- Generated $196.8 million in 3Q 2024 revenues, including $176.6 million in U.S. net product sales
- Grew SYFOVRE® (pegcetacoplan injection) demand by 7% quarter-over-quarter, with U.S. net product revenue of $152.0 million
- Increase in demand offset by higher gross-to-net adjustments in the quarter
- Following FDA feedback, on track to file an sNDA seeking approval of pegcetacoplan for C3G / primary IC-MPGN in early 2025 based on positive 6-month Phase 3 VALIANT results
- Cash and cash equivalents of $396.9 million as of September 30, 2024; projected revenues and cash expected to be sufficient to fund operations to positive cash flow
- Management to host conference call today at 8:30 a.m. ET
WALTHAM, Mass., Nov. 05, 2024 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), today announced its third quarter 2024 financial results and business highlights.
"The third quarter was marked by continued progress, including growth in SYFOVRE vial demand and the positive Phase 3 VALIANT study results in C3G and IC-MPGN. While SYFOVRE net sales fell short of expectations due to higher gross-to-net adjustments, we remain focused on reaching more geographic atrophy patients and building on our leadership in this market," said Cedric Francois, M.D., Ph.D., chief executive officer at Apellis. "With two potentially blockbuster products, a promising pipeline that is emerging and a path to profitability, Apellis is well positioned for future growth. We remain confident in our long-term strategy and the significant opportunities ahead."
Third Quarter 2024 Business Highlights and Upcoming Milestones
Ophthalmology Highlights
- SYFOVRE for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD):
- Generated $152.0 million in SYFOVRE U.S. net product revenue in the third quarter 2024.
- SYFOVRE commercial vials grew by approximately 7% quarter-over-quarter.
- SYFOVRE net selling price declined relative to previous quarters due to an increase in gross-to-net adjustments.
- Gross-to-net is expected to be stable going forward, with modest quarterly increases as is typical in a buy-and-bill model.
- Remained the market leader in GA, delivering more than 88,500 SYFOVRE doses to physician practices in the third quarter, including approximately 84,500 commercial vials and 4,000 samples.
- More than 420,000 SYFOVRE injections are estimated to have been administered through September 2024, including clinical trials.
- Effective January 1, 2025, a large Medicare Advantage plan will make SYFOVRE the only preferred product on its formulary.
- Received a negative opinion from the Committee for Medicinal Products for Human Use (CHMP) in September 2024.
- Expect regulatory decisions in the U.K., Switzerland, Canada and Australia in early 2025.
- Generated $152.0 million in SYFOVRE U.S. net product revenue in the third quarter 2024.
Paroxysmal Nocturnal Hemoglobinuria (PNH) Highlights
- EMPAVELI for the treatment of PNH:
- Generated $24.6 million in EMPAVELI U.S. net product revenue in the third quarter of 2024.
- Continued high patient compliance rates of 97%.
R&D Highlights
- C3 glomerulopathy (C3G) and primary immune complex glomerulonephritis (IC-MPGN): Following feedback from the U.S. Food and Drug Administration (FDA), based on the six-month results from the VALIANT study, Apellis remains on track to file a supplemental new drug application (sNDA) in early 2025 seeking approval of pegcetacoplan in C3G and IC-MPGN.
- The FDA did not require Apellis to wait to file with the full 52-week data.
- Detailed results from the pivotal Phase 3 VALIANT study were presented at Kidney Week 2024, highlighting the strength of the pegcetacoplan treatment effects in patients with C3G and primary IC-MPGN.
- Sobi plans to submit a marketing application with the European Medicines Agency in early 2025, and with the Japanese Health Authorities in 2025.
- Hematopoietic stem cell transplantation-associated thrombotic microangiopathy (HSCT-TMA): Sobi completed enrollment in its Phase 2 study evaluating the efficacy and safety of systemic pegcetacoplan in patients with HSCT-TMA, with topline data expected in mid-2025.
- APL-3007 (small interfering RNA silencing C3): Now expect to report topline data from the Phase 1 dose escalation study in the first quarter of 2025.
Third Quarter 2024 Financial Results
Total Revenue.
- Total revenue was $196.8 million for the third quarter of 2024, which consisted of $152.0 million of SYFOVRE U.S. net product revenue, $24.6 million of EMPAVELI U.S. net product revenue, and $20.3 million in licensing and other revenue associated with the Sobi collaboration.
