TORONTO, ONTARIO--(Marketwired - Oct 31, 2013) - Stem Cell Therapeutics Corp. (TSX VENTURE:SSS)(SCTPF), an immuno-oncology company developing cancer stem cell-related therapeutics, today announced that it has been granted Orphan Drug designation by the U.S. Food and Drug Administration (FDA) for the use of tigecycline in the treatment of acute myeloid leukemia (AML).
Orphan Drug designation is granted to therapeutics treating rare diseases affecting less than 200,000 people in the U.S. The designation entitles the sponsor to seven years of market exclusivity as well as opportunities for additional funding and expert protocol assistance.
"Orphan Drug status significantly enhances the commercial potential of tigecycline in AML, a disease which is notoriously difficult to treat," said the company's Vice President, Drug Development, Dr. Penka Petrova. "Through its unique mechanism of action and synergy with existing AML therapies, tigecycline has the potential to positively impact the standard of care in this disease."
The company's program is based on Dr. Aaron Schimmer's published findings that tigecycline, an FDA-approved antibiotic, selectively targets leukemia cells and leukemic stem cells by inhibiting mitochondrial protein synthesis and thus shutting down the cells' energy supply. A Phase I Canadian and U.S. multicenter dose-escalation clinical trial in patients with relapsed or refractory AML is nearing completion.
"The Orphan Drug designation covers tigecycline as an active ingredient and is independent of the drug formulation," added Dr. Bob Uger, the company's Chief Scientific Officer. "We are investigating improved formulations of tigecycline, which we believe will be critical for the commercialization of this technology."
About Cancer Stem Cells:
The cancer stem cell (CSC) concept postulates that the growth of tumors is driven by a rare population of dedicated cells that have stem cell-like properties, including self- renewal. While the bulk of a tumor consists of rapidly proliferating cells and differentiated cells, neither of which is capable of self-renewal, a small population of CSCs provides for long-term maintenance of the cancer. Although the CSC concept was first postulated in the 1960s, it wasn't until 1994 that proof of their existence was demonstrated, when Dr. John Dick and colleagues in Toronto isolated CSCs (known as leukemic stem cells, or LSCs) from bulk acute myeloid leukemia cells. More recently, CSCs have been identified in many other human malignancies, including solid tumors such as bladder, brain, breast, colon, ovarian and prostate cancers. There is accumulating evidence that CSCs are resistant to conventional chemotherapies and radiation. Thus, CSCs are thought to be responsible for a phenomenon well known to oncologists: most patients will experience an initial response to conventional chemotherapies but will ultimately relapse. To cure cancer CSCs need to be destroyed, but the current armament of therapies is poorly equipped to do so.
About Stem Cell Therapeutics:
Stem Cell Therapeutics Corp. (SCT) is an immuno-oncology company advancing cancer stem cell discoveries into novel and innovative cancer therapies. Building on over half a century of leading and groundbreaking Canadian stem cell research, the company is supported by established links to a group of prominent Toronto academic research institutes and cancer treatment centers, representing one of the world's most acclaimed cancer research hubs. The Company has two premier preclinical programs, SIRPaFc and a CD200 monoclonal antibody (mAb), which target two key immunoregulatory pathways that tumor cells exploit to evade the host immune system. SIRPaFc is an antibody-like fusion protein that blocks the activity of CD47, a molecule that is upregulated on cancer stem cells in AML and several other tumors. The CD200 mAb is a fully human monoclonal antibody that blocks the activity of CD200, an immunosuppressive molecule that is overexpressed by many hematopoietic and solid tumors. SCT's clinical stage programs include the recently in-licensed program focused on the structure of tigecycline, which is currently being evaluated in a multi-centre Phase I study in patients with acute myeloid leukemia (AML), as well as TTI-1612, a non- cancer stem cell asset that recently completed a 28-patient Phase I trial in interstitial cystitis ("IC") patients. For more information, visit: www.stemcellthera.com.
Caution Regarding Forward-Looking Information:
This press release may contain forward-looking statements, which reflect SCT's current expectation regarding future events. These forward-looking statements involve risks and uncertainties that may cause actual results, events or developments to be materially different from any future results, events or developments expressed or implied by such forward-looking statements. Such factors include changing market conditions; the successful and timely completion of pre-clinical and clinical studies; the establishment of corporate alliances; the impact of competitive products and pricing; new product development risks; uncertainties related to the regulatory approval process or the ability to obtain drug product in sufficient quantity or at standards acceptable to health regulatory authorities to complete clinical trials or to meet commercial demand; and other risks detailed from time to time in SCT's ongoing quarterly and annual reporting. Except as required by applicable securities laws, SCT undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.
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