• Cash Balance: $392 million as of December 31, 2024.
• Cash Runway: Sufficient to fund planned operating expenses, debt service obligations, and capital expenditures into 2028.
• SOL-1 Trial Enrollment: Completed randomization with 344 subjects across more than 100 clinical sites.
• SOL-R Trial Enrollment: Target randomization reduced from 825 to approximately 555 subjects, with 311 subjects enrolled as of January 10, 2025.
• Non-Inferiority Margin for SOL-R: Set at 4.5 letters of mean BCVA.

• Warning! GuruFocus has detected 8 Warning Signs with MRSN.

Release Date: March 03, 2025

For the complete transcript of the earnings call, please refer to the full earnings call transcript.

Positive Points

• Ocular Therapeutix Inc (NASDAQ:OCUL) received FDA approval for an amendment to their special protocol agreement for SOL-1, allowing redosing at week 52 and week 76, potentially securing a six to 12-month dosing label for AXPAXLI in wet AMD.
• The company completed randomization for the SOL-1 trial ahead of schedule, with 344 subjects enrolled across more than 100 clinical sites in the US and Argentina.
• Ocular Therapeutix Inc (NASDAQ:OCUL) has a strong financial position with a cash balance of $392 million as of December 31, 2024, and does not intend to raise additional capital this year.
• The company is well-positioned to expand AXPAXLI into other retinal indications such as non-proliferative diabetic retinopathy and diabetic macular edema, with promising early clinical results.
• Enrollment for the SOL-R trial is progressing well, with 311 subjects enrolled as of January 10, 2025, and the trial size has been reduced from 825 to 555 subjects, enhancing capital efficiency and maintaining statistical power.

Negative Points

• The timeline for reporting topline data for the SOL-1 trial has been shifted to the first quarter of 2026 due to the requirement to maintain masking until week 52.
• The company faces the challenge of demonstrating AXPAXLI's durability and effectiveness in a repeat dosing setting, which is critical for regulatory approval and commercial success.
• Ocular Therapeutix Inc (NASDAQ:OCUL) must navigate the complexities of aligning their clinical trial designs with FDA guidance, which can impact trial timelines and outcomes.
• The company is dependent on FDA feedback for advancing clinical trials in non-proliferative diabetic retinopathy and diabetic macular edema, which could affect their strategic plans.
• Despite strong financials, the company's current cash projections do not account for the impact of clinical trial activities in new indications, which could require additional resources in the future.

Q & A Highlights

Q: Can you explain the rationale behind the changes in SOL-1 and SOL-R trials, especially given the strong patient retention and redosing rates? A: Dr. Pravin Dugel, Executive Chairman, President and CEO, explained that the changes are designed to enhance efficiency and potentially accelerate the timeline for bringing AXPAXLI to market. The FDA approved a redosing amendment for SOL-1, allowing for increased flexibility and durability data. This efficiency allowed for a reduction in SOL-R's size from 825 to 555 subjects, maintaining statistical integrity while potentially speeding up the regulatory filing process.

Q: What is the purpose of redosing at week 76 in SOL-1, and will all patients receive redosing regardless of their trial arm? A: Dr. Pravin Dugel stated that the redosing at week 76 is to maximize drug exposure to meet FDA safety requirements. All patients, regardless of their trial arm or prior rescue treatments, will be redosed with the original drug they were randomized to at both week 52 and week 76.

Q: How do the changes in SOL-1 and SOL-R affect the timeline for NDA submission and the potential label for AXPAXLI? A: Dr. Pravin Dugel clarified that the primary endpoints for both SOL-1 and SOL-R remain unchanged. The NDA submission will be based on achieving these endpoints. The changes allow for more efficient recruitment and potentially a better label, with flexibility in dosing from six to 12 months, enhancing commercial prospects.

Q: What are the FDA's expectations regarding rescues in SOL-R, and how will they be evaluated? A: Dr. Pravin Dugel noted that the FDA allows the first rescue in a non-inferiority study as long as it doesn't affect the primary endpoint. Rescues will be evaluated in the context of a positive SOL-1 study, which will define the drug's durability, providing a potential advantage in regulatory review.

Q: What are the plans for advancing NPDR and DME indications following FDA feedback on clinical trial design? A: Dr. Pravin Dugel stated that the company plans to pursue both NPDR and DME indications, with trial designs dependent on upcoming FDA feedback. The company is financially disciplined and does not anticipate needing additional funding this year, with cash runway projected into 2028.

For the complete transcript of the earnings call, please refer to the full earnings call transcript.