Flush with cash, Apellis trumpets positive Phase 2 AMD results
- Apellis Pharmaceuticals, Inc. said Thursday its clinical candidate APL-2 met its primary endpoint in a Phase 3 trial in 246 patients with geographic atrophy (GA) associated with age-related macular degeneration.
- Monthly intravitreal injections of the complement C-3 inhibitor reduced the rate of GA lesion growth by 29% over 12 months, compared with placebo. The rate of growth fell by 20% with injections every other month, meeting the study's pre-specified level of significance for that measure, according to the company.
- Apellis is also looking at genetic markers from the study, and Phase 3 studies are planned in GA and paroxysmal nocturnal hemoglobinuria (PNH).
Correction: A previous version of this article incorrectly indicated the rate of lesion growth with every other month administration of APL-2 did not meet statistical significance.
Dry AMD with GA is currently an untreatable condition, and so anything that offers a reduction in progression and potentially vision maintenance will likely be well received by both patients and physicians.
Adding to the top-line results, Apellis cited a post hoc analysis that seemed to show an improved response to the drug over time.
"In addition to demonstrating a statistically significant slowing of disease over 12 months, APL-2’s effect appears to increase in the second six months of the study, slowing down the rate of degeneration by almost half," said Cedric Francois, founder and CEO of Apellis. "We plan to move forward with Phase 3 studies as soon as possible."
Just two weeks ago, Apellis closed a Series E preferred stock financing for $60 million, providing it with funds to start Phase 3 trials with APL-2 in PNH, and move development forward in other indications. Results from a Phase 1 study in autoimmune hemolytic anemia are expected later in 2017.
Development in those indications will set APL-2 up against Soliris (eculizumab), Alexion's complement inhibitor approved for PNH and atypical hemolytic uremic syndrome (aHUS). A future approval for APL-2 in PNH could up competition for Soliris, although Alexion has built a large lead. The company expects Soliris to pull in more than $3 billion in 2017.
Apellis has generated some early data that shows promise. In the Phase 1b PADDOCK study, the three patients with PNH who had never received eculizumab showed rapid corrections in lactate dehydrogenase (LDH), a key PNH marker. And in the Phase 1b PHARAOH study, six patients who had responded suboptimally to eculizumab showed increases in levels of hemoglobin and rapid corrections in LDH.
- Apellis Pharmaceuticals, Inc. Press release
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