Ocugen Q3 Earnings Call Highlights

Key Points

• Ocugen completed enrollment of 140 patients in the Phase III OCU400 trial for retinitis pigmentosa, with top-line results expected in Q1 2027. The company is targeting three BLA submissions by 2028.
• Its other late-stage programs are advancing: OCU410ST data are expected in Q2 2027, and the Phase III OCU410 trial for geographic atrophy has begun, with enrollment targeted for completion in 2027.
• Ocugen reported $84.6 million in cash and short-term investments at quarter-end, which it expects to fund operations into 2028. It also secured an OCU400 licensing deal for the Middle East and North Africa and received provisional approval for a Bahamas access pathway.

Ocugen NASDAQ: OCGN said its third-quarter progress centered on advancing three retinal gene therapy programs in late-stage development, expanding regional partnerships and establishing a European presence to support future commercialization efforts.

Chairman, Chief Executive Officer and Co-Founder Shankar Musunuri said all three of the company’s modifier gene therapy programs are now in late-stage trials. The company continues to target three biologic license application, or BLA, submissions by 2028.

OCU400 Phase III Enrollment Completed

Ocugen completed enrollment of 140 patients in its Phase III liMeliGhT trial of OCU400 for retinitis pigmentosa, a group of inherited retinal disorders. Top-line data are expected in the first quarter of 2027, followed by a planned BLA submission, according to Musunuri.

The company also said it completed process performance qualification batches intended to support both its BLA filing and commercial launch supply for OCU400.

OCU400 is designed as a one-time modifier gene therapy that seeks to address multiple gene networks rather than a single mutation. Musunuri said the company’s platform is intended to be gene-agnostic and applicable across several retinal diseases.

During the call, Chief Medical Officer Mohamed Genead discussed supporting Phase I/II data. In a post-hoc analysis of seven subjects meeting the Phase III eligibility criteria, five treated eyes, or 71.4%, were projected to meet the Phase III responder threshold of at least a two-level improvement on the LDNA scale at week 52. Ocugen also cited three-year follow-up data in which seven of eight evaluable consented treated subjects showed improvement or preservation of low-luminance visual acuity compared with untreated eyes. The company reported no serious adverse events related to OCU400 through three years.

Genead said the Phase III study was designed with assumptions of a 50% responder rate in treated eyes and a 10% responder rate in controls. He added that the company’s power analysis estimated 93.2% power even under a hypothetical scenario of a 20% responder rate for treatment versus 1% for controls.

Regional Agreements and Bahamas Access Pathway

In October, Ocugen finalized an exclusive OCU400 licensing agreement with Roots Pharmaceutical covering the Middle East and North Africa. Under the agreement, Ocugen could receive up to $4 million in combined upfront and potential initial development milestone payments, up to $255 million in potential sales milestones, and a 22% royalty on net sales. Ocugen will manufacture commercial supply under the arrangement.

The deal is Ocugen’s second regional OCU400 license agreement, following an earlier agreement with Kwangdong Pharmaceutical in South Korea.

Separately, the company said OCU400 received provisional approval and priority designation in the Bahamas under the country’s Longevity and Regenerative Therapies Act, or LARTA. Ocugen has partnered with the Global Alliance for Regenerative Medicine to coordinate patient care and is preparing sites for potential full approval.

Executive Vice President of Commercial and Business Development Abhi Gupta said Ocugen expects full approval in the Bahamas by year-end, although the company did not provide patient forecasts. He said the program would be open globally to eligible patients and that pricing would align with the company’s targeted U.S. price. Gupta added that Ocugen itself would not market the program, with a local vendor responsible for patient and healthcare-community education.

Stargardt and Geographic Atrophy Programs Advance

Ocugen’s OCU410ST program for Stargardt disease remains in the pivotal Phase II/III GARDian3 trial, with top-line data anticipated in the second quarter of 2027. Stargardt disease is a pediatric-onset retinal disorder for which there are no approved therapies, according to the company.

Genead said an independent data monitoring committee reviewed a pre-specified interim analysis at eight months and recommended continuing the study without modification. The company remains blinded to the data. He said the interim analysis involved fewer than half the planned patients, occurred at an early point in a slowly progressing disease and showed baseline heterogeneity among participants.

The 63-patient study is designed to assess change in atrophic lesion size through month 12. Genead said Ocugen powered the trial at close to 90% to detect an approximately 25% difference between treated and control eyes. The company previously reported Phase I/II results showing a 54% reduction in lesion growth, though those data came from a smaller early-stage study.

Ocugen also initiated Phase III development of OCU410 for geographic atrophy, an advanced form of dry age-related macular degeneration. The first patient was dosed in the global Phase III ArMaDa3 trial during September. The 237-subject study will randomize participants two-to-one to a single subretinal OCU410 injection or untreated control across the United States, Canada and Europe.

The primary endpoint is geographic atrophy lesion growth over 12 months. Ocugen plans to complete enrollment in 2027 and target a BLA filing in 2028. Musunuri said the company does not plan an interim analysis for ArMaDa3.

Third-Quarter Financial Results

Ocugen reported cash equivalents, restricted cash and short-term investments of $84.6 million as of Sept. 30, 2026. Chief Financial Officer Rita Johnson-Greene said the company expects its cash balance to support operations into 2028 and fund two of its three late-stage programs through anticipated BLA filings.

• Third-quarter operating expenses were $19.7 million, including $12.5 million in research and development expense and $7.2 million in general and administrative expense.
• Third-quarter operating expenses compared with $19.4 million a year earlier, when research and development expense was $11.1 million and general and administrative expense was $8.2 million.
• Net loss per common share was $0.01 for the third quarter, compared with a $0.07 loss per share in the prior-year period.
• For the first nine months of 2026, operating expenses totaled $57 million, compared with $50.5 million in the first nine months of 2025.
• Nine-month net loss per common share was $0.14, compared with $0.17 a year earlier.

Ocugen had 339.3 million common shares outstanding as of Sept. 30. Musunuri said the company’s nearest major clinical catalyst remains OCU400 top-line data expected in the first quarter of 2027, followed by anticipated OCU410ST data in the second quarter.

About Ocugen (NASDAQ:OCGN)

Ocugen, Inc NASDAQ: OCGN is a biotechnology company focused on developing gene and cell therapies for blindness diseases and vaccines for infectious diseases. The company's programs are designed to address serious conditions with limited treatment options, including inherited retinal diseases and diseases affecting the central retina.

Ocugen's pipeline includes modifier gene therapy candidates intended to treat multiple retinal disorders with a single therapeutic approach. Its lead programs include OCU400 for retinitis pigmentosa, OCU410 for geographic atrophy associated with dry age-related macular degeneration, and OCU410ST for Stargardt disease.

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