Thank you, operator. I'd like to welcome you for our conference call this morning. With me today is our Chairman and CEO, Dr. Shankar Musunuri; our CFO, Sanjay Subramanian; and our Chief Medical Officer, Dr. Dan Jorgensen.
Earlier this morning, we issued a press release with our business update in 2019 financial results. We encourage listeners to review the press release, which is available on the Ocugen website at www.ocugen.com. This call is also being recorded and a replay will be available on the Investors section of Ocugen website for approximately 45 days.
Before we begin our formal comments, I'll remind you that various remarks we make today constitute forward-looking statements pursuant to the Safe Harbor provisions of the Private Securities Litigation Reform Act of 1995, including statements related to our financial outlook, our ability to raise capital on terms acceptable to us, our product development plans for our product candidates, including the results and timing of any future preclinical studies and clinical trials for such product candidates and our ability to close the NeoCart asset sale and the timeframe associated with such closing.
These forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from our expectations and forecasts, and can be identified by words such as expect, plan, will, may, anticipate, believe, estimate, upcoming, should, intend and other words of similar meaning. Any such forward-looking statements are not guarantees of future performance.
These risks are described in the Risk Factors section in the 10-K, which will be filed after the market close today. Any information we provide on this conference call is provided only as of the day of this call, March 27, 2020 and we undertake no obligation to update any forward-looking statements we may make on this call on account of new information, future events, or otherwise.
I will now turn the call over to our Chairman and CEO, Shankar Musunuri, Shankar?
Thank you, Kelly. Good morning, everyone. And thank you for joining us this morning.
We are living in unprecedented times.
As most people, we here at Ocugen are participating, practicing social distancing and our team is working remotely. This means that for this call, we are not all in one location.
So please bear with us when we get to Q&A section.
As we coordinate, we will response.
Ocugen’s suite, product portfolio approaches ensure portfolio diversification and reduced risk with a laser-focus on eye diseases. Today, we will provide an update on the progress of our OCU300 Phase 3 clinical trial for patients with ocular graft versus host disease, as well as our breakthrough modifier gene therapy platform and the recent publication in Nature Gene Therapy. Sanjay will then share highlights of our 2019 full year financial results and our business development activities before the Q&A.
The entire management team and our advisors have been strongly focused on creating long-term value for our shareholders.
Our most advanced program is OCU300 for the treatment of ocular graft versus host disease or oGVHD for short.
As a reminder oGVHD is a severe chronic autoimmune disease that occurs in up to 60% of allergenic bone marrow transplant patients and represents a critical unmet medical need. Presently, there is no approved FDA treatment for oGVHD. We're 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 trial in this patient population.
Our Phase 3 trial continued to enroll well through mid-March. We shared in December that we had reached 50% of our planned enrollment, and I'm pleased to share that as of March 20th, we have completed over 95% of our planned enrollment. Currently we are not screening new patients due to COVID-19 pandemic.
Our clinical team is working closely with our sites to address the impact to our clinical trial, putting patient safety first above all.
We have arranged for each patient's monthly supply of study medication to be shipped directly from site-to-site to the patient, rather than risking an in-person visit to pick up the medication.
In addition, if a site is not willing or able to conduct in-person follow up visits, we are using virtual visits, which may include phone interviews to continue the safety checks built into the protocol and to collect endpoint data.
For those sites that are conducting in-person follow up visits, they have a process in place to minimize the risk to the patient.
Our protocol has built in windows around each assessment visit.
So there is some flexibility as to the exact date of the follow up visit, whether it is done remotely or in-person.
As of right now, because we are ahead of our schedule for enrollment, we still anticipate having top-line results from this study before the end of the year. The situation is rapidly changing, however, and we will continue to monitor its impact on our clinical trials.
Now, I would like to talk about our gene therapy program.
We are very excited about our modifier gene therapy platform, which has the potential to treat a variety of inherited retinal diseases with a single gene therapy product. Modifier genes or master genes, as we like to call them, are 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 or a master gene, we may have the potential to treat multiple diseases as opposed to the current approach of treating one disease or a genetic defect with one product. The preclinical data for our first gene therapy product candidate OCU400 was published in Nature Gene Therapy earlier this month. OCU400 consists of a functional copy of nuclear hormone receptor gene NR2E3 delivered to target cells in the retina using an AAV vector. The publication details efficacy results in five unique mouse models of retinitis pigmentosa or RP that underwent administration of NR2E3 by sub-retinal injection.
The five RP models tested represented five different forms of RP: PDE6B associated RP; two forms of rhodopsin associated RP; Leber Congenital Amaurosis; and Enhanced S-cone Syndrome. The study demonstrated the potency of the novel modifier gene therapy to elicit broad-spectrum therapeutic benefits in early and intermediate stages of RP.
One of the biggest advantages of our modifier gene therapy platform is that it has the potential to eliminate the need for individual gene replacement and gene editing strategies and may revolutionarize gene therapy treatment for eye diseases. Inherited retinal degenerations such as RP affect over 1.5 million people worldwide or 150 gene mutations have been associated with RP and this number represents only 60% of the RP population. The remaining 40% of RP patients cannot be genetically diagnosed, making it difficult to develop individual treatment.
We believe our modifier gene therapy has the potential to eliminate the need for developing more than 150 individual products and provide one treatment option for all RP patients.
As a reminder, in September of 2019, we entered into a strategic partnership with CanSinoBIO for the development of OCU400. CanSinoBIO is responsible for all CMC development and manufacturing of clinical supplies including all associated costs. This partnership provides Ocugen greater flexibility with its capital resources. CanSinoBIO is located in China and we are pleased to share that the team there is back at work.
While do we don't anticipate any immediate impact to our overall timeline, we continue to evaluate the global pandemic and any potential impacts this may have in the future.
We continue to plan for the commencement of a Phase 1/2a clinical trial for OCU400 in patients in 2021.
Now, before I turn the call over to Sanjay, I just want to share how excited I’m about the potential of our pipeline and the value proposition it may bring to shareholders in both the short and long-term. By addressing rare and underserved ophthalmic diseases, we bring hope to patients with our adequate treatment options.
I will now turn the call over to Sanjay to provide an update on our 2019 financials and business development activities. Sanjay?