CytRx provides overview of clinical development programs, reports 2014 Q3 financial results

Nov 4 2014

CytRx Corporation (CYTR), a biopharmaceutical research and development company specializing in oncology, today reported financial results for the three months ended September 30, 2014, and also provided an overview of recent accomplishments by and upcoming milestones for its clinical development programs.

"We had a very active third quarter characterized by the initiation of three new clinical trials evaluating aldoxorubicin as both a single agent and in combination with existing therapies in several cancer settings," said Steven A. Kriegsman, CytRx Chairman and CEO. "On the discovery and regulatory fronts, we successfully launched our new discovery laboratory in Freiburg, Germany and received several additional orphan designations from the FDA, allowing us to formally expand our global oncology franchise to include glioblastoma, small cell lung and ovarian cancers. Our development team continues to raise awareness of the aldoxorubicin program to the oncology community through data presentations at prestigious oncology medical meetings and through publications in renowned peer reviewed medical journals. We have several important clinical milestones that we are working toward in the coming months and quarters, including multiple data readouts, and we look forward to updating investors on our progress as these events unfold."

Third Quarter 2014 and Recent Highlights

Preclinical Glioblastoma (GBM) Data Published in Neoplasia. In October, a paper, titled "Therapeutic Efficacy of Aldoxorubicin in an Intracranial Xenograft Mouse Model of Human Glioblastoma," was published in the journal Neoplasia. The paper discusses previously reported positive and impressive preclinical data which demonstrated that aldoxorubicin significantly increased survival almost 2½ fold compared to doxorubicin treatment in an in vivo xenograft tumor model employing growth of human GBM tumors in mouse brains. These results led CytRx to initiate the Phase 2 clinical trial in patients with GBM discussed below. The full publication can be accessed online here.

Phase 1b/2 Aldoxorubicin Clinical Data in Soft Tissue Sarcoma (STS) Published in Cancer, the Journal of the American Cancer Society. In October, a paper, titled "A Phase 1b/2 Study of Aldoxorubicin in Patients With Soft Tissue Sarcoma," discussing the results of CytRx's completed Phase 1b/2 trial of aldoxorubicin was published online in Cancer, the prestigious, peer-reviewed journal of the American Cancer Society. The findings in this clinical trial support CytRx's current global phase 3 pivotal trial in patients with STS who have relapsed or have not responded to prior chemotherapy. The full publication can be accessed online here.

Aldoxorubicin Data Presented at 2014 CTOS Annual Meeting. In October, aldoxorubicin data were highlighted at the 2014 Connective Tissue Oncology Society (CTOS) Annual Meeting in Berlin, Germany. Sant P. Chawla, M.D., F.R.A.C.P., Director of the Sarcoma Oncology Center and principal investigator of the trial, gave a moderated paper presentation where he provided an update of CytRx's Phase 2b global clinical trial of aldoxorubicin for the treatment of first-line STS. As part of the presentation, Dr. Chawla reported that the trial had not yet reached the necessary number of survival events to report on overall survival (OS), a secondary endpoint of the trial, due to longer than expected survival of the study patients. The study will continue to track survival events and final results may be reported by the end of 2014 if the expected number of events occurs. In addition to the moderated paper presentation given by Dr. Chawla, CytRx was also selected for a poster presentation discussing the cardiac safety of aldoxorubicin and a publication-only paper discussing the pharmacokinetics of aldoxorubicin for the treatment of STS.

Appointed Steven A. Kreigsman as Chairman. In October, the CytRx Board of Directors appointed Steven A. Kriegsman, President, Chief Executive Officer and a Director of the Company, as Chairman of the Board, replacing Max E. Link, Ph.D. who passed away unexpectedly in early October.

Launched Discovery Lab to Develop Albumin-Binding Anti-Cancer Drug Platform. In October, CytRx commenced operations at its new discovery laboratory, located in Freiburg, Germany. The new laboratory will conduct discovery and translational research to create drug candidates that utilize novel linker technologies that couple chemotherapeutic agents and proteins either inside the body or externally, using albumin to concentrate drug in tumors. The discovery team, overseen by CytRx's Executive Vice President and Chief Medical Officer Daniel Levitt, M.D., Ph.D, and led by Felix Kratz, Ph.D., Vice President of Drug Discovery and inventor of aldoxorubicin, and Andre Warnecke, Ph.D., Senior Director of Drug Discovery, is expanding CytRx's novel albumin-binding anti-cancer drug pipeline. CytRx's discovery laboratory is on target to yield its first clinical candidate in 2015.

Initiated Phase 1b Clinical Trial with Combination of Aldoxorubicin and Gemcitabine in Patients with Metastatic Solid Tumors. In October, CytRx announced the initiation of enrollment in an open-label Phase 1b clinical trial designed to investigate the preliminary safety and activity of aldoxorubicin plus gemcitabine in subjects with metastatic solid tumors. The clinical trial is expected to enroll up to 30 male and female patients between the ages of 15 and 80 with advanced, unresectable, metastatic solid tumors that have either relapsed or were refractory to treatment with at least one prior chemotherapy or immunotherapy regimen and for which no standard approved therapy exists. This combination could be used to treat patients with advanced pancreatic and ovarian cancers. The Company expects to complete enrollment by the third quarter of 2015.

