Ang2

Vabysmo PFS is the first and only prefilled syringe containing a bispecific antibody, offering a convenient alternative to currently available Vabysmo vials Vabysmo has demonstrated rapid and robust vision and anatomical improvements in neovascular age-related macular degeneration (nAMD), diabetic macular edema (DME) and retinal vein occlusion (RVO) The ready-to-use Vabysmo PFS is co-packaged with the only CE-marked needle specifically designed for intravitreal injection 13 December 2024– Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the European Medicines Agency has approved Vabysmo® (faricimab) 6.0 mg single-dose prefilled syringe (PFS) for use in the treatment of neovascular or ‘wet’ age-related macular degeneration (nAMD), diabetic macular edema (DME) and macular edema following retinal vein occlusion (RVO). Together, these three conditions affect more than nine million people in the European Union (EU) and can have a devastating impact – physically, emotionally, and economically – on those affected, their families and caregivers.1-5 “Approval of the Vabysmo prefilled syringe in the EU offers a convenient way for ophthalmologists to administer this treatment for people with three of the most common causes of vision loss,” said Levi Garraway, MD, PhD, Roche’s Chief Medical Officer and Head of Global Product Development. “This simplified administration may thereby help reduce the treatment burden for patients and retina specialists.” The Vabysmo PFS provides ophthalmologists with the first and only CE-labelled needle for intravitreal injection. Vabysmo PFS delivers the same medicine as the currently available 6.0 mg Vabysmo vials in an alternative, ready-to-use format.6 More than five million doses of Vabysmo have been distributed globally since its initial US approval in 2022.6-11 Vabysmo PFS was first approved for nAMD, DME and RVO by the United States Food and Drug Administration in July 2024.7 Vabysmo PFS will be the European Union’s first and only prefilled syringe containing a bispecific antibody to treat retinal conditions that can cause blindness. Vabysmo is the first bispecific antibody approved for the eye.7,8,12 It targets and inhibits two signalling pathways linked to a number of vision-threatening retinal conditions by neutralising angiopoietin-2 (Ang-2) and vascular endothelial growth factor-A (VEGF-A). Ang-2 and VEGF-A contribute to vision loss by destabilising blood vessels, causing new leaky blood vessels to form and increasing inflammation. By blocking pathways involving Ang-2 and VEGF-A, Vabysmo is designed to stabilise blood vessels.12,13 Vabysmo is approved in more than 100 countries around the world, including the United States (US), Japan, the United Kingdom and the European Union (EU) for people with neovascular or ‘wet’ age-related macular degeneration and diabetic macular edema, and in more than 30 countries, including the US, EU and Japan, for people with macular edema following retinal vein occlusion. Review by other health authorities is ongoing.6-11 Roche is focused on saving people’s eyesight from the leading causes of vision loss through pioneering therapies. Through our innovation in the scientific discovery of new potential drug targets, personalised healthcare, molecular engineering, biomarkers and continuous drug delivery, we strive to design the right therapies for the right patients. We have the broadest retina pipeline in ophthalmology, which is led by science and informed by insights from people with eye diseases. Our pipeline includes innovative treatments across different modalities, such as antibodies, and gene and cell therapies targeting multiple vision-threatening conditions, including retinal vascular and diabetic eye diseases, geographic atrophy, and autoimmune conditions, such as thyroid eye disease and uveitic macular edema. Applying our extensive experience, we have already brought breakthrough ophthalmic treatments to people living with vision loss. Susvimo® (previously called Port Delivery System with ranibizumab) 100 mg/mL for intravitreal use via ocular implant is the first United States Food and Drug Administration-approved refillable eye implant for neovascular or ‘wet’ age-related macular degeneration that continuously delivers a customised formulation of ranibizumab over a period of months.14,15 Vabysmo® (faricimab) is the first bispecific antibody approved for the eye, which targets and inhibits two signalling pathways linked to a number