Apellis Pharmaceuticals (NASDAQ:APLS) used its presentation at the 44th Annual JPMorgan Healthcare Conference to outline its near-term commercial priorities for Syfovre and Empaveli, discuss clinical and regulatory milestones, and preview longer-term pipeline ambitions built around complement factor C3.
Company focus and 2025 as a “building year”
CEO Cedric Francois described Apellis as focused on the complement pathways, specifically complement factor C3, which he called central to the immune complement cascade and a foundation for pursuing “many therapeutic options across many therapeutic areas.” He said the company is currently self-funded and characterized 2025 as a “building year” focused on establishing a foundation around Syfovre and Empaveli while shaping Apellis’ future pipeline.
Syfovre: market position, growth drivers, and upcoming prefilled syringe filing
On Syfovre in geographic atrophy (GA), Francois said Apellis remains the market leader with roughly 60% market share and that total injections grew about 17% year over year. He also said the product is in a preferred position with many payers and emphasized dosing every two months as a way to convey benefit.
Francois described GA patient segments as including advanced GA, GA in patients who also have wet age-related macular degeneration (where patients may be co-treated with Syfovre and anti-VEGF therapy), and early GA, which he said could broaden as use in retina practices becomes more established. He indicated Apellis expects execution and clinical evidence to support growth in advanced GA and GA with wet AMD in the first half of 2026, with the potential to expand share versus its “only competitor” in the second half of 2026.
He highlighted what he described as the largest GA dataset generated to date: five years of follow-up showing that treatment can slow GA progression, which he summarized as “the ability to slow down, over the course of five years, the progression of GA by one and a half years.” He framed that as meaningful for patients typically in their 70s and 80s.
Apellis also discussed a prefilled syringe intended to simplify administration in retina practices. Francois said the clinical trial for the prefilled syringe is complete and that the company plans to file in the first half of the year, with additional timing guidance to be provided later in the year on when it could reach the market. He described current preparation as time-consuming—thawing a vial, drawing a viscous product through a filter needle, switching needles, and managing workflow—whereas the prefilled syringe could improve clinic flow and potentially provide a competitive advantage.
Syfovre: functional imaging effort and Europe strategy comments
Francois also discussed work aimed at making GA’s functional impact more tangible. He said GA studies have traditionally focused on slowing the growth of the atrophic retinal area, while linking that to real-world visual function has been challenging. He said Apellis has leveraged artificial intelligence to translate standard SD-OCT images into a “functional map” of what a patient sees, an approach he referred to as “functional OCT.” He also referenced development of an augmented reality headset intended to help family members experience the visual impact of the disease.
In response to an audience question about the lack of European Medicines Agency approval for Syfovre, Francois said Europe’s regulatory process included concerns about measuring functional benefit. He said Apellis believes it is “cracking that code” and hopes there will be paths to revisit global strategies to make Syfovre available in Europe and other markets, though he did not provide a specific timeline.
Empaveli: C3G/IC-MPGN launch progress and phase III expansion plans
Turning to Empaveli, Francois said the product—previously approved for paroxysmal nocturnal hemoglobinuria (PNH)—is now approved for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). He cited combined U.S. epidemiology of approximately 5,000 patients and said about half could ultimately be on Empaveli treatment.
Francois said the C3G/IC-MPGN approval occurred at the end of July and that the company began launching in August. He stated Apellis had achieved “more than 5% penetration after the first full quarter” and described payer reception as strong. He attributed launch momentum to Empaveli’s efficacy and safety profile and highlighted uptake in pediatric patients and in transplanted patients, noting the perceived urgency can be higher in those groups.
During Q&A, Francois also pointed to a December publication in The New England Journal of Medicine and described what he called a “trifecta” of data from the Valiant study:
- Proteinuria benefit he described as unprecedented in these patients
- Biopsy findings showing that after six months of treatment, 70% of patients had no trace of complement deposition in the kidney
- eGFR impact observed as early as six months
He also said the FDA accepted six months of data for review leading to approval, which he suggested differentiated Empaveli’s regulatory path.
For 2026, Francois said priorities for durable growth include improving patient identification and diagnosis, educating nephrologists to increase urgency in treatment decisions, and differentiating Empaveli through efficacy, safety, and real-world evidence. He highlighted safety as a key point, stating Apellis has “well north of 3,000 patient years of dosing” and has observed zero cases of encapsulated meningococcal infection on Empaveli, while noting typical expectations for the class.
Apellis also discussed two ongoing phase III programs for Empaveli in focal segmental glomerulosclerosis (FSGS) and delayed graft function (DGF), both of which Francois said are currently enrolling. He cited roughly 13,000 U.S. patients for FSGS and described DGF as affecting about 30% to 35% of deceased-donor kidney transplants in the first week post-transplant, which he said is associated with complement activation. When asked about timelines, Francois said it was too early to guide given the trials had recently started, adding that transplantation studies can be more challenging to run.
Separately, Francois previewed a gene-editing program, APL-9099, aimed at disrupting what he called a $20 billion FcRn market. He said the approach uses base editing in the liver delivered via lipid nanoparticles (LNPs), with the goal of reducing IgG levels by about 50% while maintaining albumin levels, and that an IND is planned for the second half of the year.
About Apellis Pharmaceuticals (NASDAQ:APLS)
Apellis Pharmaceuticals, Inc, traded as NASDAQ:APLS, is a clinical-stage biopharmaceutical company focused on the development of novel therapies targeting the complement cascade for the treatment of rare and debilitating diseases. The company’s research and development efforts center on modulating complement proteins to address a range of ophthalmologic, hematologic and renal conditions. Apellis leverages its proprietary compstatin technology platform to design targeted inhibitors intended to improve patient outcomes and quality of life.
The company’s lead marketed product, Syfovre (pegcetacoplan), is an intravitreal complement C3 inhibitor approved for geographic atrophy secondary to age-related macular degeneration, with ongoing investigations in other retinal disorders.
