HTRA1

PALO ALTO, Calif., April 21, 2023 /PRNewswire/ -- Kodiak Sciences Inc. (Nasdaq: KOD), a biopharmaceutical company committed to researching, developing and commercializing transformative therapeutics to treat high prevalence retinal diseases, announced today that multiple scientific presentations on its clinical and research pipeline programs will be made at the Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting, being held from April 23 – 27 in New Orleans, United States. "We look forward to sharing data across our pipeline of investigational medicines at this year's ARVO Annual Meeting," said Victor Perlroth, M.D., Chief Executive Officer of Kodiak Sciences. "Our efforts encompass Phase 3 clinical studies with our lead tarcocimab tedromer, a Phase 1 clinical study with our new bispecific KSI-501, and earlier discovery efforts. We believe the data being presented will demonstrate the robust retina-focused research and development engine at Kodiak. We remain committed to our mission of bringing new science to the prevention and treatment of high prevalence retinal diseases." The following data will be presented at the meeting: Presentation on tarcocimab tedromer (KSI-301): Abstract Title: Nonclinical Pharmacokinetics, Distribution and Excretion of 125I-KSI-301 after Intravenous Administration in Rats Session Title: Retina / RPE: New drugs, mechanisms of action and toxicity Session Date and Time: April 25, 2023; 8:45 – 10:45 AM CT Presentation Type: Poster Session Poster Number: 2606 - B0319 Presentations on KSI-501: Abstract Title: KSI-501 is a novel anti-VEGF and anti-IL-6 bispecific biopolymer conjugate to simultaneously address neovascularization and inflammation in retinal diseases Session Title: AMD New drugs, delivery systems and mechanisms of action 2 Session Date and Time: April 23, 2023; 3:45 – 5:45 PM CT Presentation Type: Poster Session Poster Number: 1153 - C0308 Abstract Title: Biological benefits of KSI-501: Novel bispecific anti-inflammatory and anti-angiogenic therapy for the treatment of both retinal vascular and inflammatory diseases Session Title: AMD anti-VEGF Session Date and Time: April 24, 2023; 3:15 – 5:00 PM CT Presentation Type: Poster Session Poster Number: 2215 - C0168 Presentations on research pipeline: Abstract Title: Development and characterization of an anti-HTRA1 antibody for dry AMD treatment Session Title: AMD New drugs, delivery systems and mechanisms of action 2 Presentation Date and Time: April 23, 2023; 3:45 – 5:45 PM CT Presentation Type: Poster Session Poster Number: 1151 - C0306 Abstract Title: Development of a modular IL-1 trap and anti-HTRA1 bispecific for the treatment of dry AMD Session Title: AMD New drugs, delivery systems and mechanisms of action 2 Presentation Date and Time: April 23, 2023; 3:45 – 5:45 PM CT Presentation Type: Poster Session Poster Number: 1152 - C0307 About tarcocimab tedromer (tarcocimab, KSI-301) Tarcocimab is an investigational anti-VEGF therapy built on Kodiak's Antibody Biopolymer Conjugate ("ABC") Platform and is designed to maintain potent and effective drug levels in ocular tissues for longer than existing available agents. Kodiak's objective with tarcocimab is to enable earlier treatment and prevention of vision loss for patients with diabetic eye diseases and to develop a new first-line agent to improve outcomes for patients with retinal vascular diseases as a whole. The tarcocimab clinical program is designed to explore 6-month durability in the majority of patients with diabetic eye disease through the GLEAM and GLIMMER Phase 3 studies in diabetic macular edema ("DME") and the GLOW Phase 3 study in non-proliferative diabetic retinopathy ("NPDR") without DME. The tarcocimab clinical program is also exploring the product's durability, efficacy and safety in retinal vein occlusion ("RVO") via the BEACON Phase 3 study and in wet age-related macular degeneration ("wet AMD") via the on-going DAYLIGHT Phase 3 study. The BEACON study met its primary endpoint in 2022, and four Phase 3 clinical studies are expected to announce topline data in 3Q2023. If successful, Kodiak plans to file a single Biologics Licensing Applications ("BLA") for tarcocimab in the four major retinal vascular disease indications. The global tarcocimab clinical program is being conducted at 150+ study sites in more than 10 countries. Kodiak is developing and owns global rights to tarcocimab. About KSI-501 Also built on Kodiak's ABC Platform, KSI-501 is an investigational, first-in-class bispecific ABC that is designed to inhibit two mechanisms implicated in retinal diseases: vascular endothelial growth factor ("VEGF") and interleukin-6 (IL-6). IL-6 is a pro-inflammatory cytokine and growth factor implicated in the pathophysiology of multiple retinal diseases and, in conditions for which anti-VEGF treatment is used, elevated levels of ocular IL-6 have been associated with poor anti-VEGF treatment response. KSI-501 is a trap-antibody fusion biopolymer