- Total revenue was $110.4 million for the third quarter of 2023, which consisted of $75.3 million of SYFOVRE U.S. net product revenue, $23.9 million in EMPAVELI U.S. net product revenue and $11.2 million in revenue associated with the Sobi collaboration.
Cost of Sales.
- Cost of sales were $33.6 million for the third quarter of 2024, compared to $22.4 million for same period in 2023.
- The increase in cost of sales was primarily driven by an increase in expenses incurred related to excess, obsolete or scrapped inventory, and an increase in expense incurred in connection with the termination of the minimum purchase obligation of PEG in September 2024, which were partially offset by a decrease in royalty expense as sales-based milestones incurred in the prior year did not recur in the current period.
R&D Expenses.
- R&D expenses were $88.6 million for the third quarter of 2024, compared to $79.4 million for the same period in 2023.
- The increase in R&D expenses was primarily attributable to an increase in program specific external costs and an increase in non-program specific external costs, which were partially offset by a decrease in compensation and related personnel costs.
Selling, General and Administrative (SG&A) Expenses.
- SG&A expenses were $122.0 million for the third quarter of 2024, compared to $145.7 million for the same period in 2023.
- The decrease was primarily attributable to decreases in personnel related costs, commercial and marketing activities, office costs, and professional and consulting fees.
Net Loss. Apellis reported a net loss of $57.4 million for the third quarter of 2024, compared to a net loss of $140.2 million for the same period in 2023.
Cash. As of September 30, 2024, Apellis had $396.9 million in cash and cash equivalents, compared to $351.2 million in cash and cash equivalents as of December 31, 2023.
- Apellis anticipates its cash, combined with expected product revenues, will be sufficient to fund its projected operating expenses and capital expenditures to positive cash flow.
Conference Call and Webcast
Apellis will host a conference call and webcast to discuss its third quarter 2024 financial results and business highlights today, November 5, 2024, at 8:30 a.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-ever 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, European Union, and other countries globally. The therapy is also under investigation for several other rare diseases across hematology and nephrology.
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.
- Retinal Vasculitis and/or Retinal Vascular Occlusion
- Retinal vasculitis and/or retinal vascular occlusion, typically in the presence of intraocular inflammation, have been reported with the use of SYFOVRE. Cases may occur with the first dose of SYFOVRE and may result in severe vision loss. Discontinue treatment with SYFOVRE in patients who develop these events. Patients should be instructed to report any change in vision without delay.
- 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® (pegcetacoplan)
BOXED WARNING: SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA
EMPAVELI, a complement inhibitor, increases the risk of serious infections, especially those caused by encapsulated bacteria, such as Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B. Life-threatening and fatal infections with encapsulated bacteria have occurred in patients treated with complement inhibitors. These infections may become rapidly life-threatening or fatal if not recognized and treated early.
- Complete or update vaccination for encapsulated bacteria at least 2 weeks prior to the first dose of EMPAVELI, unless the risks of delaying therapy with EMPAVELI outweigh the risks of developing a serious infection. Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for vaccinations against encapsulated bacteria in patients receiving a complement inhibitor.
- Patients receiving EMPAVELI are at increased risk for invasive disease caused by encapsulated bacteria, even if they develop antibodies following vaccination. Monitor patients for early signs and symptoms of serious infections and evaluate immediately if infection is suspected.
Because of the risk of serious infections caused by encapsulated bacteria, EMPAVELI is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the EMPAVELI REMS.
CONTRAINDICATIONS
- Hypersensitivity to pegcetacoplan or to any of the excipients
- For initiation in patients with unresolved serious infection caused by encapsulated bacteria including Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B
WARNINGS AND PRECAUTIONS
Serious Infections Caused by Encapsulated Bacteria
EMPAVELI, a complement inhibitor, increases a patient's susceptibility to serious, life-threatening, or fatal infections caused by encapsulated bacteria including Streptococcus pneumoniae, Neisseria meningitidis (caused by any serogroup, including non-groupable strains), and Haemophilus influenzae type B. Life-threatening and fatal infections with encapsulated bacteria have occurred in both vaccinated and unvaccinated patients treated with complement inhibitors. The initiation of EMPAVELI treatment is contraindicated in patients with unresolved serious infection caused by encapsulated bacteria.
