in 2019. The increase was primarily attributable to an increase of $18.0 million in contract manufacturing expenses in connection with the supply of pegcetacoplan for our Phase 3 clinical trials, an increase of $6.7 million in clinical trial costs associated with the continued enrollment of our Phase 3 clinical trials in PNH and GA, an increase of $9.9 million in personnel-related costs primarily due to the hiring of additional personnel, an increase of $7.8 million related to research and innovation activities and other development costs and offset by a decrease of $0.5 million in pre-clinical study expenses and device development expenses. We would expect our research and development expenses to continue to increase with the number of aggregate patients enrolled in our trials and as we may add to the number of ongoing trials for systemic pegcetacoplan.

    General and administrative expenses were $37.0 million in the third quarter of 2020, compared to $18.6 million for the same period in 2019. The increase was primarily attributable to an increase in employee-related costs of $8.2 million, an increase in professional and consulting fees and general commercial preparation activities of $9.7 million, an increase of $0.4 million in director stock compensation expense, and an increase in $0.3 million in insurance, offset by a decrease in general office costs and conference and travel-related expenses of $0.2 million. The increase in employee-related costs of $8.2 million consisted of a $5.7 million increase in salaries and benefits primarily due to the increase in the number of employees, $3.5 million related to stock expense associated with the grant of stock options and restricted stock units to employees offset by a decrease of $1.0 million in recruitment expense. The increase in other professional and consulting fees and general commercial preparation activities of $9.7 million primarily related to an increase in commercial-related activity of $6.3 million, a $2.6 million increase in legal and accounting fees, and an increase in general professional fees of $0.7 million and an increase in communication and public relations fees of $0.1 million.

    About Pegcetacoplan (APL-2)

    Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Apellis is evaluating pegcetacoplan in several clinical studies across hematology, ophthalmology, nephrology, and neurology. Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and the treatment of geographic atrophy, and received orphan drug designation for the treatment of C3 glomerulopathy (C3G) by the FDA and European Medicines Agency. For additional information regarding our clinical trials, visit https://apellis.com/our-science/clinical-trials.

    About APL-9

    APL-9 is an investigational, targeted C3 therapy designed to control the complement cascade centrally and may have the potential to treat a range of diseases caused by excessive activation of complement. APL-9 leverages the same mechanism of action as Apellis' lead compound, pegcetacoplan, but has a lower molecular weight and shorter half-life. APL-9 is designed to be intravenously administered for acute use.

    About Apellis

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit http://apellis.com.

    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 the company's clinical trials will be fully enrolled and completed when anticipated; 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 will be indicative of results that will be generated in future clinical trials; whether pegcetacoplan will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the company's clinical trials will warrant regulatory submissions and whether pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, C3G, IC-MPGN, ALS or any other indication when expected or at all; 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 2, 2020 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.



    Investor Contact:

    Sam Martin / Maghan Meyers

    Argot Partners

     / 

    212.600.1902

    Media Contact:

    Tracy Vineis



    617.420.4839







    APELLIS PHARMACEUTICALS, INC.   
    CONDENSED CONSOLIDATED BALANCE SHEETS

     
    (Amounts in thousands, except per share amounts)   
      September 30, December 31, 
       2020   2019  
    Assets (Unaudited)   
    Current assets:     
    Cash and cash equivalents $415,560  $351,985  
    Marketable Securities  312,598   -  
    Prepaid assets  11,299   19,802  
    Restricted Cash  1,255   -  
    Other current assets  1,637   1,308  
    Total current assets  742,349   373,095  
    Non-current Assets:     
    Right-of-use assets  18,545   14,110  
    Property and equipment, net  6,531   1,655  
    Other assets  909   385  
    Total assets $768,334  $389,245  
    Liabilities and Stockholders' Equity     
    Current liabilities:     
    Accounts payable $11,069  $8,361  
    Accrued expenses  60,766   54,783  
    Current portion of right of use liabilities  3,438   2,609  
    Total current liabilities  75,273   65,753  
    Long-term liabilities:     
    Convertible senior notes  353,769   142,567  
    Development derivative liability  217,778   134,839  
    Operating lease liabilities  15,674   11,857  
    Total liabilities  662,494   355,016  
    Stockholders' equity:     
    Preferred stock, $0.0001 par value; 10.0 million shares authorized, and zero shares issued and outstanding at September 30, 2020 and December 31, 2019  -   -  
    Common stock, $0.0001 par value; 200.0 million shares authorized at September 30, 2020 and December 31, 2019; 75.7 million shares issued and outstanding at June 30, 2020, and 63.9 million shares issued and outstanding at December 31, 2019  8   6  
    Additional paid in capital  1,112,203   615,850  
    Accumulated other comprehensive loss  (1,759)  (154) 
    Accumulated deficit  (1,004,612)  (581,473) 
    Total stockholders' equity  105,840   34,229  
    Total liabilities and stockholders' equity $768,334  $389,245  
          





    APELLIS PHARMACEUTICALS, INC.
    CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
            
     For the Three Months Ended September 30, For the Nine Months Ended September 30,
      2020   2019   2020   2019 
     (Unaudited)
    Revenue:       
    Licensing revenue$646  $  $646  $ 
    Total revenue 646      646    
    Operating expenses:       
    Research and development 93,207   51,319   249,584   142,497 
    General and administrative 36,991   18,629   94,909   39,578 
    Total operating expenses: 130,198   69,948   344,493   182,075 
    Net operating loss (129,552)  (69,948)  (343,847)  (182,075)
    Loss on extinguishment of debt    (293)     (1,501)
    Gain/(loss) from remeasurement of development derivative liability 2,697   (263)  (62,939)  (10,103)
    Interest income 670   1,342   3,970   3,630 
    Interest expense (9,499)  (602)  (20,327)  (1,354)
    Other income/(expense), net (16)  (61)  4   (86)
    Net loss (135,700)  (69,825)  (423,139)  (191,489)
    Other comprehensive loss:       
        Unrealized gain/(loss) on marketable securities (430)     122    
        Foreign currency loss (1,658)  (83)  (1,727)  (83)
    Total other comprehensive loss (2,088)  (83)  (1,605)  (83)
    Comprehensive loss, net of tax$(137,788) $(69,908) $(424,744) $(191,572)
    Net loss per common share, basic and diluted$(1.79) $(1.10) $(5.65) $(2.79)
    Weighted-average number of common shares used in net loss per common share, basic and diluted 75,628   63,753   74,925   68,737 
            

     







     

     

    Primary Logo

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    • Sobi obtains global co-development and exclusive ex-U.S. commercialization rights for systemic pegcetacoplan, a targeted C3 therapy
    • Apellis retains U.S. commercialization rights for systemic pegcetacoplan and worldwide commercialization rights for ophthalmological pegcetacoplan (geographic atrophy program in Phase 3)
    • The companies will jointly advance systemic pegcetacoplan in five parallel registrational programs including two new hematological studies planned to start in 2021 (CAD and HSCT-TMA). These join ongoing registrational programs in hematology (PNH), nephrology (IC-MPGN/C3G) and neurology (ALS)
    • Sobi will make an upfront payment of $250 million to Apellis and $80 million in committed development reimbursements over four years and up to
    • Sobi obtains global co-development and exclusive ex-U.S. commercialization rights for systemic pegcetacoplan, a targeted C3 therapy

    • Apellis retains U.S. commercialization rights for systemic pegcetacoplan and worldwide commercialization rights for ophthalmological pegcetacoplan (geographic atrophy program in Phase 3)
    • The companies will jointly advance systemic pegcetacoplan in five parallel registrational programs including two new hematological studies planned to start in 2021 (CAD and HSCT-TMA). These join ongoing registrational programs in hematology (PNH), nephrology (IC-MPGN/C3G) and neurology (ALS)
    • Sobi will make an upfront payment of $250 million to Apellis and $80 million in committed development reimbursements over four years and up to $915 million in regulatory and commercial milestones plus tiered double-digit royalties
    • Apellis to host conference call today at 8:30 a.m. ET

    WALTHAM, Mass. and STOCKHOLM, Sweden, Oct. 27, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) and Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) today announced a strategic collaboration to accelerate the advancement of systemic pegcetacoplan, a targeted C3 therapy, for the treatment of multiple rare diseases with high unmet need that impact more than 275,000 patients globally.

    Sobi will receive global co-development and exclusive ex-US commercialization rights for systemic pegcetacoplan. Apellis retains U.S. commercialization rights for systemic pegcetacoplan and worldwide commercial rights for ophthalmological pegcetacoplan, which is being evaluated by Apellis in two fully enrolled Phase 3 studies in geographic atrophy (GA). Pegcetacoplan targets excessive activation of C3 in the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases.

    Apellis and Sobi plan to jointly advance the clinical development of systemic pegcetacoplan in five parallel registrational programs across hematology, nephrology, and neurology. These include new registrational programs in cold agglutinin disease (CAD) and hematopoietic stem cell transplantation-associated thrombotic microangiopathy (HSCT-TMA), both of which are expected to start in 2021. By controlling complement activation centrally, pegcetacoplan offers the potential to become a transformative new therapy in several rare diseases where patients have few or no treatment options today.

    "This collaboration enables us to further expand on the broad platform potential of targeting C3 for serious rare diseases that impact hundreds of thousands of patients around the world," said Cedric Francois, M.D., Ph.D., co-founder and chief executive officer of Apellis. "We evaluated numerous companies, medium and large, and chose Sobi because of their global leadership in hematology and rare diseases, track record of successful product launches, and deep commitment to patients. Together, we will quickly advance systemic pegcetacoplan in multiple registrational programs across hematology, nephrology, and neurology while also preparing for our first potential U.S. launch in PNH. Financially, this transaction also strengthens our position, with our cash runway expected to extend into the second half of 2022."

    "We are excited to collaborate with Apellis, a leader in targeted C3 therapies. The collaboration will significantly strengthen and broaden our late-stage R&D portfolio and be a catalyst for further internationalization. The products have an excellent fit with our strategic focus on hematology and immunology," said Guido Oelkers, chief executive officer and president of Sobi. "Given the central role of C3 in the complement cascade, pegcetacoplan has the potential to become the foundation for a broader platform in rare diseases. With positive Phase 3 data in PNH, pegcetacoplan can elevate the standard of care for this debilitating blood disorder."

