Setting Sights on the COVID Vaccine Market: Shankar Musunuri, CEO, Ocugen

Biotech company Ocugen got its start developing gene therapies for various forms of blindness diseases. Its breakthrough modifier gene therapy platform has the potential to address many rare retinal diseases with one product as well as address complex diseases that affect millions of people. Its novel biologic product candidate aims to offer better therapy to patients with underserved diseases such as wet age-related macular degeneration, diabetic macular edema, and diabetic retinopathy.

In late 2020, the company expanded to address the COVID-19 pandemic by partnering with Indian drug manufacturer, Bharat Biotech.

Dr. Shankar Musunuri is chairman, CEO, and co-founder of Ocugen. Prior to co-founding the company in 2013, Dr. Musunuri held leadership roles at numerous companies ranging from big pharma to novel start-up biotechs. He spent nearly 15 years at Pfizer/Wyeth, where he played a key role as Global Operations Team Leader for the most successful launch in vaccine history, Prevnar 13.

Here he talks to Pharm Exec about his 25 years’ experience advancing and commercializing a diverse portfolio of products — and about the company's latest mission to develop, manufacture, and commercialize Bharat Biotech’s COVID-19 vaccine, COVAXIN, in the North American market.

PharmExec: Tell us briefly about your work experience before Ocugen. What inspired you to work in biotech, and why did you co-found Ocugen?

Shankar Musunuri: Healthcare has always been in my DNA and it’s a sector that continues to show us how innovation can change peoples’ lives. I wanted to be a part of something like that and moving from big pharma to biotech made sense. In biotech, we make every decision matter because our resources are limited even though our creativity is boundless. That makes it a privilege to work with such a talented and passionate group every day.

I founded Ocugen because I was inspired by what my friend, Uday Kompella, shared with me so many years ago. That so many people with blindness diseases need hope and help, and I was driven to create solutions where none existed before.

Imagine discussing your visual impairment — or pending loss of vision — with your doctor and hearing, “There’s nothing we can do.” Knowing that the way you go about daily activities will change dramatically, wondering how vision loss may affect your career, and thinking about how it will impact every other facet of your life leaves many people feeling an overwhelming sense of loss and despair.We want to change that. To offer hope. Not just for vision-related conditions, but for many diseases that seem hopeless — much like how we all felt before there were COVID vaccines on the market.

Ocugen got its start in gene therapies for various forms of visual impairment and has branched out recently into the COVID-19 vaccine space. Can you tell me about that shift?

We’re determined to innovate where we are needed and where we can do the greatest good. Our leadership team’s deep history in vaccine development meant that we could not sit by the wayside while this public health crisis unfolded. We were perfectly positioned to jump in and help meet an important need. The pandemic made it incumbent upon all of us to find solutions that have the potential to save lives and restore normalcy to our day-to-day activities.

Like many, COVID personally impacted those closest to me when my elderly mother, who’s almost 80 years old, contracted the disease. Thankfully she recovered, and I was so grateful that she received at least one dose of vaccine prior to contracting the disease, knowing that may have helped her and positively impacted her outcome.

Additionally, the Delta variant has run rampant through India; most infections there are the Delta variant. I’ve been there, personally, and saw the effect COVID has had on that country, as well as on the United States. You can’t see something like that and not step in to help in some way if you have the means, experience, network, and knowledge to do so. It is like a war out there, and we are well positioned to jump in to do our part.

Ocugen is taking on a bold approach to innovation. We’ve put together a courageous team that seeks to apply their passions and expertise wherever there’s a need, from gene therapy to addressing public health crises. We’re not afraid to forge new paths with a sense of urgency, to deliver new options to people facing disease.

Tell me more about COVAXIN, your partnership with Bharat Biotech, and how the vaccine works. It’s not an mRNA vaccine, correct?

COVAXIN is a whole-virus inactivated vaccine that creates a broad-spectrum immune response, differentiating it from other mRNA and adenovirus-based vaccines that only target the spike protein. COVAXIN is designed to build immunity against more than just the spike protein, which is what mutates in Delta and other emerging variants. In clinical studies, COVAXIN elicited strong IgG responses against the spike protein, receptor-binding domain, and the nucleocapsid protein of SARS-CoV-2. It’s also the first vaccine to date with in vivo clinical data demonstrating efficacy against the Delta variant, while most mRNA vaccine players only have in vitro data.