Complete or update vaccination against encapsulated bacteria at least 2 weeks prior to administration of the first dose of EMPAVELI, according to the most current ACIP recommendations for patients receiving a complement inhibitor. Revaccinate patients in accordance with ACIP recommendations considering the duration of therapy with EMPAVELI. Note that, ACIP recommends an administration schedule in patients receiving complement inhibitors that differs from the administration schedule in the vaccine prescribing information. If urgent EMPAVELI therapy is indicated in a patient who is not up to date with vaccines against encapsulated bacteria according to ACIP recommendations, provide the patient with antibacterial drug prophylaxis and administer these vaccines as soon as possible. The benefits and risks of treatment with EMPAVELI, as well as the benefits and risks of antibacterial drug prophylaxis in unvaccinated or vaccinated patients, must be considered against the known risks for serious infections caused by encapsulated bacteria.
Vaccination does not eliminate the risk of serious encapsulated bacterial infections, despite development of antibodies following vaccination. Closely monitor patients for early signs and symptoms of serious infection and evaluate patients immediately if an infection is suspected. Inform patients of these signs and symptoms and instruct patients to seek immediate medical care if these signs and symptoms occur. Promptly treat known infections. Serious infection may become rapidly life-threatening or fatal if not recognized and treated early. Consider interruption of EMPAVELI in patients who are undergoing treatment for serious infections.
EMPAVELI is available only through a restricted program under a REMS.
EMPAVELI REMS
EMPAVELI is available only through a restricted program under a REMS called EMPAVELI REMS, because of the risk of serious infections caused by encapsulated bacteria. Notable requirements of the EMPAVELI REMS include the following:
Under the EMPAVELI REMS, prescribers must enroll in the program. Prescribers must counsel patients about the risks, signs, and symptoms of serious infections caused by encapsulated bacteria, provide patients with the REMS educational materials, ensure patients are vaccinated against encapsulated bacteria at least 2 weeks prior to the first dose of EMPAVELI, prescribe antibacterial drug prophylaxis if patients' vaccine status is not up to date and treatment must be started urgently, and provide instructions to always carry the Patient Safety Card both during treatment, as well as for 2 months following last dose of EMPAVELI. Pharmacies that dispense EMPAVELI must be certified in the EMPAVELI REMS and must verify prescribers are certified.
Further information is available at www.empavelirems.com or 1-888-343-7073.
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-ever therapy for geographic atrophy, a leading cause of blindness around the world. We believe we have only begun to unlock the potential of targeting C3 across serious retinal, rare, and neurological diseases. For more information, please visit http://apellis.com or follow us on X (formerly 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 the results of the Company's clinical trials for EMPAVELI, SYFOVRE, or any of its future products will warrant regulatory submissions to the FDA or equivalent foreign regulatory agencies; whether systemic pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for C3G and IC-MPGN or any other indication when expected or at all; rate and degree of market acceptance and clinical utility of EMPAVELI, SYFOVRE and any future products for which we receive marketing approval will impact our commercialization efforts; whether SYFOVRE will receive approval from foreign regulatory agencies for GA when expected or at all; whether the Company's clinical trials will be completed when anticipated; whether results obtained in clinical trials will be indicative of results that will be generated in future clinical trials or in the real world setting; whether the period for which the Company believes that its cash resources will be sufficient to fund its operations; and other factors discussed in the "Risk Factors" section of Apellis' Annual Report on Form 10-K with the Securities and Exchange Commission on February 27, 2024 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:
Tracy Vineis
media@apellis.com
617.420.4839
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) | |||||||||
| September 30, | December 31, | ||||||||
| 2024 | 2023 | ||||||||
