Thank you, Kelly. Good morning, everyone. And thank you for joining us this morning.
As this is our first conference call as a public company, I would like to take this opportunity to share with you more about our mission, our pipeline programs and the opportunity to create needed new therapies for the treatment of debilitating eye diseases.
I'll review our pipeline and the progress we have made since our reverse merger in September. Sanjay will then recap our recently completed reverse merger transaction and discuss our current quarter financial results before the Q&A. The entire management team and our advisors have been strongly focused on creating long term value for our shareholders.
Our company was founded upon a mission to develop innovative therapies to address rare and underserved eye diseases.
Our most advanced program is OCU300 for the treatment of ocular graft versus host disease or oGVHD for short. oGVHD is a severe chronic autoimmune disease that occurs in up to 60% of allogeneic bone marrow transplantations and represents the critical unmet medical need.
By 2020, it is expected that there will be 53,000 patients suffering from oGVHD. Of which, there is no approved FDA treatment. These cancer patients experienced severe and debilitating pain and discomfort, which significantly diminishes their quality of life and restricts daily activities leading to disability in many cases. One patient described this disease like putting hot sauce directly into your eye and rolling your eyeball in the sand, imaging the pain that must feel like for patients who have already undergone so much in their cancer treatment.
For some patients, this disease can lead to vision loss and irreparable damage to the ocular surface.
Our product, OCU300, is brimonidine 0.18% developed in our proprietary OcuNanoE nanoemulsion formulation.
Our OcuNanoE formulation was designed to deliver drugs more efficiently to relevant ocular tissues, provide protection to the oculuar surface and potentially increase overall efficacy compared to conventional eye drops.
We are the first and only company to have received orphan drug designation from the FDA for oGVHD, and we are also the first company to conduct a Phase 3 clinical study in this patient population.
Our Phase 3 study is an 84 day randomized, double masked placebo controlled study focusing on the treatment of ocular discomfort and ocular redness in patients with oGVHD. This study has co-primary endpoints of symptom and sign.
Our symptoms endpoint is ocular discomfort based on a visual analog scale. When a patient visits the clinic, he or she will rate on a scale of 1 to 10, the amount of pain they have felt over the past 24 hours and this is recorded on the visual analog scale. The signed endpoint is ocular redness measured on a validated by Bulbar Redness score.
As physician will measure the amount of redness in the patients' eye, which indicates the severity of the information the patient is experiencing.
We are enrolling 60 patients in a 2 to 1 randomization.
So for every two patients that receive OCU300, one patient receives the placebo. These patients are referred to specialty bone marrow transplant centers several of which are active in the study. We plan to conduct a statistical review when we reach 50% of planned enrollments to ensure that the sample size is accurate, and we are more than 40% endorsed at this point in time.
We expect to have top line data available in the second half of next year.
Now I would like to talk about our gene therapy program. We're very excited about our modified gene therapy programs, which have the potential to treat variety of inherited retinal diseases at a single gene therapy product [Technical Difficulty] genes or master gene, as we like to call them, are called nuclear hormone receptor genes that are believed to play a vital role in regulating many functions within the retina, and are able to restore normal function in the retina.
What this means is that with one modifier, our master gene, we may have the potential to treat multiple diseases as opposed to the current approach of treating one disease or genetic defects at one product. Multiple animal models have shown that the expression of these master genes within the retina can stabilize retinal cells, and restore normal functional retinal cell function, demonstrating their modifier function. This technology was developed at Schepens Eye Research Institute of Massachusetts Eye and Ear, and Harvard Medical School based on the pioneering research of Dr. Neena Haider.
