APLS Apellis Pharmaceuticals Inc.

35.46
-2.17  -6%
Previous Close 37.63
Open 36
52 Week Low 27.5
52 Week High 73
Market Cap $3,452,310,106
Shares 97,357,871
Float 70,648,486
Enterprise Value $3,233,831,105
Volume 1,405,342
Av. Daily Volume 1,084,292
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Upcoming Catalysts

Drug Stage Catalyst Date
EMPAVELI (pegcetacoplan) - (OAKS)
Geographic atrophy (GA) associated with age-related macular degeneration (AMD)
NDA Filing
NDA Filing
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Drug Pipeline

Drug Stage Notes
EMPAVELI (pegcetacoplan) - (MERIDIAN)
Amyotrophic lateral sclerosis (ALS)
Phase 2
Phase 2
Phase 2 enrollment to be completed by 1H 2022.
EMPAVELI (pegcetacoplan)
C3G and IC-MPGN
Phase 3
Phase 3
Phase 3 trial to commence in 1Q 2022
EMPAVELI (pegcetacoplan) - (PRINCE)
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Phase 3
Phase 3
Phase 3 demonstrated clinically meaningful improvements in key markers of disease, noted November 4, 2021. Phase 3 results reported 46% of patients achieved hemoglobin normalization in the absence of transfusions vs. 0% for standard of care and 91% achieved transfusion avoidance vs. 6% for standard of care, noted December 13, 2021.
EMPAVELI (pegcetacoplan) - (DERBY)
Geographic atrophy (GA)
NDA Filing
NDA Filing
NDA submission due in 2Q 2022. NDA will be submitted in conjunction with OAKS trial.
EMPAVELI (pegcetacoplan) subcutaneous
Cold agglutinin disease (CAD)
Phase 3
Phase 3
Phase 3 trial to commence 1Q 2022.
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.
EMPAVELI (pegcetacoplan)
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Approved
Approved
FDA approval announced May 14, 2021.
EMPAVELI (pegcetacoplan) subcutaneous
Complement-dependent Nephropathies (CDN)
Phase 2
Phase 2
Phase 2 initial data presented at ASN November 8, 2019.

Latest News

  1. WALTHAM, Mass., Jan. 06, 2022 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in complement, today announced that the company approved the grant of equity awards to one new employee with a grant date of January 3, 2022, as equity inducement awards outside of the company's 2017 Stock Incentive Plan (but under the terms of the 0 Inducement Stock Incentive Plan) and material to the employee's acceptance of employment with the company. The equity awards were approved in accordance with Nasdaq Listing Rule 5635(c)(4).

    The employee received options to purchase 60,000 shares of Apellis common stock and 20,000 restricted stock units (RSUs). The options have an exercise price of $48.77…

    WALTHAM, Mass., Jan. 06, 2022 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in complement, today announced that the company approved the grant of equity awards to one new employee with a grant date of January 3, 2022, as equity inducement awards outside of the company's 2017 Stock Incentive Plan (but under the terms of the 0 Inducement Stock Incentive Plan) and material to the employee's acceptance of employment with the company. The equity awards were approved in accordance with Nasdaq Listing Rule 5635(c)(4).

    The employee received options to purchase 60,000 shares of Apellis common stock and 20,000 restricted stock units (RSUs). The options have an exercise price of $48.77, which is equal to the closing price of Apellis common stock on January 3, 2022, 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 employee will be fully vested on the fourth anniversary of the grant date, subject to the employee's continued employment with Apellis on such vesting dates. 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:

    Meredith Kaya



    617.599.8178 



    Primary Logo

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  2. WALTHAM, Mass., Jan. 03, 2022 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in complement, today announced that the company will present virtually at the 40th Annual J.P. Morgan Healthcare Conference on Monday, January 10, 2022 at 8:15 a.m. ET.

    The presentation and question and answer session will be available via a live webcast from the "Events and Presentations" page of the "Investors and Media" section of the company's website. A replay of the webcast will be available for 30 days following the event. A copy of the presentation slides will be also posted on the company's website beginning on Monday, January 10, 2022, at 8:00 a.m. ET.

    About Apellis
    Apellis Pharmaceuticals…

    WALTHAM, Mass., Jan. 03, 2022 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS), a global biopharmaceutical company and leader in complement, today announced that the company will present virtually at the 40th Annual J.P. Morgan Healthcare Conference on Monday, January 10, 2022 at 8:15 a.m. ET.

