12 NASDAQ Stocks with Biggest Upside Potential According to Analysts

3. Arrowhead Pharmaceuticals Inc. (NASDAQ:ARWR)

Average Upside Potential as of November 21: 145.40%

Arrowhead Pharmaceuticals Inc. (NASDAQ:ARWR) is a biopharmaceutical company that specializes in developing innovative therapies using its proprietary RNA interference (RNAi) technology to target and silence specific genes responsible for various diseases. The lead product candidate, ARO-AAT, is designed to treat alpha-1 antitrypsin deficiency, a genetic disorder that can lead to liver disease and lung problems.

It’s working on a pipeline of 20+ drugs, with the first, plozasiran, expected to launch in 2025 in familial chylomicronemia syndrome (FCS) and revolutionize the treatment of several lipid disorders. A key advantage of plozasiran is its unique mechanism of action, which allows it to significantly lower triglyceride levels, positioning it as a potential best-in-class treatment.

A recent significant milestone for the company was the successful completion of the Phase 3 PALISADE study for plozasiran. The study met its primary endpoint and key secondary endpoints, demonstrating the drug’s efficacy and safety. This achievement validates its scientific approach and positions the company for future growth and success.

Baron Opportunity Fund made the following comment about Arrowhead Pharmaceuticals, Inc. (NASDAQ:ARWR) in its Q1 2023 investor letter:

“Arrowhead Pharmaceuticals, Inc. (NASDAQ:ARWR), a biotechnology company specializing in RNA interference (RNAi) medications to treat a variety of diseases, detracted from performance. The company has lacked major wins in recent years, while also tallying some losses. Management’s late-stage drug licensing efforts to drive upfront and running royalties to bolster its balance sheet and ensure a longer cash runway have also pressured shares. Reinvigorating the company’s strategic story remains a key consideration for Arrowhead going forward. Last year, we reduced our portfolio weighting in Arrowhead but decided to maintain a foothold position. We believe Arrowhead’s efforts to target RNAi to the lungs will open a new therapeutic area of exploration. In our view, this initiative may lead to RNAi economies of scale, allow for an expansion of pipeline and collaboration opportunities, and generate a long runway for growth.”