Initiated a Global Phase 2b Clinical Trial of Aldoxorubicin for the Treatment of Relapsed/Refractory Small Cell Lung Cancer. In September, CytRx announced the initiation of a global Phase 2b clinical trial evaluating aldoxorubicin compared to topotecan in subjects with extensive-stage small cell lung cancer (SCLC) who have relapsed or were refractory to prior chemotherapy. The open-label Phase 2b clinical trial is expected to enroll approximately 132 patients (1:1 randomization) with extensive-stage SCLC who have relapsed or were refractory to prior chemotherapy. Patients will receive either aldoxorubicin or topotecan. The primary endpoint is progression-free survival (PFS) and the secondary endpoints are OS, overall response rates (partial and complete) and the safety of aldoxorubicin compared to topotecan in this population. Enrollment is expected to be completed in 2015 and PFS data are anticipated by mid-2016.

Received Multiple FDA Orphan Drug Designations for Aldoxorubicin for the Treatment of GBM, Small Cell Lung Cancer and Ovarian Cancer. In September, CytRx announced that the U.S. Food and Drug Administration (FDA) granted multiple Orphan Drug Designations for the Company's lead drug candidate, aldoxorubicin, in three indications: glioblastoma multiforme (GBM), small cell lung cancer and ovarian cancer.

Aldoxorubicin Global Phase 3 STS and Global Phase 2b Small Cell Lung Cancer Trial Designs Highlighted at ESMO 2014 Congress. In September, CytRx presented study designs and progress updates from two of its key clinical trials of aldoxorubicin, a pivotal global Phase 3 clinical trial in relapsed/refractory STS and a global Phase 2b clinical trial in small cell lung cancer, at the European Society for Medical Oncology (ESMO) 2014 Congress in Madrid, Spain.

Initiated Phase 1b Clinical Trial with Combination of Aldoxorubicin and Ifosfamide/Mesna as First-Line Treatment in Patients with Advanced Sarcomas. In September, CytRx initiated an open-label Phase 1b clinical trial designed to investigate the preliminary safety and activity of aldoxorubicin plus ifosfamide/mesna as a first-line treatment in subjects with various sarcomas. The trial is expected to enroll up to 30 male and female patients between the ages of 15 and 80 with locally advanced, unresectable, and/or metastatic STS, chondrosarcoma or certain osteosarcomas. The ifosfamide treatment regimen was devised in collaboration with the M.D. Anderson Cancer Center and the Sarcoma Oncology Center. The Company expects to complete enrollment by the third quarter of 2015.

Oral Presentation of CytRx's Aldoxorubicin Phase 2b Clinical Trial in STS Highlighted in The Lancet Oncology. In August, an oral presentation given by Dr. Chawla, titled "Randomized phase 2b trial comparing first-line treatment with aldoxorubicin versus doxorubicin in patients with advanced soft tissue sarcomas," was featured in The Lancet Oncology in its July 2014 issue (Volume 15, Issue 8) in a review of the major presentations from the 2014 American Society of Clinical Oncology (ASCO) Annual Meeting.

Upcoming Milestones

• Announce OS results from the ongoing global Phase 2b clinical trial of aldoxorubicin as a first-line treatment for STS by the end of 2014 if the expected number of events occur
• Announce preliminary results from the ongoing Phase 2 clinical trial of aldoxorubicin in Kaposi's Sarcoma in the second quarter of 2015
• Expand the oncology pipeline by developing a drug candidate that utilizes novel linker technologies that couple chemotherapeutic agents and proteins either inside the body or externally, and then concentrate drug in tumors in the second half of 2015
• Announce preliminary results from the ongoing Phase 2 clinical trial of aldoxorubicin in GBM in 2015
• Complete enrollment in the ongoing pivotal global Phase 3 clinical trial of aldoxorubicin as a second-line treatment for STS by year-end 2015, with PFS data announced in mid-2016. Subject to FDA approval, the Company projects market launch in 2017
• Complete enrollment in the ongoing Phase 1b clinical trial with combination of aldoxorubicin and ifosfamide/mesna as first-line treatment for advanced sarcomas by the third quarter of 2015 and initiate a Phase 2 study with that combination
• Complete enrollment in the ongoing Phase 1b clinical trial with combination of aldoxorubicin and gemcitabine in metastatic solid tumors by the third quarter of 2015 and initiate a Phase 2 study with that combination
• Complete enrollment in the ongoing Phase 2b clinical trial of aldoxorubicin in relapsed/refractory small cell lung cancer in 2015, with PFS data expected by mid-2016

Third Quarter 2014 Financial Results

CytRx reported cash, cash equivalents and short-term investments of $90.0 million as of September 30, 2014.

Net loss for the three months ended September 30, 2014 was $5.6 million, or $0.10 per share, compared with a net loss of $10.0 million, or $0.33 per share, for the three months ended September 30, 2013. The Company recorded a gain on warrant derivative liability of $7.3 million, as compared to a loss on warrant derivative liability of $4.0 million in the comparative 2013 period, for a difference of $11.3 million. This was partially offset by an increase in our research and development expenditures of $6.6 million.

Research and development (R&D) expenses were $10.6 million for the third quarter of 2014, and included development expenses of $9.1 million for aldoxorubicin. The remaining $1.5 million of R&D expenses were primarily related to research and development support costs. R&D costs were $4.0 million for the third quarter of 2013.

General and administrative (G&A) expenses were $2.4 million for the third quarter of 2014, compared with $2.0 million for the third quarter of 2013. G&A expenses for the third quarter of 2014 included non-cash stock-compensation expense of $0.4 million, compared to $0.2 million for the same period in 2013.