- Generated U.S. net product revenues of $18.4 million for SYFOVRE™ (pegcetacoplan injection) and $20.4 million for EMPAVELI® (pegcetacoplan)
- SYFOVRE launched in the U.S. as the first and only treatment for geographic atrophy (GA) secondary to age-related macular degeneration (AMD)
- Validation received for marketing applications for intravitreal pegcetacoplan in the EU, Canada, Australia, the United Kingdom, and Switzerland; decisions expected in the first half of 2024
- Cash and cash equivalents of $765 million as of March 31, 2023; expected cash runway into Q1 2025
WALTHAM, Mass., May 04, 2023 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in complement, today announced its first quarter 2023 financial results and business highlights.
“2023 is off to a very strong start. We received FDA approval of SYFOVRE in geographic atrophy in February and are encouraged by the early momentum we are seeing in the launch. The team is proud to be able to bring SYFOVRE to people suffering with GA who, until now, did not have a treatment option,” said Cedric Francois, M.D., Ph.D., co-founder and chief executive officer of Apellis. “Additionally, our position in the PNH market continues to expand, and we are making steady progress advancing a robust clinical pipeline, which encompasses multiple late-stage, rare disease programs as well as several programs heading into the clinic. This broad portfolio gives us a position of strength as we strive to achieve our vision of being the global leader in complement.”
Adam Townsend, chief commercial officer of Apellis, added, “I am thrilled with the initial enthusiasm that physicians and patients are showing for SYFOVRE in these early days of launch. We have built best-in-class commercial and medical teams with extensive experience in retina who went into the SYFOVRE launch well prepared and highly energized. With over 6,000 commercial vials shipped to physicians in March alone, the trajectory looks very promising.”
First Quarter 2023 Business Highlights and Upcoming Milestones
Ophthalmology Highlights
- SYFOVRE for the treatment of GA secondary to AMD:
- Apellis reported $18.4 million in SYFOVRE U.S. net product revenue for the first quarter of 2023.
- On February 17, 2023, the U.S. Food and Drug Administration (FDA) approved SYFOVRE for the treatment of GA secondary to AMD.
- The commercial launch of SYFOVRE began on March 1, 2023. As of March 31, more than 6,000 commercial vials of SYFOVRE had been shipped to physician practices.
- Marketing applications for intravitreal pegcetacoplan for the treatment of GA secondary to AMD are under review in the EU, Canada, Australia, the United Kingdom, and Switzerland. A decision by the European Medicines Agency is expected in early 2024; decisions by the local regulatory authorities in the other countries are expected in the first half of 2024.
- Apellis is exploring the potential to treat other complement-driven retina diseases with intravitreal pegcetacoplan, such as Stargardt disease.
- APL-2006: Apellis continues to advance APL-2006, a bispecific C3 and VEGF inhibitor, for the treatment of GA and wet AMD.
Paroxysmal Nocturnal Hemoglobinuria (PNH) Highlights
- EMPAVELI for the treatment of PNH:
- Apellis recorded $20.4 million in EMPAVELI U.S. net product revenue for the first quarter 2023.
- More than 200 patients with PNH were on commercial treatment with EMPAVELI as of March 31, 2023.
- In February 2023, the FDA approved the supplemental new drug application (sNDA) with the Phase 3 PRINCE results and the 48-week Phase 3 PEGASUS data.
- In March 2023, the company was notified by the FDA that the FDA would miss the Prescription Drug User Fee Act (PDUFA) target action date of March 15, 2023, for Apellis’ sNDA application for the EMPAVELI Injector. The FDA indicated that the review is ongoing, and the company is awaiting further detail on timing. EMPAVELI Injector is a compact, on-body drug delivery system that features several advancements to streamline self-administration of EMPAVELI.
- In March 2023, Sobi received approval of EMPAVELI from the Japanese Ministry of Health, Labor, and Welfare for the treatment of adults with PNH.
