An Open-label Extension (OLE) Phase 3 Trial to Assess the Safety of Intravitreal Administration of Avacincaptad Pegol (Complement C5 Inhibitor) in Patients With Geographic Atrophy Who Previously Completed Phase 3 Study ISEE2008 (GATHER2)

The purpose of this study is to assess long-term safety of avacincaptad pegol intravitreal administration for patients with geographic atrophy (GA) who completed Study ISEE2008 (GATHER2) through the Month 24 visit on study treatment (either avacincaptad pegol or Sham).

Start Date26 Sep 2022

Sponsor / Collaborator

Astellas Pharma Global Development, Inc.

NCT04435366 / CompletedPhase 3

A Phase 3 Multicenter, Randomized, Double Masked, Sham- Controlled Clinical Trial to Assess the Safety and Efficacy of Intravitreal Administration of Zimura (Complement C5 Inhibitor) in Patients With Geographic Atrophy Secondary to Age-Related Macular Degeneration

The objectives of this study are to evaluate the safety and efficacy of Zimura (avacincaptad pegol) intravitreal administration in patients with geographic atrophy secondary to age-related macular degeneration (AMD)

Start Date22 Jun 2020

Sponsor / Collaborator

IVERIC bio, Inc.

NCT03374670 / WithdrawnPhase 2

A Phase 2A Open-Label Trial to Assess the Safety of ZIMURA™ (Anti-C5) in Combination With EYLEA® in Treatment Experienced Subjects With Idiopathic Polypoidal Choroidal Vasculopathy

To asses the safety of intravitreal Zimura™ (complement factor C5 inhibitor) administered in combination with Eylea® in treatment experienced subjects with idiopathic polypoidal choroidal vasculopathy

Start Date01 Nov 2018

Sponsor / Collaborator

Ophthotech Corp. (New Jersey)

100 Clinical Results associated with Avacincaptad pegol

100 Translational Medicine associated with Avacincaptad pegol

100 Patents (Medical) associated with Avacincaptad pegol

186

Literatures (Medical) associated with Avacincaptad pegol

01 Mar 2024·Progress in retinal and eye research

Investing in vision: Innovation in retinal therapeutics and the influence on venture capital investment

Review

Author: Luneborg, Nanna ; Lohoff, Tim ; Mulder, Geert-Jan ; Hristodorov, Dmitrij ; Clark, Simon J

Since the groundbreaking approval of the first anti-VEGF therapy in 2004, the retinal therapeutics field has undergone a remarkable transformation, witnessing a surge in novel, disease-modifying therapeutics for a broad spectrum of retinal diseases, extending beyond exudative VEGF-driven conditions. The surge in scientific advancement and the pressing, unmet, medical need have captured the attention of venture capital investors, who have collectively invested close to $10 billion in research and development of new retinal therapeutics between 2004 and 2023. Notably, the field of exudative diseases has gradually shifted away from trying to outcompete anti-VEGF therapeutics towards lowering the overall treatment burden by reducing injection frequency. Simultaneously, a new era has emerged in the non-exudative field, targeting prevalent conditions like dry AMD and rare indications such as Retinitis pigmentosa. This has led to promising drug candidates in development, culminating in the landmark approval of Luxturna for a rare form of Retinitis pigmentosa. The validation of new mechanisms, such as the complement pathway in dry AMD has paved the way for the approvals of Syvovre (Apellis) and Izervay (Iveric/Astellas), marking the first two therapies for this condition. In this comprehensive review, we share our view on the cumulative lessons from the past two decades in developing retinal therapeutics, covering both positive achievements and challenges. We also contextualize the investments, strategic partnering deals, and acquisitions of biotech companies, pharmaceutical companies venture capital investors in retinal therapeutics, respectively. Finally, we provide an outlook and potentially a forward-looking roadmap on novel retinal therapeutics, highlighting the emergence of potential new intervention strategies, such as cell-based therapies, gene editing, and combination therapies. We conclude that upcoming developments have the potential to further stimulate venture capital investments, which ultimately could facilitate the development and delivery of new therapies to patients in need.

