Delving into the Latest Updates on Avacincaptad pegol with Synapse

Overview

Basic Info

Drug Type

RNA aptamer

Synonyms

Anti-C5 aptamer, Avacincaptad pegol sodium (USAN), Zimura

+ [4]

Target

C5

Mechanism

C5 inhibitors(Complement C5 inhibitors)

Therapeutic Areas

Congenital DisordersEye DiseasesOther Diseases

Active Indication

Geographic AtrophyAge Related Macular DegenerationDystrophy, Macular+ [1]

Inactive Indication

Originator Organization

Ocular Therapeutix, Inc.

Active Organization

Astellas Pharma Global Development, Inc.

Archemix Corp.Astellas Pharma US, Inc.

Inactive Organization

IVERIC bio, Inc.

Astellas Pharma, Inc.Ophthotech Corp. (New Jersey)

Drug Highest PhaseApproved

First Approval Date

US (04 Aug 2023),

Geographic Atrophy

RegulationPriority Review (US), Breakthrough Therapy (US)

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Structure/Sequence

Sequence Code 29451050

Source: *****

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.

01 Nov 2024·Ophthalmology Retina

Vision Loss Reduction with Avacincaptad Pegol for Geographic Atrophy

Article

Author: Lally, David R ; Kovach, Jaclyn L ; Chakravarthy, Usha ; Sheth, Veeral S ; Kaiser, Peter K ; Desai, Dhaval ; Chang, Margaret A ; Rachitskaya, Aleksandra ; Tang, Justin ; Zhu, Liansheng ; Clark, Julie ; Khanani, Arshad M ; Vajzovic, Lejla ; Danzig, Carl J

PURPOSE:

To evaluate the impact of reduction in geographic atrophy (GA) lesion growth on visual acuity in the GATHER trials using categorical outcome measures.

DESIGN:

Randomized, double-masked, sham-controlled phase 3 trials.

PARTICIPANTS:

Aged ≥50 years with noncenter point-involving GA and best-corrected visual acuity (BCVA) of 25 to 80 ETDRS letters in the study eye.

METHODS:

GATHER1 consisted of 2 parts. In part 1, 77 patients were randomized 1:1:1 to avacincaptad pegol (ACP) 1 mg, ACP 2 mg, and sham. In part 2, 209 patients were randomized 1:2:2 to ACP 2 mg, ACP 4 mg, and sham. In GATHER2, patients were randomized 1:1 to ACP 2 mg (n = 225) and sham (n = 223). A post hoc analysis of 12-month data for pooled ACP 2 mg and sham groups is reported.

MAIN OUTCOME MEASURES:

Proportion of study eyes that experienced ≥10-, ≥15-, or ≥20-BCVA ETDRS letter loss from baseline to month 12; time-to-event analysis of persistent vision loss of ≥10, ≥15, or ≥20 BCVA letters from baseline at ≥2 consecutive visits over 12 months; proportion of study eyes with BCVA loss to a level below driving eligibility threshold at month 12 among those eligible to drive at baseline.

RESULTS:

Lower proportions of study eyes experienced ≥10-, ≥15-, or ≥20-BCVA letter loss from baseline over 12 months with ACP 2 mg (11.6%, 4.0%, and 1.6%, respectively) versus sham (14.1%, 7.6%, and 4.5%, respectively). There was a reduction in the risk of persistent loss of ≥15 BCVA ETDRS letters with ACP 2 mg (3.4%) versus sham (7.8%) through 12 months. A lower proportion of study eyes treated with ACP 2 mg reached the threshold for driving ineligibility versus sham by 12 months.

CONCLUSIONS:

Treatment with ACP 2 mg delayed the risk of progression to persistent vision loss (i.e., ≥10-, ≥15-, and ≥20-BCVA letter loss or BCVA loss to a level below driving eligibility threshold) versus sham over 12 months.

FINANCIAL DISCLOSURE(S):

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

01 Jan 2025·INDIAN JOURNAL OF OPHTHALMOLOGY

Dry and neovascular “wet” age-related macular degeneration: Upcoming therapies

Article

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

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.