Rare Disease R&D Highlights
- Amyotrophic lateral sclerosis (ALS): In April 2023, Apellis discontinued investigational treatment with systemic pegcetacoplan in the open-label period of the Phase 2 MERIDIAN study. This decision was made following an unblinded review of the available data by an independent data monitoring committee.
- The committee concluded the available data did not support continuation of treatment. Their recommendation was not based on any safety concerns.
- Top-line results are expected in the second quarter of 2023, followed by an analysis of the full dataset, which will inform the next steps for the program.
- Immune complex membranoproliferative glomerulonephritis (IC-MPGN) and C3 glomerulopathy (C3G): Apellis continues to enroll patients in the Phase 3 VALIANT study of systemic pegcetacoplan for IC-MPGN and C3G. Top-line data from this study is expected in 2024.
- Cold agglutinin disease (CAD): Sobi, Apellis’ global co-development partner for systemic pegcetacoplan, continues to enroll patients in the Phase 3 CASCADE study of systemic pegcetacoplan for CAD.
- Hematopoietic stem cell transplantation-associated thrombotic microangiopathy (HSCT-TMA): Sobi continues to enroll patients in its Phase 2 study evaluating the efficacy and safety of systemic pegcetacoplan in patients with HSCT-TMA. Sobi expects data from this study in 2024.
- C3 inhibition + siRNA: In March 2023, Apellis submitted an investigational new drug (IND) application for its siRNA program.
Neurology R&D Highlights
- APL-1030: Apellis continues to advance pre-clinical studies with APL-1030, a first-in-class, brain-active C3 inhibitor for neurological diseases.
First Quarter 2023 Financial Results
Cash. As of March 31, 2023, Apellis had $765.1 million in cash and cash equivalents, compared to $551.8 million in cash and cash equivalents as of December 31, 2022. Apellis anticipates its cash balance, combined with cash anticipated to be generated from sales of EMPAVELI and SYFOVRE, to fund operations into the first quarter of 2025.
Total Revenue.
- Total revenue was $44.8 million for the first quarter of 2023, which consisted of $20.4 million of U.S. net product revenue of EMPAVELI, $18.4 million of U.S. net product revenue of SYFOVRE, and additional licensing and other revenue associated with the Sobi collaboration. Total revenue was $14.4 million for the first quarter of 2022, which consisted of $12.1 million in net product revenue from sales of EMPAVELI and $2.3 million in revenue associated with the Sobi collaboration.
Cost of Sales.
- Cost of sales were $7.8 million for the first quarter 2023, compared to $1.2 million for same period in 2022. Cost of sales consists primarily of costs associated with the manufacturing of EMPAVELI, royalties owed to our licensor for such sales, and certain period costs.
- Prior to receiving FDA approval for EMPAVELI in May 2021 and SYFOVRE in February 2023, costs associated with the manufacturing of EMPAVELI and SYFOVRE inventory were expensed as research and development (R&D) expense. This resulted in inventory being sold during the periods ended March 31, 2023, and March 31, 2022, for which a portion of the costs had been previously expensed prior to FDA approval.
R&D Expenses.
- R&D expenses were $110.0 million for the first quarter of 2023, compared to $90.9 million for the same period in 2022.
- The increase in R&D expenses for the first quarter 2023 was primarily attributable to higher personnel related costs due an increase in employees, an increase in research and innovation costs, an increase in other development costs, an increase in pre-clinical study expenses and an increase in device development expenses. The increases were partially offset by a decrease in clinical trial costs due to the completion of our Phase 3 DERBY and OAKS trials and a decrease in contract manufacturing expenses due primarily to the timing of drug supply and analytical activity.
General and Administrative (G&A) Expenses.
- G&A expenses were $102.1 million for the first quarter of 2023, compared to $51.2 million for the same period in 2022.