01 Jan 2024·Lancet (London, England)Q1 · MEDICINE

Complement in human disease: approved and up-and-coming therapeutics

Q1 · MEDICINE

Review

Author: Woodruff, Trent ; Kemper, Claudia ; West, Erin E ; Fremeaux-Bacchi, Veronique

The complement system is recognised as a protector against blood-borne pathogens and a controller of immune system and tissue homoeostasis. However, dysregulated complement activity is associated with unwanted or non-resolving immune responses and inflammation, which induce or exacerbate the pathogenesis of a broad range of inflammatory and autoimmune diseases. Although the merit of targeting complement clinically has long been acknowledged, the overall complement drug approval rate has been modest. However, the success of the humanised anti-C5 antibody eculizumab in effectively treating paroxysmal nocturnal haemoglobinuria and atypical haemolytic syndrome has revitalised efforts to target complement therapeutically. Increased understanding of complement biology has led to the identification of novel targets for drug development that, in combination with advances in drug discovery and development technologies, has resulted in a surge of interest in bringing new complement therapeutics into clinical use. The rising number of approved drugs still almost exclusively target rare diseases, but the substantial pipeline of up-and-coming treatment options will possibly provide opportunities to also expand the clinical targeting of complement to common diseases.

01 Dec 2023·Acta ophthalmologica

Genetic and molecular biomarkers for geographic atrophy

Review

Author: Huh, Hannah ; Shen, Jie ; den Hollander, Anneke I ; Riley-Gillis, Bridget

Abstract:

Geographic atrophy (GA) is characterized by atrophy of the retina, retinal pigment epithelium and choriocapillaris, causing a gradual loss of vision over time. Treatment options to prevent initiation or progression of GA are limited; two recently FDA‐approved inhibitors of the complement system (pegcetacoplan, avacincaptad pegol) showed a modest decrease in GA lesion growth in phase 3 clinical trials. Exploration of genetic and molecular biomarkers in GA plays a critical role in our battle against this blinding disease to improve early disease detection, to find more effective therapies, and to provide personalized care to patients. In this review, we provide a comprehensive overview of the current literature investigating genetic and molecular biomarkers for GA. Genetic studies identified multiple genes and variants that play a role in progression to GA and GA lesion growth, involving pathways such as complement activation, extracellular matrix interaction and lipid metabolism. The number of published studies assessing molecular biomarkers for GA initiation and progression in ocular matrices is limited. Several studies evaluated molecular biomarkers in the systemic circulation, showing higher levels of complement activation and a causal role of lipid subfractions in GA. Larger, well‐powered studies are needed to identify novel and validate existing biomarkers, and to investigate the potential of combining genetic and molecular markers with imaging techniques for more accurate diagnosis and monitoring of GA. The development of personalized medicine approaches based on individual genetic and molecular profiles could hold promise for more effective and targeted treatments for this devastating disease.

