Annexon Provides Update on ARCHER II Global Registrational Program in Geographic Atrophy
Annexon Inc (ANNX) has initiated patient dosing in the global Phase 3 ARCHER II trial for ANX007, a C1q inhibitor targeting geographic atrophy (GA). The trial aims to evaluate ANX007's potential to protect vision in GA patients, with topline data expected in H2 2026. New data from the Phase 2 ARCHER trial showed:
1. Significant vision protection in standard and low light conditions
2. Structural protection in key retinal areas for visual acuity
3. Up to 60% protection of photoreceptors in the central fovea
4. 29% protection of photoreceptors across the full retinal field
ANX007 is the only GA therapy candidate to receive PRIME designation in the EU and Fast Track designation from the FDA. The Phase 3 trial will enroll approximately 630 patients, using best corrected visual acuity (BCVA) protection against ≥15-letter loss as the primary outcome measure.
Annexon Inc (ANNX) ha avviato la somministrazione di farmaci ai pazienti nel trial globale di Fase 3 ARCHER II per ANX007, un inibitore del C1q mirato all'atrofia geografica (GA). L'obiettivo dello studio è valutare il potenziale di ANX007 nel proteggere la vista nei pazienti con GA, con i dati principali previsti per la seconda metà del 2026. Nuovi dati dal trial di Fase 2 ARCHER hanno mostrato:
1. Una significativa protezione della vista in condizioni di luce standard e scarsa
2. Protezione strutturale in aree retiniche chiave per l'acuità visiva
3. Fino al 60% di protezione dei fotorecettori nella fovea centrale
4. 29% di protezione dei fotorecettori nell'intero campo retinico
ANX007 è l'unico candidato terapia per GA ad aver ricevuto la designazione PRIME nell'UE e la designazione Fast Track dalla FDA. Lo studio di Fase 3 arruolerà circa 630 pazienti, utilizzando la protezione dell'acuità visiva corretta (BCVA) contro una perdita ≥15 lettere come misura primaria dell'esito.
Annexon Inc (ANNX) ha iniciado la dosificación de pacientes en el ensayo global de Fase 3 ARCHER II para ANX007, un inhibidor de C1q dirigido a la atrofia geográfica (GA). El ensayo tiene como objetivo evaluar el potencial de ANX007 para proteger la visión en pacientes con GA, con datos preliminares esperados en la segunda mitad de 2026. Nuevos datos del ensayo de Fase 2 ARCHER mostraron:
1. Protección significativa de la visión en condiciones de luz estándar y baja
2. Protección estructural en áreas retinianas clave para la agudeza visual
3. Hasta un 60% de protección de los fotorreceptores en la fóvea central
4. 29% de protección de los fotorreceptores en todo el campo retinal
ANX007 es el único candidato a terapia para GA que ha recibido la designación PRIME en la UE y la designación Fast Track de la FDA. El ensayo de Fase 3 inscribirá aproximadamente a 630 pacientes, utilizando la protección de la agudeza visual corregida (BCVA) contra una pérdida ≥15 letras como medida principal de resultado.
Annexon Inc (ANNX)는 지리적 위축(GA)을 표적하는 C1q 억제제인 ANX007에 대한 글로벌 3상 ARCHER II 시험에서 환자 투약을 시작했습니다. 이 시험은 GA 환자의 시력을 보호할 수 있는 ANX007의 잠재력을 평가하는 것을 목표로 하며, 주요 데이터는 2026년 하반기에 나올 것으로 예상됩니다. 2상 ARCHER 시험에서 신규 데이터는 다음과 같은 내용을 보여주었습니다:
1. 표준 및 저조도 조건에서의 눈 보호 효과
2. 시력 기준에 중요한 망막 영역의 구조적 보호
3. 중심 황반에서 최대 60%의 광수용체 보호
4. 전체 망막 영역에서 29%의 광수용체 보호
ANX007은 EU에서 PRIME 지정을 받은 유일한 GA 치료 후보이며 FDA에서 Fast Track 지정을 받았습니다. 3상 시험은 약 630명의 환자를 등록하며, 주요 결과 측정 지표로는 15자 이상의 손실에 대한 시력 교정(BSVA) 보호를 사용합니다.