- The increase in G&A expenses for the first quarter of 2023 was primarily attributable to an increase in employee related costs of $23.6 million, an increase in professional and consulting fees and general commercial preparation activities of $24.0 million, an increase in travel related expenses, higher office costs, and an increase in director stock option compensation.
Net Loss. Apellis reported a net loss of $177.8 million for the first quarter 2023, compared to a net loss of $138.9 million for the same period in 2022.
Conference Call and Webcast
Apellis will host a conference call and webcast to discuss its first quarter 2023 financial results and business highlights today, May 4, 2023, at 4:30 p.m. ET. To access the live call by phone, please pre-register for the call here. 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. A replay of the webcast will be available for 30 days following the event.
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®/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. It is approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in the United States, Australia, Canada, and Saudi Arabia as EMPAVELI and in the European Union and United Kingdom as Aspaveli. The therapy is also under investigation for several other rare diseases across hematology, nephrology, and neurology.
U.S. Important Safety Information for SYFOVRE™ (pegcetacoplan injection)
CONTRAINDICATIONS
- SYFOVRE is contraindicated in patients with ocular or periocular infections, and in patients with active intraocular inflammation
WARNINGS AND PRECAUTIONS
- Endophthalmitis and Retinal Detachments
- Intravitreal injections, including those with SYFOVRE, may be associated with endophthalmitis and retinal detachments. Proper aseptic injection technique must always be used when administering SYFOVRE to minimize the risk of endophthalmitis. Patients should be instructed to report any symptoms suggestive of endophthalmitis or retinal detachment without delay and should be managed appropriately.
- Neovascular AMD
- In clinical trials, use of SYFOVRE was associated with increased rates of neovascular (wet) AMD or choroidal neovascularization (12% when administered monthly, 7% when administered every other month and 3% in the control group) by Month 24. Patients receiving SYFOVRE should be monitored for signs of neovascular AMD. In case anti-Vascular Endothelial Growth Factor (anti-VEGF) is required, it should be given separately from SYFOVRE administration.
- Intraocular Inflammation
- In clinical trials, use of SYFOVRE was associated with episodes of intraocular inflammation including: vitritis, vitreal cells, iridocyclitis, uveitis, anterior chamber cells, iritis, and anterior chamber flare. After inflammation resolves, patients may resume treatment with SYFOVRE.
- Increased Intraocular Pressure
- Acute increase in IOP may occur within minutes of any intravitreal injection, including with SYFOVRE. Perfusion of the optic nerve head should be monitored following the injection and managed as needed.
ADVERSE REACTIONS
- Most common adverse reactions (incidence ≥5%) are ocular discomfort, neovascular age-related macular degeneration, vitreous floaters, conjunctival hemorrhage.
Please see accompanying full Prescribing Information for more information.
U.S. Important Safety Information for EMPAVELI
BOXED WARNING: SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA
- Meningococcal infections may occur in patients treated with EMPAVELI and may become rapidly life-threatening or fatal if not recognized and treated early. Use of EMPAVELI may predispose individuals to serious infections, especially those caused by encapsulated bacteria, such as Streptococcus pneumoniae, Neisseria meningitidis types A, C, W, Y, and B, and Haemophilus influenzae type B.
- Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for vaccinations against encapsulated bacteria.
- Vaccinate patients at least 2 weeks prior to administering the first dose of EMPAVELI unless the risks of delaying therapy with EMPAVELI outweigh the risk of developing a serious infection.
- Vaccination reduces, but does not eliminate, the risk of serious infections. Monitor patients for early signs of serious infections and evaluate immediately if infection is suspected.
- EMPAVELI is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS). Under the EMPAVELI REMS, prescribers must enroll in the program.
