Apellis Pharmaceuticals, Inc. is reporting new long-term data on its FDA-approved SYFOVRE (pegcetacoplan injection), indicated for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
Notably: This news follows 2 years since its last data readout on the therapeutic.
First, a look at Syfovre.
Its claim to fame: The therapeutic received regulatory approval in the United States in 2023 as the first treatment option for patients with GA secondary to AMD.
What it is: An intravitreally (IVT)-administered formulation dosed once every 25 to 60 days into each affected eye to potentially reduce the progression of GA.
- That dosage: 15 mg/0.1 mL
- See here for its full prescribing information.
As for this data … what study is it based on?
That would be a post-hoc analysis from GALE, a phase 3 multicenter, open-label extension study.
Its purpose: Evaluating the long-term efficacy (5 years, in total) and safety of SYFOVRE among 792 patients.
- But more specifically, investigators wanted to determine:
- Long-term incidence and severity of ocular and systemic treatment-emergent adverse events (TEAEs)
- Total area of GA lesions as measured by fundus autofluorescence (FAF)
And what do we know about the participants in GALE?
A reported 80% of the 286 nonsubfoveal GA patients included in the study had previously completed the phase 3 OAKS and DERBY studies (whose data were used to support SYFOVRE’s regulatory approval).
Specifically:
- Patients in the SYFOVRE treatment arms through Month 24 of the DERBY and OAKS studies continued on the same regimen in the GALE extension.
- Sham-treated patients from the DERBY and OAKS studies were eligible to transition to SYFOVRE treatment following Month 24—as such, a projected sham arm was used to estimate GA lesion growth without treatment between Months 24 and 60 (the 5-year mark).
Noted. Now, what is the 5-year data showing?
To start, here’s a breakdown of treatment groups:
- SYFOVRE monthly (n = 95)
- SYFVORE every-other-month (EOM): (n = 104)
- Sham: (n = 87)
Following 5 years of continuous SYFOVRE treatment, investigators found that “both (EOM) and monthly” dosings delayed GA lesion growth by an estimated 1.5 years in patients
Any numbers to back this up?
From baseline to Month 60, SYOVRE led to:
- A 31% reduction in GA lesion growth for the monthly dosing group (p < 0.0001)
- A 27% reduction in GA lesion growth for the EOM dosing group (p < 0.0001)
So what does this mean?
The takeaway: Early and continuous treatment with SYFOVRE leads to a meaningful delay in GA progression.
- As Apellis Chief Medical Office Caroline Baumal, MD, noted, this new data underscores SYFOVRE’s “durable impact of targeting C3.”
The company also emphasized that SYFOVRE’s safety profile through the 5-year period “remained consistent” with previously reported GALE data.
Talk about that prior data.
Back in 2023, we covered GALE’s 3-year findings, which determined that SYFOVRE:
- Reduced GA lesion growth with both monthly (35%; p<0.0001) and EOM (24%; p<0.0001) treatment compared to the projected sham arm
- Reduced nonsubfoveal GA lesion growth with both monthly (42%; p<0.0001) and EOM (28%; p=0.0015) treatment compared to the projected sham arm
See here for more from this coverage.
Circling back to these 5-year results, any other data to report?
That’s all for now. However, Apellis also shared plans to present more “detailed results” at a future (currently unnamed) medical meeting.
So you know what that means … stay tuned!