Pigmentary Disorder, Reticulate, With Systemic Manifestations

BEDFORD, Mass., June 07, 2023 (GLOBE NEWSWIRE) -- Ocular Therapeutix , Inc. (NASDAQ:OCUL), a biopharmaceutical company focused on the formulation, development, and commercialization of innovative therapies for diseases and conditions of the eye, today announced completion of enrollment in the HELIOS clinical trial of OTX-TKI. Using Elutyx™ technology, OTX-TKI is the Company’s axitinib intravitreal implant that is being developed for the treatment of diabetic retinopathy, wet AMD and other retinal diseases. Diabetic retinopathy is a leading cause of vision loss in working-age adults, affecting approximately 140 million individuals around the world, including 8.4 million people in the United States alone. Diabetic retinopathy can lead to vision loss and even blindness due to high levels of blood sugar in the eyes that damage the blood vessels in the retina, the light-sensitive tissue at the back of the eye. “Millions of people suffer from diabetic retinopathy. Today, the current standard of care in non-proliferative stages is primarily ‘watchful waiting’, as frequent anti-VEGF injections and office visits pose a significant treatment burden primarily for the working-age population. Many patients frequently progress to more severe vision-destroying disease before they seek treatment, making diabetic retinopathy one of the leading causes of blindness among working-age adults in the U.S.,” said Rabia Gurses Ozden, MD, Chief Medical Officer of Ocular Therapeutix. “We believe the same attributes that make OTX-TKI a compelling product candidate for the treatment of wet AMD – the ease of use of an office-based injection and the potential for long-term durability that reduces the treatment burden and need for frequent, invasive injections – could establish OTX-TKI as the standard of care in the treatment of diabetic retinopathy.” The HELIOS Phase 1 trial is a multi-center, prospective, masked, 2:1 randomized, controlled U.S.-based trial in 21 subjects evaluating 600 µg OTX-TKI dosed in a single implant containing axitinib compared to a sham injection procedure. Interim 6-month data from the trial are expected in the first quarter of 2024. The Company has been in discussions with the FDA for the clinical development of OTX-TKI for the treatment of diabetic retinopathy and has a potential pivotal design that is consistent with guidance from the FDA. Subject to favorable interim data from the ongoing clinical trial and obtaining the necessary financing to fund the trial, the Company plans to be prepared to initiate a Phase 3 pivotal trial for diabetic retinopathy as early as Q1 2024. This is in addition to the Company’s plan to be prepared for initiation of a pivotal program in wet AMD as early as Q3 of 2023, subject to obtaining the necessary financing. About OTX-TKI OTX-TKI is an investigational bioresorbable, hydrogel implant incorporating axitinib, a small molecule, multi-target, tyrosine kinase inhibitor with anti-angiogenic properties, being evaluated for the treatment of wet age-related macular degeneration (wet AMD), diabetic retinopathy and other retinal diseases. About Diabetic Retinopathy Diabetic retinopathy is a chronic, progressive disease driven by elevated levels of blood glucose and depleted levels of oxygen causing damage to blood vessels in the retina. Diabetic retinopathy is among the most common microvascular complications of diabetes and is a leading cause of blindness globally. Diabetic retinopathy can take time to develop, and may progress from early, mild, non-proliferative diabetic retinopathy (NPDR) to moderate and then severe NPDR and eventually to proliferative diabetic retinopathy (PDR). As patients progress from mild to severe NPDR, the risk of developing proliferative disease increases. PDR is the most serious stage of the disease and develops when areas of the retina that are starved for nourishment and oxygen, triggering the proliferation of new blood vessels via secretions of vascular endothelial growth factor (VEGF). Diabetic macular edema (DME), an accumulation of fluid in the macula, can occur at any stage of diabetic retinopathy. The current standard of care for NPDR is watchful waiting, although damage to blood vessels in the retina is ongoing in the background, with anti-VEGFs used more commonly when the disease has progressed to the proliferative stage. Regular eye examinations are crucial for early