The year 2020 has had quite several challenges, and the biggest of them being the coronavirus pandemic, which has affected the entire world. However, there is always some party that stands to gain from any situation. This time it looks like the pharmaceutical industry is the one benefiting as players in the industry race to come up with a working cure or vaccine against COVID-19. Some of the developments are not even related to the coronavirus. Among the pharmaceutical companies that have been achieved significant growth in the pursuit of their developments is Ocugen.
Ocugen publishes nuclear hormone receptor gene NR2E3 preclinical data
The biopharma announced on March 3 this year that it published preclinical data from research involving its nuclear hormone receptor gene NR2E3. The latter is the company's pipeline therapeutic agent and gene modifier, which it hopes to use to target numerous degenerative diseases that affect the eyes. The preclinical data was published in a journal called Nature Gene Therapy. the company also noted that the FDA awarded OCU400 (NR2E3-AAV) orphan drug designation for treating inherited retinal diseases (IRDs). This includes retinal diseases caused by NR2E3 and CEP290 mutations.
The published data highlights the findings from five different mouse versions of retinitis pigmentosa (RP) that were treated using retinal injections of NR2E3-AAV. The study shows the efficiency of an innovative modifier gene therapy to seek out a wide range of therapeutic benefits in both the early and middle stages of PP.
Later publications on March 27 revealed that Ocugen will continue to advance its OGVHD research through clinical trials. The company had achieved 95% planned enrollment of its phase 3 study of OGVHD, for which it revealed that the results would be published before the end of 2020. However, the company later announced the discontinuation of the OGVHD phase 3 trial of OCU300. The company decided to terminate the clinical trial after an independent data monitoring committee evaluated an interim sample size. The committee concluded that there was very little chance that the clinical trial would meet its primary endpoints. Ocugen made it clear that it did not halt the study due to safety concerns. It also added that it would conduct a more elaborate review of the data.
Modifier Gene Therapy Platform’s potential
Despite discontinuing the Phase 3 trial of OCU300 as a potential oGVHD treatment, the company is still banking heavily on the benefits that the modifier gene therapy platform offers. It allows the company to use a single product to address multiple illnesses. OCU400 can potentially be used to effectively restore the retina's functions that have been compromised by inherited genetic illnesses. OCU400 features a nuclear hormone receptor (NHR) gene known as NR2E3, whose delivery method to the retina is an adeno-associated viral vector. NR2E3 expression in the retina may influence the restoration of homeostasis and even deliver other benefits. These may include reducing photoreceptor degeneration, cell stabilization, and preventing the loss of vision.
OCU400 has already demonstrated retina improvements where subjects Electroretinogram response indicates recovery and vision improvement in deem and well-lit conditions. Ocugen is also optimistic that having a single product that can address multiple diseases is an important advantage because it will allow the company to secure regulatory approval more easily. It will also facilitate faster recovery of development costs since it will have multiple therapeutic indications. The company plans to launch two phase 1 and 2 clinical trials in 2022.