A Prospective, Multicenter, Open-Label, Observational Phase 4 Study to Evaluate Real-World Safety, Tolerability, and Treatment Patterns of Pegcetacoplan (Syfovre) in Patients With Geographic Atrophy Secondary to Age-Related Macular Degeneration

A Prospective, Multicenter, Open-Label, Observational Phase 4 Study to Evaluate Real-World Safety, Tolerability, and Treatment Patterns of Pegcetacoplan (Syfovre) in Patients with Geographic Atrophy Secondary to Age-Related Macular Degeneration

Start Date28 Sep 2023

Sponsor / Collaborator

Apellis Pharmaceuticals, Inc.

JPRN-jRCT2041230022 / RecruitingPhase 3IIT

A PHASE 3, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLINDED, MULTICENTER STUDY TO EVALUATE THE EFFICACY AND SAFETY OF PEGCETACOPLAN IN PATIENTS WITH C3 GLOMERULOPATHY OR IMMUNE-COMPLEX MEMBRANOPROLIFERATIVE GLOMERULONEPHRITIS - A PHASE 3 STUDY TO EVALUATE THE EFFICACY AND SAFETY OF PEGCETACOPLAN IN PATIENTS WITH C3 GLOMERULOPATHY OR IMMUNE-COMPLEX MEMBRANOPROLIFERATIVE GLOMERULONEPHRITIS

Start Date18 Aug 2023

Sponsor / Collaborator-

NCT05776472 / RecruitingNot Applicable

A Single Arm, Multicentre Observational Study to Evaluate Effectiveness of Pegcetacoplan Under Real World Conditions in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

This is a 24-month multicenter, observational study designed to describe the real world effectiveness of pegcetacoplan in patients with PNH. Patients meeting the eligibility criteria will be enrolled in the study at the enrollment visit and followed prospectively for 24 (+/- 3) months. The scope of the study is to collect both retrospective and prospective data. The main part of the study will be prospective,collecting data on effectiveness, safety, patient- and clinician-reported outcomes and health care resource use.

Start Date26 Jun 2023

Sponsor / Collaborator

Swedish Orphan Biovitrum AB

100 Clinical Results associated with Pegcetacoplan

100 Translational Medicine associated with Pegcetacoplan

100 Patents (Medical) associated with Pegcetacoplan

163

Literatures (Medical) associated with Pegcetacoplan

01 Nov 2024·Ophthalmology Retina

A Cost Effectiveness Analysis of Avacincaptad Pegol for the Treatment of Geographic Atrophy with Comparison to Pegcetacoplan

Article

Author: Al-Khersan, Hasenin ; Yannuzzi, Nicolas A ; Hoyek, Sandra ; Patel, Nimesh A ; Smiddy, William E

PURPOSE:

The purpose of this study was to evaluate the cost effectiveness of the treatment of geographic atrophy (GA) with intravitreal avacincaptad pegol (ACP) and to compare it with pegcetacoplan (PEG).

DESIGN:

Cost analysis based on data from published studies.

SUBJECTS:

None; based on data from published sham control compared with 2 treatment groups in each of the index studies.

METHODS:

Costs were based on 2022 Medicare reimbursement data for both facility (hospital-based) and nonfacility settings in Miami. Specific usage and outcomes were derived from the GATHER2 study as well as DERBY and OAKS trials. For ACP, all patients were treated every month (EM) in year 1 then randomized to every other month (EOM) or EM in year 2. Two-year models were created for patients in the facility setting for extrafoveal (ACP and PEG) and all patients (PEG).

MAIN OUTCOME MEASURES:

Cost, cost utility, and cost per area of GA (in United States dollars).

RESULTS:

The cost to treat GA with ACP in EM and EOM treatment groups over the 2 years as reported was $67 400 and $40 600, respectively. With ACP treatment over 2 years, the daily cost of delaying GA 3.4 months (EM) and 4.5 months (EOM) was $649 (EM) and $356 (EOM). The (facility-based) costs per unit area of retinal pigment epithelium saved for patients with extrafoveal GA over the 2-year period were $119 000/mm² (EM ACP) versus $54 000/mm² (EM PEG) (P < 0.001), $57 100/mm² (EOM ACP) versus $31 400/mm² (EOM PEG) (P < 0.001), and $45 300/mm² (hypothetical EOM from outset ACP).

