Ocular Therapeutix™ Reports First Quarter 2025 Results and Business Highlights
- Strong cash position of $349.7M providing runway through 2028
- Exceptional retention in SOL-1 trial with on-track timeline for 1Q 2026 topline data
- Positive FDA feedback received for AXPAXLI NPDR registrational trial design
- Potential for 6-12 month dosing regimen on label for wet AMD treatment
- Streamlined SOL-R trial execution could accelerate NDA submission timeline
- Total revenue decreased 27.6% YoY to $10.7M in Q1 2025
- Net loss of $64.1M in Q1 2025
- R&D expenses more than doubled to $42.9M vs $20.7M in Q1 2024
- DEXTENZA sales declined due to pricing strategy impact and MIPS inclusion
Insights
Ocular's AXPAXLI showing strong trial retention with potential for 6-12 month dosing intervals; pipeline expansion into diabetic eye conditions progressing.
Ocular Therapeutix's Q1 update reveals significant progress in their AXPAXLI wet AMD program with exceptional retention rates in SOL-1 and strong enrollment in SOL-R following target reduction from 825 to 555 subjects. This reduction maintains 90% statistical power while preserving the FDA-guided non-inferiority margin.
The FDA's acceptance of the SOL-1 Special Protocol Assessment amendment represents a crucial strategic advancement. By incorporating re-dosing at Weeks 52 and 76, Ocular could potentially secure a product label supporting 6-12 month dosing intervals - a remarkable improvement over current wet AMD therapies that typically require more frequent administration. This extended interval directly addresses treatment burden, a critical barrier to optimal outcomes in chronic retinal conditions.
Beyond wet AMD, the company received positive FDA feedback on a potential registrational trial design for AXPAXLI in non-proliferative diabetic retinopathy (NPDR). This expansion into diabetic eye conditions substantially enlarges Ocular's addressable market. The company is also actively planning DME development, further broadening their retinal disease footprint.
With SOL-1 topline data firmly on track for Q1 2026 and the streamlined approach to SOL-R potentially accelerating overall NDA submission timelines, Ocular appears to be executing efficiently on its clinical strategy while maintaining regulatory alignment. These developments collectively strengthen AXPAXLI's position as a potentially differentiated therapy in the retinal disease landscape.
Strong $349.7M cash position offset by concerning 27.6% DEXTENZA revenue decline and doubled R&D expenses; neutral financial outlook.
Ocular's Q1 financial results present a contradictory picture. The
The
Operating expenses have increased dramatically, with R&D costs more than doubling to
The escalating burn rate combined with declining commercial revenue creates near-term financial pressure despite the robust cash position. The company's ability to reverse DEXTENZA's sales trajectory will be crucial for maintaining their projected runway while supporting extensive clinical development. The financial outlook balances promising clinical progress against concerning commercial performance and increased spending.
AXPAXLI™ SOL trials for wet AMD progressing rapidly following recent updates to accelerate and enhance the registrational program
Following positive FDA feedback for potential AXPAXLI NPDR registrational trial, Ocular is actively planning next steps in NPDR and DME
SOL-1 retention remains exceptional as trial is on track for 1Q 2026 topline data readout
SOL-R continues to have strong enrollment through streamlined, accelerated execution
Cash balance of
BEDFORD, Mass., May 05, 2025 (GLOBE NEWSWIRE) -- Ocular Therapeutix, Inc. (NASDAQ: OCUL, “Ocular”), a fully-integrated biopharmaceutical company committed to redefining the retina experience, today reported financial results for the first quarter ended March 31, 2025, and provided recent business highlights.
“We continue to advance the SOL registrational program for our product candidate AXPAXLI in wet AMD with urgency and precision. Our unwavering focus is on redefining the retina experience. Delivering a more sustainable therapy designed to drive better long-term outcomes is central to this effort. Earlier this year, we implemented strategic regulatory updates designed to accelerate the SOL program, enhancing capital and operational efficiencies. We believe these refinements could position AXPAXLI for an unprecedented 6 to 12-month dosing regimen on the label for the treatment of wet AMD and potentially enable an earlier NDA submission. These enhancements were made in alignment with FDA feedback and preserve the scientific integrity and robust powering of our two complementary registrational studies, SOL-1 and SOL-R,” said Pravin U. Dugel, MD, Executive Chairman, President and Chief Executive Officer of Ocular Therapeutix. “In addition to our momentum in wet AMD, we are thrilled with the positive written feedback received from the FDA on the design of a potential registrational trial for AXPAXLI in NPDR. These encouraging interactions represent a meaningful step forward as we plan our next steps in both NPDR and DME.”