News (Medical) associated with Avacincaptad pegol

16 May 2024

·www.fiercepharma.com

In Big Pharma's growth rankings, Lilly took top spot from Novo Nordisk in Q1

In the first quarter, 18 of the biopharma industry's top 25 companies posted year-over-year revenue increases, led by Eli Lilly with a 28% gain. The momentum the biopharma industry showed in the fourth quarter of 2023 has continued into this year. With 18 of the industry’s top 25 companies achieving year-over-year revenue gains, the first quarter of 2024 nearly matched the final quarter of last year, when 21 of the industry's top 25 companies posted sales increases. Additionally, nine companies realized double-digit growth in the first three months of this year, which was one more than in the previous period. https://public.flourish.studio/visualisation/17990552/ Once again, the dominant players were the sellers of GLP-1 diabetes and obesity drugs. While Novo Nordisk dropped out of the top spot with its 22% year-over-year revenue increase, Eli Lilly took over as No. 1 thanks to its 28% quarterly boost. Lilly may hold the top spot for a while. Last month, GlobalData projected Lilly’s Mounjaro will overtake Novo’s Ozempic as the top blood sugar-modulating treatment, generating staggering sales of $34 billion by 2029. “Mounjaro’s novel dual mechanism not only underscores its clinical value but also its potential to become the top-selling drug in its category,” Eleni Tokali, pharma analyst at GlobalData, said in a release. “Mounjaro’s projected growth rate signifies a potential shift in market dynamics in Eli Lilly’s favor.” While Novo’s older GLP-1 medicines outsold Lilly’s by a margin of better than 2 to 1, the momentum appears to be on Lilly’s side. In the first quarter, while Ozempic generated sales of $4.3 billion, good for a 35% growth rate, Mounjaro brought in $1.8 billion, triple its haul from the first quarter of 2023. As for the companies’ obesity follow-on treatments, Novo’s Wegovy pulled in $1.3 billion, a 107% increase, while Lilly’s Zepbound accounted for $517 million in sales in its first full quarter on the market. While Novo’s 22% growth rate put it among the top companies in the industry in the first quarter, it was a decline from the gaudy 37% growth rate it enjoyed in the previous quarter. Novo’s 65.3 billion Danish kroner ($9.4 billion) in the first quarter also was a slight decline from the 65.9 billion Danish kroner the company achieved in the previous quarter. It was the first sequential drop for Novo since the second quarter of 2022, indicating it is not scaling up its manufacturing quickly enough to meet the overwhelming demand for semaglutide. The company hopes to remedy that with its acquisition of three plants from CDMO giant Catalent. Lilly’s edge came despite its own manufacturing challenges that are limiting its ability to meet the demand for its diabetes and obesity drugs. “Both Mounjaro and Zepbound are expected to remain supply-constrained as growing volume production is overwhelmed by building wholesaler backorders and increasing demand,” Third Bridge analyst Lee Brown recently wrote. Double-digit gainers Seven other companies—including three from Japan—realized double-digit growth in the first quarter, though there were asterisks next to a few of them. While revenues for Daiichi Sankyo were up by 25%, much of the boost can be attributed to the company’s sale of three antibody-drug conjugate (ADC) investigational treatments to Merck for $4.5 billion upfront. Daiichi is deferring the payments and booking them as revenue. Last year as a result of the deal, Daiichi jacked up its projection of 2024 revenue by 100 billion Japanese yen ($662 million). Another Japanese firm, Astellas, saw a 17% revenue increase in the quarter, though part of the gain can be attributed to geographic atrophy treatment Izervay. Astellas acquired the drug last year in the