CONCLUSION:

Treatment of GA with intravitreal ACP EOM was more cost effective than EM. When assessing extrafoveal lesions, ACP was less cost effective than PEG for both EM and EOM treatment.

FINANCIAL DISCLOSURE(S):

Proprietary or commercial disclosure may be found in the Footnotes and Disclosures at the end of this article.

25 Oct 2024·INDIAN JOURNAL OF OPHTHALMOLOGY

Dry and neovascular "wet" age-related macular degeneration: Upcoming therapies.

Article

Author: Yan, Audrey ; Hasan, Nasiq ; Chhablani, Jay

ABSTRACT:

The age-related macular degeneration (AMD) field is witnessing promising advancements in therapeutic options. Breakthrough drugs such as pegcetacoplan and avacincaptad have been FDA-approved for dry AMD, marking a significant development as there were no treatment options until August 2023. While several antivascular endothelial growth factor (VEGF) inhibitors have been approved for wet AMD, challenges persist with the need for frequent dosing. New treatments such as gene therapy, cell therapy, WNT pathway agonists, complement inhibitors, and anti-VEGF combination drugs are under development to address these issues. These developments are exciting and hold promise for transforming the field of medicine, offering hope for improved outcomes and enhanced patient care in managing AMD.

01 Oct 2024·Ophthalmology Retina

Retinal vasculopathy and choroiditis after pegcetacoplan injection: clinicopathologic support for a drug hypersensitivity reaction

Article

Author: Crawford, Courtney ; Avery, Robert L ; Stagner, Anna M. ; Nahar, Ankur ; Avery, Robert L. ; Carrasco, Jacqueline R ; Stagner, Anna M ; Eliott, Dean ; Pulido, Jose ; Eagle, Ralph C ; Eagle, Ralph C. ; Milman, Tatyana ; Carrasco, Jacqueline R.

PURPOSE:

To report the detailed histopathology of two enucleated eyes from two patients who developed severe visual loss associated with retinal hemorrhages, vessel sheathing and vascular nonperfusion after administration of an initial dose of intravitreal pegcetacoplan, and to propose, with supportive histopathology, the pathogenesis of the clinical syndrome previously termed hemorrhagic occlusive retinal vasculitis (HORV).

DESIGN:

Case series.

SUBJECTS:

Two enucleated eyes from two patients treated with intravitreal pegcetacoplan.

METHODS:

Retrospective, multicenter consecutive clinical-pathologic analysis.

MAIN OUTCOME MEASURES:

Histopathologic review and immunophenotypic characterization.

RESULTS:

Both patients presented with inflammation and significant vision loss nine days following the initial injection of pegcetacoplan with no subsequent improvement and underwent enucleation for pain control. Histologic examination of the enucleated eyes (patient one at 4 months post-injection and patient two at 40 days) revealed extensive vascular thrombosis, retinal hemorrhages and necrosis, and a dense inflammatory infiltrate in the uvea and variably the optic nerve, episclera, and muscle tendons composed of predominantly of T-cells, macrophages, and eosinophils. Notably, the inflammatory infiltrate was absent from the retina. In addition, one eye demonstrated multiple foci of glomerular-like vascular proliferations in the uveal tract and thrombosis with focal recanalization of vessels in the optic nerve.

CONCLUSION:

Drug-induced, immune-mediated, retinal vasculopathy and choroiditis (DIRVAC) is a rare complication following pegcetacoplan injection. Although some limitations arise in interpretation of histopathologic findings due to compensatory changes in the eyes over time (prior to enucleation), the authors propose that the combined clinical, histopathologic, and immunohistochemical findings suggest a mixed-type, delayed hypersensitivity reaction as mechanism of initial injury.