Annexon Inc (ANNX) a lancé l'administration du traitement aux patients dans l'essai mondial de Phase 3 ARCHER II pour ANX007, un inhibiteur de C1q ciblant l'atrophie géographique (GA). L'essai vise à évaluer le potentiel d'ANX007 à protéger la vision des patients atteints de GA, avec des données préliminaires attendues au second semestre 2026. De nouvelles données de l'essai de Phase 2 ARCHER ont montré :
1. Protection significative de la vision dans des conditions de lumière standard et faible
2. Protection structurelle dans des zones rétiniennes clés pour l'acuité visuelle
3. Jusqu'à 60 % de protection des photorécepteurs dans la fovéa centrale
4. 29 % de protection des photorécepteurs sur l'ensemble du champ rétinien
ANX007 est le seul candidat thérapeutique contre la GA à avoir reçu la désignation PRIME dans l'UE et la désignation Fast Track de la FDA. L'essai de Phase 3 recrutera environ 630 patients, utilisant la protection de l'acuité visuelle corrigée (BCVA) contre une perte de ≥15 lettres comme mesure principale des résultats.
Annexon Inc (ANNX) hat die Patientendosierung im globalen Phase 3 ARCHER II-Studie für ANX007, einen C1q-Inhibitor zur Behandlung von geographischer Atrophie (GA), begonnen. Die Studie zielt darauf ab, das Potenzial von ANX007 zum Schutz des Sehvermögens bei GA-Patienten zu bewerten, wobei die Ergebnisse im zweiten Halbjahr 2026 erwartet werden. Neue Daten aus der Phase 2 ARCHER-Studie zeigten:
1. Bedeutender Schutz des Sehvermögens unter Standard- und schlechten Lichtverhältnissen
2. Struktureller Schutz in wichtigen retinalen Bereichen für die Sehschärfe
3. Bis zu 60% Schutz der Fotorezeptoren in der zentralen Fovea
4. 29% Schutz der Fotorezeptoren über das gesamte RetinAGEFELD
ANX007 ist der einzige GA-Therapiekandidat, der in der EU den PRIME-Status erhalten hat und von der FDA als Fast Track eingestuft wurde. Die Phase-3-Studie wird etwa 630 Patienten einschließen und den Schutz der besten korrigierten Sehschärfe (BCVA) gegen einen Verlust von ≥15 Buchstaben als primäres Ergebnismaß verwenden.
- Initiated patient dosing in Phase 3 ARCHER II trial for ANX007 in geographic atrophy
- Phase 2 ARCHER trial showed significant vision protection in standard and low light conditions
- Demonstrated up to 60% protection of photoreceptors in the central fovea
- Received PRIME designation in EU and Fast Track designation from FDA
- Phase 3 trial to enroll approximately 630 patients
- Topline data from Phase 3 ARCHER II trial not expected until second half of 2026
Insights
The initiation of patient dosing in the Phase 3 ARCHER II trial for ANX007 in geographic atrophy (GA) is a significant milestone for Annexon. This global registrational study, with topline data expected in H2 2026, could potentially lead to the first GA therapy approved to protect vision. The trial's design, using visual protection as the primary endpoint, aligns with both FDA and EMA requirements, which is important for regulatory approval.
The new Phase 2 ARCHER trial data presented at ASRS are highly encouraging. ANX007 demonstrated statistically significant protection of vision in both standard and low light conditions, with a
The advancement of ANX007 into Phase 3 trials represents a significant value driver for Annexon (NASDAQ: ANNX). The geographic atrophy market is substantial, with over 8 million patients worldwide, presenting a large commercial opportunity. ANX007's potential best-in-class profile, demonstrated by its unique ability to protect both visual acuity and key visual structures, could lead to strong market positioning if approved.
Investors should note the regulatory advantages ANX007 has secured, including PRIME designation in the EU and Fast Track designation from the FDA. These designations could potentially accelerate the approval process and provide a competitive edge. However, with topline data expected in H2 2026, Annexon will need to ensure adequate funding through this extended development period. The company's ability to advance ANX007 while managing cash burn will be important for long-term value creation.
