APLS Apellis Pharmaceuticals Inc.

27.81
+0.71  (+3%)
Previous Close 27.1
Open 27.35
52 Week Low 16.85
52 Week High 45.04
Market Cap $2,102,546,990
Shares 75,603,991
Float 49,294,339
Enterprise Value $1,578,285,156
Volume 298,998
Av. Daily Volume 844,482
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Upcoming Catalysts

Drug Stage Catalyst Date
Pegcetacoplan (APL-2) PEGASUS
Paroxysmal Nocturnal Hemoglobinuria (PNH)
NDA Filing
NDA Filing
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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
APL-2 subcutaneous
Cold agglutinin disease (CAD)
Phase 2
Phase 2
Phase 3 trial plan update due 3Q 2020.
APL-9
COVID-19 / Acute respiratory distress syndrome (ARDS)
Phase 1/2
Phase 1/2
Phase 1/2 initiation announced May 28, 2020. Enrolling patients in the randomized, double-blind, controlled part of the trial - noted July 31, 2020.
APL-2 subcutaneous
Complement-dependent Nephropathies (CDN)
Phase 2
Phase 2
Phase 2 initial data presented at ASN November 8, 2019.

Latest News

  1. 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: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 $27.72, which is equal to the closing price of Apellis common stock on August 3, 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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    • New Drug Application (NDA) and Marketing Authorization Application (MAA) Submissions for Pegcetacoplan in Paroxysmal Nocturnal Hemoglobinuria (PNH) Planned for the Second Half of 2020
    • Phase 3 DERBY and OAKS Trials Evaluating Pegcetacoplan in Geographic Atrophy (GA) Fully Enrolled; Top-line Data Expected in the Third Quarter of 2021
    • PRINCE Trial Evaluating Pegcetacoplan in Treatment-Naïve PNH Patients Fully Enrolled; Top-line Data Expected in the First Half of 2021
    • Cash and Investments of $833 Million as of June 30, 2020

    WALTHAM, Mass., July 31, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced its second quarter 2020 financial results and…

    • New Drug Application (NDA) and Marketing Authorization Application (MAA) Submissions for Pegcetacoplan in Paroxysmal Nocturnal Hemoglobinuria (PNH) Planned for the Second Half of 2020
    • Phase 3 DERBY and OAKS Trials Evaluating Pegcetacoplan in Geographic Atrophy (GA) Fully Enrolled; Top-line Data Expected in the Third Quarter of 2021
    • PRINCE Trial Evaluating Pegcetacoplan in Treatment-Naïve PNH Patients Fully Enrolled; Top-line Data Expected in the First Half of 2021
    • Cash and Investments of $833 Million as of June 30, 2020

    WALTHAM, Mass., July 31, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced its second quarter 2020 financial results and business highlights.

    "This past quarter, Apellis continued to make remarkable progress advancing our targeted C3 therapy, pegcetacoplan, for patients living with serious, complement-driven diseases," said Cedric Francois, M.D., Ph.D., co-founder and chief executive officer of Apellis. "In PNH, we presented detailed results from the Phase 3 PEGASUS study, which showed that pegcetacoplan demonstrated substantial improvements over C5 inhibition and strengthened our belief in the potential of targeting C3 to elevate the standard of care in PNH. Following positive meetings with regulatory agencies, we are on track to submit marketing applications to the FDA and EMA later this year, and we look forward to bringing pegcetacoplan to the PNH community.

    "We also completed enrollment in three Phase 3 trials while ensuring patient safety during the pandemic. These included two pivotal trials in geographic atrophy, a leading cause of blindness that affects five million people worldwide and has no approved treatment. We look forward to top-line results from these studies, as well as from our Phase 3 PRINCE trial in treatment-naïve patients with PNH, in 2021. I am proud of all that the Apellis team continues to accomplish to bring life-changing therapies to people with debilitating diseases."



