Ocular Therapeutix

Company overview

• Integrated biopharmaceutical company focused on retina and other ophthalmic indications.
• Core platform: ELUTYX bioresorbable hydrogel technology for local, programmed-release drug delivery to the eye.
• Lead clinical asset: AXPAXLI (OTX-TKI), an axitinib-embedded intravitreal hydrogel for retinal diseases.
• Commercial product: DEXTENZA, a preservative-free dexamethasone intracanalicular insert for post-surgical ocular inflammation and pain and for ocular itching in allergic conjunctivitis in patients aged 2 and older.
• Additional program: OTX-TIC, a travoprost intracameral hydrogel for lowering intraocular pressure in open-angle glaucoma or ocular hypertension (Phase 2 completed).
• Global collaboration with AffaMed Therapeutics Limited for DEXTENZA and OTX-TIC in parts of Asia, including China, with upfront and milestone payments plus tiered royalties.

Technology and approach

• ELUTYX hydrogel platform
  • PEG-based, bioresorbable hydrogels with ester linkages that hydrolyze in the eye to release embedded drug over extended periods (months to a year or more).
  • Hydrogels are soft, lack a rigid shell, and fully bioresorb after drug release without leaving harmful byproducts.
  • Platform benefits include programmed local release, reduced treatment burden, fewer injections for some indications, and avoidance of preservative-related side effects.
• Delivery modalities enabled by ELUTYX
  • Intravitreal hydrogels (e.g., AXPAXLI) for sustained anti-angiogenic therapy.
  • Intracameral hydrogels (e.g., OTX-TIC) for sustained prostaglandin analog delivery to lower IOP.
  • Intracanalicular inserts (DEXTENZA) for surface-eye drug delivery without patient self-administration.
• Criteria for selecting active ingredients
  • Prior FDA approval for the indication, scientific rationale, patentability and freedom-to-operate, high potency, supplier availability, and compatibility with the hydrogel delivery system.

Products and lead programs

• AXPAXLI (axitinib intravitreal hydrogel)

  • Indications: wet age-related macular degeneration (wet AMD) and diabetic retinal diseases (NPDR, DME, PDR, RVO).
  • Phase 3 registrational program:
    • SOL program for wet AMD
      • SOL-1: repeat-dosing Phase 3 comparing AXPAXLI 450 µg to aflibercept 2 mg; Week 36 primary endpoint; Week 52 and Week 76 re-dosing; Week 104 safety follow-up; SPA with FDA in place.
      • SOL-R: repeat-dosing Phase 3 non-inferiority trial vs aflibercept 2 mg Q8W; primary endpoint at Week 56.
      • SOL-X: long-term open-label safety extension planned to start in Q2 2026 with a three-year follow-up for long-term safety and durability.
  • Program status and data timelines
    • SOL-1 enrolled 344 randomized, treatment-naïve subjects; topline Week 52 data expected at the Macula Society meeting (Feb 25–28, 2026). All subjects re-dosed at Week 52 and Week 76; safety follow-up continues to Week 104.
    • SOL-R targeted 555 randomized; 631 subjects were ultimately randomized; topline data expected Q1 2027.
  • Regulatory path
    • Plan to pursue an NDA under 505(b)(2) using axitinib’s prior approvals. The program includes a superiority trial (SOL-1) and a non-inferiority trial (SOL-R) as potential bases for submission.
  • Supporting data
    • Phase 1 Australia and Phase 1 US data support durability and activity, with long-term PK/PD and real-world durability discussed.

• HELIOS program (diabetic retinal disease)

  • HELIOS-3: Phase 3, approximately 930 NPDR without CI-DME subjects in a three-arm superiority trial evaluating two AXPAXLI 450 µg dosing regimens vs sham; primary endpoint is an ordinal 2-step DRSS change at Week 52.
  • HELIOS-2: potential Phase 3 trial comparing AXPAXLI 450 µg vs ranibizumab 0.3 mg (optional; not yet initiated) that could support a superiority label.
  • HELIOS-1: Phase 1 completed with data through Week 48 showing safety and DRSS improvements.

• OTX-TIC (travoprost intracameral hydrogel)

  • Status: Phase 2 completed for OAG/OHT; next steps under evaluation.
  • Phase 2 results showed sustained IOP reduction of roughly 24–30% from baseline over six months after a single implant.
  • AffaMed holds commercial rights for OTX-TIC in specified Asian territories.