conjugate designed to provide potent inhibition of (i) VEGF-mediated angiogenesis and vascular permeability through a soluble decoy receptor inhibiting the binding of VEGF-A and PLGF to their cognate receptors and (ii) IL-6 mediated inflammation through an antibody that binds soluble interleukin-6, inhibiting its binding to both soluble and membrane-bound IL-6 receptors. In cell-based assays, KSI-501 inhibits angiogenesis and also normalizes inner and outer blood retinal barriers; dual inhibition of VEGF and IL-6 by KSI-501 confers superior normalization of cell morphology and junctional biology compared to either anti-VEGF or anti-IL-6 monotherapy. We believe KSI-501 has the potential to become a new category of retinal medicines with greater therapeutic efficacy than existing therapies while also benefiting from the promising long-interval durability of Kodiak's ABC Platform. A Phase 1 study of KSI-501 is currently dosing patients in the United States to evaluate the safety, tolerability and bioactivity of KSI-501 in DME patients. About Kodiak Sciences Inc. Kodiak (Nasdaq: KOD) is a biopharmaceutical company committed to researching, developing and commercializing transformative therapeutics to treat high prevalence retinal diseases. We are focused on bringing new science to the design and manufacture of next generation retinal medicines to prevent and treat the leading causes of blindness globally. Our antibody biopolymer conjugate platform, or ABC Platform™, uses molecular engineering to merge the fields of antibody-based and chemistry-based therapies and is at the core of Kodiak's discovery engine. Kodiak's lead investigational medicine, tarcocimab tedromer, is a novel anti-VEGF antibody biopolymer conjugate being developed for the treatment of retinal vascular diseases including diabetic eye diseases, the leading cause of blindness in working-age patients in the developed world, and wet age-related macular degeneration, the leading cause of blindness in elderly patients in the developed world. The tarcocimab clinical program is designed to assess the product candidate's durability, efficacy and safety in major retinal vascular diseases in parallel, through the GLEAM and GLIMMER studies in diabetic macular edema, the BEACON study in retinal vein occlusion, the GLOW study in non-proliferative diabetic retinopathy and the DAYLIGHT study in wet age-related macular degeneration. Phase 3 data across the tarcocimab clinical program are expected in 3Q2023. Kodiak has leveraged its ABC Platform to build a pipeline of product candidates in various stages of development. KSI-501 is our dual inhibitor antibody biopolymer conjugate targeting both VEGF (VEGF-trap) and IL-6 (anti-IL-6 antibody) and is being investigated in a Phase 1 clinical study initially in patients with diabetic macular edema. We are expanding our early research pipeline to include ABC Platform based triplet inhibitors for multifactorial retinal diseases. Kodiak is based in Palo Alto, CA. For more information, please visit . Kodiak®, Kodiak Sciences®, ABC™, ABC Platform™ and the Kodiak logo are registered trademarks or trademarks of Kodiak Sciences Inc. in various global jurisdictions. Forward-Looking Statements This release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995. These forward-looking statements are not based on historical fact and include statements regarding: the expected timing and presentation of clinical study readouts; our robust retina-focused research and development engine; the potential of the ABC Platform to maintain potent and effective drug levels in ocular tissues for longer than existing available agents; the potential for KSI-501 to achieve a greater therapeutic benefit for certain patients and to provide potent inhibition of both VEGF-mediated vascular permeability and IL-6 mediated inflammation; future development plans; the objectives and potential benefits of our tarcocimab clinical program, including its potential to enable earlier treatment and prevention of vision loss for patients with diabetic eye diseases; the objectives and potential benefits of KSI-501, including its potential to be a first-in-class bispecific ABC inhibiting VEGF and IL-6 and its potential to provide extended durability; and the potential for a single BLA submission in four major retinal vascular disease indications. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as "may," "will," "should," "would," "could," "expect," "plan," "believe," "intend," "pursue," and other similar expressions among others. Any forward-looking statements are based on management's current expectations of future events and are subject to a risks and uncertainties that could cause actual results to differ materially and adversely from those in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: cessation or delay of any clinical studies and/or development of tarcocimab and/or KSI-501 may occur; the risk that KSI-501 may not inhibit VEGF and IL-6, provide extended durability or have an impact on the treatment of patients as expected; the risk that tarcocimab may not enable earlier treatment and prevention of vision loss for patients with diabetic eye diseases as expected; future potential regulatory milestones of tarcocimab/KSI-501, including those related to current and planned clinical studies, may be insufficient to support regulatory submissions or approval; adverse economic conditions may significantly impact our business and operations, including our clinical trial sites, and those of our manufacturers, contract research organizations or others with whom we conduct business; as well as the other risks identified in our filings with the Securities and Exchange Commission (SEC). For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled "Risk Factors" in our most recent Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the SEC. These forward-looking statements speak only as of the date hereof and Kodiak undertakes no obligation to update forward-looking statements, and readers are cautioned not to place undue reliance on such forward-looking statements. Kodiak®, Kodiak Sciences®, ABC™, ABC Platform™ and the Kodiak logo are registered trademarks or trademarks of Kodiak Sciences Inc. in various global jurisdictions. SOURCE Kodiak Sciences Inc.

- FDA Accepts Filing of New Drug Application and Grants Priority Review for Avacincaptad Pegol (ACP) for the Treatment of Geographic Atrophy; PDUFA Goal Date is August 19, 2023 - - Post-hoc Time-to-Event Analysis of ACP GATHER Trials Signals up to 59% Risk Reduction in Rate of Vision Loss Compared to Sham at 12 Months – - Commercial Launch Preparations for ACP Continue to Accelerate – - Conference Call and Webcast Today, March 1, 2023, at 8:00 a.m. ET - PARSIPPANY, N.J.--(BUSINESS WIRE)-- IVERIC bio, Inc. (Nasdaq: ISEE) today announced financial and operating results for the fourth quarter and full year ended December 31, 2022 and provided a general business update. “In 2022, we successfully delivered a banner year with avacincaptad pegol (ACP) achieving a statistically significant reduction in the rate of geographic atrophy (GA) progression at the 12-month pre-specified primary endpoint across two Phase 3 clinical trials,” stated Glenn P. Sblendorio, Chief Executive Officer of Iveric Bio. “We are excited to begin 2023 with the FDA’s acceptance of the filing of our new drug application (NDA) and granting of priority review for ACP for the treatment of GA secondary to age-related macular degeneration (AMD). With the achievement of these important milestones, we move closer to our goal of providing patients with a treatment for GA, a devastating disease that leads to irreversible blindness.” “We are also excited about the new post-hoc analysis highlighting the potential signal of ACP reducing the rate of vision loss by slowing progression of GA at 12 months of treatment,” stated Pravin U. Dugel, MD, President of Iveric Bio. “Our commercial leadership team brings extensive experience in launching drugs for retinal diseases with large market potential. We continue to accelerate our commercial plans and expect to have the full U.S. commercial team, including a field based sales force, hired by early April. We believe that we are well-positioned to become a leader in the development and commercialization of treatments for retinal diseases and to create long-term value for our shareholders.” Avacincaptad pegol (ACP): Complement C5 Inhibitor In February 2023, the Company announced that the FDA accepted for filing the Company’s NDA for ACP for the treatment of GA secondary to AMD. The NDA has been granted priority review with a Prescription Drug User Fee Act (PDUFA) goal date of August 19, 2023. The Company also announced that, at the time of the FDA’s acceptance letter, the FDA had not identified any potential review issues and the FDA was not currently planning to hold an Advisory Committee meeting for ACP. The Company announced today a post-hoc analysis from the ACP GATHER1 and GATHER2 clinical trials signaling up to a 59% reduction in the rate of vision loss for ACP 2 mg compared to sham at 12 months of treatment. Vision loss is defined as a loss of ≥ 15 letters (EDTRS) in Best Corrected Visual Acuity (BCVA) from baseline measured at two consecutive visits up to month 12. This time to event analysis will be presented at the Association for Research in Vision and Ophthalmology’s annual meeting from April 23-27, 2023. In November 2022, the Company announced the FDA granted Breakthrough Therapy designation (BTD) for ACP for the treatment of GA secondary to AMD. To date, ACP is the first and only investigational therapy to receive BTD status for this indication, which was granted based on the 12-month results from GATHER1 and GATHER2. The Company intends to pursue further discussions with the FDA about utilizing the GATHER1 and GATHER2 clinical trial data included in the current NDA submission to support