Geron (NASDAQ:GERN)
In the biotech sector, I'd encourage small-cap enthusiasts with the highest levels of risk tolerance to keep their eyes on clinical-stage drug developer Geron, which currently boasts a $470 million valuation and has just four Wall Street firms covering it.
There are obvious risks that come with investing in a clinical-stage pipeline. For example, there are no guarantees that any of the pipeline products will succeed, and a clinical-stage biotech could very well continue to burn through its cash on hand. In Geron's case, the added risk is that its pipeline consists solely of a single experimental drug, imetelstat. If this experimental drug fails in clinical trials or isn't approved by the Food and Drug Administration, Geron could lose most of its market value.
The reason I'd suggest you keep this relative unknown on your radar is that imetelstat did something in clinical studies that no drug has done before: It elicited partial and complete responses in patients with myelofibrosis, a rare type of bone marrow cancer that leads to scarring.
The existing treatment on pharmacy shelves for myelofibrosis is Jakafi, a JAK2 inhibitor that led to spleen size reduction in clinical trials. However, Jakafi's purpose is to lessen symptoms of the disease, not to provide any curative benefit. Imetelstat, on the other hand, led to a 41% overall response rate in an early-stage study conducted by the Mayo Clinic. In recently released results from a phase 2 pilot study, imetelstat led to the reversal of bone marrow fibrosis.
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