    As part of the collaboration, Apellis and Sobi will co-develop systemic pegcetacoplan in the following rare diseases:

    Hematology Paroxysmal nocturnal hemoglobinuria (PNH), CAD, and HSCT-TMA

    PNH represents the first potential indication to market for systemic pegcetacoplan. Marketing applications for pegcetacoplan for the treatment of PNH were submitted to the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) based on positive results from the Phase 3 PEGASUS study. Top-line results from the Phase 3 PRINCE study, which is evaluating pegcetacoplan in treatment-naïve patients with PNH, are expected in the first half of 2021.

    Sobi will lead development activities for the Phase 3 study in CAD and a potentially registrational Phase 2 study in HSCT-TMA, both planned to start in 2021.



    NephrologyImmune complex membranoproliferative glomerulonephritis (IC-MPGN) and C3 glomerulopathy (C3G)

    Apellis has initiated and will continue to lead a registrational program in IC-MPGN and C3G, which includes Phase 2 and Phase 3 studies.

    NeurologyAmyotrophic lateral sclerosis (ALS)

    Apellis has initiated and will continue to lead a potentially registrational Phase 2 study in ALS. Multiple other neurological conditions are under consideration for future clinical development.

    About the Transaction

    Sobi will make an upfront payment of $250 million to Apellis and up to $915 million in other regulatory and commercial milestone payments, and will contribute $80 million in reimbursement payments over a four-year period for research and development to support the initial development plan, which includes ongoing studies in PNH, IC-MPGN/C3G, and ALS and new studies in CAD and HSCT-TMA. Apellis will also be eligible for tiered double-digit royalties on sales ranging from high teens to high twenties. Sobi intends to finance these payments with available funds. Sobi will receive reimbursement payments for the costs incurred by Sobi in connection with the CAD and HSCT-TMA trials that Sobi will conduct. The parties have agreed to split costs 50/50 for any future global studies beyond the initial development plan.

    Per the terms of the agreement, Apellis will be responsible for all regulatory and commercial activities in the United States and the ongoing Marketing Authorization Application (MAA) review for PNH in the European Union, which will be subsequently transferred to Sobi. Sobi will be responsible for regulatory and commercial activities for systemic pegcetacoplan in ex-US markets. The co-development of systemic pegcetacoplan will be overseen by a joint development committee, and the commercial strategy will be overseen by a joint commercial committee.

    Conference Call and Webcast

    Apellis will host a conference call and webcast to discuss its collaboration with Sobi today, October 27, 2020, at 8:30 a.m. ET. To access the conference call, please dial (866) 774-0323 (local) or (602) 563-8683 (international) at least 10 minutes prior to the start time and refer to conference ID 5774165. A live audio webcast of the event and accompanying slides may also be accessed through the "Events and Presentations" page of the "Investors and Media" section of the company's website at http://investors.apellis.com/events-and-presentations. A replay of the webcast will be available for 30 days following the event.

    About Pegcetacoplan (APL-2)

    Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive complement activation, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Apellis is evaluating pegcetacoplan in several clinical studies across hematology, ophthalmology, nephrology, and neurology. Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of PNH and the treatment of geographic atrophy and received orphan drug designation for the treatment of C3G by the FDA and European Medicines Agency.

    About Pegcetacoplan for Paroxysmal Nocturnal Hemoglobinuria (PNH) 

    In October, the European Medicines Agency validated the Marketing Authorization Application (MAA) for pegcetacoplan in PNH, and an opinion from the Committee for Medicinal Products for Human Use is expected in 2021. A decision by the U.S. Food and Drug Administration regarding the acceptance of the New Drug Application (NDA) and a Prescription Drug User Fee Act (PDUFA) target action date is expected in the fourth quarter of 2020. Top-line results from the Phase 3 PRINCE study, which is evaluating pegcetacoplan in treatment-naïve patients with PNH, are expected in the first half of 2021.

    The NDA and MAA submissions for pegcetacoplan for the treatment of PNH are based on positive results from the Phase 3 PEGASUS study (APL2-302; NCT03500549), a multi-center, randomized, active-comparator controlled Phase 3 study in 80 adults with PNH. The primary objective of PEGASUS was to establish the efficacy and safety of pegcetacoplan compared to eculizumab. Pegcetacoplan is also being evaluated in the Phase 3 PRINCE study (APL2-308; NCT04085601), a randomized, multi-center, controlled study evaluating pegcetacoplan in 53 patients with PNH who had not received a complement inhibitor within three months before entering the study.

    About PNH

    PNH is a rare, chronic, life-threatening blood disorder characterized by the destruction of oxygen-carrying red blood cells through extravascular and intravascular hemolysis. Persistently low hemoglobin can result in frequent transfusions and debilitating symptoms such as severe fatigue, hemoglobinuria, and difficulty breathing (dyspnea). A retrospective analysis shows that, even on eculizumab, approximately 72% of people with PNH have anemia, a key indicator of ongoing hemolysis.1 The analysis also finds that 36% of patients require one or more transfusions a year and 16% require three or more.1



    About Cold Agglutinin Disease (CAD)

    CAD is a severe, chronic, rare blood disorder2 that currently has no approved therapies and impacts ~10,500 people across the United States and Europe.3 People living with CAD may suffer from chronic anemia, transfusion requirements, and an increased risk of life-threatening thrombotic events such as stroke.4 In people with CAD, immunoglobin M (IgM) autoantibodies cause red blood cells to agglutinate, or clump together, at temperatures below 30oC or as a result of a compromised immune system or infection.5 This activates the complement cascade to destroy healthy red blood cells through extravascular and intravascular hemolysis.6,7

    About Hematopoietic Stem Cell Transplantation Thrombotic Microangiopathy (HSCT-TMA)

    HSCT-TMA is rare blood disease that can be a fatal complication of a bone marrow transplant or HSCT.8 In HSCT-TMA, microscopic blood clots form in small blood vessels, leading to organ damage. The kidneys are commonly affected, although any organ may be involved.8 HSCT-TMA occurs in up to 40% of HSCT recipients;9 every year, there are ~9,000 allogeneic transplants in the United States and ~18,000 in the EU+.10,11 Excessive complement activation is a high-risk feature in patients with HSCT-TMA,12 and C3 is believed to play a critical role in TMA based on proinflammatory and procoagulant properties of C3a and C3b.13

    About Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN) and C3 Glomerulopathy (C3G)

    IC-MPGN and C3G are rare, debilitating kidney diseases that affect ~18,000 people in the United States and Europe.14 There are no approved therapies for the diseases, and symptoms include blood in the urine, dark foamy urine due to the presence of protein, swelling, and high blood pressure.15 Approximately 50% of people living with IC-MPGN and C3G ultimately suffer kidney failure within five to 10 years of diagnosis.16 Although IC-MPGN is considered a distinct disease from C3G, the underlying cause and progression of the two diseases are remarkably similar and include overactivation of the complement cascade, with excessive accumulation of C3 breakdown products in the kidney causing inflammation and damage to the organ. 17,18

    About Amyotrophic Lateral Sclerosis (ALS)

    ALS is a devastating neurodegenerative disease that results in progressive muscle weakness and paralysis due to the death of nerve cells, called motor neurons, in the brain and spinal cord.19, 20 The death of motor neurons leads to the progressive loss of voluntary muscle movement required for speaking, walking, swallowing and breathing.19,20 In individuals with ALS, high levels of C3 are present at the neuromuscular junction21 where motor neurons communicate directly to muscle cells. Numerous studies suggest that elevated levels of C3 present throughout the motor system of ALS patients are likely to contribute to chronic neuroinflammation and the death of motor neurons.21,22,23 There are no treatments that stop or reverse the progression of ALS, which impacts ~225,000 patients worldwide.24

    About Apellis

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit http://apellis.com.

    About Sobi

    Sobi is a specialized international biopharmaceutical company transforming the lives of people with rare diseases. Sobi is providing sustainable access to innovative therapies in the areas of hematology, immunology and specialty indications. Today, Sobi employs approximately 1,500 people across Europe, North America, the Middle East, Russia and North Africa. In 2019, Sobi's revenue amounted to SEK 14.2 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. You can find more information about Sobi at www.sobi.com.

    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 the company's clinical trials will be fully enrolled and completed when anticipated; 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 will be indicative of results that will be generated in future clinical trials; whether pegcetacoplan will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the company's clinical trials will warrant regulatory submissions and whether pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, C3G, IC-MPGN, ALS or any other indication when expected or at all; 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 July 30, 2020 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.

    Contacts:

    Apellis

    Media

    Tracy Vineis

     

    +1 617 420 4839



    Investors

    Sam Martin / Maghan Meyers

     /

    +1 212 600 1902

    Sobi

    Paula Treutiger, Head of Communication & Investor Relations

    + 46 733 666 599

    Linda Holmström, Corporate Communication & Investor Relations

    + 46 708 734 095

    Apellis

    1 McKinley C. Extravascular Hemolysis Due to C3-Loading in Patients with PNH Treated with Eculizumab: Defining the Clinical Syndrome. Blood. 2017;130:3471.

    2 Sokol RJ, Hewitt S, Stamps BK. Autoimmune haemolysis: an 18-year study of 865 cases referred to a regional transfusion centre. Br Med J (Clin Res Ed). 1981;282(6281):2023-2027.National Institute of Health (NIH), Genetic and Rare Diseases Information Center (GARD)

    3 Catenion using physician and literature consensus

    4 Website https://rarediseases.info.nih.gov/diseases/6130/cold-agglutinin-disease. Accessed November 21, 2019.

    5 Berentsen S, Ulvestad E, Langholm R, et al. Primary chronic cold agglutinin disease: a population based clinical study of 86 patients. Haematologica. 2006;91(4):460-466.

    6 Cold agglutinin disease. Genetic and Rare Diseases Information Center Web site. https://rarediseases.info.nih.gov/diseases/6130/cold-agglutinin-disease. Accessed November 21, 2019.

    7 Reynaud Q, Durieu I, Dutertre M, et al. Efficacy and safety of rituximab in auto-immune hemolytic anemia: A meta-analysis of 21 studies. Autoimmun Rev. 2015;14(4):304-313.

