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Financial Data and Key Metrics Changes - The company is currently cash neutral and on a path to profitability, with over $450 million in cash available [6][64] - The company reported a dramatic reduction in proteinuria of approximately 68% in patients with C3 glomerulopathy and immune complex membranoproliferative glomerulonephritis [10][11] - The company has been skating close to cash operational expenses, indicating strong financial health [64] Business Line Data and Key Metrics Changes - Apellis has two approved drugs: EMPAVELI for paroxysmal nocturnal hemoglobinuria (PNH) and recently approved indications for C3 glomerulopathy and immune complex membranoproliferative glomerulonephritis [5][6] - SYFOVRE, the first treatment for geographic atrophy, was approved in 2023 and has stabilized its market share at just north of 60% [6][45] Market Data and Key Metrics Changes - The prevalence of C3G and IC-MPGN in the U.S. is approximately 5,000 patients, with about 50% progressing to end-stage renal disease over ten years [9] - The company has achieved around 5% market penetration with 225 start forms for the new indications within five months [14][32] Company Strategy and Development Direction - The company is focused on educating nephrologists about its new drug indications and differentiating its products from competitors [16][19] - Future trials are planned for FSGS and delayed graft function, with expectations of positive outcomes based on the underlying disease mechanisms [26][28] Management's Comments on Operating Environment and Future Outlook - Management noted that the competitive landscape for PNH has not led to significant patient loss, with 15% of patients returning to EMPAVELI after trying oral alternatives [24] - The company anticipates a strong 2026, with ongoing efforts to improve physician education and patient access to treatments [64][77] Other Important Information - The company is developing a siRNA combination therapy that aims to enhance the efficacy of SYFOVRE and potentially allow for less frequent dosing [67][69] - There is a focus on leveraging machine learning and AI to improve patient imaging and tracking disease progression [58][61] Q&A Session Summary Question: What is the opportunity with C3G and IC-MPGN? - The company highlighted the significant unmet need in these kidney diseases and the potential for its drug to provide a new treatment option [9][10] Question: How will the company increase its market share in the 5,000 prevalent pool? - Management emphasized the importance of education and building relationships with nephrologists to drive adoption [16][19] Question: What is the current status of SYFOVRE in the market? - The company reported stabilization in market share and ongoing efforts to communicate the drug's benefits to the retina community [40][45] Question: What are the timelines for upcoming studies? - The company indicated that trials for FSGS and delayed graft function have just opened, with updates expected next year [28][29] Question: How is the company addressing the impact of patient copay assistance foundations? - Management acknowledged the significant impact on patient access and is focusing on physician education to mitigate these challenges [75][76]

Financial Data and Key Metrics Changes - The company is currently cash neutral and on a path to profitability, with over $450 million in cash available [6][64] - The company reported a dramatic reduction in proteinuria of approximately 68% in patients with C3 glomerulopathy and immune complex membranoproliferative glomerulonephritis [10][11] - The company has been skating close to cash operational expenses, indicating strong financial health [64] Business Line Data and Key Metrics Changes - Empaveli, the first approved drug, has received additional approvals for C3 glomerulopathy and immune complex membranoproliferative glomerulonephritis, expanding its market potential [5][6] - Syfovre, the first treatment for geographic atrophy, has stabilized its market share at just north of 60% [45] - The company has achieved approximately 5% penetration into the 5,000 prevalent pool of C3G and IC-MPGN patients in the U.S. [32] Market Data and Key Metrics Changes - The prevalence of C3G and IC-MPGN in the U.S. is approximately 5,000 patients, with about 50% expected to progress to end-stage renal disease over ten years [9] - The company has seen significant adoption in the pediatric population, with a growing interest from adult nephrologists and transplant centers [19] Company Strategy and Development Direction - The company is focused on educating nephrologists about its products, particularly in the pediatric and transplant nephrology communities [16][19] - Future trials are planned for FSGS and delayed graft function, with expectations of positive outcomes based on the underlying disease mechanisms [26][27] - The company aims to redefine how the retina community understands geographic atrophy and improve patient management through new imaging technologies [56][57] Management's Comments on Operating Environment and Future Outlook - Management noted that the competitive landscape for PNH has not led to significant patient loss, with some patients returning to Empaveli after trying oral alternatives [24] - The company anticipates a recovery in the geographic atrophy market as practices stabilize and education efforts continue [41][43] - Management expressed confidence in the financial health and future profitability based on current commercial products and expense profiles [64] Other Important Information - The company is developing a siRNA combination therapy that aims to enhance the efficacy of Syfovre and extend dosing intervals [67][68] - There is ongoing concern regarding patient copay assistance foundations, which have significantly impacted patient access to treatments [75] Q&A Session Summary Question: What is the opportunity with C3G and IC-MPGN? - The company highlighted the significant unmet need in these kidney diseases and the potential for its drug to provide effective treatment [9][10] Question: How will the company increase its market share in the prevalent pool? - Education and awareness among nephrologists are key strategies to increase adoption and market share [16][19] Question: What is the current status of Syfovre in the market? - Syfovre has stabilized its market share and is expected to grow as practices adapt to new treatment paradigms [45][41] Question: What are the timelines for upcoming trials? - Trials for FSGS and delayed graft function have just opened, with updates expected next year [28][29] Question: How does the company plan to address the impact of copay assistance issues? - The focus is on physician education to facilitate patient access to treatments despite copay challenges [75]

