Dystrophy, Macular

PLANEGG-MARTINSRIED, Germany I January 20, 2025 I Formycon AG (FSE: FYB, Prime Standard, “Formycon”) and its licensing partner Klinge Biopharma GmbH (“Klinge”) today jointly announce that the European Commission has granted central marketing authorization for FYB203 (Aflibercept), a biosimilar to Eylea ® 1 , under the brand names AHZANTIVE ® 2 and Baiama ® 3 . The approval encompasses the treatment of Age-Related Neovascular (wet) Macular Degeneration (nAMD) and other serious retinal diseases such as Diabetic Macular Edema (DME), visual impairment due to Myopic Choroidal Neovascularisation (CNV) and Macular Edema following Retinal Vein Occlusion (RVO). The decision of the European Commission follows the positive opinion of the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) from November 2024 and applies to all countries in the European Economic Area (EEA), including the 27 member states of the European Union (EU) as well as Iceland, Liechtenstein, and Norway. Dr. Stefan Glombitza, CEO of Formycon AG , commented: “The EU approval of FYB203, our biosimilar for Eylea ® , marks another milestone for Formycon and is based on the expertise and dedication of our entire team. As our second ophthalmic biosimilar, FYB203 significantly expands therapeutic options for patients with severe retinal diseases. With AHZANTIVE ® and Baiama ® , we are improving access to high-quality and affordable therapies that contribute sustainably to enhancing patients’ quality of life.” Aflibercept inhibits the vascular endothelial growth factor (VEGF), which is responsible for the excessive formation of blood vessels in the retina, thereby impairing vision. In 2023, the reference product Eylea ® achieved global sales of approximately USD 9 billion 4 , highlighting the significance and necessity of a cost-effective alternative like FYB203. In mid-January 2025, Formycon and Teva Pharmaceuticals International GmbH (Teva) signed a licensing agreement for the semi-exclusive commercialization of FYB203 across major parts of Europe and Israel. Concurrently, Formycon has concluded an agreement with Teva for product supply. FYB203 was already approved by the U.S. Food and Drug Administration (FDA) in June 2024. 1) Eylea ® is a registered trademark of Regeneron Pharmaceuticals Inc. 2) AHZANTIVE ® is a registered trademark of Klinge Biopharma GmbH 3) Baiama ® is a registered trademark of Klinge Biopharma GmbH 4) Source: https://investor.regeneron.com/news-releases/news-release-details/regeneron-reports-fourth-quarter-and-full-year-2023-financial/ About Formycon: Formycon AG (FSE: FYB) is a leading, independent developer of high-quality biosimilars, follow-on products of biopharmaceutical medicines. The company focuses on therapies in ophthalmology, immunology, immuno-oncology and other key disease areas, covering almost the entire value chain from technical development through clinical trials to approval by the regulatory authorities. For commercialization of its biosimilars, Formycon relies on strong, well-trusted and long-term partnerships worldwide. With FYB201/Ranibizumab, Formycon already has a biosimilar on the market in Europe, the USA, and the MENA-region. Two further biosimilars, FYB202/ustekinumab and FYB203/aflibercept, received FDA and European Commission approval; FYB202 is also approved in the UK and Canada. Another four biosimilar candidates are currently in development. With its biosimilars, Formycon is making an important contribution to providing as many patients as possible with access to highly effective and affordable medicines. Formycon AG, headquartered in Munich, is listed in the Prime Standard of the Frankfurt Stock Exchange: FYB / ISIN: DE000A1EWVY8 / WKN: A1EWVY and is part of the SDAX and TecDAX selection indices. Further information can be found at: https://www.formycon.com/ About Biosimilars: Since their introduction in the 1980s, biopharmaceutical drugs have revolutionized the treatment of serious and chronic diseases. By 2032, many of these drugs will lose their patent protection – including 45 blockbusters with an estimated total annual global turnover of more than 200 billion US dollars. Biosimilars are successor products to biopharmaceutical drugs for which market exclusivity has expired. They are approved in highly regulated markets such as the EU, the USA, Canada, Japan and Australia in accordance with strict regulatory procedures. Biosimilars create competition and thus give more patients access to biopharmaceutical therapies. At the same time, they reduce costs for healthcare providers. Global sales of biosimilars currently amount to around 21 billion US dollars. Analysts assume that sales could rise to over 74 billion US dollars by 2030. SOURCE: Formycon

