Fortress Biotech, Inc. (NASDAQ:FBIO) (“Fortress”), an innovative biopharmaceutical company, today announced that Oncogenuity, Inc. (“Oncogenuity”), a new Fortress partner company, has entered into an exclusive worldwide licensing agreement with Columbia University to develop novel oligonucleotides for the treatment of genetically driven cancers. The proprietary platform produces oligomers, now known as “ONCOlogues,” that are capable of binding gene sequences 1,000 times more effectively than complementary native DNA. The technology comes from the labs of Gary Schwartz, M.D., Division Chief, Hematology/Oncology, and Jeffrey Rothman, M.D., Ph.D., Assistant Professor of Medicine.
ONCOlogues are sensitive to a single base pair mismatch, resistant to degradation and use a proprietary delivery sequence to enter cells. ONCOlogues’ selectivity enables Oncogenuity to target genetically driven cancers caused by mutations without impacting wild-type (“WT”) DNA sequences, potentially limiting off-target toxicity. In addition, this allows ONCOlogues to target mutations that have historically been considered “un-druggable.”
Oncogenuity has established proof-of-concept in a pre-clinical setting for various cancer types. The company’s most advanced program is targeting the KRAS mutation G12D, which was previously considered un-druggable and plays a significant role in various cancer types with substantial unmet need, including pancreatic and colorectal. Given the platform’s ability to target any mutation, Oncogenuity will continue to evaluate other mutations simultaneously. The company anticipates additional data publications in the coming 12 months.
Additionally, Oncogenuity is exploring the platform’s potential to treat coronaviruses. Coronaviruses have single-stranded RNA genomes, making them strong targets for ONCOlogues. The company is studying replacement sequences, which could help combat COVID-19 and provide proof-of-concept as a treatment for coronaviruses. These ongoing experiments would validate ONCOlogues as a possible treatment for COVID-19, as well as potentially expedite the discovery of treatments for future coronavirus outbreaks.
Lindsay A. Rosenwald, M.D., Fortress’ Chairman, President and Chief Executive Officer, said, “We are excited to work with the excellent scientists and physicians at Columbia University again. Our last joint effort with Columbia University led to the formation of our partner company, Caelum Biosciences, Inc. (“Caelum”). Since formation, Caelum has raised approximately $60 million in development funding from a number of sources, with additional amounts available upon the satisfaction of certain milestones and will be initiating two registration clinical trials in the next several weeks. Building upon our success with Caelum, we are grateful to Columbia University for entrusting us to develop this highly innovative technology using oligonucleotides to target genetically driven cancers and coronaviruses. Using a targeted genetic approach to treat cancer has become essential to limiting toxicity and treating patients effectively. This technology has the potential to target mutations that have previously been considered un-druggable. Oncogenuity will aggressively pursue the development of ONCOlogues to ultimately provide patients with new, safe and effective treatment options.”
Scientific Co-Founder Jeffrey Rothman, M.D., Ph.D., said, “Through rigorous statistical, mechanical and molecular modeling, combined with gene sequence data, we are able to create sequence-specific, targeted therapeutics against oncogenes, which are the cause of and specific to tumor cells. Until now, achieving this goal had been considered nearly impossible. However, with these novel design features, we now have the ability to target cancer while potentially avoiding side effects, which are the main cause of dose-limitation, by design. There is much potential because we are able to target multiple genes and therefore, multiple cancers. Moreover, due to their single-strand format, application toward viral targets such as in COVID are even more facile given their easier accessibility. We are excited and determined to pursue this endeavor with Fortress Biotech and very much welcome their continued support.”