UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 Form 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Quarterly Period Ended March 31, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF SECURITIES EXCHANGE ACT OF 1934 For the transition period from to ________ Commission File Number: 001-34079 Opus Genetics, Inc. (Exact name of Registrant as specified in its charter) | Delaware | 11-3516358 | | --- | --- | | ( ...

Exhibit 99.1 Opus Genetics Announces Financial Results for First Quarter 2025 and Provides Corporate Update ARVO presentation highlights 12-month results from Phase 1/2 study that support potential of OPGx-LCA5 gene therapy to restore meaningful vision Pediatric cohort of LCA5 study ongoing with initial multi-patient data anticipated in Q3 2025 OPGx-BEST1 on track for IND filing and initiation of Phase 1/2 trial with early data expected in Q1 2026 Leading healthcare investors provide funding to deliver on k ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K ☒ Annual report pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934. For the Fiscal Year Ended December 31, 2024 or ☐ Transition report pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934. For the transition period from _____ to _____ Commission File No. 001-34079 Opus Genetics, Inc. (Exact name of registrant as specified in its charter) Delaware 11-3516358 (State or other jurisdiction of ...

Financial Performance - The company reported a net loss of approximately $56.8 million for the year ended December 31, 2024[6] - Net cash used in operating activities for the year ended December 31, 2024 was approximately $25.6 million[6] Cash Position - The cash and cash equivalents balance as of December 31, 2024 was approximately $30.3 million[6]

Financial Performance - The company reported a net loss of $7.5 million for Q3 2024, compared to a net income of $5.6 million in Q3 2023, and a net loss of $22.4 million for the nine months ended September 30, 2024, compared to a net loss of $5.2 million for the same period in 2023[179]. - The net loss for Q3 2024 was $7.5 million, compared to a net income of $5.6 million in Q3 2023, reflecting a significant year-over-year change of $13.1 million[192]. - For the nine months ended September 30, 2024, license and collaborations revenue was $6.7 million, down $10.7 million from $17.4 million in the same period of 2023[201]. - The loss from operations for the nine months ended September 30, 2024 was $24.0 million, compared to a loss of $5.1 million in the same period of 2023, indicating a deterioration of $18.9 million[201]. - Cash used in operating activities for the nine months ended September 30, 2024 was $18.1 million, attributed to a net loss of $22.4 million, partially offset by $2.6 million in non-cash operating expenses[229]. - The company reported a net decrease in cash and cash equivalents of $13.9 million for the nine months ended September 30, 2024, compared to a decrease of $284,000 in 2023[227]. - For the nine months ended September 30, 2023, the company reported a net loss of $5.2 million, with cash used in operating activities amounting to $5.7 million[230]. Revenue and Expenses - License and collaborations revenue for Q3 2024 was $3.9 million, a decrease of $8.1 million from $11.9 million in Q3 2023, primarily due to a one-time $10.0 million milestone payment in the prior year[192]. - Total operating expenses for Q3 2024 were $11.9 million, an increase of $6.3 million from $5.5 million in Q3 2023, driven by higher research and development costs[192]. - Research and development expenses for Q3 2024 were $9.0 million, up $5.5 million from $3.5 million in Q3 2023, mainly due to increased clinical costs and drug manufacturing expenses[197]. - General and administrative expenses for Q3 2024 were $2.9 million, an increase of $0.8 million from $2.1 million in Q3 2023, attributed to personnel-related costs and business development expenses[193]. - Total operating expenses for the nine months ended September 30, 2024 were $30.7 million, an increase of $8.2 million from $22.5 million in the same period of 2023[201]. - Research and development expenses for the nine months ended September 30, 2024 were $19.8 million, an increase of $6.0 million from $13.8 million in the same period of 2023[201]. - General and administrative expenses increased to $10.9 million for the nine months ended September 30, 2024, compared to $8.7 million in 2023, with a $2.2 million increase attributed mainly to legal support and business development activities[204]. Cash and Financing - As of September 30, 2024, cash and cash equivalents totaled $36.6 million, sufficient to fund operations for at least twelve months beyond the filing date[209]. - The company entered into a common stock purchase agreement with Lincoln Park for an equity line financing of up to $50 million over 30 months, with $5.2 million raised through September 30, 2024[211]. - The company has raised approximately $67.8 million through various equity offerings and $8.5 million through convertible notes to fund operations[210]. - The company expects to satisfy its cash requirements through cash on hand, future equity and debt financings, and reimbursement payments until it generates adequate revenue from commercial