Core Viewpoint - Ocuphire Pharma, Inc. has announced an all-stock acquisition of Opus Genetics, creating a biotech company focused on gene therapies for inherited retinal diseases (IRDs) [1] Company Overview - The combined entity will be renamed Opus Genetics, effective October 23, 2024, and will trade on Nasdaq under the ticker "IRD" starting October 24, 2024 [1] - The merger expands the pipeline to include multiple assets from an adeno-associated virus (AAV)-based gene therapy portfolio for IRDs and Phentolamine Ophthalmic Solution 0.75% for presbyopia and dim light vision disturbances [1] Clinical Development - Ocuphire will seek a partner to advance the clinical development of APX3330 for non-proliferative diabetic retinopathy, redirecting resources to the acquired gene therapy programs [2] - The most advanced gene therapy candidate, OPGx-LCA5, is in an ongoing open-label, dose-escalation Phase 1/2 trial for LCA5, showing early clinical proof-of-concept with visual improvement in all three adult patients [2] - Enrollment of the first pediatric patients in the Phase 1/2 trial is expected in Q1 2025, with initial data anticipated in the same quarter [2] - The expected cash runway for the combined company has been extended into 2026 [2] Share Issuance and Ownership - Ocuphire issued 5.2 million shares and 14.1 thousand shares of convertible preferred stock, resulting in Ocuphire owning approximately 58% and Opus Genetics owning approximately 42% of the combined company [3] - Following the announcement, OCUP stock experienced a decline of 13.5%, trading at $1.15 [3]

Core Insights - The acquisition of Opus Genetics by Ocuphire Pharma creates a leading clinical-stage company focused on gene therapy treatments for rare inherited retinal degenerations [1][2] - The combined company will be renamed Opus Genetics, Inc. and will trade under the ticker symbol "IRD" on Nasdaq starting October 24, 2024 [1][2] - The merger is expected to accelerate the development of a promising pipeline of gene therapies, with significant clinical milestones anticipated in 2025 [2][6] Company Overview - Ocuphire Pharma is a clinical-stage ophthalmic biopharmaceutical company developing therapies for retinal and refractive disorders, including adeno-associated virus (AAV)-based gene therapies [9] - The most advanced gene therapy candidate, OPGx-LCA5, is in a Phase 1/2 trial and has shown visual improvement in all three adult patients with late-stage disease [3][4] - The combined company will focus on multiple assets, including Phentolamine Ophthalmic Solution 0.75% for presbyopia and dim light disturbances [2][9] Clinical Development - The OPGx-LCA5 Phase 1/2 trial is ongoing, with pediatric patient enrollment expected in Q1 2025 and initial data anticipated in Q3 2025 [4][6] - The LYNX-2 Phase 3 trial for dim light disturbances and the VEGA-3 Phase 3 trial for presbyopia are on track for top-line data in Q1 and H1 2025, respectively [1][6] - The company plans to seek a strategic partner for the development of APX3330, an oral small-molecule inhibitor for non-proliferative diabetic retinopathy [1][2] Financial Aspects - The acquisition involved the issuance of 5.2 million shares of common stock and 14.1 thousand shares of convertible preferred stock to Opus Genetics' stockholders [7] - Following the acquisition, pre-acquisition stockholders of Ocuphire will own approximately 58% and those of Opus Genetics will own about 42% of the combined company's fully diluted capitalization [7] - The expected cash runway for the combined company has been extended into 2026, allowing for continued clinical development [6]

Core Insights - Ocuphire Pharma, Inc. announced the publication of full results from two pivotal Phase 3 clinical trials (MIRA-2 and MIRA-3) for RYZUMVI™ (Phentolamine Ophthalmic Solution 0.75%) demonstrating its efficacy in reversing pharmacologically-induced mydriasis with a favorable safety profile [1][4][10] Group 1: Clinical Trial Results - In the MIRA-2 trial, 48.9% of subjects treated with Phentolamine achieved reversal of mydriasis at 90 minutes compared to 6.6% in the placebo group (p<0.0001) [2] - In the MIRA-3 trial, 58% of subjects treated with Phentolamine achieved the primary endpoint versus 6% in the placebo group (p<0.0001) [2] - Significant improvements in pupil diameter were observed as early as 60 minutes post-administration, with effects lasting up to 24 hours [2][3] Group 2: Safety Profile - The most common treatment-emergent adverse events for Phentolamine were mild and transient, including conjunctival hyperemia (11.2%), instillation site discomfort (10.9%), and dysgeusia (3.6%) [3] - Fewer subjects treated with Phentolamine (8-11%) had residual dilation at 24 hours compared to 28-34% in the placebo group (p<0.0001) [3] Group 3: Future Developments - Phentolamine Ophthalmic Solution 0.75% is being evaluated in ongoing Phase 3 trials for presbyopia (VEGA-3) and dim light vision disturbances (LYNX-2), with top-line data expected in the first half of 2025 [5][10] - The VEGA-3 trial is actively enrolling participants, and the LYNX-2 trial is also in progress [5][10] Group 4: Company Overview - Ocuphire Pharma is focused on developing small molecule therapies for retinal and refractive eye disorders, with RYZUMVI™ being a key late-stage product candidate [1][10] - The company is responsible for managing the ongoing clinical trials and has a partnership for the commercialization of Phentolamine [9][10]