CONTRAINDICATIONS
- Hypersensitivity to pegcetacoplan or to any of the excipients
- Not currently vaccinated against certain encapsulated bacteria, unless the risks of delaying EMPAVELI treatment outweigh the risks of developing a bacterial infection with an encapsulated organism
- Unresolved serious infection caused by encapsulated bacteria including Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae
WARNINGS AND PRECAUTIONS
Serious Infections Caused by Encapsulated Bacteria
The use of EMPAVELI may predispose individuals to serious, life-threatening, or fatal infections caused by encapsulated bacteria, including Streptococcus pneumoniae, Neisseria meningitidis types A, C, W, Y, and B, and Haemophilus influenzae type B (Hib). To reduce the risk of infection, all patients must be vaccinated against these bacteria according to the most current ACIP recommendations for patients with altered immunocompetence associated with complement deficiencies. Revaccinate patients in accordance with ACIP recommendations considering the duration of therapy with EMPAVELI.
For patients without known history of vaccination, administer required vaccines at least 2 weeks prior to receiving the first dose of EMPAVELI. If immediate therapy with EMPAVELI is indicated, administer required vaccine as soon as possible and provide patients with 2 weeks of antibacterial drug prophylaxis.
Closely monitor patients for early signs and symptoms of serious infection and evaluate patients immediately if an infection is suspected. Promptly treat known infections. Serious infection may become rapidly life-threatening or fatal if not recognized and treated early. Consider discontinuation of EMPAVELI in patients who are undergoing treatment for serious infections.
EMPAVELI REMS
Because of the risk of serious infections, EMPAVELI is available only through a restricted program under a REMS. Under the EMPAVELI REMS, prescribers must enroll in the program and must counsel patients about the risk of serious infection, provide the patients with the REMS educational materials, and ensure patients are vaccinated against encapsulated bacteria. Enrollment and additional information are available by telephone: 1-888-343-7073 or at www.empavelirems.com.
Infusion-Related Reactions
Systemic hypersensitivity reactions (e.g., facial swelling, rash, urticaria) have occurred in patients treated with EMPAVELI. One patient (less than 1% in clinical studies) experienced a serious allergic reaction which resolved after treatment with antihistamines. If a severe hypersensitivity reaction (including anaphylaxis) occurs, discontinue EMPAVELI infusion immediately, institute appropriate treatment, per standard of care, and monitor until signs and symptoms are resolved.
Monitoring PNH Manifestations after Discontinuation of EMPAVELI
After discontinuing treatment with EMPAVELI, closely monitor for signs and symptoms of hemolysis, identified by elevated LDH levels along with sudden decrease in PNH clone size or hemoglobin, or reappearance of symptoms such as fatigue, hemoglobinuria, abdominal pain, dyspnea, major adverse vascular events (including thrombosis), dysphagia, or erectile dysfunction. Monitor any patient who discontinues EMPAVELI for at least 8 weeks to detect hemolysis and other reactions. If hemolysis, including elevated LDH, occurs after discontinuation of EMPAVELI, consider restarting treatment with EMPAVELI.
Interference with Laboratory Tests
There may be interference between silica reagents in coagulation panels and EMPAVELI that results in artificially prolonged activated partial thromboplastin time (aPTT); therefore, avoid the use of silica reagents in coagulation panels.
