Retinal Dystrophies

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.

Subretinal injection of ATSN-201 was well tolerated in all patients in first cohort with no serious adverse events reported Enrollment is ongoing in first clinical trial utilizing AAV.SPR, the company’s novel spreading capsid March 13, 2024 -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced dosing has been initiated in the second cohort of the LIGHTHOUSE study, a Phase I/II clinical trial evaluating subretinal injection of ATSN-201 for the treatment of X-linked retinoschisis (XLRS). ATSN-201, a best-in-class gene therapy product candidate, leverages AAV.SPR, the company’s novel spreading capsid, to achieve therapeutic levels of gene expression in photoreceptors of the central retina while avoiding the surgical risks of foveal detachment. ATSN-201 was well tolerated in all three XLRS patients in the first cohort and no serious adverse events were reported. The Data and Safety Monitoring Board recommended proceeding with the second cohort, in which patients will receive the next dose level of ATSN-201. "We are encouraged by the excellent safety observed with the low dose of ATSN-201 in the LIGHTHOUSE study, which represents the first clinical utilization of AAV.SPR. Additionally, initial indications of structural and functional improvement observed within and beyond the injection areas suggest successful lateral spread of the vector,” said Kenji Fujita, MD, Chief Medical Officer of Atsena Therapeutics. “Advancing into the second cohort brings us one step closer to potentially bringing a much-needed one-time treatment to patients with XLRS, an inherited retinal disease that causes progressive vision loss and can lead to blindness.” The LIGHTHOUSE study is a Phase I/II, open-label, dose-escalation and dose-expansion clinical trial evaluating the safety and tolerability of ATSN-201 in male patients ages 6 and older with a clinical diagnosis of XLRS caused by pathogenic or likely pathogenic mutations in RS1. Enrollment in the LIGHTHOUSE study is ongoing. For more information, visit ClinicalTrials.gov (Identifier: NCT05878860). XLRS is a monogenic X-linked disease caused by mutations in the RS1 gene which encodes retinoschisin, a protein secreted primarily by photoreceptors. RS1 is localized to the extracellular surface of rods, cones, and bipolar cells. XLRS is characterized by schisis, or abnormal splitting of retinal layers, which causes impaired visual acuity that is not correctable with glasses and leads to progressive vision loss and ultimately blindness. XLRS primarily affects males and is typically diagnosed in early childhood. Approximately 30,000 males in the U.S. and EU have XLRS, for which there are currently no approved treatments. AAV.SPR, one of Atsena’s novel capsids, spreads laterally beyond the subretinal injection site to enable safe and efficient transduction of the central retina (where schisis cavities predominate in XLRS patient retinas) when injected into areas outside the macula. A preclinical study in non-human primates demonstrated that AAV.SPR promotes transgene expression well beyond subretinal injection bleb margins. This is in stark contrast to benchmark AAV vectors, which remain confined to the original bleb margins. At clinically relevant doses, AAV.SPR efficiently transduces foveal cones without the need for surgical detachment and does not cause inflammation. For more information about the preclinical study and how AAV.SPR works, visit https://atsenatx.com/our-approach/laterally-spreading-aav/. Atsena Therapeutics is a clinical-stage gene therapy company developing best-in-class treatments for the reversal or prevention of blindness from inherited retinal diseases. The company’s lead program is evaluating ATSN-201 in an ongoing Phase I/II clinical trial for X-linked retinoschisis (XLRS), a progressive genetic condition affecting boys and men that is typically diagnosed in childhood. Another ongoing Phase I/II clinical trial is evaluating ATSN-101 for Leber congenital amaurosis type 1 (LCA1), one of the most common causes of blindness in children. Atsena’s pipeline is powered by novel adeno-associated virus (AAV) technology tailored to overcome the hurdles presented by inherited retinal diseases. Founded by pioneers in ocular gene therapy, Atsena is led by an experienced team dedicated to addressing the needs of patients with vision loss. The content above comes from the network. if any infringement, please contact us to modify.

March 12, 2024 -- Arctic Therapeutics ehf, an Icelandic drug development company and partner Nacuity Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company developing treatments for retinitis pigmentosa, cataracts and other diseases caused by oxidative stress, today announced approval by the European Medicines Agency (EMA) for initiation of the first clinical trial evaluating AT-001 (Nacuity’s NPI-001) for the treatment of Hereditary Cystatin C Amyloid Angiopathy (HCCAA) in Iceland. HCCAA is an ultra rare, serious condition characterized by the accumulation of amyloid in the brain's blood vessels, which can lead to cerebral hemorrhage, stroke, and progressive neurological deterioration, including dementia. Current treatment options are severely limited, focusing primarily on symptom management rather than addressing the underlying cause of the disease. AT-001 is a differentiated, purified, prescription-quality, GMP-grade N-acetylcysteine amide (NACA). “EMA's approval paves the way for a comprehensive investigation into the safety and efficacy of a novel therapeutic approach designed to halt or reverse the progression of HCCAA. Our study aims to explore the potential of AT-001 in modifying the disease's course, improving patient outcomes, and ultimately, saving lives,” said Ivar Hakonarson, CEO and co-founder of Arctic. In September 2022, Nacuity and Arctic entered into an exclusive licensing agreement and strategic partnership for the development and commercialization of Nacuity’s NPI-001 under the name AT-001 for HCCAA in Iceland and related forms of amyloid angiopathies (CAAs) worldwide. Nacuity is also currently evaluating NPI-001 in Phase 1/2 clinical trials in Australia for retinitis pigmentosa associated with Usher syndrome and cystinosis. “Our proprietary NPI-001 has potential to address the myriad diseases and conditions where oxidative stress plays a role. EMA approval for the evaluation of NPI-001 tablets for treatment of HCCAA represents most importantly, a step forward in addressing HCCAA, a life threatening and underserved rare disease, and secondly, a key pharmaceutical quality milestone for the companies in the global development of NPI-001,” said G. Michael Wall, Ph.D., SVP & CSO, Nacuity. Nacuity Pharmaceuticals, Inc. (http://www.nacuity.com/), is a clinical-stage leader in innovative treatments for oxidative stress. The company’s powerful, targeted therapies aim to attentuate oxidative tissue damage, a driver of blinding eye diseases and a broad spectrum of serious chronic conditions. Nacuity has three highly differentiated clinical programs ongoing in retinitis pigmentosa, cataracts and cystinosis with the potential to be first-of-a-kind therapies and gateways to wider applications. Nacuity has operations in Fort Worth, TX, USA, and Australia, and extensive managerial and scientific domain expertise as well as backing from Foundation Fighting Blindness (https://www.fightingblindness.org/) and its venture arm RD Fund (https://www.retinaldegenerationfund.org). Arctic Therapeutics ehf (AT) (https://www.arctictherapeutics.com/) is an Icelandic drug development company, established in 2015 as a spin-off from the US-based Center for Applied Genomics (CAG), a research center at the Children's Hospital of Philadelphia. AT leverages the transformative power of applied genomics, steering the course of drug development towards safer, more effective treatments for some of the world’s most challenging diseases. The company has operations in Iceland, USA, and multiple collaborations across Europe. The content above comes from the network. if any infringement, please contact us to modify.