    The presentation and question and answer session will be available via a live webcast from the "Events and Presentations" page of the "Investors and Media" section of the company's website. A replay of the webcast will be available for 30 days following the event. A copy of the presentation slides will be also posted on the company's website beginning on Monday, January 10, 2022, at 8:00 a.m. ET.

    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:

    Meredith Kaya

     

    617.599.8178



    Primary Logo

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  3. STOCKHOLM, Dec. 15, 2021 /PRNewswire/ --

    • The first targeted C3 therapy approved in the EU
    • Approval based on results from head-to-head PEGASUS phase 3 study where Aspaveli demonstrated superiority to eculizumab in improving haemoglobin levels1
    • Aspaveli will have market exclusivity for paroxysmal nocturnal haemoglobinuria (PNH) based on orphan drug designation

    Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) and Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) announced today that the European Commission (EC) has approved Aspaveli® (pegcetacoplan), the first and only targeted C3 therapy, for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH) who are anaemic after treatment with a C5 inhibitor for at least three months. Based…

    STOCKHOLM, Dec. 15, 2021 /PRNewswire/ --

    • The first targeted C3 therapy approved in the EU
    • Approval based on results from head-to-head PEGASUS phase 3 study where Aspaveli demonstrated superiority to eculizumab in improving haemoglobin levels1
    • Aspaveli will have market exclusivity for paroxysmal nocturnal haemoglobinuria (PNH) based on orphan drug designation

    Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) and Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) announced today that the European Commission (EC) has approved Aspaveli® (pegcetacoplan), the first and only targeted C3 therapy, for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH) who are anaemic after treatment with a C5 inhibitor for at least three months. Based on the recommendation from the European Medicines Agency's Committee for Orphan Medicinal Products, the EC determined that pegcetacoplan continues to meet the criteria for the orphan drug designation status granted in 2017 for the treatment of PNH.

    PNH is a rare, chronic and life-threatening blood disorder where uncontrolled complement activation leads to the destruction of oxygen-carrying red blood cells through intravascular haemolysis and extravascular haemolysis. Characterised by persistently low haemoglobin, PNH can result in frequent transfusions and debilitating symptoms such as severe fatigue caused by anaemia. Despite improvements in haemolytic activity with C5 inhibitor treatment, approximately 72 per cent of people with PNH treated with C5 inhibitors remain anaemic, according to a retrospective and a cross-sectional study.2,3

    "The European Commission's approval of Aspaveli is a milestone for people living with PNH across Europe," said," Guido Oelkers, CEO and President at Sobi. "The symptoms of PNH can significantly impact quality of life. In addition, despite current therapy, many people still require frequent blood transfusions. We are now working with EU member states to provide access to this important medicine as quickly as possible."

    "As the first and only targeted C3 therapy in Europe, Aspaveli has the potential to elevate the standard of care for patients living with PNH," said Federico Grossi, MD, PhD, Chief Medical Officer of Apellis. "Today's approval represents the first new class of complement medicines in Europe in over a decade, building on the launch of this important treatment in the United States."

    The approval is based on the results from the head-to-head PEGASUS phase 3 study, which evaluated the efficacy and safety of Aspaveli compared to eculizumab at 16 weeks in adults with PNH who had persistent anaemia despite treatment with eculizumab. The full safety and efficacy results were published in The New England Journal of Medicine in March 2021.1

    Orphan drug designation is granted to therapies that treat a serious disease that affects fewer than five in 10,000 people in the EU and provide a significant benefit over existing treatments. Aspaveli will have market exclusivity based on orphan drug designation for PNH.

    About Aspaveli®/Empaveli™

    Aspaveli/Empaveli (pegcetacoplan) is a 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. Aspaveli is approved in the EU as an orphan drug for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH) who are anaemic after treatment with a C5 inhibitor for at least three months and in the United States as Empaveli for the treatment of adults with PNH. The therapy is also under investigation for several other rare diseases across haematology, nephrology, and neurology.

    About the Sobi and Apellis Collaboration

    Sobi and Apellis have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-US commercialisation rights for systemic pegcetacoplan, and Apellis has exclusive US commercialisation rights for systemic pegcetacoplan and retains worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy (GA).

    About Apellis

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

    About Sobi™

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

    Contacts Sobi

    To contact the Sobi Investor Relations Team, click here. For Sobi Media contacts, click here.