treatment of GA associated with earlier stage disease, including in patients with intermediate AMD (iAMD). The Company does not believe it needs to conduct a new clinical trial of ACP in patients with iAMD. The Company plans to submit marketing authorization applications (MAAs) to the European Medicines Agency (EMA) and the UK Medicines and Healthcare Regulatory Agency (MHRA) in 2023, subject to feedback from planned interactions with regulatory authorities in Europe, which the Company expects to have during the first half of 2023. The Company is planning to explore collaboration opportunities for the further development and potential commercialization of ACP outside the United States. The Company initiated an open-label extension (OLE) study for patients who completed their month 24 visits in the GATHER2 trial, with the aim of providing patients longer-term access to ACP and collecting additional safety data. Patient enrollment in STAR, the Company’s Phase 2b screening clinical trial of ACP for the treatment of autosomal recessive Stargardt disease (STGD1), is ongoing. IC-500: HtrA1 (high temperature requirement A serine peptidase 1 protein) Inhibitor The Company is developing IC-500, its HtrA1 inhibitor product candidate, for GA and potentially other age-related retinal diseases. The Company is conducting additional preclinical studies to optimize formulation, dosage and delivery of IC-500 and planning for IND-enabling toxicology studies. The Company expects to submit an investigational new drug application (IND) to the FDA in the first half of 2024. Gene Therapy Programs in Orphan Inherited Retinal Diseases (IRDs) The Company is continuing to advance its minigene programs for Leber’s Congenital Amaurosis type 10 (CEP290), autosomal recessive Stargardt Disease (ABCA4) and Usher’s Syndrome type 2A (USH2A). As part of the Company’s previously stated strategy, in December 2022 the Company entered into an asset purchase agreement with Opus Genetics Inc. (Opus), pursuant to which Opus acquired all of the Company’s rights to IC-100, the Company’s former preclinical product candidate for Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa, and IC-200, the Company’s former preclinical product candidate for BEST1-Related IRDs. Corporate Update In December 2022, the Company raised approximately $324.3 million in net proceeds in an underwritten public offering of common stock. In late December 2022, the Company’s Board of Directors (the Board) elected Pravin U. Dugel, MD, President of Iveric Bio, to the Board, effective as of January 1, 2023. Dr. Dugel has been instrumental in helping to shape the Company’s business strategy and in overseeing the development and regulatory strategy for ACP since he joined the Company in 2020. Fourth Quarter and Year Ended 2022 Financial Results As of December 31, 2022, the Company had approximately $646.8 million in cash, cash equivalents and available-for-sale securities. The Company estimates that its cash, cash equivalents, available-for-sale securities and committed loan facilities will be sufficient to fund its planned capital expenditure requirements, debt service obligations and operating expenses for at least the next 12 months. This estimate does not include any potential new borrowings under its term loan facility with Hercules Capital and Silicon Valley Bank, beyond the $25.0 million that the Company plans to borrow during 2023 based on its achievement of the performance milestone related to the FDA's acceptance of its NDA for filing. 2022 Q4 Financial Highlights R&D Expenses: Research and development expenses were $35.8 million for the quarter ended December 31, 2022, compared to $25.1 million for the same period in 2021. For the year ended December 31, 2022, research and development expenses were $117.0 million compared to $85.1 million for the same period in 2021. Research and development expenses increased primarily due to the continued progress of the Company’s GATHER2 trial, increased manufacturing activities for ACP, and increases in personnel costs associated with additional research and development staffing, including share-based compensation, offset by decreases in costs associated with the Company’s gene therapy programs. G&A Expenses: General and administrative expenses were $27.1 million for the quarter ended December 31, 2022 compared to $8.0 million for the same period in 2021. For the year ended December 31, 2022, general and administrative expenses were $72.9 million, compared to $29.7 million for the same period in 2021. General and administrative expenses increased year over year primarily due to increases in commercial preparation expenses for ACP and costs associated with additional staffing for commercial preparation, including share-based compensation. Net Loss: The Company reported a net loss for the quarter ended December 31, 2022, of $59.1 million, or ($0.47) per diluted share, compared to a net loss of $33.0 million, or ($0.29) per