How this biotech plans to go up against big pharma with its eye disease products

Shankar Musunuri has spent time in both the pharmaceutical world and the biotech arena – after 15 years at Pfizer (NYSE:PFE), he went on to launch several biotech start-ups. As co-founder, chairman & chief executive of Ocugen, he is looking to bring eye disease products to the patients that big pharma’s drugs have left out.

“There’s so much unmet need when you look into retinal diseases,” Musunuri told Drug Delivery Business News.

The Malvern, Penn.-based company has four compounds in its pipeline at various stages of development. It’s most advanced candidate, OCU300, is gearing up for Phase III trials in 2018.

The brimonidine nanoemulsion is a indicated for ocular graft versus host disease, a complication that occurs in as many as 60% of patients who have had a bone marrow transplant or allogenic hematological stem cell transplantation.

It’s second candidate, OCU310, combines brimonidine, a drug usually designed for patients with glaucoma, and a steroid.

Ocugen is hoping that its brimonidine-steroid combination can help treat the millions of people in the U.S. suffering from dry eye disease, bringing it directly into competition with companies like Shire (NSDQ:SHPG) and Allergan (NYSE:AGN).

There are just two products on the market for dry eye disease – Shire’s Xiidra and Allergan’s Restasis – and doctors say they wish there were better drugs available for a broader array of patients, according to Musunuri.

Ophthalmologists have told the CEO that they are specifically looking for drugs that work quickly and boast multiple mechanisms of action.

“There’s still a significant number of patients out there who really need therapeutics for dry eye disease,” he said.

With some of Ocugen’s products, like it’s OCU100 compound designed to delay the progression of retinitis pigmentosa, there is no other drug currently on the market – so there’s no immediate competition. Going up against a billion dollar product like Restasis is a different story. But Musunuri isn’t worried.

“They’re not helping the entire population,” he said.

As an example, he pointed to anti-VEGF therapies for macular degeneration, like Regeneron‘s (NSDQ:REGN) Eylea or Roche‘s (OTC:RHHBY) Lucentis. These products, according to Musunuri, are only effective in less than half of the two million people with AMD.

Before Eylea or Lucentis came to the market, there was nothing to help these patients. “Hat’s off to them – however, they’re only effective in about 30% of the population,” Musunuri said. “The majority of people are looking for new therapies. That’s where we come in.”

Ocugen is hoping to carve out a competitive space for themselves with a product that has a distinct mechanism of action compared to Eylea and Lucentis. It’s OCU200 product is made up of two proteins, tumstatin and transferrin, fused together and delivered via intra-vitreal injection.

Getting a drug into the eye presents a unique delivery problem. Most people would rather avoid having needles stuck into their eyes multiple times every year. While some companies are focused on creating implants or drug-eluting polymers to deliver drugs to the eye, Musunuri is taking a different approach.

“The goal is to get something to the market to help patients as soon as possible, because they don’t have anything today,” he said. So for now, the company’s strategy is to bring their injectable drugs to the market.

But as for the future of drug-delivery for eye disease products, Ocugen is betting on gene therapy. Earlier this month the company announced that it obtained an exclusive global license to develop and sell products based on technology from the Schepens Eye Research Institute of the Massachusetts Eye & Ear Infirmary.

The platform tech aims to modify disease progression by regulating gene networks that control retinal homeostasis, according to Ocugen, using therapeutic nuclear hormone receptors.

The goal is to boost compliance by giving patients the option to receive a single injection every few years, rather than multiple injections each year.

Looking ahead, Ocugen is preparing for a busy year: Musunuri is hoping to bring one of Ocugen’s biologics to the clinic and the Phase II dry eye disease study is slated to wrap up at the end of the first quarter. Also next year, the company wants to bring its oGVHT compound into Phase III trials.

As Ocugen builds out its pipeline for front- and back-of-the-eye products, Musunuri believes the relatively-young biotech can create a competitive space for itself among big pharma.

“We will distinguish ourselves based on how our product works,” he said.

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