Summary of Ocular Therapeutix Conference Call Company Overview - **Company**: Ocular Therapeutix (NasdaqGM: OCUL) - **Focus**: Development of innovative therapies for retinal diseases, particularly through their lead program AXPAXLI Key Points and Arguments Clinical Trials and Drug Development - Ocular Therapeutix is currently conducting two pivotal Phase III clinical trials: SOL-1 (superiority study) and SOLAR (non-inferiority study) [3][10] - SOL-1 results are expected in Q1 2026, while SOLAR results are anticipated in the first half of 2027 [3][4] - AXPAXLI is a TKI (tyrosine kinase inhibitor) on an attunable hydrogel platform, showing promising results in studies conducted in Australia and the U.S. [5][6] - AXPAXLI is reported to be 100 times more potent and 50 times more selective than other TKIs [5] Unmet Needs in Retinal Diseases - There is a significant dropout rate of 40% in wet macular degeneration treatments, leading to blindness for many patients [6] - AXPAXLI aims to provide a sustainable treatment option, allowing for annual injections instead of monthly or bi-monthly visits [8] - For diabetic retinopathy, less than 1% of patients currently receive treatment, despite the high risk of complications [7][30] Confidence in Clinical Trial Design - The design of SOL-1 and SOLAR trials follows FDA guidelines, with a focus on de-risking patient populations [12][15] - SOL-1 has a rescue-free rate of 100% at six months and 80% at twelve months, indicating strong efficacy [11] - The superiority label is considered a significant advantage, as it differentiates AXPAXLI from other anti-VEGF treatments [16][18] Regulatory Pathway - Ocular Therapeutix plans to utilize a 505(b)(2) regulatory pathway for expedited approval, leveraging existing FDA-approved components of AXPAXLI [28][29] - The company expects to submit for approval immediately after successful SOLAR results [28] Market Opportunity - The target market for diabetic retinopathy is estimated to be 3.5 times larger than that for wet macular degeneration, with a substantial unmet need [30] - AXPAXLI is designed to address both diabetic retinopathy and diabetic macular edema, potentially leading to a broad label for treatment [32][38] Commercial Strategy - Ocular Therapeutix has an existing product, Dextenza, which provides a foundation for scaling up their commercial efforts in ophthalmology [39] - The company is well-capitalized and does not currently see a need for partnerships or licensing opportunities for the commercialization of AXPAXLI [40] Additional Important Information - The company has a strong intellectual property position, with extensions to 2044, enhancing the attractiveness of AXPAXLI [17] - The novel ordinal endpoint for diabetic retinopathy studies has been accepted by the FDA, increasing the likelihood of successful outcomes in future trials [34][35] This summary encapsulates the critical insights from the Ocular Therapeutix conference call, highlighting the company's strategic direction, clinical advancements, and market potential in the field of retinal diseases.

Company Participation in Investor Conferences - Ocular Therapeutix, Inc. will participate in several investor conferences in November and December 2025, including the Jefferies Global Healthcare Conference and the Piper Sandler 37th Annual Healthcare Conference [1][2] Leadership Presentation Details - Pravin U. Dugel, MD, Executive Chairman, President and CEO, will present at both conferences, with the Jefferies conference scheduled for November 18, 2025, and the Piper Sandler conference on December 3, 2025 [2] Product Pipeline and Clinical Trials - Ocular Therapeutix is focused on redefining the retina experience with its investigational product AXPAXLI™ (OTX-TKI), currently in Phase 3 clinical trials for wet age-related macular degeneration (wet AMD) and planning to initiate a Phase 3 program for non-proliferative diabetic retinopathy (NPDR) imminently [3] - The company also leverages its proprietary ELUTYX™ technology in its commercial product DEXTENZA, an FDA-approved corticosteroid for treating ocular inflammation and pain, and in the investigational product OTX-TIC, which has completed a Phase 2 clinical trial for open-angle glaucoma or ocular hypertension [4]