diluted share, for the same period in 2021. For the year ended December 31, 2022, the Company reported a net loss of $185.2 million or ($1.53) per diluted share, compared to a net loss of $114.5 million or ($1.12) for the same period in 2021. Conference Call/Webcast Information Iveric Bio will host a conference call/webcast to discuss the Company’s financial and operating results and provide a business update. The call is scheduled for March 1, 2023, at 8:00 a.m. Eastern Time. To participate in this conference call, dial 1-888-317-6003 (USA) or 1-412-317-6061 (International), passcode 1375589. A live, listen-only audio webcast of the conference call can be accessed on the Investors section of the Iveric Bio website at . A replay will be available approximately two hours following the live call for two weeks. The replay number is 1-877-344-7529 (USA Toll Free), passcode 4132187. About Iveric Bio Iveric Bio is a science-driven biopharmaceutical company focused on the discovery and development of novel treatments for retinal diseases with significant unmet medical needs. The Company is committed to having a positive impact on patients’ lives by delivering high-quality, safe and effective treatments designed to address debilitating retinal diseases including earlier stages of age-related macular degeneration. For more information on the Company, please visit . Forward-looking Statements Any statements in this press release about the Company’s future expectations, plans and prospects constitute forward-looking statements for purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995. Forward-looking statements include any statements about the Company’s strategy, future operations and future expectations and plans and prospects for the Company, and any other statements containing the words “anticipate,” “believe,” “estimate,” “expect,” “intend”, “goal,” “may”, “might,” “plan,” “predict,” “project,” “seek,” “target,” “potential,” “will,” “would,” “could,” “should,” “continue,” and similar expressions. In this press release, the Company’s forward looking statements include statements about its expectations regarding the regulatory pathway for and potential approval of ACP for the treatment of GA secondary to AMD, including its plans to submit MAAs to the European Medicines Agency and the UK Medicines and Healthcare Products Agency, statements regarding the Company’s commercial plans and strategy for ACP, the clinical relevance of the clinical data from its GATHER1 and GATHER2 trials of ACP in GA, including from post-hoc analyses that it conducted, its development and regulatory strategy for ACP and its other product candidates, including its plans for the use of ACP in treating patients with intermediate AMD, the timing, progress and results of clinical trials and other research and development activities and regulatory submissions, the potential safety and efficacy and commercial potential of its product candidates, its projected use of cash, cash equivalents, marketable securities and its committed loan facilities and the sufficiency of its cash resources, and statements regarding the Company’s business development and hiring plans. 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, expectations for regulatory matters, interpretation of clinical trial results by the scientific and medical community, the initiation, progress and success of research and development programs and clinical trials, reliance on clinical trial sites, contract development and manufacturing organizations and other third parties, developments from the Company’s competitors and the marketplace for the Company’s products, need for and availability of additional financing, negotiation and consummation of business development transactions, the hiring of personnel and other factors discussed in the “Risk Factors” section contained in the quarterly and annual reports that the Company files with the Securities and Exchange Commission. Any forward-looking statements represent the Company’s views only as of the date of this press release. The Company anticipates that subsequent events and developments may cause its views to change. 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 except as required by law. IVERIC bio, Inc. Selected Financial Data (unaudited) (in thousands, except per share data) Three Months Ended December 31, Year Ended December 31, 2022 2021 2022 2021 Statements of Operations Data: Operating expenses: Research and development $ 35,841 $ 25,096 $ 117,012 $ 85,068 General and administrative 27,130 8,001 72,894 29,689 Total operating expenses 62,971 33,097 189,906 114,757 Loss from operations (62,971 ) (33,097 ) (189,906 ) (114,757 ) Interest income, net 1,449 61 2,264 245 Gain on sale of IC100 & IC200 2,369 - 2,369 - Other income (expense), net 92 3 62 (10 ) Loss before income tax benefit (59,061 ) (33,033 ) (185,211 ) (114,522 ) Income tax benefit - - - - Net loss $ (59,061 ) $ (33,033 ) $ (185,211 ) $ (114,522 ) Net loss per common share: Basic and diluted $ (0.47 ) $ (0.29 ) $ (1.53 ) $ (1.12 ) Weighted average common shares outstanding: Basic and diluted 125,367 115,073 121,037 101,866 December 31, 2022 December 31, 2021 (in thousands) Balance Sheets Data: Cash, cash equivalents and marketable securities $ 646,835 $ 381,749 Total assets $ 666,823 $ 389,358 Term loan, net $ 96,568 $ - Total liabilities $ 132,166 $ 28,830 Additional paid-in capital $ 1,399,555 $ 1,040,098 Accumulated deficit $ (864,806 ) $ (679,595 ) Total stockholders' equity $ 534,657 $ 360,528 ISEE-G