sales[248]. Clinical Development - The company’s most advanced gene therapy candidate, OPGx-LCA5, is in a Phase 1/2 clinical trial, with early data showing visual improvement in all three adult patients[166]. - Enrollment of the first pediatric patients in the LCA5 Phase 1/2 trial is expected in Q1 2025, with initial data anticipated in Q3 2025[167]. - The company plans to seek a strategic partner for the late-stage diabetic retinopathy program APX3330 due to capital requirements and developmental timelines[165]. - The company anticipates ongoing expenses related to clinical work for LCA5, BEST1, and other product candidates, as well as regulatory approvals and manufacturing contracts[179]. - The company expects research and development expenses to increase in the coming years due to higher costs associated with later-stage clinical trials[187]. Regulatory and Commercialization - The company received a $10 million milestone payment under the Viatris License Agreement following FDA approval of RYZUMVI for pharmacologically-induced mydriasis in September 2023[170]. - RYZUMVI was commercialized by Viatris in April 2024, following its approval[170]. - The company aims to establish a sales, marketing, and distribution infrastructure for future commercialization efforts[179]. - The company does not expect to generate significant revenue until RYZUMVI sales become material or regulatory approval is obtained for other product candidates[234]. - The company anticipates recognizing revenue from reimbursement for research and development services under the Viatris License Agreement, with a total of $35 million in one-time non-refundable payments and a $10 million milestone payment received to date[233]. Internal Controls and Legal Matters - As of September 30, 2024, the company's disclosure controls and procedures were evaluated as effective by its principal executive officer and principal financial officer[258]. - There were no changes in the company's internal control over financial reporting during the quarter ended September 30, 2024, that materially affected its internal control[259]. - The company is not currently involved in any legal proceedings that are likely to materially affect its business or financial results[260].

Merger Agreement Details - The merger agreement involves OCUPHIRE PHARMA, INC. and OPUS GENETICS INC., with the effective date set for October 22, 2024[1]. - The first merger will result in OPUS GENETICS INC. becoming a wholly owned subsidiary of OCUPHIRE PHARMA, INC.[11]. - Following the first merger, OPUS GENETICS INC. will merge into ORANGE MERGER SUB II, LLC, with the latter being the surviving entity[12]. - The transactions are intended to qualify as a "reorganization" under Section 368(a) of the Code[13]. - The Parent Board has unanimously determined that the contemplated transactions are fair and in the best interests of its stockholders[14]. - The aggregate merger consideration to be paid by Parent for all outstanding shares of Company Capital Stock at Closing will include 5,237,063 shares of Parent Common Stock and 14,145.374 shares of Parent Convertible Preferred Stock, representing 42% of the outstanding capital stock of Parent immediately following the Merger[30]. - The First Merger will result in the Company continuing as the surviving corporation, and the Second Merger will lead to the formation of a new entity named "OPUSTX LLC" as the surviving entity[24][29]. - The First Merger is set to be consummated remotely via electronic exchange of documents on the Closing Date, with the Second Merger to occur no earlier than the tenth Business Day after the Closing Date[26]. - All directors and officers of Parent and certain Company stockholders are executing lock-up agreements and support agreements to facilitate the merger[20][21]. - The Company stockholders have irrevocably approved the merger and waived any rights to receive payment of the fair value of their capital stock under the Delaware General Corporation Law[18]. - The merger will result in the issuance of shares of Parent Common Stock and Parent Convertible Preferred Stock to Company stockholders, with each Parent Preferred Stock Payment Share convertible into 1,000 shares of Parent Common Stock[30]. - The First Effective Time will occur upon the filing of the First Certificate of Merger with the Secretary of State of Delaware, while the Second Effective Time will follow the filing of the Second Certificate of Merger[26][29]. - The certificate of incorporation of Parent will be amended to change its name to "Opus Genetics, Inc." following the First Effective Time[27]. - The merger is structured to ensure that no fractional shares of Parent Common Stock will be issued, with cash payments made for any fractional shares[33]. - The Company will transmit a Stockholder Notice to each stockholder who did not execute the Stockholder Written Consent immediately following the execution of the Agreement[19]. - At the First Effective Time, each share of common stock of First Merger Sub will be converted into one share of common stock of the First Step Surviving Corporation[34]. - The Preferred Stock Exchange Ratio will be adjusted to reflect any changes in outstanding shares due to stock dividends or other