company’s largest-ever M&A deal, a $5.9 billion buyout of Iveric Bio. Amgen’s sales were up 22% from the first quarter of 2023. But of the firm's $1.3 billion year-over-year revenue bump, $914 million can be chalked up to products gained in the company’s buyout of Horizon Therapeutics. Companies that achieved mostly organic double-digit growth were AstraZeneca (17%), Vertex (13%), Takeda (10%) and Novartis (10%). AZ and Vertex also showed their momentum with sequential revenue increases of 6% and 7%, respectively. AstraZeneca was boosted by its wealth of blockbusters that are in their prime, including heart treatment Farxiga, rare disease drug Ultomiris and cancer therapies Calquence, Imfinzi and Tagrisso. Vertex continues to climb behind its cystic fibrosis drugs while laying the groundwork for the launch of sickle cell disease gene therapy Casgevy. Single-digit gainers Several giants of the biopharma industry are among the companies that posted single-digit revenue increases in the first quarter, including Merck (9%), GSK (6%), Bristol Myers Squibb (5%), Gilead (5%), Teva (4%), Sanofi (2%), Johnson & Johnson (2%) and AbbVie (1%). Merck’s rise paralleled the continued growth of mega-blockbuster Keytruda, which was up 20% to $6.9 billion in the quarter. Meanwhile, HPV vaccine Gardasil was up 14% to $2.2 billion. Together, the two products accounted for 58% of the company’s revenue of $15.9 billion for the quarter. GSK and J&J showed growth after a year in which they shed their consumer health units in an industry trend where companies are becoming increasingly focused on their prescription drug units. Gilead’s 5% increase came after it was down 4% in the previous quarter, owing largely to an 18% boost in its oncology products and a 9% increase in its liver disease portfolio. On the downside, in addition to reporting its revenue increase, BMS also revealed a massive cost-cutting plan in which it will lay off more than 2,000 employees. The news came months after the company pulled off pricey acquisitions of Mirati, Karuna and RayzeBio in an M&A splurge. Seven in decline in Q1 Of the companies that saw sales declines in the first quarter, the new surprise member is Regeneron, which had a 1% drop despite the continued growth of Dupixent. The decline for the New York biotech is due largely to a drop in sales for eye disease treatment Eylea. The company’s Eylea partner Bayer also was down by 3% in the period, continuing a trend for the troubled German company which is undergoing a massive restructuring under CEO Bill Anderson. With quarterly sales falling by 19%, Pfizer was the only company in the industry that sustained a double-digit decrease in the first quarter. The fall, of course, is due to declining demand for its COVID products. That effect should now be in the rear-view mirror, the company said. There were plenty of positive numbers for Pfizer as its $14.9 billion revenue haul was $1 billion higher than the analyst consensus. It also was the third straight quarter in which Pfizer had a sequential revenue increase. Excluding sales of COVID products, the company was up 11% in the most recent quarter. Two other companies that saw sales declines in the quarter—Roche at 6% and Merck KGaA at 5%—also chalked it up to the loss of revenue from COVID products and related services. Roche’s drop came despite skyrocketing sales of its eye disease treatment Vabysmo, which has quickly taken market share from Eylea. Other companies that suffered revenue declines were Biogen, at 7%, and Viatris at 2%. With sales falling for its multiple sclerosis products, Biogen sees the trend continuing in 2024 as it is projecting an annual revenue decline for the fifth straight year. Viatris’ decline also reflects an overall trend for the company which has seen annual revenues fall each year since 2021 when it was formed by merging generics powers Mylan and Upjohn.