Financial Data and Key Metrics Changes - The company is currently cash neutral and on a path to profitability, with over $450 million in cash available [6][61] - The company reported a dramatic reduction in proteinuria of approximately 68% in patients with C3 glomerulopathy and immune complex membranoproliferative glomerulonephritis [10] Business Line Data and Key Metrics Changes - Apellis has two approved drugs: Empaveli for paroxysmal nocturnal hemoglobinuria (PNH) and recently for C3 glomerulopathy and immune complex membranoproliferative glomerulonephritis, and Syfovre for geographic atrophy [5][6] - The company has achieved approximately 5% market penetration in the C3G and IC-MPGN indications, with 225 start forms reported by the end of the year [13][31] Market Data and Key Metrics Changes - The prevalence of C3G and IC-MPGN in the U.S. is approximately 5,000 patients, with about 50% expected to progress to end-stage renal disease over ten years [8] - The company has seen a significant adoption of Empaveli in the pediatric population, with interest growing among adult nephrologists [18] Company Strategy and Development Direction - The company is focused on educating nephrologists about its new drug indications and the unique benefits of its therapies compared to oral alternatives [15][17] - Future trials are planned for FSGS and delayed graft function, with expectations of positive outcomes based on the underlying disease mechanisms [25][26] Management's Comments on Operating Environment and Future Outlook - Management noted that 2025 was a challenging year for retina practices due to funding issues affecting patient access to treatments, but they expect a gradual recovery [39][40] - The company is optimistic about the long-term data from the Gale study and the potential for Syfovre to improve patient outcomes in geographic atrophy [41][42] Other Important Information - The company is developing a siRNA combination therapy that aims to enhance the efficacy of Syfovre and potentially allow for less frequent dosing [64][66] - Management emphasized the importance of new imaging technologies to better track disease progression and treatment efficacy in retinal diseases [51][54] Q&A Session Summary Question: What is the potential for growth in PNH? - Management indicated that despite the introduction of oral alternatives, many patients have returned to Empaveli, demonstrating its strong efficacy and safety profile [22][23] Question: How does the company plan to increase market share in C3G and IC-MPGN? - The strategy focuses on education and building relationships with nephrologists, particularly in pediatric and transplant settings [15][18] Question: What is the current market share for Syfovre? - The company has stabilized its market share just north of 60%, with ongoing efforts to maintain and grow this position [43] Question: What are the timelines for upcoming trials? - Trials for FSGS and delayed graft function have just opened, with updates expected next year [27][28] Question: How is the company addressing the impact of patient copay assistance foundations? - Management acknowledged the significant impact of funding issues on patient access and is focusing on physician education to mitigate these challenges [72][74]

Core Insights - Apellis Pharmaceuticals Inc. is highlighted as a promising investment opportunity due to the positive long-term benefits of its drug SYFOVRE for treating geographic atrophy (GA) secondary to age-related macular degeneration (AMD) [1][3] Group 1: Drug Efficacy and Study Results - A post hoc analysis of the Phase 3 GALE extension study indicates that SYFOVRE can delay GA lesion growth by approximately 1.5 years with both monthly and bi-monthly administration compared to sham treatment [2][3] - SYFOVRE is the first FDA-approved therapy for geographic atrophy, targeting C3 to control the complement cascade effectively [3] Group 2: Disease Impact - Geographic atrophy is a progressive and irreversible disease that leads to blindness, affecting over 1 million Americans and 5 million people globally [2]

Core Insights - Apellis Pharmaceuticals Inc. is considered one of the most oversold biotech stocks to invest in, with a Neutral rating maintained by Mizuho and a price target reduction from $24 to $19 [1] Financial Performance - In Q3 2025, Syfovre's revenue was $150.9 million, slightly down from $152.0 million in Q3 2024, but there was a 4% quarter-over-quarter increase in injections [2] - Apellis Pharmaceuticals reported that Syfovre holds over 60% market share in the geographic atrophy (GA) treatment area, with 52% of new patient starts in Q3 2025 [2] - Mizuho has lowered its revenue projections for Syfovre, now estimating a peak annual revenue of about $800 million, which is 25-30% lower than consensus estimates [2] Company Overview - Apellis Pharmaceuticals Inc. is a commercial-stage biopharmaceutical company focused on discovering, developing, and commercializing novel therapeutic compounds for diseases with high unmet needs [2]

