Arrowhead Pharmaceuticals Requests Regulatory Clearance to Initiate Phase 1/2a Study of ARO-ALK7 for the Treatment of Obesity
Arrowhead Pharmaceuticals (NASDAQ: ARWR) has filed for regulatory clearance to begin a Phase 1/2a clinical trial of ARO-ALK7, its second RNAi therapeutic for obesity treatment. The trial, AROALK7-1001, will evaluate safety and efficacy in up to 90 obese adults. The study includes two parts: monotherapy assessment and combination therapy with tirzepatide. ARO-ALK7 targets the ACVR1C gene in adipose tissue, aiming to reduce ALK7 production. Preclinical studies showed reduced body weight and fat mass while preserving lean muscle. The company now has two clinical-stage RNAi candidates for obesity treatment, demonstrating its leadership in delivering siRNA to multiple tissues using its TRiM™ platform.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) ha presentato una richiesta di autorizzazione per avviare uno studio clinico di Fase 1/2a per ARO-ALK7, il suo secondo farmaco terapeutico RNAi per il trattamento dell'obesità. Lo studio, AROALK7-1001, valuterà la sicurezza e l'efficacia su un massimo di 90 adulti obesi. La ricerca comprende due parti: la valutazione della monoterapia e la terapia combinata con tirzepatide. ARO-ALK7 mira a colpire il gene ACVR1C nel tessuto adiposo, con l'obiettivo di ridurre la produzione di ALK7. Gli studi preclinici hanno mostrato una diminuzione del peso corporeo e della massa grassa, preservando al contempo la massa muscolare magra. L'azienda ora dispone di due candidati RNAi in fase clinica per il trattamento dell'obesità, dimostrando la sua leadership nella somministrazione di siRNA a più tessuti mediante la piattaforma TRiM™.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) ha presentado una solicitud para obtener la autorización regulatoria para iniciar un ensayo clínico de Fase 1/2a de ARO-ALK7, su segunda terapia RNAi para el tratamiento de la obesidad. El ensayo, AROALK7-1001, evaluará la seguridad y la eficacia en hasta 90 adultos obesos. El estudio incluye dos partes: la evaluación de la monoterapia y la terapia combinada con tirzepatida. ARO-ALK7 tiene como objetivo el gen ACVR1C en el tejido adiposo, buscando reducir la producción de ALK7. Los estudios preclínicos mostraron una disminución del peso corporal y de la masa grasa, mientras se preservaba la masa muscular magra. La compañía ahora cuenta con dos candidatos RNAi en etapa clínica para el tratamiento de la obesidad, demostrando su liderazgo en la entrega de siRNA a múltiples tejidos utilizando su plataforma TRiM™.
Arrowhead Pharmaceuticals (NASDAQ: ARWR)는 비만 치료를 위한 두 번째 RNAi 치료제인 ARO-ALK7의 1/2a상 임상 시험을 시작하기 위한 규제 승인을 신청했습니다. 이 시험인 AROALK7-1001은 최대 90명의 비만 성인에서 안전성과 유효성을 평가합니다. 연구는 단독 요법 평가와 tirzepatide와의 병합 요법의 두 부분으로 나뉩니다. ARO-ALK7은 지방 조직의 ACVR1C 유표를 목표로 하여 ALK7 생산을 줄이는 것을 목표로 합니다. 전임상 연구에서는 지방량과 체중을 감소시키고, 근육량은 보존되는 결과를 보였습니다. 이 회사는 현재 비만 치료를 위한 두 가지 RNAi 후보 물질을 임상 단계에서 보유하고 있으며, TRiM™ 플랫폼을 사용하여 다양한 조직에 siRNA를 전달하는 선두 주자로 자리매김하고 있습니다.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) a déposé une demande d'autorisation réglementaire pour commencer un essai clinique de Phase 1/2a concernant ARO-ALK7, sa deuxième thérapie RNAi pour le traitement de l'obésité. L'essai, AROALK7-1001, évaluera la sécurité et l'efficacité chez jusqu'à 90 adultes obèses. L'étude comprend deux parties : l'évaluation de la monothérapie et la thérapie combinée avec le tirzepatide. ARO-ALK7 cible le gène ACVR1C dans le tissu adipeux, visant à réduire la production d'ALK7. Les études précliniques ont montré une réduction du poids corporel et de la masse grasse tout en préservant la masse musculaire maigre. L'entreprise dispose désormais de deux candidats RNAi en phase clinique pour le traitement de l'obésité, démontrant son leadership dans la délivrance de siRNA à plusieurs tissus grâce à sa plateforme TRiM™.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) hat eine Genehmigung zur Durchführung einer Phase 1/2a-Studie für ARO-ALK7, seine zweite RNAi-Therapie zur Behandlung von Fettleibigkeit, beantragt. Die Studie, AROALK7-1001, wird Sicherheit und Wirksamkeit bei bis zu 90 fettleibigen Erwachsenen bewerten. Die Untersuchung umfasst zwei Teile: die Bewertung der Monotherapie und die Kombinationstherapie mit Tirzepatid. ARO-ALK7 zielt auf das ACVR1C-Gen im Fettgewebe ab und hat das Ziel, die ALK7-Produktion zu reduzieren. Vorläufige Studien zeigten eine Verringerung des Körpergewichts und der Fettmasse, während die mageren Muskeln erhalten blieben. Das Unternehmen verfügt jetzt über zwei RNAi-Kandidaten in der klinischen Phase zur Behandlung von Fettleibigkeit und demonstriert damit seine Führungsposition in der Bereitstellung von siRNA für verschiedene Gewebe unter Verwendung seiner TRiM™-Plattform.