Reported Positive GATHER2 Results for Avacincaptad Pegol in Geographic Atrophy Planned NDA Submission Moved Up to End of This Year Received Favorable Feedback from FDA on Intermediate AMD Development Plan Conference Call and Webcast Today, November 3, 2022, at 8:00 a.m. ET PARSIPPANY, N.J.--(BUSINESS WIRE)-- IVERIC bio, Inc. (Nasdaq: ISEE) today announced financial and operating results for the third quarter ended September 30, 2022 and provided a general business update. “With the positive data from our GATHER2 clinical trial reported in the third quarter reinforcing the positive data from GATHER1, avacincaptad pegol (ACP) became the first and only investigational therapy in geographic atrophy (GA) to achieve its 12-month, prespecified, primary endpoint in two independent pivotal, Phase 3 clinical trials,” stated Glenn P. Sblendorio, Chief Executive Officer of Iveric Bio. “We are excited to announce that we are ahead of schedule in preparing our new drug application (NDA) for ACP for the treatment of GA and we are moving up our submission timeline to the end of this year.” “As we close out the year and look forward to 2023, we are working diligently to make ACP available to physicians and their patients with GA as expeditiously as possible, subject to regulatory review and approval,” stated Pravin U. Dugel, MD, President of Iveric Bio. “We continue to build our U.S. launch readiness plan and prepare for potential commercialization of ACP. We also continue to explore future development opportunities for ACP in earlier patient populations and to invest in lifecycle initiatives such as sustained release delivery technologies for ACP.” Avacincaptad pegol (ACP also known as Zimura): Complement C5 Inhibitor In September 2022, the Company announced that GATHER2, the Company’s second Phase 3 clinical trial of ACP for the treatment of GA, met its pre-specified primary endpoint at 12 months with statistical significance and a favorable safety profile. Results from GATHER1, the Company’s first Phase 3 clinical trial of ACP for the treatment of GA, and GATHER2, as well as the Company’s Special Protocol Assessment with the U.S. Food and Drug Administration (FDA), provide the basis for an NDA, which the Company plans to submit to the FDA by the end of this year. The Company is also planning to submit a marketing authorization application (MAA) to the European Medicines Agency in 2023, subject to feedback from planned interactions with regulatory authorities in Europe. The GATHER2 topline results for ACP were presented in two oral sessions as part of the Retina Subspecialty Day at the American Academy of Ophthalmology 2022 Annual Meeting on September 30, 2022. The Company recently initiated an open-label extension (OLE) trial for patients who completed their month 24 visits in the GATHER2 trial, with the aim of providing patients access to ACP and collecting additional safety data. Patients will be treated with ACP for 18 months or until potential regulatory approval of ACP in the applicable region, whichever is earlier. The Company received favorable feedback from the FDA on its development plans for intermediate AMD. The Company is continuing to engage with the FDA regarding its development plans and strategy for this important patient population. In July 2022, a post-hoc analysis from the GATHER1 clinical trial was presented at the Annual Meeting of the American Society of Retina Specialists. In the post-hoc analysis, ACP was observed to be associated with a reduction in GA lesion growth compared to sham across all distances from the foveal center point. Patient enrollment in STAR, the Company’s Phase 2b screening clinical trial of ACP for the treatment of autosomal recessive Stargardt disease (STGD1), is ongoing. IC-500: HtrA1 (high temperature requirement A serine peptidase 1 protein) Inhibitor The Company plans to conduct additional preclinical studies to optimize formulation, dosage and delivery of IC-500. As a result, the Company does not expect to submit an investigational new drug application for IC-500 mid-next year, as it had previously planned. The Company remains committed to this program and will provide additional information as it becomes available. Gene Therapy Programs in Orphan Inherited Retinal Diseases (IRDs) As the Company focuses its efforts and resources on the development and potential commercialization of ACP, the Company is currently