New clinical results from Ocugen could help the company reach an ambitious target of filing for three gene therapy approvals within the next few years.
Topline data from a phase 2 study showed that its treatment for the eye disease geographic atrophy reduced lesions by 31% in the optimal dose. The outcome marked an improvement over the “15% and 22% reductions reported for currently approved therapies,” Ocugen said.
With those results in hand, the retinal specialist is now aiming to usher the candidate, OCU410, into phase 3 trials, joining its two other late-stage contenders for rare eye conditions.
Gene therapies hold enormous therapeutic potential in the retinal space. The first gene therapy for an inherited disease to win an FDA nod, Spark Therapeutics’ Luxturna, was approved in 2017 for a rare form of blindness.
“Probably 100 years from now, they’ll say gene therapy was one of the greatest discoveries of the 21st century,” said Shankar Musunuri, CEO and co-founder of Ocugen.
But winning broad market traction for gene therapies has been another story. In particular, payers have grappled with the multimillion-dollar price tags they often carry. So far, just one gene therapy — Novartis’ Zolgensma — has crossed the blockbuster threshold.
The commercial challenges have made Wall Street skeptical, Musunuri said.
Now, Ocugen hopes to change that perspective by helping create a “paradigm shift” in the typical drug development and commercialization path for gene therapies — potentially drawing a roadmap for other drugmakers to follow.
A broad platform and speedy clinical trials
Part of Ocugen’s special sauce lies in its modifier gene therapy platform. Because of its gene agnostic capabilities, the platform could develop therapies for a range of inherited retinal diseases.
“It’s not gene specific like CRISPR or traditional gene therapy,” Musunuri told PharmaVoice in 2024. “We are embarking on something really big that nobody has done before.”
Because genes “don’t work in isolation,” Musunuri said, Ocugen is going beyond the approach of targeting one mutation. For example, its lead candidate OCU400 treats retinitis pigmentosa, a group of inherited diseases, by delivering a functional copy of a modifier gene. With the help of this “master regulator” the treatment can “reset the homeostasis, restore the function and also create a healthy environment, that’s important for long-term vision restoration,” he said.
Ultimately, OCU400 could treat more than 100 mutations associated with RP, Musunuri said. Ocugen is gunning to file for an approval for the phase 3 candidate this year.
Ocugen’s third late-stage therapy was developed for a genetic retinal disease called Stargardt. The candidate has won several designations from the FDA and could enter the market by 2028.
As it advances three leading candidates, Ocugen has a specific competitive advantage for an up-and-coming biotech — the speed at which retinal therapies reveal results.
“We can show impact in one year compared to a control group,” Musuruni said.
Phase 2 trials for drugs last up to two years on average and up to five in phase 3, according to the FDA.
Of course, it’s the final phase of development that could prove most challenging.
Global ambitions and a smaller price tag
Rather than focusing on just an FDA nod, Ocugen has been coordinating with regulators in other parts of the world, including Europe and Japan, to set the stage for scoring more approvals quickly. The goal is to run one trial that would generate the needed data for multiple countries.
The company is also setting the clinical stage for administering therapies by expanding training for retinal surgeons. Ocugen’s therapies are currently administered in about 20 facilities, Musunuri said, and the company has plans to also create a training center for surgeons.
“We are investing heavily to get the infrastructure right,” Musunuri said.
If the therapies are approved, the next sticking point could be pricing.
While Ocugen’s candidates target rare conditions, its therapy for geographic atrophy, a type of dry age-related macular degeneration, has the potential to treat 2 million to 3 million patients in the U.S. and EU, Musunuri said — a much larger population than other gene therapies that have hit the market. If approved, Ocugen will have to then navigate the challenging pricing dynamics that have dogged other gene therapies.
But Ocugen is approaching those decisions as an opportunity to demonstrate how gene therapies can be priced for a larger patient population.
“We don’t have to charge millions,” Musunuri said. “We can show the market that we are leading the way for large populations and show shareholders how we can have commercial success.”
Because vision loss diseases cost the U.S. economy an estimated $134 billion per year, Musunuri said, he believes the company has a good pitch to make about the value of its one-and-done treatments.
“We are not in it for a spike in revenue or a one-time flash in the pan,” he said. “Ultimately, these are durable therapies.”
