MALVERN, PA — Ocugen Inc. (Nasdaq: OCGN) has signed a licensing agreement with Roots Pharmaceutical that could generate up to $259 million in upfront and milestone payments, plus royalties, giving the Malvern biotechnology company a commercial partner for its experimental blindness treatment across the Middle East and North Africa.
The agreement grants Roots Pharmaceutical exclusive regional rights to OCU400, Ocugen’s investigational gene therapy for retinitis pigmentosa, an inherited retinal disease that causes progressive vision loss and can lead to blindness.
Under the terms, Ocugen is eligible to receive an upfront licensing fee and initial development milestone payments totaling up to $4 million, along with as much as $255 million in additional payments tied to sales milestones.
Ocugen will also receive royalties equal to 22% of net OCU400 sales generated by Roots Pharmaceutical. The company will retain responsibility for manufacturing and supplying the treatment under a separate supply agreement.
The transaction establishes a regional commercialization arrangement for OCU400 while the therapy remains in late-stage clinical development. Its commercial potential depends on successful clinical results and regulatory approvals.
Ocugen is evaluating OCU400 in its Phase 3 liMeliGhT clinical trial, with topline results expected in the first quarter of 2027.
The company plans to pursue regulatory submissions following the trial, including a Biologics License Application with the U.S. Food and Drug Administration and a Marketing Authorization Application in Europe.
OCU400 is designed as a potential one-time gene therapy for retinitis pigmentosa, a condition for which Ocugen is seeking to address substantial unmet treatment needs.
The company estimates that retinitis pigmentosa is 10 to 20 times more prevalent in the Middle East and North Africa than in the United States and European Union, creating a potentially significant regional market if the therapy receives regulatory clearance.
“By partnering with Roots Pharmaceutical, an established leader with strong reach across the Middle East and North Africa, we are expanding our ability to bring our potential one-time gene therapy to patients in a region where retinitis pigmentosa is highly prevalent,” said Shankar Musunuri, Ocugen’s chairman, chief executive officer and co-founder.
Roots Pharmaceutical, working with strategic partner Al-Dhow International Holding, will provide regional commercialization capabilities and healthcare relationships intended to support access to the treatment.
Islam Zayed, chief executive officer and co-founder of Roots Pharmaceutical, said the companies will work to establish the diagnostic and treatment infrastructure required to deliver gene therapy across regional markets.
“We look forward to working closely with Ocugen to build the diagnosis-to-treatment pathway that gene therapy requires across our markets,” Zayed said.
The agreement also creates an opportunity for the companies to explore additional collaboration involving Ocugen’s ophthalmology pipeline, although the disclosed licensing terms apply to OCU400.
For Ocugen, the arrangement provides a potential source of development and commercial revenue while extending its international distribution strategy ahead of a pivotal clinical trial readout.
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