detection and prevention of diabetic retinopathy. There were an estimated 8.4 million cases of diabetic retinopathy in the United States in 2022 according to Market Scope, of which 3.3 million cases were moderate to severe non-proliferative, growing at an approximately 2% compound annual growth rate through 2027. Overall, there are an estimated 141.2 million cases of diabetic retinopathy globally, growing at a compound annual growth rate of 3%. Prevalence of diabetic retinopathy is expected to increase due to population growth and increasing incidence of diabetes throughout much of the world. About Ocular Therapeutix, Inc. Ocular Therapeutix, Inc. is a biopharmaceutical company focused on the formulation, development, and commercialization of innovative therapies for diseases and conditions of the eye using its proprietary bioresorbable hydrogel-based formulation technology. Ocular Therapeutix’s first commercial drug product, DEXTENZA®, is an FDA-approved corticosteroid for the treatment of ocular inflammation and pain following ophthalmic surgery and ocular itching associated with allergic conjunctivitis. Ocular Therapeutix’s earlier stage development assets include: OTX-TKI (axitinib intravitreal implant), currently in Phase 1 clinical trials for the treatment of wet AMD and diabetic retinopathy; OTX-TIC (travoprost intracameral implant), currently in a Phase 2 clinical trial for the treatment of primary open-angle glaucoma or ocular hypertension; and OTX-DED (dexamethasone intracanalicular insert) for the short-term treatment of the signs and symptoms of dry eye disease; and OTX-CSI (cyclosporine intracanalicular insert) for the chronic treatment of dry eye disease, both of which have completed Phase 2 clinical trials. Forward Looking Statements Any statements in this press release about future expectations, plans, and prospects for the Company, including the commercialization of DEXTENZA® or any of the Company’s products or product candidates; the development and regulatory status of the Company’s product candidates, such as the Company’s development of, timing of, and prospects for approvability of OTX-TKI for the treatment of retinal diseases including wet AMD and diabetic retinopathy including the timing of planned pivotal clinical trials, OTX-TIC for the treatment of primary open-angle glaucoma or ocular hypertension, OTX-DED for the short-term treatment of the signs and symptoms of dry eye disease, and OTX-CSI for the chronic treatment of dry eye disease; the Company’s plans to advance the development of its product candidates or preclinical programs; the Company’s ability to fund the planned and future development of its product candidates, whether through strategic alliances or other fundraising; the potential utility of any of the Company’s product candidates; the size of potential markets for the Company’s product candidates; 2023 financial guidance, including estimated net product revenue; the sufficiency of the Company’s cash resources; and other statements containing the words "anticipate," "believe," "estimate," "expect," "intend", "goal," "may", "might," "plan," "predict," "project," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors. Such forward-looking statements involve substantial risks and uncertainties that could cause the Company’s preclinical and clinical 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, the timing and costs involved in commercializing DEXTENZA or any product or product candidate that receives regulatory approval, including the conduct of post-approval studies, the ability to retain regulatory approval of DEXTENZA or any product or product candidate that receives regulatory approval, the ability to maintain and the sufficiency of product, procedure and any other reimbursement codes for DEXTENZA, the initiation, timing, conduct and outcomes of clinical trials, whether clinical trial data will be indicative of the results of subsequent clinical trials in the same or other indications or that interim data will be indicative of the full data from a clinical trial, uncertainties as to the timing and availability of data from clinical trials and expectations for regulatory submissions and approvals, the Company’s ability to enter into and perform its obligations under collaborations and the performance of its collaborators under such collaborations, the Company’s scientific approach and general development progress, the availability or commercial