Arrowhead Pharmaceuticals (NASDAQ:ARWR) executives said the company is entering a key period marked by new regulatory approvals, early commercial progress for REDEMPLO and upcoming Phase 3 data in severe hypertriglyceridemia, during a fireside chat at RBC Capital Markets’ 2026 Global Healthcare Conference.
Vince Anzalone, Arrowhead’s vice president of investor relations, said the company is “on the most solid footing” in its history, citing regulatory progress for REDEMPLO and a strengthened balance sheet. He said the product, approved by the U.S. Food and Drug Administration in November of last year, has since received approval in China, Australia and Canada, as well as a positive CHMP opinion in Europe. Arrowhead expects European Commission approval shortly, he said.
Anzalone also pointed to the company’s first full quarter of commercial sales for REDEMPLO, which he characterized as encouraging. He said dynamics in the market for FCS, the rare portion of severe hypertriglyceridemia, are favorable for both Arrowhead and its competitor.
SHASTA-3 and SHASTA-4 Readout Expected in Q3
The most closely watched near-term catalyst discussed at the conference was the expected readout from SHASTA-3 and SHASTA-4, Phase 3 studies evaluating Arrowhead’s therapy in severe hypertriglyceridemia. Anzalone said the company expects to complete the studies toward the end of June, with data expected in the third quarter.
James Hamilton, Arrowhead’s chief medical officer, said the company remains “cautiously optimistic” that the studies will show statistical significance on acute pancreatitis. He said Arrowhead is confident the studies will be sufficiently powered based on blinded event rates.
Hamilton also discussed a change in how acute pancreatitis events are adjudicated. Arrowhead moved from the strict Atlanta criteria used in the PALISADE study to a modified approach that includes definite, probable and possible acute pancreatitis events. He said the change was made before any acute pancreatitis events were adjudicated and does not alter the statistical analysis.
The modified scale, Hamilton said, has been accepted by regulators in the U.S. and Europe, key opinion leaders and major journals. He added that it is the same scale used by Arrowhead’s competitor, which may allow for more direct comparison.
Anzalone said that after the last patient’s last visit, Arrowhead will need to lock the database and conduct the analysis, a process he described as typically taking four to eight weeks.
SHASTA-5 Positioned as Additional Support
Arrowhead also discussed SHASTA-5, a study designed specifically to evaluate acute pancreatitis events as the primary endpoint in high-risk patients. Hamilton described the trial as a “belt and suspenders” approach that could help with payers, particularly because it is focused on acute pancreatitis.
Hamilton said enrollment has picked up after the company broadened entry criteria. Initially, patients needed stricter triglyceride and acute pancreatitis history requirements. The revised criteria include at least one acute pancreatitis event within the past five years, a history of triglycerides above 880 and screening triglycerides above 500. Hamilton said the broader criteria should help identify more eligible patients without losing the ability to capture events.
Hamilton said data from SHASTA-5 could arrive in late 2027 or early 2028, though he cautioned that timing is difficult to predict because the trial is event-driven. Anzalone emphasized that SHASTA-5 is not required for submission for approval in severe hypertriglyceridemia, either in the U.S. or abroad.
Safety Signals and Commercial Opportunity
Executives addressed investor questions about liver fat and hemoglobin A1c signals seen in the broader APOC3 drug class. Hamilton said Arrowhead has not yet seen unblinded liver fat data from SHASTA-3 and SHASTA-4. He said the company’s hypothesis is that liver fat increases observed by a competitor may be more related to the competitor’s antisense oligonucleotide than to the target itself, though he did not rule out an on-target mechanism.
Hamilton said Arrowhead did not see an increase in liver fat at the 25-milligram dose being used as the go-to-market dose in pivotal studies. At a 50-milligram dose in an earlier study, he said there was about a 2% absolute increase in liver fat. Anzalone said the 25-milligram dose was selected because it appeared maximally active, not due to safety concerns.
On hemoglobin A1c, Hamilton said the company has seen an approximately 0.25% absolute increase, mostly in patients who were diabetic at baseline. He said the issue is generally manageable through adjustments to diabetes medications.
Anzalone said Arrowhead continues to view REDEMPLO as a $3 billion to $4 billion peak revenue opportunity. He said discussions with physicians and payers have reinforced the company’s belief that the APOC3 class is viewed as valuable, adding that some sell-side analysts now estimate the overall class could reach $9 billion to $10 billion annually.
Pipeline Focus Remains Cardiometabolic
Beyond REDEMPLO, Anzalone said Arrowhead has about $1.8 billion in cash and is positioned to support a broad development agenda. He highlighted zodasiran as the company’s hoped-for second approved product and said Arrowhead expects a readout later this year for a dual-function molecule designed to silence PCSK9 and APOC3 for mixed hyperlipidemia.
Hamilton also discussed Arrowhead’s ARO-INHBE program, noting that monotherapy weight loss has been modest, while reductions in liver fat and visceral fat have been more notable. He said the strongest weight-loss signal appeared in combination with tirzepatide in patients with type 2 diabetes, a group that can be harder to treat with GLP-1 therapies alone. Hamilton said Arrowhead is still evaluating regulatory endpoints for combination therapy in weight loss, including whether visceral fat reduction could support a path forward.
Asked about Biogen’s recent tau data, Hamilton said the results were generally supportive of the tau hypothesis, particularly because Biogen is moving the molecule into Phase 3. He said Arrowhead’s own approach uses siRNA-mediated knockdown and subcutaneous dosing rather than intrathecal administration, which he described as a potential advantage.
Anzalone said Arrowhead’s capital allocation will be increasingly weighted toward cardiometabolic programs, while the company may be more aggressive in partnering non-core assets. He said the company has more than 20 drugs in clinical studies and recently completed a deal with Madrigal on a MASH asset targeting PNPLA3.
About Arrowhead Pharmaceuticals (NASDAQ:ARWR)
Arrowhead Pharmaceuticals, Inc is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of RNA interference (RNAi) therapeutics. Since its founding in 2008, Arrowhead has leveraged its proprietary delivery platform—known internally as the Advanced RNAi Compound (ARC) technology—to silence disease-causing genes in patients suffering from genetically defined diseases. The company's approach aims to offer durable, targeted treatments across a range of therapeutic areas.
The company's pipeline includes multiple candidates in various stages of development.
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