Ocugen, Inc. has announced that its Investigational New Drug (IND) application for OCU500, an innovative inhaled mucosal vaccine candidate for COVID-19, is now in effect following review by the U.S. Food and Drug Administration (FDA). This clearance paves the way for a Phase 1 clinical trial to evaluate the safety, tolerability, and immunogenicity of OCU500.
The Phase 1 trial will be sponsored and conducted by the National Institute of Allergy and Infectious Diseases (NIAID), a part of the National Institutes of Health. The study will assess OCU500 administered via two routes: inhalation into the lungs and intranasally as a spray. The trial is anticipated to start in the second quarter of 2025.
Novel Mucosal Vaccine Approach
OCU500 is based on a chimpanzee adenovirus-vectored (ChAd36) technology. This approach aims to stimulate mucosal immunity, attacking the virus at its entry points in the nose and mouth, potentially offering better and longer-lasting protection. Earlier studies using similar technology via inhalation showed increased mucosal and systemic antibodies and a durable immune response for up to one year, using one-fifth the dose compared to intramuscular administration.
"We are grateful for our ongoing collaboration with NIAID and pleased to bring our novel vaccine technology into the clinic through Project NextGen," said Mike Shine, Senior Vice President, Commercial at Ocugen. "COVID-19 remains a real public health concern, and an increasing number of studies are showing the benefit of mucosal vaccines that attack the virus where it enters the body—through the nose and mouth—to give better and longer protection. We look forward to this important next step in potentially providing a more durable and safer option to help prevent infection and transmission of COVID-19 regarding various variants of concern."
Trial Design and Objectives
The Phase 1 trial will enroll 80 adult subjects aged 18 to 64 years. Participants will be divided into low-dose and high-dose groups, with each group further split between inhalation and intranasal administration routes. The primary endpoint is to determine the safety of OCU500, while secondary and exploratory endpoints include assessing antibody production and the incidence of breakthrough COVID-19 infections.
Addressing the Ongoing Threat of COVID-19
Despite the end of the pandemic, COVID-19 continues to pose a significant public health burden. The Centers for Disease Control (CDC) estimates that between October 1, 2024, and January 11, 2025, there were 4.4 to 7.9 million COVID-19 infections in the U.S., leading to 120,000 to 210,000 hospitalizations and 14,000 to 25,000 deaths.
Future Development and Expansion
Ocugen intends to leverage this mucosal platform to develop vaccines for other respiratory threats, including seasonal influenza, bird flu, and respiratory syncytial virus (RSV). According to Dr. Arun Upadhyay, Chief Scientific Officer at Ocugen, the vector technology allows for the incorporation of single or multiple antigens into a single vector, enabling rapid development of respiratory vaccines in response to future outbreaks.
The original ChAd36 vector was licensed from Washington University in St. Louis. Dr. Michael Diamond, Professor of Medicine and Co-Director of the Center for Vaccines and Immunity to Microbial Pathogens at Washington University School of Medicine, stated, "We believe this vector is ideal for mucosal administration and can be designed to carry COVID-19 strains as well as influenza and antigens from other respiratory viruses."
Project NextGen Support
This project is supported by Project NextGen, a $5 billion multi-government agency initiative aimed at developing next-generation vaccines and therapeutics to combat COVID-19. NIAID, with funding from Project NextGen, will cover the full cost of the Phase 1 clinical trial. Ocugen will provide clinical trial materials and retain full right of reference to the findings, which the company believes will support the further development of OCU500.
