As CEO bounces back from Covid-19, Apellis says FDA meeting clears path to file for an OK on Soliris rival
After holding up the company more than 4 months after reporting positive head-to-head data against the blockbuster drug Soliris, Apellis $APLS says it’s cleared things up with the FDA and is ready to file for an approval with their rival PNH drug pegcetacoplan.
The Waltham, MA-based biotech — whose CEO, Cedric Francois, has recovered from a bout with Covid-19 — put out the word Thursday morning that execs had a “successful outcome of the pre-NDA meeting” with the agency and they’re ready to press ahead with their filing in the second half of this year.
The unexpected delay marred Apellis’ positive readout back in early January, as researchers tracked an “improvement in adjusted means of 3.8 g/dL of hemoglobin at week 16 (p<0.0001). At week 16, pegcetacoplan-treated patients (n=41) had an adjusted mean hemoglobin increase of 2.4 g/dL from a baseline of 8.7 g/dL, compared to eculizumab-treated patients (n=39) who had a change of -1.5 g/dL from a baseline of 8.7 g/dL.”
The hitch, though, was spelled out in an SEC filing. The agency told them that:
(F)or the approval of a new treatment for PNH, hemoglobin stabilization in conjunction with change in transfusion dependence constitute accepted clinical benefit, but that a rise in hemoglobin levels may not translate to clinical benefit in patients who entered the trial with high hemoglobin levels, such as permitted by the inclusion criteria of the PEGASUS trial, and who do not require transfusions.
Apellis didn’t spell out the agency’s new position, but evidently no longer believes the FDA may have trouble with its application.
Phil Nadeau at Cowen, meanwhile, says he’s expecting data from a study involving treatment-naive patients in the early part of next year after Covid-19 delayed some of the company’s timelines on development. He noted this morning:
(T)here had remained a chance that FDA would want additional data beyond PEGASUS and that the NDA filing would have to wait until results from the second Phase III trial in treatment-naive PNH patients in early 2021. With this morning’s announcement, that possibility has been removed.
Nadeau is expecting an approval with peak sales ahead of $400 million a year.
As we reported a couple of months ago, Francois posted that he was one of the first to be infected by coronavirus during the first wave. A spokesperson for the company tells me:
Cedric has been fully recovered for some time now with a high level of antibodies, and is excited for the future of Apellis and to see all the collaboration between biotechs and healthcare companies during this pandemic.