    Apellis

    Media:

    Lissa Pavluk



    +1.617.977.6764

    Investors:

    Meredith Kaya



    +1.617.599.8178

    _________________________________________________

    Reference

    1.  Hillmen P, Szer J, Weitz I, et al. Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria. N Engl J Med. DOI: 10.1056/NEJMoa2029073.

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

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

    4.  Risitano et al. 2009.

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

    https://news.cision.com/swedish-orphan-biovitrum-ab/r/aspaveli---pegcetacoplan--approved-in-eu-as-orphan-drug-for-treatment-of-pnh,c3472815

    The following files are available for download:

    https://mb.cision.com/Main/14266/3472815/1510273.pdf

    Aspaveli® (pegcetacoplan) approved in EU as orphan drug for treatment of PNH

    Cision View original content:https://www.prnewswire.com/news-releases/aspaveli-pegcetacoplan-approved-in-eu-as-orphan-drug-for-treatment-of-pnh-301445216.html

    SOURCE Swedish Orphan Biovitrum AB

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    • The first targeted C3 therapy approved in the EU
    • Approval based on results from head-to-head PEGASUS Phase 3 study where Aspaveli demonstrated superiority to eculizumab in improving hemoglobin levels1
    • Aspaveli will have market exclusivity for paroxysmal nocturnal hemoglobinuria (PNH) based on orphan drug designation

    WALTHAM, Mass. and STOCKHOLM, Sweden, Dec. 15, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) and Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) announced today that the European Commission (EC) has approved Aspaveli® (pegcetacoplan), the first and only targeted C3 therapy, for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) who are anemic after treatment with a C5 inhibitor…

    • The first targeted C3 therapy approved in the EU
    • Approval based on results from head-to-head PEGASUS Phase 3 study where Aspaveli demonstrated superiority to eculizumab in improving hemoglobin levels1
    • Aspaveli will have market exclusivity for paroxysmal nocturnal hemoglobinuria (PNH) based on orphan drug designation

    WALTHAM, Mass. and STOCKHOLM, Sweden, Dec. 15, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) and Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) announced today that the European Commission (EC) has approved Aspaveli® (pegcetacoplan), the first and only targeted C3 therapy, for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) who are anemic after treatment with a C5 inhibitor for at least three months. Based on the recommendation from the European Medicines Agency's Committee for Orphan Medicinal Products, the EC determined that pegcetacoplan continues to meet the criteria for the orphan drug designation status granted in 2017 for the treatment of PNH.

    PNH is a rare, chronic and life-threatening blood disorder where uncontrolled complement activation leads to the destruction of oxygen-carrying red blood cells through intravascular hemolysis and extravascular hemolysis. Characterized by persistently low hemoglobin, PNH can result in frequent transfusions and debilitating symptoms such as severe fatigue caused by anemia. Despite improvements in hemolytic activity with C5 inhibitor treatment, approximately 72% of people with PNH treated with C5 inhibitors remain anemic, according to a retrospective and a cross-sectional study.2,3

    "The European Commission's approval of Aspaveli is a milestone for people living with PNH across Europe," said Guido Oelkers, chief executive officer and president at Sobi. "The symptoms of PNH can significantly impact quality of life. In addition, despite current therapy, many people still require frequent blood transfusions. We are now working with EU member states to provide access to this important medicine as quickly as possible."

    "As the first and only targeted C3 therapy in Europe, Aspaveli has the potential to elevate the standard of care for patients living with PNH," said Federico Grossi, M.D., Ph.D., chief medical officer of Apellis. "Today's approval represents the first new class of complement medicines in Europe in over a decade, building on the launch of this important treatment in the United States."

    The approval is based on the results from the head-to-head PEGASUS Phase 3 study, which evaluated the efficacy and safety of Aspaveli compared to eculizumab at 16 weeks in adults with PNH who had persistent anaemia despite treatment with eculizumab. The full safety and efficacy results were published in The New England Journal of Medicine in March 2021.1

    Orphan drug designation is granted to therapies that treat a serious disease that affects fewer than five in 10,000 people in the EU and provide a significant benefit over existing treatments. Aspaveli will have market exclusivity based on orphan drug designation for PNH.

    About Aspaveli®/Empaveli™

    Aspaveli®/Empaveli™ (pegcetacoplan) is a 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. Aspaveli is approved in the EU as an orphan drug for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) who are anemic after treatment with a C5 inhibitor for at least three months and in the United States as EMPAVELI for the treatment of adults with PNH. The therapy is also under investigation for several other rare diseases across hematology, nephrology, and neurology.