Acquisition

01 May 2024

·www.biospace.com

Apellis Announces 11 Oral Presentations at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting

WALTHAM, Mass., May 01, 2024 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced that 14 abstracts, 11 of which are oral presentations, will be presented at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting to be held May 5 - 9 in Seattle. The data continue to reinforce the robust efficacy and safety pro SYFOVRE® (pegcetacoplan injection) for geographic atrophy (GA) secondary to age-related macular degeneration (AMD). “The 11 oral presentations at ARVO speak to the continued excitement surrounding SYFOVRE, which is the most chosen GA treatment by retina specialists,” said Caroline Baumal, M.D., chief medical officer, Apellis. “We are excited to share data that continue to highlight SYFOVRE’s ability to slow disease progression with increasing effects over time and its potential to preserve visual function longer.” Oral presentations: Long-term efficacy and safety of pegcetacoplan over 36 months: results from 12 months of the GALE open-label extension study – Ashkan Abbey, M.D. – Sunday, May 5 – 1:45‑2:00 PM PDT (encore) Pegcetacoplan vs avacincaptad pegol in geographic atrophy: an anchored matching-adjusted indirect comparison of three phase 3 trials – Marco Zarbin, M.D., Ph.D. – Thursday, May 9 – 2:15‑2:30 PM PDT (encore) Pegcetacoplan delays time to loss of central macular sensitivity: a microperimetry analysis of the phase 3 OAKS study – Rishi Singh, M.D., FASRS – Thursday, May 9 – 3:30‑3:45 PM PDT (encore) The SD-OCT correlates of fundus autofluorescence in geographic atrophy: a comprehensive point-to-point analysis of fundus autofluorescence with SD-OCT using phase 3 trials DERBY and OAKS – Konstantinos Balaskas, M.D. – Sunday, May 5 – 2:15‑2:30 PM PDT Variability of perilesional FAF patterns associated with automated OCT-based PR/RPE loss ratios in GA progression in the phase 3 OAKS/DERBY trials – Julia Mai, M.D. – Sunday, May 5 – 4:45‑5:00 PM PDT Contrast sensitivity function using a prototype gaze tracking-based virtual reality headset in age-related macular degeneration (AMD) – Deepayan Kar, Ph.D., MS – Monday, May 6 – 8:30‑8:45 AM PDT A deep-learning approach to identifying quantitative OCT biomarkers predictive of visual deficit measured by microperimetry at 24 months in OAKS trial – Dun Jack Fu, Ph.D., BMBCh – Tuesday, May 7 – 9:30‑9:45 AM PDT AI-based qOCT analysis of phase 3 trials OAKS & DERBY data on the effect of pegcetacoplan on geographic atrophy – Pallavi Bagga, Ph.D. – Tuesday, May 7 – 2:15‑2:30 PM PDT Predictive value of the photoreceptor/RPE loss ratio on OCT in the management of geographic atrophy (GA) for patient selection and monitoring of therapeutic efficacy – Ursula Schmidt‑Erfurth, M.D. – Tuesday, May 7 – 2:30‑2:45 PM PDT Rate of response to treatment in eyes with geographic atrophy treated with Pegcetacoplan in a within-patient comparison using automated AI-based OCT biomarker quantifications in OAKS, DERBY and GALE – Gregor S. Reiter, M.D., Ph.D. – Thursday, May 9 – 2:30‑2:45 PM PDT IRIS® Registry analysis of anti-VEGF treatment in patients with coexisting neovascular age-related macular degeneration and geographic atrophy – Theodore Leng, M.D., FACS – Thursday, May 9 – 3:15‑3:30 PM PDT Poster presentations: Prevalence of outer retinal tubulation (ORT) and association with geographic atrophy (GA) growth among patients in the OAKS and DERBY trials – Kensington Hatcher, O.D., FAAO – Monday, May 6 – 3:00‑4:45 PM PDT Loss of independence in geographic atrophy (GA) - results from the MOSAIC study among patients and caregivers in Europe – Beverly Lui, PharmD – Thursday, May 9 – 8:00‑9:45 AM PDT Deep learning-based segmentation of geographic atrophy in fundus autofluorescence: external validation in imaging acquired in a clinical trial – Souvick Mukherjee, Ph.D. – Thursday, May 9 – 8:00‑9:45 AM PDT About SYFOVRE® (pegcetacoplan injection) SYFOVRE® (pegcetacoplan injection) is the first-ever approved therapy for geographic atrophy (GA). By targeting C3, SYFOVRE is designed to provide comprehensive control of the complement cascade, part of the body’s immune system. SYFOVRE is approved in the United States for the treatment of GA secondary to age-related macular degeneration. About Geographic Atrophy (GA) Geographic atrophy (GA) is an advanced form of age-related macular degeneration and a leading cause of blindness worldwide, impacting more than one million Americans and five million people worldwide.1,2 It is a progressive and