Type: Second-generation C3 inhibitor designed to control the complement cascade centrally at C3. According to Apellis, APL-9 could treat a range of diseases caused by excessive or uncontrolled activation of complement.
2021 Status: DEVELOPMENT ENDS IN SEVERE COVID-19—Apellis said March 4 it will no longer pursue development of APL-9 for severe COVID-19, following an interim review of mortality data from the Phase I/II APL9-COV-201 trial (NCT04402060) by the study’s independent data monitoring committee, which found no meaningful reduction in the overall mortality rate in patients treated with APL-9 in combination with standard of care therapy compared to standard of care alone. No safety signals were observed by the committee.
“While the mortality results from this study were not what we had hoped, we extend our heartfelt thanks to the patients and their families, healthcare providers, and investigators who joined us in working to address an urgent public health need,” Lukas Scheibler, PhD, chief innovation officer at Apellis, said in a statement. “We remain confident in the potential of targeting C3 for complement-driven diseases and are committed to bringing transformative treatments to patients.”
Enrollment has been completed, and the trial had reached the last patient visit. Apellis said it plans to provide full results “in a scientific forum” following completion of a full data analysis.
2020 Status: Apellis said October 15 that observational study results found a correlation between COVID-19 severity and complement overactivation, a key immune response. Preliminary open-label safety data from six patients in the Phase I/II APL9-COV-201 trial supported advancement of APL-9 in severe COVID-19.
On May 28, Apellis initiated the Phase I/II APL9-COV-201 trial, designed to assess of APL-9 in COVID-19 patients with respiratory failure including acute respiratory distress syndrome (ARDS). The randomized, multi-center, controlled study is expected to enroll 66 hospitalized patients with COVID-19 and respiratory failure who require oxygen supplementation or mechanical ventilation.
The trial’s primary objective is to evaluate the safety of APL-9 as an add-on to the current standard of care. Secondary objectives include evaluating length of stay in the hospital, overall survival, time on oxygen therapy or mechanical ventilation, and markers of complement activation, organ failure, and blood clotting.
“ARDS and harmful blood clots are associated with mortality in COVID-19, and we believe that dysregulation of complement plays a key role in both of these conditions,” Apellis’ chief innovation officer Lukas Scheibler, PhD, said in a statement.
COVID-19: 300 Candidates and Counting
To navigate through the >300 potential therapeutic and vaccine options for COVID-19, GEN has grouped the candidates into four broad categories based on their developmental and (where applicable) clinical progress:
● FRONT RUNNER – the most promising therapeutics/vaccines based on clinical progress, favorable data or both.
● DEFINITELY MAYBE – earlier phases with promising partners, or more advanced candidates in development that have generated uneven data
● KEEPING AN EYE ON… – interesting technology, attracting notable partners, or both, but preliminary data.
● TOO SOON TO TELL – longshots pending additional experimental and/or clinical data.
GEN has also tagged the most common treatment types: