CytRx Corporation (NASDAQ:CYTR) announced that, in response to a request from the company, the U.S. Food and Drug Administration (FDA) has agreed to a Type B pre-NDA meeting at which the company will seek input on its planned New Drug Application (NDA) for aldoxorubicin as a new second-line treatment for patients with soft tissue sarcomas (STS). Assuming a positive outcome from this pre-NDA meeting, CytRx expects to submit an NDA for aldoxorubicin to the FDA in the last quarter of 2017, and, subject to FDA approval, bring aldoxorubicin to market next year.
“Based on positive, statistically significant results from our pivotal Phase 3 trial evaluating aldoxorubicin compared to investigator’s choice in patients with previously treated relapsed or refractory sarcomas, we requested, and were recently granted, a Type B meeting with the FDA,” said Daniel Levitt, M.D., Ph.D., Chief Operating Officer and Chief Medical Officer of CytRx. “The purpose of this meeting is to share the Phase 3 data with the Agency and discuss the regulatory path forward for aldoxorubicin. In our view, this meeting is an important next step toward our goal of bringing aldoxorubicin to the thousands of patients in need of a new treatment for relapsed soft tissue sarcoma.”
Overview of the Updated Phase 3 Trial and Results
The randomized, controlled Phase 3 trial enrolled a total of 433 patients at 79 clinical sites. Patients with metastatic, locally advanced or unresectable soft tissue sarcomas who had either not responded to, or who had progressed following treatment with one or more systemic regimens of non-adjuvant chemotherapy were randomized 1:1 to be treated with aldoxorubicin or the investigator’s choice of an approved chemotherapeutic regimen, including doxorubicin, ifosfamide, dacarbazine, pazopanib (Votrient®), or gemcitabine plus docetaxel. The primary endpoint of the study is PFS. Secondary endpoints include overall survival, response rates, disease control rates and safety.
In November 2016, CytRx reported positive results which demonstrated a statistically significant improvement in progression-free survival (PFS) between aldoxorubicin and investigator’s choice therapy in 246 patients with leiomyosarcoma and liposarcoma, (p=0.007). The hazard ratio (HR) was 0.62 (95% CI 0.44-0.88), representing a 38% reduction in the risk of tumor progression for patients receiving aldoxorubicin versus investigator’s choice. Leiomyosarcoma and liposarcoma are the two most common types of STS and accounted for 57% of the patients enrolled in the trial. Aldoxorubicin also demonstrated a statistically significant improvement in PFS over investigator’s choice in 312 patients treated in North America (p=0.028; HR=0.71, 95% CI 0.53-0.97). Notably, aldoxorubicin performed better than investigator’s choice for the entire study population and narrowly missed statistical significance (p=0.12; HR=0.81, 95% CI 0.64-1.06). All responses were determined by an independent, blinded central lab assessment of scans.
Aldoxorubicin did not cause clinically significant cardiac, renal, or hepatic toxicities. For the global trial population, the most commonly reported adverse events were neutropenia and anemia consistent with prior clinical trials with aldoxorubicin. Grade 3 or higher adverse events were manageable with supportive care and occurred at a rate of 61% for patients receiving aldoxorubicin and 46% in patients treated with investigator’s choice. Importantly, treatment-emergent adverse events leading to discontinuation occurred in 4.2% of patients treated with aldoxorubicin, compared to 6.3% for patients receiving investigator’s choice.
Patients continue to be followed for overall survival (OS), a secondary endpoint, and CytRx expects the OS data to be available in 2017. (Original Source)
Shares of CytRx are rising 11.37% to $0.430, or up $0.044 in pre-market trading Wednesday. CYTR has a 1-year high of $3.66 and a 1-year low of $0.36. The stock’s 50-day moving average is $0.48 and its 200-day moving average is $0.73.
On the ratings front, FBR analyst Christopher James reiterated a Buy rating on CYTR, with a price target of $3, in a report issued on November 11. The current price target represents a potential upside of 669% from where the stock is currently trading.
According to TipRanks.com, James has a yearly average loss of 14.2%, a 30% success rate, and is ranked #4244 out of 4351 analysts.
CytRx Corp. operates as a biopharmaceutical research and development company specializing in oncology. It provides clinical development of aldoxorubicin oncology pipeline for the treatment of cancer.