Shares of Cytrx Corporation (CYTR) soared 20% on the news that their cancer drug, Aldoxorubicin, achieved positive phase 2 results in patients with brain cancer. Phase 2 is to show clinical efficacy but Cytrx hit the ball out of the park. This is because 12 patients enrolled in the trial showed tumor shrinkage in the brain, and one of the 12 patients had a complete response. A complete response is where the tumor is completely gone and the patient has achieved a full cure of their cancer. This phase 2 trial is expected to enroll up to 28 patients in total who will be receiving Aldoxorubicin to treat their brain cancer. The primary endpoint of the study will be progression-free survival at at 6-month time point along with overall survival upon completion of the trial. Progression-free survival is how long the patient goes in the trial without their cancer advancing or becoming worse. Overall survival measures the amount of time the patient goes through to the end of the trial.

The type of brain cancer in these patients is known as Glioblastoma multiforme. Glioblastoma multiforme is the most aggressive form of brain cancer in which patients have limited treatment options. Some other treatment options include chemotherapy along with surgery, but both carry significant risks to the patient. Aldoxorubicin is a chemotherapeutic agent in a way because it is derived from doxorubicin. Cytrx has been able to combine doxorubicin with an acid linker known as EMCH that creates the compound Aldoxorubicin. This combination allows the drug to be more efficacious in a variety of cancers, reduce toxicity (minimize side effects), and work more quickly to reach the cancerous target.

With this compound Cytrx can target many other types of cancers as well. The company's lead drug compound is currently testing Aldoxorubicin in patients with soft tissue sarcoma in a phase 3 trial. This treatment is used as a second-line type of treatment which occurs when patients have failed with primary treatment of a placebo compound.