Ocugen, Inc. Announces Signing of Binding Term Sheet for the License of OCU400 Modifier Gene Therapy for Retinitis Pigmentosa in Korea

Rhea-AI Summary

Ocugen (NASDAQ: OCGN) has signed a binding term sheet for licensing OCU400, its modifier gene therapy for retinitis pigmentosa (RP), to a leading Korean pharmaceutical company. The agreement includes upfront fees and near-term development milestone payments of up to $11 million, plus sales milestones of $1 million for every $15 million in net sales. Ocugen will receive a 25% royalty on net sales and will manufacture the commercial supply. The deal targets approximately 15,000 RP patients in Korea. OCU400 is currently in Phase 3 clinical development with a planned Biologics License Application filing by mid-2026. This regional licensing strategy aligns with Ocugen's business development approach to partner with established companies while preserving rights in larger markets.

Positive

• Significant financial terms with up to $11 million in upfront and near-term milestone payments
• Attractive 25% royalty rate on net sales in Korean market
• Additional revenue stream through manufacturing and supply agreement
• Strategic entry into Korean market while preserving rights for larger markets
• Clear market opportunity with 15,000 RP patients in Korea

Negative

• Limited to Korean market only
• Phase 3 trials still ongoing with BLA filing not expected until mid-2026
• Final agreement yet to be executed within 60-day timeframe

Insights

Biotechnology Licensing Strategist

Ocugen secures valuable Korean licensing deal for OCU400 gene therapy with strong financial terms and strategic regional approach.

Ocugen has signed a binding term sheet for a potentially lucrative licensing agreement in Korea for OCU400, their modifier gene therapy for retinitis pigmentosa. The financial structure is notably robust for a regional deal, featuring upfront fees and near-term milestones totaling $11 million, substantial sales milestones of $1 million for every $15 million in net sales (potentially exceeding $150 million over 10 years), and an impressive 25% royalty rate on net sales.

This deal structure demonstrates significant confidence in OCU400's commercial potential. The 25% royalty rate is particularly noteworthy, as pharmaceutical licensing deals typically range from 10-20% for late-stage assets. Ocugen maintaining manufacturing rights further enhances their value capture from this agreement.

The Korean market, with approximately 15,000 RP patients, represents a strategic target that balances meaningful revenue potential with focused market access requirements. This regional approach allows Ocugen to monetize smaller markets while preserving rights to larger territories, creating a stepping-stone commercialization strategy that generates near-term capital while maintaining long-term upside.

With OCU400 advancing through Phase 3 trials and targeting BLA filing by mid-2026, this agreement provides validation from an established pharmaceutical player and strengthens Ocugen's financial position during the critical late-stage development period. The binding term sheet indicates high confidence in finalizing the full agreement within 60 days, suggesting alignment on key terms and mutual commitment to the partnership.

Ophthalmic Gene Therapy Specialist

This licensing agreement for OCU400 represents a significant development in addressing retinitis pigmentosa, a debilitating inherited retinal disease with limited treatment options. OCU400 employs a modifier gene therapy approach targeting nuclear hormone receptors (NHRs), which function as master regulators of multiple functions within the retina. This mechanism potentially addresses multiple genetic mutations through a single therapy—a key advantage over single-gene replacement approaches.

The Korean market, with an estimated 15,000 RP patients, offers a well-defined patient population for targeted commercialization. The high royalty rate (25%) suggests the partner recognizes OCU400's differentiated therapeutic approach and potential efficacy.

The advancement to Phase 3 clinical development indicates promising earlier clinical results, though specific efficacy data isn't mentioned in this release. The projected BLA filing timeline of mid-2026 aligns with typical late-stage ophthalmology development programs and suggests confidence in the ongoing clinical pathway.

For patients with RP—who typically experience progressive vision loss leading to blindness—OCU400 could potentially address the underlying disease mechanism rather than merely managing symptoms. The modifier gene approach could be particularly valuable given the heterogeneous genetic nature of RP (caused by mutations in over 100 different genes), potentially offering a broader solution than single-gene therapies that can only address specific mutations.

• Upfront fees and near-term development milestone payments totaling up to $11 million
• Sales milestones of $150 million or more in first 10 years of commercialization
• Royalties equaling 25% of net sales
• Ocugen to manufacture and supply OCU400

MALVERN, Pa., June 05, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced the signing of 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—Ocugen’s novel modifier gene therapy for retinitis pigmentosa (RP).

Pursuant to the term sheet, under the license agreement Ocugen will receive upfront license fees and near-term development milestones equaling up to $11 million. The Company will be entitled to sales milestones of $1 million for every $15 million of net sales in Korea in addition to a royalty of 25% on net sales of OCU400 generated by Ocugen’s partner. Additionally, Ocugen will manufacture commercial supply of OCU400 under terms of a supply agreement.

There are an estimated 15,000 individuals in the Republic of Korea with RP. OCU400 provides the opportunity for our partner to help thousands of patients and become a leader in gene therapy in Korea.

“This regional licensing agreement is aligned with our business development strategy to partner with well-established companies in their respective countries and regions—leveraging their networks and know-how to treat as many RP patients as possible,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “A regional approach preserves Ocugen’s rights to larger geographies to maximize total patient reach while also generating return for our shareholders.”

Additional details will be available once the definitive agreement between the parties is executed, which is expected to occur within the next 60 days.

Ocugen is currently advancing OCU400 through Phase 3 clinical development with a target Biologics License Application filing of mid-2026.

About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene therapies to address major blindness diseases and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to address significant unmet medical need for large patient populations through our gene-agnostic approach. Discover more at www.ocugen.com and follow us on X and LinkedIn.

Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding the terms of the definitive license and timing of a definitive agreement or if a definitive agreement will be executed at all or the anticipated benefits to Ocugen of the definitive license agreement, qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that a definitive agreement for the license will be delayed or not executed at all, or that, if executed, it will not be on terms described above, the risk that contemplated license agreement, if executed, will not lead to the current anticipated benefits to Ocugen, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; the ability of OCU400 to perform in humans in a manner consistent with nonclinical or preclinical study data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.

Contact:
Tiffany Hamilton
AVP, Head of Communications
Tiffany.Hamilton@ocugen.com

FAQ

What are the financial terms of OCGN's OCU400 licensing deal in Korea?

The deal includes up to $11 million in upfront fees and near-term milestones, $1 million sales milestones for every $15 million in net sales, and 25% royalties on net sales. Ocugen will also manufacture the commercial supply.

How many patients could benefit from OCU400 in Korea?

There are approximately 15,000 individuals with retinitis pigmentosa (RP) in the Republic of Korea who could potentially benefit from OCU400.

When does Ocugen expect to file for OCU400 approval?

Ocugen is targeting a Biologics License Application (BLA) filing by mid-2026, as the therapy is currently in Phase 3 clinical development.

What is OCU400 designed to treat?

OCU400 is a novel modifier gene therapy designed to treat retinitis pigmentosa (RP), a genetic eye disease that causes vision loss.

Why did OCGN choose a regional licensing strategy for OCU400?

The regional approach allows Ocugen to partner with established local companies while preserving rights in larger markets, maximizing patient reach and shareholder returns.