In addition, COVAXIN uses a novel adjuvant developed by a U.S. company in collaboration with the National Institute of Allergy and Infectious Diseases. In clinical studies, COVAXIN showed strong cellular responses due to this adjuvant. Cellular responses for a vaccine are important for long-term memory and durability of vaccines.

COVAXIN was built on the same decades-old technology platform used to produce the Polio, Influenza, Hepatitis A, and Rabies vaccines, which may offer peace of mind to those hesitant to take new mRNA vaccines. It’s our hope that we can help overcome some of the vaccine hesitancy with this tried-and-true technology. Broad, protective vaccines such as COVAXIN have the ability to minimize or eliminate viral escape and can be a much-needed tool to help control the pandemic.

COVAXIN isalready approved under emergency use authorizations in 16 countries, with more than 75 million doses administered globally. Through our collaboration with Bharat Biotech, we are responsible for the clinical development, registration, manufacturing, distribution, and commercialization of COVAXIN for the U.S. and Canadian markets. We’ve filed a new drug submission to Canada, and it’s currently under active review for potential approval. We’re also currently in discussion with the U.S. FDA to determine the best regulatory path forward to bring this vaccine to the U.S. market. We’re also eagerly anticipating an answer from the World Health Organization on the status of Bharat’s emergency use listing application.

What other therapeutic areas are Ocugen focusing on?

Ocugen is developing amodifier gene therapy to potentially produce one product that can treat many diseases. Where traditional approaches to gene therapy target one individual gene mutation (and therefore, one disease) at a time, our novel approach targets nuclear hormone genes, which regulate multiple functions within the retina.

Currently, we’re developing the following gene therapies:

  • OCU400 for the treatment of both retinitis pigmentosa and Leber congenital amaurosis, which together consists of about 175 mutations
  • OCU410 for the treatment of dry age-related macular degeneration, which affections nine to 10 million patients in the U.S. alone and is a significant unmet medical need

We also have a novel biologic, called OCU200, for the treatment of major retinal diseases such as diabetic macular edema, diabetic retinopathy, and wet age-related macular degeneration. Combined, the conditions OCU200 is aimed at treating have an estimated global market size of more than $10 billion. We are planning to initiate a Phase 1/2 trial of OCU200 in 2022.

In all, we plan to initiate four Phase 1/2 trials over the next 18 months.

What’s the biggest challenge for Ocugen in the next 1–3 years?

When it comes to vaccines, one of our immediate challenges is tohelp re-establish public trust in medicine and science. It’s our hope that providing people with options like COVAXIN, which has been developed with familiar technology and offers efficacy against variants like Delta, will help improve vaccination rates.Additionally, now that we know boosters will be needed, we think the strong adverse event profile demonstrated in clinical trials, along with its broad protection against variants, will make COVAXIN an appealing option.

On the ophthalmology front, as we prepare to begin our gene therapy clinical trials in the U.S. and continue to expand into new therapeutic areas and face new challenges, we aim to remain true to the spirit and the passion of why we began. We will continue to go where there is need and to drive science to new heights to help our patients.

What has working in biotech throughout the course of the pandemic taught you?

Certainly, the pandemic has taught us all a great deal about ourselves, and about how we react individually and collectively in the face of crisis. One thing I have learned is that it’s important for companies like ours to act with urgency and resolve to effect change.

We must recognize that our communities want us to leverage science for the betterment of people. We remind ourselves that nothing is impossible with science; it’s just a matter of figuring things out. That also means we have to push ourselves to keep the quality up while being agile and working at speed. We’ve all seen how the pandemic has shortened development timescales. We’re constantly applying that thinking and mindset.

And finally, I’ve learned to make sure that everyone in our organization knows that they count. We are explorers and risk takers with a lot of passion and commitment. Everyone hears from me and my leadership our appreciation and a sincere “Thank you.”