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Just caught Fulcrum's earnings drop - mixed bag honestly. Stock tanked 13% after they reported Q4 results, but the real story is their Pociredir trial data for sickle cell disease actually looking solid.
So Fulcrum announced 12-week results from their Phase 1b PIONEER trial and the numbers are pretty interesting. Patients on the 20mg dose saw fetal hemoglobin jump from 7.1% to 19.3% in just 12 weeks - that's a 12.2% increase. They're also seeing improvements in hemolysis markers and some encouraging trends in reducing vaso-occlusive crises. For a rare disease treatment, this kind of response is the kind of thing biotech investors usually get excited about.
On the financial side, Fulcrum's cash position actually strengthened - they raised $164M in a December offering and now have $352M in cash and securities. They're saying that's enough runway through 2029, which is solid for a clinical-stage company burning through development costs. The net loss did widen to $74.9M for full year 2025 vs $9.7M prior year, but that's typical when you're scaling trials.
The FDA already gave Pociredir Fast Track and Orphan Drug designation for SCD, which helps with the approval pathway. Fulcrum's planning to kick off a potential registration-enabling trial in H2 2026 pending FDA feedback. Could be a catalyst if Phase 2 data holds up. Interesting risk/reward if you're into biotech plays.