“Backed by a world-class team, strong capital position, and disciplined execution, we believe we are in an outstanding position as we pursue our goal of becoming a leading retina company,” concluded Dr. Dugel.
Recent Achievements and Upcoming Milestones:
- SOL-1 (Phase 3, wet AMD) retention remains exceptional and the vast majority of rescues, evaluated on a masked basis, continue to be per protocol. Topline data are on track for 1Q 2026. In March, the Company announced that the FDA accepted an amendment to the SOL-1 Special Protocol Assessment (SPA) agreement that includes re-dosing of all subjects at Weeks 52 and 76 with their respective initial treatment of AXPAXLI or aflibercept (2 mg). Subjects will remain masked and are then followed for safety until the end of year two. This optimized design enhances the potential to achieve label flexibility for dosing AXPAXLI every 6 to 12 months and should provide valuable insights into AXPAXLI’s long-term durability.
- SOL-R (Phase 3, wet AMD) enrollment continues to be strong following the recent reduction in target randomization to approximately 555 subjects (previously 825). The strong SOL-1 subject retention observed to date and re-dosing amendment have enabled Ocular to reduce the size of the SOL-R non-inferiority study while continuing to meet the FDA requirements for long-term safety data. Streamlining the execution of SOL-R is expected to accelerate the trial readout and, if successful, the AXPAXLI wet AMD NDA submission timeline. SOL-R remains robustly powered at
90% with the non-inferiority margin of -4.5 ETDRS letter per FDA guidance. - Written feedback received from FDA on AXPAXLI registrational trial in non-proliferative diabetic retinopathy (NPDR). The FDA provided positive feedback to Ocular on a potential registrational trial design for AXPAXLI in NPDR. Following recent changes more broadly within the FDA, Ocular has not noticed any disruption in the cadence and nature of its dialogue with the Agency to date and continues to maintain productive interactions. The Company is actively planning next steps in the development of AXPAXLI for NPDR and diabetic macular edema (DME) and expects to provide further details at a later date.
First Quarter Ended March 31, 2025, Financial Results:
Total cash and cash equivalents were
Total net revenue was
The Company’s net product revenue was
The Company anticipates that net product revenue on a quarterly basis should increase for the remainder of 2025, driven primarily by expected increases in the number of units sold, as clinicians adjust to the impact of MIPS, and as the Company increases sales efforts directed towards HOPDs, which are receiving separate payment for DEXTENZA in 2025 after being ineligible for separate payments in 2024.
Research and development expenses for the first quarter of 2025 were
Selling and marketing expenses were
General and administrative expenses were
Net loss for the first quarter of 2025 was
Outstanding shares as of May 1, 2025, were approximately 159.3 million.
About AXPAXLI
AXPAXLI™ (also known as OTX-TKI) is an investigational, bioresorbable, intravitreal hydrogel incorporating axitinib, a small molecule, multi-target, tyrosine kinase inhibitor with anti-angiogenic properties, being evaluated for the treatment of wet AMD, diabetic retinopathy, diabetic macular edema, and other retinal diseases.
About the SOL-1 Study
The registrational Phase 3 SOL-1 trial (NCT06223958) is designed to evaluate the safety and efficacy of AXPAXLI in a multi-center, double-masked, randomized (1:1), parallel group study that involves more than 100 clinical trial sites located in the U.S. and Argentina. In December 2024, the trial completed randomization of 344 evaluable treatment-naïve subjects with a diagnosis of wet AMD in the study eye.