ADVERSE REACTIONS
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
[TABLES TO FOLLOW]
| APELLIS PHARMACEUTICALS, INC. | |||||||
| CONDENSED CONSOLIDATED BALANCE SHEETS | |||||||
| (Amounts in thousands, except per share amounts) | |||||||
| (Unaudited) | |||||||
| March 30, | December 31, | ||||||
| 2023 | 2022 | ||||||
| Assets | |||||||
| Current assets: | |||||||
| Cash and cash equivalents | $ | 765,083 | $ | 551,801 | |||
| Accounts receivable | 31,505 | 7,727 | |||||
| Inventory | 85,484 | 85,714 | |||||
| Prepaid assets | 36,559 | 36,350 | |||||
| Restricted cash | 1,275 | 1,273 | |||||
| Other current assets | 34,349 | 36,658 | |||||
| Total current assets | 954,255 | 719,523 | |||||
| Non-current assets: | |||||||
| Right-of-use assets | 17,854 | 18,747 | |||||
| Property and equipment, net | 5,967 | 6,148 | |||||
| Other assets | 793 | 15,799 | |||||
| Total assets | $ | 978,869 | $ | 760,217 | |||
| Liabilities and Stockholders' Equity | |||||||
| Current liabilities: | |||||||
| Accounts payable | $ | 31,494 | 37,342 | ||||
| Accrued expenses | 71,251 | 95,139 | |||||
| Current portion of development liability | 30,071 | 29,504 | |||||
| Current portion of right of use liabilities | 5,828 | 5,625 | |||||
| Total current liabilities | 138,644 | 167,610 | |||||
| Long-term liabilities: | |||||||
| Long-term development liability | 321,713 | 315,647 | |||||
| Convertible senior notes | 92,809 | 92,736 | |||||
| Right-of-use liabilities | 13,233 | 14,352 | |||||
| Other liabilities | 347 | — | |||||
| Total liabilities | 566,746 | 590,345 | |||||
| Commitments and contingencies (Note 14) | — | — | |||||
| Stockholders' equity: | |||||||
| Preferred stock, $0.0001 par value; 10,000 shares authorized and zero shares issued and outstanding at March 31, 2023 and December 31, 2022 | — | — | |||||
| Common stock, $0.0001 par value; 200,000 shares authorized at March 31, 2023 and December 31, 2022; 116,179 shares issued and outstanding at March 31, 2023, and 110,772 shares issued and outstanding at December 31, 2022 | 12 | 11 | |||||
| Additional paid-in capital | 2,899,524 | 2,479,596 | |||||
| Accumulated other comprehensive loss | (775 | ) | (875 | ) | |||
| Accumulated deficit | (2,486,638 | ) | (2,308,860 | ) | |||
| Total stockholders' equity | 412,123 | 169,872 | |||||
| Total liabilities and stockholders' equity | $ | 978,869 | $ | 760,217 | |||
| APELLIS PHARMACEUTICALS, INC. | |||||||
| CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS | |||||||
| (Amounts in thousands, except per share amounts) | |||||||
| (Unaudited) | |||||||
| For the Three Months Ended March 31, | |||||||
| 2023 | 2022 | ||||||
| Revenue: | |||||||
| Product revenue, net | $ | 38,800 | $ | 12,109 | |||
| Licensing and other revenue | 6,046 | 2,272 | |||||
| Total revenue: | 44,846 | 14,381 | |||||
| Operating expenses: | |||||||
| Cost of sales | 7,809 | 1,247 | |||||
| Research and development | 110,027 | 90,945 | |||||
| General and administrative | 102,093 | 51,187 | |||||
| Operating expenses: | 219,929 | 143,379 | |||||
| Net operating loss | (175,083 | ) | (128,998 | ) | |||
| Interest income | 5,393 | 98 | |||||
| Interest expense | (7,529 | ) | (8,538 | ) | |||
| Other expense, net | (277 | ) | (289 | ) | |||
| Net loss before taxes | (177,496 | ) | (137,727 | ) | |||
| Income tax expense | 282 | 1,208 | |||||
| Net loss | $ | (177,778 | ) | $ | (138,935 | ) | |
| Other comprehensive (loss)/gain: | |||||||
| Unrealized loss on marketable securities | — | (52 | ) | ||||
| Foreign currency gain | 100 | 83 | |||||
| Total other comprehensive income/(loss) | 100 | 31 | |||||
| Comprehensive loss, net of tax | $ | (177,678 | ) | $ | (138,904 | ) | |
| Net loss per common share, basic and diluted | $ | (1.56 | ) | $ | (1.42 | ) | |
| Weighted-average number of common shares used in net loss per common share, basic and diluted | 113,872 | 98,069 | |||||
