Stargardt Disease

Industry Veteran with Extensive Experience in Retinal Disease Research and Strong Track Record in Advancing Programs Through Clinical Development ALPHARETTA, Ga., March 18, 2024 (GLOBE NEWSWIRE) -- Clearside Biomedical, Inc. (Nasdaq: CLSD), a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS®), announced today that Victor Chong, M.D., MBA was appointed Chief Medical Officer on March 14, 2024. Dr. Chong is a board-certified retinal specialist with more than 25 years of experience as a retinal physician and navigating all phases of ophthalmic drug development. George Lasezkay, Pharm.D., J.D., President and Chief Executive Officer of Clearside, commented, “Victor is a well-known and well-respected retinal clinician and scientist, who is a strategically focused and visionary leader. We are delighted to have him join the Clearside team. Victor has worked on small molecules, biologics, oligonucleotides, and gene therapy across the spectrum of retinal diseases, including age-related macular degeneration (AMD), diabetic macular edema (DME), diabetic retinopathy (DR) and inherited retinal diseases, such as XL Retinitis Pigmentosa and Stargardt Disease. He has extensive experience advancing drug candidates through all stages of drug development and has been involved in numerous clinical trials as principal investigator, including the development of ranibizumab and aflibercept for wet AMD. Victor is well-suited to spearhead our suprachoroidal product development activities, including most importantly, the upcoming ODYSSEY Phase 2b wet AMD clinical trial data analysis and the planning for our Phase 3 program.” “As a retinal physician with broad clinical experience, I believe that suprachoroidal administration using Clearside’s SCS Microinjector® represents an important and innovative approach for delivery of drugs directly to the back of the eye to treat retinal diseases,” said Dr. Chong. “With the upcoming ODYSSEY data and extensive partnership portfolio, this is an exciting time to join Clearside. I look forward to working with the Clearside team to advance the CLS-AX program and develop a potential new treatment option for patients with wet AMD.” Dr. Chong served most recently as VP, Global Head of Retina DAS at Johnson & Johnson (J&J) Innovative Medicine (formerly Janssen), where he oversaw strategic decisions from preclinical to late clinical development, business development and external innovation, and coordination with the commercial organization. Prior to J&J, he was Global Head of Medicine, Retinal Health at Boehringer Ingelheim (BI), where he provided the clinical link in discovery and translational medicine and was responsible for clinical development and medical affairs in ophthalmology. His role at BI included moving 11 molecules into clinical development, covering five indications, including wet AMD, DME, DR, diabetic macular ischemia (DMI), and geographic atrophy. His laboratory was the first to link systemic complement activation, in particular C3, to AMD and he has published over 150 peer-reviewed publications. Previously, Dr. Chong served as Head of Department and Consultant Ophthalmic Surgeon of Oxford Eye Hospital, a part of the Oxford University Hospitals. He was also Co-director of Ophthalmic Education at Oxford University. Dr. Chong graduated from the University of Glasgow Medical School (MBChB) with the Neil Arnott Prize, finished his ophthalmic training at Moorfields Eye Hospital and completed a retinal fellowship at the Institute of Ophthalmology and Moorfields Eye Hospital, London. He also completed a postdoctoral cell biology fellowship at the University of Iowa. Dr. Chong holds designations as a Fellow of Royal College of Ophthalmologists (FRCOphth), and Fellow of Hong Kong Academy of Medicine (FHKAM in Ophthalmology). He earned an M.D. by research in Ophthalmology from King’s College, an MBA from Quantic School of Business and Technology and a MPhil in Cell Biology and Pathology from University College London. About Clearside Biomedical, Inc. Clearside Biomedical, Inc. is a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS®). Clearside’s SCS injection platform, utilizing the Company’s patented SCS Microinjector®, enables an in-office, repeatable, non-surgical procedure for the targeted and compartmentalized delivery of a wide variety of therapies to the macula, retina, or choroid to potentially preserve and improve vision in patients with sight-threatening eye diseases. Clearside is developing