    Business Highlights and Upcoming Milestones
    :

    Subcutaneous Pegcetacoplan (APL-2)

    • In July 2020, Apellis announced the completion of enrollment in the global Phase 3 PRINCE study, which is evaluating pegcetacoplan in patients with PNH who are treatment-naïve, meaning they had not received a complement inhibitor within three months before entering the trial. The company expects to report top-line results in the first half of 2021.
    • In June 2020, Apellis presented detailed results from the Phase 3 PEGASUS study in an oral presentation at the 25th Congress of the European Hematology Association (EHA). New data from the pivotal study showed that the benefit of pegcetacoplan treatment was consistent across the study population, both in patients who had low or no transfusion requirements and high transfusion requirements. Pegcetacoplan also demonstrated higher normalization rates in key markers of hemolysis and clinically meaningful improvements in FACIT-fatigue score compared to eculizumab at 16 weeks.
    • Apellis plans to submit an NDA to the U.S. Food and Drug Administration (FDA) and a MAA to the European Medicines Agency (EMA) for pegcetacoplan for the treatment of PNH in the second half of the year.
    • The company also expects to report 48-week top-line results from the PEGASUS trial by the end of the year.
    • Apellis plans to disclose the strategy and timing for further clinical development of pegcetacoplan for patients with cold agglutinin disease (CAD) and C3 glomerulopathy (C3G) in the third quarter.

    Intravitreal Pegcetacoplan

    • In July 2020, Apellis announced that it completed enrollment in the Phase 3 DERBY and OAKS studies investigating intravitreal pegcetacoplan for the treatment of GA secondary to age-related macular degeneration (AMD). The company expects to announce top-line results from both trials in the third quarter of 2021.

    COVID-19 Clinical Program

    • In May 2020, Apellis announced that it had initiated a Phase 1/2 clinical study of APL-9 in COVID-19 patients with respiratory failure including acute respiratory distress syndrome. An independent data monitoring committee (DMC) reviewed safety data from the first six patients who received open-label APL-9 and recommended that the study continue. Apellis is now enrolling an additional 60 patients into the randomized, double-blind, controlled part of the study.

    Corporate & Other Highlights:

    • In May 2020, Apellis announced it had entered into subscription agreements with a number of institutional investors for a private placement of $300.0 million aggregate principal amount of 3.500% convertible senior notes due in 2026, for an aggregate purchase price of $328.9 million.

    Second Quarter 2020 Financial Results:

    As of June 30, 2020, Apellis had $833 million in cash, cash equivalents, and short-term marketable securities, compared to $352 million in cash and cash equivalents as of December 31, 2019.

    Apellis reported a net loss of $118.6 million for the second quarter of 2020, compared to a net loss of $71.1 million for the second quarter of 2019.

    Research and development expenses were $87.1 million in the second quarter of 2020, compared to $50.7 million for the same period in 2019. The increase was primarily attributable to an increase of $8.8 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 $11.0 million in personnel-related costs primarily due to the hiring of additional personnel, an increase of $4.6 million related to research and innovation activities, and an increase of $6.2 million in other development costs, and offset by a decrease of $0.9 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 subcutaneous pegcetacoplan.

    General and administrative expenses were $28.4 million in the second quarter of 2020, compared to $12.8 million for the same period in 2019. The increase was primarily attributable to an increase in employee-related costs of $9.9 million, an increase in professional and consulting fees and general commercial preparation activities of $5.7 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 $0.7 million. The increase in employee-related costs of $9.9 million consisted of a $6.1 million increase in salaries and benefits primarily due to the increase in the number of employees, $3.6 million related to stock expense associated with the grant of stock options and restricted stock units to employees, and $0.2 million in recruitment expense. The increase in other professional and consulting fees and general commercial preparation activities of $5.7 million primarily related to an increase in commercial-related activity of $4.8 million, an increase of $0.9 million in general consulting fees, a $0.3 million increase in accounting, an increase in communication and public relations fees of $0.1 million, and offset by a decrease of $0.4 million legal fees.

    About Pegcetacoplan (APL-2)

    Pegcetacoplan is the only investigational targeted C3 therapy in late-stage clinical trials. It is designed to control 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 geographic atrophy (GA), paroxysmal nocturnal hemoglobinuria (PNH), cold agglutinin disease, and C3 glomerulopathy. Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of GA and the treatment of PNH. For additional information regarding our clinical trials, visit https://apellis.com/our-science/clinical-trials/.

