Pegcetacoplan Study Cliffhanger
Last updated: May 2023
Cliffhangers can be exciting! Will the cavalry get there in time? Will the people be rescued? We wait on the edges of our seats to find out.
Pegcetacoplan clinical trial
There has been a bit of a cliffhanger playing out with “my” study medication, pegcetacoplan – the drug formerly known as APL-2. Will this medication make it in time to become the first treatment for dry AMD (age-related macular degeneration)? Will the FDA decide to approve it?
And, important for me, will there be a “sequel” so I can make the next step in my diabolical plan? I have been waiting with bated breath!
Phase 2
The story starts several years ago. In the phase 2 trials, things looked really promising. Phase 2 is “proof of concept” and the results suggested APL-2 was going to be pretty effective at slowing down the rate of progression of the disease.1
Apellis, the pharmaceutical company, announced there had been a 29% reduction in the rate of lesion growth at the end of 12 months. That meant the people in the clinical trial were “going blind” at 71 mph and not 100 mph. Encouraging!1
Phase 3
Fast forward to phase 3. That is the phase of the study I was in. Phase 3 is another proof of concept phase but with many more people. Unfortunately, the results of the phase 3 studies were not as impressive. The 2, different experimental groups, DERBY and OAKES, were more lackluster with DERBY missing the cut all together.1
In other words the difference between the rate of growth of the lesions in treated eyes and the rate of lesion growth in the untreated eyes were not great enough to recommend the treatment in the DERBY people.1
The verdict
There was talk of going back to the drawing board as it were. Maybe running more subjects over a longer period of time would improve the findings. My doctor said one option might be to submit the data they were gathering from us in the long-term study that I am in now.
The way I understand it, they decided to try a different track. Someone did some statistical magic and, with the approval of the FDA, combined the results of the 2, concurrent groups that were run in phase 3. Put together, the results of the studies meet significance and APL-2 stands a good chance of being approved. Maybe.1
Hopeful
So why am I pretty optimistic the calvary will get here in time? For one, the mood at my study location is pretty upbeat. While they are not supposed to, and don’t, share, I get the feeling they see APL-2 making the cut and being approved by the FDA.
Also, I got some of this information initially from multiple investment blogs. One put APL-2 as their lead story. Why do that if they do not see it as a hot tip? If the investment scuttlebutt is correct, by the time this page is published, APL-2 may have reached approval. The drug will become the first treatment for dry AMD in history!
Are you interested?
Would I opt for APL-2 if I were not in the study? Probably. While the 17% slowing in lesion growth rate in one of the studies is not amazing, it is better than nothing and potentially could be better than that for some people.1
Look at phase 2 results to get an idea of how it worked on others. And if you don’t want to go with APL-2, wait for a few months. Iveric Bio is projected to apply for approval for Zimura early in 2023.1
There is lots of hope for the new year!

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