its own pipeline of small molecule product candidates for administration via its SCS Microinjector. The Company’s lead program, CLS-AX (axitinib injectable suspension), for the treatment of neovascular age-related macular degeneration (wet AMD), is in Phase 2b clinical testing. Clearside developed and gained approval for its first product, XIPERE® (triamcinolone acetonide injectable suspension) for suprachoroidal use, which is available in the U.S. through a commercial partner. Clearside also strategically partners its SCS injection platform with companies utilizing other ophthalmic therapeutic innovations. For more information, please visit clearsidebio.com and follow us on LinkedIn and TwitterX. Cautionary Note Regarding Forward-Looking Statements Any statements contained in this press release that do not describe historical facts may constitute forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements may be identified by words such as “believe”, “expect”, “may”, “plan”, “potential”, “will”, and similar expressions, and are based on Clearside’s current beliefs and expectations. These forward-looking statements include statements regarding the clinical development of CLS-AX, the expected timing of topline results from the ODYSSEY clinical trial, and the potential benefits of CLS-AX and other product candidates using Clearside’s SCS Microinjector®. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements. Risks and uncertainties that may cause actual results to differ materially include uncertainties inherent in the conduct of clinical trials, Clearside’s reliance on third parties over which it may not always have full control and other risks and uncertainties that are described in Clearside’s Annual Report on Form 10-K for the year ended December 31, 2023, filed with the U.S. Securities and Exchange Commission (SEC) on March 12, 2024 and Clearside’s other Periodic Reports filed with the SEC. Any forward-looking statements speak only as of the date of this press release and are based on information available to Clearside as of the date of this release, and Clearside assumes no obligation to, and does not intend to, update any forward-looking statements, whether as a result of new information, future events or otherwise. Investor and Media Contacts: Jenny Kobin Remy Bernarda ir@clearsidebio.com (678) 430-8206 Source: Clearside Biomedical, Inc. A photo accompanying this announcement is available at Victor Chong, M.D., MBA Victor Chong, M.D., MBA was appointed as Chief Medical Officer for Clearside Biomedical.

Tinlarebant is Belite Bio’s orally administered tablet intended to slow disease progression in patients affected with Stargardt Disease (STGD1) and Geographic Atrophy (GA) in advanced Dry Age-related Macular Degeneration (Dry AMD)Data from a 24-month Phase 2 trial in adolescent STGD1 subjects showed a sustained lower atrophic lesion growth in Tinlarebant-treated subjects compared to ProgStar participants possessing similar baseline characteristics (aged ≤18 years) (p<0.001)In the Phase 2 trial, 42% of Tinlarebant-treated subjects (5 out of 12) did not develop atrophic retinal lesions during the 24-month treatment periodEnrollment of a pivotal global Phase 3 trial of Tinlarebant in adolescent STGD1 subjects (“DRAGON”) has been completed with 104 subjects across 11 countriesInterim data from the “DRAGON” trial expected in 4Q 2024Dosed first subject in pivotal global Phase 3 trial of Tinalrebant in GA subjects (“PHOENIX”) and received approval to initiate PHOENIX trial in eight countriesAs of December 31, 2023, the Company had $88.2 million in cash.Conference Call and Webcast on Tuesday, March 12, 2024, at 4:30 p.m. ET SAN DIEGO, March 11, 2024 (GLOBE NEWSWIRE) -- Belite Bio, Inc (NASDAQ: BLTE) (“Belite Bio” or the “Company”), a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical needs, today announced its audited financial results for full-year 2023 and the unaudited and unreviewed financial results for the quarter ended December 31, 2023, and provided a business update. “2023 was a productive year for Belite, from the initiation of our Phase III trial in GA, enrollment completion of our Phase III Stargardt disease trial, to the exciting 24-month data from our Phase II Stargardt disease trial,” said Dr. Tom Lin, Chairman and CEO of Belite Bio. “These milestones continue to underscore the therapeutic potential of Tinlarebant, not only in Stargardt disease, for which there is still no approved treatment, but for patients