    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, 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 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

    Media Contact:

    Tracy Vineis



    617.420.4839





    APELLIS PHARMACEUTICALS, INC.   
    CONDENSED CONSOLIDATED BALANCE SHEETS   
    (Amounts in thousands, except per share amounts)   
      June 30, December 31, 
       2020   2019  
    Assets (Unaudited)   
    Current assets:     
    Cash and cash equivalents $317,348  $351,985  
    Marketable Securities  515,476   -  
    Prepaid assets  17,925   19,802  
    Restricted Cash  1,248   -  
    Other current assets  1,828   1,308  
    Total current assets  853,825   373,095  
    Non-current Assets:     
    Right-of-use assets  12,959   14,110  
    Property and equipment, net  2,416   1,655  
    Other assets  906   385  
    Total assets $870,106  $389,245  
    Liabilities and Stockholders' Equity     
    Current liabilities:     
    Accounts payable $9,927  $8,361  
    Accrued expenses  45,399   54,783  
    Current portion of right of use liabilities  2,664   2,609  
    Total current liabilities  57,990   65,753  
    Long-term liabilities:     
    Convertible senior notes  348,842   142,567  
    Development derivative liability  220,475   134,839  
    Operating lease liabilities  10,735   11,857  
    Total liabilities  638,042   355,016  
    Stockholders' equity:     
    Preferred stock, $0.0001 par value; 10.0 million shares authorized, and zero shares issued and outstanding at June 30, 2020 and December 31, 2019  -   -  
    Common stock, $0.0001 par value; 200.0 million shares authorized at June 30, 2020 and December 31, 2019; 75.6 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  1,100,639   615,850  
    Accumulated other comprehensive loss  329   (154) 
    Accumulated deficit  (868,912)  (581,473) 
    Total stockholders' equity  232,064   34,229  
    Total liabilities and stockholders' equity $870,106  $389,245  
          





    APELLIS PHARMACEUTICALS, INC.       
    CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS       
            
      For the Three Months Ended June 30, For the Six Months Ended June 30,
       2020   2019   2020   2019 
                     
      (Unaudited)
    Operating expenses:        
    Research and development $87,094  $50,698  $156,377  $91,178 
    General and administrative  28,414   12,778   57,918   20,949 
    Operating loss  (115,508)  (63,476)  (214,295)  (112,127)
    Loss on extinguishment of debt  -   -   -   (1,208)
    Gain/(loss) from remeasurement of development derivative liability  2,770   (9,104)  (65,636)  (9,840)
    Interest income  1,025   1,421   3,300   2,288 
    Interest expense  (6,909)  (159)  (10,828)  (753)
    Other (expense)/income, net  5   228   20   (25)
    Net loss  (118,617)  (71,090)  (287,439)  (121,665)
    Other comprehensive gain/(loss):        
      Unrealized gain on marketable securities $(842) $-  $552  $- 
      Foreign currency gain/(loss)  161   (1)  (69)  1 
    Total other comprehensive gain/(loss)  (681)  (1)  483   1 
    Comprehensive loss, net of tax $(119,298) $(71,091) $(286,956) $(121,664)
    Net loss per common share, basic and diluted $(1.57) $(1.12) $(3.85) $(2.01)
    Weighted-average number of common shares used in net loss per common share, basic and diluted  75,550   63,264   74,635   60,581 
             

     

     

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  2. WALTHAM Mass., July 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 three new employees with a grant date of July 1, 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 employees' acceptance of employment with the company. The equity awards were approved on February 19, 2020, February 21, 2020 and May 13, 2020, in accordance with Nasdaq Listing Rule 5635(c)(4).

    The employees received options to purchase 43,400 shares of Apellis common stock. The options…

    WALTHAM Mass., July 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 three new employees with a grant date of July 1, 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 employees' acceptance of employment with the company. The equity awards were approved on February 19, 2020, February 21, 2020 and May 13, 2020, in accordance with Nasdaq Listing Rule 5635(c)(4).

    The employees received options to purchase 43,400 shares of Apellis common stock. The options have an exercise price of $31.96, which is equal to the closing price of Apellis common stock on July 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. 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.

