Ocugen introduced NeoCart, which is designed to repair full-thickness lesions of the knee cartilage in adults, as a Phase III cell therapy platform technology.
On Tuesday morning, Pennsylvania-based Ocugen announced it is diversifying its pipeline by introducing NeoCart as a Phase III cell therapy platform technology.
The technology is designed to repair full-thickness lesions of the knee cartilage in adults. This follows the U.S. Food and Drug Administration’s recent decision to grant a Regenerative Medicine Advanced Therapy (RMAT) designation for NeoCart.
The platform is a three-dimensional tissue-engineered disc of new cartilage. NeoCart is manufactured by growing chondrocytes, the cells responsible for maintaining cartilage health. The chondrocytes are derived from the patient and built on a unique scaffold. They indicate the technology platform can speed healing and decrease pain by rebuilding damaged knee cartilage.
“We’re excited that NeoCart has received this RMAT designation, an important regulatory milestone, especially as we view this product as an enabling technology in cell and regenerative therapy for orthopedic indications,” said Shankar Musunuri, Ph.D., chairman, chief executive officer and co-founder of Ocugen. “Our next step will be working with the FDA to construct the Phase III program to bring this innovation to this emerging treatment area.”
Musunuri added, “People living with articular cartilage lesions literally have holes in their knees that are extremely difficult to heal, and without proper treatment, they’re at high risk of getting osteoarthritis. We believe that NeoCart offers the potential for an innovative new option where treatments in this area are still limited and results are not optimal.”
RMAT is part of the 21st Century Cures Act, designed to expedite the development and review of regenerative medicine therapies. It is similar to having both a Breakthrough Therapy designation and Fast Track designation, including early interactions with the FDA.
Most recent coverage of Ocugen has revolved around its partnership with India’s Bharat Biotech on the COVID-19 vaccine, Covaxin. On Monday, Ocugen reported the FDA had lifted a second clinical hold on the company’s Phase II/III clinical trial of the vaccine. Ocugen had originally paused the study after the World Health Organization expressed concerns about an inspection of one of Bharat Biotech’s manufacturing facilities. The FDA followed along, but has now lifted the hold, allowing the trial to continue.
One of the probable advantages of the Covaxin vaccine is that it is a highly purified and inactivated vaccine of the entire virus. Unlike most COVID-19 vaccines, which use one type of technology or another to introduce the virus’s spike protein to the human immune system, Covaxin introduces the entire virus — albeit a dead one — to the human immune system.
Variants to SARS-CoV-2, such as Delta, Omicron and several Omicron subvariants, primarily involve mutations in the spike protein. In theory, Covaxin does not have problems with spike mutations because it is training the immune system to recognize the entire virus, not just the spike protein.
Ocugen has rights to market Covaxin in the U.S., Canada and Mexico. In April, Ocugen added Mexico to the deal. As of April 18, it was under review in Mexico. As of its first-quarter financial release on May 6, 2022, discussions with Health Canada were still ongoing.
Ocugen had submitted an emergency use request (EUA) to the FDA for the use of Covaxin in pediatric patients in late 2021, even though the agency had recommended it seek full approval rather than an EUA. In March, however, the FDA rejected the EUA request in patients ages 2 to 18. Shortly afterward, Ocugen shared real-world safety data from 36 million teenagers who have already received Covaxin globally. The company also provided the FDA with in vitro data suggesting the vaccine provides “robust neutralization” against the Delta and Omicron variants.
