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WALTHAM, Mass., Feb. 21, 2023 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in complement, today announced its fourth quarter and full year 2022 financial results and business highlights.
“2022 was another year of remarkable execution for Apellis as we moved closer toward our goal of bringing SYFOVRE to patients with GA worldwide and continued to elevate the standard of care in PNH with EMPAVELI. Last week, we were thrilled to announce the FDA approval of SYFOVRE as the first and only treatment approved for patients living with GA, and we are now ready to launch in the U.S.,” said Cedric Francois, M.D., Ph.D., co-founder and chief executive officer of Apellis. “With two commercial products, a robust pipeline of multiple late-stage programs, and a portfolio of pre-clinical assets heading towards the clinic, we believe we are in a strong position for 2023 and beyond.”
Dr. Francois continued, “With our first two approvals in less than two years, we believe we have only begun to unlock the potential of targeting C3 to treat some of the most challenging diseases that patients face. We look forward to building on this momentum.”
Fourth Quarter and Full Year 2022 Business Highlights and Upcoming Milestones:
Ophthalmology Highlights
Paroxysmal Nocturnal Hemoglobinuria (PNH) Highlights
Rare Disease R&D Highlights
NeurologyR&D Highlights
Fourth Quarter and Full Year 2022 Financial Results:
Cash. As of December 31, 2022, Apellis had $551.8 million in cash and cash equivalents, compared to $700.6 million in cash, cash equivalents, and short-term marketable securities as of December 31, 2021.
Total Revenue.
Cost of Sales.
R&D Expenses.
General and Administrative (G&A) Expenses.
Net Loss (Income). Apellis reported a net loss of $166.0 million and $652.2 million for the fourth quarter and full year 2022, respectively, compared to a net loss of $147.9 million and $746.4 million for the same periods in 2021.
About SYFOVRE™ (pegcetacoplan injection)
SYFOVRE™ (pegcetacoplan injection) is the first and only approved therapy for geographic atrophy (GA). By targeting C3, SYFOVRE is designed to provide comprehensive control of the complement cascade, part of the body’s immune system. SYFOVRE is approved in the United States for the treatment of GA secondary to age-related macular degeneration.
About EMPAVELI® (pegcetacoplan)
EMPAVELI® (pegcetacoplan) is the first and only approved therapy for PNH 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 SYFOVRE™ (pegcetacoplan injection)
CONTRAINDICATIONS
WARNINGS AND PRECAUTIONS
ADVERSE REACTIONS
Please see accompanying full Prescribing Information for more information.
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
Most common adverse reactions in patients with PNH (incidence ≥10%) were injection-site reactions, infections, diarrhea, abdominal pain, respiratory tract infection, pain in extremity, hypokalemia, fatigue, viral infection, cough, arthralgia, dizziness, headache, and rash.
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 combines courageous science and compassion to develop life-changing therapies for some of the most challenging diseases patients face. We ushered in the first new class of complement medicine in 15 years and now have two approved medicines targeting C3. These include the first and only therapy for geographic atrophy, a leading cause of blindness around the world. With nearly a dozen clinical and pre-clinical programs underway, we believe we have only begun to unlock the potential of targeting C3 across many serious diseases. For more information, please visit http://apellis.com or follow us on Twitter and LinkedIn.
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. 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 SYFOVRE will be commercially available when expected; whether clinical trials of SYFOVRE indicate an apparent positive effect that is greater than the actual positive effect, whether SYFOVRE will receive approval from foreign regulatory agencies for GA when expected or at all; 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 systemic pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for CAD, C3G, IC-MPGN, HSCT-TMA, ALS or any other indication when expected or at all; whether, if Apellis’ products receive approval, they will be successfully distributed and marketed; and other factors discussed in the “Risk Factors” section of Apellis’ Annual Report on Form 10-K filed with the Securities and Exchange Commission on February 21, 2023 and the risks described in other filings that Apellis may make with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Apellis specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.
