On January 8, 2015 CytRx Corporation reported positive interim results from its ongoing Phase 2 clinical trial evaluating the safety and efficacy of aldoxorubicin for the treatment of Kaposi’s Sarcoma (KS) in HIV-infected patients (Press release CytRx, JAN 8, 2015, View Source [SID:1234501287]). The clinical results reported are from 9 patients, 6 of which carried the worst KS prognosis. Also, 4 of the 9 patients had received from 1 to 8 prior liposomal doxorubicin (Doxil) cycles. Efficacy results revealed that all 9 patients (100%) exhibited a decrease in skin lesions and in the number of cancer cells expressing the KS virus DNA. Of the 6 patients with lung tumors, 4 patients (66%) demonstrated either a partial or complete response, with no patients (0%) demonstrating progression. Data thus far show that aldoxorubicin can be detected in all tumor biopsies 24 hours following drug administration. Preliminary safety results showed only 2 patients (22%) experienced a grade 4 adverse event (transient neutropenia and anemia). Other adverse events were mild and most were unrelated to aldoxorubicin.

“The current standard-of-care for severe dermatological and systemic KS is Doxil, however, many patients experience significant toxicity, or exhibit minimal to no clinical response to this agent,” said Steven Kriegsman, Chairman and CEO of CytRx. “Aldoxorubicin has demonstrated effectiveness against a range of tumors in the phase 2 clinical trial, thus we are optimistic in regard to its potential as a treatment for KS. This data provides important insight into both the safety and efficacy of aldoxorubicin in this indication, and the lack of toxicity of our drug in these patients is very impressive. We look forward to further data using aldoxorubicin in KS as this study continues.”

Assuming a positive outcome of the ongoing Phase 2 clinical trial, CytRx plans to discuss with the FDA a potential pathway for the registration of aldoxorubicin for use in KS. The Company intends to submit the Phase 2 KS data for presentation at the 2015 ASCO (Free ASCO Whitepaper) annual meeting.

This open-label Phase 2 clinical trial is expected to enroll up to 30 patients, randomly assigned to three equally sized treatment arms which will receive aldoxorubicin at 50, 100 or 150 mg/m2 by 30-minute intravenous infusion. Because the KS patients in the study have compromised immune systems, the aldoxorubicin dosages administered in the trial are lower than those administered in the Company’s clinical testing of aldoxorubicin in patients with soft tissue sarcomas. Patients with advanced KS receive aldoxorubicin on day 1, then every 3 weeks until evidence of tumor progression, unacceptable toxicity or withdrawal of consent. The primary objectives of preliminary efficacy include evaluation of the size, number and nodularity of skin lesions, change in size and number of lung lesions and changes in the number of tumor cells that express the KS virus DNA (Human Herpes Virus 8). The Company is also evaluating the level of aldoxorubicin uptake into lesions. Safety is being assessed through monitoring of adverse events and the ability to remain on assigned treatment. The trial is being conducted at the Louisiana State University Health Sciences Center in New Orleans, LA. Possible additional sites are being considered to expedite enrollment.

KS is an orphan indication in the U.S.