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Core Insights - Ocular Therapeutix reported a quarterly loss of $0.37 per share, which was better than the Zacks Consensus Estimate of a loss of $0.39, but worse than a loss of $0.26 per share a year ago, indicating an earnings surprise of +5.13% [1] - The company generated revenues of $14.54 million for the quarter ended September 2025, surpassing the Zacks Consensus Estimate by 8.89%, although this represents a decline from $15.43 million in the same quarter last year [2] - Ocular Therapeutix shares have increased by approximately 33.6% year-to-date, outperforming the S&P 500's gain of 16.5% [3] Earnings Outlook - The future performance of Ocular Therapeutix's stock will largely depend on management's commentary during the earnings call and the company's earnings outlook [4][6] - The current consensus EPS estimate for the upcoming quarter is -$0.36 on revenues of $14.77 million, and for the current fiscal year, it is -$1.48 on revenues of $53.44 million [7] Industry Context - The Medical - Drugs industry, to which Ocular Therapeutix belongs, is currently ranked in the top 36% of over 250 Zacks industries, suggesting a favorable outlook compared to lower-ranked industries [8]

Financial Data and Key Metrics Changes - The company ended Q3 2025 with approximately $345 million in cash, not including around $445 million in net proceeds from an October equity financing [48] - The financial strength provides flexibility for top-line data from registrational programs and preparation for commercialization [50] Business Line Data and Key Metrics Changes - The registrational studies for XPAXLY in Wet AMD (SOL1 and SOLAR) are progressing well, with SOLAR reaching its target randomization of 555 subjects [6][31] - The SOL1 trial has over 95% patient retention, which is exceptional for retina trials [27] Market Data and Key Metrics Changes - The global annual anti-VEGF market is estimated at approximately $15 billion, with significant untapped opportunities in Wet AMD and diabetic retinal disease [16] - In diabetic retinopathy, fewer than 1% of the 6.4 million NPDR patients in the U.S. receive treatment, indicating a large market expansion opportunity [17] Company Strategy and Development Direction - The company aims to redefine the retina experience through a strategic triad: potential superiority label, market expansion, and immediate adoptability of XPAXLY [10][52] - The Helios program is designed to pursue a broad diabetic retinopathy label, including DME, with a single registrational program [47] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential of XPAXLY to break the cycle of pricing pressures and commoditization in the retina market [12][14] - Positive feedback from payers indicates a strong market preparation for XPAXLY, emphasizing its potential to deliver better outcomes and lower treatment burdens [49] Other Important Information - The company is committed to maintaining high trial integrity and has received no safety signals to date from independent monitoring [30] - The design of the Helios trials includes a novel ordinal endpoint that captures a broader range of patient responses, which is expected to increase the probability of success [40][43] Q&A Session Summary Question: Initial label expectations for Wet AMD - Management expects a superiority label based on SOL1, with flexibility in dosing intervals [59][60] Question: Expected patient populations in Helios trials - Management indicated confidence in obtaining a broad label that encompasses all diabetic eye disease without needing separate DME trials [68] Question: Progress of NPDR studies - Management confirmed that the process for NPDR studies began immediately after funding, leveraging existing site relationships for efficient enrollment [90] Question: Risks associated with the ordinal DRSS endpoint - Management expressed great confidence in the ordinal endpoint, citing overwhelming success in previous studies [96][98] Question: Enrollment timeline for Helios 2 trial - Management believes there will be no issues completing the trials efficiently due to high demand and enthusiasm from both patients and investigators [105]

Financial Data and Key Metrics Changes - The company ended Q3 2025 with approximately $345 million in cash, not including about $445 million in net proceeds from an October equity financing [26] - The company has an expected cash runway into 2028, providing financial flexibility for ongoing clinical programs and potential commercialization [27] Business Line Data and Key Metrics Changes - The registrational programs for XPAXLY in Wet AMD and diabetic retinopathy are progressing well, with SOL1 on track for top-line data in Q1 2026 and SOLar reaching its target randomization of 555 subjects [28][29] - The SOL1 trial has over 95% patient retention, indicating strong engagement and compliance [15] Market Data and Key Metrics Changes - The global annual anti-VEGF market is estimated at approximately $15 billion, with significant opportunities for expansion due to high treatment discontinuation rates in Wet AMD and low treatment rates in diabetic retinopathy [9][10] - The company aims to capture a larger addressable market by reducing treatment burden and improving adherence, potentially reaching