We wrote earlier about this very promising biotech company (you might want to revisit that to see the big potential), and today, they’re off to the races. Here is the news. This is a very promising company..
Sequenom, Inc. (NASDAQ:SQNM – News), a leading provider of genetic-analysis and molecular diagnostic solutions, announced additional, positive results from screening studies using the Company’s noninvasive circulating cell-free fetal (ccff) nucleic acid SEQureDx™ Technology, which enables the detection of fetal aneuploidy, including Down syndrome from maternal blood, at its Analyst Briefing in New York City. Among the data presented, Sequenom’s test demonstrated complete concordance with clinical results (no false positives and no false negatives) in both first and second trimester samples (over 200 samples announced today and in excess of 400 prospective samples to-date). Sequenom executives were joined by a panel of leading scientists and clinicians to discuss these study results and updates in the development of noninvasive prenatal diagnostics.
“These data expand upon the data we announced in June and underscore the potential for our SEQureDx Technology to transform current clinical practice for prenatal diagnostics as a primary screening tool for Trisomy 21. Furthermore, these results support the potential for our test to be used in the first trimester,” said Harry Stylli, Ph.D., Sequenom’s President and Chief Executive Officer. “In addition, our announcement earlier today regarding our acquisition of the Center for Molecular Medicine, a CLIA-certified molecular diagnostics laboratory, and our partnership with Spectrum Health and the Van Andel Research Institute, provides us with important infrastructure and commercialization control. We are delighted with our progress in bringing to market an important, noninvasive screening test for Down syndrome, as well as a broader menu of molecular diagnostic tests. These results are very promising, and we look forward to continuing the clinical development and validation progress to launch in the first half of 2009.”
Elizabeth Dragon, Ph.D., Senior Vice President of Research and Development at Sequenom, presented data from blinded studies performed at Sequenom involving 219 new clinical samples collected prospectively, showing that its proprietary test for Down syndrome correctly identified 100% of all Down syndrome samples (i.e. sensitivity or detection rate), without any false-positive outcomes (i.e. specificity). The SEQureDx prototype test also demonstrated its ability to correctly identify a Down syndrome positive sample in the first trimester, confirmed by chorionic villus sampling (CVS), a current testing standard that requires the harvesting of placental tissue cells.
Sequenom indicated that with the addition of new SNPs in PLAC4 and a recently discovered gene, the SEQureDx Trisomy 21 test should increase its coverage from 93% to greater than 95% in the US population. The Company has also identified novel markers for Trisomy 18 that have passed its initial selection criteria, and other chromosomes, and intends to develop these markers into new tests.
The Company expects to continue its current development activities through the end of 2008, at which time the Company will initiate a multi-site 3,000 to 5,000-sample laboratory developed test (LDT) validation study, which is expected to be completed and submitted for publication at the time of the anticipated commercial launch in June 2009. To facilitate the LDT validation study, Sequenom also indicated that the company will be collaborating with new clinical partners who perform in excess of 12,000 amniocenteses and 3,000 CVS per year. In addition, Sequenom announced sponsorship of the RNA Noninvasive Aneuploidies (“RNA”) study, a landmark, multi-center, prospective study involving up to 10,000 samples from first and second trimester pregnancies using the SEQureDx technology, managed and analyzed by an independent third-party.
During the Analyst Briefing, management also highlighted key upcoming milestones for the prenatal diagnostics business, including:
* Confirm 10-week or earlier gestational age testing
* Evaluate integration of T18 (Edward’s syndrome) assay into the first generation test
* Initiate T21 LDT clinical validation study evaluating 3,000 to 5,000 samples
* Complete T21 testing of up to 800 additional high prevalence specimens by year-end 2008
* Initiate “RNA” multi-center study involving 10,000 high prevalence patient samples
* Submission of key data for publication
* Commercial launch of T21 test in first half of 2009
Sequenom’s analyst briefing included the following speakers and topics:
* Harry Stylli, Ph.D., Chief Executive Officer, Sequenom Business Overview and Update
* Charles R. Cantor, Ph.D., Chief Scientific Officer, New applications of the Sequenom Platform
* Yury Khudyakov, Ph.D., Chief, Molecular Epidemiology and Bioinformatics Laboratory, Division of Viral Hepatitis, Centers for Disease Control and Prevention, Novel Molecular Technologies for Public Health
* Betty Dragon, Ph.D., Senior Vice President, R&D, Noninvasive Prenatal Detection of Genetic Abnormalities Using SEQure Dx™ Technology
* Daniel H. Farkas, PhD, HCLD, FACB, Executive Director, Center for Molecular Medicine, Real-world Genetic Testing: Translating Science Into Routine Clinical Tests
* Gary S. Riordan, Vice President, Regulatory Affairs and Quality, FDA Regulatory Environment
* Jacob A. Canick, Ph.D., Professor of Pathology and Laboratory Medicine, Brown University Medical School, Design And Implementation of a Landmark, Multicenter, Prospective Clinical Study To Validate The Safe And Accurate Detection Of Down Syndrome Using SEQure Dx™ Technology
* Allan T. Bombard, M.D., FACOG, FACS, FACMG, reproductive geneticist, Chief Medical Officer, Sharp Mary Birch Hospital, Are We Facing a Revolution in Noninvasive Prenatal Genetic Diagnostics?
A recording of the webcast of the event will be available on the Company Web site at www.sequenom.com until October 7, 2008.
Sequenom’s Proprietary Noninvasive Prenatal Diagnostics
Sequenom’s commercial opportunities in prenatal diagnostics are built upon its SEQureDx technologies and are enabled by the pioneering inventions and associated intellectual property rights that it has exclusively licensed from Isis Innovation Ltd., the technology transfer company of the University of Oxford, as well as The Chinese University of Hong Kong. Sequenom’s portfolio of noninvasive prenatal diagnostic patent rights and patent applications is platform-independent, includes genetic-analysis methods using circulating cell-free fetal nucleic acids from maternal serum, plasma or whole blood, and also includes a portfolio of methylation and nucleic-acid markers. Sequenom holds exclusive rights in territories including the United States, Europe, Australia, Canada, Japan and Hong Kong. Sequenom is actively expanding its intellectual property position with new technology and new territories. Because Sequenom’s license rights are platform-independent, the rights provide exclusivity (with the narrow exception in Europe for RT-PCR-based Rhesus D tests) for development and commercialization of noninvasive prenatal screens and tests on any platform and are not limited to the Company’s MassARRAY platform.
You might want to wait until the dust has settled a bit, but we have a feeling this thing wants to move significantly higher…