• DEXTENZA (dexamethasone intracanalicular insert)

  • Commercial product since 2019 for post-surgical inflammation and pain; approved for ocular itching in allergic conjunctivitis in patients aged 2+ following a 2021 sNDA.
  • Commercial model: sold through specialty distributors for ambulatory surgery centers, hospital outpatient departments, and physician offices; supported by a dedicated reimbursement and sales force.
  • Usage and safety: nearly 750,000 eyes treated since launch; adverse events reported in approximately 2 per 10,000 patients.
  • AffaMed has rights to develop and commercialize DEXTENZA in specified markets in Asia.

Commercial, manufacturing, and intellectual property

• Manufacturing footprint (Bedford, MA)
  • ~20,000 sq ft facility for manufacturing and packaging DEXTENZA.
  • ~71,000 sq ft facility (plus expansion) for drug products and manufacturing of AXPAXLI materials and associated devices.
  • Strategy emphasizes internal manufacturing control to support commercial scale and to inform potential second-source outsourcing decisions.
• Market context
  • AXPAXLI targets large addressable markets in exudative retinal diseases, including wet AMD and diabetic retinal diseases.
  • DEXTENZA has an established commercial footprint and a defined safety profile.
• Intellectual property
  • Over 300 issued or pending U.S. and foreign patents and applications related to DEXTENZA, OTX-TIC, AXPAXLI, and the hydrogel technology.
  • Licensing and IP governance with Incept, LLC under original and amended agreements, with joint and controlled prosecution arrangements in defined areas.

Partnerships and licensing economics

• AffaMed License Agreement for DEXTENZA and OTX-TIC in Asia
  • Upfront payment: $12.0 million.
  • Development, regulatory, and commercial milestone payments: up to $91.0 million aggregate.
  • Royalties: tiered, in the low-teens to low-twenties percent on net sales, per product and jurisdiction.
  • Cost-sharing: sponsor generally bears development costs; AffaMed covers certain territory-specific and regulatory costs and may reimburse some global study data or local studies per the agreement.
  • Earlier payments and milestones:
    • $1.0 million milestone tied to FDA sNDA approval for DEXTENZA allergic conjunctivitis (Q4 2021).
    • $2.0 million clinical support payment related to OTX-TIC Asia development (Q2 2022).
    • $1.0 million milestone upon NMPA approval of AffaMed’s Phase 3 registrational study in China (Q2 2023).
• Incept license arrangements
  • Exclusive worldwide rights in a broad ophthalmic field with royalties on net sales in the low- to mid-single-digit range, and possible upfront payments in exchange for royalty adjustments.
  • Joint IP arrangements around shape-changing and Incept-related IP, with prosecution responsibilities allocated by agreement.

Regulatory and clinical development framework

• FDA engagement includes SPA agreements for SOL-1 and HELIOS-3.
• A 505(b)(2) NDA pathway is planned for AXPAXLI.
• Trial designs include long-term safety extensions (SOL-X) and consideration of combination approaches with existing anti-VEGF therapies.
• Pricing and reimbursement considerations, including PDUFA user fees and post-approval requirements, are part of regulatory planning.

Other program and platform context

• DEXTENZA is the first FDA-approved drug-eluting intracanalicular insert delivering dexamethasone for up to 30 days.
• ELUTYX-based materials have clinical use history in the human body since 1992, with safety data across more than five million patients in multiple FDA-approved treatments.

Summary

Ocular Therapeutix develops and commercializes ophthalmic therapies using the ELUTYX hydrogel platform to provide long-acting, local drug delivery to the eye. The company’s portfolio includes a marketed product (DEXTENZA) and development-stage assets (AXPAXLI for retinal disease and OTX-TIC for glaucoma), supported by strategic licensing partnerships in Asia and a broad IP estate. Key program milestones include active Phase 3 registrational programs for AXPAXLI (SOL-1, SOL-R, SOL-X) and ongoing clinical work for HELIOS-3 in diabetic retinal disease. Financial highlights from licensing arrangements include an AffaMed upfront of $12.0 million and potential milestone payments up to $91.0 million, plus tiered royalties.