Financial Performance - Generated $196.8 million in total revenue for Q3 2024, including $152.0 million from SYFOVRE U.S. net product sales and $24.6 million from EMPAVELI U.S. net product sales [1][8] - SYFOVRE U.S. net product revenue grew to $152.0 million in Q3 2024, up from $75.3 million in Q3 2023 [8] - Total revenue increased by 78% year-over-year, from $110.4 million in Q3 2023 to $196.8 million in Q3 2024 [8] - Net loss decreased to $57.4 million in Q3 2024, compared to a net loss of $140.2 million in Q3 2023 [12] SYFOVRE Performance and Market Position - SYFOVRE demand grew by 7% quarter-over-quarter, with U.S. net product revenue of $152.0 million in Q3 2024 [1][3] - More than 88,500 SYFOVRE doses were delivered to physician practices in Q3 2024, including approximately 84,500 commercial vials and 4,000 samples [3] - SYFOVRE remains the market leader in geographic atrophy (GA), with over 420,000 injections administered through September 2024, including clinical trials [3] - A large Medicare Advantage plan will make SYFOVRE the only preferred product on its formulary starting January 1, 2025 [3] R&D and Regulatory Updates - On track to file a supplemental new drug application (sNDA) for pegcetacoplan in C3G and IC-MPGN in early 2025, based on positive 6-month Phase 3 VALIANT results [1][5] - Detailed results from the Phase 3 VALIANT study were presented at Kidney Week 2024, highlighting strong treatment effects in patients with C3G and primary IC-MPGN [5] - Sobi plans to submit a marketing application with the European Medicines Agency in early 2025 and with Japanese Health Authorities in 2025 [6] Cash Position and Financial Outlook - Cash and cash equivalents stood at $396.9 million as of September 30, 2024, up from $351.2 million as of December 31, 2023 [1][13] - The company anticipates that its cash, combined with expected product revenues, will be sufficient to fund operations to positive cash flow [1][13] EMPAVELI Performance - EMPAVELI generated $24.6 million in U.S. net product revenue in Q3 2024, with a high patient compliance rate of 97% [4] - EMPAVELI is approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in the U.S., European Union, and other countries globally [16] Pipeline and Future Milestones - Enrollment completed in the Phase 2 study evaluating systemic pegcetacoplan in patients with HSCT-TMA, with topline data expected in mid-2025 [6] - Topline data from the Phase 1 dose escalation study of APL-3007 (small interfering RNA silencing C3) is expected in Q1 2025 [6]