potential of the Company’s product candidates, the Company’s ability to meet supply demands, the Company’s ability to generate its projected net product revenue and in-market sales on the timeline expected, if at all, the sufficiency of cash resources, the Company’s existing indebtedness, the ability of the Company’s creditors to accelerate the maturity of such indebtedness upon the occurrence of certain events of default, any additional financing needs, the Company’s ability to recruit and retain key personnel, and other factors discussed in the “Risk Factors” section contained in the Company’s quarterly and annual reports on the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company’s views as of the date of this press release. The Company anticipates that subsequent events and developments will cause the Company’s views to change. However, 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, whether as a result of new information, future events or otherwise, except as required by law. These forward-looking statements should not be relied upon as representing the Company’s views as of any date subsequent to the date of this press release. Investors Ocular Therapeutix Donald Notman Chief Financial Officer dnotman@ocutx.com or ICR Westwicke Chris Brinzey, 339-970-2843 Managing Director chris.brinzey@westwicke.com Media ICR Westwicke Ben Shannon, 443-213-0495 ben.shannon@westwicke.com

Point-of-Care AI-DR is the first AI-assisted system which can be integrated into handheld and desktop fundus cameras used to detect diabetic retinopathy and diabetic macular edema HSINCHU, Nov. 10, 2022 /PRNewswire/ -- The Industrial Technology Research Institute (ITRI), Taiwan's largest and one of the world's leading high-tech applied research institutions, today introduced Point-of-Care AI-DR, the first AI-assisted system which can be integrated into handheld and desktop fundus cameras commonly used in hospitals and clinics to detect diabetic retinopathy (DR) and diabetic macular edema (DME). Point-of-Care AI-DR is a 2022 R&D 100 Awards winner in the software/services award category. Continue Reading Point-of-Care AI-DR is the first AI-based solution that can locate four main lesions, classify five severity levels of DR, and determine whether a patient should be referred to an ophthalmologist. Point-of-Care AI-DR diagnoses DR and DME in 5 to 10 seconds and instantly assesses image quality before diagnosis to prevent misinterpretation by the AI. It marks lesions and assesses the corresponding severity levels based on the fundus images. Further, Point-of-Care AI-DR can identify 14 other common ocular fundus abnormalities, including retinal related diseases, changes in blood vessels, and optic nerve related diseases. Point-of-Care AI-DR is easy to use, so general physicians can perform early screening for DR and DME complications. It also can determine whether a patient should be referred to an ophthalmologist. Screening through general physicians increases screening rates, reduces treatment costs and improves patient outcomes by enabling early detection. According to the International Diabetes Foundation, 537 million adults globally between 20 and 79 years old were living with diabetes in 2021, and the number is projected to increase to 643 million by 2030 and 783 million by 2045. According to the U.S. National Eye Institute, more than half of people with diabetes will develop DR and about one in 15 will develop DME. By identifying lesions and severity stages of DR and DME, Point-of-Care AI-DR improves diabetes health monitoring and management and helps lower the risks of vision loss or blindness caused by diabetic eye complications. Physicians can effectively grasp the patient's diabetes development status by tracking the changes in fundus symptoms over time. This is crucial for diabetes management, because the changes in fundus symptoms can be monitored more reliably than blood sugar levels, which patients can alter immediately before examinations. "Point-of-Care AI-DR is a collaboration of human and artificial intelligence through the expertise of ophthalmologists and AI analysis. It uses complementary medical AI models to perform individual diagnostic tasks such as classifying and detecting symptoms to improve the efficiency of overall interpretation," said Dr. Pang-An Ting, ITRI general director of Information and Communications Research Laboratories. To create Point-of-Care AI-DR, ITRI engaged 50 