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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) 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.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) is set to participate in major healthcare events in May 2021. Key dates include the RBC Capital Markets Global Healthcare from May 18-20, with a presentation by CEO Chris Anzalone on May 18 at 11:30 a.m. ET, and the UBS Global Healthcare Virtual Conference from May 24-26, featuring another chat by Anzalone on May 26 at 3:00 p.m. ET. For presentation materials and details, visit the Arrowhead website.
Arrowhead Pharmaceuticals (ARWR) reported its fiscal second-quarter financials for the period ending March 31, 2021. The company achieved a revenue of $32.8 million, up from $23.5 million in the previous year, resulting in a net loss of $26.8 million compared to $19.8 million in the same quarter last year. Operating expenses increased to $61 million, driven by research and development costs. Significant clinical updates include positive interim results for the ARO-AAT therapy and submissions for INDs for ARO-ANG3 and ARO-APOC3 treatments.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) has announced promising interim results from its AROAAT2002 Phase 2 clinical study of ARO-AAT, an RNAi therapeutic for alpha-1 antitrypsin deficiency (AATD). After 48 weeks, 80% of patients showed significant improvement in fibrosis, while total intra-hepatic Z-AAT decreased by 77-97%. Following 24 weeks of treatment, two patients also improved. Arrowhead plans to share further data at a medical congress and is pursuing accelerated approval pathways. The collaboration with Takeda continues to deliver encouraging outcomes.
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