Revenue Generation - SYFOVRE generated $152.0 million and $444.0 million in U.S. net product revenue for the three and nine months ended September 30, 2024, respectively[133]. - EMPAVELI generated $24.6 million and $74.7 million in U.S. net product revenue for the three and nine months ended September 30, 2024, respectively, along with $5.0 million and $13.9 million in royalties from Sobi[135]. - For the three months ended September 30, 2024, the company recognized net product revenue of $176.6 million, compared to $99.2 million for the same period in 2023, representing a 78% increase[183]. - The net product revenue for the nine months ended September 30, 2024, included $24.6 million from EMPAVELI and $152.0 million from SYFOVRE sales[183]. - Total revenue for the three months ended September 30, 2024, was $196.8 million, an increase of 78% from $110.4 million in the same period of 2023[184]. - Net product revenue for the nine months ended September 30, 2024, was $518.8 million, a 128% increase from $227.6 million in the same period of 2023[200]. - Licensing and other revenue for the three months ended September 30, 2024, was $20.3 million, up 81% from $11.2 million in the same period of 2023[186]. - Licensing and other revenue increased to $50.1 million for the nine months ended September 30, 2024, compared to $22.6 million for the same period in 2023, representing a growth of 121%[201]. Financial Performance - The company has incurred net losses of $57.4 million and $140.2 million for the three months ended September 30, 2024 and 2023, respectively, with an accumulated deficit of $3.0 billion as of September 30, 2024[141]. - Net operating loss improved to $(47.3) million for the three months ended September 30, 2024, compared to $(137.1) million in the same period of 2023, a 66% improvement[184]. - Interest income decreased to $2.9 million for the three months ended September 30, 2024, down 42% from $5.0 million in the same period of 2023[195]. - Interest expense increased to $12.5 million for the three months ended September 30, 2024, up 71% from $7.3 million in the same period of 2023[196]. - Interest income decreased to $9.4 million for the nine months ended September 30, 2024, down from $16.4 million in 2023, a decline of 43%[209]. - Interest expense increased to $28.9 million for the nine months ended September 30, 2024, compared to $22.2 million in 2023, an increase of 30%[210]. Expenses - Research and development expenses are expected to rise as product candidate development programs progress, although specific program costs have not been tracked historically[177]. - Research and development expenses increased by 12% to $88.6 million for the three months ended September 30, 2024, compared to $79.4 million in the same period of 2023[189]. - Research and development expenses decreased by $33.9 million to $251.2 million for the nine months ended September 30, 2024, a decline of 12% from $285.1 million in 2023[204]. - Selling, general and administrative expenses decreased by 16% to $122.0 million for the three months ended September 30, 2024, from $145.6 million in the same period of 2023[193]. - Selling, general and administrative expenses increased by $20.5 million to $379.6 million for the nine months ended September 30, 2024, reflecting a 6% increase from $359.1 million in 2023[207]. - The company anticipates an increase in selling, general, and administrative expenses to support ongoing commercial activities and potential product commercialization[180]. Financing and Cash Flow - The Sixth Street Financing Agreement provides for a senior secured term loan facility of up to $475.0 million, with an initial draw of $375.0 million[144]. - The net proceeds from the initial draw of the Credit Facility were approximately $358.2 million, net of $16.8 million of issuance costs[146]. - The company has financed operations primarily through approximately $2.6 billion in net proceeds from public and private offerings, $392.0 million in payments and royalties from Sobi, and $532.5 million under various credit arrangements[140]. - Net cash used in operating activities was $107.2 million for the nine months ended September 30, 2024, compared to $496.9 million in 2023, indicating a significant reduction in cash outflow[219][220]. - Net cash used in operating activities for the nine months ended September 30, 2024 was $(107.2) million, a significant improvement compared to $(496.9) million for the same period in 2023, reflecting a reduction of approximately 78%[222]. - Net cash used in investing activities during the nine months ended September 30, 2024 was $(0.4) million, down from $(0.7) million in the same period of 2023, indicating a decrease of about 43%[221]. - Net cash provided by financing activities was $153.2 million for the nine months ended September 30, 2024, compared to $398.4 million for the same period in 2023, representing a decline of approximately 62%[223]. - The company expects its current cash and cash equivalents of $396.9 million will be sufficient to fund projected operating expenses and capital expenditures for at least the next 12 months[225]. - As of September 30, 2024, the company had cash and cash equivalents of $396.9 million, primarily in money market funds and U.S. Government obligations[231]. Product Development - The VALIANT trial demonstrated a 68% reduction in proteinuria in C3G and IC-MPGN patients compared to placebo (p < 0.0001)[136]. - The company is developing additional product candidates, including APL-3007, a small interfering RNA in Phase 1 clinical trial, and an oral complement inhibitor in preclinical development[138]. - The company plans to submit a supplemental new drug application to the FDA in early 2025 for systemic pegcetacoplan[137]. - The company is focusing on the ongoing development of systemic pegcetacoplan and prioritizing research initiatives on high potential opportunities[224]. - The company is investing substantial resources in the commercial infrastructure for SYFOVRE for GA and the development of product candidates[226]. - If cash generated from sales of EMPAVELI and SYFOVRE is insufficient, the company may need to seek external funding sources, including equity offerings or debt financings[228]. - The company may need to delay or limit product development if it cannot generate sufficient funds from sales or raise additional capital[229]. Inventory and Assets - The remaining pre-FDA approved inventory as of September 30, 2024, was valued at $15.4 million, primarily consisting of raw materials[188]. - As of September 30, 2024, the remaining pre-FDA approved inventory was valued at $15.4 million, primarily consisting of raw materials[203]. - As of September 30, 2024, the company held Convertible Notes in principal amount of $425.4 million, which had not been canceled[165]. - The company has not called for redemption of any Convertible Notes as of September 30, 2024[160]. - The conditional conversion feature of the Convertible Notes was not triggered as of September 30, 2024, meaning they are not convertible during the quarter ending December 31, 2024[165]. Market and Economic Factors - An immediate 10% change in interest rates would not materially affect the fair market value of the company's investment portfolio due to its short-term duration and low risk profile[231].