irreversible disease caused by the growth of lesions, which destroy the retinal cells responsible for vision. The vision loss caused by GA severely impairs independence and quality of life by making it difficult to participate in daily activities. On average, it takes only 2.5 years for GA lesions to start impacting the fovea, which is responsible for central vision.3 U.S. Important Safety Information for SYFOVRE® (pegcetacoplan injection) CONTRAINDICATIONS SYFOVRE is contraindicated in patients with ocular or periocular infections, and in patients with active intraocular inflammation WARNINGS AND PRECAUTIONS Endophthalmitis and Retinal Detachments Intravitreal injections, including those with SYFOVRE, may be associated with endophthalmitis and retinal detachments. Proper aseptic injection technique must always be used when administering SYFOVRE to minimize the risk of endophthalmitis. Patients should be instructed to report any symptoms suggestive of endophthalmitis or retinal detachment without delay and should be managed appropriately. Retinal Vasculitis and/or Retinal Vascular Occlusion Retinal vasculitis and/or retinal vascular occlusion, typically in the presence of intraocular inflammation, have been reported with the use of SYFOVRE. Cases may occur with the first dose of SYFOVRE and may result in severe vision loss. Discontinue treatment with SYFOVRE in patients who develop these events. Patients should be instructed to report any change in vision without delay. Neovascular AMD In clinical trials, use of SYFOVRE was associated with increased rates of neovascular (wet) AMD or choroidal neovascularization (12% when administered monthly, 7% when administered every other month and 3% in the control group) by Month 24. Patients receiving SYFOVRE should be monitored for signs of neovascular AMD. In case anti-Vascular Endothelial Growth Factor (anti-VEGF) is required, it should be given separately from SYFOVRE administration. Intraocular Inflammation In clinical trials, use of SYFOVRE was associated with episodes of intraocular inflammation including: vitritis, vitreal cells, iridocyclitis, uveitis, anterior chamber cells, iritis, and anterior chamber flare. After inflammation resolves, patients may resume treatment with SYFOVRE. Increased Intraocular Pressure Acute increase in IOP may occur within minutes of any intravitreal injection, including with SYFOVRE. Perfusion of the optic nerve head should be monitored following the injection and managed as needed. ADVERSE REACTIONS Most common adverse reactions (incidence ≥5%) are ocular discomfort, neovascular age-related macular degeneration, vitreous floaters, conjunctival hemorrhage. Please see accompanying full Prescribing Information for more information. About Apellis Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that combines courageous science and compassion to develop life-changing therapies for some of the most challenging diseases patients face. We ushered in the first new class of complement medicine in 15 years and now have two approved medicines targeting C3. These include the first-ever therapy for geographic atrophy, a leading cause of blindness around the world. We believe we have only begun to unlock the potential of targeting C3 across serious retinal, rare, and neurological diseases. For more information, please visit or follow us on Twitter and LinkedIn. Apellis Forward-Looking Statement Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements” within the meaning of The Private Securities Litigation Reform Act of 1995. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors and other factors discussed in the “Risk Factors” section of Apellis’ Annual Report on Form 10-K with the Securities and Exchange Commission on February 27, 2024 and the risks described in other filings that Apellis may make with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Apellis specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise. Media Contact: Lissa Pavluk media@apellis.com 617.977.6764 Investor Contact: Meredith Kaya meredith.kaya@apellis.com 617.599.8178 1Rudnicka AR, Jarrar Z, Wormald R, et al. Age and gender variations in age-related macular degeneration prevalence in populations of European ancestry: a meta analysis. Ophthalmology 2012;119:571–580. 2Wong WL, Su X, Li X, et al. Global prevalence of age-related macular degeneration and disease burden projection for 2020 and 2040: a systematic review and meta-analysis. Lancet Glob Health 2014;2:e106–116. 3Lindblad AS, et al, and AREDS Research Group. Arch Ophthalmol. 2009;127(9):1168-1174.