- First RNAi-based therapy directly targeting gene expression in adipose tissue
- Preclinical studies showed positive results in reducing body weight and fat mass while preserving lean muscle
- Potential complementary therapy to existing obesity treatments
- Expanded portfolio with two clinical-stage RNAi candidates for obesity
- Early-stage development (Phase 1/2a) with significant time before potential commercialization
- Regulatory approval pending for clinical trial initiation
- Will face competition from established obesity treatments like tirzepatide
Insights
The regulatory submission for ARO-ALK7's Phase 1/2a trial marks a significant advancement in obesity treatment research. The drug's novel mechanism targeting adipose tissue through RNAi represents a pioneering approach, distinct from current GLP-1 therapies. Key differentiators include:
- Direct targeting of adipose tissue gene expression
- Potential preservation of lean muscle mass
- Strong genetic validation from human loss-of-function carriers
The planned combination study with tirzepatide is particularly noteworthy, as it could lead to enhanced weight loss outcomes. The 90-patient trial design will provide important safety and efficacy data in both monotherapy and combination settings. This development positions Arrowhead as a potential player in the
This development strengthens Arrowhead's position in the rapidly growing obesity therapeutics market. Having two clinical-stage candidates (ARO-ALK7 and ARO-INHBE) with differentiated mechanisms demonstrates strategic portfolio diversification. The company's proprietary TRiM™ platform showcases technological leadership in RNAi delivery systems. The decision to target obesity aligns with market trends, as GLP-1 drugs have shown significant commercial success. The combination study with tirzepatide could potentially create a best-in-class treatment paradigm, opening doors for partnership opportunities with major pharmaceutical companies.
- In preclinical studies, investigational ARO-ALK7 silenced Activin receptor-like kinase 7 (ALK7) expression in adipose tissue, which led to reduced body weight and fat mass with preservation of lean muscle
“Arrowhead now has two clinical stage RNAi-based candidates, ARO-ALK7 and ARO-INHBE, for the treatment of obesity. Both have a novel mechanism of action, with ARO-INHBE inhibiting a ligand and ARO-ALK7 inhibiting a receptor, to intervene in a known pathway that signals the body to store fat in adipose tissue. Both targets are also supported by human genetics, where loss-of-function carriers have favorable body composition and metabolic characteristics compared to non-carriers,” said James Hamilton, M.D., Chief of Discovery and Translational Medicine at Arrowhead. “The upcoming Phase 1/2a clinical studies will evaluate single and multiple ascending doses as a monotherapy in obese patients as well as multiple doses in obese patients with or without type 2 diabetes in combination with incretin therapy. We believe ARO-INHBE and ARO-ALK7 have attractive profiles that may potentially fill gaps in the current standard of care and complement Arrowhead’s sharpened focus on development and commercialization of RNAi-based therapies in the cardiometabolic space.”
ARO-ALK7 is designed to silence adipocyte expression of the ACVR1C gene to reduce production of Activin receptor-like kinase 7 (ALK7), which acts as a receptor in a pathway that regulates energy homeostasis in adipose tissue. In large genetic datasets, reduced ACVR1C expression has been associated with healthier adipose distribution and reduced risk of obesity-related metabolic complications. Treatment with investigational ARO-ALK7 has the potential to reduce visceral adiposity and improve lipid and glycemic parameters.
An application for approval to initiate the clinical trial was submitted to the New Zealand Medicines and Medical Devices Safety Authority for review by the Standing Committee on Therapeutic Trials. Pending clearance, Arrowhead intends to proceed with AROALK7-1001, A Phase 1/2a first-in-human dose-escalating study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ARO-ALK7 in up to 90 adult volunteers with obesity. Part 1 of the study is designed to assess single and multiple doses of ARO-ALK7 monotherapy, and Part 2 of the study is designed to assess ARO-ALK7 in combination with tirzepatide, a subcutaneously administered GLP-1/GIP receptor co-agonist that has been approved in
About Arrowhead Pharmaceuticals
Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing.
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Source: Arrowhead Pharmaceuticals, Inc.
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Arrowhead Pharmaceuticals, Inc.
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ir@arrowheadpharma.com
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Source: Arrowhead Pharmaceuticals, Inc.
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