Arrowhead Pharmaceuticals has begun laying the groundwork for its entry into a rare disease market, kicking off an awareness campaign focused on the unmet need its drug candidate is positioned to serve.
The biotech is developing plozasiran, a siRNA candidate designed to silence APOC3, for the treatment of conditions including familial chylomicronemia syndrome (FCS). People with the rare genetic disorder have a mutation that results in very high levels of triglycerides in the blood.
Because APOC3 is involved in triglyceride transport, plozasiran’s mechanism could help people with FCS. The candidate is trailing Ionis antisense prospect olezarsen in the race to market but Arrowhead believes its asset may be differentiated, in part because of its effect on potentially fatal acute pancreatitis.
Arrowhead’s “We'll Get There Soon” campaign reflects its potential benefits of plozasiran. The campaign logo uses “500N” instead of “SOON.” During clinical development, Arrowhead has targeted patients who have triglycerides of 500 mg/dL or more at screening. The decision reflects evidence that 500 mg/dL is the threshold above which acute pancreatitis becomes a bigger risk.
Below the “500N” graphic, the campaign website states “imagine lowering the risk of acute pancreatitis by achieving the guideline-recommended goal of triglycerides <500 mg/dL.” The rest of the website lists the current treatment options and their limitations—and looks forward to future interventions that may be more effective.
The “future strategies” section is focused on siRNA, the technology used in Arrowhead’s candidate. The section explains how siRNA acts on molecular machinery and poses the question “what if RNA silencing held the key to managing refractory, extremely high triglycerides?”
Arrowhead, which timed the launch to coincide with FCS Awareness Day, kicked off the campaign at an event at its headquarters. The in-person launch was supported by activities on social media. Posts about the event are the company’s first, and currently only, updates on Facebook and Instagram.