of vision-threatening retinal conditions by neutralising angiopoietin-2 and vascular endothelial growth factor-A.7,8,12,13 Vabysmo is approved around the world for people living with nAMD, diabetic macular edema and macular edema following retinal vein occlusion.6-11 Lucentis® (ranibizumab injection)* was the first treatment approved to improve vision in people with certain retinal conditions.16 Founded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world’s largest biotechnology company and the global leader in in-vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in Diagnostics and Pharma with data insights from the clinical practice. For over 125 years, sustainability has been an integral part of Roche’s business. As a science-driven company, our greatest contribution to society is developing innovative medicines and diagnostics that help people live healthier lives. Roche is committed to the Science Based Targets initiative and the Sustainable Markets Initiative to achieve net zero by 2045. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. References [1] National Institute for Health and Care Excellence. Macular degeneration – age-related: how common is it? [Internet; cited December 2024]. Available from: https://cks.nice.org.uk/topics/macular-degeneration-age-related/background-information/prevalence. [2] Bright Focus Foundation. Age-related macular degeneration: facts & figures. [Internet; cited December 2024]. Available from: https://www.brightfocus.org/macular/article/age-related-macular-facts-figures. [3] European Commission. Diabetes prevention. [Internet; cited December 2024]. Available from: https://knowledge4policy.ec.europa.eu/health-promotion-knowledge-gateway/diabetes-prevention. [4] Im JHB, et al. Prevalence of diabetic macular edema (DME) based on optical coherence tomography in people with diabetes: A systematic review and meta-analysis. Survey of Ophthalmology. 2022 Jul-Aug;67(4):1244-1251. [5] Li JQ, et al. Prevalence of retinal vein occlusion (RVO) in Europe: a systematic review and meta-analysis. Ophthalmologica. 2019;241(4):183-189. [6] European Medicines Agency. Summary of product characteristics, Vabysmo. 2024. [Internet; cited December 2024]. Available from: https://www.ema.europa.eu/en/documents/product-information/vabysmo-epar-product-information_en.pdf. [7] United States (US) Food and Drug Administration (FDA). Highlights of prescribing information, Vabysmo. 2024 [Internet; cited December 2024]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/761235s005lbl.pdf. [8] Medicines and Healthcare products Regulatory Agency approves faricimab through international work-sharing initiative. [Internet; cited December 2024]. Available from: https://www.gov.uk/government/news/mhra-approves-faricimab-through-international-work-sharing-initiative. [9] Chugai obtains regulatory approval for Vabysmo, the only bispecific antibody in the ophthalmology field, for additional indication of macular edema associated with RVO. [Internet; cited December 2024]. Available from: https://www.chugai-pharm.co.jp/english/news/detail/20240326160000_1054.html. [10] Chugai obtains regulatory approval for Vabysmo, the first bispecific antibody in ophthalmology, neovascular age-related macular degeneration (nAMD) and DME. [Internet; cited December 2024]. Available from: https://www.chugai-pharm.co.jp/english/news/detail/20220328160002_909.html. [11] Roche data on file. [12] Heier JS, et al. Efficacy, durability, and safety of intravitreal faricimab up to every 16 weeks for nAMD (TENAYA and LUCERNE): two randomised, double-masked, phase III, non-inferiority trials. The Lancet. 2022; 399:729-40. [13] Wykoff C, et al. Efficacy, durability, and safety of intravitreal faricimab with extended dosing up to every 16 weeks in patients with DME (YOSEMITE and RHINE): Two randomised, double-masked, phase III trials. The Lancet. 2022; 399:741-755. [14] US FDA. Highlights of prescribing information, Susvimo. 2021. [Internet; cited December 2024]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/761197s000lbl.pdf. [15] Holekamp N, et al. Archway randomised phase III trial of the Port Delivery System with ranibizumab for nAMD. Ophthalmology. 2021. [16] US FDA. Highlights of prescribing information, Lucentis. 2014. [Internet; cited December 2024]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2012/125156s0069s0076lbl.pdf. The content above comes from the network. if any infringement, please contact us to modify.