ophthalmologists who collected and labeled 150,000 fundus images as the training data for robust AI models. As a result, Point-of-Care AI-DR outperforms existing products in terms of sensitivity (>98%) and specificity (>96%) in DR diagnosis and helps non-ophthalmologists perform rapid point-of-care DR screening like ophthalmologists. It can support all existing fundus cameras worldwide and be used to create different types of solutions such as edge AI systems, standalone web applications, and private/public cloud-based services. Point-of-Care AI-DR is the first AI-based solution for DR and DME detection that can: Locate four main lesions: microaneurysms, hemorrhages, soft exudates, and hard exudates, and detect two anatomical landmarks: optic disc and macular area Classify five severity levels of diabetic retinopathy: no DR, mild NPDR, moderate NPDR, severe NPDR and PDR Produce binary classifications of DR and DME so general practitioners can provide data-informed decisions for referrals to ophthalmologists Point-of-Care AI-DR is available for licensing. One example of Point-of-Care AI-DR licensing is VeriSee DR by Acer. Companies interested in licensing should contact ITRI. About ITRI Industrial Technology Research Institute (ITRI) is one of the world's leading technology R&D institutions aiming to innovate a better future for society. Founded in 1973, ITRI has played a vital role in transforming Taiwan's industries from labor-intensive into innovation-driven. To address market needs and global trends, it has launched its 2030 Technology Strategy & Roadmap and focuses on innovation development in Smart Living, Quality Health, and Sustainable Environment. It also strives to strengthen Intelligentization Enabling Technology to support diversified applications. Over the years, ITRI has been dedicated to incubating startups and spinoffs, including well-known names such as UMC and TSMC. In addition to its headquarters in Taiwan, ITRI has branch offices in the U.S., Europe, and Japan in an effort to extend its R&D scope and promote international cooperation across the globe. For more information, please visit . SOURCE Industrial Technology Research Institute

Virtual Webinar to Take Place Friday, October 14th @ 11 AM ET Topline Results from ZETA-1 Phase 2b Trial of APX3330 Expected Later in 4Q 2022 FARMINGTON HILLS, Mich., Oct. 06, 2022 (GLOBE NEWSWIRE) -- Ocuphire Pharma, Inc. (Nasdaq: OCUP), a clinical-stage ophthalmic biopharmaceutical company focused on developing and commercializing therapies for the treatment of refractive and retinal eye disorders, today announced that it will host a key opinion leader (KOL) webinar highlighting oral APX3330, its lead drug candidate for diabetic retinopathy (DR) and diabetic macular edema (DME), on Friday, October 14, 2022 at 11AM to 12:30PM Eastern Time. The event will feature KOLs Peter Kaiser, MD, from the Cleveland Clinic, Caroline Baumal, MD, from Tufts Medical Center, and David Lally, MD, from New England Retina Consultants. KOL presentations will set the stage for a discussion on Ocuphire’s APX3330, a first-in-class, novel orally administered small molecule inhibitor of VEGF and inflammation currently in a completed multi-center, randomized, double-masked, placebo-controlled ZETA-1 Phase 2b trial for DR and DME. The discussion will highlight the unmet need and current treatment landscape for DR/DME. New data will be presented on study demographics and 24-week masked safety data from 103 enrolled diabetic retinopathy patients. The KOLs will share insights on the trial design, endpoints, and safety and efficacy expectations for a new novel oral treatment in DR. Topline results from ZETA-1 are expected later in the 4th quarter. A live question & answer session will follow. To register for the event, please click here. Dr. Peter Kaiser, MD is a clinical research expert, serving as Study Chairman of five major, multi-center, international clinical trials, and principal investigator in numerous studies for Age-related Macular Degeneration (AMD), DR, and other retinal disorders. He is the Editor-in-Chief of Retinal Physician, Associate Editor of International Ophthalmology Clinics, and serves on the editorial boards of Retina, Retina Today, and Ocular Surgery News. He is a major contributor to medical literature, having authored seven textbooks and more than 250 peer-reviewed papers, and has been recognized by the American Academy of Ophthalmology and American