Arrowhead Pharmaceuticals Q4 Earnings Call Highlights

Key Points

• REDEMPLO could reach a much larger U.S. market: Arrowhead’s supplemental application for severe hypertriglyceridemia is in an FDA pilot program with a six-month priority-review framework, and the company is preparing for a potential launch in Q2 2027. It estimates a 3 million to 3.5 million U.S. patient population and projects $3 billion to $4 billion in annual sales over time.
• Early demand is growing, but prescriptions must convert to paid treatment: New-to-brand prescriptions rose to 950 by fiscal year-end and have since increased by more than 300, while weekly prescriptions are running at roughly 50 to 60. Management identified reimbursement and prior authorization as key hurdles to conversion.
• Spending drove a wider fiscal 2026 loss: Arrowhead’s net loss widened to $724.2 million as operating expenses increased, including a one-time $215 million priority-review-voucher expense. The company ended the year with nearly $1.2 billion in cash and investments and refinanced its debt with a $400 million loan due in 2031.

Arrowhead Pharmaceuticals NASDAQ: ARWR is preparing for a potential U.S. launch of its drug REDEMPLO in a larger patient population in the second quarter of 2027, as the company works to turn growing prescription demand for the medicine into paid treatments.

On its fiscal 2026 earnings call, management said the company submitted a supplemental application on Nov. 2 seeking approval for REDEMPLO, also known as plozasiran, in severe hypertriglyceridemia, or SHTG. The Food and Drug Administration has accepted the application into a pilot program that provides a six-month priority review framework and the possibility of an earlier decision.

Arrowhead currently sells REDEMPLO for familial chylomicronemia syndrome, or FCS, a narrower market. The company reported approximately $6.6 million in net product revenue for the fiscal year ended Sept. 30, while its full-year net loss widened to $724.2 million from $1.6 million a year earlier.

Prescription growth and reimbursement

CEO Chris Anzalone said Arrowhead had received 950 cumulative new-to-brand patient prescriptions as of Sept. 30, up 63% from the prior quarter’s end. The company has since received more than 300 additional prescriptions, while the number of distinct prescribers has grown from more than 640 at year-end to more than 860.

Andy Davis, senior vice president and head of the global cardiometabolic franchise, said recent trends supported approximately 50 to 60 new prescriptions per week, compared with a previously cited rate of 20 to 30. More than 160 million people in the U.S. have coverage for eligible patients under applicable payer policies, he said.

Management cautioned that written prescriptions are not the same as paid claims. Patients and physicians must navigate benefit verification and prior authorization, among other steps, before prescriptions can become paid shipments. Anzalone described that conversion process as a “choke point.”

Davis said Arrowhead has a quick-start bridge program and a patient assistance program for FCS, allowing patients to begin treatment while coverage is being established. The company expects to have a similar quick-start program for SHTG if the drug is approved for that use.

Asked about a competitor’s softer-than-expected early SHTG launch, Anzalone said it had not changed Arrowhead’s expectations. He attributed some of the competitor’s experience to communication and commercial execution and said Arrowhead was already engaging payers ahead of a possible approval.

Arrowhead plans to track new-to-brand prescriptions, distinct prescribers and covered lives as early indicators of an SHTG launch. Anzalone said it was too early to provide financial guidance without an FDA decision, a final label and more experience with payers.

SHTG opportunity and clinical data

Arrowhead estimates that 3 million to 3.5 million people in the U.S. have triglyceride levels above 500 mg/dL. Its initial focus would be a higher-risk group of approximately 1 million people, including patients with very high triglycerides or a prior history of acute pancreatitis.

The company continues to project that REDEMPLO could generate $3 billion to $4 billion in annual U.S. sales over time in SHTG. Davis said Arrowhead intends to maintain its current U.S. wholesale acquisition cost of $45,000 per patient per year across FCS and SHTG, if the broader indication is approved.

In the SHASTA-3 and SHASTA-4 trials, median triglyceride reductions from baseline at month 12 were 79% and 81%, respectively, management said. A pooled analysis showed a 78% reduction in cumulative acute pancreatitis events versus placebo. Among patients with a prior history of acute pancreatitis, the event-rate reduction was 91%.

Chief Medical Officer James Hamilton said treatment-emergent adverse event rates were similar in the pooled active and placebo groups. Worsening glycemic control was reported more frequently with plozasiran, at 14.3%, versus 8.7% with placebo; Hamilton said there was no progressive worsening of average blood sugar levels over time. The FDA will determine the final label if it approves the application.

Loss widens as spending rises

Fiscal 2026 revenue fell to $450.8 million from $829.4 million a year earlier, when Arrowhead recognized substantial collaboration revenue tied largely to its Sarepta and Sanofi arrangements. The latest year included $317 million from the Sarepta collaboration and $89 million from Novartis.

Operating expenses rose to approximately $1.16 billion from $731 million. The increase included a one-time $215 million expense for a priority review voucher purchased in September. Chief Financial Officer Dan Apel said the voucher is no longer needed for the SHTG application following acceptance into the FDA pilot program and is eligible for sale, although Arrowhead has not entered into a sale agreement.

Cash and investments totaled nearly $1.2 billion at fiscal year-end, up from approximately $900 million a year earlier. On Nov. 2, Arrowhead refinanced its existing Sixth Street facility with a $400 million senior secured term loan due in 2031. Apel said the new loan carries a lower interest rate and eliminates required cash sweeps tied to product sales and partnership proceeds.

Pipeline milestones ahead

Arrowhead expects data in the second half of 2027 from its phase 3 YOSEMITE study of zodasiran in homozygous familial hypercholesterolemia. If results support it, the company has a goal of filing a new drug application by year-end 2027.

For ARO-DIMER-PA, an experimental therapy designed to silence both PCSK9 and APOC3, Hamilton said additional clinical data are expected around mid-2027. Arrowhead is considering an initial approval strategy based on LDL cholesterol lowering, subject to regulatory agreement.

The company also expects additional data in 2027 for ARO-MAPT, its experimental central nervous system drug. In an interim healthy-volunteer analysis, mean cerebrospinal fluid total tau fell 73% by day 105 under the regimen studied. Hamilton said Arrowhead is discussing studies in frontotemporal dementia and progressive supranuclear palsy, while gathering further data before advancing later-stage Alzheimer’s development.

About Arrowhead Pharmaceuticals (NASDAQ:ARWR)

Arrowhead Pharmaceuticals is a biotechnology company that develops medicines using RNA interference (RNAi), a biological process that can reduce the production of specific proteins by silencing the genes that encode them. Its proprietary Targeted RNAi Molecule (TRiM) platform is designed to deliver RNAi treatments to targeted tissues. The company's research and development programs address a range of conditions, including liver, cardiometabolic, pulmonary and neurological diseases. Its work centers on investigational medicines and drug-development partnerships.

This instant news alert was generated by narrative science technology and financial data from MarketBeat in order to provide readers with the fastest reporting and unbiased coverage. Please send any questions or comments about this story to contact@marketbeat.com.