Roche’s blockbuster Vabysmo is vying for ophthalmological dominance against Regeneron’s Eylea. Image credit: Shutterstock / Marlon Trottmann. The European Medicines Agency (EMA) has approved Roche’s Vabysmo (faricimab) in single-dose prefilled syringe form for three retinal conditions that cause blindness. Roche’s ready-to-use drug is indicated for the treatment of neovascular or ‘wet’ age-related macular degeneration (nAMD), diabetic macular oedema (DME) and macular oedema following retinal vein occlusion (RVO), as per a 13 December press release. Shares in Roche opened 0.5% down at market open on 13 December, likely due to the separate event of Saratoga Research & Investment Management decreasing its position in the pharma company yesterday by 3.5%. The Swiss drugmaker has a market cap of $203.1bn. Blockbuster Vabysmo has already been available in Europe for the same three diseases in 6.0mg Vabysmo vials. The drug received its most recent approval in July this year when RVO was added to its EU label. Both the prefilled syringe and vials deliver faricimab, a bispecific antibody that blocks vascular endothelial growth factor-A (VEGF-A) and angiopoietin-2 (Ang-2). Blocking these two protein pathways stabilises blood vessels and prevents fluid leakage, the latter being the cause of vision loss in patients with the conditions. Together, nAMD, DME and RVO affect more than nine million people in the EU, according to Roche. AMD is the leading cause of vision loss in people over 50 years of age. A 2022 report by nonprofit Retina International estimated the economic burden of late-stage AMD in Germany was €7.6bn. Regeneron Pharmaceuticals ’ Eylea (aflibercept) currently dominates the ophthalmologic market in Europe. Similar to Vabysmo, it is also available in both vials and prefilled syringes, and administered via intravitreal injection. Eylea, along with a higher-dose version at 8mg, generated global sales of $9.38bn – down 3% from 2022. Vabysmo’s sales, meanwhile, grew 324% to bring in $2.64bn. Eylea took another hit in October when Regeneron lost an attempt to block the launch of a biosimilar developed by Pavblu. The US Food and Drug Administration (FDA) has already approved multiple aflibercept biosimilars this year. GlobalData’s Pharma Intelligence Centre predicts a continued decrease in sales over the next five years for Eylea amid increased competition from Vabysmo and biosimilars. By 2030, Roche’s candidate is forecast to reach global sales of $8.78bn, whilst Eylea stutters to $2bn. Roche’s chief medical officer Levi Garraway said: “Approval of the Vabysmo prefilled syringe in the EU offers a convenient way for ophthalmologists to administer this treatment for people with three of the most common causes of vision loss.” “This simplified administration may thereby help reduce the treatment burden for patients and retina specialists.”

PHILADELPHIA--( BUSINESS WIRE )--Exegenesis Bio, a clinical stage global gene therapy company is pleased to announce that US FDA has granted Orphan Drug Designation (“ODD”) to EXG110, a novel gene therapy for Fabry disease, a rare lysosomal disorder that results in excessive deposition of lipids in tissues, eventually leading to renal failure, cardiac disease and strokes. EXG110 is a one-time treatment that delivers the genetic payload directly to liver and heart cells with improved efficacy, safety and dosing. The first patient has been dosed in a China clinical trial and the company plans to initiate a US clinical trial. The FDA’s Office of Orphan Drug Products grants orphan drug designation to support the development of medicines for rare disorders that affect fewer than 200,000 people in the U.S. Orphan drug designation qualifies a company for incentives, including tax credits for qualified clinical trials, exemptions from user fees, and the potential for seven years of market exclusivity after approval. “The FDA’s decision to grant orphan drug designation to EXG110 highlights the need for better approaches to treat Fabry disease, a debilitating condition that affects thousands of people worldwide. We believe this is validation of our rigorous science, our innovation culture and our strong commitment to rare disease patients,” stated Dr. Zhenhua Wu, CEO of Exegenesis Bio. “This is another milestone in our journey to become a truly Global Gene Therapy company. We recently shared updates on our other clinical programs and technology platforms at the European Society for Gene and Cell Therapy meeting, including updates on EXG102/202, a clinical stage gene therapy for wet Age-Related Macular Degeneration (wAMD) and EMC023, a muscle-specific capsid that enables targeted delivery of gene therapies for muscle diseases.” EXG102 is an investigational gene therapy for wAMD that is administered by subretinal injection. Exegenesis Bio is developing a next generation version, EXG202, that incorporates the same genetic cargo, but is delivered to deep retinal cells by a highly-specific ocular capsid that enables administration by intravitreal injection, a less invasive, non-surgical approach. EXG202 has demonstrated a 3 to 5-fold increase in retinal transduction efficiency over currently known ocular-specific capsids in a non-human primate study. “Our unique approach to wAMD is built on three pillars: (1) targeting multiple pathways involved in neovascular disease, including VEGF