millions of untreated patients [11][28] Company Strategy and Development Direction - The company is focused on achieving a superiority label for XPAXLY, which could differentiate it from existing anti-VEGF therapies and insulate it from pricing pressures [7][8] - The strategy includes a triad approach: potential superiority, market expansion, and immediate adoptability, aiming to redefine the retina treatment landscape [27][28] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the clinical strategy and market potential for XPAXLY, reinforced by positive feedback from payers and investors [26][27] - The company is committed to delivering long-lasting, clinically impactful therapies for retinal diseases, with a focus on early treatment to improve long-term outcomes [5][19] Other Important Information - The Helios program for diabetic retinopathy aims to pursue a broad label that encompasses both NPDR and DME, addressing a significant unmet need in the market [20][25] - The company has engaged with payers representing a large portion of U.S. commercial lives, receiving positive feedback on the potential of XPAXLY [14] Q&A Session Summary Question: What will the initial label for Wet AMD look like? - Management expects a superiority label based on SOL1, with flexibility for dosing intervals and repeat dosing [33][34] Question: What percentage of enrolled patients in Helios trials will have non-center-involved DME? - Management indicated confidence in achieving a broad label that includes all diabetic eye diseases based on previous study data [36][39] Question: What is the progress of NPDR studies? - Management confirmed that the process for NPDR studies began immediately after the recent financing, leveraging existing site relationships for efficient enrollment [56][59] Question: Are there risks associated with the ordinal two-step DRSS endpoint? - Management expressed great confidence in the ordinal endpoint, citing strong data from previous studies to support its validity [61][63] Question: How long will it take to complete enrollment for Helios 2? - Management believes there will be no issues in completing the trials efficiently due to high demand and enthusiasm from both patients and investigators [66]

Financial Data and Key Metrics Changes - The company ended Q3 2025 with approximately $345 million in cash, not including around $445 million in net proceeds from an October equity financing [25] - The financial strength provides the company with a cash runway into 2028, allowing for flexibility in pursuing clinical programs and preparing for commercialization [26] Business Line Data and Key Metrics Changes - The registrational programs for XPAXLY in Wet AMD and diabetic retinopathy are progressing well, with SOL1 on track for top-line data in Q1 2026 and SOLAR reaching its target randomization of 555 subjects [27][28] - The Helios program for diabetic retinopathy is designed to capture a broad label, addressing both NPDR and DME, with a focus on early treatment to prevent disease progression [19][24] Market Data and Key Metrics Changes - The global annual anti-VEGF market is estimated at approximately $15 billion, with significant opportunities for expansion as many patients remain untreated [8] - In Wet AMD, up to 40% of patients discontinue therapy within the first year, highlighting a substantial market opportunity for XPAXLY to improve adherence and reduce treatment burden [9] Company Strategy and Development Direction - The company aims to redefine the retina experience through a strategic triad: potential superiority label, market expansion, and immediate adoptability of XPAXLY [5][27] - The focus is on achieving a superiority label that distinguishes XPAXLY from existing anti-VEGF therapies, potentially insulating it from pricing pressures and enhancing market dynamics [6][7] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the clinical strategy and market potential of XPAXLY, reinforced by positive feedback from payers and investors [25][26] - The company is committed to delivering long-lasting, clinically impactful therapies for retinal diseases, with a focus on reducing treatment burden and improving patient outcomes [4][12] Other Important Information - The company has engaged with payers representing over 75% of U.S. commercial lives, receiving positive feedback on the potential of XPAXLY to deliver meaningful clinical differentiation [13] - The SOL1 trial has achieved over 95% patient retention, indicating strong engagement and compliance within the study [14] Q&A Session Summary Question: How is the initial label for Wet AMD expected to look? - Management anticipates a superiority label based on SOL1, with flexibility for dosing intervals and repeat dosing [32][33] Question: What percentage of enrolled