MALVERN, PA, USA I January 16, 2025 I Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that the first patient has been dosed in the OCU200 Phase 1 clinical trial for diabetic macular edema (DME). “OCU200 has the potential to change the treatment landscape for DME, diabetic retinopathy (DR), and wet age-related macular degeneration (wet AMD) with its unique mechanism of action, binding the active component—tumstatin—to integrin receptors that play a crucial role in disease pathogenesis,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-Founder of Ocugen. “OCU200 holds the promise to benefit all DME patients, including the 30-40% of patients who do not respond to current anti-VEGF therapies.” The OCU200 Phase 1 clinical trial is a multicenter, open-label, dose-escalation study to assess drug safety via intravitreal injection in three cohorts: low dose (0.025 mg), medium dose (0.05 mg), and high dose (0.1 mg). All subjects will receive a total of two intravitreal injections of OCU200 six weeks apart. Patient follow-up will take place up to three months after the last injection. Approximately 12 million people in the United States and 130 million people worldwide are affected by DME, DR or wet AMD. Patients affected by these diseases share common symptoms, such as blurriness in vision and progressive vision loss as the disease progresses. The formation of fragile and leaky new blood vessels leads to fluid accumulation in and around the retina, causing damage to vision. “I am seeing an increasing rate of vision-threatening diseases associated with diabetes at my clinic and am eager to provide a new therapeutic option to these patients,” said Dr. David Almedia, Vitreoretinal Surgeon and Clinician Scientist, President and CEO of Erie Retina Research, and Founder and President of Case X Global in Erie, Pennsylvania. “There remains a considerable unmet medical need for DME and DR patients with currently available anti-VEGF treatments.” OCU200 is a recombinant fusion protein that consists of two parts connected by a linker: tumstatin, the active component, acts as an anti-inflammatory, anti-VEGF agent by binding to integrin receptors; and transferrin, which targets the drug to the choroid and retina by binding transferrin receptors on endothelial cells. These features will potentially enable OCU200 to reduce the vascular permeability, inflammation, and neovascularization that drive the pathophysiology of DME, DR, and wet AMD at a significantly lower dose compared to currently approved therapies. “We are enthusiastic about getting patients started in the OCU200 Phase 1 clinical trial and sharing not only safety but preliminary efficacy data as the study progresses,” said Dr. Huma Qamar, Chief Medical Officer at Ocugen. “OCU200 brings an innovative biologic candidate to Ocugen’s ophthalmology portfolio targeting blindness diseases.” The Company intends to pursue approval to use OCU200 as a first-line therapy for DME, DR, and wet AMD. About Ocugen, Inc. Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patients’ lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on X and LinkedIn . SOURCE: Ocugen

Jan. 14, 2025 -- PulseSight Therapeutics SAS, an ophthalmology biotech company developing disruptive non-viral vectorized therapies with minimally-invasive delivery technology, today announces it has submitted a Clinical Trial Authorization (CTA) to Agence Nationale de Sécurité du Médicament et des produits de santé (ANSM) for a Phase I trial (PST-611-CT1) assessing the safety and tolerability of PST-611 in humans. PST-611-CT1 is a first-in-human single ascending dose trial aiming to confirm the favorable safety profile of the drug and validate the maximal dose for the Phase II proof-of-concept study, in six to twelve dry age-related macular degeneration (AMD)/geographic atrophy (GA) patients. The study PST-611-CT1 is expected to start early Q2 2025 with a readout anticipated by the end of 2025 or early 2026. AMD is the leading cause of central vision loss in the elderly, affecting 200 million people worldwide. AMD's pathogenesis is complex and involves the dysregulation of iron homeostasis, leading to an excess of free iron, which results in inflammation, oxidative stress, and cell death. PST-611 is a first-in-class non-viral vectorized therapy for the treatment of dry AMD/GA, expressing human transferrin, a highly potent iron regulator, to restore normal iron homeostasis. The PST-611-CT1 Phase I trial builds on PulseSight’s previous clinical demonstration of the favorable safety profile of its electrotransfection delivery system and plasmid technology, as well as preclinical experiments demonstrating the beneficial effects of PST-611 in reducing oxidative stress and inflammation, and preserving the integrity of the retinal pigment epithelium, with the potential of preventing retinal degeneration and vision loss. Judith Greciet, CEO of PulseSight Therapeutics, said, “This first in human study is a significant milestone for the clinical development of PST-611, which we believe holds the potential to become a major new treatment option for patients with dry AMD/GA. Our goal is to confirm the safety of our drug candidate to then rapidly move into a Phase II proof-of-concept study, to demonstrate transferrin’s ability to protect retinal cells from atrophy and preserve vision.” Dirk Sauer, Chairman of the Board of PulseSight Therapeutics, said, “This is a pivotal moment as we progress towards clinical studies with our lead program PST-611. PST-611 has shown unique features in terms of efficacy, safety and durability of effect in a robust preclinical plan. This program is the first milestone in the validation of our candidate’s potential to be truly life-changing for dry AMD/GA patients.” PulseSight is supported in its clinical trial execution by expert CRO ILife Consulting. AMD develops with aging. It is a disease with progressive, painless loss of central vision with a strong burden on patients’ everyday life, impacting their ability to read, recognize faces and see objects, and is ultimately leading to irreversible central vision loss. Dry AMD is the most common form of AMD, progressing through successive stages into the late form of dry AMD also called geographic atrophy (GA). Wet AMD is a less common type of late AMD causing faster vision loss. Any stage of dry AMD can turn into wet AMD. In all its forms, AMD represents a compelling unmet need for more effective and durable treatment options, with a large and growing market, estimated to reach $27.5 Billion by 2031. PulseSight is clinical-stage biotech company committed to developing disruptive non-viral vectorized therapies with minimally-invasive delivery technology to protect and improve the vision of patients with retinal disease with a focus on age-related macular degeneration (AMD) including wet AMD and geographic atrophy (GA) secondary to dry AMD. Already clinically validated for its safety and sustained activity, PulseSight’s technology platform delivers DNA plasmids encoding therapeutic proteins into the ciliary muscle using an electro-transfection system. The ciliary muscle cells act as biofactories, expressing therapeutic proteins that reach the retina with high distribution, providing a safe and long-lasting treatment for major eye diseases. PST-611 encodes the human transferrin protein, a crucial regulator of iron homeostasis and holds the potential to effectively address key pathological mechanisms in dry AMD/GA, whilst requiring re-treatment only every four to six months. This program is expected to enter the clinic by Q2 2025. The content above comes from the network. if any infringement, please contact us to modify.