subtypes A, B, C, D and Angiopoietin 2; (2) packaging our cargo in a proprietary capsid that targets photoreceptors and retinal pigment epithelium more efficiently, and (3) administering the treatment by intravitreal injection, a safe, non-surgical procedure,” emphasizes Dr. Wu. “We believe this will result in a "Best-In-Class" treatment for wAMD and other ocular diseases. We are currently conducting clinical trials in USA and China and hope to share further data in the coming months.” EMC023 is Exegenesis Bio’s proprietary muscle-specific capsid that targets and transduces skeletal muscle tissues more effectively than current benchmark AAV muscle-specific capsids. EMC023 has demonstrated increased skeletal muscle transduction in a non-human primate study – up to 3-fold increase in vector genome copies and up 13-fold increase in transgene mRNA levels. “We believe that we have discovered one of the most specific skeletal muscle targeting capsids known to date,” states Dr. Wu. We are excited to explore the potential of this novel delivery platform in a variety of muscle diseases, including Duchenne Muscular Dystrophy.” “I would like to congratulate our global team for accelerating progress on all of our pipeline programs and platform technologies. We have built a company with full in-house R&D, CMC, Quality and Regulatory capabilities, and we now have three clinical stage programs in USA and/or China: (i) wet Age Related Macular Degeneration, (ii) Spinal Muscular Atrophy, and (iii) Fabry Disease. Our team is operating with a sense of urgency to bring these much-needed treatments to patients worldwide,” emphasizes Dr. Wu. Exegenesis Bio plans to provide further R&D updates on Fabry Disease, wAMD, Spinal Muscular Atrophy and Muscle Tropic Capsid programs during JP Morgan week in San Francisco, Jan 13-16, 2025. About Fabry Disease Fabry disease is a rare X-linked lysosomal disorder that results in excessive deposition of lipids in the tissues. Researchers have identified hundreds of mutations causing Fabry disease in the gene for alpha-Gal A, located on the X chromosome. Deficiency of alpha-Gal A is the main cause for the lysosomal buildup of glycosphingolipids. Progressive glycolipid accumulation can lead to renal failure, cardiac disease and strokes. About wet Age Related Macular Degeneration (wAMD) Nearly 20 million people in the US are living with Age-Related Macular Degeneration (AMD), a progressive eye disease caused by damage to the macula/retina that can lead to blurred vision and blindness. Almost 2 million people have a more severe form of the disease, “wet” AMD (wAMD), which is caused by abnormal blood vessel growth and blood or fluid leakage into the macula, leading to scarring and rapid loss of central vision. wAMD accounts for approximately 90 percent of all AMD-related blindness worldwide. About Duchenne Muscular Dystrophy (DMD) Duchenne muscular dystrophy is a genetic disorder characterized by the progressive loss of muscle. It is a multi-systemic condition, affecting many parts of the body, resulting in deterioration of the skeletal, heart, and lung muscles. Over a million people are living with DMD and other muscle dystrophies worldwide. About EXG110 Gene Therapy for Fabry Disease EXG110 is a one-time treatment for Fabry disease in children and adults, engineered with a proprietary capsid to increase delivery to target heart and liver cells, potentially improving efficacy and safety, and enabling lower dosing. The first patient has been dosed in China and a US clinical trial is planned in 2025. About EXG102-031 Subretinal Injection for wet Age Related Macular Degeneration EXG102-031 is a rAAV-based gene therapy expressing a therapeutic fusion protein that is able to bind/neutralize all known subtypes of Vascular Endothelial Growth Factor (VEGF A, B, C, D) and Angiopoietin-2, which are known to stimulate abnormal blood vessel formation and vascular leakage in the retina. The US and China based clinical trials will evaluate safety and tolerability, as well as visual acuity and central retinal thickness in patients with wAMD. About EXG202 Intravitreal Injection for wet Age Related Macular Degeneration EXG202 is a next generation gene therapy that incorporates the same genetic cargo as EXG102-031 – and is delivered to deep retinal cells by a highly-specific ocular capsid that enables administration by intravitreal injection, a less invasive, non-surgical approach. EXG202 has demonstrated a 3 to 5-fold increase in retinal transduction efficiency over currently known ocular-specific capsids in a non-human primate study About Exegenesis Bio Exegenesis Bio is a clinical stage global gene therapy company with operations in Philadelphia, Boston, Singapore and China. The company’s innovative gene therapy pipeline is based on proprietary capsids, promoters and unique protein engineering designs. Three programs have advanced to the clinical stage: (1) wAMD in USA and China, (2) Spinal Muscular Atrophy (SMA) in China and (3) Fabry disease in China. Exegenesis Bio has built state-of-the-art cGMP manufacturing facilities that include 500L and 2,000L disposable bioreactors for viral vectors and 30L disposable fermenters for plasmids. The company employs over 200 scientific and operations staff worldwide. Please contact us to discuss US and Global collaboration opportunities. Company Website: www.ExegenesisBio.com