Cerecor Announces CERC-002 Significantly Reduced Respiratory Failure and Mortality in Phase 2 Clinical Trial in Patients Hospitalized with COVID-19 ARDS

Rhea-AI Summary

Cerecor Inc. announced final efficacy data for CERC-002 from a Phase 2 trial in COVID-19 ARDS patients. The study met its primary endpoint, showing a statistically significant reduction in respiratory failure and mortality (p=0.044) at Day 28. Notably, patients over 60 exhibited the highest efficacy (p=0.042). An approximately 50% reduction in mortality was observed at both 28 and 60 days (22.5% vs 10.8%). CERC-002 was well-tolerated, with no serious adverse effects reported. Cerecor has applied for FDA Breakthrough Therapy and Fast Track Designations, aiming for Emergency Use Authorization.

Positive

• CERC-002 met its primary efficacy endpoint with a p-value of 0.044 in reducing respiratory failure and mortality.
• Efficacy was notably higher in patients over 60 years old (p=0.042).
• Approximately 50% reduction in mortality observed at both 28 and 60 days (22.5% vs 10.8%).
• CERC-002 was well-tolerated with no drug-related serious adverse effects reported.
• Cerecor has submitted for FDA Breakthrough Therapy and Fast Track Designations.

Negative

• None.

• Final analysis inclusive of the 60-day safety update in the randomized placebo-controlled study demonstrated CERC-002 was statistically significant in reducing respiratory failure and mortality at Day 28 in patients hospitalized with COVID-19 associated pneumonia and mild to moderate acute respiratory distress syndrome (ARDS), the primary endpoint, (p=0.044)
  
• Efficacy was highest in patients over the age of 60, 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
• Results were observed on top of standard of care including high dose steroids (>90%) and remdesivir (>65%)
• Cerecor has applied for FDA Breakthrough Therapy and Fast Track Designations, and plans to meet with FDA to discuss potential path to Emergency Use Authorization
  

ROCKVILLE, Md. and CHESTERBROOK, Pa., March 02, 2021 (GLOBE NEWSWIRE) -- Cerecor Inc. (NASDAQ: CERC), a biopharmaceutical company focused on becoming a leader in the development and commercialization of treatments for rare and orphan diseases, today 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.”

A presentation of these data updates can be found on the Investors section of the Cerecor website linked here.

CERC-002 (anti-LIGHT monoclonal antibody)

CERC-002 is a fully human anti-LIGHT or tumor necrosis factor superfamily member 14 (TNFSF14) monoclonal antibody licensed from Kyowa Kirin Co., Ltd. It is the only clinical stage anti-LIGHT therapy and has the potential to treat a number of LIGHT-associated immune diseases including cytokine storm-induced COVID-19 ARDS. It is currently in development for pediatric onset Crohn’s disease and cytokine storm induced COVID-19 ARDS. Cerecor has also developed a validated, high sensitivity serum/plasma free LIGHT assay in collaboration with Myriad RBM.

Role of LIGHT in Acute Inflammatory Response

LIGHT (homologous to Lymphotoxin, exhibits inducible expression and competes with HSV glycoprotein D for binding to herpesvirus entry mediator, a receptor expressed on T lymphocytes) is a cytokine with inflammatory actions encoded by the TNFSF14 gene. LIGHT plays an important role in regulating immune responses in the lung, gut and skin. It stimulates T Cell and B Cell response as well as induces the release of other cytokines such as IL-1, IL-6, IL-8, IL-10, TNF and GM-CSF. Therefore, LIGHT potentially plays a key role in immune responses to viral pneumonia and other diseases.

About Cerecor

Cerecor is a biopharmaceutical company focused on becoming a leader in the development and commercialization of treatments for rare and orphan diseases. The company is advancing its clinical-stage pipeline of innovative therapies that address unmet patient needs within rare and orphan diseases. The company's rare disease pipeline includes CERC-801, CERC-802 and CERC-803, which are in development for congenital disorders of glycosylation and CERC-006, an oral mTORc1/c2 inhibitor in development for the treatment of complex lymphatic malformations. The company is also developing two monoclonal antibodies, CERC-002, and CERC-007. CERC-002 targets the cytokine LIGHT (TNFSF14) and is in clinical development for treatment of severe pediatric-onset Crohn's disease, and COVID-19 acute respiratory distress syndrome. CERC-007 targets the cytokine IL-18 and is in clinical development for the treatment of Still’s disease (adult onset Still’s disease (AOSD) and systemic juvenile idiopathic arthritis (sJIA)), and multiple myeloma (MM). CERC-006, 801, 802 and 803 have all received Orphan Drug Designation and Rare Pediatric Disease Designation, which makes all four eligible for a priority review voucher upon FDA approval.

For more information about Cerecor, please visit www.cerecor.com.

Forward-Looking Statements

This press release may include forward-looking statements made pursuant to the Private Securities Litigation Reform Act of 1995. Forward-looking statements are statements that are not historical facts. Such forward-looking statements are subject to significant risks and uncertainties that are subject to change based on various factors (many of which are beyond Cerecor’s control), which could cause actual results to differ from the forward-looking statements. Such statements may include, without limitation, statements with respect to Cerecor’s plans, objectives, projections, expectations and intentions and other statements identified by words such as “projects,” “may,” “might,” “will,” “could,” “would,” “should,” “continue,” “seeks,” “aims,” “predicts,” “believes,” “expects,” “anticipates,” “estimates,” “intends,” “plans,” “potential,” or similar expressions (including their use in the negative), or by discussions of future matters such as: the development of product candidates or products; timing and success of trial results and regulatory review; potential attributes and benefits of product candidates; and other statements that are not historical. These statements are based upon the current beliefs and expectations of Cerecor’s management but are subject to significant risks and uncertainties, including: drug development costs, timing and other risks, including reliance on investigators and enrollment of patients in clinical trials, which might be slowed by the COVID-19 pandemic; regulatory risks; Cerecor's cash position and the need for it to raise additional capital; general economic and market risks and uncertainties, including those caused by the COVID-19 pandemic; and those other risks detailed in Cerecor’s filings with the Securities and Exchange Commission. Actual results may differ from those set forth in the forward-looking statements. Except as required by applicable law, Cerecor expressly disclaims any obligations or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in Cerecor’s expectations with respect thereto or any change in events, conditions or circumstances on which any statement is based.

For media and investor inquiries

Chris Brinzey
Westwicke, an ICR Company
chris.brinzey@westwicke.com
339-970-2843

or

James Harrell
Investor Relations
Chief Commercial Officer
Cerecor Inc.
jharrell@cerecor.com
623.439.2220 office

¹ Perlin DS, Zafir-Lavie I, Roadcap L, et al Levels of the TNF-Related Cytokine LIGHT Increase in Hospitalized COVID-19 Patients with Cytokine Release Syndrome and ARDS. mSphere. 2020 Aug 12;5(4):e00699-20.

FAQ

What were the results of the Phase 2 trial for CERC-002?

The Phase 2 trial for CERC-002 showed statistically significant efficacy in reducing respiratory failure and mortality at Day 28 (p=0.044) in COVID-19 patients with ARDS.

How did CERC-002 perform in older patients?

CERC-002 demonstrated the highest efficacy in patients over 60 years old, with a significant p-value of 0.042.

What is the mortality trend observed in the trial?

An approximately 50% trend in mortality reduction was observed at both Day 28 and Day 60, dropping from 22.5% to 10.8%.

What are the next steps for Cerecor regarding CERC-002?

Cerecor has applied for FDA Breakthrough Therapy and Fast Track Designations and plans to discuss the potential path for Emergency Use Authorization.