Our first product candidate in our modified gene therapy platform is OCU400. OCU400 consists of a functional copy of the nuclear hormone receptor gene, NR2E3, delivered to target cells in the retina using an AAV vector. In a mouse model of six different retinal diseases, NR2E3 delivery to retinal cells reversed disease progression and restored retinal structures. OCU400 has already received two orphan drug designations from the FDA; one for treating patients with NR2E3 mutation-associated retinal diseases; and the second one, which was announced on September 18th, for treating patients with CEP290 mutation-associated retinal diseases. We plan to initiate a Phase 1/2a clinical study of OCU400 by 2021.
On September 30th, we announced our strategic partnership with CanSinoBIO on the development of this program. Under this strategic collaboration, CanSinoBIO will be responsible for all CMC developments and manufacturing of clinical supplies, including all associated costs. This partnership provides Ocugen greater flexibility within its capital resources. CanSinoBIO also maintains the option to support commercial manufacturing for us.
In this agreement, we have provided commercialization rights to CanSinoBIO in the Greater China territory, and will receive high single digit royalties on net sales. We recognize the importance of a reliable manufacturing partnership for gene therapy clinical trials and commercialization. We believe this partnership with CanSinoBIO with the state-of-the-art facilities and world-class team provides us with a clear path to advance our development and manufacturing processes to reach the clinic with significantly reduced capital and resources requirements.
The second program in our modifier gene therapy platform is OCU410, utilizing the nuclear hormone receptor genes RORA with an AAV vector delivery platform for the treatment of dry AMD. Dry AMD is a complex disease caused by multiple pathways, such as inflammation, oxidative stress and imbalance in lipid metabolism. Currently, there are no approved therapies to treat this disease, which affects about 9 million people in the U.S.
In the past, many clinical trials were not successful due to the inability to target multiple pathways associated with this disease. Experts believe that the products that can control multiple pathways will be able to control dry AMD. It is known that RORA gene, being a master gene, regulates multiple pathways associated with dry AMD pathogenesis. It has potential to increase survival of photoreceptors and the retinal pigment layer and by doing so, it could slow or reverse the disease progression of retinal degeneration associated with dry AMD. Currently, this program is in preclinical development.
Now I will move on to our biological program, OCU200. OCU200 is our retinal program focused on wet age related macular degeneration, or wet-AMD. Wet-AMD is a severe site threatening disease caused by the abnormal growth and infiltration of new leaky blood vessels in the retina, and is the leading cause of blindness in people over the age of 55 in the U.S. and Europe. It has been estimated that approximately 11 million patients in the U.S. have some form of AMD, and approximately 1.1 million of those patients suffer from wet-AMD.
OCU200, our program for wet-AMD is a fusion protein consists of two naturally occurring molecules, tumstatin and transferrin. We believe we are the first company to focus on a macro molecule target integrins for ocular diseases. This integral targeting based approach has been actually explored in a variety of other disease treatments, such as cancer, autoimmune disease, angiogenic and fibrotic treatments, and preclinical studies OCU200, demonstrated superior efficacy compared to anti-VEGF therapies in reducing choroid neovascularization lesion areas in laser-induced rats and mice CNV models.
We believe these results highlight the potential for OCU200 to deliver disease modification to patients with diabetic macular edema and diabetic retinopathy, which also have a significant impact to patients globally. Existing anti-VEGF treatments are suboptimally effective in a majority of these patients. We believe that OCU200 with its distinct mechanism for action can be effective in a majority of these patients. This program is in preclinical development and targeted to enter into the clinics in 2021.
As you can see, Ocugen has developed a diversified ophthalmology portfolio with programs in both clinical and preclinical development, which allow us to broaden the patients we serve, focusing on those with retinal diseases that can be treated with our gene therapy platform and biologics, and those with ocular surface diseases oGVHD. I am excited about the potential of our pipeline and the value proposition it may bring to shareholders in both the short and long-term. We believe we have clear development pacts for these programs, along with key value driving inflection points in the years ahead.
Now I'll turn the call over to Sanjay to provide an update regarding our merger with Histogenics, the recent warrant restructuring, and our quarterly financial results. Sanjay?