The superiority study has an eight-week loading segment prior to randomization. During the loading segment, subjects who have 20/80 vision or better and who satisfy other enrollment criteria receive two doses of aflibercept (2 mg) at Week -8 and Week -4. Eligible subjects who achieve best corrected visual acuity (BCVA) of 20/20 at Day 1 or gain at least 10 early treatment diabetic retinopathy study (ETDRS) letters at Day 1 are then randomized to receive a single dose of AXPAXLI or a single dose of aflibercept (2 mg). At Week 52 and at Week 76, all subjects are re-dosed with their respective initial treatment of AXPAXLI or aflibercept (2 mg). Subjects will be followed for safety until the end of Year 2. Throughout the study, subjects are assessed monthly. Trial subjects and designated study personnel will remain masked through the end of Year 2. The clinical trial protocol requires that, during the study, subjects in either arm meeting pre-specified rescue criteria will receive a supplemental dose of aflibercept (2 mg).
The primary endpoint of SOL-1 is the proportion of subjects who maintain visual acuity, defined as a loss of <15 ETDRS letters of BCVA, at Week 36. Subjects will continue to be evaluated for durability up to Week 52. The study is being conducted under a Special Protocol Assessment (SPA) agreement with the FDA.
About the SOL-R Study
The registrational Phase 3 SOL-R trial (NCT06495918) is designed to evaluate the safety and efficacy of AXPAXLI in a multi-center, double-masked, randomized (2:2:1), three-arm study that will involve sites located in the U.S. and the rest of the world. The trial is intended to randomize approximately 555 subjects who are treatment-naïve or were diagnosed with wet AMD in the study eye within about four months prior to enrollment.
This non-inferiority trial reflects a patient enrichment strategy over the six months prior to randomization that includes three screening doses of any anti-VEGF therapy, excluding brolucizumab-dbll, and monitoring to exclude those subjects with significant retinal fluid fluctuations. Subjects that continue to meet eligibility will enter a run-in period and receive two loading doses of aflibercept (2 mg) prior to Day 1. Subjects in the first arm receive a single dose of AXPAXLI at Day 1 and are re-dosed at Weeks 24, 48, and 72. Subjects in the second arm receive aflibercept (2 mg) on-label every eight weeks. Subjects in the third arm receive a single dose of aflibercept (8 mg) at Day 1 and are re-dosed at Weeks 24, 48, and 72, aligned with the AXPAXLI treatment arm for adequate masking. Subjects will be followed for safety until the end of Year 2. Throughout the study, subjects are assessed monthly. Trial subjects and designated study personnel will remain masked through the end of Year 2. Subjects in any arm that meet pre-specified rescue criteria will receive a supplemental dose of aflibercept (2 mg). The pre-specified rescue criteria include loss of ≥ 10 letters of BCVA from baseline or a combination of worsening anatomical measures and BCVA loss.
The primary endpoint of SOL-R is to demonstrate non-inferiority in mean BCVA change from baseline between the AXPAXLI and on-label aflibercept (2 mg) arms at Week 56. As per the protocol agreed to by the FDA, the non-inferiority margin for the lower bound is -4.5 letters of mean BCVA when compared to aflibercept (2 mg) dosed every eight weeks. In a written Type C response received in August 2024, and a subsequent written response received in December 2024, the FDA agreed that the SOL-R repeat dosing wet AMD study, with a primary endpoint at Week 56, should be appropriate as an adequate and well-controlled study in support of a potential New Drug Application and product label for wet AMD.
About Wet AMD
Wet age-related macular degeneration (wet AMD) is a leading cause of severe, irreversible vision loss affecting approximately 14.5 million individuals globally and 1.7 million in the United States alone (2024 Market Scope® Retinal Pharmaceuticals Market Report). Wet AMD causes vision loss due to abnormal new blood vessel growth and hyperpermeability and associated retinal vascularity in the macula, which is primarily stimulated by local upregulation of vascular endothelial growth factor (VEGF). Without prompt and continuous treatment to control this exudative activity, patients develop irreversible vision loss. With proper treatment, patients may maintain visual function for a period of time and may temporarily regain lost vision. Challenges with current therapies include pulsatile, repeated intraocular injections, treatment-related adverse events and up to
About Ocular Therapeutix, Inc.