patients in Helios trials will have non-center-involved DME? - Management indicated that they do not have specific percentages yet but expressed confidence in obtaining a broad label that includes all diabetic eye disease [35][38] Question: What is the progress of the SOLar study? - Management reported that SOLar has reached its target randomization of 555 patients, with a focus on thoughtful patient selection to ensure stability [40][43] Question: What details will be shared in the SOL1 top line? - Management stated that the primary goal of SOL1 is to achieve a superiority label, and they will provide data that supports confidence in the success of SOLar [47][49] Question: How will the NPDR studies be accelerated? - Management noted that they are leveraging existing site relationships and expertise to ensure efficient enrollment and execution of the NPDR studies [51][52] Question: Are there risks associated with the ordinal two-step DRSS endpoint? - Management expressed great confidence in the ordinal endpoint, citing strong data from previous studies to support its validity [57][59]

Clinical Trials and Product Development - AXPAXLI is currently in Phase 3 clinical trials for wet age-related macular degeneration (wet AMD) and plans to initiate a Phase 3 program for non-proliferative diabetic retinopathy (NPDR) imminently[113]. - The SOL-1 trial has randomized 344 subjects with a primary endpoint of maintaining visual acuity at Week 36, with over 95% retention rate among participants[120][121]. - The SOL-R trial has achieved its randomization target of 555 subjects, with topline data expected in the first half of 2027[127]. - The HELIOS-2 and HELIOS-3 trials for NPDR will evaluate AXPAXLI's efficacy using a novel ordinal primary endpoint, aiming for 6- and 12-month dosing intervals[135][136]. - AXPAXLI has the potential to be the first product for wet AMD with a superiority label compared to a single injection of anti-VEGF, with redosing potentially every 12 months[131]. - The SOL-1 trial is conducted under a special protocol assessment agreement with the FDA, with topline results expected in Q1 2026[121]. - The HELIOS-2 trial will include approximately 432 subjects, comparing AXPAXLI 450 μg to ranibizumab 0.3 mg, with a primary endpoint assessed at Week 52[138][140]. - The HELIOS-3 trial will involve approximately 930 subjects, comparing two dosing regimens of AXPAXLI to a sham comparator, with the same primary endpoint as HELIOS-2[141][143]. - The company plans to leverage the 505(b)(2) NDA review pathway for AXPAXLI, potentially shortening the review timeline by two months[131]. Financial Performance - The company reported net product revenue of $14.5 million for the three months ended September 30, 2025, a decrease of $0.8 million or 5.2% compared to $15.3 million for the same period in 2024[152]. - For the nine months ended September 30, 2025, net product revenue was $38.6 million, reflecting a decrease of $7.8 million or 16.8% from $46.4 million in the same period in 2024[152]. - The company reported a net loss of $201.3 million for the nine months ended September 30, 2025, compared to $193.5 million in 2024[218]. - The net loss for the three months ended September 30, 2025, was $69.4 million, compared to a net loss of $36.5 million for the same period in 2024, representing an increase of $32.9 million[174]. - The company anticipates continued increases in selling and marketing expenses as it supports the commercialization of DEXTENZA and prepares for the potential launch of AXPAXLI[204]. Research and Development Expenses - Research and development expenses increased by $15.3 million to $52.4 million for the three months ended September 30, 2025, compared to $37.1 million for the same period in 2024[174]. - Total research and development expenses for the nine months ended September 30, 2025, were $146.3 million, compared to $86.6 million in 2024, reflecting an increase of $59.7 million[196]. - Direct research and development expenses for products and candidates were $35.9 million for the three months ended September 30, 2025, compared to $18.2 million in 2024, an increase of $17.7 million[179]. - Direct research and development expenses for products and candidates were $96.2 million for the nine months ended September 30, 2025, compared to $42.0 million in 2024, an increase of $54.2 million[198]. - The company anticipates an increase in research and development expenses in the future as it supports the continued development of its product candidates[164]. Sales and Marketing - In-Market Sales for DEXTENZA were approximately 48,000 units in the three months ended September 30, 2025, an increase of approximately 6,000 units compared to the same period in 2024[154]. - Selling and marketing expenses rose to $13.1 million