suffering from GA, for which an oral treatment would be game changing. As we enter 2024, we remain focused on our mission to develop oral therapies for eye diseases with significant unmet medical needs and are well positioned to execute with our strong balance sheet. We expect to share additional analysis from our Phase 2 Stargardt disease trial at the annual meeting of The Association for Research in Vision and Ophthalmology (ARVO) in May, and to receive one-year interim data from our Phase 3 DRAGON trial later this year. We also look forward to sharing additional enrollment updates on our Phase III PHOENIX trial in GA as the year progresses.” Full Year 2023 Business Highlights and Upcoming Milestones: Clinical Highlights Tinlarebant (LBS-008) is designed to be an oral, potent, once-daily retinol binding protein 4 (RBP4) antagonist that decreases RBP4 levels in the blood and reduces vitamin A (retinol) delivery to the eye without disrupting systemic retinol delivery to other tissues. Vitamin A is critical for normal vision but can accumulate as toxic byproducts in individuals affected with STGD1 and GA (the advanced form of Dry AMD) leading to retinal cell death and loss of vision. Stargardt disease (STGD1): Accumulation of cytotoxic vitamin A byproducts (bisretinoids) has been implicated in the onset and progression of STGD1. Tinlarebant has been granted Fast Track and Rare Pediatric Disease (RPD) designations by the U.S. Food and Drug Administration (FDA) and orphan drug designation (ODD) in the U.S., Europe, and Japan for STGD1. There are currently no FDA-approved treatments for STGD1.LBS-008-CT02 Trial: Completed, open label, 24-month Phase 2 trial in adolescent STGD1 subjects and presented end of trial data at the American Association of Ophthalmology (AAO) annual meeting in November 2023 Tinlarebant was safe and well tolerated with no withdrawals due to adverse eventsThe 24-month data showed a sustained lower atrophic lesion growth in Tinlarebant-treated subjects compared to ProgStar participants possessing similar baseline characteristics (aged ≤18 years) (p<0.001)42% of Tinlarebant-treated subjects (5 out of 12) did not develop atrophic retinal lesionsVisual acuity was stabilized in the majority of subjects during the trial with a mean loss of five letters following 24 months of treatment (a loss of <10 letters is not considered clinically significant) DRAGON Trial: Ongoing, 24 month, randomized (2:1, active: placebo), double-masked, placebo-controlled, global, multi-center, pivotal Phase 3 trial in adolescent STGD1 subjects Completed enrollment with 104 subjects in 11 countriesPrimary efficacy endpoint is slowing of atrophic lesion growth rate; safety and tolerability will also be assessedInterim data expected in 4Q 2024 Geographic Atrophy (GA): GA, the advanced form of Dry AMD, is a chronic degenerative disease of the retina that leads to blindness in the elderly. Accumulation of toxic vitamin A byproducts (bisretinoids) has been implicated in the progression of GA. There are currently no FDA-approved, orally administered treatments for GA. PHOENIX Trial: Ongoing, 24-month, randomized (2:1, active: placebo; n~430 subjects), double-masked, placebo-controlled, global, multi-center, pivotal Phase 3 trial in patients with GA Primary efficacy endpoint is slowing of atrophic lesion growth rate; safety and tolerability will also be assessedFirst patient dosed in the third quarter of 2023Has enrolled 56 subjectsInterim data expected at mid-point of the trial Corporate Highlights Completed an underwritten public offering of American Depositary Shares of the Company (“ADSs”) and warrants (the “Follow-on Offering”) for gross proceeds of $30 million in June 2023Initiated an at-the-market offering program of additional ADSs (the “ATM Offering”) with an aggregate offering price of up to $100 million in June 2023Raised $22 million from the exercise of warrants issued in the Follow-on Offering and $29 million from ATM Offering as of December 31, 2023 Audited Full Year 2023 and Unaudited and Unreviewed Fourth Quarter 2023 Financial Results: Cash: As of December 31, 2023, the Company had $88.2 million in cash, as compared with $42.1 million on December 31, 2022. R&D Expenses: For the three months ended December 31, 2023, research and development expenses were $4.9 million compared to $5.2 million for the same period in 2022. The decrease resulted primarily from higher R&D expenses in the fourth quarter of 2022 as the DRAGON trial reached certain