On May 2, 2023 Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) reported financial results for its fiscal second quarter ended March 31, 2023 (Press release, Arrowhead Research Corporation, MAY 2, 2023, View Source [SID1234630824]). The company is hosting a conference call today, May 2, 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

•Reported interim results from an ongoing Phase 1/2 clinical study of ARO-RAGE, an investigational RNAi therapeutic for treatment for inflammatory pulmonary diseases, such as asthma. These data represent the first clinical demonstration of the potential utility of Arrowhead’s proprietary Targeted RNAi Molecule (TRiMTM) platform optimized for delivery to the lungs. Key results from Part 1 of the study in normal healthy volunteers included the following:
oMean maximum reduction in soluble RAGE (sRAGE) as measured in serum after two doses of 92 mg on Day 1 and Day 29 was 80% with a maximum reduction of 90%
Duration of pharmacologic effect persisted for at least 6 weeks
oMean reduction in sRAGE as measured in bronchoalveolar lavage fluid (BALF) at Day 31 after a single dose of 92 mg was 75% with a maximum reduction of 92%

o Mean maximum reduction of 56% with a maximum reduction of 68% in serum sRAGE was also observed after a single dose of 92 mg
o Safety and tolerability

Overall, no patterns of adverse changes in any clinical safety parameters
No reported serious or severe adverse events
No dropouts related to drug or related to adverse events

oThese results include 4 of 5 escalating dose levels. Data are not yet available for single or multiple dose cohorts at 184 mg, the highest dose being tested
•Announced a planned R&D Day on June 1, 2023, which will include presentations on cardiometabolic, pulmonary, and newly announced central nervous system (CNS) pipeline programs
oThe company expanded its TRiMTM platform to include an optimized intrathecal administration for CNS delivery with good distribution throughout the brain and in all relevant brain cell types. The first development candidate to utilize this new delivery platform, ARO-SOD1, is on track for a clinical trial application filing in the third quarter of 2023 to begin clinical studies
oIn preclinical studies, ARO-SOD1 achieved 95% spinal cord tissue mRNA knockdown after a single intrathecal dose in human SOD1 transgenic rats and maintained greater than 80% spinal cord tissue mRNA knockdown three months after a single intrathecal dose in non-human primates
•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 (TAK-999/ARO-AAT) for the treatment of alpha-1 antitrypsin deficiency associated liver disease (AATD-LD)
•Received Fast Track designation from the U.S. Food and Drug Administration (FDA) for ARO-APOC3 for reducing triglycerides in adult patients with familial chylomicronemia syndrome
•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)

•Initiated dosing in a Phase 1/2a single ascending dose and multiple ascending dose clinical study of ARO-MMP7, Arrowhead’s third pulmonary targeted investigational RNAi therapeutic being developed as a potential treatment for idiopathic pulmonary fibrosis
•Reported interim results from an ongoing Phase 1/2 clinical study of ARO-C3, an investigational RNAi therapeutic for treatment of various complement mediated diseases, demonstrating mean reductions of 88% in C3 and 91% in AH50, a marker of alternative complement pathway hemolytic activity
•Regained rights to ARO-PNPLA3, formerly called JNJ-75220795, which was part of a 2018 research collaboration and option agreement between Arrowhead and Janssen Pharmaceuticals, Inc. ARO-PNPLA3 is an investigational RNAi therapeutic developed using Arrowhead’s proprietary TRiMTM platform and designed to reduce liver expression of patatin-like phospholipase domain containing 3 (PNPLA3) as a potential treatment for patients with NASH
•Announced topline results with Takeda from the Phase 2 SEQUOIA clinical study of investigational fazirsiran for the treatment of AATD-LD and provided an outline of a Phase 3 study that was co-developed by Takeda and Arrowhead and will be conducted by Takeda. Key results from SEQUOIA included the following:

oFibrosis regression observed in 50% of patients receiving fazirsiran

oMedian reductions of 94% of Z-AAT accumulation in the liver and mean reduction of 68% in histologic globule burden

oTreatment emergent adverse events were generally well balanced between fazirsiran and placebo groups
oResults consistent with AROAAT-2002 open-label study previously published in The New England Journal of Medicine