    8 Dvorak C, et al. Transplant-Associated Thrombotic Microangiopathy in Pediatric Hematopoietic Cell Transplant Recipients: A Practical Approach to Diagnosis and Management. Frontiers in Pediatrics. Vol 7, article 133. (2019)

    9 Jodele S, et al. Diagnostic and risk criteria for HSCT-associated thrombotic microangiopathy: a study in children and young adults. Blood. 124(4): 645–653 (2014)

    10 Current Uses and Outcomes of Hematopoietic Cell Transplantation (HCT): CIBMTR Summary Slides

    11 Passweg et al, BMT. 2019, 38: 1575–1585

    12 Jodele S, et al. Complement blockade for TA-TMA: lessons learned from a large pediatric cohort treated with eculizumab. Blood. 135 (13): 1049–1057. (2020)

    13 Noris M, et al. STEC-HUS, atypical HUS and TTP are all diseases of complement activation. Nature Reviews Nephrology. 8, 622–633 (2012)

    14 ClearView Analysis using physician and literature consensus.

    15 Complement 3 Glomerulopathy (C3G). National Kidney Foundation Website. https://www.kidney.org/atoz/content/complement-3-glomerulopathy-c3g. Accessed November 21, 2019.

    16 C3 glomerulopathy. National Institute of Health, Genetics Home Reference. https://ghr.nlm.nih.gov/condition/c3-glomerulopathy#resources. Accessed November 21, 2019.

    17 Noris M, Donadelli R, Remuzzi G. Autoimmune abnormalities of the alternative complement pathway in membranoproliferative glomerulonephritis and C3 glomerulopathy. Pediatr Nephrol. 2019 Aug;34(8):1311-1323.

    18 Cook HT. Evolving complexity of complement-related diseases: C3 glomerulopathy and atypical haemolytic uremic syndrome. Curr Opin Nephrol Hypertens. 2018 May;27(3):165-170.

    19 National Institute of Neurological Disorders and Stroke. (2020). Amyotrophic Lateral Sclerosis Fact Sheet. Retrieved from https://www.ninds.nih.gov/Disorders/Patient-Caregiver-Education/Fact-Sheets/Amyotrophic-lateral-Sclerosis-ALS-Fact-Sheet

    20 ALS Association. What is ALS? Retrieved June 2020 from https://www.als.org/understanding-als/what-is-als

    21 Bahia El Idrissi N, et al. J Neuroinflammation. 2016;13(1):72.4 Sta M, et al. Neurobiol Dis. 2011;42(3):211-220.

    22 Woodruff, et al.,  PNAS January 7, 2014 111 (1) E3-E4

    23 Lee, et al Journal of Neuroinflammation volume 15: 171 (2018)25 Arthur K et al. Nat Commun, 2016, Vol 7, article 12408

    24 Arthur K et al. Nat Commun, 2016, Vol 7, article 12408

    Primary Logo

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    • First data for 18 months of treatment with intravitreal pegcetacoplan demonstrates continued slowing of GA lesion growth beyond 12 months

    • Post hoc analysis demonstrated a 52% decrease in mean lesion growth in seven patients with bilateral GA comparing treated eye vs. untreated fellow eye at 18 months (p=0.01)

    • Top-line data from Phase 3 DERBY and OAKS studies expected in Q3 2021

    WALTHAM, Mass., Oct. 19, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced 18-month data from the Phase 1b APL2-103 study of pegcetacoplan (APL-2) in patients with advanced geographic atrophy (GA) secondary to age-related macular degeneration (AMD) and low vision…

    • First data for 18 months of treatment with intravitreal pegcetacoplan demonstrates continued slowing of GA lesion growth beyond 12 months



    • Post hoc analysis demonstrated a 52% decrease in mean lesion growth in seven patients with bilateral GA comparing treated eye vs. untreated fellow eye at 18 months (p=0.01)



    • Top-line data from Phase 3 DERBY and OAKS studies expected in Q3 2021



    WALTHAM, Mass., Oct. 19, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced 18-month data from the Phase 1b APL2-103 study of pegcetacoplan (APL-2) in patients with advanced geographic atrophy (GA) secondary to age-related macular degeneration (AMD) and low vision. GA is a complement-driven eye disease1,2 that can cause blindness, affects approximately five million people globally3,4 and has no approved treatment. The study, which enrolled 12 patients with bilateral GA (disease in both eyes), was initiated to assess the safety of the Phase 3 formulation of pegcetacoplan (15mg/0.1mL).

    Patients were dosed monthly with pegcetacoplan in one eye using the fellow eye as an untreated control. Apellis previously reported results at Month 12 in nine patients demonstrating a trend in reduced GA lesion growth in treated eyes versus untreated fellow eyes. The current post hoc analysis reports on the seven Phase 1b study patients for whom data were available for at least 18 months. In this population, the growth rate of GA lesions in the treated eye was on average 52% (mean square root) slower than the opposite untreated eye (p=0.01). It has been shown that lesions in both eyes tend to grow at the same rate in patients with bilateral GA.5 Of the 12 enrolled patients, there were no reported cases of inflammation and, as previously reported, one patient (8%) developed new-onset exudation at Month 12.

    "It is exciting to see data for the efficacy of intravitreal pegcetacoplan at 18 months among patients with GA," said Charles Wykoff, M.D., Ph.D., Director of Research, Retina Consultants of Houston, and Investigator of Apellis' Phase 1b and Phase 3 GA trials. "These data align with the Phase 2 FILLY results, where patients with bilateral GA in the monthly treated population had a significant reduction in growth relative to their untreated fellow eye in a post hoc analysis at 12 months. Finally, while this is a limited number of patients, I am encouraged to see that the benefit of pegcetacoplan in slowing GA growth seems to be maintained through Month 18. I believe that a 52% reduction in GA lesion growth at Month 18 is likely to be highly clinically meaningful."

    Post hoc analysis at 18 months

    Figure 1. Mean (± SE) change from baseline in square root GA lesion measured by fundus autofluorescence in the study eye (SE) and fellow eye (FE). Percentage difference and p value represents the comparison in GA growth between the study and fellow eye at each timepoint.



    The ongoing pegcetacoplan development program in GA includes the Phase 1b APL2-103 study and the Phase 3 DERBY and OAKS studies. The patient population enrolled in the Phase 1b study is similar to DERBY and OAKS but allowed for more advanced disease with a wider range of baseline lesion size and lower baseline visual acuity.

    The DERBY and OAKS studies were initiated in 2019 with the pegcetacoplan formulation tested in this Phase 1b study and top-line data are expected in the third quarter of 2021.

    About the APL2-103 study

    The APL2-103 study is a 12-patient Phase 1b, multicenter, open label, single arm, 24-month clinical trial to assess the safety of monthly intravitreal (IVT) injections of pegcetacoplan in patients diagnosed with advanced geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The primary outcome measures include incidence and severity of ocular and systemic treatment-emergent adverse events (TEAEs).

    About the FILLY study

    The FILLY study was a 246-patient, Phase 2, multicenter, randomized, single-masked, sham-controlled clinical trial evaluating pegcetacoplan in patients with GA secondary to AMD conducted at over 40 clinical sites in the United States, Australia and New Zealand. Pegcetacoplan was administered as an intravitreal injection monthly or every other month (EOM) for 12 months, followed by six months of monitoring after the end of treatment. The primary efficacy endpoint was the change in GA lesion area from baseline to Month 12 in pegcetacoplan-treated patients compared to sham.

    About the DERBY and OAKS studies

    The DERBY and OAKS studies are 600-patient prospective, international, multicenter, randomized, double-masked, sham-injection controlled Phase 3 studies assessing the efficacy and safety of multiple IVT injections of pegcetacoplan in patients with GA secondary to AMD. For more information, please visit https://gastudy.com/.

    About Pegcetacoplan (APL-2)

    Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive complement activation, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Apellis is evaluating pegcetacoplan in several clinical studies across hematology, ophthalmology, nephrology, and neurology. Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and the treatment of geographic atrophy, and received orphan drug designation for the treatment of C3 glomerulopathy by the FDA and European Medicines Agency. For additional information regarding our clinical trials, visit https://apellis.com/our-science/clinical-trials.

    About Geographic Atrophy (GA) 

    GA is an advanced form of age-related macular degeneration (AMD), a leading cause of blindness. Excessive complement activation drives irreversible lesion growth in GA,6 and C3 is the only target to precisely control complement overactivation. Pegcetacoplan, studied in early and late-stage trials comprising a total of approximately 1,500 patients, is the only targeted C3 inhibitor being evaluated in late-stage trials to control lesion growth in GA.7

    GA lesions affect the central portion of the retina, known as the macula, which is responsible for central vision. GA is progressive and irreversible, leading to central visual impairment and permanent loss of vision. Based on published studies, approximately one million people have GA in the United States and five million people have GA globally.1,2 There are currently no approved treatments for GA.

    About Apellis 

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop best-in-class and first-in-class therapies for a broad range of debilitating diseases that are driven by uncontrolled or excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit http://apellis.com.

    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 the company's clinical trials will be fully enrolled and completed when anticipated; 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 will be indicative of results that will be generated in future clinical trials; whether pegcetacoplan will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the company's clinical trials will warrant regulatory submissions and whether pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, C3G, IC-MPGN, ALS, or severe COVID-19 or any other indication when expected or at all; 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 July 30, 2020 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:

    Mark Dole

     

    617.997.3484

    Investor Contact: 

    Sam Martin / Maghan Meyers

    Argot Partners

     / 

    212.600.1902

    ________________________________________________________________________________________________________________________________

    1 Weber, BHF, Issa, PC, et al. The Role of the Complement System in Age-Related Macular Degeneration. Dtsch Arztebl Int 2014; 111(8): 133–8. 



    2 Heesterbeek, TJ, Lechanteur YTE, et al. Complement activation levels are related to disease stage in AMD. Invest Ophthalmol Vis Sci. 2020; 61(3): 18. 



    3 Rudnicka AR, Jarrar Z, Wormald R, et al. Age and gender variations in age-related macular degeneration prevalence in populations of European ancestry: a meta-analysis. Ophthalmology 2012; 119:571–580.



    4 Wong WL, Su X, Li X, et al. Global prevalence of age-related macular degeneration and disease burden projection for 2020 and 2040: a systematic review and meta-analysis. Lancet Glob Health 2014; 2:e106–116.

    5 Sunness JS, et al. The long-term natural history of geographic atrophy from age-related macular degeneration: enlargement of atrophy and implications for interventional clinical trials. Ophthalmology. 2007 Feb; 114(2):271-7.

    6 Seddon, JM, Rosner, B. Validated prediction models for macular degeneration progression and predictors of visual acuity loss identify high-risk individuals. Am J Ophthalmol 2019;198:223–261. 

    7 Yates, JRW, Sepp T, et al. Complement C3 Variant and the Risk of Age-Related Macular Degeneration. N Engl J Med 2007; 357.