While I typically focus on cancer treatments, the approach used in ANX007 for geographic atrophy (GA) shares similarities with some targeted therapies in oncology. The drug's mechanism as a C1q inhibitor targets the complement cascade, which is involved in inflammation and tissue damage in various diseases, including some cancers.
The disease-modifying potential of ANX007, as evidenced by its protection of photoreceptors and visual acuity, is particularly intriguing. In oncology, we strive for treatments that not only slow disease progression but also preserve organ function. ANX007's ability to protect both structure (photoreceptors) and function (vision) in GA patients mirrors this ideal. The dose-dependent response observed in the Phase 2 trial is also encouraging, as it suggests a clear biological effect. However, as with any novel therapy, long-term safety data from the Phase 3 trial will be important to fully assess the risk-benefit profile of ANX007.
Patient Dosing Initiated in Phase 3 ARCHER II Trial of C1q Inhibitor ANX007; Topline Data Expected in Second Half 2026
Additional Data from Phase 2 ARCHER Trial Demonstrated Both Significant Vision Protection in Standard and Low Light Conditions, and Significant Structural Protection in Regions of the Eye Important for Visual Acuity
BRISBANE, Calif., Aug. 05, 2024 (GLOBE NEWSWIRE) -- Annexon, Inc. (Nasdaq: ANNX), a biopharmaceutical company advancing novel complement therapies for neuroinflammatory diseases of the body, brain and eye, today announced patient dosing was initiated in the global pivotal Phase 3 ARCHER II trial evaluating ANX007 for the treatment of geographic atrophy (GA), the only GA pivotal program utilizing visual protection as the global regulatory-aligned primary end point. Additionally, Annexon announced new data regarding protection of vision and vision-related retinal structure from the Phase 2 ARCHER trial, supporting the disease modifying potential of ANX007 for the treatment of GA. These data were recently presented on July 18, 2024, at the American Society of Retina Specialists (ASRS) Annual Scientific meeting.
GA is a chronic, progressive neurodegenerative disease that is the leading cause of blindness for over 8 million elderly people worldwide, resulting in the loss of independence and major disruption in their daily lives. Although there are two currently FDA-approved treatments to slow disease progression in GA, there are currently no approved treatments indicated to protect vision in GA.
“ANX007 is the only investigational medicine for GA to date to show in a randomized clinical trial significant protection of both visual acuity and key visual structures in regions of the eye essential for vision,” said Douglas Love, president and chief executive officer of Annexon. “These data underscore ANX007’s potential best-in-class profile and make all the more exciting the initiation of the ARCHER II global, pivotal Phase 3 study designed to treat patients impacted by this devastating disease at a vulnerable stage in their lives. Assuming success in the ARCHER II trial, ANX007 has the potential to be the first GA therapy approved to protect vision.”
ARCHER II is a global, randomized, sham-controlled Phase 3 trial with topline data expected in the second half of 2026. The ANX007 registrational program has received regulatory alignment with both the Food and Drug Administration (FDA) and the European Medicines Agency (EMA) on key study elements, including on using, for the first-time in GA, best corrected visual acuity (BCVA) protection against ≥15-letter loss as the primary outcome measure (equivalent to three lines on a standardized eye chart). In ARCHER II, ANX007 will be compared to sham control in a well-powered study with a robust safety database that is expected to enroll approximately 630 patients. ANX007 is the only therapeutic candidate for GA to date to receive Priority Medicine (PRIME) designation in the European Union, and has been granted Fast Track designation from the FDA.
Eleonora Lad, MD, PhD, Vice Chair of Clinical Research at Duke Eye Center added, “Treatments for GA have primarily focused on addressing lesion growth by measuring protection of the retinal pigment epithelium (RPE), supportive cells that do not detect light across the retina. Unfortunately, current treatments have not translated to protection of clinically meaningful vision for patients. The new analyses of the Phase 2 ARCHER trial are very encouraging, demonstrating protection of photoreceptors in the central fovea, the region of the retina needed for important activities such as reading, driving and seeing faces. These data shed new insights into the mechanism of ANX007 to potentially modify disease activity, leading to protection of vision loss in GA.”