257

News (Medical) associated with Pegcetacoplan

03 Dec 2024

·www.prnewswire.com

Sobi's strength in haematology to be showcased at ASH 2024

STOCKHOLM, Dec. 3, 2024 /PRNewswire/ -- New data from Sobi® and partners will be presented at the 66th Annual Meeting of the American Society of Hematology (ASH) in San Diego, CA (USA) from the 7th - 10th of December 2024. During the meeting several analyses will be presented on haemophilia A, haemophagocytic lymphohistiocytosis (HLH), myelofibrosis, paroxysmal nocturnal haemoglobinuria (PNH), and immune thrombocytopenia (ITP). "Haemophilia A has a lasting impact on joint health and quality of life. At ASH 2024, three oral presentations will reveal important new data about long-term outcomes with ALTUVOCT® prophylaxis," said Lydia Abad-Franch, MD, MBA, Head of Research, Development, and Medical Affairs (RDMA), and Chief Medical Officer at Sobi. "We will also present new data spanning different severe and debilitating rare diseases, including an oral presentation on the real-world effectiveness of Doptelet® in treating people with ITP, demonstrating our broader commitment to advancing innovative treatments for people with rare haematological diseases. We look forward to sharing these findings in San Diego." Key data to be presented at ASH 2024 About ALTUVOCT® ALTUVOCT® (efanesoctocog alfa) [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein] (formerly BIVV001) is the first high-sustained FVIII replacement therapy with the potential to deliver near-normal factor activity levels for a significant part of the week, improving bleed protection in a once-weekly dose for people with haemophilia A. ALTUVOCT builds on the established Fc fusion technology by innovatively adding a region of von Willebrand factor and XTEN® polypeptides to extend its time in circulation. It is the only therapy that has been shown to break through the von Willebrand factor ceiling, which imposes a half-life limitation on current factor VIII therapies. The European Commission granted Orphan Drug designation in June 2019. It is approved and marketed as ALTUVOCT by Sobi in Europe. It is approved and marketed as ALTUVIIIO™ [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl] by Sanofi in the United States, Japan, and Taiwan. About the Sanofi and Sobi collaboration Sobi and Sanofi collaborate on the development and commercialisation of Alprolix® and Elocta®/Eloctate®. The companies also collaborate on the development and commercialisation of ALTUVOCT® (efanesoctocog alfa), or ALTUVIIIO™ in the US. Sobi has final development and commercialisation rights in the Sobi territory (essentially Europe, North Africa, Russia, and most Middle Eastern markets). Sanofi has final development and commercialisation rights in North America and all other regions in the world excluding the Sobi territory. About Gamifant® Gamifant (emapalumab) is an anti-interferon gamma (IFNγ) monoclonal antibody that binds to and neutralises IFNγ. In the USA, Gamifant is indicated for the treatment of adult and paediatric (newborn and older) patients with primary haemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. Primary HLH is a rare syndrome of hyperinflammation that usually occurs within the first year of life and can rapidly become fatal unless diagnosed and treated. The FDA approval is based on data from the phase 2/3 studies (NCT01818492 and NCT02069899). Gamifant is indicated for administration through intravenous infusion over one hour twice per week until haematopoietic stem cell transplantation (HSCT). About Vonjo® Vonjo (pacritinib), a JAK-1 sparing JAK2/IRAK1/ACVR1 inhibitor, is approved for the treatment of adults with intermediate- or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 109/L in the United States. This indication is approved under FDA accelerated approval based on spleen volume reduction. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). Currently, a Phase 3 study (PACIFICA) is being conducted to assess pacritinib in patients with myelofibrosis and severe thrombocytopenia as a post-marketing requirement. About Aspaveli®/ Empaveli® Aspaveli/Empaveli (pegcetacoplan) is a targeted C3 and C3b inhibitor designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is approved for the treatment of paroxysmal nocturnal haemoglobinuria (PNH) as Aspaveli/Empaveli in the United States, European Union, and other countries globally. Currently, a Phase 3 trial (VALIANT) is being conduted to assess the efficacy and safety of pegcetacoplan in patients with C3G and primary IC-MPGN. Aspaveli/Empaveli is also under investigation for several other rare diseases across haematology and nephrology. About the Sobi and Apellis collaboration Sobi and Apellis have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-U.S. commercialisation rights for systemic pegcetacoplan, and Apellis has exclusive U.S. commercialisation rights for systemic pegcetacoplan and worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy. About Doptelet® Doptelet (avatrombopag) is an orally administered thrombopoietin receptor agonist (TPO-RA) that mimics the biologic effects of TPO in stimulating the development and maturation of megakaryocytes, resulting in increased platelet count. It is approved in over 30 countries worldwide, including the EU and the US, for the treatment of severe thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo an invasive procedure, and for the treatment of thrombocytopenia in adult patients with primary chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment. Chronic ITP is a rare autoimmune bleeding disorder characterised by low number of platelets. The incidence of primary ITP in adults is 3.3/100,000 adults per year with a prevalence of 9.5 per 100,000 adults. About Sobi® Sobi® is a specialised international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of haematology, immunology, and specialty care, Sobi has approximately 1,800 employees across Europe, North America, the Middle East, Asia, and Australia. In 2023, revenue amounted to SEK 22.1 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn. Contacts For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. This information was brought to you by Cision The following files are available for download: WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Drug ApprovalPhase 3ASHOrphan DrugLicense out/in