milestones in such quarter. For the year ended December 31, 2023, research and development expenses were $24.8 million compared to $8.9 million for the same period in 2022. The increase in research and development expenses was primarily attributable to increases in expenses related to conducting the DRAGON and PHOENIX trials. G&A Expenses: For the three months ended December 31, 2023, general and administration expenses were $2.1 million compared to $1.5 million for the same period in 2022. The increase resulted primarily from an increase in share-based compensation granted in 2023. For the year ended December 31, 2023, general and administration expenses were $6.8 million compared to $4.0 million for the same period in 2022. The increase resulted also primarily from an increase in share-based compensation granted in 2023 and an increase in professional service fees. Net Loss: For the three months ended December 31, 2023, the Company reported a net loss of $7.0 million or ($0.25) per share compared to $6.8 million or ($0.27) per share for the same period in 2022. For the year ended December 31, 2023, the Company reported a net loss of $31.6 million or ($1.19) per share, compared to a net loss of $12.6 million or ($0.63) per share for the same period in 2022. Webcast Information Belite Bio will host a webcast on Tuesday, March 12, 2023, at 4:30 p.m. Eastern Time to discuss the Company’s financial results and provide a business update. To join the webcast, please visit https://wsw.com/webcast/cc/blte2/1419894. A replay will be available for approximately 90 days following the event at the Company’s Investor Relations website at https://investors.belitebio.com/presentations-events/events. About Belite Bio Belite Bio is a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting retinal degenerative eye diseases which have significant unmet medical needs such as (i) atrophic age-related macular degeneration (AMD), commonly known as Geographic Atrophy (GA) in advanced dry AMD, and (ii) autosomal recessive Stargardt disease type 1, or STGD1, in addition to specific metabolic diseases. For more information, follow us on Twitter, Instagram, LinkedIn, Facebook, or visit us at www.belitebio.com. Important Cautions Regarding Forward Looking Statements This press release contains forward-looking statements about future expectations and plans, as well as other statements regarding matters that are not historical facts. These statements include but are not limited to statements regarding the potential implications of clinical data for patients, and Belite Bio’s advancement of, and anticipated preclinical activities, clinical development, regulatory milestones, and commercialization of its product candidates, and any other statements containing the words “expect”, “hope” and similar expressions. Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including but not limited to Belite Bio’s ability to demonstrate the safety and efficacy of its drug candidates; the clinical results for its drug candidates, which may not support further development or regulatory approval; the timing to complete relevant clinical trials and/or to receive the interim/final data of such clinical trials; the content and timing of decisions made by the relevant regulatory authorities regarding regulatory approval of Belite Bio’s drug candidates; the potential efficacy of Tinlarebant, as well as those risks more fully discussed in the “Risk Factors” section in Belite Bio’s filings with the U.S. Securities and Exchange Commission. All forward-looking statements are based on information currently available to Belite Bio, and Belite Bio undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law. BELITE BIO, INCCONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS(Amounts in thousands of US Dollars, except share and per share amounts) For the Year Ended December 31, 2022 2023 (Audited) (Audited) Expenses Research and development 8,869 24,844 General and administrative 3,952 6,824 Total operating expenses 12,821 31,668 Loss from operations (12,821) (31,668)Other income Total other income, net 173 45 Loss before income tax (12,648) (31,623)Income tax expense - 9 Net loss (12,648) (31,632)Other comprehensive income (loss) Foreign currency translation adjustments, net of nil tax (196) 18 Total comprehensive loss (12,844) (31,614)Weighted average number of ordinary shares used in per share calculation: - Basic and Diluted 19,976,596 26,593,673 Net loss per ordinary share - Basic