     

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    • Observational study in 41 patients hospitalized with COVID-19 found that nearly all patients had elevated systemic levels of C3a, a marker for C3 activation; median C3a levels were 3.7 times the upper limit of normal
    • In a Phase 1/2 interventional study, preliminary open-label safety results in first six patients support advancement of APL-9, an investigational targeted C3 therapy, for severe COVID-19; additional 60-patient randomized, double-blind, controlled study cohort is currently enrolling

    WALTHAM, Mass., Oct. 15, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced observational study results that found a correlation between COVID-19…

    • Observational study in 41 patients hospitalized with COVID-19 found that nearly all patients had elevated systemic levels of C3a, a marker for C3 activation; median C3a levels were 3.7 times the upper limit of normal
    • In a Phase 1/2 interventional study, preliminary open-label safety results in first six patients support advancement of APL-9, an investigational targeted C3 therapy, for severe COVID-19; additional 60-patient randomized, double-blind, controlled study cohort is currently enrolling

    WALTHAM, Mass., Oct. 15, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced observational study results that found a correlation between COVID-19 severity and complement overactivation, which is a key immune response. Additionally, preliminary open-label safety data from six patients in a Phase 1/2 study support advancement of APL-9, an investigational targeted C3 therapy designed for acute interventions, for severe COVID-19. These results also showed that key markers of inflammation were within or near normal range at the end of the APL-9 treatment period.

    Observational Study Results

    In the 41-patient observational study, data from 40 patients with evaluable blood samples demonstrated that increased complement activation showed a correlation with disease severity. C3a, C4a, Bb and terminal complement complex (TCC) levels each correlated statistically with COVID-19 severity as quantified by the National Early Warning Score (NEWS), a widely used assessment for the need for critical care. A similar correlation was observed with lactate dehydrogenase (LDH), C-reactive protein (CRP) and cytokine interleukin 6 (IL-6).

    In addition, 39 (97.5%) patients had substantially elevated systemic levels of C3a, a marker for C3 activation, with median C3a levels 3.7 times the upper limit of normal. Multiple complement pathways were activated as evidenced by systemic elevations of C4a, Bb and TCC. Complement overactivation is known to worsen respiratory diseases by killing healthy cells and causing further complications such as blood clots and multiorgan failure, making it difficult for patients to recover.

    "These study results demonstrate that multiple complement pathways are going into overdrive in severe cases of COVID-19, reinforcing the critical need for therapies that can regulate overactive complement and improve patient outcomes in COVID-19," said Lukas Scheibler, Ph.D., Chief Innovation Officer of Apellis. "Targeting C3 has the potential to control complement activation across all three major pathways due to its central location, so we believe it is a strong target to continue exploring." 

    Observational study levels of complement and inflammatory markers

    n = 40Median

    (range)
    ULNMultiple of

    ULN (median)
    # patients >ULN (%

    of patients)
    Bb, µg/mL1.37

    (0.44, 6.22)
    1.490.916 (40.0%)
    C3, g/L1.36

    (0.82, 2.03)
    1.620.85 (12.5%)
    C3a, ng/mL182

    (34.8, 679.8)
    49.43.739 (97.5%)
    C4, g/L0.39

    (0.08, 1.11)
    0.520.77 (17.5%)
    C4a, ng/mL1612

    (658, 3000)
    12511.329 (72.5%)
    TCC, ng/mL187

    (109, 1034)
    2440.811 (27.5%)
    IL-6, pg/mL19.8

    (2.22, 613.37)
    11.91.727 (67.5%)
    CRP, mg/L50.8

    (0.8, 361.5)
    412.736 (90.0%)
    dLDH, U/L224

    (107, 850)
    1801.230 (75.0%)

    n=number of patients with evaluable assessments for each parameter. CRP, C-reactive protein; IL-6, interleukin 6; LDH, lactate dehydrogenase; TCC, terminal complement complex; ULN, upper limit of normal.

    Preliminary Results from the Phase 1/2 Study

    Apellis also announced preliminary results from the first part of the interventional Phase 1/2 study of APL-9 in patients with severe COVID-19. Of the six patients enrolled, all patients had elevated C3a and CRP levels and five of the six patients had elevated LDH levels at baseline. Five patients completed the study and were discharged from the hospital. Additionally, in these patients, C3a, LDH and CRP levels were within the normal reference range by the end of the APL-9 treatment period. Three patients experienced four treatment-emergent adverse events (TEAEs), which were all considered unrelated to study treatment. One serious adverse event of respiratory failure, which was considered unrelated to the study treatment, led to death from failed intubation.

    Based on the results, an independent data monitoring committee recommended continuing the investigation of APL-9 in this study. Apellis is currently enrolling an additional 60 patients with COVID-19 and respiratory failure who require oxygen supplementation or mechanical ventilation into the randomized, double-blind, controlled phase of the study to evaluate the safety and efficacy of APL-9 (as add-on to standard care for up to 21 days).

    Detailed results from both studies will be submitted for future scientific publication.

    About the Observational Study

    The observational exploratory study evaluated the relationship between markers of complement activation and disease severity in 41 patients with COVID-19. Patients in the observational study had blood drawn at baseline and at subsequent visits for up to six weeks. The primary objective of the observational study was to quantify systemic concentrations of the complement activation products Bb, C3a, C4a and terminal complement complex (TCC) and correlate them to COVID-19 severity. Secondary objectives were to quantify concentrations of C3 and C4 in serum and proinflammatory T-cell populations (CD4, CD8 and TH17) in whole blood and correlate them to COVID-19 severity. The exploratory objectives were to quantify the concentrations of interleukin 6 (IL-6) and C-reactive protein (CRP) in plasma and serum, respectively, and correlate them to COVID-19 severity. Additional detail and study results can be found on the Events and Presentations section of the Apellis website.

    About the Interventional Phase 1/2 Study

    The Phase 1/2 study (NCT04402060) is designed to evaluate the safety and feasibility of targeting C3 with APL-9 as an add-on therapy to standard of care in 66 patients with severe COVID-19. Patients included in the study have respiratory failure requiring oxygen supplementation or invasive or noninvasive mechanical ventilation. Patients are treated with APL-9 via intravenous (IV) infusion with an initial dose of 540 mg in the first 24 hours of dosing, followed by 360 mg/day on all subsequent days. The primary objective of the study is to assess the safety of APL-9 as an add-on to the current standard of care. Secondary objectives include evaluating length of stay in the hospital, overall survival, time on oxygen therapy or mechanical ventilation, and markers of complement activation, organ failure and coagulation (blood clotting). In the preliminary phase, six patients received open-label APL-9 treatment either until the end of study drug administration at day 7 or resolution of acute respiratory distress syndrome (ARDS). Based on the results, an independent data monitoring committee recommended continuing the investigation of APL-9 in this study. Apellis is enrolling an additional 60 patients into the randomized, double-blind, controlled phase of the study to evaluate the safety and efficacy of APL-9 (as add-on to standard care for up to 21 days). Additional detail and preliminary results can be found on the Events and Presentations section of the Apellis website.

    About APL-9

    APL-9 is an investigational drug designed to control the complement cascade centrally at C3 and may have the potential to treat a range of diseases caused by excessive or uncontrolled activation of complement. APL-9 leverages the same mechanism of action as Apellis' lead compound, pegcetacoplan (APL-2), but has a lower molecular weight and shorter half-life. APL-9 is designed to be intravenously administered for acute use.



    About Apellis 

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop best-in-class and first-in-class therapies for a broad range of debilitating diseases that are driven by uncontrolled or excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit http://apellis.com.

    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 the company's clinical trials will be fully enrolled and completed when anticipated; 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 will be indicative of results that will be generated in future clinical trials; whether pegcetacoplan will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the company's clinical trials will warrant regulatory submissions and whether pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, C3G, IC-MPGN, ALS, or severe COVID-19 or any other indication when expected or at all; 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 July 30, 2020 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: 

    Lissa Pavluk



    617.977.6764

    Investor Contact:

    Sam Martin / Maghan Meyers

    Argot Partners

     / 

    212.600.1902

     

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    • Results demonstrate a greater than 65% mean reduction in proteinuria at week 48 in C3G patients treated with pegcetacoplan
    • Sustained improvements were seen across key clinical measures at 48 weeks
    • There are no approved medicines for C3G, a rare disease that often leads to kidney failure

    WALTHAM, Mass., Oct. 09, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that positive results from the Phase 2 DISCOVERY study evaluating pegcetacoplan, a targeted C3 therapy, in patients with C3 glomerulopathy (C3G) will be presented at the American Society of Nephrology (ASN) Kidney Week on October 22, 2020. The 48-week results show that patients treated…

    • Results demonstrate a greater than 65% mean reduction in proteinuria at week 48 in C3G patients treated with pegcetacoplan

    • Sustained improvements were seen across key clinical measures at 48 weeks
    • There are no approved medicines for C3G, a rare disease that often leads to kidney failure

    WALTHAM, Mass., Oct. 09, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that positive results from the Phase 2 DISCOVERY study evaluating pegcetacoplan, a targeted C3 therapy, in patients with C3 glomerulopathy (C3G) will be presented at the American Society of Nephrology (ASN) Kidney Week on October 22, 2020. The 48-week results show that patients treated with pegcetacoplan demonstrated sustained improvements across key clinical measures including a greater than 65% mean reduction in proteinuria, which is an important marker of renal damage. C3G is a rare, complement-driven kidney disease with no approved medicines.

    In five C3G patients treated with pegcetacoplan, mean (SE) proteinuria decreased from 3.48 (0.82) mg/mg at baseline to 0.93 (0.27) mg/mg at week 48, a decrease of 73.3%, as measured by 24-hour urine protein-to-creatinine ratio (uPCR). Importantly, this reduction in proteinuria was accompanied by a corresponding increase in mean serum albumin. Other biomarkers improved, including an observed increase in mean serum C3 and stabilization of renal function, as measured by mean serum creatinine. No serious or severe adverse events were reported, and pegcetacoplan was well tolerated overall.

    "There is an urgent need for treatments for C3G, a disease that ultimately leads to kidney failure for about half of people living with the disease. These positive results highlight the potential of pegcetacoplan to make a meaningful difference for the C3G community and further strengthen our confidence in targeting C3 across multiple complement-driven diseases," said Federico Grossi, M.D., Ph.D., Chief Medical Officer of Apellis. "We are working quickly to progress a registrational program of our targeted C3 therapy for people living with C3G."

    Earlier this week, Apellis announced that it was advancing Phase 2 and Phase 3 studies of pegcetacoplan in patients with C3G or immune complex membranoproliferative glomerulonephritis (IC-MPGN), another rare, complement-driven kidney disease with no approved medicines.