Ocular Therapeutix, Inc. is a fully-integrated biopharmaceutical company committed to redefining the retina experience. AXPAXLI™ (also known as OTX-TKI), Ocular’s investigational product candidate for retinal disease, is an axitinib intravitreal hydrogel based on its ELUTYX™ proprietary bioresorbable hydrogel-based formulation technology. AXPAXLI is currently in Phase 3 clinical trials for wet age-related macular degeneration (wet AMD).
Ocular’s pipeline also leverages the ELUTYX technology in its commercial product DEXTENZA®, an FDA-approved corticosteroid for the treatment of ocular inflammation and pain following ophthalmic surgery in adults and pediatric patients and ocular itching associated with allergic conjunctivitis in adults and pediatric patients aged two years or older, and in its investigational product candidate PAXTRAVA™ (also known as OTX-TIC), which is a travoprost intracameral hydrogel that is currently in a Phase 2 clinical trial for the treatment of open-angle glaucoma or ocular hypertension.
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The Ocular Therapeutix logo and DEXTENZA® are registered trademarks of Ocular Therapeutix, Inc. AXPAXLI™, PAXTRAVA™, ELUTYX™, and Ocular Therapeutix™ are trademarks of Ocular Therapeutix, Inc.
Forward-Looking Statements
Any statements in this press release about future expectations, plans, and prospects for the Company, including the commercialization of DEXTENZA; the development, regulatory status of and regulatory submissions regarding the Company’s product candidates; the design of, and the timing of the screening, enrollment and randomization of patients in and the availability of data from the Company’s SOL-1 and SOL-R Phase 3 clinical trials of AXPAXLI (also known as OTX-TKI) for the treatment of wet AMD; the Company’s plans to advance the development of AXPAXLI, including in additional indications such as NPDR and DME, and its other product candidates; the potential utility or adoption, if approved, of any of the Company’s product candidates; the Company’s cash runway and the sufficiency of the Company’s cash resources; and other statements containing the words “anticipate”, “believe”, “estimate”, “expect”, “intend”, “designed”, “goal”, “may”, “might”, “plan”, “predict”, “project”, “target”, “potential”, “will”, “would”, “could”, “should”, “continue”, and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors. Such forward-looking statements involve substantial risks and uncertainties that could cause the Company’s development programs, future results, performance, or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the timing and costs involved in commercializing any product or product candidate that receives regulatory approval; the ability to retain regulatory approval of any product or product candidate that receives regulatory approval; the ability to maintain and the sufficiency of product, procedure and any other reimbursement codes for DEXTENZA; the initiation, design, timing, conduct and outcomes of ongoing and planned clinical trials; the risk that the FDA will not agree with the Company’s interpretation of the written agreement under the Special Protocol Assessment for the SOL-1 trial; the risk that the FDA may not agree that the protocol and statistical analysis plan of SOL-R or that the data generated by the SOL-1 and SOL-R trials support marketing approval, even if the trials are successful; the risk that the Company and the FDA may not agree on the registrational pathway for any of its product candidates; uncertainty as to whether the data from earlier clinical trials will be predictive of the data of later clinical trials, particularly later clinical trials that have a different design or utilize a different formulation than the earlier trials, whether preliminary or interim data from a clinical trial (including masked safety or masked rescue data from the Company’s SOL-1 trial) will be predictive of final data from such trial, or whether data from a clinical trial assessing a product candidate for one indication will be predictive of results in other indications; availability of data from clinical trials and expectations for regulatory submissions and approvals; the Company’s scientific approach and general development progress; uncertainties inherent in estimating the Company’s cash runway, future expenses and other financial results, including its ability to fund future operations, including clinical trials; the Company’s existing indebtedness and the ability of the Company’s creditors to accelerate the maturity of such indebtedness upon the occurrence of certain events of default; and other factors discussed in the “Risk Factors” section contained in the Company’s quarterly and annual reports on file with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company’s views as of the date of this press release. The Company anticipates that subsequent events and developments may cause the Company’s views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so, whether as a result of new information, future events or otherwise, except as required by law. These forward-looking statements should not be relied upon as representing the Company’s views as of any date subsequent to the date of this press release.
Investors & Media
Ocular Therapeutix, Inc.