Core Insights - Apellis is in a strong position with a foundational technology targeting the complement cascade, specifically C3, which is believed to optimally control complement dysregulation [2] - The company has achieved three drug approvals in approximately 3 to 3.5 years, indicating a robust pipeline and growth potential [2] Drug Approvals - The first approval was for EMPAVELI, targeting paroxysmal nocturnal hemoglobinuria [3] - The second approval was for SYFOVRE, which addresses geographic atrophy, received about 2.5 years ago [3] - Recently, EMPAVELI received a second approval for C3G and IC-MPGN, bringing the total indications to four [3]

Summary of Apellis Pharmaceuticals Conference Call Company Overview - **Company**: Apellis Pharmaceuticals (NasdaqGS:APLS) - **Key Products**: - **Empaveli**: Approved for paroxysmal nocturnal hemoglobinuria, C3G, and IC-MPGN - **Syfovre**: Approved for geographic atrophy Industry Context - **Market Dynamics**: The company operates in the ophthalmology and nephrology sectors, focusing on complement inhibition therapies. Core Points and Arguments Product Performance - **Syfovre**: - Achieved a run rate of approximately **$600 million** after two and a half years post-launch [3] - Facing headwinds due to copay funding and free product distribution, estimated impact of **$40 million** in the first three quarters of the year [9][10] - Efficacy messages from studies, such as saving **1.5 years** of lesion growth, are being emphasized to drive growth [10][11] - **Empaveli**: - Initial launch for C3G and IC-MPGN has been successful with **152 start forms** in the first two months [4][7] - Expected to reach **225 start forms** by the end of the fourth quarter, indicating strong market entry [7][8] - The product is positioned as a disease-modifying therapy, with a focus on pediatric and adult populations [26][27] Market Strategy - **Growth Initiatives**: - Plans to enhance referral patterns from general ophthalmology to retinal specialists [16][17] - Introduction of a prefilled syringe to improve physician workflow and patient compliance [12] - Development of an OCTF program to facilitate patient monitoring and treatment discussions [13] Financial Outlook - **Revenue and Expenses**: - Current cash usage is averaging **$5 million-$10 million** per quarter, with a total of **$500 million** in cash reserves [60] - The company is approaching cash flow neutrality, with a target of **$20 million** in quarterly revenue to achieve this [60] Challenges and Risks - **Competitive Landscape**: The company faces competition in both ophthalmology and nephrology, with the need to establish strong clinical guidelines for Empaveli [30][31] - **Patient Access**: Ongoing challenges with copay funding and patient assistance programs may impact growth [18][19] Future Expectations - **Market Potential**: The total addressable market (TAM) for Empaveli is estimated at **5,000 patients**, with a potential revenue of approximately **$500,000** per patient [51][55] - **Guidance for Next Year**: The company is considering providing guidance but has not committed to specific figures yet [56] Additional Important Insights - **Patient Journey**: The urgency for treatment varies between pediatric and adult populations, with pediatric nephrologists showing a high sense of urgency for early intervention [41][42] - **Compliance Factors**: The twice-weekly dosing of Empaveli is seen as a significant advantage for patient compliance compared to daily oral medications [36][37] This summary encapsulates the key points discussed during the conference call, highlighting the company's strategic positioning, product performance, market challenges, and future outlook.

Core Insights - Apellis Pharmaceuticals announced positive long-term data from the GALE extension study, showing that SYFOVRE® significantly delays the progression of geographic atrophy (GA) secondary to age-related macular degeneration (AMD) [1][2][3] Study Results - The analysis revealed that both monthly and every-other-month administration of SYFOVRE delayed GA lesion growth by approximately 1.5 years compared to a projected sham treatment [2][3] - The safety profile of SYFOVRE remained consistent over the five-year period, aligning with previously reported data [3] Study Design - The GALE study is a Phase 3, multicenter, open-label extension study involving 792 patients, aimed at evaluating the long-term efficacy and safety of SYFOVRE in GA patients [5] - More than 80% of participants from the earlier OAKS and DERBY studies transitioned into the GALE study [5] Mechanism of Action - SYFOVRE targets C3 in the complement cascade, providing comprehensive control of the immune response, which is crucial for managing GA [10] Disease Impact - Geographic atrophy is a leading cause of blindness, affecting over one million Americans and five million people globally, with significant implications for patients' quality of life [11]

Core Insights - Apellis Pharmaceuticals will present at the Stifel 2025 Healthcare Conference on November 12, 2025, at 2:40 p.m. ET [1] - The company has developed two C3-targeting medicines approved for treating four serious diseases, marking significant advancements in complement science [2] Company Overview - Apellis Pharmaceuticals is a global biopharmaceutical company focused on complement science to create therapies for challenging diseases [2] - The company introduced the first new class of complement medicine in 15 years, including the first therapy for geographic atrophy and the first treatment for patients with C3G or primary IC-MPGN [2] - Apellis believes there is significant potential in targeting C3 for various serious diseases [2]