Arrowhead Pharmaceuticals develops and commercializes RNA interference medicines that silence genes associated with intractable diseases. The company’s TRiM™ platform is used to design targeted RNAi therapeutics for tissues including liver, lung, muscle, adipose and central nervous system tissue.
Recurring ARWR news covers REDEMPLO® (plozasiran), an siRNA medicine approved in the United States and Australia as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome. Company updates also address plozasiran data across hypertriglyceridemia, regulatory actions in additional geographies, pricing and market-access strategy, fiscal results, investor events, and clinical or partnering developments involving programs such as ARO-PNPLA3, ARO-INHBE and ARO-ALK7.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) has filed for regulatory clearance to begin a Phase 1/2a clinical trial of ARO-ALK7, its second RNAi therapeutic for obesity treatment. The trial, AROALK7-1001, will evaluate safety and efficacy in up to 90 obese adults. The study includes two parts: monotherapy assessment and combination therapy with tirzepatide. ARO-ALK7 targets the ACVR1C gene in adipose tissue, aiming to reduce ALK7 production. Preclinical studies showed reduced body weight and fat mass while preserving lean muscle. The company now has two clinical-stage RNAi candidates for obesity treatment, demonstrating its leadership in delivering siRNA to multiple tissues using its TRiM™ platform.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) has announced its participation in multiple scientific conferences during December 2024. The company will present at the Piper Sandler Healthcare Conference (Dec 3) and Evercore ISI HealthCONx Conference (Dec 4). Notable presentations include the PALISADE Phase 3 study results of plozasiran for Familial Chylomicronemia Syndrome at several events, including the APA/JPS/CAP/IAP Joint Meeting and WCIRDC.
The company will also present research on siRNA for neurodegenerative disorders and complement-targeted therapies at the RNA at the Bench and Bedside IV and Complement-Based Drug Development Summit respectively.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) reported its fiscal year 2024 results, highlighting significant financial and R&D developments. The company secured major financing through a $825 million licensing agreement with Sarepta Therapeutics and a $500 million credit facility with Sixth Street, extending cash runway into 2028. Key achievements include submitting their first NDA for plozasiran and receiving Breakthrough Therapy designation from FDA. However, financial results showed a significant increase in net loss to $599.5 million compared to $205.3 million in 2023, with revenue declining to $3.6 million from $240.7 million previous year.
Sarepta Therapeutics announces a global licensing and collaboration agreement with Arrowhead Pharmaceuticals for multiple siRNA programs. Sarepta will obtain exclusive worldwide rights to four clinical-stage and three preclinical-stage programs targeting muscle, CNS, and rare pulmonary disorders. The deal includes a $500 million upfront payment and $325 million equity investment at a 35% premium to Arrowhead, plus future milestone payments and royalties. Additionally, both companies entered a discovery partnership for six targets. Sarepta's Board has approved a $500 million share repurchase program. The transaction is expected to close in early 2025.
Arrowhead Pharmaceuticals announced a global licensing and collaboration agreement with Sarepta Therapeutics. The deal includes an immediate $825 million payment ($500M cash + $325M equity investment at 35% premium), plus $250 million over five years. Arrowhead is eligible for $300 million in near-term clinical trial milestone payments and up to $10 billion in future milestones plus royalties.
The agreement covers multiple clinical and preclinical programs in rare genetic diseases affecting muscle, CNS, and lungs. Sarepta can select up to six new targets using Arrowhead's TRiM™ platform. The transaction extends Arrowhead's cash runway into 2028 and is expected to close in early 2025.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) has submitted a New Drug Application (NDA) to the FDA for plozasiran, targeting familial chylomicronemia syndrome (FCS), a severe genetic disease with no current FDA-approved treatments. The submission is based on positive Phase 3 PALISADE study results, which showed an 80% median reduction in triglycerides and an 83% reduction in acute pancreatitis risk compared to placebo. The company plans to submit additional regulatory applications in 2025. The drug demonstrated promising efficacy in treating FCS, a condition characterized by extremely high triglyceride levels and increased risk of acute pancreatitis.
Arrowhead Pharmaceuticals presented new data from Phase 3 PALISADE study and open-label extension (OLE) of MUIR and SHASTA-2 studies for plozasiran at AHA24. The PALISADE study showed plozasiran induced significant triglyceride reductions of approximately -80% at 25mg dose, with similar efficacy in both genetically confirmed and clinically diagnosed FCS patients. In the OLE studies, extended treatment with 25mg plozasiran demonstrated mean triglyceride reductions up to -73% in MUIR and -86% in SHASTA-2 patients through 15 months. The drug was generally well-tolerated across studies.
Arrowhead Pharmaceuticals (NASDAQ:ARWR) has announced it will host a webcast and conference call on November 26, 2024, at 4:30 p.m. ET to discuss its financial results for the 2024 fiscal year ended September 30, 2024. Investors can access the live audio webcast through the Events and Presentations page on the company's website. A replay will be available approximately two hours after the call concludes. Analysts wishing to participate must register through the provided link to receive dial-in details and a personalized PIN code.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) has announced its participation in several major healthcare conferences in November 2024. The company will present research on multiple drug candidates including Plozasiran at the American Conference on Pharmacometrics and American Heart Association Scientific Sessions. Key presentations focus on treatments for hypertriglyceridemia and familial chylomicronemia syndrome.
The company will also showcase research on Fazirsiran for alpha-1 antitrypsin deficiency-associated liver disease at The Liver Meeting 2024. Additional presentations are scheduled at the UBS Global Healthcare Conference, TIDES Europe, and Jefferies London Healthcare Conference, covering various aspects of their therapeutic pipeline and platform technologies.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) launched 'We'll Get There Soon,' a disease awareness campaign for Familial Chylomicronemia Syndrome (FCS) on FCS Awareness Day. The initiative includes a website, www.LowerTriglycerides.com, and social media presence to support the FCS community. The campaign features Julie Klueckman's story, highlighting the challenges of living with FCS, including a decade-long diagnosis journey, multiple hospitalizations, and the need to maintain an extremely low-fat diet of up to 20 grams daily. The campaign aims to educate about maintaining triglyceride levels below 500 mg/dL to reduce acute pancreatitis risk and bring hope to those affected by this rare condition.