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]. - 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]. - 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 in 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 in 2023, indicating a significant increase in operational losses[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]. - 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]. Cash and Financing - As of September 30, 2024, the company had cash and cash equivalents of $36.6 million, which is expected to fund operations for at least twelve months[209]. - The company has funded operations through equity financings totaling $67.8 million, including $21.15 million from the merger with Rexahn Pharmaceuticals, and received $45.0 million in license fee and milestone payments related to the Viatris License Agreement[178]. - The company entered into a common stock purchase agreement with Lincoln Park, allowing for the purchase of up to $50 million of shares over a 30-month term, with $5.2 million raised through September 30, 2024[211]. - The company has received a total of approximately $67.8 million from various equity offerings and $8.5 million from convertible notes to fund operations[210]. - 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]. Research and Development - The most advanced gene therapy candidate, OPGx-LCA5, is in an ongoing Phase 1/2 clinical trial, with early data showing visual improvement in all three adult patients[166]. - The company’s gene therapy candidate OPGx-BEST1 is planned for a first-in-man clinical trial in 2025, targeting IRDs associated with mutations in the BEST1 gene[168]. - APX3330, an oral small-molecule inhibitor for diabetic retinopathy, is in development, with a Phase 2 trial showing favorable safety and tolerability[175]. - The company expects research and development expenses to increase over the next several years as clinical development progresses, particularly for LCA5, BEST1, and PS[187]. - 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]. - Research and development expenses rose to $19.8 million for the nine months ended September 30, 2024, up from $13.8 million in 2023, reflecting a $6 million increase primarily due to higher manufacturing and toxicology costs for APX3330[205]. Regulatory and Milestones - The FDA approved Phentolamine Ophthalmic Solution 0.75% (RYZUMVI) for pharmacologically-induced mydriasis in September 2023, triggering a $10 million milestone payment under the Viatris License Agreement[170]. - 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 expected[233]. - The company does not expect to generate significant revenue until RYZUMVI sales become material or regulatory approval is obtained for other product candidates[234]. Operational Expenses - Total operating expenses for Q3 2024 were $11.9 million, an increase of $6.3 million compared to $5.5 million in Q3 2023, driven by higher research and development costs[192]. - 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]. - Other income for Q3 2024 was $0.5 million, primarily from interest income related to cash and cash equivalents[200]. - Other income for the nine months ended September 30, 2024 was $1.6 million, primarily from interest income, compared to $1.2 million in 2023[208]. Company Status and Controls - As of September 30, 2024, the company had an accumulated deficit of $103.9 million[179]. - The company is not currently involved in any legal proceedings that are likely to materially affect its business or financial results[260]. - 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].

Financial Performance - Net loss for the quarter ended September 30, 2024, was $7.5 million, or $(0.29) per basic and diluted share, compared to net income of $5.6 million, or $0.26 per share, in Q3 2023[13]. - Net loss for September 2024 was $7,526,000, compared to a net income of $5,561,000 in September 2023[24]. - Basic net loss per share for September 2024 was $0.29, compared to earnings of $0.26 per share in September 2023[24]. - The accumulated deficit increased to $(103,863,000) as of September 30, 2024, compared to $(81,466,000) at the end of December 2023[23]. Revenue and Expenses - License and collaborations revenue for the three months ended September 30, 2024, was $3.9 million, a decrease from $11.9 million in the same period of 2023, primarily due to a one-time $10 million milestone in 2023[9]. - License and collaborations revenue for September 2024 was $3,867,000, a decrease of 67.7% compared to $11,935,000 in September 2023[24]. - Total operating expenses increased to $11,876,000 in September 2024, up 114.5% from $5,549,000 in September 2023[24]. - General and administrative expenses for the three months ended September 30, 2024, were $2.9 million, up from $2.1 million in the same period of 2023[11]. - Research and development expenses for the three months ended September 30, 2024, were $9.0 million, compared to $3.5 million for the same period in 2023, reflecting increased clinical costs and other R&D activities[12]. - Research and development expenses rose to $8,982,000 in September 2024, a significant increase of 157.5% from $3,494,000 in September 2023[24]. Assets and Liabilities - Total liabilities as of September 30, 2024, were $6,089,000, an increase of 50.5% from $4,042,000 at the end of December 2023[22]. - Total current liabilities increased to $6,089,000 as of September 30, 2024, compared to $4,042,000 at the end of December 2023[22]. - Total stockholders' equity decreased to $34,300,000 as of September 30, 2024, down 31.2% from $49,906,000 at the end of December 2023[23]. Corporate Developments - The acquisition of Opus Genetics was completed on October 22, 2024, with the issuance of 5,237,063 shares of common stock and 14,145.374 shares of Series A Preferred Stock[3]. - The combined company has an expanded pipeline that includes seven assets from the AAV-based gene therapy portfolio[4]. - The company anticipates filing a Clinical Trial Authorization for OPGx-BEST1 in Germany in 2025[6]. - Four clinical data readouts are expected in 2025, including Phase 3 studies for Phentolamine Ophthalmic Solution 0.75%[2]. Clinical Trials - The most advanced gene therapy candidate, OPGx-LCA5, has shown visual improvement in three out of three adult patients in a Phase 1/2 trial, with pediatric enrollment expected in Q1 2025[5].