WALTHAM, Mass., Aug. 09, 2021 (GLOBE NEWSWIRE) --  Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced its second quarter 2021 financial results and business highlights.

“The second quarter was an exceptional period for Apellis, underscored by the approval of EMPAVELI –– the first FDA-approved targeted C3 therapy –– for adults with PNH and a strong start to our U.S. commercial launch. We and our partner Sobi were also thrilled to report positive data from our Phase 3 PRINCE study in treatment-naïve PNH patients, emphasizing the potential for EMPAVELI to redefine treatment for all adults with PNH,” said Cedric Francois M.D., Ph.D., co-founder and chief executive officer of Apellis. “We are looking forward to the Phase 3 DERBY and OAKS readouts in September. GA is a leading cause of blindness worldwide, and we are excited about the potential to bring the first treatment to the millions of patients affected by this disease.”

“In addition to the broad platform potential of our targeted C3 therapy, our world-class research team is advancing multiple new molecular entities across several modalities, and we entered an exclusive collaboration with Beam Therapeutics to discover transformative therapies using its base editing technology. With our expanded pipeline across rare disease, ophthalmology, and neurology, we believe we are well positioned for long-term, global leadership in complement,” Dr. Francois continued.

Second Quarter 2021 Business Highlights and Upcoming Milestones:

Rare Disease

Ophthalmology

Neurology

Pipeline Expansion

Corporate Highlights

Second Quarter 2021 Financial Results:

As of June 30, 2021, Apellis had $599.0 million in cash, cash equivalents, and short-term marketable securities, compared to $877.6 million in cash, cash equivalents, and short-term marketable securities as of December 31, 2020.

Apellis reported a net loss of $219.2 million for the second quarter of 2021, compared to a net loss of $118.6 million for the same period in 2020.

Net product revenues for the second quarter of 2021 were $0.6 million, which reflects recorded sales of EMPAVELI upon approval by the FDA in May 2021 through June 30, 2021. Apellis did not have any net product revenue in the quarter ended June 30, 2020.

Research and development (R&D) expenses were $145.9 million in the second quarter of 2021, compared to $87.1 million for the same period in 2020. The increase in R&D expense was primarily attributable to $50.0 million associated with the Beam collaboration, increased quality and medical affairs expenses, personnel-related costs primarily due to the hiring of additional personnel, an increase in clinical trial costs associated with the ongoing Phase 3 studies and the preparation and commencement of our clinical trials in other indications and increased pre-clinical study expenses as we continue to perform research related to our product candidates. These expenses were offset by contra R&D expense related to the Sobi transaction and a decrease in contract manufacturing expenses due primarily to the timing of drug supply and analytical activity as well as the capitalization of inventory following FDA approval of EMPAVELI.

General and administrative (G&A) expenses were $49.0 million in the second quarter of 2021, compared to $28.4 million for the same period in 2020. The increase in G&A expenses for the second quarter 2021 was primarily attributable to general commercial preparation activities, an increase in employee-related costs, professional and consulting fees, director stock compensation expense, and insurance.

Conference Call and Webcast
Apellis will host a conference call and webcast to discuss its second quarter 2021 financial results and business highlights today, August 9, 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 5762347. 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 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

CONTRAINDICATIONS

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 Apellis

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

Forward-Looking Statements

Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements” within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements in respect of the expected closing of the exchanges. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including whether the conditions for the closing of the exchanges will be satisfied and other factors discussed in the “Risk Factors” section of Apellis’ Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on August 9, 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:
Meredith Kaya
meredith.kaya@apellis.com
617.599.8178

Media Contact:
Tracy Vineis
media@apellis.com
617.420.4839

    
APELLIS PHARMACEUTICALS, INC.
CONDENSED CONSOLIDATED BALANCE SHEETS 
(Amounts in thousands, except per share amounts)
  June 30, December 31, 
   2021   2020  
Assets (Unaudited)   
Current assets:     
Cash and cash equivalents $270,764  $565,779  
Marketable securities  328,246   311,869  
Accounts receivable  719   -  
Inventory  3,731   -  
Prepaid assets  18,866   11,400  
Restricted cash  1,572   1,266  
Other current assets  31,228   26,878  
Total current assets  655,126   917,192  
Non-current Assets:     
Right-of-use assets  21,939   17,719  
Property and equipment, net  6,750   6,803  
Other assets  16,097   18,855  
Total assets $699,912  $960,569  
Liabilities and Stockholders' Equity     
Current liabilities:     
Accounts payable $5,339  $8,477  
Accrued expenses  134,303   111,935  
Current portion of development derivative liability  14,110   4,230  
Current portion of right of use liabilities  4,035   3,685  
Total current liabilities  157,787   128,327  
Long-term liabilities:     
Convertible senior notes  386,471   358,830  
Development derivative liability  278,022   253,638  
Operating lease liabilities  19,125   15,217  
Total liabilities  841,405   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 June 30, 2021 and December 31, 2020  -   -  
Common stock, $0.0001 par value; 200,000 shares authorized at June 30, 2021 and December 31, 2020; 80,956 shares issued and outstanding at June 30, 2021, and 76,130 shares issued and outstanding at December 31, 2020  8   8  
Additional paid-in capital  1,173,512   1,131,013  
Accumulated other comprehensive loss  (1,829)  (117) 
Accumulated deficit  (1,313,184)  (926,347) 
Total stockholders' equity  (141,493)  204,557  
Total liabilities and stockholders' equity $699,912  $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 June 30, For the six months ended June 30,
  2021   2020   2021   2020 
    
  (Unaudited)
  (Unaudited)
Revenue:       
Product revenue, net$623  $—  $623  $— 
Total revenue: 623   —   623   — 
Operating expenses:       
Research and development 95,943   87,094   179,955   156,377 
Cost of research collaboration 50,000   -   50,000   - 
General and administrative 48,967   28,414   89,546   57,918 
Total operating expenses: 194,910   115,508   319,501   214,295 
Net operating loss (194,287)  (115,508)  (318,878)  (214,295)
Loss on conversion of debt —   —   (39,487)  — 
Loss from remeasurement of development derivative liability (21,180)  2,770   (38,264)  (65,636)
Interest income 104   1,025   237   3,300 
Interest expense (3,767)  (6,909)  (7,941)  (10,828)
Other income, net (61)  5   1,483   20 
Net loss (219,191)  (118,617)  (402,850)  (287,439)
Other comprehensive gain/(loss):       
    Unrealized gain/(loss) on marketable securities (35)  (842)  44   552 
    Foreign currency gain/(loss) (174)  161   (1,756)  (69)
Total other comprehensive gain/(loss) (209)  (681)  (1,712)  483 
Comprehensive loss, net of tax$(219,400) $(119,298) $(404,562) $(286,956)
Net loss per common share, basic and diluted$(2.72) $(1.57) $(5.04) $(3.85)
Weighted-average number of common shares used in net loss per common share, basic and diluted 80,654   75,550   79,938   74,635