    About the Sobi and Apellis Collaboration

    Sobi and Apellis have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-US commercialization rights for systemic pegcetacoplan, and Apellis has exclusive US commercialization rights for systemic pegcetacoplan and retains worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy (GA).

    About Apellis 

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

    About Sobi™

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

    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 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 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 8, 2021 and the risks described in other filings that Apellis may make with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Apellis specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

    Contacts:



    Apellis

    Media:

    Lissa Pavluk 

     

    617.977.6764



    Investors: 

    Meredith Kaya



    617.599.8178

    Sobi

    To contact the Sobi Investor Relations Team, click here. For Sobi Media contacts, click here.

    References

    1. Hillmen P, Szer J, Weitz I, et al. Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria. N Engl J Med. DOI: 10.1056/NEJMoa2029073.
    2. McKinley C. Extravascular Hemolysis Due to C3-Loading in Patients with PNH Treated with Eculizumab: Defining the Clinical Syndrome. Blood. 2017;130:3471.
    3. Dingli ASH 2020 Abstract/ p.1/ Methods/ ln.1-2; p.2/ Results/ln.7-9; ln.14-1 


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    • Improvements demonstrated in treatment-naïve patients and patients with baseline hemoglobin levels greater than or equal to 10.0 g/dL
    • Data were presented at the American Society of Hematology (ASH) Annual Meeting

    WALTHAM, Mass. and STOCKHOLM, Sweden, Dec. 13, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) and Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) announced today new data demonstrating that Empaveli™/Aspaveli® (pegcetacoplan) provides consistent, sustained normalization of clinical measures across patients with paroxysmal nocturnal hemoglobinuria (PNH) who are treatment-naïve and patients with baseline hemoglobin levels greater than or equal to 10.0 g/dL. The data were presented at the American Society…

    • Improvements demonstrated in treatment-naïve patients and patients with baseline hemoglobin levels greater than or equal to 10.0 g/dL
    • Data were presented at the American Society of Hematology (ASH) Annual Meeting

    WALTHAM, Mass. and STOCKHOLM, Sweden, Dec. 13, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) and Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) announced today new data demonstrating that Empaveli™/Aspaveli® (pegcetacoplan) provides consistent, sustained normalization of clinical measures across patients with paroxysmal nocturnal hemoglobinuria (PNH) who are treatment-naïve and patients with baseline hemoglobin levels greater than or equal to 10.0 g/dL. The data were presented at the American Society of Hematology Annual Meeting (ASH) taking place December 11 - 14, 2021.

    Sustained Normalization and Superior Improvements of Clinical Measures in Treatment-Naïve Patients

    New data from the Phase 3 PRINCE study in treatment-naïve patients with PNH showed that treatment with EMPAVELI resulted in sustained and superior improvements in the co-primary endpoints of hemoglobin stabilization through Week 26 and reduction in lactate dehydrogenase (LDH) compared to standard of care, which did not include complement inhibitors, at Week 26.

    Improvements were seen as early as two weeks after starting treatment with EMPAVELI and patients showed sustained normalization across key markers of disease through Week 26:

    • 46% of EMPAVELI patients achieved hemoglobin normalization in the absence of transfusions vs. 0% for standard of care (p<0.0010), reaching a mean hemoglobin level of 12.8 g/dL from a mean baseline of 9.4 g/dL
    • Mean LDH levels rapidly fell from 9.5x the upper limit of normal (ULN) to below 1.5x the ULN by Week 2, normalized by Week 4, and were maintained through Week 26 with EMPAVELI
    • 91% of EMPAVELI patients achieved transfusion avoidance vs. 6% for standard of care (p<0.0001), demonstrating superiority

    The safety profile of EMPAVELI was consistent with previous studies. At Week 26, 9% of patients in the EMPAVELI group experienced a serious adverse event (SAE) compared to 17% on standard of care. No cases of thrombosis or meningococcal infection were reported in either group. The most common adverse events reported during the study in the EMPAVELI and standard of care groups, respectively, were injection site reaction (30% vs. 0%), hypokalemia (13% vs.11%), dizziness (11% vs. 0%), and fever (9% vs. 0%).

    "The data presented at ASH add to a robust body of evidence that underscores the consistent efficacy and safety of EMPAVELI across a broad range of adults with PNH," said Federico Grossi, M.D., Ph.D., chief medical officer of Apellis. "EMPAVELI has the potential to elevate the standard of care for adults with PNH regardless of prior treatment or baseline hemoglobin levels."

