NEW YORK—Fortress Biotech, Inc. announced today that Oncogenuity, Inc., a new partner company, has entered into an exclusive worldwide licensing agreement with Columbia University to develop novel oligonucleotides for the treatment of genetically driven cancers.
“We are excited to work with the excellent scientists and physicians at Columbia University again,” said Lindsay A. Rosenwald, M.D., chairman, president and chief executive officer of Fortress Biotech. “Our last joint effort with Columbia University led to the formation of our partner company, Caelum Biosciences, Inc. 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,” Rosenwald continued. “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.”
The technology comes from the Columbia University labs of Gary Schwartz, M.D., division chief of Hematology/Oncology, and Jeffrey Rothman, M.D., Ph.D., assistant professor of Medicine. The proprietary platform produces oligomers, now known as ONCOlogues, that are reportedly capable of binding gene sequences a thousand times more effectively than complementary native DNA.
ONCOlogues are sensitive to a single base pair mismatch, resistant to degradation and use a proprietary delivery sequence to enter cells. The selectivity of the ONCOlogues enables Oncogenuity to target genetically driven cancers caused by mutations without impacting wild-type DNA sequences. This could potentially limit off-target toxicity. It also allows for targeting mutations that have historically been considered undruggable.
Oncogenuity has established proof-of-concept in a preclinical setting for various cancer types. The company’s most advanced program is targeting the KRAS mutation G12D, which plays a significant role in various cancer types with substantial unmet need — including pancreatic and colorectal. Oncogenuity plans to evaluate other mutations simultaneously, and anticipates additional data publications within the next year.
Oncogenuity is also 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 they believe could help combat COVID-19 and provide proof-of-concept as a coronavirus treatment. 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.
“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,” noted scientific co-founder Jeffrey Rothman, M.D., Ph.D. “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.”