So here's a classic biotech puzzle: Fulcrum Therapeutics Inc. (FULC) announced some pretty encouraging data for its sickle cell disease drug on Tuesday, and the stock... went down. Not a little down, either—it fell over 10%. Welcome to the sometimes-illogical world of clinical trial reactions.
The company released initial results from the 20 mg dose group in its Phase 1b PIONEER trial for a drug called pociredir. The headline number is fetal hemoglobin, or HbF. This is the good kind of hemoglobin that babies have, and getting adults with sickle cell to produce more of it is a key therapeutic goal. In this latest cohort, the mean absolute HbF increased by 12.2% at Week 12. That's up from an 8.6% increase seen in the earlier 12 mg group. Even better, 58% of patients hit HbF levels of 20% or more.
Digging a bit deeper, the proportion of red blood cells actually containing this fetal hemoglobin (called F-cells) basically doubled, going from a mean of 31% at the start to 63% at Week 12. That's important because F-cells are more resistant to the sickling and destruction that causes so many problems in this disease. The data also showed improvements in markers related to red blood cell breakdown and anemia.
On the safety front—always a huge focus—things looked clean. The company reported no serious adverse events related to pociredir. One patient did drop out on the first day due to a serious event, but it was deemed unrelated to the drug. That patient is counted in the safety analysis but not the main effectiveness data.
So, with solid efficacy signals and a clean safety sheet, why the stock drop? It's hard to say for sure. Sometimes the market had even higher expectations baked in. Sometimes it's just profit-taking after a run-up. Or maybe investors are looking ahead to the long regulatory path still to come.
Speaking of the path ahead, Fulcrum laid out its next steps. The plan is to start a trial that could support asking for regulatory approval in the second half of 2026, pending talks with health authorities. They also plan to chat with the European Medicines Agency around mid-2026. For patients already in the current trial, Fulcrum is setting up an extension study to see how long the benefits last.
Financially, the company seems well-positioned for the long haul. It ended 2025 with over $352 million in cash and equivalents, which it says funds operations into 2029.
Wall Street's take is a bit of a mixed bag but generally supportive. The average price target among analysts is $12.80, and the stock carries a consensus Buy rating. Recent moves include JP Morgan starting coverage with an Overweight rating and a $20 target in January, and Truist Securities raising its target to $18 in December. Not everyone is a bull, though; B of A Securities has an Underperform rating with a $7 target.
Despite the promising science, Fulcrum's shares finished Tuesday at $9.80, down 10.01%. It's a reminder that in biotech, good data doesn't always equal a green day on the stock chart—at least not immediately.
