Imetelstat: Blood cancer drug succeeds in late-stage study

Geron said on Wednesday its experimental blood cancer drug helped more patients achieve independence from blood transfusions when compared with a placebo in a late-stage trial, sending its shares up 50%.

Geron’s lead drug, imetelstat, was being studied in patients with a type of myelodysplastic syndromes (MDS), a difficult to treat family of blood cancers. Patients with the disease require frequent blood transfusions to manage their anemia.

The company plans to submit applications for approval in the United States in mid-2023 and in Europe in the second half of 2023, and expects to commercially launch the drug in 2024.

It anticipates a peak market potential of $1.2 billion in the United States and some key EU countries, Chief Executive Officer John Scarlett told Reuters.

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’s myelofibrosis drug, Jakafi.