    Presentation Details at ASN Kidney Week 2020

    C3 Inhibition with Pegcetacoplan Targets the Underlying Disease Process of C3 Glomerulopathy (C3G) and Improves Proteinuria, ePoster # PO1852 – Oct. 22 at 10:00 a.m. ET.

    About the DISCOVERY Study

    The DISCOVERY study is a Phase 2, open-label trial designed to evaluate the preliminary efficacy and safety of pegcetacoplan in patients with complement-driven renal diseases. Eight C3G patients were enrolled into the study. Three patients were excluded from the analysis at 48 weeks due to self-reported non-compliance or study drug interruption.

    The primary endpoint in the DISCOVERY study is change from baseline in proteinuria at week 48 as measured by 24-hour urine protein-to-creatinine ratios (uPCR). Secondary endpoints include analysis of serum C3 and estimated glomerular filtration rate (eGFR).

    About Pegcetacoplan (APL-2)

    Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive complement activation, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Apellis is evaluating pegcetacoplan in several clinical studies across hematology, ophthalmology, nephrology, and neurology. Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and the treatment of geographic atrophy, and received orphan drug designation for the treatment of C3G by the FDA and European Medicines Agency. For additional information regarding our clinical trials, visit https://apellis.com/our-science/clinical-trials.

    About C3 Glomerulopathy (C3G) and Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN)

    C3G and IC-MPGN are rare, debilitating kidney diseases that affect ~18,000 people in the United States and Europe.1 There are no approved therapies for the diseases, and symptoms include blood in the urine, dark foamy urine due to the presence of protein, swelling and high blood pressure.2 Approximately 50% of people living with C3G and IC-MPGN ultimately suffer kidney failure within five to 10 years of diagnosis.3 Although IC-MPGN is considered a distinct disease from C3G, the underlying cause and progression of the two diseases are remarkably similar and include overactivation of the complement cascade, with excessive accumulation of C3 breakdown products in the kidney causing inflammation and damage to the organ.4,5

    About Apellis 

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop best-in-class and first-in-class therapies for a broad range of debilitating diseases that are driven by uncontrolled or excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit http://apellis.com.



    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 the company's clinical trials will be fully enrolled and completed when anticipated; 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 will be indicative of results that will be generated in future clinical trials; whether pegcetacoplan will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the company's clinical trials will warrant regulatory submissions and whether pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, C3G, IC-MPGN, ALS or any other indication when expected or at all; 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 July 30, 2020 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.

    Contacts:

    Media

    Tracy Vineis



    +1 617 420 4839



    Investors

    Sam Martin / Maghan Meyers

    /

    +1 212 600 1902

    1 ClearView Analysis using physician and literature consensus.

    2 Complement 3 Glomerulopathy (C3G). National Kidney Foundation Web site. https://www.kidney.org/atoz/content/complement-3-glomerulopathy-c3g. Accessed November 21, 2019.

    3 C3 glomerulopathy. National Institute of Health, Genetics Home Reference. https://ghr.nlm.nih.gov/condition/c3-glomerulopathy#resources. Accessed November 21, 2019.

    4 Noris M, Donadelli R, Remuzzi G. Autoimmune abnormalities of the alternative complement pathway in membranoproliferative glomerulonephritis and C3 glomerulopathy. Pediatr Nephrol. 2019 Aug;34(8):1311-1323.

    5 Cook HT. Evolving complexity of complement-related diseases: C3 glomerulopathy and atypical haemolytic uremic syndrome. Curr Opin Nephrol Hypertens. 2018 May;27(3):165-170.

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  21. WALTHAM, Mass., Oct. 07, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company pioneering targeted C3 therapies, today announced that the company approved the grant of equity awards to four new employees with grant date of October 1, 2020, as equity inducement awards outside of the company's 2017 Stock Incentive Plan (but under the terms of the 2020 Inducement Stock Incentive Plan) and material to the employees' acceptance of employment with the company. The equity awards were approved on July 1, 2020, September 8, 2020, September 11, 2020, and September 25, 2020, in accordance with Nasdaq Listing Rule 5635(c)(4).

    The employees received options to purchase 25,962 shares of Apellis common…

    WALTHAM, Mass., Oct. 07, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company pioneering targeted C3 therapies, today announced that the company approved the grant of equity awards to four new employees with grant date of October 1, 2020, as equity inducement awards outside of the company's 2017 Stock Incentive Plan (but under the terms of the 2020 Inducement Stock Incentive Plan) and material to the employees' acceptance of employment with the company. The equity awards were approved on July 1, 2020, September 8, 2020, September 11, 2020, and September 25, 2020, in accordance with Nasdaq Listing Rule 5635(c)(4).

    The employees received options to purchase 25,962 shares of Apellis common stock and 4,275 restricted stock units (RSUs). The options have an exercise price of $31.77, which is equal to the closing price of Apellis common stock on October 1, 2020, the grant date of the options. One-fourth of the shares underlying the employee options will vest on the one year anniversary of the grant date and thereafter 1/48th of the shares underlying the employee options will vest monthly, such that the shares underlying the options granted to the employees will be fully vested on the fourth anniversary of the grant date, subject to the employees' continued employment with Apellis on such vesting dates.   Each RSU will vest as to 25% of the shares underlying the RSU award on the first anniversary of the grant date and as to an additional 25% of the shares underlying the RSU award annually thereafter, subject to each such employee's continued employment on each vesting date.

    About Apellis

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop best-in-class and first-in-class therapies for a broad range of debilitating diseases that are driven by uncontrolled or excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit http://apellis.com.

    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 the company's clinical trials will be fully enrolled and completed when anticipated; 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 will be indicative of results that will be generated in future clinical trials; whether pegcetacoplan or APL-9 will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the company's clinical trials will warrant regulatory submissions and whether pegcetacoplan or APL-9 will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, C3G, COVID-19 with respiratory failure including ARDS or any other indication when expected or at all; 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 July 30, 2020 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.

    Investor Contact:

    Sam Martin / Maghan Meyers

    Argot Partners

     / 

    212.600.1902 

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    • Advancing Phase 2 and Phase 3 studies of the company's targeted C3 therapy in C3G / IC-MPGN, rare kidney diseases with no approved medicines. First patient in Phase 2 study expected to be dosed by the end of the year
    • Potentially registrational Phase 2 study in ALS to enroll ~200 adults globally. First patient expected to be dosed by the end of the year

    WALTHAM, Mass., Oct. 05, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that it has initiated registrational programs of pegcetacoplan, a targeted C3 therapy, for people living with C3 glomerulopathy (C3G) or immune complex membranoproliferative glomerulonephritis (IC-MPGN), rare diseases…

    • Advancing Phase 2 and Phase 3 studies of the company's targeted C3 therapy in C3G / IC-MPGN, rare kidney diseases with no approved medicines. First patient in Phase 2 study expected to be dosed by the end of the year

    • Potentially registrational Phase 2 study in ALS to enroll ~200 adults globally. First patient expected to be dosed by the end of the year

    WALTHAM, Mass., Oct. 05, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that it has initiated registrational programs of pegcetacoplan, a targeted C3 therapy, for people living with C3 glomerulopathy (C3G) or immune complex membranoproliferative glomerulonephritis (IC-MPGN), rare diseases that can lead to kidney failure within five to 10 years, and amyotrophic lateral sclerosis (ALS), a devastating neurodegenerative disease that leads to progressive muscle weakness and paralysis. Uncontrolled activation of the complement cascade, a part of the body's immune system, is believed to play a role in the progression of these serious diseases.

    "People and families living with C3G, IC-MPGN, and ALS are in significant need of new medicines. We believe that controlling complement activation centrally, at the level of C3, has the potential to offer an important new therapeutic approach for each of these rare diseases," said Cedric Francois, M.D., Ph.D., co-founder and chief executive officer of Apellis. "Our data in C3G support continued advancement of pegcetacoplan, and numerous studies suggest that elevated levels of C3 may play a role in the progression of ALS. These new trials build on the broad platform potential of targeting C3, and we are working urgently to advance these programs for patients."

    Nephrology: Registrational Program for C3G / IC-MPGN

    C3G / IC-MPGN are rare kidney diseases that impact ~18,000 patients in the United States and Europe1 and currently have no approved therapies. C3 deposition in the kidneys is present in both C3G and IC-MPGN; however, substantial immunoglobulin deposition is also seen in IC-MPGN.2,3

    Apellis is advancing a registrational program in C3G / IC-MPGN beginning with the Phase 2 NOBLE study, a randomized, controlled trial in 12 patients with post-transplant disease recurrence that will focus on the histopathology of the kidneys. The first patient is expected to be dosed in this study by the end of 2020. Apellis also plans to begin a Phase 3 study in the first half of 2021 with reduction in proteinuria as its primary endpoint.

    With these trials, Apellis will be the only company actively studying a potential treatment for patients with IC-MPGN, a disease where both the alternative and classical pathways have been implicated, which supports targeting the complement cascade centrally at C3 as a potential therapeutic option.

    The company is advancing pegcetacoplan in C3G / IC-MPGN based on data in C3G from the Phase 2 DISCOVERY trial. Data at week 12 were positive; results at 48 weeks will be presented at a scientific congress later this year.

    Neurology: Potentially Registrational Phase 2 Study in ALS

    Apellis is expanding the development of pegcetacoplan into neurology with a potentially registrational Phase 2 study in ALS, a disease that impacts approximately 225,000 patients worldwide.4

    In individuals with ALS, high levels of activated C3 are present at the neuromuscular junction5 where the neurons communicate directly to muscle cells. Multiple studies suggest that the elevated levels of C3 present in ALS may be responsible for the chronic neuroinflammation that leads to motor neuron death.5,6,7

    Apellis has initiated the MERIDIAN study, a randomized, placebo-controlled trial of pegcetacoplan in approximately 200 adults with sporadic ALS, and expects to dose the first patient by the end of 2020. The primary endpoint of the study is the Combined Assessment of Function and Survival (CAFS) rank scores at week 52.

    About the Phase 2 NOBLE study in C3G / IC-MPGN

    The Phase 2 NOBLE study (APL2-C3G-204; NCT04572854) is a multicenter, open-label, randomized, controlled study designed to evaluate the efficacy and safety of pegcetacoplan in up to 12 adults who have post-transplant recurrence of C3G or IC-MPGN. Study participants will be randomized in a 3:1 ratio to receive pegcetacoplan or maintain standard of care for 12 weeks and then all patients in the study will receive pegcetacoplan from week 13 to week 52.