108

News (Medical) associated with Avacincaptad pegol

26 Nov 2024

·endpts.com

Aviceda aims to raise $200M; Nerviano gets back rights to PARP1 inhibitor

Plus, news about Maat Pharma, Secretome Therapeutics, 35Pharma, Medigene and Doron Therapeutics: Aviceda Therapeutics’ $200M financing: The Boston-area biotech is looking to raise a $200 million equity round, according to an SEC filing on Friday. The company recently completed enrollment for a Phase 2b study testing its investigational drug, called AVD-104, against Astellas’ Izervay in patients with geographic atrophy secondary to age-related macular degeneration. — Kyle LaHucik Nerviano’s dealmaking: Nerviano Medical Sciences will reacquire the worldwide rights to a PARP1 inhibitor dubbed NMS-293 from Merck KGaA. The drug is in a Phase 2 study in relapsed glioblastoma. The Italian biotech had received $65 million in upfront and option fees, the companies disclosed in September 2022. Earlier this month, NMS also bought a Phase 2-ready PARP7 inhibitor from Ribon Therapeutics ., which has shuttered. Terms of both deals were undisclosed. — Kyle LaHucik MaaT Pharma touts positive Phase 1b data for its ALS drug: The topline data shared Tuesday showed that the trial met its primary endpoint, assessing the safety and tolerability of the drug, dubbed MaaT033, with multiple doses in ALS patients over two months. More data are expected early next year. According to the company, the data safety and monitoring board for the trial supported moving the drug into a Phase 2 study. — Katherine Lewin Secretome Therapeutics raises $20.4M for heart therapies: The cell therapy company plans to use the proceeds to launch two Phase 1 trials for its lead asset, STM-01, in heart failure with preserved ejection fraction and dilated cardiomyopathy. The studies are expected to start by the end of the year. — Ayisha Sharma 35Pharma’s $35M Series C: Frazier Life Sciences led the financing round for the clinical-stage biopharma, which wants to use the money to support clinical proof-of-concept studies for its candidate in pulmonary hypertension and get its asset through a Phase 1 trial in HFpEF and obesity. — Katherine Lewin Medigene to lay off approximately 40% of its workforce: Along with the layoffs, the company is refocusing its capital on R&D programs with the “highest potential for return on invested capital.” As of Dec. 31, 2023, the company had 86 employees. — Katherine Lewin Doron Therapeutics bags $11M in Series A: The company said the funds will support the Chapel Hill, NC-based biotech as it advances its regenerative medicine candidate, Motys, into Phase 3 development for symptomatic knee osteoarthritis. The asset could offer “potent and durable effects from a single injection” as an alternative to surgical interventions, according to Doron. — Ayisha Sharma

Phase 2Phase 1Cell TherapyPhase 3

20 Nov 2024

·www.pharmaceutical-technology.com

FDA issues CRL to Astellas’ IZERVAY sNDA for geographic atrophy

IZERVAY is an intravitreal solution to treat geographic atrophy secondary to age-related macular degeneration. Credit: fizkes/Shutterstock. The US Food and Drug Administration (FDA) has issued a complete response letter (CRL) for the supplemental new drug application (sNDA) of Astellas Pharma ’s IZERVAY (avacincaptad pegol intravitreal solution), a treatment for geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The FDA’s feedback is unrelated to IZERVAY’s safety or efficacy and centres on statistical aspects concerning the proposed labelling language. Astellas is seeking further FDA clarification and will resolve the regulator’s concerns promptly. The application aimed to update the US prescribing information for IZERVAY with positive two-year outcomes from the Phase III GATHER2 trial. This trial investigated the efficacy and safety of IZERVAY with monthly and alternate monthly dosing over two years. However, the FDA has indicated that it cannot approve the application in its current state by the Prescription Drug User Fee Act (PDUFA) action date set on 19 November 2024. In August 2023, the US FDA approved IZERVAY for GA secondary to AMD. The drug met its primary endpoint in the two Phase III, double-masked, sham-controlled, multicentre, and randomised GATHER1 and GATHER2 trials. These involved more than 700 subjects and were designed to evaluate the efficacy and safety of a monthly 2mg intravitreal administration of IZERVAY. In the first 12 months of the trials, 286 individuals in GATHER1 and 448 in GATHER2 were randomised to receive either IZERVAY 2mg or a sham treatment monthly. The primary efficacy endpoints were measured by fundus autofluorescence at baseline, month six, and month 12, with safety evaluated across both pivotal studies. Astellas Pharma biopharma and ophthalmology development senior vice-president Marci English stated: “Astellas stands by the clinical profile of IZERVAY, the only FDA-approved GA treatment that consistently demonstrated statistically significant slowing of GA across two pivotal Phase III studies. “While this is a disappointment for patients and physicians who rely on IZERVAY for the management of a chronic, progressive disease that can lead to irreversible vision loss, Astellas is unwavering in our commitment to the ophthalmology space and will continue to work with the FDA to advance solutions for those suffering from GA.”

Phase 3Clinical ResultNDA

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

100 Deals associated with Avacincaptad pegol

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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.

