Ocugen (NASDAQ:OCGN) Chief Executive Officer Shankar Musunuri outlined the company’s late-stage ophthalmology gene therapy pipeline, saying the company is targeting three biologics license application filings by 2028 across retinitis pigmentosa, Stargardt disease and geographic atrophy.
Speaking during a company session moderated by Annabelle, Musunuri said Ocugen’s strategy differs from traditional gene therapy approaches because its programs are designed to regulate broader retinal disease pathways rather than replace a single defective gene. He described the company’s therapies as “master regulator” gene therapies intended to reset homeostasis in retinal cells and create a healthier environment for photoreceptors and retinal pigment epithelial cells to survive.
Pipeline Focuses on Retinal Diseases With Large Patient Populations
- OCU400 for retinitis pigmentosa, an inherited retinal disease caused by defects in more than 100 genes.
- OCU410ST for Stargardt disease, a rare inherited retinal disease.
- OCU410 for geographic atrophy, the late stage of dry age-related macular degeneration.
Musunuri said geographic atrophy affects roughly 2 million to 3 million patients across the United States and European Union, while retinitis pigmentosa affects about 300,000 patients in those markets and Stargardt disease affects about 100,000. He said the company is pursuing “first-in-class gene therapies” that could potentially be one-time treatments.
For retinitis pigmentosa, Musunuri said Ocugen’s approach could address the broader disease population with a single product, in contrast to mutation-specific therapies that would require separate products for different genetic causes. He said OCU400 uses the modified gene NR2E3, while OCU410 and OCU410ST use RORA delivered through an AAV vector.
OCU400 Top-Line Data Expected in First Quarter
Musunuri said enrollment in Ocugen’s phase 3 trial of OCU400 for retinitis pigmentosa was completed this year. The study includes 140 patients randomized on a 2:1 basis, with about 95 patients in the treated group and the remainder in an untreated control group.
The company expects top-line results in the first quarter of next year. Musunuri said OCU400 has RMAT designation, making the program eligible for rolling submission. He said Ocugen plans to begin the rolling submission in the third quarter of this year by submitting nonclinical and chemistry, manufacturing and controls sections, with the clinical module to follow within weeks of top-line data.
“That will start the six-month accelerated clock for potential approval in late next year,” Musunuri said.
The primary assessment in the retinitis pigmentosa trial is a mobility test similar to one used for Luxturna, according to Musunuri. He said the test measures whether patients can perform better across lighting conditions, including low-light environments, compared with untreated controls.
Musunuri said the trial includes patients from early to advanced stages of disease, though patients must still have remaining retinal tissue and vision. He said the company’s goal is to obtain a broad retinitis pigmentosa indication regardless of genetic mutation.
Stargardt and Geographic Atrophy Programs Advance
For Stargardt disease, Musunuri said OCU410ST is in a phase 2/3 registration trial. The company anticipates an interim outcome analysis in the third quarter of this year to evaluate whether any sample size adjustment is needed. If the program remains on track, he said top-line results could be available in the second quarter, with a BLA filing after that.
Musunuri said both the retinitis pigmentosa and Stargardt programs have alignment with the European Medicines Agency, and that the single studies being conducted in the United States are expected to be sufficient to support market authorization applications in the European Union.
In Stargardt disease, Musunuri said the primary endpoint is lesion growth over one year compared with untreated controls. He said the company is aiming to show a slowing of lesion expansion, though longer-term observations from earlier studies have suggested some patients may experience broader visual improvements over time. He also noted that patients in the gene therapy studies are followed annually for five years for safety monitoring.
For geographic atrophy, Musunuri said Ocugen recently released “very promising” phase 2 data and is working with the FDA and EMA on a global phase 3 trial design. He said the proposed trial would include lesion growth as the primary endpoint and ellipsoid zone as a secondary endpoint tied to visual function. Ocugen expects regulatory alignment by the third quarter and is targeting potential BLA approval in 2028.
Company Says Cash Runway Extends Into 2028
Asked about the company’s financial position, Musunuri said Ocugen recently raised gross proceeds of $130 million through a convertible financing. He said the company’s cash runway now extends into 2028.
Musunuri said that funding is expected to support two BLA filings next year and the initiation of the geographic atrophy phase 3 trial. He also noted that the Stargardt program has rare pediatric disease designation and said a priority review voucher, if granted upon approval, could be worth $150 million to $200 million.
“We are in a very good cash position,” Musunuri said, adding that the company’s focus is on executing its three retinal gene therapy programs.
About Ocugen (NASDAQ:OCGN)
Ocugen Inc is a clinical-stage biopharmaceutical company focused on discovering, developing and commercializing gene therapies to treat rare inherited retinal diseases, as well as vaccines designed to address unmet needs in infectious diseases. Headquartered in Malvern, Pennsylvania, the company applies its proprietary gene therapy platform to create novel treatments aimed at preserving and restoring vision, while leveraging strategic partnerships to broaden its vaccine pipeline.
In its gene therapy portfolio, Ocugen is advancing multiple programs targeting retinal disorders.