seeking potential collaborators or licensees for the future development and potential commercialization of IC-100, the Company’s product candidate for Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa (RHO-adRP) and IC-200, the Company’s product candidate for BEST1-Related IRDs. The Company is continuing its minigene programs for Leber’s Congenital Amaurosis type 10 (CEP290), autosomal recessive Stargardt Disease (ABCA4) and Usher’s Syndrome (USH2A). Non-Dilutive Debt Financing Facility In July 2022, the Company entered into a term loan debt financing facility with Hercules Capital, Inc. (Hercules Capital) and Silicon Valley Bank (SVB) providing the Company with total borrowing capacity of up to $250 million in non-dilutive debt financing. In July 2022, the Company borrowed $50 million at the close of the facility. The Company believes it has satisfied the first performance milestone under that term loan facility, which would allow it to borrow an additional $50 million. The Company plans to borrow this additional amount in the fourth quarter of 2022. Third Quarter Financial Results and 2022 Cash Guidance As of September 30, 2022, the Company had $321 million in cash, cash equivalents and available for sale securities, which reflects the impact of its $50 million initial borrowing under its term loan debt financing facility with Hercules and SVB. The Company estimates its year-end 2022 cash, cash equivalents and available for sale securities to range between $265 and $275 million. The Company estimates that its cash, cash equivalents, available for sale securities and committed loan facilities will be sufficient to fund its planned capital expenditure requirements, debt service obligations and operating expenses through at least mid-2024. These estimates are based on the Company’s current business plan, including the continuation of its ongoing clinical development programs for ACP in GA and STGD1, including the recently initiated OLE trial, evaluating ACP for intermediate AMD, preparation and submission of an NDA and an MAA for ACP in GA, continuing preparations for potential commercialization of ACP in GA in the United States, pursuing DelSiTech’s silica-based sustained release delivery technology and exploring additional sustained release delivery technologies for ACP, and the advancement of its IC-500 development program as currently planned. These estimates do not include any potential new borrowings under the term loan facility with Hercules and SVB, including the $50 million that the Company plans to borrow in the fourth quarter of this year. Also excluded from these estimates are any potential approval or sales milestones payable to Archemix Corp. or any potential expenses for actual commercial launch of ACP, such as associated sales force expenses, any additional expenditures related to potentially studying ACP in indications outside of GA, STGD1 and intermediate AMD, or resulting from the potential in-licensing or acquisition of additional product candidates or technologies, or any associated development the Company may pursue. 2022 Q3 Financial Highlights R&D Expenses: Research and development expenses were $25.0 million for the quarter ended September 30, 2022, compared to $17.9 million for the same period in 2021. For the nine months ended September 30, 2022, research and development expenses were $81.2 million compared to $60.0 million for the same period in 2021. Research and development expenses increased primarily due to the continued progress of the Company’s GATHER2 trial, increased manufacturing activities for ACP, and increases in personnel costs, including share-based compensation associated with additional research and development staffing, offset by decreases in costs associated with the Company’s gene therapy programs. G&A Expenses: General and administrative expenses were $17.5 million for the quarter ended September 30, 2022, compared to $6.6 million for the same period in 2021. For the nine months ended September 30, 2022, general and administrative expenses were $45.8 million compared to $21.7 million for the same period in 2021. General and administrative expenses increased primarily due to increases in personnel costs, including share-based compensation associated with staffing for commercial preparation. Net Loss: The Company reported a net loss for the quarter ended September 30, 2022, of $42.4 million, or ($0.35) per diluted share, compared to a net loss of $24.6 million, or $(0.23) per diluted share, for the same period in 2021. For the nine months ended September 30, 2022, the Company reported a net loss of $126.2 million or ($1.05) per diluted share, compared to a net loss of $81.5 million or ($0.84) for the same period in 2021. Conference Call/Webcast Information Iveric Bio will host a conference call/webcast to discuss the Company’s financial and operating results and provide a business update. The call is scheduled for November 3, 2022, at 8:00 a.m. Eastern