MALVERN, PA — Ocugen, Inc. (NASDAQ: OCGN) reported third-quarter 2025 financial results and provided an update on its advancing portfolio of late-stage gene therapies for inherited retinal diseases. The biotechnology company remains on track to file three Biologics License Applications (BLAs) within the next three years, positioning itself as a potential leader in modifier gene therapy.
The company’s lead programs, OCU400 for retinitis pigmentosa and OCU410ST for Stargardt disease, continue to progress toward regulatory milestones. Enrollment in the Phase 3 liMeliGhT trial for OCU400 is nearing completion, while the Phase 2/3 GARDian3 pivotal confirmatory trial for OCU410ST has reached 50% enrollment. Ocugen expects to file for U.S. and European approvals between 2026 and 2027.
“With two late-stage modifier gene therapies on track to meet 2026 and 2027 BLA/MAA filings, it’s remarkable to look back and recognize we only began dosing the first patient in the Phase 1/2 OCU400 clinical trial in 2022,” said Dr. Shankar Musunuri, chairman, CEO, and co-founder of Ocugen. “This progress not only reinforces our commitment to file three BLAs in the next three years, but it also brings us closer to addressing the incredible unmet medical needs that exist for patients facing vision loss.”
In September, Ocugen entered an exclusive licensing agreement with Kwangdong Pharmaceutical Co., Ltd. for commercialization rights to OCU400 in South Korea. The deal includes up to $7.5 million in upfront and milestone payments, plus royalties of 25% on net sales. Sales milestones will add $1.5 million for every $15 million generated, with projections of more than $180 million in revenue during the first decade of commercialization. Ocugen retains global manufacturing rights and plans additional regional partnerships.
The OCU410ST trial also achieved a key regulatory milestone in August when the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) accepted a single U.S.-based trial as sufficient for a Marketing Authorization Application (MAA). Interim results are expected by mid-2026, with full data anticipated in 2027.
Financially, Ocugen reported $32.9 million in cash, cash equivalents, and restricted cash as of September 30, 2025, compared to $58.8 million at year-end 2024. Total operating expenses rose to $19.4 million in the third quarter, up from $14.4 million a year earlier, reflecting higher research and development spending.
The company closed a $20 million registered direct offering in August, with potential for another $30 million in proceeds if accompanying warrants are exercised in full. Ocugen expects its current cash runway to extend through the second quarter of 2026.
“We will continue to pursue financing opportunities along with strategic business development to fund the Company into commercialization,” Musunuri said.
Upcoming milestones include full Phase 2 data from OCU410 for geographic atrophy in early 2026, interim data for OCU410ST mid-year, and top-line Phase 3 results for OCU400 by late 2026.
With advancing clinical progress and regulatory validation, Ocugen’s modifier gene therapy platform continues to move closer to redefining treatment options for patients affected by rare and blinding retinal diseases.
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