Society of Retina Specialists (ASRS) with Senior Achievement Awards and is listed as one of the “Best Doctors in America.” Dr. Kaiser graduated magna cum laude with Highest Honors from Harvard College and Harvard Medical School. He completed an ophthalmology residency at the Massachusetts Eye and Ear Infirmary, and a vitreoretinal fellowship at Bascom Palmer Eye Institute before joining the vitreoretinal department of the Cole Eye Institute, Cleveland, Ohio. Caroline R. Baumal, MD is a Professor of Ophthalmology, Co-Director of the Retina Service and Co-Director of the medical retina fellowship at New England Eye Center, Tufts Medical Center in Boston, MA. Her clinical interests include age-related macular degeneration, diabetic retinopathy, retinal circulatory disorders, complex vitreoretinal surgery, and retinopathy of prematurity (ROP). She has received multiple awards from various societies including the American Academy of Ophthalmology, and is on the editorial board for Retina Cases and Brief Reports and Ophthalmic Surgery, Lasers, and Imaging (OSLI) Retina. Dr. Baumal has authored over 170 publications, 33 book chapters on retinal diseases and recently edited the book Treatment of Diabetic Retinopathy. Dr. Baumal completed medical school and ophthalmology residency at the University of Toronto after undergraduate studies at McGill University. She completed two fellowships: one at New England Eye Center, Boston in Medical Retina and Lasers, and another in Vitreoretinal Diseases and Surgery at Wills Eye Hospital in Philadelphia. Dr. Baumal is Board Certified by the American Board of Ophthalmology. Dr. David Lally is the Director of the Retina Research Institute at New England Retina Consultants. He is an attending surgeon at Baystate Medical Center in Springfield, Massachusetts and Assistant Professor in the Department of Surgery at the University of Massachusetts Medical School-Baystate. Dr. Lally is a clinical trial expert who has participated as an investigator in more than 40 clinical trials for retinal diseases. He lectures nationally and has authored over 30 peer-reviewed papers published in journals including the New England Journal of Medicine (NEJM), JAMA Ophthalmology, and Ophthalmology Retina. He also serves as a manuscript reviewer for several ophthalmology journals. Dr. Lally is an active member of the American Society of Retina Specialists with the Fellow of the American Society of Retina Specialist (FASRS) award designation. Dr. Lally graduated from Vanderbilt University and received a medical degree from Jefferson Medical College. He completed an ophthalmology residency at the Wills Eye Hospital in Philadelphia followed by a vitreoretinal fellowship at Tufts/New England Eye Center and Ophthalmic Consultants of Boston. About Diabetic Retinopathy Diabetes, a worldwide epidemic, is the leading cause of blindness among adults age 20 to 74. DR is the most common diabetic complication that affects the eyes and is manifested when chronically elevated blood sugar levels cause damage to blood vessels in the retina. DR affects over 8 million patients in the U.S. and 93 million patients worldwide. This problem is projected to worsen as the number of individuals at risk of developing diabetes increases. There are two major types of DR: (1) non-proliferative DR (NPDR) and (2) proliferative DR (PDR). NPDR is an earlier, more typical stage of DR that can progress to more severe forms of DR if untreated and if the underlying diabetes remains uncontrolled. PDR is a more advanced stage of DR that is characterized by retinal neovascularization that, if left untreated, can lead to permanent damage and blindness. When DR is in its early stages, blood vessels in the retina are damaged and can leak fluid into the retina, a complication called diabetic macular edema (DME). Fluid from DME and hemorrhage of the abnormal blood vessels formed in PDR can interfere with vision and can cause irreversible visual impairment due to retinal scarring and retinal detachment. Despite the approval of intravitreal injection therapies for DR, patients with DR are not widely treated. About APX3330 APX3330 is a first-in-class, small molecule, oral inhibitor of the transcription factor regulator Ref-1 (reduction-oxidation effector factor-1). With a novel dual mechanism of action, APX3330 blocks the downstream pathways regulated by Ref-1 – including those involving angiogenesis (VEGF) and inflammation (NFkB) – to