Time. To participate in this conference call, dial 1-888-317-6003 (USA) or 1-412-317-6061 (International), passcode 8170771. A live, listen-only audio webcast of the conference call can be accessed on the Investors section of the Iveric Bio website at . A replay will be available approximately two hours following the live call for two weeks. The replay number is 1-877-344-7529 (USA Toll Free), passcode 6056402. About Iveric Bio Iveric Bio is a science-driven biopharmaceutical company focused on the discovery and development of novel treatments for retinal diseases with significant unmet medical needs. The Company is committed to having a positive impact on patients’ lives by delivering high-quality, safe and effective treatments designed to address debilitating retinal diseases including earlier stages of age-related macular degeneration. Forward-looking Statements Any statements in this press release about the Company’s future expectations, plans and prospects constitute forward-looking statements for purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995. Forward-looking statements include any statements about the Company’s strategy, future operations and future expectations and plans and prospects for the Company, and any other statements containing the words “anticipate,” “believe,” “estimate,” “expect,” “intend”, “goal,” “may”, “might,” “plan,” “predict,” “project,” “seek,” “target,” “potential,” “will,” “would,” “could,” “should,” “continue,” and similar expressions. In this press release, the Company’s forward looking statements include statements about its expectations regarding the robustness and clinical relevance of the clinical data from its GATHER1 and GATHER2 trials of ACP in GA, its development and regulatory strategy for ACP and its other product candidates, including its plans to submit an NDA to the U.S. Food and Drug Administration and an MAA to the European Medicines Agency for ACP and its plans for evaluating ACP in patients with intermediate AMD, the timing, progress and results of clinical trials and other research and development activities and regulatory submissions, the potential utility of its product candidates and sustained release delivery technologies for ACP, its projected use of cash, cash equivalents, marketable securities and its committed loan facilities and the sufficiency of its cash resources, and statements regarding the Company’s commercial plans and strategy. 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, expectations for regulatory matters, interpretation of clinical trial results by the scientific and medical community, the initiation, progress and success of research and development programs and clinical trials, reliance on clinical trial sites, contract development and manufacturing organizations and other third parties, developments from the Company’s competitors, and the marketplace for the Company’s products, need for and availability of additional financing and negotiation and consummation of business development transactions and other factors discussed in the “Risk Factors” section contained in the quarterly and annual reports that the Company files with the Securities and Exchange Commission. Any forward-looking statements represent the Company’s views only as of the date of this press release. The Company anticipates that subsequent events and developments may cause its views to change. 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 except as required by law. IVERIC bio, Inc. Selected Financial Data (unaudited) (in thousands, except per share data) Three Months Ended September 30, Nine Months Ended September 30, 2022 2021 2022 2021 Statements of Operations Data: Operating expenses: Research and development $ 24,967 $ 17,935 $ 81,171 $ 59,972 General and administrative 17,545 6,648 45,764 21,688 Total operating expenses 42,512 24,583 126,935 81,660 Loss from operations (42,512 ) (24,583 ) (126,935 ) (81,660 ) Interest income, net 200 42 815 184 Other expense, net (39 ) (10 ) (30 ) (13 ) Loss before income tax benefit (42,351 ) (24,551 ) (126,150 ) (81,489 ) Income tax benefit - - - - Net loss $ (42,351 ) $ (24,551 ) $ (126,150 ) $ (81,489 ) Net loss per common share: Basic and diluted $ (0.35 ) $ (0.23 ) $ (1.05 ) $ (0.84 ) Weighted average common shares outstanding: Basic and diluted 120,277 105,217 119,578 97,370 September 30, 2022 December 31, 2021 (in thousands) Balance Sheets Data: Cash, cash equivalents and marketable securities $ 320,535 $ 381,749 Total assets $ 331,790 $ 389,358 Term loan, net $ 47,649 $ - Total liabilities $ 72,264 $ 28,830 Additional paid-in capital $ 1,065,545 $ 1,040,098 Accumulated deficit $ (805,745 ) $ (679,595 ) Total stockholders' equity $ 259,526 $ 360,528 ISEE-G View source version on businesswire.com: Contacts Investor Contact: Kathy Galante Senior Vice President, Investor Relations kathy.galante@ivericbio.com or Source: IVERIC bio, Inc. 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