for the three months ended September 30, 2025, up from $10.6 million in 2024, reflecting a year-over-year increase of $2.5 million[181]. - Selling and marketing expenses increased to $41.0 million for the nine months ended September 30, 2025, up from $30.8 million in 2024, reflecting a year-over-year increase of $10.2 million[202]. Cash Flow and Liquidity - As of September 30, 2025, the company had cash and cash equivalents of $344.8 million and outstanding notes payable of $82.5 million under the Barings Credit Facility[214]. - The company raised approximately $445.4 million from the sale of 37,909,018 shares of common stock in October 2025[214]. - Cash provided by financing activities for the nine months ended September 30, 2025, was $112.3 million, a significant decrease from $327.6 million in the same period of 2024[234]. - The company has a total borrowing capacity of $82.5 million under the Barings Credit Facility, which has been fully drawn down[228]. - As of September 30, 2025, cash and cash equivalents totaled $344.8 million, indicating a strong liquidity position despite operational cash outflows[246]. Operating Expenses - Total costs and operating expenses for the three months ended September 30, 2025, were $83.2 million, an increase of $21.8 million from $61.4 million in the same period in 2024[174]. - The company expects to incur significant operating losses as it advances clinical trials for AXPAXLI, including the SOL-1 and SOL-R trials, and initiates new trials[219]. - Net cash used in operating activities for the nine months ended September 30, 2025, was $150.6 million, compared to $95.2 million for the same period in 2024, reflecting an increase in operating expenses to $239.0 million[229][230]. - The net loss for the nine months ended September 30, 2025, was $201.3 million, attributed to increased operating expenses and partially offset by $38.7 million in net revenue[229]. - The company incurred non-cash adjustments of $40.8 million during the nine months ended September 30, 2025, primarily from stock-based compensation[229]. Interest and Other Financial Metrics - Interest income decreased to $3.7 million for the three months ended September 30, 2025, down from $5.7 million in 2024, a decrease of $2.0 million[187]. - Interest income decreased to $11.0 million for the nine months ended September 30, 2025, down from $15.6 million in 2024, a decline of $4.6 million year-over-year[208]. - Interest expense decreased to $9.0 million for the nine months ended September 30, 2025, compared to $10.5 million in 2024, reflecting a decrease of $1.5 million year-over-year[209]. - Total GTN Provisions for the three months ended September 30, 2025, were 52.5% of gross DEXTENZA product sales, up from 39.0% in the same period of 2024[176]. - GTN Provisions for the nine months ended September 30, 2025, were 51.4% of gross DEXTENZA product sales, up from 38.3% in 2024[192].

Financial Performance - Total net revenue for Q3 2025 was $14.5 million, a 5.8% decrease from $15.4 million in Q3 2024[6] - The net loss for Q3 2025 was $(69.4) million, or $(0.38) per share, compared to a net loss of $(36.5) million, or $(0.22) per share, in Q3 2024[11] - Total revenue for the three months ended September 30, 2025, was $14,544 million, a decrease of 5.2% compared to $15,425 million for the same period in 2024[42] - Product revenue, net for the nine months ended September 30, 2025, was $38,573 million, down 16.9% from $46,441 million in 2024[42] - The net loss for the three months ended September 30, 2025, was $69,418 million, compared to a net loss of $36,493 million for the same period in 2024[42] - Net loss per share, basic, for the three months ended September 30, 2025, was $0.38, compared to $0.22 for the same period in 2024[42] - Total costs and operating expenses for the three months ended September 30, 2025, were $83,242 million, an increase of 35.5% from $61,423 million in 2024[42] - Collaboration revenue for the three months ended September 30, 2025, was negligible at $0 million, down from $78 million in 2024[42] - Interest income for the three months ended September 30, 2025, was $3,729 million, a decrease of 34.0% from $5,653 million in 2024[42] - Total other income (expense), net, for the three months ended September 30, 2025, was $(720) million, a significant decrease from $9,505 million in 2024[42] Expenses - Research and development expenses for Q3 2025 were $52.4 million, up from $37.1 million in Q3 2024, reflecting increased clinical trial costs[8] - Selling and marketing expenses rose to $13.1 million in Q3 2025, compared to $10.6 million in Q3 2024, due to expanded marketing efforts for AXPAXLI[9] - General and administrative expenses increased to $16.0 million in Q3 2025 from $12.2 million in Q3 2024, primarily