Dompé’s ‘Oxervate’ U.S. sales surpass $1.7 billion; RGN-259 seeks differentiation with room-temperature storage and 4-week dosing. SEER-2 top-line results expected mid-November; success would signal entry into the monopolized orphan ocular disease market.
HLB Therapeutics (115450) is positioning its neurotrophic keratitis (NK) candidate, "RGN-259," to challenge the U.S. market currently dominated by Italian firm Dompé’s "Oxervate." The company is bracing for the mid-November announcement of top-line results from its U.S. Phase 3 "SEER-2" clinical trial.
The key competitive advantages of RGN-259 are its room-temperature storage capability and a shorter treatment regimen. Compared to existing therapies, the drug is easier to store and distribute, and its simplified administration is expected to significantly improve patient compliance.
According to Dompé Holdings' 2025 annual financial report, Oxervate’s U.S. revenue last year exceeded $1.724 billion, excluding discounts and rebates. This marks an approximately 53% increase from the $1.127 billion recorded in 2024, equivalent to roughly 2.3 trillion KRW when adjusted for exchange rates.
Oxervate is currently the only FDA-approved prescription eye drop for the treatment of NK. Dompé’s report indicates that distribution discounts and government rebates totaled approximately $38 million and $60 million, respectively—roughly 5.7% of the stated U.S. revenue. Deducting these items, the net revenue remains at approximately $1.626 billion, or about 94% of gross sales.
This resilience in pricing within a rare disease market—where no direct approved competitors currently exist—highlights the commercial potential that RGN-259 is targeting. The growth trend in these sales serves as a key benchmark for estimating the future commercial value of RGN-259.
[Source: Oxervate’s 2025 U.S. Revenue; Dompé Holdings 2025 Annual Financial Report]
Frozen/Refrigerated Oxervate vs. Room-Temperature RGN-259
Neurotrophic keratitis is a rare and intractable ocular disease characterized by impaired corneal sensation—often due to trigeminal nerve damage—and reduced corneal epithelial regenerative capacity. Because the condition blunts pain and sensation even when the cornea is damaged, patients often miss critical windows for treatment. Left untreated, the disease can progress to corneal epithelial defects, ulcers, perforations, and eventually vision loss or blindness.
Approved by the FDA in 2018, Oxervate uses cenegermin-bkbj (a recombinant human nerve growth factor, or rhNGF) as its active ingredient. Patients must administer the drops six times a day for eight weeks. The storage and usage procedures are notably complex: pharmacies must keep the product frozen, and patients must store unopened vials in a refrigerator. The administration process also requires a specialized adapter and pipette.
In contrast, RGN-259 is based on thymosin beta-4. HLB Therapeutics emphasizes its potential for a 4-week treatment course and the use of single-use, room-temperature stable vials, making it as user-friendly as common artificial tears.
If RGN-259 demonstrates efficacy and safety in its Phase 3 trials and secures FDA approval, analysts suggest it could secure a strong competitive edge over Oxervate by addressing critical pain points in distribution logistics and patient adherence.
The November SEER-2 Milestone
The critical gateway to commercialization for RGN-259 is the global SEER-2 Phase 3 trial. Conducted by HLB Therapeutics’ U.S. subsidiary, ReGenTree, the study involved 50 medical institutions, including 41 hospitals in the U.S. and 9 across three European countries.
Patient dosing is now complete, and the contract research organization (CRO) is currently validating the data. On August 26, the company announced that the CRO had slated the delivery of top-line data for mid-November. The company plans to make an official announcement immediately upon receiving the results.
In the previous European Phase 3 trial, SEER-3, the drug failed to reach statistical significance in recovery rates compared to the placebo group after four weeks of treatment. The company attributed this to a higher-than-expected recovery rate in the placebo group, and has since conducted a rigorous re-examination of individual patient data and consulted with external experts to analyze the causes.
HLB Therapeutics asserts that it has refined its clinical operations and evaluation processes to prevent a recurrence of the SEER-3 outcome in the SEER-2 trial. However, whether these improvements translate into proven efficacy remains to be seen in the upcoming top-line report.
Potential for Off-Label Use in Dry Eye Syndrome
Market interest also extends to the potential expansion of RGN-259 beyond NK, particularly into the dry eye disease market.
Market research firm Grand View Research projects the global dry eye treatment market to grow from $6.7 billion in 2025 to $7.1 billion in 2026, eventually reaching $11.1 billion by 2033 with an annual growth rate of 6.6%. This equates to an expansion from approximately 9.6 trillion KRW to 15 trillion KRW by 2033.
In the U.S., once a drug is FDA-approved, clinicians have the flexibility to prescribe it "off-label" for other conditions based on medical evidence and patient status. Consequently, if RGN-259 wins FDA approval for NK, it could theoretically be utilized for dry eye patients based on physician discretion.
However, off-label expansion is not automatic. It would require supporting evidence, insurance coverage, established prescription patterns, and incorporation into clinical treatment guidelines.
RGN-259 has a history of clinical development for dry eye syndrome. While the developers noted potential improvements in signs and symptoms for certain patient subgroups in past studies, it did not meet the pre-specified primary endpoints. Any future application for dry eye syndrome will depend on additional clinical evidence and clinical judgment in practice.
Eyes on Potential Licensing Deals
For now, all eyes are on the November SEER-2 results. Demonstrating statistically significant efficacy over the placebo would allow HLB Therapeutics to solidify its FDA regulatory strategy while actively weighing its entry into the U.S. NK market.
The company previously disclosed that it had signed non-disclosure agreements (NDAs) for licensing discussions with several global pharmaceutical firms during its European Phase 3 trial, and that it is awaiting the results to resume these talks. The actual terms of any potential licensing deal will depend on the SEER-2 findings and subsequent negotiations.
If RGN-259 provides positive efficacy and safety data in November, it will provide HLB Therapeutics with a bridge into a multi-trillion won market currently monopolized by Oxervate. Conversely, a failure to prove efficacy would necessitate a comprehensive overhaul of its clinical, regulatory, and commercialization strategies.
