On August 7, 2023 Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) reported financial results for its fiscal third quarter ended June 30, 2023 (Press release, Arrowhead Research Corporation, AUG 7, 2023, View Source [SID1234633862]). The company is hosting a conference call today, Aug. 7, 2023, at 4:30 p.m. ET to discuss the results.
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Webcast and Conference Call and Details
Investors may access a live audio webcast on the Company’s website at View Source A replay of the webcast will be available approximately two hours after the conclusion of the call.
For analysts that wish to participate in the conference call, please register at https://register.vevent.com/register/BIf9cd65655e5444119df91cd4891157f8. Once registered, you will receive the dial-in number and a personalized PIN code that will be required to access the call.
Selected Recent Events
•Hosted a Research & Development Day to discuss progress towards the company’s "20 in 25" goal to grow its pipeline of RNAi therapeutics that leverage the proprietary Targeted RNAi Molecule (TRiMTM) platform to a total of 20 clinical stage or marketed products in the year 2025. Key updates included:
•New Phase 1 clinical data on ARO-RAGE showing a continued dose response in healthy volunteers with a single inhaled dose of 184 mg achieving mean knockdown of 90% and a maximum of 95% in bronchoalveolar lavage fluid (BALF)
•Serum sRAGE reductions in asthma patients consistent with effects seen in healthy volunteers at the 44 mg dose, the only dose level with results currently available in asthma patient cohorts
•TRiMTM platform for intrathecal administration for central nervous system (CNS) delivery demonstrates 90-95% dose-dependent mRNA knockdown in disease-relevant spinal cord and cortex brain regions in non-human primates
•TRiMTM platform for adipose tissue delivery achieved up to 98% knockdown and maintained greater than 85% knockdown over 31 weeks
•TRiMTM dimer platform for delivery to hepatocytes achieved equivalent or better knockdown of two separate target genes with longer duration than monomer mixture
•Completed enrollment of the global PALISADE Phase 3 clinical trial evaluating ARO-APOC3 for the treatment of familial chylomicronemia syndrome. The company anticipates that the primary portion of the study will be complete in the second quarter of 2024 with a data readout shortly thereafter
•Presented interim data from the ongoing Phase 2 GATEWAY clinical study of ARO-ANG3 in patients with homozygous familial hypercholesterolemia at the 91st European Atherosclerosis Society Congress showing that ARO-ANG3 achieved 44-48% mean reductions in LDL-C on top of continued standard of care
•Presented updated results from the Phase 2 SEQUOIA clinical study of investigational fazirsiran (TAK-999/ARO-AAT) for the treatment of liver disease associated with alpha-1 antitrypsin deficiency at the European Association for the Study of the Liver (EASL) Congress 2023
•Filed an application to initiate a Phase 1 clinical trial of ARO-SOD1, an RNAi-based investigational medicine designed to reduce expression of superoxide dismutase 1 (SOD1) in the CNS as a potential treatment for patients with amyotrophic lateral sclerosis (ALS) caused by SOD1 mutations. ARO-SOD1 is the first therapeutic candidate designed for delivery to the CNS to enter clinical studies that leverages the TRiMTM platform
•Filed an application to initiate a Phase 1/2 clinical trial of ARO-DUX4, the company’s investigational RNAi therapeutic being developed as a potential treatment for patients with facioscapulohumeral muscular dystrophy (FSHD). ARO-DUX4 is the first clinical candidate utilizing the TRiMTM platform to target disease associated genes in skeletal muscle
•Earned a $30 million milestone payment from GSK (LSE/NYSE: GSK) following the start of GSK’s Phase 2b trial of GSK4532990, formerly called ARO-HSD, an investigational RNAi therapeutic for the treatment of patients with non-alcoholic steatohepatitis (NASH)
•Earned a $40 million milestone payment from Takeda (TSE:4502/NYSE:TAK) after the first patient was dosed in the Phase 3 REDWOOD clinical study of fazirsiran