decrease abnormal activation of both angiogenesis, and of inflammatory pathways that are implicated across several ocular diseases, including DR, DME, and age-related macular degeneration (AMD). APX3330 has shown a favorable safety and tolerability pro 11 clinical trials conducted in healthy, hepatitis, and cancer subjects prior to the current Phase 2 ZETA-1 trial in diabetic retinopathy. The most recent interim analysis of masked safety data from ZETA-1 trial was presented by Dr. Michael Allingham, M.D., Ph.D., at the American Society of Retina Specialists’ (ASRS) 40th Annual Scientific Meeting in July 2022. These data indicated that oral APX3330 continued to demonstrate a favorable safety pro with the prior trials comprising hundreds of patients. Across all the trials, the safety findings represent over 9000 subject-days of exposure at the target dose of 600 mg/day. About Ocuphire Pharma Ocuphire is a publicly traded (Nasdaq: OCUP), clinical-stage, ophthalmic biopharmaceutical company focused on developing and commercializing small-molecule therapies for the treatment of refractive and retinal eye disorders. The Company’s lead product candidate, Nyxol® eye drops (0.75% phentolamine ophthalmic solution), is a once-daily, preservative-free eye drop formulation of phentolamine mesylate, a non-selective alpha-1 and alpha-2 adrenergic antagonist designed to reduce pupil size, and is being developed for several indications, including reversal of pharmacologically-induced mydriasis (RM), presbyopia and dim light or night vision disturbances (NVD). Nyxol has been studied in 12 completed clinical trials, with positive data reported from the MIRA-2 and MIRA-3 registration trials and the MIRA-4 pediatric safety trial for the treatment of RM. Ocuphire also reported positive top-line data from the VEGA-1 Phase 2 trial of Nyxol for treatment of presbyopia, which evaluated both Nyxol as a single agent and Nyxol with low dose pilocarpine (“LDP”) 0.4% as adjunctive therapy. The Company recently announced positive top-line results from the LYNX-1 Phase 3 trial of Nyxol for night vision disturbances (NVD). Ocuphire’s second product candidate, APX3330, is an oral tablet designed to inhibit angiogenesis and inflammation pathways relevant to retinal and choroidal vascular diseases, such as diabetic retinopathy (DR) and diabetic macular edema (DME). APX3330 has been studied in 11 Phase 1 and 2 trials. Please visit to learn more about Ocuphire’s ongoing APX3330 Phase 2b trial in DR/DME ZETA-1 (NCT04692688) and completed Nyxol trials: Phase 3 registration trial in NVD LYNX-1 (NCT04638660), Phase 3 registration trials in RM MIRA-2 (NCT04620213) and MIRA-3 (NCT05134974), MIRA-4 Phase 3 pediatric safety study (NCT05223478), and Phase 2 trial in presbyopia VEGA-1 (NCT04675151). For more information, visit . Forward Looking Statements Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, the success and timing of planned regulatory filings, including the timing of the results of the ZETA-1 Phase 2b trial and the market for Ocuphire’s indications including DR. These forward-looking statements are based upon Ocuphire’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, including, without limitation: (i) the success and timing of regulatory submissions and pre-clinical and clinical trials, including enrollment and data readouts; (ii) regulatory requirements or developments; (iii) changes to clinical trial designs and regulatory pathways; (iv) changes in capital resource requirements; (v) risks related to the inability of Ocuphire to obtain sufficient additional capital to continue to advance its product candidates and its preclinical programs; (vi) legislative, regulatory, political and economic developments, (vii) changes in market opportunities, (viii) the effects of COVID-19 on clinical programs and business operations, (ix) the success and timing of commercialization of any of Ocuphire’s product candidates and (x) the maintenance of Ocuphire’s intellectual property rights. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including the risk factors detailed in documents that have been and may be filed by Ocuphire from time to time with the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Ocuphire undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made. Contacts Corporate Mina Sooch, President & CEO Ocuphire Pharma, Inc. ir@ocuphire.com Investors Corey Davis, Ph.D. LifeSci Advisors cdavis@lifesciadvisors.com