similar changes[35]. - At the Second Effective Time, each share of common stock of the First Step Surviving Corporation will be canceled without any conversion or payment[36]. - All holders of Company Capital Stock will cease to have rights as stockholders immediately prior to the First Effective Time[37]. - The Exchange Fund will include Parent Common Stock, Parent Convertible Preferred Stock, and cash for fractional shares[38]. - Dissenting Shares will not convert into the Merger Consideration but will receive the appraised value under the DGCL[44]. - Company Options will be canceled without payment at the First Effective Time[46]. - Company Convertible Notes will be converted into 6,223,538.51 shares of Series Seed-1 Preferred Stock at the First Effective Time[47]. Company Financials and Operations - The Company is duly incorporated and has all necessary corporate power to conduct its business[51]. - The Company has no subsidiaries and does not own any capital stock or equity interests in other entities[53]. - The Company has 77,341,000 authorized shares of Common Stock, with 6,548,709 shares issued and outstanding[63]. - The Company has 65,236,000 authorized shares of Preferred Stock, with 56,053,000 designated as Series Seed Preferred Stock and 33,382,450 shares issued and outstanding[63]. - The Company maintains accurate books and records reflecting its assets and liabilities, ensuring reasonable assurance over financial reporting[73]. - The Company has reserved 5,576,465 shares of Common Stock for issuance under the 2021 Stock Option Plan, with 21,500 shares already issued[66]. - The Company has not declared or paid any dividends or repurchased any shares of its capital stock since the date of the Unaudited Interim Balance Sheet[76]. - The Company has not identified any significant deficiencies or material weaknesses in its internal accounting controls since inception[75]. - The Company has not conducted any mergers, consolidations, or similar transactions except for the Contemplated Transactions[76]. - The Company has not incurred or guaranteed any indebtedness for borrowed money since the date of the Unaudited Interim Balance Sheet[76]. - The Company Financials fairly present the financial position and operating results as of the specified dates[72]. - The Company does not hold any shares of its capital stock in its treasury[63]. - The Company has no undisclosed liabilities as of the date hereof, except for those disclosed in the Unaudited Interim Balance Sheet[81]. - The Company owns all tangible properties and assets used in its business, free and clear of any encumbrances, except for permitted encumbrances[82]. - The Company does not own any real property and has valid leasehold interests in all leased properties[83]. - The Company has identified all material registered intellectual property (IP) and has no pending or threatened challenges to its validity[85]. - The Company exclusively owns or has exclusively licensed all material IP necessary for its business operations[86]. - The Company has not experienced any data breaches or security violations related to sensitive data since its inception[92]. - The Company has a list of material contracts in effect, including those requiring payments exceeding $50,000[94]. - The Company has not breached any material contracts that would allow other parties to cancel or seek damages, ensuring all material contracts are valid and enforceable[98]. - The Company holds all required Governmental Authorizations necessary for its operations, and no permits have been revoked or suspended[101]. - There are no pending or threatened investigations against the Company regarding compliance with drug regulations, indicating a strong legal standing[103]. - All clinical studies conducted by the Company have adhered to applicable regulations, with no studies terminated for safety or noncompliance reasons[105]. - The Company has complied with all laws related to patient health information, including HIPAA, and has not received any allegations of noncompliance[109]. - Manufacturing operations for product candidates have been conducted in accordance with good manufacturing practices, with no significant compliance issues reported[111]. - The Company has made available all material correspondence with regulatory bodies, ensuring transparency in its compliance efforts[112]. - As of the date of the Agreement, there are no pending legal proceedings that could interfere with the contemplated transactions[113]. - The Company has timely filed all required Tax Returns and all such returns are correct and complete in all material respects[117]. - All amounts of Taxes due and owing by the Company have been fully and timely paid, and unpaid Taxes did not materially exceed the reserve for Tax liability[118]. - There are no pending or ongoing audits, assessments, or other actions for any material amount of Taxes of the Company[120]. - The Company has not incurred any Liability for Taxes outside the Ordinary Course of Business since the date of the Unaudited Interim Balance Sheet[118]. - The Company has not participated in any transaction that constitutes a "listed transaction" under the Code[127]. Employee and Labor Compliance - The Company has provided a true and