TransMolecular Receives Orphan Drug Designation for Non-radiolabeled TM601 for the Treatment of Malignant Glioma
CAMBRIDGE, Mass.--(Business Wire)--TransMolecular, Inc., a biotechnology company focused on targeted
therapies for cancer, today announced that the FDA has granted Orphan
Drug Designation for the non-radiolabeled version of its anti-cancer
compound TM601, which is currently entering clinical trials for the
treatment of malignant glioma. The company had previously received
Orphan designation for the radiolabeled version, 131I-TM601, which
recently completed patient enrollment in a Phase 2 clinical trial in
glioma and a Phase 1 trial in multiple tumor types.
"We are pleased to receive orphan drug status for TM601 in
malignant glioma," stated Michael Egan, President and Chief Executive
Officer of TransMolecular. "The TM601 platform has performed very well
in recent Phase 1 and 2 clinical trials, showing specific tumor
targeting in both primary and metastatic disease of multiple tumor
types. This adds to a strong clinical rationale supporting its
therapeutic promise, and we look forward to initiating Phase 1
clinical trials with the non-radiolabeled form of this drug candidate
in malignant glioma. This designation is part of TransMolecular's
strategy to advance this program so that patients with this
poor-prognosis disease may eventually have a new treatment option
available to them."
The FDA grants Orphan Drug Designation to promising products that
address rare diseases affecting fewer than 200,000 Americans annually.
If non-radiolabeled TM601 receives FDA approval for malignant glioma,
this designation will entitle TransMolecular to exclusive marketing
rights for the compound for the treatment of malignant glioma for
seven years following the NDA approval. Orphan Drug Designation
provides financial and regulatory incentives for companies pursuing
less common diseases.
About TM601
TM601 is a synthetic version of chlorotoxin, a naturally occurring
peptide derived from scorpion venom, which is highly specific in
targeting both primary tumors and metastases. TM601 targets and binds
to receptors expressed on tumor cells but not on normal, healthy
cells. As TM601 binds primarily with the tumor cell receptor sites, it
also delivers a targeted dose of radiation, killing the tumor cell
without affecting nearby healthy cells. The data obtained from
preclinical and clinical data also suggest that native TM601 may
affect a tumor's ability to grow and spread without added radiation,
so the therapeutic potential as a non-radiolabeled peptide is also
being explored. The Company's robust development plan for TM601
reflects its broad platform potential for multiple applications in
cancer. The FDA has granted it orphan drug status for patients with
high-grade and malignant glioma, as well as a Fast Track designation.
About Glioma
In the U.S., an estimated 20,500 new cases of brain and/or nervous
system tumors were expected to be diagnosed in 2007. Of primary brain
tumors, malignant glioma is the most common tumor type and is the
second most common cause of cancer-related mortality in the 15-to-44
age group. In patients with grade III anaplastic glioma, the median
survival is three-to-five years; however, median survival in patients
with grade IV glioma or glioblastoma multiforme is less than a year.
Despite over twenty-five years of intensive research and a variety of
chemotherapy, radiotherapy, and surgical approaches, the prognosis for
these tumors has not changed significantly. Primary brain tumors
remain one of the most aggressive and difficult-to-treat cancers.
About TransMolecular, Inc.
TransMolecular, Inc. is a privately held, venture capital backed
biotechnology company committed to discovering, developing and
commercializing novel and proprietary products to diagnose and treat
cancers that have inadequate treatment alternatives. TransMolecular's
product pipeline is based on a protein platform that employs a
therapeutically active polypeptide derived from scorpion venom. The
company is currently exploring the use of this platform for broad
applications to diagnose and treat cancers and other human diseases.
More information can be found at www.transmolecular.com.
This press release contains forward-looking statements. The
Company wishes to caution the reader of this press release that actual
results may differ from those discussed in the forward-looking
statements and may be adversely affected by, among other things, risks
associated with litigation, clinical trials, the regulatory approval
process, reimbursement policies, commercialization of new
technologies, intellectual property, and other factors.
TransMolecular
Michael Egan, 617-995-3050
President and CEO
or
Media
MacDougall Biomedical Communications
Jennifer Greenleaf, 781-235-3060
Copyright Business Wire 2008
