Vertex Pharmaceuticals ( NASDAQ:VRTX ) just hit a major roadblock. The biotech heavyweight's much-anticipated non-opioid painkiller, suzetrigine, flopped in its latest Phase 2 trial for chronic lower back pain. The results? Pain scores for patients on suzetrigine barely edged out those on a placebo, with a reduction of 2.02 vs. 1.98 on a standard pain scale. That tiny margin wasn't enough to impress Wall Streetor investors. Shares nosedived 14% on Thursday, wiping out more than $10 billion in market value. Ouch.

Here's the kicker: Vertex isn't giving up. They're pressing ahead with a Phase 3 trial, banking on tweaks to trial designs to tackle what they're calling a placebo response problem. Some analysts remain hopeful, but plenty aren't buying it. RBC Capital Markets went as far as calling this a near worst-case scenario for the drug's potential in chronic pain. Still, the FDA is reviewing suzetrigine for acute pain with a decision due by January 30, 2025so there's a glimmer of hope for investors banking on the drug as a non-addictive alternative to opioids.

If suzetrigine makes it to market, it could be the first new class of pain medication in over 20 yearsa big deal given the ongoing opioid crisis. The drug works by blocking pain signals at their source instead of rewiring the brain's reward centers like traditional opioids. But with competitors circling and skepticism mounting, Vertex has its work cut out. Investors are watching closely, but for now, the mood is cautious. The big question: Can Vertex turn this potential breakthrough into the multibillion-dollar business they promised?

This article first appeared on GuruFocus .