Core Insights - Ocuphire Pharma, Inc. is advancing its clinical trials for Phentolamine Ophthalmic Solution 0.75%, targeting presbyopia and visual disturbances under low light conditions [1][3][6] - The VEGA-3 Phase 3 trial has commenced, with top-line data expected in the first half of 2025, while the LYNX-2 Phase 3 trial is also ongoing, with data anticipated in the first quarter of 2025 [1][4][5] Company Overview - Ocuphire Pharma is a clinical-stage ophthalmic biopharmaceutical company focused on developing therapies for retinal and refractive eye disorders [11] - The company's lead product candidate, APX3330, is in development for diabetic retinopathy, alongside Phentolamine Ophthalmic Solution 0.75% for presbyopia and dim light vision disturbances [11] Product Development - Phentolamine Ophthalmic Solution 0.75% is designed as a non-invasive alternative to traditional corrective measures for presbyopia, which affects a significant portion of the aging population [2][9] - The solution works by reducing pupil size without the risks associated with other treatments, potentially improving visual function in low light conditions [6][8] Clinical Trials - The VEGA-3 trial involves 545 participants and aims to evaluate the efficacy of Phentolamine in improving near visual acuity [4] - The LYNX-2 trial is focused on participants who have undergone keratorefractive surgery and are experiencing visual disturbances, with a target enrollment of 200 participants [5] Market Potential - Presbyopia affects an estimated 128 million Americans and over 2 billion people globally, indicating a substantial market opportunity for effective treatments [9] - Currently, there are no FDA-approved treatments for visual disturbances under low light conditions, positioning Phentolamine as a potential first-in-class option [6]

Financial Performance - Ocuphire Pharma reported a net loss of $7.8 million and $14.9 million for the three and six months ended June 30, 2024, respectively, compared to a net loss of $5.0 million and $10.8 million for the same periods in 2023[136]. - The company has an accumulated deficit of $96.3 million as of June 30, 2024[136]. - License and collaborations revenue decreased to $1.1 million for the three months ended June 30, 2024, down from $3.7 million in the same period of 2023, primarily due to reduced PS research and development services[150]. - License and collaborations revenue for the six months ended June 30, 2024 was $2.8 million, down from $5.4 million in the same period of 2023, reflecting a decrease in PS research and development services[157]. - General and administrative expenses for the three months ended June 30, 2024 were $3.4 million, a decrease of $1.0 million compared to $4.3 million in the prior year, mainly due to reduced payroll-related costs[151]. - General and administrative expenses for the six months ended June 30, 2024 were $8.0 million, an increase of $1.4 million compared to $6.6 million in the prior year[159]. - Research and development expenses increased to $6.1 million for the three months ended June 30, 2024, compared to $4.7 million in the same period of 2023, driven by higher manufacturing and toxicology costs for APX3330[154]. - Research and development expenses for the six months ended June 30, 2024 were $10.8 million, slightly up from $10.3 million in the same period of 2023, primarily due to increased manufacturing costs for APX3330[160]. - Total operating expenses for the three months ended June 30, 2024 were $9.4 million, slightly higher than $9.1 million in the same period of 2023[150]. - Other income, net for the three months ended June 30, 2024 was $0.6 million, compared to $0.4 million in the same period of 2023, mainly from interest income[155]. - Other income, net for the six months ended June 30, 2024 was $1.2 million, up from $0.8 million in the same period of 2023, primarily due to interest income[161]. - For the six months ended June 30, 2024, the company reported a net cash used in operating activities of $13.0 million, attributed to a net loss of $14.9 million[176]. Product Development - Ocuphire's lead product candidate, APX3330, is in development for diabetic retinopathy, which affects approximately 10 million diabetics and is projected to impact over 14 million Americans by 2050[127]. - The FDA approved Phentolamine Ophthalmic Solution 0.75% (RYZUMVI) in September 2023, triggering a $10 million milestone payment under the Viatris License Agreement[131]. - RYZUMVI was commercialized by Viatris in April 2024, following a comprehensive clinical trial program involving over 600 subjects[131]. - The VEGA-2 Phase 3 study for presbyopia achieved its primary endpoint, and the VEGA-3 Phase 3 clinical trial has begun recruiting[134]. - Ocuphire has in-licensed APX2009 and APX2014, which are second-generation analogs of APX3330, targeting other retinal diseases[129]. - The company anticipates recognizing revenue from the Viatris License Agreement and potential future milestone and royalty payments, but significant revenue generation is uncertain until product sales materialize[179]. - The development of APX3330 is subject to uncertainties, and the company cannot assure future profitability or positive cash flow from operating activities[183]. Funding and Capital Structure - Ocuphire has funded operations primarily through equity financings totaling $67.3 million, including $21.15 million from a merger with Rexahn Pharmaceuticals[136]. - The company expects to finance its cash needs through a combination of equity and debt financings, as well as collaborations and licensing arrangements[135]. - The company raised approximately $67.3 million from various equity offerings and $8.5 million from convertible notes, including a one-time non-refundable payment of $35 million and a $10 million milestone payment related to the Viatris License Agreement[163]. - The company entered into a Purchase Agreement with Lincoln Park Capital Fund, allowing for the purchase of up to $50 million of common stock over a 30-month term[164]. - The company has a shelf registration effective for an aggregate offering price of up to $175 million, with $26 million raised through an At-The-Market program since inception[165]. - The company may face dilution of stockholder ownership if additional capital is raised through equity or convertible debt securities[182]. - The company expects to satisfy short-term and long-term obligations through cash on hand, future equity and debt financings, and reimbursement payments[191]. Legal and Regulatory Matters - The company is not currently a party to any legal proceedings that are likely to materially affect its business or financial results[204]. - The company has a full valuation allowance on net deferred tax assets due to uncertainty of future taxable income[196]. - The company has not triggered any milestone or royalty payments as of the date of the report[187]. Miscellaneous - As of June 30, 2024, the company had cash and cash equivalents of $41.4 million, sufficient to fund operations for at least twelve months[162]. - As of June 30, 2024, the company had 5,665,838 Series A Warrants outstanding, with an initial exercise price of $4.4795 per share[172]. - The company has no significant cash flows from investing activities during the reported periods[178]. - Research and development expenses related to the development of PS are fully reimbursed by Viatris[183]. - The company has potential milestone payments totaling up to $11 million and sales milestone payments of up to $20 million under the Apexian Sublicense Agreement[186]. - The company has a non-cancellable operating lease with a base rent of $3,000 per month expiring on December 31, 2024[185]. - The company recognizes license and collaborations revenue based on the stand-alone price of each performance obligation[194].