and Diluted $(0.63) $(1.19) For the Three Months Ended December 31, 2022 2023 (Unaudited and Unreviewed) (Unaudited and Unreviewed) Expenses Research and development 5,226 4,862 General and administrative 1,495 2,093 Total operating expenses 6,721 6,955 Loss from operations (6,721) (6,955)Other expense Total other expense, net (62) (36)Loss before income tax (6,783) (6,991)Income tax expense - - Net loss (6,783) (6,991)Other comprehensive income (loss) Foreign currency translation adjustments, net of nil tax 127 133 Total comprehensive loss (6,656) (6,858)Weighted average number of ordinary shares used in per share calculation: - Basic and Diluted 24,889,136 28,316,251 Net loss per ordinary share - Basic and Diluted $(0.27) $(0.25) BELITE BIO, INCCONSOLIDATED BALANCE SHEETS(Amounts in thousands of US Dollars, except share amounts) December 31 2022 2023 (Audited) (Audited) ASSETS Current Assets Cash $42,089 $88,157 Other receivables — 818 Prepayments and other current assets 716 947 Other receivables due from related parties 2 18 Total current assets 42,807 89,940 Property and equipment, net 541 490 Prepayments and other non-current assets 31 3,297 Security deposits 88 104 Operating lease right-of-use asset, net 806 811 TOTAL ASSETS $44,273 $94,642 LIABILITIES AND SHAREHOLDERS’ EQUITY Current liabilities Accrued expenses and other liabilities 1,906 3,325 Operating lease liabilities – current 198 308 Total current liabilities 2,104 3,633 Non-current liabilities Operating lease liabilities –non – current 668 578 TOTAL LIABILITIES 2,772 4,211 Shareholders’ equity Ordinary shares, par value of US$0.0001 per share; 400,000,000 shares authorized; 24,898,908 and 29,184,475 shares issued; 24,898,908 and 29,149,444 shares outstanding as of December 31, 2022 and 2023, respectively 3 3 Additional paid-in capital 81,761 162,305 Accumulated other comprehensive loss (392) (374)Accumulated deficit (39,871) (71,503)Total shareholders’ equity 41,501 90,431 TOTAL LIABILITIES AND SHAREHOLDERS’ EQUITY $44,273 $94,642 Media and Investor Relations Contact:Jennifer Wuir@belitebio.comJulie Fallonbelite@argotpartners.com

Encinitas, California--(News - March 8, 2024) - Kiora Pharmaceuticals, Inc. (NASDAQ: KPRX), ("Kiora" or the "Company") announced the publication of results from a Phase 1 double-masked study of KIO-101, a potent, non-steroidal anti-inflammatory agent. KIO-101 contains the same active molecule as KIO-104, which belongs to a family of potent anti-inflammatory agents, The study showed that a 12-day treatment of KIO-101 topically at multiple doses was well tolerated in healthy volunteers and patients with inflammation of the eye. There was a significant decrease in conjunctival hyperemia in the treatment group compared to the placebo group. Conjunctival hyperemia, a sign of ocular inflammation, is due to a variety of non-infectious and infectious causes. The paper was published in the journal, Pharmaceutics (2024, 16(3), 367 | ) Kiora's KIO-100 family of compounds contain the same proprietary active molecule but are uniquely formulated for a specific condition and region of the eye being targeted. KIO-101 is the designation for topical delivery and KIO-104 is the designation for intravitreal injection (directly into the eye). Kiora's active molecule belongs to a class of drugs known as DHODH inhibitors, including teriflunomide, an FDA-approved drug that has been prescribed to hundreds of thousands of patients with multiple sclerosis. DHODH inhibitors are believed to reduce the production of T-cells, a type of immune cell that, in certain conditions like autoimmune disease, can cause damaging inflammation. Delivering KIO-101 or KIO-104 locally in the eye aims to reduce T-cell-related inflammation without the associated potential side effects of systemic anti-inflammatory drugs. "These results support the therapeutic potential of our molecule to address inflammation in the eye," explained Eric Daniels, MD, Chief Development Officer, Kiora. "A Phase 2 trial of KIO-104 is expected to start later this year to treat posterior non-infectious uveitis, a rare inflammatory condition of the retina. Because uveitis can lead to vision loss, there's an important need for new treatment options beyond chronic steroid injections or systemic autoimmune disease drugs currently being used today. Beyond uveitis, we see an opportunity to target additional retinal inflammatory diseases that could similarly benefit from a non-steroidal therapeutic option." The published study