    The primary endpoint of the study is the proportion of patients with reduction in C3c staining on renal biopsy after 12 weeks of treatment with pegcetacoplan. Secondary endpoints include an evaluation of safety, the proportion of patients with reduction in C3c staining on renal biopsy after 52 weeks of treatment, and the proportion of patients achieving at least a 50% reduction in proteinuria.

    About the Phase 2 MERIDIAN Study in ALS

    The Phase 2 MERIDIAN study (APL2-ALS-206) is a potentially registrational, randomized, placebo-controlled, multicenter study that was designed to evaluate the efficacy and safety of pegcetacoplan in approximately 200 adults with sporadic ALS. Study participants will be randomized in a 2:1 ratio to receive pegcetacoplan or placebo while continuing to receive their existing standard of care treatment for ALS. After one year, all patients in the study will receive pegcetacoplan. To reduce the burden on people living with ALS and their caregivers, the trial has been designed to minimize the number of in-clinic visits, with approximately six clinic visits in the first year and four in the open-label second year.

    The primary endpoint of the study is the Combined Assessment of Function and Survival (CAFS) rank scores at week 52. Key secondary endpoints include measures of lung function, muscle strength, and quality of life.

    About Pegcetacoplan (APL-2)

    Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive or uncontrolled complement activation, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Apellis is evaluating pegcetacoplan in several clinical studies including paroxysmal nocturnal hemoglobinuria (PNH), geographic atrophy (GA), cold agglutinin disease (CAD), and C3 glomerulopathy (C3G). Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of PNH and the treatment of GA, and received orphan drug designation for the treatment of C3G by the FDA and European Medicines Agency. For additional information regarding our clinical trials, visit https://apellis.com/our-science/clinical-trials.

    About C3 Glomerulopathy (C3G) and Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN)

    C3G and IC-MPGN are rare, debilitating kidney diseases that affect ~18,000 people in the United States and Europe.1 There are no approved therapies for the diseases, and symptoms include blood in the urine, dark foamy urine due to the presence of protein, swelling and high blood pressure.8 Approximately 50% of people living with C3G and IC-MPGN ultimately suffer kidney failure within five to 10 years of diagnosis.9 Although IC-MPGN is considered a distinct disease from C3G, the underlying cause and progression of the two diseases are remarkably similar and include overactivation of the complement cascade, with excessive accumulation of C3 breakdown products in the kidney causing inflammation and damage to the organ.2,3

    About Amyotrophic Lateral Sclerosis (ALS)

    ALS is a devastating neurodegenerative disease that results in progressive muscle weakness and paralysis due to the death of nerve cells, called motor neurons, in the brain and spinal cord.10,11 The death of motor neurons leads to the progressive loss of voluntary muscle movement required for speaking, walking, swallowing and breathing.10,11 In individuals with ALS, high levels of C3 are present at the neuromuscular junction5 where motor neurons communicate directly to muscle cells. Numerous studies suggest that elevated levels of C3 present throughout the motor system of ALS patients are likely to contribute to chronic neuroinflammation and the death of motor neurons.5,6,7 There are no treatments that stop or reverse the progression of ALS, which impacts ~225,000 patients worldwide.4

    About Apellis

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop best-in-class and first-in-class therapies for a broad range of debilitating diseases that are driven by uncontrolled or excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit http://apellis.com.

    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 the company's clinical trials will be fully enrolled and completed when anticipated; 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 will be indicative of results that will be generated in future clinical trials; whether pegcetacoplan will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the company's clinical trials will warrant regulatory submissions and whether pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, C3G, IC-MPGN, ALS or any other indication when expected or at all; 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 July 30, 2020 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



    +1 617 420 4839

    Investor Contact:

    Sam Martin / Maghan Meyers

    /

    +1 212 600 1902

    1 ClearView Analysis using physician and literature consensus.

    2 Noris M, Donadelli R, Remuzzi G. Autoimmune abnormalities of the alternative complement pathway in membranoproliferative glomerulonephritis and C3 glomerulopathy. Pediatr Nephrol. 2019 Aug;34(8):1311-1323.

    3 Cook HT. Evolving complexity of complement-related diseases: C3 glomerulopathy and atypical haemolytic uremic syndrome. Curr Opin Nephrol Hypertens. 2018 May;27(3):165-170.

    4 Arthur K et al. Nat Commun, 2016, Vol 7, article 12408

    5 Bahia El Idrissi N, et al. J Neuroinflammation. 2016;13(1):72.4 Sta M, et al. Neurobiol Dis. 2011;42(3):211-220.

    6 Woodruff, et al., PNAS January 7, 2014 111 (1) E3-E4

    7 Lee, et al Journal of Neuroinflammation volume 15: 171 (2018)

    8 Complement 3 Glomerulopathy (C3G). National Kidney Foundation Website. https://www.kidney.org/atoz/content/complement-3-glomerulopathy-c3g. Accessed November 21, 2019.

    9 C3 glomerulopathy. National Institute of Health, Genetics Home Reference. https://ghr.nlm.nih.gov/condition/c3-glomerulopathy#resources. Accessed November 21, 2019.

    10 National Institute of Neurological Disorders and Stroke. (2020). Amyotrophic Lateral Sclerosis Fact Sheet. Retrieved from https://www.ninds.nih.gov/Disorders/Patient-Caregiver-Education/Fact-Sheets/Amyotrophic-lateral-Sclerosis-ALS-Fact-Sheet

    11 ALS Association. What is ALS? Retrieved June 2020 from https://www.als.org/understanding-als/what-is-als

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    • Post hoc analysis of Phase 2 FILLY study shows 39% reduction in rate of progression from nascent GA, an earlier form of disease, to GA in patients treated with pegcetacoplan monthly vs. sham
    • First ever observation of slowed nascent GA progression in a Phase 2 study signals potential benefit of earlier intervention with pegcetacoplan in patients with GA
    • Data support hypothesis that targeting C3 with pegcetacoplan addresses an underlying cause of disease, excessive complement activation, as evidenced by slowed progression of nascent GA to GA

    WALTHAM, Mass., Oct. 03, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced the results of a post hoc…

    • Post hoc analysis of Phase 2 FILLY study shows 39% reduction in rate of progression from nascent GA, an earlier form of disease, to GA in patients treated with pegcetacoplan monthly vs. sham

    • First ever observation of slowed nascent GA progression in a Phase 2 study signals potential benefit of earlier intervention with pegcetacoplan in patients with GA

    • Data support hypothesis that targeting C3 with pegcetacoplan addresses an underlying cause of disease, excessive complement activation, as evidenced by slowed progression of nascent GA to GA

    WALTHAM, Mass., Oct. 03, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced the results of a post hoc analysis of the Phase 2 FILLY study investigating intravitreal pegcetacoplan (APL-2) for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The post hoc analysis found that monthly treatment with pegcetacoplan significantly reduced the rate of progression from nascent GA to GA in areas of the retina outside of existing GA lesions. The data were presented today in a late-breaking oral session at the European Society of Retina Specialists 2020 Virtual (EURETINA).

    Pegcetacoplan is the only targeted C3 therapy in Phase 3 clinical trials for GA, a complement-driven eye disease1,2 that causes blindness, affects approximately five million people globally3,4 and has no approved treatment.

    • In the post hoc analysis, progression from nascent GA to GA was observed in 50.0% of the pegcetacoplan-treated group and 81.8% of the sham-treated group (p=0.02).

    • The 39% reduction in progression from nascent GA to GA indicates that pegcetacoplan slows the rate of progression of AMD in areas of the retina outside of GA lesions.

    • Progression from large drusen (deposits in the retina that are a hallmark of AMD) to nascent GA or GA at Month 12 was observed in 22.6% of patients in the pegcetacoplan-treated group and 33.3% of patients in the sham-treated group (p=0.31).

    "Our findings from this post-hoc analysis demonstrate that intravitreal pegcetacoplan, a targeted C3 therapy, significantly lowers the rate of progression from nascent GA to GA in patients when compared to sham controls," said SriniVas Sadda, M.D., President & Chief Scientific Officer of the Doheny Eye Institute and lead investigator. "This study provides exciting evidence to support further exploration of the potential of pegcetacoplan for earlier intervention in the course of GA."

    The post hoc analysis, a collaboration with the Doheny Image Reading Research Lab, included FILLY patients from the monthly pegcetacoplan-treated group (n=42) and sham-treated group (n=69) who completed the Month 12 study visit and who did not develop exudative or neovascular AMD. The objective of the analysis was to investigate the effects of pegcetacoplan on complement-driven progression of AMD outside of GA lesions.

    "Patients with geographic atrophy experience changes in the retina that progress from the earlier stages of AMD to the beginning of atrophy and ultimately irreversible vision loss," said Federico Grossi, M.D., Ph.D., Chief Medical Officer of Apellis. "This post hoc analysis of the FILLY study demonstrates that pegcetacoplan slows early disease progression and may have the potential to delay the onset of GA and vision loss in patients."

    Earlier this year, Apellis announced the completion of patient enrollment in the ongoing Phase 3 DERBY and OAKS studies investigating pegcetacoplan in patients with GA secondary to AMD. Top-line results from these pivotal trials are expected in Q3 2021.

    About Pegcetacoplan (APL-2)

    Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive complement activation, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Apellis is evaluating pegcetacoplan in several clinical studies including paroxysmal nocturnal hemoglobinuria (PNH), geographic atrophy (GA), cold agglutinin disease (CAD), and C3 glomerulopathy (C3G). Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of PNH and the treatment of GA. For additional information regarding our clinical trials, visit https://apellis.com/our-science/clinical-trials.

    About Geographic Atrophy (GA) 

    GA is an advanced form of age-related macular degeneration (AMD), a leading cause of blindness. Excessive complement activation drives irreversible lesion growth in GA5, and C3 is the only target to precisely control complement overactivation. Pegcetacoplan, studied in early and late-stage trials comprising a total of approximately 1,500 patients, is the only targeted C3 inhibitor being evaluated in late-stage trials to control lesion growth in GA.6

    GA lesions affect the central portion of the retina, known as the macula, which is responsible for central vision. GA is progressive and irreversible, leading to central visual impairment and permanent loss of vision. Based on published studies, approximately one million people have GA in the United States and 5 million people have GA globally.1,2 There are currently no approved treatments for GA.