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Indication	Highest Phase	Country/Location	Organization	Date
Age Related Macular Degeneration	Phase 3	US	Astellas Pharma Global Development, Inc.	26 Sep 2022
Age Related Macular Degeneration	Phase 3	AR	Astellas Pharma Global Development, Inc.	26 Sep 2022
Age Related Macular Degeneration	Phase 3	AU	Astellas Pharma Global Development, Inc.	26 Sep 2022
Age Related Macular Degeneration	Phase 3	AT	Astellas Pharma Global Development, Inc.	26 Sep 2022
Age Related Macular Degeneration	Phase 3	BE	Astellas Pharma Global Development, Inc.	26 Sep 2022
Age Related Macular Degeneration	Phase 3	BR	Astellas Pharma Global Development, Inc.	26 Sep 2022
Age Related Macular Degeneration	Phase 3	CA	Astellas Pharma Global Development, Inc.	26 Sep 2022
Age Related Macular Degeneration	Phase 3	CO	Astellas Pharma Global Development, Inc.	26 Sep 2022
Age Related Macular Degeneration	Phase 3	HR	Astellas Pharma Global Development, Inc.	26 Sep 2022
Age Related Macular Degeneration	Phase 3	CZ	Astellas Pharma Global Development, Inc.	26 Sep 2022

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02686658 \| NCT04435366 (GATHER2, EURETINA2024) Manual	Phase 2/3	Geographic Atrophy	-	Avacincaptad Pegol 2 mg	ygvahjmrqa(hqdfboaqfj) = nsksqaoyvs hxyscvystc (zzqtjiwywq )	Positive	19 Sep 2024
GATHER2 (PRNewswire) Manual	Phase 3	Age Related Macular Degeneration	-	IZERVAY	uadllkkyff(lafjinrraz) = 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. dfsdwmzkmc (ctjrywquan ) View more	Positive	04 Nov 2023
				IZERVAY
EURETINA2023	Not Applicable	Geographic Atrophy	-	Avacincaptad pegol 2 mg	dknllvrmzf(diiuwshcrw) = fuktlecylm gqdsarlixc (yqrhomhpyi, 0.56 - 0.15-2.06)	-	05 Oct 2023
EURETINA2023	Not Applicable	-	-	Avacincaptad pegol 2 mg	vepriqxouk(bzsnmuyjwy) = xszyijjcsn nuleniiawv (dilejrazun ) View more	-	05 Oct 2023
NCT02686658 \| NCT04435366 (GATHER2, FDA) Manual	Phase 2/3	Geographic Atrophy	624	IZERVAY (GATHER1)	zikzjzcdxp(wvctnqbtfw) = odoglbylqi vxwdnixtkz (teoamneici )	Positive	04 Aug 2023
				Sham (GATHER1)	zikzjzcdxp(wvctnqbtfw) = bfgbqahenb vxwdnixtkz (teoamneici )
NCT05571267 (CTgov)	Phase 2	Age Related Macular Degeneration	1	Zimura+avastin (Zimura and Avastin)	vjkytvrznh(zyocjadwcc) = exssalboii mhonbdgzrr (yqdbjspdfj, qkbwfcqjic - cljmxdhiwv) View more	-	03 Apr 2023
				Zimura+Lucentis (Zimura and Lucentis)	eepdprxhls(rsihhklpgu) = rpdajqnccj ckstahndwr (tquqevziqv, wvoutfqouh - epvbjnopeu) View more
NCT04435366 (GATHER2, Corporate Publications) Manual	Phase 3	Geographic Atrophy	447	Avacincaptad pegol	xanimnkswz(hbdlkgontw) = mhoawerpfw jezodnxifd (tygaujbqax ) View more	Positive	06 Sep 2022
				Sham	xanimnkswz(hbdlkgontw) = nnrgnsydwy jezodnxifd (tygaujbqax ) View more
GATHER1 (ARVO2022)	Phase 2/3	Geographic Atrophy	-	Avacincaptad pegol 2 mg	flexfenfil(vwahsthyhq) = wcycufwyzu itjpywzmei (thpnuznzxb )	-	01 May 2022
				avacincaptad pegol (Sham)	flexfenfil(vwahsthyhq) = jomlshtnfp itjpywzmei (thpnuznzxb )
NCT03362190 (CTgov)	Phase 2	Wet age-related macular degeneration	64	Zimura+Lucentis (Cohort 1)	pdzlisuyvb(mcksuaogva) = sgkjfbjttg fcnltndnoh (exhbsucrhe, fnhmthbnau - zmylgxobns) View more	-	02 Feb 2022
				Zimura+Lucentis (Cohort 2)	pdzlisuyvb(mcksuaogva) = ldasmljevd fcnltndnoh (exhbsucrhe, bguwrvgxcn - ljyybovges) View more
GATHER1 (AAO2021)	Not Applicable	-	286	Avacincaptad Pegol 2mg	qaezpkmvuc(bjuqgejyvf) = zbauhuotbk qojxhqbuzc (tnxqdpbger )	-	13 Nov 2021
				Avacincaptad Pegol 4mg	qaezpkmvuc(bjuqgejyvf) = tqkoblkbcj qojxhqbuzc (tnxqdpbger )