involved a double-masked, placebo-controlled, randomized, parallel-group design that consisted of two parts. In Part I, 24 healthy volunteers received single or multiple administrations of KIO-101 eye drops in ascending doses (0.05%, 0.15% or 0.30%). Part II involved 21 patients with conjunctival hyperemia who received either 0.15% KIO-101 eye drops or vehicle (2:1) twice daily for 12 consecutive days. The findings include the following: KIO-101 eye drops were well tolerated in all subjects. There were no serious adverse events. All adverse events were transient and considered mild to moderate. In the highest dose cohort (0.30%), epistaxis occurred in two subjects after multiple instillations. In Part II, after 12 days of treatment with 0.15% KIO-101, conjunctival hyperemia decreased by 1.1 ± 0.27 points in the KIO-101 treated group versus 0.6 ± 0.79 points in the placebo group (p = 0.0385). From baseline to day 12, the Ocular Surface Disease Index Questionnaire (OSDI) score decreased by 20.3 points in the treatment group, while in the placebo group decreased by 14.0 points (p=ns). Kiora is planning a Phase 2 clinical trial of KIO-104 to be administered by intravitreal injection directly into the retina for the treatment of posterior non-infectious uveitis. The Company previously reported positive results from a Phase 1 study of KIO-104 for treatment of posterior non-infectious uveitis. Posterior non-infectious uveitis is a rare autoimmune disease characterized by inflammation of the retina, choroid, vitreous, or optic nerve, and can result in severe vision loss. It can result from many underlying causes, which may include the ocular manifestation of certain autoimmune diseases. About Kiora Pharmaceuticals Kiora Pharmaceuticals is a clinical-stage biotechnology company developing and commercializing products for the treatment of orphan retinal diseases. KIO-301 is being developed for the treatment of retinitis pigmentosa, choroideremia, and Stargardt disease. It is a molecular photoswitch that has the potential to restore vision in patients with inherited and/or age-related retinal degeneration. KIO-104 is being developed for the treatment of posterior non-infectious uveitis. It is a next-generation, non-steroidal, immuno-modulatory, and small-molecule inhibitor of dihydroorotate dehydrogenase. In addition to news releases and SEC filings, we expect to post information on our website, , and social media accounts that could be relevant to investors. We encourage investors to follow us on Twitter and LinkedIn as well as to visit our website and/or subscribe to email alerts. Forward-Looking Statements Some of the statements in this press release are "forward-looking" and are made pursuant to the safe harbor provision of the Private Securities Litigation Reform Act of 1995. These "forward-looking" statements include statements relating to, among other things, the potential of the KIO-100 family to address inflammation in the eye, the expected timing of a Phase 2 trial of KIO-104, and the development and commercialization efforts and other regulatory or marketing approval efforts pertaining to Kiora's development-stage products, including KIO-104, as well as the success thereof, where such approvals or success may not be obtained or achieved on a timely basis or at all. These statements involve risks and uncertainties that may cause results to differ materially from the statements set forth in this press release, including, among other things, the ability to conduct clinical trials on a timely basis, the ability to obtain any required regulatory approvals, whether future trials will yield similar results for participants, market and other conditions, and certain risk factors described under the heading "Risk Factors" contained in Kiora's Annual Report on Form 10-K filed with the SEC on March 23, 2023, or described in Kiora's other public filings. Kiora's results may also be affected by factors of which Kiora is not currently aware. The forward-looking statements in this press release speak only as of the date of this press release. Kiora expressly disclaims any obligation or undertaking to release publicly any updates or revisions to such statements to reflect any change in its expectations with regard thereto or any changes in the events, conditions, or circumstances on which any such statement is based, except as required by law. Contact Information: Investors@kiorapharma.com To view the source version of this press release, please visit