correct list of all current employees and independent contractors, including compensation details[144]. - The Company is in compliance with all applicable Laws regarding labor and employment practices[147]. - There are no pending audits or investigations involving any Company Benefit Plan[137]. - The Company has not been a party to any collective bargaining agreement or similar labor organization[145]. - The Company has not made any election under Section 965(h) of the Code[123]. - The Company has not implemented any "plant closing" or "mass layoff" since its inception, ensuring compliance with the WARN Act[148]. - The Company has been in material compliance with all COVID-19-related laws and regulations, including those from the Occupational Safety and Health Administration and the Centers for Disease Control[148]. Environmental and Regulatory Compliance - The Company has complied with all applicable Environmental Laws and has not received any notices of non-compliance since its inception[149]. - The Company has delivered accurate and complete copies of all material insurance policies, and no cancellations or denials of coverage have been reported since its inception[151]. - There are no undisclosed transactions with affiliates that would require disclosure under Item 404 of Regulation S-K[153]. - The Company has provided all material environmental reports and assessments to the Parent prior to the date of the Agreement[149]. - The Company has not been subject to any investigations regarding potential violations of Anti-Bribery Laws[155]. Parent Company Financials - Each holder of Company Preferred Stock is an accredited investor as defined in Regulation D[157]. - The Company has made no representations or warranties outside of those expressly set forth in the Agreement[158]. - The Company has obtained all necessary corporate approvals for the execution and delivery of the Agreement[168]. - As of the Reference Date, Parent has 125,000,000 authorized shares of common stock, with 26,198,444 shares issued and outstanding[174]. - Parent has 10,000,000 authorized shares of preferred stock, none of which have been issued[174]. - Parent has outstanding options for the purchase of 4,759,037 shares and warrants for 7,204,299 shares of common stock[177][178]. - Parent's financial statements comply with GAAP and fairly present its consolidated financial position as of the respective dates[184]. - Parent has maintained a system of internal control over financial reporting designed to provide reasonable assurance regarding the reliability of financial reporting[190]. - As of the date of the Agreement, there are no unresolved comments from the SEC regarding Parent's SEC Documents[187]. - Parent has not received any comment letters from the SEC regarding delisting or maintenance of listing on Nasdaq since December 31, 2022[187]. - Parent's cash and cash equivalents as of the close of business on the Business Day preceding the date of the Agreement have not materially changed[193]. - Parent has not been classified as a "shell company" under Section 12b-2 of the Exchange Act[194]. - All distributions and repurchases of Parent Common Stock have been undertaken in compliance with applicable laws and regulations[181]. - Parent and its Subsidiaries have conducted business only in the Ordinary Course of Business since the date of the Parent Balance Sheet[195]. - No dividends were declared or paid, and no shares were repurchased or reacquired, except for stock plan exercises[195]. - Parent has not incurred or guaranteed any indebtedness for borrowed money since the date of the Parent Balance Sheet[197]. - No material transactions outside the Ordinary Course of Business have been entered into, except in connection with the Contemplated Transactions[197]. - Parent has not made any expenditures exceeding $100,000 individually or $300,000 in the aggregate[199]. - As of the date hereof, Parent has no undisclosed liabilities that would be material to the Parent[200]. - Parent has not obtained a loan under the Paycheck Protection Program under the CARES Act[200].

Core Insights - The RD Fund has announced the all-stock acquisition of Opus Genetics, Inc. by Ocuphire Pharma, creating a new entity named Opus Genetics focused on gene therapies for inherited retinal diseases (IRDs) [1][3] - The combined company will trade on Nasdaq under the ticker symbol "IRD" [1] - This acquisition marks the first RD Fund portfolio company to enter public markets, validating the venture philanthropy model [3] Company Overview - Opus Genetics was launched in 2021 by the RD Fund to develop gene therapies for IRDs, with initial seed financing from the RD Fund [1][2] - The RD Fund aims to accelerate the approval of therapies for retinal diseases and provide alternative revenue sources for the Foundation Fighting Blindness [4] Financial and Development Highlights - Opus has developed a pipeline of early-stage preclinical assets, including advancing OPGx-LCA5 into a Phase 1/2 trial, which showed safety and visual improvement in early onset retinal degeneration [2] - The RD Fund has raised over $915 million to support research for preventing and treating retinal degenerative diseases [5]

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]