Ocugen Initiates Dosing in GARDian3 Trial, Signs $11mln Korea Deal.

• Ocugen initiated dosing in OCU410ST Phase 2/3 GARDian3 clinical trial • OCU400 Phase 3 liMeliGhT clinical trial is actively dosing patients • OrthoCellix reverse merger to focus capital on modifier gene therapy platform • Signed binding term sheet for exclusive Korean rights to OCU400 with $11 million upfront fees and milestone payments • Ocugen reported second quarter 2025 financial results today at 8:30 a.m. ET

Ocugen, Inc. (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, reported its second quarter 2025 financial results today at 8:30 a.m. ET. The company also provided updates on its clinical trials and business developments.

In the quarter, Ocugen initiated dosing in the OCU410ST Phase 2/3 GARDian3 clinical trial, a pivotal confirmatory trial for Stargardt disease. The trial follows the FDA’s agreement to proceed with the Phase 2/3 trial after granting Rare Pediatric Disease Designation (RPDD) to OCU410ST. This designation underscores the urgent need to address Stargardt disease, a significant unmet medical need affecting approximately 100,000 people in the U.S. and Europe combined and approximately 1 million globally.

The company is actively dosing patients in the OCU400 Phase 3 liMeliGhT clinical trial, which remains on track for BLA and MAA submissions in 2026. This is the only broad retinitis pigmentosa (RP) gene-agnostic trial to address multiple genetic mutations with a single therapeutic approach. The European Medicines Agency has granted eligibility to submit the OCU400 Marketing Authorization Application (MAA) through the centralized procedure.

Ocugen has also made significant strides in its business development strategy. The company signed a binding term sheet to negotiate and enter into a licensing agreement with a well-established leader in the pharmaceutical and healthcare sector in Korea for exclusive Korean rights to OCU400. The agreement includes upfront fees and near-term development milestone payments totaling up to $11 million, with additional sales milestones and a royalty of 25% on net sales of OCU400 generated by Ocugen’s partner. This regional approach preserves Ocugen’s rights to larger geographies while generating return for shareholders.

The company has also announced a proposed reverse merger with OrthoCellix, a wholly-owned subsidiary, and Carisma Therapeutics, Inc. to create a Nasdaq-listed, late clinical-stage regenerative cell therapy company with a first-in-class technology platform, focused on orthopedic diseases. The combined company will focus on the development of OrthoCellix’s NeoCart® technology for the treatment of articular knee cartilage defects.

Ocugen reported a $0.05 net loss per common share for the three months ended June 30, 2025, compared to a $0.06 net loss per common share for the same period in 2024. The company’s cash, cash equivalents, and restricted cash totaled $27.3 million as of June 30, 2025, providing cash runway into the first quarter of 2026. Total operating expenses for the three months ended June 30, 2025, were $15.2 million, including research and development expenses of $8.4 million and general and administrative expenses of $6.8 million.

The company’s executive leadership team will host a conference call and webcast today to discuss the financial results and recent business highlights. The call will be open to all listeners and a replay will be available on the Ocugen investor site for approximately 45 days following the event.

References:

[1] https://ir.ocugen.com/news-releases/news-release-details/ocugen-provides-business-update-second-quarter-2025-financial