OCULAR THERAPEUTIX, INC Q3 FY2026 earnings call

Dr. Pravin Dugel, CEO, outlined the company's strategic position and recent operational milestones:

• Regulatory Strategy: Ocular plans to submit the New Drug Application (NDA) for Xpaxly in wet age-related macular degeneration (wet AMD) in Q4 2026. The submission will utilize the FDA’s 505(b)(2) pathway, potentially shortening the review timeline by up to 60 days compared to traditional NME pathways. • Clinical Data & SOL1 Success: The Phase 3 SOL1 trial demonstrated superiority over a single injection of aflibercept 2 mg. Key data points include approximately 75% of patients maintaining vision at week 36 and two-thirds remaining rescue-free through week 52 following a single injection. • SOLAR Trial Adjustments: To protect statistical integrity during an interim safety analysis, the primary efficacy endpoint for the Phase 3 SOLAR trial was moved from week 56 to week 40. This change incurs only a minimal alpha penalty (0.0001) and ensures all patients complete efficacy assessments before safety unmasking. Week 56 and 96 remain key secondary endpoints. • Durability Evidence: Post-hoc analyses show that approximately 71% of subjects were quantitatively dry (using a stringent AI-based algorithm defining dryness as ≤10 nanoliters of fluid) at week 8, with ~70% remaining dry through week 36. Early persistent fluid or variability was identified as a negative predictor for durability. • Commercial Preparation: The company has engaged with 100% of top-priority national and regional payer accounts covering ~84% of commercial lives. Pre-approval exchanges indicate strong interest in Xpaxly’s potential for fewer injections and superior disease control. • Board Expansion: Dr. Riyadh Sharif joined the board to bring global commercial expertise, while Dr. Sung-Se Hong retired. This reflects a shift toward preparing for commercial launch capabilities. • Pipeline Progress: Enrollment continues in Helios 3, the Phase 3 trial for non-proliferative diabetic retinopathy (NPDR). Additionally, the long-term extension study SOLEX modified its dosing cadence from 24 weeks to 48 weeks to reflect confidence in annual dosing potential.

Ocular Therapeutix is currently a clinical-stage biopharmaceutical company with no commercial revenue. The transcript does not provide specific financial performance metrics (revenue or absolute costs) for product segments, as the company’s primary focus remains on the development of its investigational drug candidate, Xpaxly (OTXTKI). Financial highlights are limited to the balance sheet position.

• NDA Submission: Management reaffirmed guidance to submit the NDA for Xpaxly in wet AMD in the fourth quarter of 2026. • Approval Expectations: Management expressed high confidence that Xpaxly will receive FDA approval based on the SOL1 data package alone, though they noted SOLAR data may be valuable for international regulatory approvals. • Safety Database: Confirmed that the NDA will include safety data from >300 patients with at least one year of follow-up, incorporating repeat-dosing data from both SOL1 and SOLAR. • No Specific Revenue Guidance: As the product is not yet approved, no specific revenue or profit guidance was provided. Cash runway is expected to extend into 2028 based on current assumptions, excluding full commercialization expenses.

• Regulatory Risk: While aligned with the FDA, there is inherent risk in the approval process. The use of the 505(b)(2) pathway requires strict adherence to regulatory expectations to avoid delays or major amendments that could reset review clocks. • Clinical Trial Integrity: The decision to move the SOLAR primary endpoint carries a small statistical alpha penalty. Failure to maintain masking or operational bias could compromise the trial's validity. • Competitive Landscape: Although management claims Xpaxly has no direct late-stage competitor, the landscape is evolving. Recent failures of peer TKI programs highlight risks associated with potency and trial design if not executed correctly. • Patient Selection Dependency: Efficacy is heavily dependent on patient selection. Patients with early persistent fluid or variability showed worse outcomes, suggesting a subset of the population may not benefit as significantly. • Commercial Execution Risk: Transitioning from a clinical-stage to a commercial entity involves significant execution risk, including physician adoption, payer reimbursement, and infrastructure building.

Q: What data drove the decision to extend the SOLEX dosing interval from 24 weeks to 48 weeks, and will this apply to all patients? / A: The change is driven by high confidence in Xpaxly’s durability observed in SOL1, where two-thirds of challenging patients remained rescue-free at 12 months with a single injection. It is not automatic for all; it provides evidence for clinicians that once-a-year dosing is viable for stable patients. The goal is to offer a roadmap showing how bolus therapies can transition to long-term maintenance, leveraging the drug's sustained release profile.

Q: Did the FDA request the change of the SOLAR primary endpoint from week 56 to week 40? / A: No, the change was initiated proactively by Ocular after discussions with the FDA began post-Type C meeting in May. The primary driver was to protect statistical integrity by ensuring all patients crossed the efficacy assessment point before the interim safety unmasking at week 52. This prevents any potential operational bias and aligns with FDA requirements for trial integrity.

Q: Will the NDA submission rely solely on SOL1, or is SOLAR required for US approval? / A: Management stated with increasing confidence that US FDA approval can be achieved based on SOL1 data alone. However, SOLAR results are expected to be important for regulatory agencies outside the US. The NDA includes SOLAR safety data at week 52 to meet the requirement of >300 exposed patients, but efficacy data from SOLAR will remain masked until week 96 to preserve trial value.

Q: How does Ocular distinguish Xpaxly from competitors in the competitive landscape? / A: Management emphasizes three pillars: TKI potency (Excedinib is ~100x more potent than competitors against VEGFR2), the bioresorbable hydrogel delivery vehicle, and rigorous patient selection/trial design. They view Xpaxly as a maintenance therapy complementary to bolus anti-VEGF agents used for initial disease control, rather than a direct competitor for the initiation phase.

Q: What is the status of the repeat dosing data for the NDA package? / A: The NDA will include comprehensive repeat dosing safety data. All patients in SOL1 received repeat dosing at week 52, and all active patients in SOL1, SOLAR, and SOLEX have received multiple injections. This provides a robust safety database exceeding the FDA’s requirement for long-term exposure evaluation.