Arrowhead Pharma Stock Price, News & Analysis

Arrowhead Pharmaceuticals (NASDAQ: ARWR) presented preclinical data on ARO-ALK7, its investigational RNAi therapeutic targeting ALK7 for obesity treatment, at the Keystone Symposia on Obesity and Adipose Tissue in February 2025. The company received regulatory clearance to begin a Phase 1/2a clinical trial in New Zealand, with dosing expected in Q2 2025.

Preclinical studies showed dose-dependent reductions in ALK7 mRNA in adipose tissue, with approximately 80% knockdown at 0.3 mg/kg and 91% at 1.5 mg/kg in non-human primates. In diet-induced obese mice, ARO-ALK7 suppressed body weight gain by 40% and reduced fat mass by approximately 50% while preserving lean mass.

Notably, when combined with tirzepatide (a GLP-1/GIP receptor co-agonist), ARO-ALK7 showed additive effects on weight and fat loss while ameliorating tirzepatide's lean mass reduction. The fat loss mechanism involves increased energy expenditure and lipolysis without affecting food intake. Toxicology studies in rats showed the treatment was generally well-tolerated.

Arrowhead Pharmaceuticals (NASDAQ: ARWR) reported its fiscal 2025 first quarter results, highlighting significant developments. The company secured a major licensing agreement with Sarepta Therapeutics, receiving $825 million upfront ($500M cash, $325M equity investment) plus $250 million over five years, with potential additional payments of $300 million and approximately $10 billion in milestones.

The FDA accepted Arrowhead's NDA for plozasiran for familial chylomicronemia syndrome treatment, with a PDUFA date of November 18, 2025. Phase 3 PALISADE study showed positive results with significant reductions in triglycerides.

Financial results showed revenue of $2.5 million, down from $3.55 million year-over-year. Net loss widened to $173.1 million ($1.39 per share) compared to $132.9 million ($1.24 per share) in the previous year. Total cash resources stood at $552.9 million as of December 31, 2024.

Arrowhead Pharmaceuticals (NASDAQ: ARWR) has completed its global licensing and collaboration agreement with Sarepta Therapeutics (NASDAQ: SRPT). The deal includes a $500 million upfront payment and $325 million through Sarepta's purchase of Arrowhead stock at $27.25 per share. Additionally, Arrowhead will receive $250 million in annual $50 million installments over 5 years.

The agreement covers multiple clinical-stage programs including ARO-DUX4 for facioscapulohumeral muscular dystrophy, ARO-DM1 for myotonic dystrophy, ARO-MMP7 for idiopathic pulmonary fibrosis, and ARO-ATXN2 for spinocerebellar ataxia 2. Three preclinical programs are also included. Arrowhead can earn up to $300 million in near-term payments for ARO-DM1 study enrollment, plus development milestones of $110-410 million and sales milestones of $500-700 million per program, along with tiered royalties up to low double digits.

Arrowhead Pharmaceuticals (NASDAQ: ARWR) has announced a webcast and conference call scheduled for February 10, 2025, at 4:30 p.m. ET. During this event, the company will discuss its financial results for the fiscal 2025 first quarter ended December 31, 2024.

Investors can access the live audio webcast through the Events and Presentations page in the Investors section of Arrowhead's website. A replay will be available approximately two hours after the call concludes. Analysts wishing to participate in the conference call must register through the provided link to receive dial-in information and a personalized PIN code.

Arrowhead Pharmaceuticals (NASDAQ: ARWR) announced FDA acceptance of its New Drug Application (NDA) for plozasiran, targeting familial chylomicronemia syndrome (FCS), a severe and rare genetic disease. The FDA set a PDUFA date of November 18, 2025, with no advisory committee meeting currently planned.

The NDA is supported by positive Phase 3 PALISADE study results, which met its primary endpoint and key secondary endpoints. The study demonstrated that plozasiran achieved an 80% median reduction in triglycerides at the 25mg dose and an 83% reduction in acute pancreatitis risk compared to placebo. The most common side effects included abdominal pain, COVID-19, nasopharyngitis, and nausea.

Arrowhead plans to submit additional regulatory applications for plozasiran FCS treatment in 2025 and is preparing for commercial launch pending FDA approval.

Arrowhead Pharmaceuticals (NASDAQ: ARWR) has initiated a Phase 1/2a clinical trial for ARO-INHBE, its RNAi therapeutic candidate for obesity treatment. The company has dosed the first subjects and recently filed for regulatory clearance of a second obesity candidate, ARO-ALK7. Both therapeutics target a pathway that signals fat storage in adipose tissue.

Preclinical studies of ARO-INHBE showed promising results in reducing body weight and fat mass, with a potential advantage of better lean muscle mass preservation compared to existing obesity treatments. The Phase 1/2 study will evaluate ARO-INHBE both as a monotherapy in part 1 and in combination with tirzepatide in part 2, targeting patients with obesity.

Arrowhead Pharmaceuticals (NASDAQ: ARWR) announced the approval of inducement grants for 20 new employees by its Board of Directors on December 13, 2024. The grants, approved under NASDAQ Listing Rule 5635(c)(4), collectively provide up to 44,350 restricted stock units. These grants are separate from the company's stockholder-approved equity incentive plans and will vest annually over a four-year period.

Arrowhead Pharmaceuticals (NASDAQ: ARWR) announced interim results from their Phase 1/2a clinical study of ARO-CFB, an RNAi therapeutic targeting complement factor B for complement mediated diseases. The data, presented at the 8th Complement-Based Drug Development Summit, showed significant efficacy with:

- Up to 90% reduction in circulating CFB protein with over 3 months duration
- Near complete inhibition of alternative pathway activity (100% mean reduction) at 200mg and 400mg doses
- Generally well-tolerated safety profile with mostly mild adverse events

The company plans to complete Part 1 of the study and proceed to Part 2, focusing on patients with immunoglobulin A nephropathy, the most common glomerular disease worldwide.

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.