Arrowhead reports positive ARO-C3 study results for IgAN

PASADENA, Calif. - Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR), a $2.14 billion market cap biotechnology company currently trading near its 52-week low of $16.57, has announced promising results from a clinical trial of its investigational drug ARO-C3, aimed at treating IgA nephropathy (IgAN), a kidney disease. The Phase 1/2 study demonstrated significant reductions in key markers of disease activity and proteinuria, a sign of kidney damage. According to InvestingPro data, analysts anticipate sales growth in the current year, with five analysts recently revising their earnings estimates upward.

Patients in the trial received three subcutaneous doses of ARO-C3 and were monitored through Day 169. The treatment led to a mean sustained reduction of complement component 3 (C3) by at least 87%, with a maximum mean reduction of 89%. Complement activity, measured by serum AH50 and Wieslab AP, showed mean sustained reductions of more than 76% and 89%, respectively. While the company is quickly burning through cash, InvestingPro analysis shows its liquid assets exceed short-term obligations with a healthy current ratio of 6.09.

Additionally, the study reported a mean reduction of 41% in the spot urine protein-to-creatinine ratio (UPCR) by week 24, indicating an improvement in a key indicator of kidney health. Importantly, ARO-C3 was generally well-tolerated by patients, with no serious treatment-emergent adverse events or discontinuations due to side effects. The most common adverse events were headache, cough, and nasopharyngitis.

James Hamilton, M.D., MBA, Chief Medical Officer and Head of R&D at Arrowhead, highlighted the potential of ARO-C3, noting the "durable and near complete inhibition of the alternative complement pathway" and the improvement in proteinuria. He indicated that further data from the study is expected to be shared at a medical meeting later in 2025.

ARO-C3 is designed to reduce the production of C3 by the liver, which could potentially treat various complement-mediated renal diseases. By silencing C3, the drug aims to modulate the activation of the complement cascade, a part of the immune system that can contribute to tissue injury and disease progression when dysregulated.

The AROC3-1001 Phase 1/2a study is a first-in-human trial assessing the safety, tolerability, pharmacokinetics, and pharmacodynamics of ARO-C3 in healthy volunteers and patients with complement-mediated renal disease.

This news article is based on a press release statement from Arrowhead Pharmaceuticals, Inc. Looking ahead, analysts have set price targets ranging from $21 to $80, reflecting mixed opinions about the company’s prospects. For deeper insights into Arrowhead’s financial health and growth potential, investors can access comprehensive analysis and 8 additional ProTips through InvestingPro’s detailed research reports, available for over 1,400 US stocks.

In other recent news, Arrowhead Pharmaceuticals has presented promising preclinical data for its investigational RNAi-based therapeutics, ARO-INHBE and ARO-ALK7, aimed at treating obesity and metabolic diseases. These therapies, now progressing into clinical trials, have shown significant reductions in fat mass in preclinical studies. Arrowhead’s strategic focus was further sharpened by the completion of a transaction with Sarepta Therapeutics, bringing in approximately $1.4 billion in upfront and near-term payments, which will support the company’s cardiometabolic programs. RBC Capital Markets has maintained an Outperform rating on Arrowhead, with a price target of $42, citing the company’s strengthened financial position and focused pipeline. Meanwhile, Chardan Capital Markets has reiterated a Buy rating with a $60 price target, reflecting confidence in Arrowhead’s clinical trials for plozasiran, a treatment for severe hypertriglyceridemia. BofA Securities has adjusted its price target for Arrowhead to $41, maintaining a Buy rating, following the company’s latest earnings report and strategic pivot to prioritize zodasiran in its cardiometabolic portfolio. Additionally, Arrowhead has expanded its support for the Familial Chylomicronemia Syndrome (FCS) community by launching a new website and white paper, providing educational resources and patient support. These developments highlight Arrowhead’s continued commitment to advancing RNAi-based therapies and addressing unmet medical needs.

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