Phase 3Drug Approval

29 Apr 2024

·www.biospace.com

Inflammasome Therapeutics Announces First Patient Dosed in Geographic Atrophy (GA) Clinical Trial

Phase 1 investigator-sponsored clinical trial “Evaluation of Kamuvudine-8 in Subjects with Geographic Atrophy (K8 for GA)” for a blinding condition resulting from dry age-related macular degeneration (dry AMD). Open label 26-week study designed to evaluate the safety and treatment efficacy of K8, a novel neuroprotectant that will target the underlying cause of vision loss in GA. Expected to treat up to five subjects with intravitreous injection of intraocular implant designed to last for three months. Marks second clinical trial underway for Inflammasome Therapeutics that uses the company’s proprietary new class of drugs. Trial for DME also recruiting (ClinicalTrials.gov ID NCT05699759). Kamuvudines are shown in pre-clinical research to inhibit the underlying cause of vision loss in GA. NEWTON, Mass.--(BUSINESS WIRE)-- Inflammasome Therapeutics ( ), a private company developing a new class of inflammasome inhibitor drugs, Kamuvudines, as therapies for prevalent, degenerative diseases, announced the first patient has been dosed in a first-in-class clinical trial for dry AMD. The Phase 1 trial (ClinicalTrials.gov ID NCT06164587) is sponsored by the University of Kentucky and expected to enroll up to five patients with GA due to age-related macular degeneration (AMD). This press release features multimedia. View the full release here: Inflammasome's K8, a tiny sustained release implant that provides slow, consistent release of the drug K8 (inflammasome inhibitor drug Kamuvudine) directly in the back of the eye for a period of three months. (Photo: Business Wire) Trial participants will receive a tiny, sustained release implant (illustration is available) that will provide slow, consistent release of the drug K8 directly into the back of the eye. K8 was specifically designed for retinal delivery and the implants and injector system were crafted to deliver this particular drug. This combined drug and delivery strategy allows high therapeutic doses to be maintained in the eye while the drug is undetectable in systemic circulation. “This marks the second trial underway with our Kamuvudines in ophthalmology,” said Dr. Paul Ashton, President and CEO of Inflammasome Therapeutics, developers of both the compounds and delivery systems for administration. GA affects approximately one million individuals in the US and more than eight million worldwide. In GA, multiple toxic substances (such as complement, amyloid beta, retrotransposons, iron, and reactive oxygen species) build up in the eye and trigger inflammasome activation that then causes cells in the macula to slowly die (atrophy). This is a very similar process to what goes on in the brain and nervous system in patients with ALS, Alzheimer’s disease and multiple sclerosis. Last year, two drugs (Syfovre and Izervay) were approved for GA that target one of these toxic substances, complement. The drugs do not target other toxic elements and only modestly slow progression of the disease. Unfortunately, they also increase the risk of developing wet AMD. Furthermore, the drugs require an intraocular injection every four to eight weeks. There is tremendous interest in developing potential GA treatments. There are 38 other interventional clinical trials for GA registered in clinicaltrials.gov., almost all of which target individual toxic substances, but not the underlying cause of the atrophy, inflammasome activation. “That’s where we believe we provide a distinctive and significant difference,” said Dr. Ashton. “Our Kamuvudines have been shown in pre-clinical studies to block inflammasome activation caused by multiple toxic pathways – complement, amyloid beta, iron overload, retrotransposons, etc. If we can block inflammasome activation in the clinic, we believe we can have a profound effect on the disease by blocking multiple pathways.” Dr. Ashton confirmed that the implications for treatments in other neuroinflammatory diseases like Alzheimer’s Disease, ALS and Multiple Sclerosis are “extremely interesting. We have Kamuvudines specifically designed for neurological diseases that penetrate into the brain and Central Nervous System from a simple oral tablet. Inflammasome Therapeutics is expected to begin clinical trials in some of these diseases soon as well,” he affirmed. Inflammasome Therapeutics’ co-founder, Dr. Jayakrishna Ambati, has spent more than a decade developing Kamuvudines and identifying their role in the inhibition of inflammasome activity that is being found to be the underlying cause of many diseases. In a series of publications in journals such as Science and Nature, he has described the basic research on GA and pre-clinical development of Kamuvudines: Dr. Ambati and Dr. Ashton co-founded Inflammasome Therapeutics in 2016 to develop therapies for prevalent, degenerative diseases and to develop novel delivery technologies for the sustained release of therapeutic agents and compounds. The company combines scientific excellence with proven development expertise and works to develop products via a mixture of licensing agreements and internal development, including work with Boehringer Ingelheim and the Gates Foundation.

Phase 1Drug Approval

100 Deals associated with Avacincaptad pegol

External Link

KEGG	Wiki	ATC	Drug Bank
D11748	-	-	DB15165

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Geographic Atrophy	US	Astellas Pharma US, Inc.	04 Aug 2023

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Age Related Macular Degeneration	Phase 3	US	IVERIC bio, Inc.	22 Jun 2020
Age Related Macular Degeneration	Phase 3	AR	IVERIC bio, Inc.	22 Jun 2020
Age Related Macular Degeneration	Phase 3	AU	IVERIC bio, Inc.	22 Jun 2020
Age Related Macular Degeneration	Phase 3	AT	IVERIC bio, Inc.	22 Jun 2020
Age Related Macular Degeneration	Phase 3	BE	IVERIC bio, Inc.	22 Jun 2020
Age Related Macular Degeneration	Phase 3	BR	IVERIC bio, Inc.	22 Jun 2020
Age Related Macular Degeneration	Phase 3	CA	IVERIC bio, Inc.	22 Jun 2020
Age Related Macular Degeneration	Phase 3	CO	IVERIC bio, Inc.	22 Jun 2020
Age Related Macular Degeneration	Phase 3	HR	IVERIC bio, Inc.	22 Jun 2020
Age Related Macular Degeneration	Phase 3	CZ	IVERIC bio, Inc.	22 Jun 2020

