Arrowhead Pharmaceuticals Analysis: $1.7B Raised
What is Arrowhead Pharmaceuticals?
RNAi technology targeting disease-causing genes
Product Features & Capabilities
- TRiM™ platform for RNA interference
- Plozasiran (ARO-APOC3) for severe hypertriglyceridemia
- Zodasiran (ARO-ANG3) for dyslipidemia
- ARO-RAGE for inflammatory pulmonary diseases
- Clinical trial services
Investment Focus
Arrowhead Pharmaceuticals is primarily focused on several therapeutic areas, including cardiometabolic diseases, pulmonary diseases, and inflammatory pulmonary diseases. Specific conditions they are targeting within these areas include severe hypertriglyceridemia, dyslipidemia, cardiovascular disease, obesity, and idiopathic pulmonary fibrosis.
Other Considerations
Key Innovations
- Plozasiran (ARO-APOC3) - Designed to reduce the production of Apolipoprotein-CIII (ApoC3) to lower triglyceride levels in patients with severe hypertriglyceridemia and familial chylomicronemia syndrome. It is currently in Phase 3 clinical trials and has received Breakthrough Therapy designation from the FDA.
- Zodasiran (ARO-ANG3) - Targets angiopoietin-like protein 3 (ANGPTL3) to lower serum LDL and triglyceride levels, currently in Phase 2 clinical trials.
- Olpasiran (AMG 890) - Aims to reduce lipoprotein(a) levels linked to cardiovascular diseases, also in Phase 3 clinical trials.
- Fazirsiran (ARO-AAT) - Developed for treating liver disease associated with alpha-1 antitrypsin deficiency, currently in Phase 3 clinical trials.
- ARO-ATXN2 - Investigational therapy targeting the toxic ATXN2 protein for the treatment of spinocerebellar ataxia type 2, currently in Phase 1 trials. Arrowhead utilizes its TRiM™ (Targeted RNAi Molecule) platform, which allows for tissue-specific targeting and simplifies manufacturing processes, potentially reducing costs and improving safety.
Regulatory Approvals
Arrowhead Pharmaceuticals has received several regulatory approvals and designations for its products, particularly for its investigational drug plozasiran. Key approvals include: 1. Acceptance of a New Drug Application (NDA) by the U.S. Food and Drug Administration (FDA) for plozasiran, aimed at treating familial chylomicronemia syndrome. 2. FDA Breakthrough Therapy Designation for plozasiran, which facilitates expedited development and review processes due to its potential to address unmet medical needs.
Additionally, Arrowhead has filed for regulatory clearances to initiate various Phase 1/2a clinical trials for other products, including ARO-ALK7, ARO-DM1, and ARO-INHBE.