    Clinically Meaningful Improvements in Patients with Near-Normal Baseline Hemoglobin Levels

    A new post hoc analysis across studies from the EMPAVELI PNH clinical development program showed that EMPAVELI-treated patients with baseline hemoglobin levels greater than or equal to 10.0 g/dL demonstrated clinically meaningful improvements across key markers of disease. The analysis included data from patients who were treatment-naïve and patients that remained anemic despite stable treatment with eculizumab, a C5 inhibitor.

    Detailed data showed:

    • EMPAVELI increased mean hemoglobin levels to 13.9 g/dL, 12.1 g/dL, and 12.7 g/dL from a mean baseline of 11.3 g/dL, 10.2 g/dL, and 10.4 g/dL in the PRINCE, PEGASUS, and PADDOCK studies, respectively
    • EMPAVELI demonstrated mean improvements from baseline in the Functional Assessment of Chronic Illness Therapy (FACIT)-fatigue score of 9.9 points, 13.3 points, and 6.6 points in the PRINCE, PEGASUS, and PADDOCK studies. A three-point improvement is generally considered to be clinically meaningful

    "The presented data reinforce the efficacy and safety profile of pegcetacoplan in PNH," said Ravi Rao, head of research and development and chief medical officer at Sobi. "We are committed to improve care, and to make a difference in the lives of people with this rare blood disease."

    About the PRINCE Study

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

    About the PEGASUS Study

    The PEGASUS study (NCT03500549) was 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 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 EMPAVELI 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 EMPAVELI twice weekly or their current dose of eculizumab (n=39). All participants completing the randomized controlled period (n=77) opted to enter the open-label EMPAVELI treatment period.

    About the PADDOCK Study

    PADDOCK (NCT02588833) was a multicenter, open-label, multiple ascending dose, Phase 1b study in 23 adults with paroxysmal nocturnal hemoglobinuria (PNH) who have never received eculizumab. The primary objective of this study, designed with two cohorts, was to establish the safety and efficacy of 270 mg of EMPAVELI administered daily by subcutaneous injection in adults with PNH. Patients in Cohort One received a suboptimal dose of 180 mg of EMPAVELI once daily for 28 days and subjects in Cohort Two received 270 mg of EMPAVELI once daily for up to one year.

    About Empaveli™/Aspaveli® (pegcetacoplan)

    Empaveli™/Aspaveli® (pegcetacoplan) is a 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. EMPAVELI is approved in the United States for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH). The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion for Aspaveli, which is the European trade name for pegcetacoplan, for the treatment of adults with PNH who are anemic after treatment with a C5 inhibitor for at least three months. The positive opinion from the CHMP is now referred to the European Commission for an approval decision. The therapy is also under investigation for several other rare diseases across hematology, nephrology, and neurology.

    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 pneumoniaeNeisseria 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 pneumoniaeNeisseria meningitidis types A, C, W, Y, and B, and Haemophilus influenzae type B (Hib). To reduce the risk of infection, all patients must be vaccinated against these bacteria according to the most current ACIP recommendations for patients with altered immunocompetence associated with complement deficiencies. Revaccinate patients in accordance with ACIP recommendations considering the duration of therapy with EMPAVELI.

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

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

    EMPAVELI REMS

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

    Infusion-Related Reactions

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

    Monitoring PNH Manifestations after Discontinuation of EMPAVELI

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

    Interference with Laboratory Tests

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

    ADVERSE REACTIONS

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

    USE IN SPECIFIC POPULATIONS

    Females of Reproductive Potential

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

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

    About Paroxysmal Nocturnal Hemoglobinuria (PNH)

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

    About the Apellis and Sobi Collaboration

    Sobi and Apellis have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-US commercialisation rights for systemic pegcetacoplan, and Apellis has exclusive US commercialisation rights for systemic pegcetacoplan and retains worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy (GA).

    About Apellis

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

    About Sobi™

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

    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 the factors discussed in the "Risk Factors" section of Apellis' Quarterly Report on Form 10-Q with the Securities and Exchange Commission on November 8, 2021 and the risks described in other filings that Apellis may make with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Apellis specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

    Contacts:



    Apellis

    Media:

    Lissa Pavluk 

     

    617.977.6764



    Investors: 

    Meredith Kaya



    617.599.8178

    Sobi



    To contact the Sobi Investor Relations Team, click here. For Sobi Media contacts, click here.



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