Data highlights from the ARCHER Phase 2 analyses presented at the ASRS Annual meeting include:
ANX007 provided broad-based protection of vision vs. sham-treated eyes
- Statistically significant and dose dependent protection of vision measured by best corrected visual acuity (BCVA) protection against ≥15-letter loss at month 12, which represents three lines on the standard Early Treatment of Diabetic Retinopathy Study (ETDRS) eye chart and is a widely accepted clinically meaningful assessment of visual acuity (
21.3% (sham) vs.5.6% (ANX007 monthly); p = 0.0021) - Statistically significant protection of vision in low light conditions, low luminance visual acuity (LLVA) at month 12, which is an important assessment of visual performance for everyday activity (LLVA ≥15-letter loss at month 12:
20.3% (sham) vs.7.6% (ANX007 monthly); p = 0.022)
ANX007 provided structural protection in regions of the retina critical for visual acuity
- Photoreceptors are neurons in the retina responsible for detecting light, and thus protecting their structure is essential for protecting vision
- ANX007 provided up to
60% protection of photoreceptors compared to sham within the central 1.5 mm of the fovea, as measured by ellipsoid zone (EZ) through 12 months (p = 0.0319*) - ANX007 provided
29% protection of photoreceptors across the full retinal field as measured by EZ through 12 months (p = 0.017*) - ANX007 demonstrated a trend of protection against RPE loss in the central foveal subfield, the region where RPE loss best correlates with vision loss (
18% protection of RPE loss, p = 0.40*). RPE loss is a measure that lags behind photoreceptor loss.
*nominal p-value
About ANX007
ANX007 is a non-pegylated antigen-binding fragment (Fab) antibody designed as a first-in-kind therapeutic to selectively inhibit C1q, a key driver of neurodegeneration. In geographic atrophy (GA), C1q binds to photoreceptor synapses early in the disease process, causing inflammation, synapse loss, and neuronal damage that results in vision loss. Vision loss precedes the loss of retinal pigment epithelium (RPE), the traditional biomarker used for previous Food and Drug Administration (FDA) approvals in GA. Intravitreal administration of ANX007 fully stops C1q and activation of its inflammatory pathway. In the Phase 2 ARCHER trial, ANX007 was shown to preserve vision on multiple measures and provided significant structural protection within the retina, particularly of photoreceptors in the central fovea that are important to visual acuity. Additionally, ANX007 was generally well-tolerated through month 12, with no increase in choroidal neovascularization (CNV) rates between the treated and sham arms and no events of retinal vasculitis reported. ANX007 has been granted Fast Track designation from the FDA and is the first therapeutic candidate for the treatment of GA to receive Priority Medicine (PRIME) designation in the European Union, which provides early and proactive support to developers of promising medicines that may offer a major therapeutic advantage over existing treatments or benefit to patients without treatment options.
About Phase 3 ARCHER II Trial
ARCHER II is a global, randomized, double-masked, sham-controlled Phase 3 trial expected to enroll approximately 630 patients with geographic atrophy (GA) secondary to age-related macular degeneration who will be randomized 2:1 to receive a monthly dose of ANX007 or sham procedure. The primary endpoint is the prevention of ≥15-letter loss of best corrected visual acuity (BCVA), which represents three lines on the standard Early Treatment of Diabetic Retinopathy Study (ETDRS) eye chart. The primary analysis will occur between 12 and 18 months from dosing initiation based on the accumulation of target events (patients in the overall study experiencing BCVA ≥15-letter loss on consecutive visits). Proportion of patients experiencing BCVA ≥15-letter loss is a well-established functional endpoint that has served as the basis for numerous ophthalmology drug approvals by the Food and Drug Administration (FDA) and European Medicines Agency (EMA). Secondary endpoints in ARCHER II include safety, low-luminance visual acuity (LLVA), and photoreceptor integrity (EZ). Topline data are expected in the second half of 2026.