Media Contact:
Lissa Pavluk
media@apellis.com
617.977.6764
Investor Contact:
Meredith Kaya
meredith.kaya@apellis.com
617.599.8178
| APELLIS PHARMACEUTICALS, INC. | |||||||
| CONDENSED CONSOLIDATED BALANCE SHEETS | |||||||
| (Amounts in thousands, except per share amounts) | |||||||
| December 31, | December 31, | ||||||
| 2022 | 2021 | ||||||
| Assets | (Unaudited) | ||||||
| Current assets: | |||||||
| Cash and cash equivalents | $ | 551,801 | $ | 640,192 | |||
| Marketable securities | — | 60,358 | |||||
| Accounts receivable | 7,727 | 10,103 | |||||
| Inventory | 85,714 | 16,286 | |||||
| Prepaid assets | 36,350 | 24,868 | |||||
| Restricted cash | 1,273 | 1,563 | |||||
| Other current assets | 36,658 | 70,677 | |||||
| Total current assets | 719,523 | 824,047 | |||||
| Non-current assets: | |||||||
| Right-of-use assets | 18,747 | 19,901 | |||||
| Property and equipment, net | 6,148 | 6,177 | |||||
| Other assets | 15,799 | 31,640 | |||||
| Total assets | $ | 760,217 | $ | 881,765 | |||
| Liabilities and Stockholders' Equity | |||||||
| Current liabilities: | |||||||
| Accounts payable | 37,342 | $ | 16,909 | ||||
| Accrued expenses | 95,139 | 103,239 | |||||
| Current portion of development liability | 29,504 | 7,584 | |||||
| Current portion of right of use liabilities | 5,625 | 4,115 | |||||
| Total current liabilities | 167,610 | 131,847 | |||||
| Long-term liabilities: | |||||||
| Long-term development liability | 315,647 | 345,151 | |||||
| Convertible senior notes | 92,736 | 189,024 | |||||
| Right-of-use liabilities | 14,352 | 17,081 | |||||
| Total liabilities | 590,345 | 683,103 | |||||
| Commitments and contingencies (Note 16) | |||||||
| Stockholders' equity: | |||||||
| Preferred stock, $0.0001 par value; 10,000 shares authorized and zero shares issued and outstanding at December 31, 2022 and 2021 | — | — | |||||
| Common stock, $0.0001 par value; 200,000 shares authorized at December 31, 2022 and 2021; 110,772 and 97,524 shares issued and outstanding at December 31, 2022 and 2021 | 11 | 10 | |||||
| Additional paid-in capital | 2,479,596 | 1,857,430 | |||||
| Accumulated other comprehensive loss | (875 | ) | (2,090 | ) | |||
| Accumulated deficit | (2,308,860 | ) | (1,656,688 | ) | |||
| Total stockholders' equity | 169,872 | 198,662 | |||||
| Total liabilities and stockholders' equity | $ | 760,217 | $ | 881,765 | |||
| APELLIS PHARMACEUTICALS, INC. | |||||||||||||||
| CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS | |||||||||||||||
| (Amounts in thousands, except per share amounts) | |||||||||||||||
| For the Three Months Ended December 31, | Year Ended December 31, | ||||||||||||||
| 2022 | 2021 | 2022 | 2021 | ||||||||||||
| (Unaudited) | (Unaudited) | ||||||||||||||
| Revenue: | |||||||||||||||
| Product revenue, net | $ | 19,653 | $ | 9,210 | $ | 65,092 | $ | 15,147 | |||||||
| Licensing and other revenue | 3,010 | 51,080 | $ | 10,330 | $ | 51,416 | |||||||||
| Total revenue: | 22,663 | 60,290 | 75,422 | 66,563 | |||||||||||
| Operating expenses: | |||||||||||||||
| Cost of sales | 2,925 | 45 | 5,636 | 200 | |||||||||||
| Research and development | 99,423 | 78,180 | 387,236 | 345,869 | |||||||||||
| Cost of research collaboration | — | 25,000 | — | 75,000 | |||||||||||
| License expense | — | 5,000 | — | 5,000 | |||||||||||
| General and administrative | 84,368 | 41,462 | 277,163 | 176,771 | |||||||||||
| Operating expenses: | 186,716 | 149,687 | 670,035 | 602,840 | |||||||||||
| Net operating income/(loss) | (164,053 | ) | (89,397 | ) | (594,613 | ) | (536,277 | ) | |||||||
| Loss on conversion of debt | — | — | (32,890 | ) | (100,589 | ) | |||||||||
| Loss from remeasurement of development derivative liability | — | (55,192 | ) | — | (97,675 | ) | |||||||||
| Interest income | 4,575 | 37 | 8,914 | 418 | |||||||||||
| Interest expense | (7,738 | ) | (3,018 | ) | (32,626 | ) | (13,241 | ) | |||||||
| Other (expense)/income, net | (246 | ) | (169 | ) | (288 | ) | 1,362 | ||||||||
| Net loss before taxes | (167,462 | ) | (147,739 | ) | (651,503 | ) | (746,002 | ) | |||||||
| Income tax expense | (1,471 | ) | 196 | 669 | 352 | ||||||||||
| Net income/(loss) | $ | (165,991 | ) | $ | (147,935 | ) | $ | (652,172 | ) | $ | (746,354 | ) | |||
| Other comprehensive (loss)/gain: | |||||||||||||||
| Unrealized (loss)/gain on marketable securities | 382 | 9 | (1 | ) | 9 | ||||||||||
| Unrealized gain on pension plans | 1,646 | — | 1,646 | — | |||||||||||
| Foreign currency gain/(loss) | 124 | 71 | (430 | ) | (1,982 | ) | |||||||||
| Total other comprehensive income/(loss) | 2,152 | 80 | 1,215 | (1,973 | ) | ||||||||||
| Comprehensive loss, net of tax | $ | (163,839 | ) | $ | (147,855 | ) | $ | (650,957 | ) | $ | (748,327 | ) | |||
| Net loss per common share, basic and diluted | $ | (1.50 | ) | $ | (1.61 | ) | $ | (6.15 | ) | $ | (8.84 | ) | |||
| Weighted-average number of common shares used in net loss per common share, basic and diluted | 110,629 | 92,149 | 106,114 | 84,421 | |||||||||||