| Assets | (Unaudited) | ||||||||
| Current assets: | |||||||||
| Cash and cash equivalents | $ | 396,864 | $ | 351,185 | |||||
| Accounts receivable | 279,011 | 206,442 | |||||||
| Inventory | 121,058 | 146,362 | |||||||
| Prepaid assets | 23,809 | 38,820 | |||||||
| Restricted cash | 1,373 | 1,114 | |||||||
| Other current assets | 11,782 | 22,408 | |||||||
| Total current assets | 833,897 | 766,331 | |||||||
| Non-current assets: | |||||||||
| Total operating expenses: | 17,060 | 16,745 | |||||||
| Property and equipment, net | 3,379 | 4,345 | |||||||
| Long-term inventory | 46,080 | - | |||||||
| Other assets | 1,450 | 1,309 | |||||||
| Total assets | $ | 901,866 | $ | 788,730 | |||||
| Other (expense)/income, net | |||||||||
| Current liabilities: | |||||||||
| Accounts payable | 42,730 | 37,516 | |||||||
| Accrued expenses | 139,835 | 127,806 | |||||||
| Current portion of development liability | - | 75,830 | |||||||
| Current portion of lease liabilities | 6,792 | 6,441 | |||||||
| Deferred revenue | 1,903 | - | |||||||
| Total current liabilities | 191,260 | 247,593 | |||||||
| Long-term liabilities: | |||||||||
| Long-term development liability | - | 239,817 | |||||||
| Long-term credit facility | 358,982 | - | |||||||
| Convertible senior notes | 93,263 | 93,033 | |||||||
| Lease liabilities | 11,411 | 11,454 | |||||||
| Other liabilities | 9,829 | 2,312 | |||||||
| Total liabilities | 664,745 | 594,209 | |||||||
| Commitments and contingencies (Note 13) | |||||||||
| Stockholders' equity: | |||||||||
| Preferred stock, $0.0001 par value; 10,000 shares authorized, and zero shares issued and outstanding at September 30, 2024 and December 31, 2023 | - | - | |||||||
| Common stock, $0.0001 par value; 200,000 shares authorized at September 30, 2024 and December 31, 2023; 122,069 shares issued and outstanding at September 30, 2024, and 119,556 shares issued and outstanding at December 31, 2023 | 12 | 12 | |||||||
| Additional paid-in capital | 3,239,262 | 3,035,539 | |||||||
| Accumulated other comprehensive loss | (3,140 | ) | (3,542 | ) | |||||
| Accumulated deficit | (2,999,013 | ) | (2,837,488 | ) | |||||
| Total stockholders' equity | 237,121 | 194,521 | |||||||
| Total liabilities and stockholders' equity | $ | 901,866 | $ | 788,730 | |||||
| APELLIS PHARMACEUTICALS, INC. | ||||||||||||||||
| CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS | ||||||||||||||||
| (Amounts in thousands, except per share amounts) | ||||||||||||||||
| For the Three Months Ended September 30, | For the Nine Months Ended September 30, | |||||||||||||||
| 2024 | 2023 | 2024 | 2023 | |||||||||||||
| (Unaudited) | (Unaudited) | |||||||||||||||
| Revenue: | ||||||||||||||||
| Product revenue, net | $ | 176,571 | $ | 99,182 | $ | 518,782 | $ | 227,626 | ||||||||
| Licensing and other revenue | 20,259 | 11,217 | 50,057 | 22,588 | ||||||||||||
| Total revenue: | 196,830 | 110,399 | 568,839 | 250,214 | ||||||||||||
| Operating expenses: | ||||||||||||||||
| Cost of sales | 33,557 | 22,410 | 76,867 | 38,598 | ||||||||||||
| Research and development | 88,569 | 79,421 | 251,216 | 285,105 | ||||||||||||
| Selling, general and administrative | 121,984 | 145,648 | 379,571 | 359,114 | ||||||||||||
| Total operating expenses: | 244,110 | 247,479 | 707,654 | 682,817 | ||||||||||||
| Net operating loss | (47,280 | ) | (137,080 | ) | (138,815 | ) | (432,603 | ) | ||||||||
| Loss on extinguishment of development liability | - | - | (1,949 | ) | - | |||||||||||
| Interest income | 2,889 | 4,989 | 9,377 | 16,385 | ||||||||||||
| Interest expense | (12,532 | ) | (7,310 | ) | (28,857 | ) | (22,179 | ) | ||||||||
| Other (expense)/income, net | 70 | (603 | ) | (405 | ) | (946 | ) | |||||||||
| Net loss before taxes | (56,853 | ) | (140,004 | ) | (160,649 | ) | (439,343 | ) | ||||||||
| Income tax expense | 592 | 233 | 876 | 709 | ||||||||||||
| Net loss | $ | (57,445 | ) | $ | (140,237 | ) | $ | (161,525 | ) | $ | (440,052 | ) | ||||
| Other comprehensive gain/(loss): | ||||||||||||||||
| Foreign currency translation | 222 | (269 | ) | 402 | (190 | ) | ||||||||||
| Total other comprehensive income | 222 | (269 | ) | 402 | (190 | ) | ||||||||||
| Comprehensive loss, net of tax | $ | (57,223 | ) | $ | (140,506 | ) | $ | (161,123 | ) | $ | (440,242 | ) | ||||
| Net loss per common share, basic and diluted | $ | (0.46 | ) | $ | (1.17 | ) | $ | (1.31 | ) | $ | (3.73 | ) | ||||
| Weighted-average number of common shares used in net loss per common share, basic and diluted | 124,234 | 120,292 | 123,698 | 117,827 | ||||||||||||