    About FILLY

    The FILLY study was a 246-patient, Phase 2, multicenter, randomized, single-masked, sham-controlled clinical trial evaluating pegcetacoplan in patients with GA secondary to AMD conducted at over 40 clinical sites in the United States, Australia and New Zealand. Pegcetacoplan was administered as an intravitreal injection monthly or every other month (EOM) for 12 months, followed by six months of monitoring after the end of treatment. The primary efficacy endpoint was the change in GA lesion area from baseline to Month 12 in pegcetacoplan-treated patients compared to sham.

    About DERBY and OAKS

    DERBY (621 patients enrolled) and OAKS (638 patients enrolled) are Phase 3, multicenter, randomized, double-masked, sham-controlled studies to compare the efficacy and safety of intravitreal pegcetacoplan with sham injections in patients with GA secondary to AMD. The primary objective of the studies is to evaluate the efficacy of pegcetacoplan compared to sham injection in patients with GA secondary to AMD assessed by change in the total area of GA lesions from baseline as measured by fundus autofluorescence (FAF).

    About Apellis 

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop best-in-class and first-in-class therapies for a broad range of debilitating diseases that are driven by uncontrolled or excessive activation of the complement cascade, including those within hematology, ophthalmology, and nephrology. For more information, please visit http://apellis.com.

    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 the company's clinical trials will be fully enrolled and completed when anticipated; 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 will be indicative of results that will be generated in future clinical trials; whether pegcetacoplan will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the company's clinical trials will warrant regulatory submissions and whether pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, C3G or any other indication when expected or at all; 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 July 30, 2020 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:

    Mark Dole



    617.997.3484

    Investor Contact: 

    Sam Martin / Maghan Meyers

    Argot Partners

     / 

    212.600.1902

    __________________________________

    1 Weber, BHF, Issa, PC, et al. The Role of the Complement System in Age-Related Macular Degeneration. Dtsch Arztebl Int 2014; 111(8): 133–8. 



    2 Heesterbeek, TJ, Lechanteur YTE, et al. Complement activation levels are related to disease stage in AMD. Invest Ophthalmol Vis Sci. 2020;61(3):18. 



    3 Rudnicka AR, Jarrar Z, Wormald R, et al. Age and gender variations in age-related macular degeneration prevalence in populations of European ancestry: a meta-analysis. Ophthalmology 2012;119:571–580.



    4 Wong WL, Su X, Li X, et al. Global prevalence of age-related macular degeneration and disease burden projection for 2020 and 2040: a systematic review and meta-analysis. Lancet Glob Health 2014;2:e106–116.

    5 Seddon, JM, Rosner, B. Validated prediction models for macular degeneration progression and predictors of visual acuity loss identify high-risk individuals. Am J Ophthalmol 2019;198:223–261. 

    6 Yates, JRW, Sepp T, et al. Complement C3 Variant and the Risk of Age-Related Macular Degeneration. N Engl J Med 2007;357.

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  22. WALTHAM, Mass., Sept. 24, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company will present at Stifel's 2020 Immunology and Inflammation Virtual Summit on Thursday, October 1, 2020 at 9:00 a.m. ET.

    Cedric Francois, M.D., Ph.D., co-founder and chief executive officer of Apellis, will participate in a panel discussion titled "The Evolution of Complement Targeted Therapies." The event will be available live via webcast from the "Events and Presentations" page of the "Investors and Media" section of the company's website at https://investors.apellis.com/events-and-presentations. A replay of the webcast will be available…

    WALTHAM, Mass., Sept. 24, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company will present at Stifel's 2020 Immunology and Inflammation Virtual Summit on Thursday, October 1, 2020 at 9:00 a.m. ET.

    Cedric Francois, M.D., Ph.D., co-founder and chief executive officer of Apellis, will participate in a panel discussion titled "The Evolution of Complement Targeted Therapies." The event will be available live via webcast from the "Events and Presentations" page of the "Investors and Media" section of the company's website at https://investors.apellis.com/events-and-presentations. A replay of the webcast will be available for 90 days following the event.

    About Apellis

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop best-in-class and first-in-class therapies for a broad range of debilitating diseases that are driven by uncontrolled or excessive activation of the complement cascade, including those within hematology, ophthalmology, and nephrology. For more information, please visit http://apellis.com.

    Investor Contact:

    Sam Martin / Maghan Meyers

    Argot Partners

    /

    212.600.1902

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    • New analysis of age-related macular degeneration (AMD) progression in Phase 2 FILLY study accepted as late-breaking oral presentation at European Society of Retina Specialists Congress (EURETINA)
    • Pegcetacoplan targets C3, and has the potential to control the irreversible lesion growth in GA, an advanced form of AMD and leading cause of blindness

    WALTHAM, Mass., Sept. 21, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that a post hoc analysis of the Phase 2 FILLY study investigating intravitreal pegcetacoplan (APL-2) for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD) has been accepted as…

    • New analysis of age-related macular degeneration (AMD) progression in Phase 2 FILLY study accepted as late-breaking oral presentation at European Society of Retina Specialists Congress (EURETINA)
    • Pegcetacoplan targets C3, and has the potential to control the irreversible lesion growth in GA, an advanced form of AMD and leading cause of blindness

    WALTHAM, Mass., Sept. 21, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that a post hoc analysis of the Phase 2 FILLY study investigating intravitreal pegcetacoplan (APL-2) for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD) has been accepted as a late-breaking oral presentation by EURETINA 2020 Virtual.

    This new analysis of the FILLY study demonstrates that pegcetacoplan impacts the progression of nascent GA,1,2 the earlier stage of disease that precedes atrophy, in areas of the retina outside of GA lesions. The data are from a collaboration with the Doheny Image Reading Research Lab, and will be presented by SriniVas Sadda, M.D. at the "Late Breaking & Reviews" oral session.

    "We are excited that this post hoc analysis of the FILLY study was selected by EURETINA as a late-breaking presentation. These new data highlight the potential of targeting C3 with pegcetacoplan to transform the treatment of GA, a disease that leads to irreversible vision loss and has no approved treatments," said Federico Grossi, M.D., Ph.D., Chief Medical Officer of Apellis.

    Featured Presentation

    Impact of Pegcetacoplan on Progression of Nascent Atrophy in AMD, October 3, 5:05 p.m. CET.

    About Pegcetacoplan (APL-2)

    Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive complement activation, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Apellis is evaluating pegcetacoplan in several clinical studies including paroxysmal nocturnal hemoglobinuria (PNH), geographic atrophy (GA), cold agglutinin disease (CAD), and C3 glomerulopathy (C3G). Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of PNH and the treatment of GA. For additional information regarding our clinical trials, visit https://apellis.com/our-science/clinical-trials.

    About Geographic Atrophy (GA) 

    GA is an advanced form of age-related macular degeneration (AMD), a leading cause of blindness. Excessive complement activation drives irreversible lesion growth in GA3, and C3 is the only target to precisely control complement overactivation. Pegcetacoplan, studied in early and late-stage trials comprising a total of approximately 1500 patients, is the only targeted C3 inhibitor being evaluated in late-stage trials to control lesion growth in GA.4

    GA lesions affect the central portion of the retina, known as the macula, which is responsible for central vision. GA is progressive and irreversible, leading to central visual impairment and permanent loss of vision. Based on published studies, approximately one million people have GA in the United States and 5 million people have GA globally.5.6  There are currently no approved treatments for GA.

    About FILLY

    The FILLY study was a 246-patient, Phase 2, multicenter, randomized, single-masked, sham-controlled clinical trial evaluating pegcetacoplan in patients with GA secondary to AMD conducted at over 40 clinical sites in the United States, Australia and New Zealand. Pegcetacoplan was administered as an intravitreal injection monthly or every other month (EOM) for 12 months, followed by six months of monitoring after the end of treatment. The primary efficacy endpoint was the change in GA lesion area from baseline to month 12 in pegcetacoplan-treated patients compared to sham.

    About Apellis 

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop best-in-class and first-in-class therapies for a broad range of debilitating diseases that are driven by uncontrolled or excessive activation of the complement cascade, including those within hematology, ophthalmology, and nephrology. For more information, please visit http://apellis.com.

    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 the company's clinical trials will be fully enrolled and completed when anticipated; 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 will be indicative of results that will be generated in future clinical trials; whether pegcetacoplan will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the company's clinical trials will warrant regulatory submissions and whether pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, C3G or any other indication when expected or at all; 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 July 30, 2020 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:

    Mark Dole

     

    617.997.3484

    Investor Contact: 

    Sam Martin / Maghan Meyers

    Argot Partners

     / 

    212.600.1902

    1 Schaal, KB, Gregori, G., Rosenfeld, PJ. En face optical coherence tomography imaging for the detection of nascent geographic atrophy. Am J Ophthalmol 2017;174: 145–154.

    2 Guymer, RH, et al. Incomplete retinal pigment epithelial and outer retinal atrophy in age-related macular degeneration. Ophthalmology 2020;127:394-409.

    3 Seddon, JM, Rosner, B. Validated prediction models for macular degeneration progression and predictors of visual acuity loss identify high-risk individuals. Am J Ophthalmol 2019;198:223–261. 

    4 Yates, JRW, Sepp T, et al. Complement C3 Variant and the Risk of Age-Related Macular Degeneration. N Engl J Med 2007;357.

    5 Weber, BHF, Issa, PC, et al. The Role of the Complement System in Age-Related Macular Degeneration. Dtsch Arztebl Int 2014; 111(8): 133–8. 



    6 Heesterbeek, TJ, Lechanteur YTE, et al. Complement activation levels are related to disease stage in AMD. Invest Ophthalmol Vis Sci. 2020;61(3):18. 

     

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  23. WALTHAM, Mass., Sept. 15, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) and a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for pegcetacoplan for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in the United States and European Union, respectively.

    "Pegcetacoplan has demonstrated its potential to elevate the standard of care in PNH and the submissions of the U.S. and EU marketing applications represent an important step in our efforts to bring the first targeted C3 therapy to people…

    WALTHAM, Mass., Sept. 15, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) and a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for pegcetacoplan for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in the United States and European Union, respectively.

    "Pegcetacoplan has demonstrated its potential to elevate the standard of care in PNH and the submissions of the U.S. and EU marketing applications represent an important step in our efforts to bring the first targeted C3 therapy to people with PNH as quickly as possible," said Federico Grossi, M.D., Ph.D., Chief Medical Officer of Apellis. "Building on our progress with the FDA and EMA, we are also proud that the TGA has granted pegcetacoplan orphan drug designation for the treatment of patients with PNH in Australia."

