Ocugen Inc. (NASDAQ:OCGN) has announced that it has decided to stop the third phase OCU300 trials for ocular Graft versus Host Disease (oGVHD).
Ocugen discontinues OCU300 trials for failing to meet co-primary endpoints
The company decided to discontinue the third phase study based on data from analysis of pre-planned initial sample size that was carried by an independent Data Monitoring Committee. The data demonstrated that the OCU300 trial could not meet expected co-primary endpoints once completed. However, the study was not halted based on safety concerns, and full data will be analyzed once available.
Shankar Musunuri, the Ocugen CEO, Chairman, and Co-Founder, indicated that they were disappointed to discontinue the study, and this is and news, especially of people suffering from oGVHD. The CEO acknowledged the efforts of investigators, patients, and their families that took part in the clinical trial. Musunuri added that the company is dedicated to advancing its transformative therapies. For now, they will redirect resources to their novel modifier gene therapy platform as well as biologic programs for people who have blindness.
Ocugen’s modifier gene therapy platform aims at generating therapies that can treat various retinal diseases such as inherited retinal diseases. The company has based the gene therapy platform on nuclear hormone receptors that can restore homeostasis. These are the main biological retina processes, and as a result, it is capable of rescuing photoreceptors from degenerating.
Ocugen in a better financial position after discontinuing OCU300 study
The CEO confirmed that the company is on track to enter clinical trials in 2021 for its first gene therapy product OCU400, that targets hereditary retinal diseases. Equally, Ocugen’s other pipeline products, which include OCU200 and OCU400, focusing on major retinal diseases, are expected to enter clinics by 2022.
Most importantly, following the discontinuation of the third phase OCU300 study and with proceeds from the ATM program, the company is well-positioned financially. The CEO confirmed that they believe they have adequate cash for funding current operations into Q4 of 2020.