FDA Grants Orphan Drug Status to Aldoxorubicin

CytRx receives multiple FDA Orphan Drug Designations for aldoxorubicin for the treatment of glioblastoma, small cell lung cancer and ovarian cancer

/ Author:  / Reviewed by: Joseph V. Madia, MD Beth Bolt, RPh

CytRx Corporation (NASDAQ: CYTR), a biopharmaceutical research and development company specializing in oncology, today announced that the U.S. Food and Drug Administration (FDA) has granted multiple Orphan Drug Designations for the Company's lead drug candidate, aldoxorubicin.

Aldoxorubicin was granted the orphan drug status in three indications: glioblastoma multiforme (GBM), small cell lung cancer and ovarian cancer. Aldoxorubicin is CytRx's modified version of the widely-used chemotherapeutic agent, doxorubicin.

"The FDA's decision to grant Orphan Drug designation for aldoxorubicin in these three new indications is a key milestone for the aldoxorubicin clinical development program and a clear reflection of the high unmet medical need for new treatments in these cancer types," said Steven A. Kriegsman, CytRx President and Chief Executive Officer.  "These designations are also a testament to the team's regulatory and development expertise and part of our core strategy to bring aldoxorubicin to patients worldwide as rapidly as possible.  We look forward to reporting top-line results from both our Phase 2 GBM trial and our Phase 2 Kaposi's sarcoma trial in the first half of 2015."

In the U.S., under the Orphan Drug Act, the FDA's Office of Orphan Products Development (OOPD) grants orphan drug status to a drug intended to treat a rare disease or condition, which is generally a disease that affects fewer than 200,000 individuals in the country.  Upon approval, if received, the designation provides aldoxorubicin with certain benefits, including seven years of U.S. market exclusivity in the specified indications if the sponsor complies with certain FDA requirements.  Additional incentives for the sponsor include tax credits related to qualified clinical trial expenses and a possible exemption from FDA application fees.

Aldoxorubicin is currently being studied in a pivotal global Phase 3 clinical trial evaluating the efficacy and safety of aldoxorubicin as a second-line treatment for patients with soft tissue sarcoma (STS) under a Special Protocol Assessment with the FDA.  CytRx is also evaluating aldoxorubicin in two Phase 2 clinical trials, one in patients with late-stage GBM and the other in HIV-related Kaposi's sarcoma. The Company expects to start a global phase 2b trial in patients with relapsed small cell lung cancer this month and is undertaking a phase 1b combination study of aldoxorubicin plus gemcitabine as a potential precursor to a trial in relapsed ovarian cancer.

About SCLC

An estimated 1.6 million new cases of lung cancer are diagnosed worldwide each year.  In the Western world, approximately 13-15% of cases are SCLC, a deadly form of lung cancer associated with tobacco use.  The five year survival rate is less than 7%, in part because an estimated 70% of patients have extensive disease at diagnosis.  According to the National Cancer Institute, more than 30,000 new cases will be diagnosed in the USA in 2014.  The estimated 2014 SCLC incidences for Europe and Asia are over 58,000 and 136,000, respectively. 

About Glioblastoma Multiforme (Brain Cancer)

Glioblastoma is the most common and most malignant brain tumor in adults and afflicts more than 12,000 new patients in the U.S. annually. Despite surgical resection, radiotherapy and chemotherapy, the median survival after diagnosis is approximately 12 to 14 months. Limited efficacy of chemotherapeutic agents has been attributed to several contributing factors including insufficient drug delivery to the tumor site through the blood-brain barrier.

About Ovarian Cancer

Ovarian cancer is the ninth most common cancer and accounts for 3% of all cancers in women. Ovarian cancer ranks as the fifth most frequent cause of death by cancer among women, accounting for more deaths than any other cancer of the female reproductive system. One out of every 71 women is at risk of developing ovarian cancer, and one out of every 95 women dies from ovarian cancer.  In the U.S. alone, there will be approximately 22,000 new cases of ovarian cancer diagnosed in 2014.  Worldwide, ovarian cancer is diagnosed annually in nearly 250,000 women, and is responsible for 140,000 deaths each year.

About Aldoxorubicin

The widely used chemotherapeutic agent doxorubicin is delivered systemically and is highly toxic, which limits its dose to a level below its maximum therapeutic benefit. Doxorubicin also is associated with many side effects, especially the potential for damage to heart muscle at cumulative doses greater than 450 mg/m2. Aldoxorubicin combines doxorubicin with a novel single-molecule linker that binds directly and specifically to circulating albumin, the most plentiful protein in the bloodstream. Protein-hungry tumors concentrate albumin, thus increasing the delivery of the linker molecule with the attached doxorubicin to tumor sites. In the acidic environment of the tumor, but not the neutral environment of healthy tissues, doxorubicin is released. This allows for greater doses (3 ½ to 4 times) of doxorubicin to be administered while reducing its toxic side effects. In studies thus far there has been no evidence of clinically significant effects of aldoxorubicin on heart muscle, even at cumulative doses of drug well in excess of 2,000 mg/m2.

About CytRx Corporation

CytRx Corporation is a biopharmaceutical research and development company specializing in oncology. CytRx currently is focused on the clinical development of aldoxorubicin (formerly known as INNO-206), its improved version of the widely used chemotherapeutic agent doxorubicin. CytRx has initiated under a special protocol assessment a pivotal Phase 3 global trial with aldoxorubicin as a therapy for patients with soft tissue sarcomas whose tumors have progressed following treatment with chemotherapy, and recently announced that it has received approval from the FDA to continue dosing patients with aldoxorubicin until disease progression in that clinical trial. CytRx has initiated a Phase 2 clinical trial in HIV-related Kaposi's sarcoma, a Phase 2 clinical trial in patients with late-stage glioblastoma (brain cancer), and a Phase 1b trial in combination with ifosfamide in patients with soft tissue sarcoma. CytRx plans to initiate a Phase 2b clinical trial in patients with SCLC this month.  CytRx has completed a global Phase 2b clinical trial with aldoxorubicin as a first-line therapy for STS, a Phase 1b/2 clinical trial primarily in the same indication, a Phase 1b clinical trial of aldoxorubicin in combination with doxorubicin in patients with advanced solid tumors and a Phase 1b pharmacokinetics clinical trial in patients with metastatic solid tumors. CytRx plans to expand its pipeline of oncology candidates at its laboratory facilities in Freiburg, Germany, based on novel linker technologies that can be utilized with multiple chemotherapeutic agents and may allow for greater concentration of drug at tumor sites. For more information about CytRx Corporation, visit www.cytrx.com.

Review Date: 
September 29, 2014