Geron's blood cancer drug succeeds in late-stage study

Geron Corp's shares rose 50% after the company announced that its experimental blood cancer drug helped more patients achieve blood transfusion independence when compared to a placebo in a late-stage trial. Imetelstat, Geron's lead drug, was being tested in patients with a type of myelodysplastic syndrome (MDS), a difficult-to-treat family of blood cancers. To manage their anaemia, patients with the disease require frequent blood transfusions. The company intends to submit applications for approval in the United States in mid-2023 and in Europe in the second half of 2023, with the drug expected to hit the market in 2024.

According to Chief Executive Officer John Scarlett, the company anticipates a peak market potential of $1.2 billion in the United States and some key EU countries. Nearly 40% of the 118 patients who were on the drug showed independence from transfusion for eight weeks, compared with 15% of the 60 patients on placebo, the company said in a statement. The results suggest that treatment with the drug "may be altering the course of the disease," Chief Medical Officer Faye Feller said in a statement.

The drug also met its secondary goal, with 28% of the patients using the drug not needing transfusion for 24 weeks, compared with 3.3% of the patients on placebo. Adverse effects observed during the late-stage trial were consistent with previous trials, with low count of platelets and white blood cells among the most common. The company said these adverse effects were not uncommon. Geron is also testing the drug for myelofibrosis, a rare type of bone marrow cancer, and expects an interim analysis in 2024. If approved, the drug would compete with Incyte Corp's myelofibrosis drug, Jakafi.