Arrowhead Pharmaceuticals : ARWR FQ2 2025 Script FINAL

Published: 2025-05-12 17:08:08 ET ARWR

Published on 05/12/2025 at 17:08

Operator

Ladies and gentlemen, welcome to the Arrowhead Pharmaceuticals conference call. Throughout today's recorded presentation all participants will be in a listen-only mode. After the presentation, there will be an opportunity to ask questions. I will now hand the conference call over to Vince Anzalone, Vice President of Investor Relations for Arrowhead. Please go-ahead Vince.

Vince Anzalone

Good afternoon and thank you for joining us today to discuss Arrowhead's results for its fiscal 2025 second quarter ended March 31, 2025.

With us today from management are president and CEO Dr. Chris Anzalone, who will provide an overview; Dr. Bruce Given, interim chief medical scientist, who will provide an update on our cardiometabolic pipeline; Andy Davis, senior vice president and head of global cardiometabolic franchise, who will provide an update on commercialization activities; Dr. James Hamilton, chief medical officer and head of R&D, who will discuss our earlier stage development programs; and Ken Myszkowski, our outgoing chief financial officer who is retiring this week, who will give a review of the financials. We also welcome Dan Apel, our incoming

CFO, who is also with us on the call today. Following management's prepared remarks, we will open the call to questions.

Before we begin, I would like to remind you that comments made during today's call contain certain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than statements of historical fact are forward-looking statements and are subject to numerous risks and uncertainties that could cause actual results to differ materially from those expressed in any forward-looking statements. For further details concerning these risks and uncertainties, please refer to our SEC filings, including our most recent annual report on Form 10-K and our quarterly reports on Form 10-Q.

I'd now like to turn the call over to Christopher Anzalone, President and CEO of the Company. Chris?

Chris Anzalone

Thanks Vince. Good afternoon everyone and thank you for joining us today.

Before I start, I want to say thank you to Ken and wish him the best in his retirement. Ken has been a valuable member of the Arrowhead team, and he retires at a time of great financial strength for the company. The finance organization that Ken built over the years is very capable and provides strong support to our ambitious development and commercialization plans. From all of us at Arrowhead, thank you for all the important contributions over the last 16 years.

I am also excited that Dan Apel will join us as our new CFO at a critical time for Arrowhead. We expect to make the transition from development stage to commercial stage, with the planned launch of plozasiran this year, pending regulatory review and approval. Dan is an accomplished pharmaceutical executive who can make an immediate and important impact on our business.

Let's now talk about our business and the progress we're making toward our short, mid, and long-term goals. Arrowhead is at an important point both in terms of capabilities and potential value as we drive our organization toward our first commercial launch, which we anticipate this year. Following this, we expect multiple additional independent and partner launches over the following few years. The combination of:

commercial expansion,

our extraordinarily productive discovery engine,

the increasingly validated nature of our platforms and RNAi modality,

our large pipeline of clinical-stage assets,

our strong balance sheet,

and clear access to additional non-dilutive capital

together provide us with a level of upside potential and stability that I believe is a rarity in our industry. This is always attractive, but is even more valuable at a time when biotech markets have been depressed for the past several years and near-term capital markets are uncertain at best. As the current biotech market weakness causes people to weigh the tradeoff of stability versus the potential for explosive value growth, I think we have the tools for both.

I view our value proposition in layers.

Layer 1 is Plozasiran. It constitutes our primary near- and mid-term value driver and provides a strong base for us. Plozasiran has shown to be a potent triglyceride-lowering agent across multiple clinical studies in hundreds of patients. We believe there are 3 - 4 million people in the US alone who suffer from severe hypertriglyceridemia (or SHTG), as defined by fasting triglyceride levels above 500 mg/deciliter. We are preparing to launch into a small subgroup of this population, patients with familial chylomicronemia syndrome (or FCS), and have a PDUFA date of November 18, 2025. We also completed the submission of a Marketing Authorization Application or MAA with the EMA and are working through additional planned submissions in other select geographies.

The P3 data supporting our regulatory submissions were consistent and encouraging. Genetically-defined and clinically-defined FCS patients responded similarly, with reductions in tryglycerides of about 80% from baseline; approximately 75% and 50% of patients had triglycerides go below 880 and 500 mg/dl, respectively, which are discussed in guidelines and the academic literature as important goals for minimizing pancreatitis risk. These are truly impressive levels to achieve in FCS patients, as the mean baseline triglyceride level in the study was approximately 2500 mg/dL. Plozasiran was generally well-tolerated and showed triglyceride reductions in 100% of patients treated at the primary endpoint of 10 months.

Our hope of treating FCS patients is important. This is an historically underserved population and we believe plozasiran could be an important medicine for them.

However, we view this as just the beginning.

SHASTA-3, SHASTA-4, and MUIR-3 are P3 studies designed to support a supplemental NDA and other applications on a global basis to enable us to treat the

broader SHTG patient population. These studies are moving rapidly and we believe they could be fully-enrolled this summer. We are also in the process of initiating SHASTA-5, which is an outcomes study to specifically evaluate the risk reduction of acute pancreatitis in high-risk patients with SHTG. We think this is an innovative strategy to potentially demonstrate meaningful value for patients, physicians, and payors.

Our second layer of value may be our initial obesity candidates, and initial CNS candidates. Regarding the former, ARO-INHBE is currently dosing in obese patients and we expect ARO-ALK7 to begin dosing in obese patients shortly. Both are designed to intervene in a biological pathway regulating fat storage. ARO-INHBE targets hepatocytes with the same TRiMTMplatform used in several ongoing clinical studies that has been in thousands of patients. It is designed to reduce hepatocyte expression of Activin E, which is a ligand for adipose ALK7.

ARO-ALK7 is the first in industry adipocyte-targeted siRNA with a new TRiMTMplatform that, in animal models, has shown good uptake in adipose tissue and high levels of target gene knockdown with a long duration of effect that may enable Q4 month, Q6 month, or less frequent administration. ARO-ALK7 is designed to reduce expression of the ALK7 receptor itself in adipose tissue.

Both programs demonstrated substantial reductions in visceral fat mass versus control while simultaneously preserving lean mass in animal models. Both targets are also supported by human genetics, where loss-of-function carriers have favorable body composition and metabolic characteristics compared to non-carriers, without any apparent safety cost. It's a very intriguing pathway that we believe may fill some important gaps left by standard of care obesity treatments, addressing some of the shortcomings of the GLP1/GIP class. The possibility of

Disclaimer

Arrowhead Pharmaceuticals Inc. published this content on May 12, 2025, and is solely responsible for the information contained herein. Distributed via Public Technologies (PUBT), unedited and unaltered, on May 12, 2025 at 21:07 UTC.