due to higher personnel-related costs[10] - Research and development expenses increased significantly to $52,358 million for the three months ended September 30, 2025, compared to $37,054 million in 2024, reflecting a 41.2% increase[42] Cash and Assets - Cash balance as of September 30, 2025, was $344.8 million, with an additional $445 million in net proceeds from an equity offering completed on October 1, 2025, providing a runway into 2028[3][5] - The company reported total current assets of $386,980,000 as of September 30, 2025, a decrease of 12.2% from $440,987,000 on December 31, 2024[40] - The company has a cash balance of $344,772,000 as of September 30, 2025, down from $392,102,000 on December 31, 2024, representing a decrease of 12.1%[40] Liabilities and Deficit - Total liabilities increased to $152,653,000 as of September 30, 2025, compared to $142,591,000 on December 31, 2024, reflecting a rise of 7.5%[40] - The accumulated deficit grew to $(1,092,369,000) as of September 30, 2025, up from $(891,084,000) on December 31, 2024, indicating a significant increase in losses[40] Clinical Trials and Product Development - The SOL-1 Phase 3 trial for AXPAXLI is on track to report topline data in Q1 2026, with over 95% retention of randomized subjects[4] - The SOL-R Phase 3 trial achieved its target randomization of 555 subjects and is expected to report topline data in 1H 2027[1][4] - The HELIOS registrational program for AXPAXLI in diabetic retinopathy is set to begin imminently, utilizing a novel primary endpoint agreed upon with the FDA[7] - AXPAXLI is currently in Phase 3 clinical trials for wet age-related macular degeneration (wet AMD) and plans to initiate a Phase 3 clinical program for non-proliferative diabetic retinopathy (NPDR) imminently[34] - The primary endpoint of the HELIOS-2 trial is the ordinal DRSS 2-step change status at Week 52 from baseline, with approximately 432 subjects participating[26][25] - The HELIOS-3 trial will involve approximately 930 subjects and is designed as a three-arm study to evaluate the safety and efficacy of AXPAXLI[27] - The SOL-X trial aims to evaluate long-term safety and efficacy of AXPAXLI in wet AMD over a 36-month period[21] Product Sales - DEXTENZA end-user unit sales grew by 9.7% in Q3 2025, with net product revenue increasing by 8.5% compared to the previous quarter[6] - The company’s product DEXTENZA is FDA-approved for treating ocular inflammation and pain, contributing to its revenue stream[35] - The company anticipates potential regulatory submissions and approvals for its product candidates, including AXPAXLI, OTX-TIC, and others[37]

Core Insights - Ocular Therapeutix is advancing its investigational product AXPAXLI™ for retinal diseases, with significant milestones achieved in clinical trials and a strong financial position to support ongoing and future studies [1][2][5] Clinical Development - The SOL-R trial for AXPAXLI in wet age-related macular degeneration (wet AMD) has successfully randomized 555 subjects, with topline data expected in the first half of 2027 [1][3] - The SOL-1 trial, also for wet AMD, is on track to report topline data in the first quarter of 2026, with over 95% retention of randomized subjects [3][4] - The HELIOS program for AXPAXLI in diabetic retinopathy (NPDR) is set to begin imminently, utilizing a novel primary endpoint agreed upon with the FDA [2][7] Financial Performance - As of September 30, 2025, the company reported a cash balance of $344.8 million, supplemented by approximately $445 million from a recent equity offering, providing a financial runway into 2028 [5][11] - Total net revenue for the third quarter of 2025 was $14.5 million, reflecting a 5.8% decrease from the same quarter in 2024, attributed to a challenging reimbursement environment [6][11] - Research and development expenses increased to $52.4 million in Q3 2025, driven by ongoing clinical trials and the SOL-X extension study [7][11] Market Opportunity - The annual market for anti-VEGF therapies is estimated at approximately $15 billion, with potential expansion opportunities in treating millions of undertreated wet AMD patients and the NPDR population, which is three times more prevalent than wet AMD [2][30] - The company aims to address the high discontinuation rates of current treatments, which can reach up to 40% within a year, by offering a more sustainable treatment option with AXPAXLI [30][31] Strategic Initiatives - The company is focused on enhancing its clinical trial designs and patient selection processes to increase the probability of success and align with real-world treatment goals [2][4] - Ocular Therapeutix plans to invest in infrastructure to support increased manufacturing capacity and future growth, leveraging its strong cash position [2][5]