Statistically significant 68% (p<0.0001) proteinuria reduction compared to placebo, with reduction observed as early as Week 4 All secondary endpoints favored treatment with pegcetacoplan, including: Stabilized eGFR, a key measure of kidney function 71% of patients achieved zero C3c staining intensity, demonstrating complete clearance of C3c deposits Results consistent across subgroups including C3G and IC-MPGN, adolescent and adult patients, and native and post-transplant kidneysDemonstrated favorable safe ...

William Blair has initiated coverage on Apellis Pharmaceuticals, Inc. APLS and notes that Apellis was the first company to successfully target complement C3, which has led to the approval of two therapies: Empaveli (systemic pegcetacoplan) for paroxysmal nocturnal hemoglobinuria and Syfovre (intravitreal pegcetacoplan) for geographic atrophy secondary to age-related macular degeneration.William Blair writes that although Empaveli has established a solid position in the PNH market, short-term challenges are ...

Hey there! I'm a biotech and healthcare investment analyst with several years of bedside clinical experience and a Master of Business Administration. I utilize risk-return charts and DCF analyses to highlight investment opportunities and risks. I'm a big fan of the barbell strategy: 90% in safe assets like Treasuries and ETFs, and 10% in high-growth stocks—keeping risk management front and center. Since markets are pretty efficient and returns can be random, I draw inspiration from 'Superforecasting' and 'A ...

Thank you for reading my research on Seeking Alpha. If you want to learn even more about my method and how I discover these investment opportunities, please check out my subscription marketplace service, Compounding Healthcare , and sign up for a free trial.It has been almost a year since my previous Apellis Pharmaceuticals, Inc. (NASDAQ: APLS ) article , where I discussed the company's C3 inhibitor technology, as well as the prospects for their flagship treatment, pegcetacoplan. Despite my trepidation abou ...

Apellis Pharmaceuticals (APLS) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has confirmed its June 2024 negative opinion on the regulatory filing for intravitreal pegcetacoplan to treat geographic atrophy (GA) secondary to age-related macular degeneration (AMD). Shares of the company lost 11.5% on Friday in response.Management expressed deep disappointment in the outcome, stating that millions of GA patients in the EU remain without treatmen ...

WALTHAM, Mass., Sept. 20, 2024 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has confirmed its June 2024 negative opinion on the marketing authorization application of intravitreal pegcetacoplan for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration. “We are deeply disappointed by this outcome, which leaves millions of Europeans with GA ...

Met the primary endpoint, achieving statistically significant 68% (p<0.0001) reduction in proteinuria compared to placebo in a broad study population Positive results consistent across all subgroups, including C3G and IC-MPGN, adolescent and adult patients, and native and post-transplant kidneys Demonstrated favourable safety, consistent with established profile Companies plan to submit data for regulatory approval in the US and EU STOCKHOLM, Aug. 8, 2024 /PRNewswire/ -- Sobi® (STO: SOBI) and Apellis Pha ...

Met the primary endpoint, achieving statistically significant 68% (p<0.0001) reduction in proteinuria compared to placebo in a broad study populationPositive results consistent across all subgroups, including C3G and IC-MPGN, adolescent and adult patients, and native and post-transplant kidneysDemonstrated favourable safety, consistent with established profileCompanies plan to submit data for regulatory approval in the US and EU STOCKHOLM, Aug. 8, 2024 /PRNewswire/ -- Sobi® (STO: SOBI) and Apellis Pharmaceu ...