    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 April 29, 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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    • Pivotal Phase 3 DERBY and OAKS studies enrolled a total of 1,259 patients
       
    • Top-line results expected in Q3 2021
       
    • Pegcetacoplan targets C3 to control the irreversible lesion growth in GA, a leading cause of blindness

    WALTHAM Mass., July 07, 2020 (GLOBE NEWSWIRE) --  Apellis Pharmaceuticals Inc., (NASDAQ:APLS), a global biopharmaceutical company pioneering targeted C3 therapies, today announced that it completed enrollment in the Phase 3 DERBY and OAKS studies investigating intravitreal pegcetacoplan (APL-2), a targeted C3 therapy, for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). GA causes blindness and is a progressive complement-driven eye disease1,2 that affects approximately five million people…

    • Pivotal Phase 3 DERBY and OAKS studies enrolled a total of 1,259 patients

       
    • Top-line results expected in Q3 2021

       
    • Pegcetacoplan targets C3 to control the irreversible lesion growth in GA, a leading cause of blindness

    WALTHAM Mass., July 07, 2020 (GLOBE NEWSWIRE) --  Apellis Pharmaceuticals Inc., (NASDAQ:APLS), a global biopharmaceutical company pioneering targeted C3 therapies, today announced that it completed enrollment in the Phase 3 DERBY and OAKS studies investigating intravitreal pegcetacoplan (APL-2), a targeted C3 therapy, for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). GA causes blindness and is a progressive complement-driven eye disease1,2 that affects approximately five million people globally.3,4 There are no approved therapies for GA.

    "Completing enrollment in the Phase 3 DERBY and OAKS studies is a critical milestone that moves us one step closer to bringing the first treatment for geographic atrophy to patients. We are extremely grateful to all patients participating in these groundbreaking studies," said Federico Grossi, M.D., Ph.D., Chief Medical Officer of Apellis. "People living with GA need a treatment and we believe targeting the complement system at C3 with pegcetacoplan has the potential to control the excessive complement activation that drives the irreversible growth of GA lesions that cause blindness. We look forward to seeing top-line results in the third quarter of 2021."

    A total of 1,259 patients are enrolled in DERBY and OAKS, which are pivotal randomized Phase 3 studies designed to compare the efficacy and safety of intravitreal pegcetacoplan with sham treatment in patients with GA secondary to AMD. The primary objective of the studies is to evaluate the reduction in growth of GA lesion size, measured by fundus autofluorescence (FAF) at month 12 compared to baseline.

    "Geographic atrophy leads to irreversible vision loss, a decline in the quality of life, and remains a major unmet need for our patients. Achieving this critical milestone reflects the importance of bringing this targeted C3 therapy to our GA patients. I want to thank my co-investigators for completing the enrollment of DERBY and OAKS, especially during the ongoing pandemic. I look forward to seeing the results next year with the hope that pegcetacoplan will enable clinicians to treat this debilitating disease," said Jeffrey S. Heier, MD, Principal Investigator of the DERBY study and Co-President & Medical Director, Director of Retinal Research, Ophthalmic Consultants of Boston.

    In July 2018, the U.S. Food and Drug Administration (FDA) granted pegcetacoplan Fast Track Designation for the treatment of GA. The Phase 3 DERBY and OAKS studies were initiated based on positive results from the Phase 2 FILLY study, which showed that monthly pegcetacoplan treatment resulted in a 29% (p=0.008) reduction in the rate of GA lesion growth compared to sham injections at 12 months.

    For more information about the DERBY and OAKS studies, visit www.clinicaltrials.gov: (NCT03525600) and (NCT03525613).

    About Pegcetacoplan (APL-2)

    Pegcetacoplan is the only investigational targeted C3 therapy in late-stage clinical trials. It is designed to control 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 geographic atrophy (GA), paroxysmal nocturnal hemoglobinuria (PNH), cold agglutinin disease, and C3 glomerulopathy. Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of GA and the treatment of PNH. 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), the leading cause of blindness. Excessive complement activation drives the 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 1500 patients, is the only targeted C3 inhibitor being evaluated in patients 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 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 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 compared to sham.

