APLS Apellis Pharmaceuticals Inc.

53.06
+8.8  (+20%)
Previous Close 44.26
Open 49.01
52 Week Low 25.49
52 Week High 58.47
Market Cap $4,271,158,669
Shares 80,496,771
Float 53,787,386
Enterprise Value $3,249,078,084
Volume 6,234,599
Av. Daily Volume 791,174
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Upcoming Catalysts

Drug Stage Catalyst Date
APL-2 PRINCE
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Phase 3
Phase 3
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APL-2 (pegcetacoplan) - DERBY
Geographic atrophy (GA)
Phase 3
Phase 3
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APL-2 OAKS
Geographic atrophy (GA) associated with age-related macular degeneration (AMD)
Phase 3
Phase 3
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Drug Pipeline

Drug Stage Notes
Empaveli (pegcetacoplan)
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Approved
Approved
FDA approval announced May 14, 2021.
APL-2 subcutaneous
Cold agglutinin disease (CAD)
Phase 2
Phase 2
Phase 3 trial to commence 2H 2021.
Pegcetacoplan
C3G and IC-MPGN
Phase 2
Phase 2
Phase 3 trial to commence 2H 2021.
APL-9
COVID-19 / Acute respiratory distress syndrome (ARDS)
Phase 1/2
Phase 1/2
Phase 1/2 trial to be discontinued following Independent data monitoring committee (DMC) review which found no meaningful reduction in the overall mortality rate.
Pegcetacoplan (MERIDIAN)
Amyotrophic lateral sclerosis (ALS)
Phase 2
Phase 2
Phase 2 initiation of dosing announced November 19, 2020.
APL-2 subcutaneous
Complement-dependent Nephropathies (CDN)
Phase 2
Phase 2
Phase 2 initial data presented at ASN November 8, 2019.

Latest News

    • 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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  1. 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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  2. 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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