Company Performance - Ocuphire Pharma reported a quarterly loss of $0.30 per share, slightly worse than the Zacks Consensus Estimate of a loss of $0.29, and compared to a loss of $0.24 per share a year ago, indicating a decline in performance [1] - The company posted revenues of $1.11 million for the quarter ended June 2024, missing the Zacks Consensus Estimate by 38.22%, and down from $3.67 million in the same quarter last year [2] - Over the last four quarters, Ocuphire Pharma has surpassed consensus EPS estimates only once and has topped consensus revenue estimates just once [2] Stock Performance - Ocuphire Pharma shares have lost approximately 45% since the beginning of the year, contrasting with the S&P 500's gain of 12.1% [3] - The current consensus EPS estimate for the upcoming quarter is -$0.29 on revenues of $1.89 million, and for the current fiscal year, it is -$1.16 on revenues of $7.34 million [7] Industry Outlook - The Medical - Biomedical and Genetics industry, to which Ocuphire Pharma belongs, is currently ranked in the top 29% of over 250 Zacks industries, suggesting a favorable industry outlook [8] - Empirical research indicates a strong correlation between near-term stock movements and trends in earnings estimate revisions, which can impact Ocuphire Pharma's stock performance [5]

Diabetic retinopathy (DR) affects approximately 10 million of the 38 million Americans with diabetes and is the leading cause of blindness in working-age adults. DR is projected to impact over 14 million Americans by 2050. DR is classified as either non-proliferative DR (NPDR), the early stage of the disease in which symptoms may be mild or non-existent, and proliferative (PDR), the more advanced stage of diabetic eye disease that can be highly symptomatic and is associated with loss of vision. Approximatel ...

CEO to participate in ARVO SIG panel on oral medications for retinal diseases FARMINGTON HILLS, Mich., July 10, 2024 (GLOBE NEWSWIRE) -- Ocuphire Pharma, Inc. (Nasdaq: OCUP), a clinical-stage ophthalmic biopharmaceutical company focused on developing and commercializing small-molecule therapies for the treatment of patients with retinal and refractive eye disorders, today announced it will participate in the Association for Research in Vision and Ophthalmology Special Interest Group (ARVO SIG) panel and the ...

FARMINGTON HILLS, Mich., June 06, 2024 (GLOBE NEWSWIRE) -- Ocuphire Pharma, Inc. (Nasdaq: OCUP) ("Ocuphire"), a clinical-stage biopharmaceutical company focused on developing small molecule therapies for the treatment of patients with retinal and refractive eye disorders, today announced that clinical updates on the Company's lead candidate APX3330 for diabetic retinopathy (DR) will be featured in upcoming presentations at the Clinical Trials at the Summit meeting on June 8 in Park City, Utah, and the Retin ...

FARMINGTON HILLS, Mich., June 06, 2024 (GLOBE NEWSWIRE) -- Ocuphire Pharma, Inc. (Nasdaq: OCUP) ("Ocuphire"), a clinical-stage biopharmaceutical company focused on developing small molecule therapies for the treatment of patients with retinal and refractive eye disorders, today announced that clinical updates on the Company's lead candidate APX3330 for diabetic retinopathy (DR) will be featured in upcoming presentations at the Clinical Trials at the Summit meeting on June 8 in Park City, Utah, and the Retin ...