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Business Update
Signs Collaboration Agreement with Novartis
On September 2, 2025, Arrowhead Pharmaceuticals, Inc. (NASDAQ:ARWR) announced a global collaboration and licensing agreement with Novartis (NYSE:NVS) for ARO-SNCA, a preclinical stage siRNA candidate that targets alpha-synuclein for the treatment of synucleinopathies (e.g., Parkinson’s Disease), and for additional collaboration targets utilizing Arrowhead’s TRiM™ platform. Arrowhead will receive a $200 million upfront payment and is eligible to receive up to $2 billion in milestone payments along with royalties on commercial sales. For the additional programs under the agreement, Arrowhead will conduct and complete preclinical research activities that are required for a clinical trial application (CTA) filing, with Novartis then assuming control over the development, manufacturing, and commercialization activities. The collaboration agreement is expected to close in the second half of 2025.
Ionis Announced Positive Phase 3 Results in sHTG
On September 2, 2025, Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) announced positive topline results from the Phase 3 CORE and CORE2 trials of olezarsen in patients with severe hypertriglyceridemia (sHTG). The company reported that olezarsen demonstrated a statistically significant placebo-adjusted mean reduction in fasting triglycerides of up to 72% and a statistically significant reduction in acute pancreatitis events of 85%. Adverse events were generally balanced across treatment groups and serious adverse events occurred less frequently in the olezarsen group compared to placebo. We anticipate Ionis submitting a supplemental new drug application (sNDA) to the U.S. FDA before the end of 2025, as olezarsen is already approved for the treatment of familial chylomicronemia syndrome (FCS).
We believe these data provide an excellent read-through for plozasiran in sHTG, for which Arrowhead is currently conducting the SHASTA-3 and SHASTA-4 trials. Those trials are both fully enrolled and we anticipate them completing in mid-2026, with a planned sNDA filing in the fourth quarter of 2026. An NDA for plozasiran for the treatment of FCS is currently under evaluation by the FDA with a PDUFA date of November 18, 2025.
Sarepta Collaboration Update
On August 13, 2025, Arrowhead announced that it had elected to receive approximately $50 million worth of Arrowhead common stock and approximately $50 million in cash from Sarepta Therapeutics (NASDAQ:SRPT) to satisfy the $100 million milestone payment owed to Arrowhead that was tied to enrollment of certain cohorts in the Phase 1 trial of ARO-DM1. The $50 million worth of Arrowhead common stock was placed into treasury to reduce the number of shares outstanding. The trigger for a second milestone payment of $200 million from Sarepta is expected before the end of calendar 2025, and that will continue to be payable in cash. Following the $100 million milestone payment, Sarepta then sold the balance of its remaining holdings of Arrowhead common stock in a separate negotiated block trade.
Conclusion
Arrowhead continues to form important collaboration agreements for a number of its pipeline candidates, which is good for both securing the company’s balance sheet along with getting as many drugs into the clinical as possible. We anticipate the company will continue to form additional strategic partnerships in the future.
Since we believe that plozasiran is a superior drug to olezarsen, both in terms of target engagement and dosing schedule, the positive results for olezarsen announced by Ionis in sHTG gives us further confidence that the SHASTA trials will have a positive readout. We look forward to Arrowhead announcing results from the SHASTA trials in mid-2026.
Lastly, as we had anticipated, the recent issues for Sarepta have not precluded the company from fulfilling its financial obligations with Arrowhead thus far, and we don’t foresee any risks to their collaboration in the future. After taking into account the share reduction and the Novartis deal into our model, our valuation is now $74 per share.
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