Pennsylvania biotech Ocugen has raised $7.5 million in a series B round as it fixes its sights on advancing a trio of eye disease treatments into clinical trials.
The new fundraising will help fund additional testing of Ocugen's lead candidate OCU300 for ocular graft-versus-host disease (GVHD)—which has no approved therapies in the U.S.—and help bring two preclinical candidates for retinitis pigmentosa (OCU100) and wet age-related macular degeneration (OCU200) into the clinic for the first time.
Turkish drugmaker Abdi Ibrahim and the Kazakhstan-based JSC Lancaster Group were the co-leaders of the round, along with existing backers Frank Leo, formerly with Cardinal Health, and Bipeptek founder John Zhang, who are both directors of the company.
Ocugen is biotech entrepreneur Shankar Musunuri's latest venture, having previously set up Nuron Biotech and Iogenetics. He set up the company with co-founder Uday Kompella, an ophthalmology and bioengineering specialist at the University of Colorado-Anschutz Medical Campus who invented OCU100 and OCU200, in 2013.
OCU300—a University of Illinois at Chicago (UIC) project—was licensed to flesh out the portfolio last year, and the new cash will be needed if Ocugen is to stick to its plan to start a phase 3 program in the next few months.
Ocular GVHD is a sight-threatening complication seen in around 40%-60% of patients who undergo donor (allogeneic) stem cell transplants as part of blood cancer treatment, and is marked by dry eye, infections and ocular scarring.
At the moment it is typically managed with artificial tears in mild cases and steroids—which can cause complications such as elevated pressure in the eye, cataracts and eye infections—in patients with more serious symptoms.
OCU300 is a repurposed drug that could take advantage of a shorter regulatory path to market if it fulfills its promise in additional trials, but for now it's not divulging the identity of the active ingredient, saying merely that it has "an established track record in ocular applications." The phase 1/2 candidate has been shown in preliminary testing to improve symptoms in 90% of ocular GVHD patients, according to the biotech.
It's not the only company pitching at the disease however. Roche's Genentech unit is also exploring therapies for ocular GVHD, including a recombinant form of DNAse I which is in phase I/II testing, while other groups are testing new formulations of a range of established drugs including the immunosuppressants cyclosporine and tacrolimus.
The cash injection will also help Ocugen advance OCU100—a recombinant form of lens epithelium-derived growth factor (LEDGF)—into its first trial for retinitis pigmentosa, an inherited eye disease that affects around 100,000 people in the U.S. and typically leads to blindness in middle age. The drug has orphan status from the FDA.
Meanwhile, OCU200 is an anti-angiogenic tumstatin fusion protein designed to block new blood vessel formation in wet AMD via a mechanism that differs from VEGF inhibitors such as Regeneron's Eylea and Roche's Lucentis, the mainstay of current drug treatment for the condition.
Ocugen has also been awarded a U.S. patent on a formulation of alpha agonist compounds with potential for the treatment of dry eye syndrome.
"We are very proud of the progress we have made with our pipeline and steadfast in our plans to advance these products toward commercialization," said Musunuri. "We are pleased to have closed this round of financing and are appreciative of the confidence shown by our new investors, all of which have proven track records of success in the pharmaceutical industry."