Getting renal with oncology markers
SAN DIEGO—Complementing and enhancing its franchise in tumor marker assets for hematological cancers, TrovaGene Inc., a developer of trans-renal molecular diagnostics, has obtained an exclusive worldwide license to mutations of the SF3B1 splicing factor. These mutations have been shown to be associated with disease progression and chemotherapy response in patients suffering from chronic lymphocytic leukemia (CLL).
TrovaGene has been building a franchise of proprietary markers in hematological oncology, including mutations in the nucleophosmin gene (NPM1), which are today widely used in the diagnosis of acute myeloid leukemia (AML), and the BRAF V600E mutation for diagnostic use in hairy cell leukemia.
"We believe that SF3B1 mutations have the potential to become key components of standard diagnostic panels, with clinical utility in the management of patients suffering from CLL," noted Antonius Schuh, TrovaGene's CEO, in the news release about the license. "We plan to offer laboratory-developed tests to detect SF3B1 mutations and to identify opportunities for the development of in-vitro diagnostic products incorporating our proprietary markers."
TrovaGene has what it maintains is "a dominant patent position as relates to transrenal molecular testing," and this latest license, Schuh tells ddn, is an important addition to making the use of urine-based tests more common in dealing with cancer, as opposed to blood tests.
TrovaGene has U.S. and European patent applications and issued patents that cover testing for HPV and other infectious diseases, cancer, transplantation, prenatal and genetic testing. In addition, it owns worldwide rights to NPM1, which is used as an informative biomarker for AML.
TrovaGene touts very highly tests that look for cell-free nucleic fragments that pass through the kidney and are detectable in the urine, because targeting transrenal markers will allow TrovaGene and its partners to develop genetic tests that rely on easy-to-obtain urine samples rather than more invasive methods like bone marrow biopsies or even traditional blood testing.
"Obviously, there is a convenience factor, since it's easier to get urine samples than it is to even get blood samples," Schuh says. "But also, when you look at tumor-specific mutations, these may be rare events that you might see only once in a milliliter of blood. So the value proposition is that you can obtain virtually limitless large volumes of urine effectively as often as you want, in a way you never could with blood or plasma samples. We're offering not just value or convenience but also enabling people to address important health problems and advance personalized medicine."
Schuh also notes that moving forward with its trans-renal molecular diagnostics products is helped by the recent acquisition of a high-complexity CLIA lab in San Diego, which, he tells ddn, "means there is a commercial platform from which we can roll out many types of tests under CLIA guidelines."
TrovaGene also noted in its announcement of the licensing coup that research results suggest that SF3B1 mutations represent important incremental diagnostic markers beyond TP53 disruptions and NOTCH1 mutations in CLL patients, and may also provide a therapeutic target for SF3B1 inhibitors, which are currently in preclinical development.
TrovaGene notes that such finding were recently published in the Dec. 22 issue of Blood, the journal of the American Society of Hematology, in an article titled "Mutations of the SF3B1 splicing factor in chronic lymphocytic leukemia: association with progression and fludarabine-refractoriness" by Davide Rossi and colleagues.
Rossi and Gianluca Gaidano at the Amedeo Avogadro University in Novara, Italy, led the research team, in collaboration with their colleague Roberto Foa at the Sapienza University in Rome, which discovered the SF3B1 mutations.
"Since CLL is the most frequent type of leukemia in adults, the discovery of SF3B1 mutations in this disease will affect a large number of patients worldwide," Gaidano says. "In particular, SF3B1 mutations may contribute to the early identification of patients destined to fail standard treatment, who instead might benefit from more aggressive therapeutic strategies. Future goals in SF3B1 research include the development of a robust molecular assay for diagnosis and the exploitation of SF3B1 as a therapeutic target for this leukemia."