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04435366 (CTgov)	Phase 3	Age Related Macular Degeneration \| Geographic Atrophy	448	Avacincaptad Pegol (Avacincaptad Pegol Treatment Group)	luwrjmnvgl(ropotcxxwy) = entkfouvzr fobmihyhov (nkjryclfyk, ktqjqoxaua - qrvgvalnug) View more	-	28 Dec 2023
				Sham (Sham Treatment Group)	luwrjmnvgl(ropotcxxwy) = czlbqahlhm fobmihyhov (nkjryclfyk, hildlwmcjv - xffscewvhn) View more
GATHER2 (PRNewswire) Manual	Phase 3	Age Related Macular Degeneration	-	IZERVAY	hdubwdqokb(hdfkqlajjj) = IZERVAY was well tolerated over 2 years, with one case each of non-serious intraocular inflammation (IOI) and culture-positive endophthalmitis. There were no cases of ischemic neuropathy or retinal vasculitis. grcobdensk (aazucrtzuj ) View more	Positive	04 Nov 2023
				IZERVAY
NCT02686658 \| NCT04435366 (GATHER2, FDA) Manual	Phase 2/3	Geographic Atrophy	624	IZERVAY (GATHER1)	fydhsymhse(slxlhvsqoe) = ozpeunzhwl qektpfivrk (hxhimsjxvm )	Positive	04 Aug 2023
				Sham (GATHER1)	fydhsymhse(slxlhvsqoe) = cmjyzwvlfl qektpfivrk (hxhimsjxvm )
NCT05571267 (CTgov)	Phase 2	Age Related Macular Degeneration	1	Zimura (Zimura and Avastin)	xchfgujrmz(cbxvyktykm) = csnotutzib whnbvinqks (qmlnplxdgv, kxwkhuknre - ygqwwsmuok) View more	-	03 Apr 2023
				Zimura+Lucentis (Zimura and Lucentis)	llzweblgjm(xrrtgbclpw) = hmbqaenuvr jhonfcybky (nlsywrubkb, toslpgostn - rmjedwudiu) View more
NCT04435366 (GATHER2, Corporate Publications) Manual	Phase 3	Geographic Atrophy	447	Avacincaptad pegol	ncitozddhw(bxwkkqbjjw) = vqgretvgvr oknitgdzlw (dqeagsdcxd ) View more	Positive	06 Sep 2022
				Sham	ncitozddhw(bxwkkqbjjw) = oukirwxibp oknitgdzlw (dqeagsdcxd ) View more
GATHER1 (ARVO2022)	Phase 2/3	Geographic Atrophy	-	Avacincaptad pegol 2 mg	icvtxsqqly(ntetoocxme) = kgvdsaxbrz hksmxsuiwg (oeuvzficcf )	-	01 May 2022
				avacincaptad pegol	icvtxsqqly(ntetoocxme) = bmkgcjbihi hksmxsuiwg (oeuvzficcf )
NCT03362190 (CTgov)	Phase 2	Wet age-related macular degeneration	64	Zimura+Lucentis (Cohort 1)	mnbrcbtaki(jadewogapx) = uhutgfffny bmtxrqnvzg (smhbmvuctw, czqsftstsx - wtfscihxjg) View more	-	02 Feb 2022
				Zimura+Lucentis (Cohort 2)	mnbrcbtaki(jadewogapx) = iebmbsbdea bmtxrqnvzg (smhbmvuctw, tduewbqujr - esovgnebni) View more
GATHER1 (AAO2021)	Not Applicable	-	286	Avacincaptad Pegol 2mg	guoqgnaffo(lyqagzsunt) = helqealtnt avhsxgdjdf (rklesbczln )	-	13 Nov 2021
				Avacincaptad Pegol 4mg	guoqgnaffo(lyqagzsunt) = ljvvjfwtoq avhsxgdjdf (rklesbczln )
ARVO2021	Not Applicable	Geographic Atrophy	-	Avacincaptad pegol 2 mg	llxmbvntwe(acztchbxtk) = There were no significant differences in best-corrected visual acuity or low-luminance visual acuity between avacincaptad and sham-treated patients nkgiyjkbak (syjggqhaoj ) View more	Positive	01 Jun 2021
				Avacincaptad pegol 4 mg
NCT02686658 (GATHER1, Pubmed \| Ophthalmology)	Phase 2/3	Age Related Macular Degeneration	286	Avacincaptad pegol 2 mg	tsqvmjjbyk(heizlhkicc) = There were no avacincaptad pegol-related adverse events (AEs) or inflammation ticuimgoem (tkzrwpweet ) View more	-	01 Sep 2020
				Avacincaptad pegol 4 mg

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