Company: ARWR
Filing Date: 2025-10-10
Form Type: 10-K/A
Source: 0000879407-25-000013
Chunk: 17

Company: ARROWHEAD PHARMACEUTICALS, INC.
Filing Date: 2025-10-10
Form: 10-K/A
Chunk 17
---
 treatment for spinocerebellar ataxia 2 (SCA2). SCA2 is a progressive cerebellar ataxia with instability of stance, speech and swallow disorder, pain, spasticity, and ocular signs, caused by gain of function of mutant expanded polyQ ATXN2 protein. The Company is currently investigating ARO-ATXN2 in a Phase 1 clinical trial.

• Study Name: Study of ARO-ATXN2 Injection in Adults With Spinocerebellar Ataxia Type 2 A Phase 1 Placebo-Controlled Dose Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-ATXN2 in Adult Subjects With Spinocerebellar AtaxiaType 2 ClinicalTrials.gov Identifier: NCT06672445

ARO-HTT is designed to reduce the expression of the Huntingtin gene as a potential treatment for Huntington’s disease.

ARO-ATXN1 is designed to reduce the expression of the ATXN1 gene as a potential treatment for spinocerebellar ataxia 1 (SCA1).

ARO-ATXN3 is designed to reduce the expression of the ATXN3 gene as a potential treatment for spinocerebellar ataxia 3 (SCA3).

Takeda Pharmaceuticals U.S.A., Inc.

Fazirsiran (formerly ARO-AAT) is a clinical-stage RNAi therapeutic candidate for the treatment of liver disease associated with alpha-1 antitrypsin deficiency. ARO-AAT is designed to knock down the Alpha-1 antitrypsin (“AAT”) gene transcript and reduce the hepatic production of the mutant AAT protein.

• Study Name: Study to Check the Safety of Fazirsiran and Learn if Fazirsiran Can Help People With Liver Disease and Scarring (Fibrosis) Due to an Abnormal Version of Alpha-1 Antitrypsin Protein A Randomized, Double-blind, Placebo-Controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Fazirsiran in the Treatment of Alpha-1 Antitrypsin Deficiency-Associated Liver Disease With METAVIR Stage F2 to F4 Fibrosis ClinicalTrials.gov Identifier: NCT05677971

• Study Name: An Extension Study to Learn About the Long-Term Safety of Fazirsiran and if F