Onconova Therapeutics, Inc. today announced promising results from two
clinical trials in patients with advanced Myelodysplastic Syndrome (MDS)
treated with ON 01910.Na. These trials are being conducted at the
National Heart, Lung, and Blood Institute (NHLBI) in Bethesda, MD, and
St. Vincent’s Comprehensive Cancer Center in New York. In these studies,
treatment with ON 01910.Na resulted in significant decrease in blast
count (cancer cells) in bone marrow without significant toxicity in
these high risk MDS patients. After treatment, MDS patients with
cytogenetic abnormalities showed fewer abnormal (aneuploid) cells, and
many patients showed improved normal blood cell counts, (i.e., had an
improvement in cytopenias). These findings were discussed in an oral
presentation (December 6th) and a poster (December 7th)
at the Annual Meeting of the American Society of Hematology (ASH) in New
Orleans.
ON 01910.Na is a novel, targeted small molecule anti-cancer compound in
Phase I and II clinical trials for MDS and solid tumors at several major
centers in the U.S. and abroad. More than 190 patients have been treated
in these clinical trials.
The positive clinical trial data were presented by Elaine Sloand, M.D.,
lead investigator for the trial at the National Heart, Lung, and Blood
Institute. These studies were conducted, in part, with a “bench to
bedside” translational award from the NHLBI and exemplify a mechanism
and biomarker aided approach to development of new anti-cancer drugs.
“These findings are very promising and reaffirm our efforts to
investigate ON 01910.Na for high risk MDS, which is difficult to treat,
and for which few therapeutic options are available,” said Azra Raza,
M.D., lead investigator for the trial at St. Vincent’s. Dr. Raza’s
studies were also featured in a poster presentation.
“We are very pleased to see progress with our lead compound, for which a
broad U.S. patent was issued in October,” said Mr. Michael Hoffman,
Chairman of Onconova. “These encouraging results have led to the
initiation of MDS trials at three additional sites, Mt. Sinai Medical
Center, Stanford University Cancer Center and Moffitt Cancer Center. We
anticipate additional clinical findings from ongoing trials of ON
01910.Na in single agent and combination therapy for solid tumor
patients.”
ON 01910.Na was recently designated an “orphan drug” for treatment of
MDS by the Food and Drug Administration. If ON 01910.Na is approved for
the treatment of patients with high-risk MDS in the U.S., orphan drug
designation could provide Onconova with potential market exclusivity for
seven years. In addition, a drug candidate designated by the FDA as an
orphan-drug product may qualify for subsidies on regulatory fees and tax
incentives and may be eligible for research grant funding to assist in
further clinical development.
Mechanism of Action and Biomarker
Findings Presented at ASH