Arrowhead Pharmaceuticals (NASDAQ:ARWR) is moving into a more commercial phase while continuing to expand its RNA interference pipeline, Chief Executive Officer Chris Anzalone said during a presentation with BofA pharma and biotech analyst Jason Gerberry at the BofA Annual Healthcare Conference.
Anzalone described Arrowhead as an “and” company rather than an “or” company, saying the company intends to build its commercial capabilities without slowing its research operations. He said Arrowhead can now address seven different cell types, with five currently in clinical studies, and remains focused on RNAi innovation.
The company also expects several pipeline readouts later this year, including initial data from its dimer, or bispecific, platform and from its CNS platform.
REDEMPLO Launch Off to a Faster-Than-Expected Start
A major focus of the discussion was REDEMPLO, Arrowhead’s commercial launch in familial chylomicronemia syndrome, or FCS. Anzalone said the launch “has gone well so far,” noting that Arrowhead had recorded more than 400 prescriptions after one full quarter of sales. He said adoption has been “a bit more rapid” than the company expected.
Anzalone emphasized that FCS remains an education market. While some patients with genetic FCS are diagnosed, he said Arrowhead believes that group represents a small portion of the broader FCS population. The company is also focused on patients with clinical FCS, who do not have known genetic mutations associated with traditional FCS but still have very high triglycerides and a substantially increased risk of pancreatitis.
He said many patients currently categorized as having severe hypertriglyceridemia, or sHTG, may be more appropriately diagnosed as having clinical FCS. That overlap, he said, is informing Arrowhead’s approach as it looks toward a broader sHTG opportunity.
SHASTA Data Expected in Third Quarter
Arrowhead is awaiting pivotal data from the SHASTA-3 and SHASTA-4 studies in sHTG, with topline results expected after last patient, last visit near the end of June and subsequent database lock and analysis. Anzalone said the primary endpoint is triglyceride lowering and expressed confidence based on prior experience.
“This essentially works in 100% of people,” Anzalone said, referring to triglyceride reduction in the company’s FCS phase 3 study, where he said there were no non-responders. He contrasted that with what he described as about 20% non-responders for a competitor’s product.
The company is also watching whether the studies show a reduction in acute pancreatitis, a key secondary endpoint. Anzalone said Arrowhead was encouraged by pancreatitis data from competitor Ionis and is “cautiously optimistic” that SHASTA-3 and SHASTA-4 may show a benefit. However, he noted that the event numbers are relatively small and that “funny things can happen with small numbers.”
Arrowhead also has SHASTA-5, an ongoing event-driven study designed to show an improvement in pancreatitis. Anzalone said if SHASTA-3 and SHASTA-4 show a pancreatitis benefit, Arrowhead could decide to stop SHASTA-5, but no decision has been made. He said the timing for SHASTA-5 is difficult to predict because it is event-driven.
Anzalone said pancreatitis data would not be gating for U.S. payers, in Arrowhead’s view, but could matter more outside the United States, where some geographies may require acute pancreatitis data on the label for reimbursement.
Pricing and Market Opportunity
Gerberry asked about pricing dynamics in a two-player APOC3-modulating market that includes Ionis’ TRYNGOLZA. Anzalone said Arrowhead does not expect to aggressively discount REDEMPLO and views the product less as a triglyceride-lowering drug and more as a pancreatitis drug.
He said Arrowhead is comfortable with a list price at a slight premium to TRYNGOLZA, citing quarterly dosing, safety profile and historical triglyceride reduction. Anzalone said he believes the sHTG market can support two competitors and that having two companies educating physicians and payers may help the category develop more rapidly.
Asked about peak U.S. sales potential, Anzalone said he would be “a bit more aggressive” than a $3 billion peak estimate referenced by Gerberry, saying he sees a potential $3 billion to $4 billion peak for REDEMPLO. However, he cautioned that the market will take time to develop because of the need for physician education.
Obesity and Cardiometabolic Pipeline
Arrowhead also discussed ARO-INHBE, an obesity-related program the company is studying in combination with GLP-1 therapies. Anzalone said Arrowhead has long viewed INHBE as a potential combination therapy rather than a monotherapy.
He said early data in an obese diabetic population showed a doubling of fat reduction and a tripling of liver fat reduction, which he called “eye-opening.” Arrowhead is designing phase 2 studies and expects to begin them this year, with plans to study INHBE as a potential MASH therapy and obesity therapy.
Anzalone also highlighted ARO-ALK7, which targets the Activin E/ALK-7 axis. He said Arrowhead expects more data in the second half of this year and is interested in safety, target knockdown, potential weight loss effects and fat distribution. He said successful adipose delivery in humans could open additional targets and potential dimer approaches.
Arrowhead also expects data from its PCSK9/APOC3 dimer in the third quarter. Anzalone said the company believes the program could address roughly 20 million people in the U.S. with mixed hyperlipidemia.
CNS Platform and Capital Position
Anzalone said Arrowhead expects initial data from ARO-MAPT, its first subcutaneously administered CNS drug, later this year. The initial readout will be in healthy volunteers and will focus on whether animal results translate to humans, including tau knockdown and tolerability.
If the readout is positive, Anzalone said Arrowhead could move quickly into additional CNS programs, with multiple candidates potentially entering clinical studies in 2027 and possibly one by late 2026.
On partnering, Anzalone said Arrowhead reported about $1.8 billion in cash on its most recent quarterly update and is positioned to advance its current programs itself. He said the company currently has no interest in partnering plozasiran, zodasiran or the PCSK9/APOC3 dimer, though he acknowledged that partnership views could change in the future.
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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