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Apellis Pharmaceuticals: Benefit/Risk Ratio Worsens

Healthcare Explorer profile picture
Healthcare Explorer


  • One year of treatment with APL-2 has NO impact on best corrected visual acuity (BCVA).
  • Treated patients continue to convert to wet AMD even after stopping therapy.
  • Treatment thus far demonstrates no clinical benefit but certainly gives plenty of adverse events.

Apellis Pharmaceuticals (NASDAQ:APLS) presented “updated” data from their FILLY trial at the 2018 Macula Society in late February (slides). Here, they showed some additional tidbits that could be of interest, but as I reviewed the presentation, for the first time, they revealed the actual best corrected visual acuity (BCVA) for patients in their trial. If you recall, the primary endpoint of the FILLY trial is LS mean change from baseline in square root GA (geographic atrophy). This is a long way of saying the change in size of the lesion. The company is using the area of atrophy as a surrogate for visual acuity. As I have written before, the reason that the FDA allows this surrogate is that it is believed that the size of the lesion would reasonably predict changes in visual acuity. However, since no one has been able to really change or reverse the lesions, we actually don’t know how good of a surrogate it would be nor the magnitude of the change that is required to show a measurable difference. For the first time, we actually have data that shows some data on this - and it isn't good.

FILLY Trial Design

Briefly, the trial compared, on a 1:1:1 basis, sham, every other month (EOM), and monthly injections of APL-2 for one year. After one year, treatment was stopped and patients were followed for another six months. I think the purpose of this observation period was for safety and to see what happened to lesions after stopping treatment. The most obvious predicted outcome is that the lesions resume their pretreatment growth rate. This seems to be what happened. This data isn’t all that interesting, but the safety data and BCVA data were shown for the first time.

BCVA impact of treatment

For the first time, the

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