Apellis Pharmaceuticals (NASDAQ:APLS) executives outlined a three-part strategy for the company at TD Cowen’s 46th Annual Healthcare Conference, emphasizing a “durable and resilient” SYFOVRE franchise in geographic atrophy (GA), an expanding EMPAVELI launch in rare kidney diseases, and a pipeline that includes a gene-editing collaboration aimed at the FcRn class.
Management highlights three pillars
Chief Executive Officer Cedric Francois said 2025 was a year focused on “building a good foundation” for the company’s next phase, with Apellis now organized around three pillars:
- SYFOVRE (GA): Management characterized the GA business as durable and said the company plans to continue providing a best-in-class therapy.
- EMPAVELI (nephrology): Apellis is in the midst of what it described as an “exciting launch” for C3G and IC-MPGN, alongside ongoing phase III trials in focal segmental glomerulosclerosis (FSGS) and delayed graft function (DGF).
- Pipeline and innovation: Francois highlighted a planned move into the clinic in the second half of the year with what he described as the first gene-editing program for the FcRn class in collaboration with Beam, framing it as a potential “once-and-done treatment.”
Why revenue declined year-over-year in 2025
On EMPAVELI’s initial indication, paroxysmal nocturnal hemoglobinuria (PNH), Sullivan said revenue was essentially flat at roughly $18 million to $20 million per quarter. However, he pointed to “very exciting” growth following approvals in C3G and IC-MPGN, describing the launch as continuing with “great momentum” exiting 2025 and with no anticipated near-term inflection.
On SYFOVRE, Sullivan cited disruption stemming from changes in patient co-pay assistance. He said patients relying on assistance were affected when co-pay assistance organizations “were no longer being funded,” driving some patients to free goods or discontinuation and causing broader disruption across retinal practices. Despite that, he noted Apellis saw 17% growth in injections, which management framed as evidence that underlying demand for GA treatment continued to grow.
Sullivan also referenced an inventory build at the end of the fourth quarter of 2024 that contributed to a lower revenue figure in the first quarter of 2025, though he said it was less significant than the co-pay issue.
SYFOVRE launch status: market share, safety, and data narrative
Francois said SYFOVRE is near the three-year anniversary of launch and emphasized that only a small percentage of GA patients have been treated to date. He also said the company recently presented five-year data in GA and argued the dataset supports meaningful long-term benefit for patients who remain on therapy.
On market dynamics, Francois said SYFOVRE’s share has been “remarkably stable” at about a 60%/40% split in favor of SYFOVRE for the past year to year-and-a-half, while share of new injections has been closer to 50%/50%. He attributed some of that stability to what he described as better real-world treatment persistence on SYFOVRE relative to a competitor.
Discussing injection cadence using a dataset from what he described as the “largest private equity group” accounting for about 30% of Apellis’ volume, Francois said the average interval between injections was about eight weeks for SYFOVRE versus about seven weeks for the competitor, with slightly higher injections per patient apparent in that dataset.
On safety, Francois addressed concerns about vasculitis during the launch period. He said Apellis’ pharmacovigilance work suggested the event was very rare and not meaningfully different from other intravitreal injections. He cited 2025 experience of approximately 370,000 injections across about 32,000 new patients, with two vasculitis cases that he said both resolved back to baseline.
Looking ahead, executives highlighted potential catalysts they believe could support broader adoption, including a prefilled syringe—which Francois described as effectively a “new launch” that could make administration more efficient. Francois also discussed “functional OCT” work intended to better demonstrate disease impact and treatment value to families and physicians using standard OCT imaging.
EMPAVELI kidney launch: early penetration and differentiation
Francois said EMPAVELI achieved more than 5% penetration in its first full quarter in a combined estimated epidemiology of approximately 5,000 patients with C3G and IC-MPGN. He said Apellis believes it is on a path to treat as many as half of that patient population at peak.
Management acknowledged investor debate about an early “bolus” of warehouse patients. Francois said an initial bolus is typical in diseases with high unmet need, citing expanded access and patients who had been waiting, and said the company is now seeing “linear steady growth” consistent with early launch expectations.
In discussing competition, Francois said EMPAVELI is the only product approved on-label for pediatric patients and also has approval in the post-transplant population, while competition is primarily in adults. He also highlighted what he described as an established safety record of “well north of 3,000 patient years” of dosing experience and said the company has observed zero encapsulated meningococcal infections to date, which he contrasted with infection rates he said are seen with vaccinated C5 inhibitors. He said Apellis is preparing a publication on that point.
Regarding the route of administration—oral versus injection—Francois said that in life-threatening kidney diseases, convenience may be less important than avoiding progression to hemodialysis and managing relapse risk post-transplant.
Pipeline readouts and cash runway discussion
Francois also described a phase II program combining a subcutaneous siRNA against C3 (referred to as 3007) with intravitreal SYFOVRE, aiming for a readout “throughout the course of next year.” He said the goal is to improve outcomes versus SYFOVRE alone, particularly by further slowing retinal pigment epithelium (RPE) cell loss and potentially enabling a shift from every-two-month dosing to every-three-month visits through a combination of intravitreal SYFOVRE and a subcutaneous injection administered in-practice. He said proof-of-concept would be a significant additional slowdown on the primary endpoint beyond SYFOVRE alone, with a functional OCT secondary endpoint intended to capture patient benefit.
On financial resources, Sullivan said Apellis ended the year with $466 million in cash and received a final $25 million installment in the first quarter tied to monetization of a Sobi royalty. He also said the company expects another milestone payment from Sobi triggered when reimbursement is obtained in Europe for C3G and IC-MPGN, which he said would bring cash to “over $500 million.” Sullivan noted interest expense of roughly $10 million per quarter and said the company has a $94 million convertible obligation to address by the end of September, adding that Apellis has “a range of opportunities” to manage it.
As the discussion concluded, management reiterated focus areas for the coming year, pointing to SYFOVRE’s trajectory, continued EMPAVELI launch execution, and the planned second-half clinical entry for the FcRn gene-editing collaboration.
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.
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