Cerecor Inc. a biopharmaceutical company focused on becoming a leader in the development and commercialization of treatments for rare and orphan diseases, announced final efficacy data including 60-day mortality from their recently completed US-based, multi-center (10 sites), Phase 2 trial (NCT04412057) of the human anti-LIGHT (TNFSF14) monoclonal antibody CERC-002. This analysis updates the preliminary topline data reported on January 5, 2021. All patients in this trial were hospitalized with COVID-19 associated pneumonia and mild-to-moderate acute respiratory distress syndrome (“ARDS”). A total of 83 patients were randomized 1:1 to receive standard of care at the sites plus either a single dose of 1,200 mg of CERC-002 or placebo subcutaneously. Due to the protocol allowing patients to receive high flow oxygen prior to randomization, 62 patients were included in the intention-to-treat (ITT) analysis of the primary endpoint.
The final analysis inclusive of the 60-day safety update demonstrated the trial met its primary efficacy endpoint (proportion of patients alive and free of respiratory failure over the 28-day study period) compared to placebo in COVID-19 patients with ARDS treated with a single dose of the anti-LIGHT monoclonal antibody CERC-002 (n=62, p=0.044). Efficacy was highest in a prespecified subpopulation of patients over the age of 60 (n=34, p=0.042), the population most vulnerable to severe complications and death with COVID-19 infection.
At both the 28-day and the 60-day final timepoints, an approximately 50% trend in mortality reduction (22.5% vs 10.8%) was observed.
CERC-002 showed statistically significant efficacy on top of corticosteroids and standard of care treatments in COVID-19 ARDS: over 90% of patients received concomitant systemic corticosteroids and over 65% received remdesivir. CERC-002 was well-tolerated. No drug-related serious adverse effects (SAEs) were reported in the trial, and there was no increase in infections in CERC-002 treated patients.
Cerecor has submitted applications to the U.S. Food and Drug Administration (FDA) for Breakthrough Therapy and Fast Track Designations. The Company plans to meet with FDA to discuss the potential path to Emergency Use Authorization (EUA) and approval.
“We would like to thank the patients who participated in this landmark study. These results not only validate our hypothesis that elevated LIGHT is a key driver of morbidity and mortality in COVID-19 ARDS1, but also provide strong initial evidence that LIGHT neutralization with CERC-002 in this setting may be able to save lives and can spare patients from the need for ventilatory support,” said H. Jeffrey Wilkins, MD, Chief Medical Officer of Cerecor. “These results are all the more encouraging given that this novel targeted therapy demonstrated efficacy and excellent tolerability despite being administered over and above standard of care including high dose systemic corticosteroids.”
“Despite progress with vaccinations, thousands of people in the US and around the world continue to die of COVID-19 related cytokine release syndrome and ARDS every day. There is an urgent need for new safe and effective therapies to treat these patients,” said Dr. David Perlin, Ph.D., Chief Scientific Officer and Senior Vice President of the Center for Discovery and Innovation at the Hackensack Meridian School of Medicine. “The need is all the greater given the increasing levels of COVID-19 mutation and resistance emerging in variants. Even with widespread vaccination such therapies will be required for many years to come.”