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Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) is a leading clinical-stage biotechnology company dedicated to developing innovative medicines that address intractable diseases through gene silencing. The company utilizes an extensive array of RNA chemistries and efficient delivery systems to harness the power of RNA interference (RNAi) mechanisms. This approach allows for the rapid, deep, and durable knockdown of target genes, thereby reducing the production of specific proteins that cause diseases.
Arrowhead's groundbreaking therapies are at the forefront of genetic-based treatments, presenting potential life-changing solutions for patients. Notable among their technologies is the Targeted RNAi Molecule (TRiMTM) platform. This platform employs ligand-mediated delivery for tissue-specific targeting while maintaining structural simplicity. The TRiMTM platform is built upon over a decade of research in actively targeted drug delivery, offering advantages such as simplified manufacturing processes and enhanced specificity.
The company's diverse portfolio includes promising treatments for conditions such as Hepatitis B, Alpha-1 Antitrypsin Deficiency, thrombosis and angioedema, clear-cell and renal cell carcinoma, and cardiovascular diseases. Arrowhead is continuously engaged in advancing its clinical programs and forging strategic partnerships to bring its novel therapies to market.
With headquarters in Pasadena, California, Arrowhead Pharmaceuticals remains committed to transforming the landscape of genetic medicine and improving patient outcomes worldwide. Their dedication to scientific innovation and patient care positions them as a pivotal player in the biotechnology sector.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) has announced a voluntary pause of the AROENaC1001 clinical study, a Phase 1/2 trial for its RNA interference therapy targeting cystic fibrosis. This decision follows preliminary findings from chronic toxicology studies indicating unexpected local lung inflammation in rats. The company has halted new patient screenings and further dosing while waiting for comprehensive data from ongoing toxicology studies. Despite this setback, Arrowhead remains focused on advancing its other eight clinical candidates and expanding its preclinical initiatives.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) has initiated dosing for the first patient in its Phase 2b clinical trial, AROANG3-2001, aimed at evaluating the safety and efficacy of ARO-ANG3 for treating mixed dyslipidemia. The study will enroll about 180 participants and assess three dose levels (50 mg, 100 mg, and 200 mg) against a placebo. ARO-ANG3 targets the inhibition of ANGPTL3 to reduce triglycerides and LDL cholesterol, potentially addressing cardiovascular risks in patients with mixed dyslipidemia.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) announced positive interim results from the AROAAT2002 study, showcasing 48-week liver biopsy outcomes for the investigational RNAi therapeutic ARO-AAT, developed in collaboration with Takeda. The study observed significant reductions in mutant AAT protein levels, with a median decrease of 80.1% in total Z-AAT. Patients also showed improvements in liver fibrosis, with 6 out of 9 achieving stage improvements. ARO-AAT was well tolerated, indicating a promising therapeutic option for patients suffering from alpha-1 antitrypsin deficiency.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) presented promising preclinical data for ARO-DUX4, an RNA interference therapeutic targeting facioscapulohumeral muscular dystrophy (FSHD) at the 28th Annual FSHD Society International Research Congress. The company plans to seek regulatory approval in Q3 2021 to commence clinical trials. ARO-DUX4 demonstrated deep knockdown of DUX4 in mouse models, preventing muscle degeneration and related symptoms. The therapy addresses a significant unmet need in FSHD, where no effective treatments exist.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) announced positive interim results from the Phase 1/2 study of ARO-HSD, an RNA interference therapeutic targeting liver diseases, including nonalcoholic steatohepatitis (NASH). The study showed a mean 84% reduction in hepatic HSD17B13 mRNA and an average 46% decrease in alanine aminotransferase (ALT) levels after just two doses. The treatment was well-tolerated, with no significant safety concerns reported. These findings position ARO-HSD as a promising candidate for later-stage clinical studies, reinforcing HSD17B13's role as a therapeutic target.
Arrowhead Pharmaceuticals and Horizon Therapeutics have entered into a global collaboration for ARO-XDH, an investigational RNAi therapeutic for uncontrolled gout. Arrowhead will handle preclinical development, while Horizon will manage clinical development and commercialization. Arrowhead will receive $40 million upfront, with potential total milestone payments of up to $660 million plus royalties in the low- to mid-teens range on product sales. This partnership aims to address the unmet treatment needs in gout, affecting over nine million patients in the U.S.
Arrowhead Pharmaceuticals and Horizon Therapeutics have formed a global collaboration for ARO-XDH, an investigational RNA interference therapy aimed at treating uncontrolled gout. Under the agreement, Arrowhead will manage preclinical development, while Horizon will handle clinical development and commercialization. Arrowhead will receive an upfront payment of $40 million and could earn up to $660 million in milestones, plus royalties on sales. The partnership aims to address the significant unmet treatment needs in gout, affecting over nine million patients in the U.S.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) has commenced patient dosing in the Phase 2b trial AROAPOC3-2001 to evaluate ARO-APOC3, aimed at treating severe hypertriglyceridemia (SHTG). The study will involve around 300 participants with triglyceride levels ≥ 500 mg/dL. Initial Phase 1 results suggested ARO-APOC3 could reduce triglyceride levels by up to 92%, maintaining efficacy for over 12 weeks. This trial seeks to determine the optimal dosing for future studies.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) has announced its participation in several key upcoming events in June 2021. Highlights include:
- Jefferies Virtual Healthcare Conference: June 3, 2:30 p.m. ET - Fireside chat with CEO Chris Anzalone.
- Goldman Sachs Global Healthcare Conference: June 10, 2:10 p.m. ET - Fireside chat with CEO Chris Anzalone.
- The International Liver Congress 2021: June 23-26 - Presentations on ARO-HSD and ARO-AAT, among others.
For more details, visit Arrowhead's website.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) reported that Janssen Pharmaceuticals has exercised its option for ARO-JNJ1, leading to a $10 million option exercise fee for Arrowhead. This agreement, part of a collaboration initiated in 2018, allows Janssen to develop and commercialize ARO-JNJ1, an RNAi therapeutic candidate. Arrowhead anticipates additional milestone payments and royalties from the commercial sales of ARO-JNJ1. The CEO expressed optimism about the collaboration, highlighting its productivity in addressing chronic hepatitis B and potential future programs.