Bill Slattery
Vice President, Investor Relations
bslattery@ocutx.com
| Ocular Therapeutix, Inc. | ||||||||
| Consolidated Balance Sheets | ||||||||
| (in thousands, except share and per share data) | ||||||||
| March 31, | December 31, | |||||||
| 2025 | 2024 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 349,681 | $ | 392,102 | ||||
| Accounts receivable, net | 25,221 | 32,388 | ||||||
| Inventory | 3,269 | 3,040 | ||||||
| Prepaid expenses and other current assets | 9,523 | 13,457 | ||||||
| Total current assets | 387,694 | 440,987 | ||||||
| Property and equipment, net | 10,784 | 9,389 | ||||||
| Restricted cash | 1,614 | 1,614 | ||||||
| Operating lease assets | 5,828 | 5,945 | ||||||
| Total assets | $ | 405,920 | $ | 457,935 | ||||
| Liabilities and Stockholders’ Equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 4,626 | $ | 4,176 | ||||
| Accrued expenses and other current liabilities | 31,080 | 35,117 | ||||||
| Deferred revenue | 64 | 128 | ||||||
| Operating lease liabilities | 2,156 | 1,933 | ||||||
| Total current liabilities | 37,926 | 41,354 | ||||||
| Other liabilities: | ||||||||
| Operating lease liabilities, net of current portion | 4,866 | 5,345 | ||||||
| Derivative liability | 13,852 | 13,246 | ||||||
| Deferred revenue, net of current portion | 14,000 | 14,000 | ||||||
| Notes payable, net | 69,202 | 68,505 | ||||||
| Other non-current liabilities | 144 | 141 | ||||||
| Total liabilities | 139,990 | 142,591 | ||||||
| Commitments and contingencies | ||||||||
| Stockholders’ equity: | ||||||||
| Preferred stock, | — | — | ||||||
| Common stock, | 16 | 16 | ||||||
| Additional paid-in capital | 1,221,051 | 1,206,412 | ||||||
| Accumulated deficit | (955,137 | ) | (891,084 | ) | ||||
| Total stockholders’ equity | 265,930 | 315,344 | ||||||
| Total liabilities and stockholders’ equity | $ | 405,920 | $ | 457,935 | ||||
| Ocular Therapeutix, Inc. | |||||||
| Consolidated Statements of Operations and Comprehensive Loss | |||||||
| (in thousands, except share and per share data) | |||||||
| Three Months Ended | |||||||
| March 31, | |||||||
| 2025 | 2024 | ||||||
| Revenue: | |||||||
| Product revenue, net | $ | 10,634 | $ | 14,715 | |||
| Collaboration revenue | 64 | 59 | |||||
| Total revenue, net | 10,698 | 14,774 | |||||
| Costs and operating expenses: | |||||||
| Cost of product revenue | 1,262 | 1,326 | |||||
| Research and development | 42,857 | 20,735 | |||||
| Selling and marketing | 14,148 | 10,183 | |||||
| General and administrative | 16,348 | 14,147 | |||||
| Total costs and operating expenses | 74,615 | 46,391 | |||||
| Loss from operations | (63,917 | ) | (31,617 | ) | |||
| Other income (expense): | |||||||
| Interest income | 3,826 | 3,922 | |||||
| Interest expense | (2,984 | ) | (4,051 | ) | |||
| Change in fair value of derivative liabilities | (978 | ) | (5,152 | ) | |||
| Loss on extinguishment of debt | — | (27,950 | ) | ||||
| Total other expense, net | (136 | ) | (33,231 | ) | |||
| Net loss | $ | (64,053 | ) | $ | (64,848 | ) | |
| Net loss per share, basic | $ | (0.38 | ) | $ | (0.49 | ) | |
| Weighted average common shares outstanding, basic | 169,396,989 | 132,021,945 | |||||
| Net loss per share, diluted | $ | (0.38 | ) | $ | (0.49 | ) | |
| Weighted average common shares outstanding, diluted | 169,396,989 | 132,021,945 | |||||
FAQ
What were Ocular Therapeutix (OCUL) Q1 2025 financial results?
What is the status of OCUL's AXPAXLI SOL trials for wet AMD?
How long will OCUL's current cash position last?
What was the FDA's response to OCUL's AXPAXLI NPDR trial design?
Why did OCUL's DEXTENZA sales decline in Q1 2025?
