FDA Lifts Partial Hold On Imetelstat Study

The FDA and Geron Crop have announced that the FDA has lifted the partial hold on the clinical study testing of imetelstat, Geron’s myelofibrosis drug. Myelofibrosis is a rare blood cancer. The Geron Crop early stage imetelstat drug study is sponsored by the Mayo Clinic and it was stopped by the FDA in March because of concerns about liver toxicity issues.

The FDA wanted to investigate the extent of the liver damage and the potential for reversibility of that damage. The study had 79 patients, 20 of which have dropped out. Geron has stated that it intends to continue the clinical study, and that enrolled patients will continue to be treated with the imetelstat drug. In response to the FDA’s lifting of the partial hold, Geron’s shares rose as high as 33 percent.

In a study last year, imetelstat cured 22 percent of myelofibrosis patients, and it showed immense potential despite needing more testing before it hit the market. Myelofibrosis can cause scarring of the bone marrow leading to symptoms such as anemia, weakness, fatigue, and enlarged spleen and liver. Some patients with myelofibrosis live symptom free for years, but most get progressively worse and some develop leukemia.

Imetelstat is the only drug currently under investigation by Geron. Geron once focused on stem cell therapies, but the company has since shifted focus to cancer treatments. The company had also been researching imetelstat’s potential in treating thrombocythemia and multiple myeloma. Those trials were also suspended in March and have not yet been given clearance to continue research.