Plans for an injection-controlled study, ARROW, to assess the prevention of ≥15-letter loss of BCVA, are also ongoing.
About Geographic Atrophy
Geographic atrophy (GA) is an advanced form of dry age-related macular degeneration (AMD), an eye disease that is the leading cause of blindness in the elderly. GA is a chronic progressive neurodegenerative disorder of the retina involving the loss of photoreceptor synapses and cells in the outer retina. GA affects an estimated one million people in the United States and eight million people globally, severely limiting their independence and causing frustration, anxiety and emotional hardship. Effective treatments that preserve vision are still needed, as no currently approved therapies have been shown in clinical trials to significantly protect vision.
About Annexon
Annexon Biosciences (Nasdaq: ANNX) is a biopharmaceutical company advancing a late-stage clinical platform of novel therapies for people living with devastating classical complement-mediated neuroinflammatory diseases of the body, brain, and eye. Annexon’s novel scientific approach targets upstream C1q to block the classical complement inflammatory cascade before it starts, and its therapeutic candidates are designed to provide meaningful benefits across multiple autoimmune, neurodegenerative and ophthalmic diseases. With proof-of concept data in Guillain-Barré syndrome, Huntington’s disease and geographic atrophy, Annexon is rigorously advancing its mid-to late-stage clinical trials to bring their potential treatments to patients as quickly as possible. To learn more visit annexonbio.com.
Forward Looking Statements
This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. In some cases, you can identify forward-looking statements by terminology such as “aim,” “anticipate,” “assume,” “believe,” “contemplate,” “continue,” “could,” “design,” “due,” “estimate,” “expect,” “goal,” “intend,” “may,” “objective,” “plan,” “positioned,” “potential,” “predict,” “seek,” “should,” “suggest,” “target,” “on track,” “will,” “would” and other similar expressions that are predictions of or indicate future events and future trends, or the negative of these terms or other comparable terminology. All statements other than statements of historical facts contained in this press release are forward-looking statements. These forward-looking statements include, but are not limited to, the ability of ANX007 to block upstream C1q, the clinical and regulatory status of ANX007; ANX007’s distinct potential neuroprotective mechanism of action and potential to provide protection from vision loss; the potential of ANX007 to become best-in-class treatment for GA; the potential therapeutic benefit of ANX007; the anticipated timing of data from the Phase 3 ARCHER II trial and plans for the Phase 3 ARROW trial; and Annexon’s ability to rigorously advance mid- to late-stage clinical trials and continue development of the company’s portfolio. Forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties that could cause actual results and events to differ materially from those anticipated, including, but not limited to, risks and uncertainties related to: the ongoing off-treatment follow-up portion of the ARCHER trial and final results from the ARCHER trial; the company’s history of net operating losses; the company’s ability to obtain necessary capital to fund its clinical programs; the early stages of clinical development of the company’s product candidates; the effects of public health crises on the company’s clinical programs and business operations; the company’s ability to obtain regulatory approval of and successfully commercialize its product candidates; any undesirable side effects or other properties of the company’s product candidates; the company’s reliance on third-party suppliers and manufacturers; the outcomes of any future collaboration agreements; and the company’s ability to adequately maintain intellectual property rights for its product candidates. These and other risks are described in greater detail under the section titled “Risk Factors” contained in the company’s Annual Report on Form 10-K and Quarterly Reports on Form 10-Q and the company’s other filings with the SEC. Any forward-looking statements that the company makes in this press release are made pursuant to the Private Securities Litigation Reform Act of 1995, as amended, and speak only as of the date of this press release. Except as required by law, the company undertakes no obligation to publicly update any forward-looking statements, whether as a result of new information, future events or otherwise.
Investor Contact:
Joyce Allaire
LifeSci Advisors, LLC
jallaire@lifesciadvisors.com
Media Contact:
Sheryl Seapy
Real Chemistry
949-903-4750
sseapy@realchemistry.com
FAQ
What is the primary endpoint for Annexon's Phase 3 ARCHER II trial for ANX007?
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