19 Nov 2024

·endpts.com

FDA rejects Astellas’ effort to support longer use of eye drug Izervay

The FDA has denied Astellas’ attempt to bolster the label for its vision loss treatment Izervay with additional two-year data. Izervay, the centerpiece of Astellas’ $5.9 billion acquisition of Iveric Bio , was approved last year to treat geographic atrophy secondary to age-related macular degeneration. The label calls for monthly dosing capped at 12 months, but Astellas had hoped that additional data would support longer use. Astellas announced in April that it submitted two-year data from a Phase 3 trial evaluating safety and efficacy in patients dosed monthly and every other month. But on Tuesday, it said the FDA issued a complete response letter over “a statistical matter related to labeling language proposed by Astellas.” An Astellas spokesperson declined to elaborate further on the rejection, and said the company is “seeking further clarification” from regulators. The company said the FDA’s decision was “unrelated to the safety and benefit/risk” of Izervay. “After discussions with the FDA, Astellas plans to resubmit our application,” the spokesperson said. Izervay competes with Apellis’ Syfovre, which was approved several months before Izervay and has no limitations on treatment duration. However, Apellis’ launch was complicated by rare but serious retinal inflammation concerns. Astellas said on its second-quarter earnings call in October that it estimates 60% of new patients are being placed on Izervay. Astellas has also faced challenges expanding outside of the US. The company pulled Izervay’s marketing application in the European Union last month after regulators expressed concerns about its efficacy. “Although the studies provided by the applicant showed that Izelvay slowed the growth of geographic atrophy lesions, the Agency considered that this effect did not lead to clinically meaningful improvements in visual function for patients,” the European Medicines Agency said. Astellas told Endpoints News Tuesday that it is “exploring potential pathways including resubmission in Europe.”

AcquisitionDrug ApprovalPhase 3Clinical Result

19 Nov 2024

·www.globenewswire.com

Ocugen Announces Compelling Preliminary Data for OCU410—a Single Dose Novel Modifier Gene Therapy to Treat Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration

MALVERN, Pa., Nov. 19, 2024 (GLOBE NEWSWIRE) -- 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 positive preliminary efficacy and safety data from the Phase 1 dose-escalation portion of the Phase 1/2 OCU410 ArMaDa clinical trial for geographic atrophy (GA), secondary to dry age-related macular degeneration (dAMD). Key findings include: no drug-related serious adverse events, reduced lesion growth, preservation of retinal tissue, and—most importantly—there was a positive effect on the functional visual measure of low luminance visual acuity (LLVA). Currently, there are approximately three million people living with GA in the United States (U.S.) and Europe combined. Patients in the U.S. have only one option available, anti-complement therapy, which requires multiple injections and only addresses one aspect of the disease. There remains no treatment option for GA in Europe. The OCU410 Phase 1 trial is evaluating nine patients in three dose cohorts (low, medium, and high). The following data was observed for the three patients in the low dose cohort at six months: Considerably slower lesion growth (21.4%) from baseline in treated vs. untreated fellow eyes that followed the natural history of the disease. This result is favorable when compared to published data on pegcetacoplan injected every month or every other month over six months.OCU410 treatment showed increasing preservation of retinal tissue around the GA lesions of treated eyes over six months, which also compared favorably to published data on pegcetacoplan given monthly and every other month.100% of the OCU410 treated eyes showed stabilization of visual function demonstrating treatment benefit as measured by LLVA. “Currently approved treatments for GA have not shown significant benefit in visual function. More importantly, we often do not realize the logistical challenge and emotional burden both patients and their caregivers must endure for every month or every other month visits,” said Syed M. Shah, MD, FACS, Director of Retina Service, Vice Chair for Research & Digital Health at Emplify Health – La Crosse, Wisconsin. “Based on the science and preliminary data, OCU410 has the potential to improve structural as well as functional outcomes. This ‘one-and-done’ treatment paradigm can be a gamechanger for how we treat patients with GA.” “OCU410 addresses multiple aspects of the disease beyond the complement pathway,” said Dr. Huma Qamar, Chief Medical Officer at Ocugen. “The latest OCU410 data emphasizes the potential of novel modifier gene therapy as a one-time treatment for dAMD. We remain very encouraged by the latest safety and efficacy data and positive patient outcomes.” Ocugen also announced promising data from the Phase 1/2 OCU410ST GARDian clinical trial for Stargardt disease and data on Leber congenital amaurosis (LCA) from the Phase 1/2 OCU400 clinical trial. All these findings, as well as commentary from study investigators and patient perspectives, were shared at the Company’s recent Clinical Showcase. The data affirms the potential for modifier gene therapy to address both rare inherited retinal diseases and blindness diseases affecting millions. A full replay of the showcase is available on the Events section of the Ocugen website. For more information about Ocugen’s ongoing clinical trials, please contact clinical.request@ocugen.com 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 patient’s 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. Cautionary Note on Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, strategy, business plans and objectives for Ocugen’s clinical programs, plans and timelines for the preclinical and clinical development of Ocugen’s product candidates, including the therapeutic potential, clinical benefits and safety thereof, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, the ability to initiate new clinical programs; statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our annual and periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release. Contact:Tiffany HamiltonHead of CommunicationsTiffany.Hamilton@ocugen.com

Phase 1Clinical ResultGene Therapy

100 Deals associated with Pegcetacoplan

External Link

KEGG	Wiki	ATC	Drug Bank
D11613	Pegcetacoplan	B03XA	-

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Geographic Atrophy	US	Apellis Pharmaceuticals, Inc.	17 Feb 2023
Hemoglobinuria, Paroxysmal	US	Apellis Pharmaceuticals, Inc.	14 May 2021

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Cold Agglutinin Disease	Phase 3	US	Swedish Orphan Biovitrum AB	20 Oct 2022
Cold Agglutinin Disease	Phase 3	JP	Swedish Orphan Biovitrum AB	20 Oct 2022
Cold Agglutinin Disease	Phase 3	AT	Swedish Orphan Biovitrum AB	20 Oct 2022
Cold Agglutinin Disease	Phase 3	BE	Swedish Orphan Biovitrum AB	20 Oct 2022
Cold Agglutinin Disease	Phase 3	CA	Swedish Orphan Biovitrum AB	20 Oct 2022
Cold Agglutinin Disease	Phase 3	FI	Swedish Orphan Biovitrum AB	20 Oct 2022
Cold Agglutinin Disease	Phase 3	GE	Swedish Orphan Biovitrum AB	20 Oct 2022
Cold Agglutinin Disease	Phase 3	DE	Swedish Orphan Biovitrum AB	20 Oct 2022
Cold Agglutinin Disease	Phase 3	HU	Swedish Orphan Biovitrum AB	20 Oct 2022
Cold Agglutinin Disease	Phase 3	IT	Swedish Orphan Biovitrum AB	20 Oct 2022