    The NDA and MAA submissions are based on results from the Phase 3 PEGASUS study, which met its primary endpoint, demonstrating the superiority of pegcetacoplan to eculizumab with a statistically significant improvement in hemoglobin levels at 16 weeks, as well as higher normalization rates across key markers of hemolysis and clinically meaningful improvement in FACIT-fatigue score. The safety profile of pegcetacoplan was comparable to eculizumab in the study. The FDA and EMA decisions on acceptance of the NDA and MAA submissions are expected in Q4 2020.

    In another regulatory milestone, the Australian Therapeutic Goods Administration (TGA) granted orphan drug designation to pegcetacoplan in PNH. This designation in Australia is granted to therapies for serious rare diseases that have the potential to provide a significant benefit in comparison to approved treatments. Apellis plans to submit a marketing application in Australia in the fourth quarter of 2020.

    About Pegcetacoplan (APL-2) 

    Pegcetacoplan is an investigational, targeted C3 inhibitor designed to regulate excessive complement activation, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Apellis is evaluating pegcetacoplan in several clinical studies including paroxysmal nocturnal hemoglobinuria (PNH), geographic atrophy (GA), cold agglutinin disease (CAD), and C3 glomerulopathy (C3G). Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of PNH and the treatment of GA. For additional information regarding our clinical trials, visit https://apellis.com/our-science/clinical-trials.

    About Paroxysmal Nocturnal Hemoglobinuria (PNH) 

    PNH is a rare, chronic, life-threatening blood disorder characterized by the destruction of oxygen-carrying red blood cells through extravascular and intravascular hemolysis. Persistently low hemoglobin can result in frequent transfusions and debilitating symptoms such as severe fatigue and difficulty breathing (dyspnea). Retrospective studies show that, even on eculizumab, approximately 70% of people with PNH have low hemoglobin levels,1,2 and 36% require one or more transfusions a year.2

    About Apellis 

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop best-in-class and first-in-class therapies for a broad range of debilitating diseases that are driven by uncontrolled or excessive activation of the complement cascade, including those within hematology, ophthalmology, and nephrology. For more information, please visit http://apellis.com.

    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 the company's clinical trials will be fully enrolled and completed when anticipated; 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 will be indicative of results that will be generated in future clinical trials; whether pegcetacoplan will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the company's clinical trials will warrant regulatory submissions and whether pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, C3G or any other indication when expected or at all; 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 July 30, 2020 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: 

    Mark Dole 

     

    617.420.4839

    Investor Contact:  

    Sam Martin / Maghan Meyers 

    Argot Partners 

     /  

    212.600.1902

    ____________________________________ 

    1.  Risitano AM, Notaro R, Marando L, et al. (2009) Complement fraction 3 binding on erythrocytes as additional mechanism of disease in paroxysmal nocturnal hemoglobinuria patients treated by eculizumab. Blood. 2009 Apr 23;113(17):4094-100.  

    2.  McKinley C. Extravascular Hemolysis Due to C3-Loading in Patients with PNH Treated with Eculizumab: Defining the Clinical Syndrome. Blood. 2017;130:3471.

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  24. WALTHAM, Mass., Sept. 04, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company pioneering targeted C3 therapies, today announced that the company approved the grant of equity awards to four new employees with grant date of September 1, 2020, as equity inducement awards outside of the company's 2017 Stock Incentive Plan (but under the terms of the 2020 Inducement Stock Incentive Plan) and material to the employees' acceptance of employment with the company. The equity awards were approved on April 30, 2020, July 14, 2020, July 30, 2020, and August 4, 2020, in accordance with Nasdaq Listing Rule 5635(c)(4).

    The employees received options to purchase 151,342 shares of Apellis common stock…

    WALTHAM, Mass., Sept. 04, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company pioneering targeted C3 therapies, today announced that the company approved the grant of equity awards to four new employees with grant date of September 1, 2020, as equity inducement awards outside of the company's 2017 Stock Incentive Plan (but under the terms of the 2020 Inducement Stock Incentive Plan) and material to the employees' acceptance of employment with the company. The equity awards were approved on April 30, 2020, July 14, 2020, July 30, 2020, and August 4, 2020, in accordance with Nasdaq Listing Rule 5635(c)(4).

    The employees received options to purchase 151,342 shares of Apellis common stock. The options have an exercise price of $30.45, which is equal to the closing price of Apellis common stock on September 1, 2020, the grant date of the options. One-fourth of the shares underlying the employee options will vest on the one year anniversary of the grant date and thereafter 1/48th of the shares underlying the employee options will vest monthly, such that the shares underlying the options granted to the employees will be fully vested on the fourth anniversary of the grant date, subject to the employees' continued employment with Apellis on such vesting dates.

    About Apellis

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop best-in-class and first-in-class therapies for a broad range of debilitating diseases that are driven by uncontrolled or excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit http://apellis.com.

    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 the company's clinical trials will be fully enrolled and completed when anticipated; 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 will be indicative of results that will be generated in future clinical trials; whether pegcetacoplan or APL-9 will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the company's clinical trials will warrant regulatory submissions and whether pegcetacoplan or APL-9 will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, C3G, COVID-19 with respiratory failure including ARDS or any other indication when expected or at all; 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 July 30, 2020 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.

    Investor Contact:

    Sam Martin / Maghan Meyers

    Argot Partners

     / 

    212.600.1902

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  25. WALTHAM, Mass., Sept. 02, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) a global biopharmaceutical company pioneering targeted C3 therapies, today announced that the company will participate in the following upcoming investor conferences in September:

    • Baird 2020 Global Healthcare Conference on Wednesday, September 9, 2020 at 2:35 p.m. ET
    • Citi 15th Annual BioPharma Virtual Conference on Thursday, September 10, 2020 at 9:50 a.m ET and 3:20 p.m. ET
    • Cantor Virtual Global Healthcare Conference on Tuesday, September 15, 2020 at 2:40 p.m. ET

    Cedric Francois, M.D., Ph.D., co-founder and chief executive officer of Apellis, will participate in a fireside chat at the Baird 2020 Global Healthcare Conference, an ophthalmology panel…

    WALTHAM, Mass., Sept. 02, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) a global biopharmaceutical company pioneering targeted C3 therapies, today announced that the company will participate in the following upcoming investor conferences in September:

    • Baird 2020 Global Healthcare Conference on Wednesday, September 9, 2020 at 2:35 p.m. ET
    • Citi 15th Annual BioPharma Virtual Conference on Thursday, September 10, 2020 at 9:50 a.m ET and 3:20 p.m. ET
    • Cantor Virtual Global Healthcare Conference on Tuesday, September 15, 2020 at 2:40 p.m. ET

    Cedric Francois, M.D., Ph.D., co-founder and chief executive officer of Apellis, will participate in a fireside chat at the Baird 2020 Global Healthcare Conference, an ophthalmology panel discussion titled "Seeing 20/20 in 2020" at the Citi 15th Annual BioPharma Virtual Conference, a fireside chat at the Citi 15th Annual BioPharma Virtual Conference, and a fireside chat at the Cantor Virtual Global Healthcare Conference.

    The events will be available via live webcast from the "Events and Presentations" page of the "Investors and Media" section of the company's website at https://investors.apellis.com/events-and-presentations. Replays of the webcasts will be available for 90 days following the event.

    About Apellis

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. By pioneering targeted C3 therapies, we aim to develop best-in-class and first-in-class therapies for a broad range of debilitating diseases that are driven by uncontrolled or excessive activation of the complement cascade, including those within hematology, ophthalmology, and nephrology.

    Investor Contact:

    Sam Martin / Maghan Meyers

    Argot Partners

    /

    212.600.1902

    Primary Logo

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  26. WALTHAM, Mass., Aug. 21, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company pioneering targeted C3 therapies, today announced that the company approved the grant of an equity award to a new employee with a grant date of August 17, 2020, as an equity inducement award outside of the company's 2017 Stock Incentive Plan (but under the terms of the 2020 Inducement Stock Incentive Plan) and material to the employee's acceptance of employment with the company. The equity award was approved on April 20, 2020, in accordance with Nasdaq Listing Rule 5635(c)(4).

    The employee received an option to purchase 111,560 shares of Apellis common stock. The option has an exercise price of $29.76, which is…

    WALTHAM, Mass., Aug. 21, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company pioneering targeted C3 therapies, today announced that the company approved the grant of an equity award to a new employee with a grant date of August 17, 2020, as an equity inducement award outside of the company's 2017 Stock Incentive Plan (but under the terms of the 2020 Inducement Stock Incentive Plan) and material to the employee's acceptance of employment with the company. The equity award was approved on April 20, 2020, in accordance with Nasdaq Listing Rule 5635(c)(4).

    The employee received an option to purchase 111,560 shares of Apellis common stock. The option has an exercise price of $29.76, which is equal to the closing price of Apellis common stock on August 17, 2020, the grant date of the option. One-fourth of the shares underlying the employee option will vest on the one year anniversary of the grant date and thereafter 1/48th of the shares underlying the employee option will vest monthly, such that the shares underlying the option granted to the employee will be fully vested on the fourth anniversary of the grant date, subject to the employee's continued employment with Apellis on such vesting dates.

    About Apellis

    Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop best-in-class and first-in-class therapies for a broad range of debilitating diseases that are driven by uncontrolled or excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology. For more information, please visit http://apellis.com.

    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 the company's clinical trials will be fully enrolled and completed when anticipated; 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 will be indicative of results that will be generated in future clinical trials; whether pegcetacoplan or APL-9 will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the company's clinical trials will warrant regulatory submissions and whether pegcetacoplan or APL-9 will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, C3G, COVID-19 with respiratory failure including ARDS or any other indication when expected or at all; 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 July 30, 2020 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.

    Investor Contact:

    Sam Martin / Maghan Meyers

    Argot Partners

     / 

    212.600.1902

    Primary Logo

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  27. WALTHAM, Mass., Aug. 07, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company pioneering targeted C3 therapies, today announced that the company approved the grant of equity awards to two new employees with grant date of August 3, 2020, as equity inducement awards outside of the company's 2017 Stock Incentive Plan (but under the terms of the 2020 Inducement Stock Incentive Plan) and material to the employees' acceptance of employment with the company. The equity awards were approved on March 26, 2020 and March 27, 2020, in accordance with Nasdaq Listing Rule 5635(c)(4).

    The employees received options to purchase 13,400 shares of Apellis common stock. The options have an exercise price of…

    WALTHAM, Mass., Aug. 07, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:<