    In this study, pegcetacoplan met its primary endpoint of reducing the growth rate of the GA lesion (measured as square root transformation of GA lesion area) compared to sham after 12 months of treatment.  Pegcetacoplan administered monthly via intravitreal injection showed a 29% (p=0.008) reduction in the rate of GA lesion growth compared to sham after 12 months of treatment. With every other month administration of pegcetacoplan, a 20% (p=0.067) reduction was observed compared to sham. Statistical significance was defined as p<0.1 for this study.

    Pegcetacoplan was generally well-tolerated in the Phase 2 FILLY trial. Over the full 18-month study period, a total of 26 cases of exudative AMD were reported by the investigators. These were seen more frequently in the pegcetacoplan-treated patients (18 in the monthly treatment group, 7 in the every other month treatment group and 1 in the sham control group). No negative impact on visual acuity was observed. The results of the FILLY study were published in Ophthalmology, the journal of the American Academy of Ophthalmology.

    About Apellis

    Apellis Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development of novel therapeutic compounds for the treatment of a broad range of life-threatening or debilitating autoimmune diseases based upon complement immunotherapy through the inhibition of the complement system at the level of C3. Apellis is the first company to advance chronic therapy with a C3 inhibitor into clinical trials. For additional information about Apellis and pegcetacoplan, please visit http://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 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 April 29, 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.

    6Yates, 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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    • PRINCE study enrolled 53 patients. Trial designed to further establish the potential of pegcetacoplan, a targeted C3 therapy, in all patients living with PNH

    • Top-line results expected in early 2021

    WALTHAM, Mass., July 02, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company pioneering targeted C3 therapies, today announced the completion of enrollment in the global Phase 3 PRINCE study, which is evaluating pegcetacoplan (APL-2) in patients with paroxysmal nocturnal hemoglobinuria (PNH) who are treatment-naïve, meaning they had not received a complement inhibitor within three months before entering the trial.

    "Patient safety during this global pandemic has been our top priority as we worked…

    • PRINCE study enrolled 53 patients. Trial designed to further establish the potential of pegcetacoplan, a targeted C3 therapy, in all patients living with PNH



    • Top-line results expected in early 2021

    WALTHAM, Mass., July 02, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company pioneering targeted C3 therapies, today announced the completion of enrollment in the global Phase 3 PRINCE study, which is evaluating pegcetacoplan (APL-2) in patients with paroxysmal nocturnal hemoglobinuria (PNH) who are treatment-naïve, meaning they had not received a complement inhibitor within three months before entering the trial.

    "Patient safety during this global pandemic has been our top priority as we worked to finalize enrollment in this trial, and we'd like to thank the patients and their families for participating in the PRINCE study as well as investigators for helping to ensure the well-being of all study participants," said Federico Grossi, M.D., Ph.D., Chief Medical Officer. "PNH patients can suffer debilitating fatigue and transfusion dependence even when treated with currently available therapies. Based on positive data announced earlier this year, we believe pegcetacoplan, our targeted C3 therapy, has the potential to redefine treatment for all patients with PNH by controlling both intravascular and extravascular hemolysis."

    The PRINCE study is a Phase 3, randomized, multicenter, open-label, controlled trial. The study was designed to evaluate the efficacy of pegcetacoplan in 48 adult patients who are treatment-naïve, showed evidence of hemolysis (elevated LDH ≥ 1.5x ULN) and had hemoglobin levels that were less than the lower limit of normal at the time of their screening. Primary outcome measures, to be evaluated after 26 weeks of treatment, include hemoglobin stabilization in the absence of transfusion and reduction in lactate dehydrogenase (LDH) level. A total of 53 patients were enrolled in the study.

    Based on positive results from the pivotal PEGASUS study of pegcetacoplan in PNH, the company plans to submit marketing applications for pegcetacoplan for the treatment of PNH both in the United States and the European Union in the second half of 2020.

    For more information about the Phase 3 PRINCE study, visit www.clinicaltrials.gov (NCT04085601).

    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, and C3 glomerulopathy. 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 www.apellis.com/clinical-trials.html.1

    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. 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. 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 April 29, 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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