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ASH2024	Not Applicable	Hemoglobinuria, Paroxysmal	-	Complement Inhibitor Experienced	razwhlkyve(echkaqaoyo) = oxmbxpywuy jzisxtfmco (rzkzcvgbrp, 1.7) View more	-	09 Dec 2024
				Complement Inhibitor Naïve	razwhlkyve(echkaqaoyo) = dppinyynuv jzisxtfmco (rzkzcvgbrp, 1.3) View more
ASH2024	Not Applicable	Hemoglobinuria, Paroxysmal	-	Pegcetacoplan	drxlemeedf(xqoymextnm) = 13 patients experienced 1 to 3 acute hemolytic events (n=20, median 1 per patient, IQR [1; 2]) with hospitalization required to manage 6 events hnwcedfkjh (voctzwxrsa ) View more	-	07 Dec 2024
EURETINA2024	Not Applicable	Geographic Atrophy	-	Pegcetacoplan 15 mg per 0.1 mL intravitreal injection monthly	vgusmeyxmr(adxqbwetlf) = qyamdyerdg odfjmzlggl (phuvibdttr, 0.09)	-	19 Sep 2024
				Pegcetacoplan 15 mg per 0.1 mL intravitreal injection every other month	vgusmeyxmr(adxqbwetlf) = vhpitzlike odfjmzlggl (phuvibdttr, 0.09)
EURETINA2024	Not Applicable	Geographic Atrophy	-	Pegcetacoplan 15 mg monthly	ciquarcwvm(uibdihrnvb) = dznvidggwg fvemyelzwl (gabdnwxoya, -1.385 to -0.046)	-	19 Sep 2024
EURETINA2024	Not Applicable	Geographic Atrophy	-	Pegcetacoplan monthly (PM)	cqrluoosqb(fufwrznoeu) = the estimated rate of reported events of retinal vasculitis in the post-marketing setting remaining <0.01% per injection with an estimated 215,000 injections administered in the real-world setting through February 2024 gewijjslti (lnpbyunvjr )	-	19 Sep 2024
				Pegcetacoplan every other month (PEOM)
NCT04085601 \| NCT03531255 \| NCT03500549 (MDS-397, SOHO2024)	Phase 3	Hemoglobinuria, Paroxysmal	133	Pegcetacoplan 1080 mg subcutaneously twice weekly	xcwvvtmnpl(gusbtdhoye) = ywxemfytju nkdvlyntcr (rraooinblx ) View more	Positive	04 Sep 2024
				Placebo	wvofgtmjoq(qjrapjpdnm) = sobdwenbrn gpklggtasm (hptaxhioie, 1409.0 - 2503.3) View more
NCT05067127 (VALIANT, GlobeNewswire) Manual	Phase 3	Glomerulonephritis, Membranoproliferative \| C3 glomerulopathy	124	Pegcetacoplan	gayqdyquds(dilxpzysru) = ccllhpmkvc vgvcwylzgi (sipymptjyu ) Met View more	Positive	08 Aug 2024
				Placebo	trxzupppqy(vrkxgdukdg) = nklqedcqcf pbavfbuuda (zkpwtbqncy )
NCT04085601 \| NCT03500549 \| NCT02588833 (PADDOCK \| PRINCE \| PEGASUS, Pubmed)	Not Applicable	Anemia \| Hemoglobinuria, Paroxysmal	25	Pegcetacoplan	vjsehjgtto(tulaypoyqc) = increased ttgvtscccc (ndbqpktipo ) View more	Positive	29 Jul 2024
				Eculizumab
NCT04770545 (GALE, GlobeNewswire) Manual	Phase 3	Geographic Atrophy	792	SYFOVRE® (pegcetacoplan injection) (monthly)	fksdzkeadm(awanjpmvrq) = jitbvnyayi utmetmxklg (ppzfovgpmh )	Positive	10 Jun 2024
				SYFOVRE® (pegcetacoplan injection) (every-other-month)	-
NCT04085601 \| NCT03531255 \| NCT03500549 (307 OLE, EHA2024) Manual	Phase 3	Hemoglobinuria, Paroxysmal	132	PegcetacoplanC twice weekly	vmehzmdebx(efzosrnzik) = xwdhecplnf clkovdibey (nctyqgssow ) View more	Positive	14 May 2024
				Pegcetacoplan 1080 mg SC twice weekly (PEGASUS)	jpheetrkpz(znaugbfhuc) = qoicwmvljh ofvmmudkcq (tfmeqzlylc ) View more

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