Nearly four months after teaming up with Celgene, Presage Biosciences has snagged another major cancer player as a partner: Bristol-Myers Squibb. The Big Pharma will use Presage’s microdosing technology to assess early-stage cancer targets in phase 0—a stage in drug development designed to bridge preclinical and clinical work.
“A well-recognized issue in cancer drug development is that, based on recent statistics, less than 10% of drugs that enter phase 1 actually benefit patients enough to receive FDA approval and enter the market,” Presage CEO Rich Klinghoffer, Ph.D., told FierceBiotech. “Looking at that suggests that the drugs we are investigating in the lab and look quite outstanding in a lab setting … are not panning out. It points to a fundamental flaw in the way we are currently assessing and developing early-stage oncology drugs.”
One solution is to do a phase 0 study, an idea the FDA introduced in 2006 to address the failure of animal models to accurately predict how drugs will work in humans. This approach involves injecting people with microdoses of the experimental drug—about 1/100 of the anticipated effective dose, Klinghoffer said. But administering tiny doses systemically can make it difficult to measure efficacy.
“My understanding, based on talking to a number of folks in the biopharma industry is that (a typical phase 0) didn’t provide enough value to insert it into the drug development pipeline,” Klinghoffer said.
Presage’s CIVO platform is designed to deliver up to eight different microdoses of drugs or drug combinations directly into the tumor. Each drug or combination is injected alongside fluorescent tracking microspheres, which allow investigators to track each dose. This way, the tiny doses don't disperse into the body, and investigators know exactly where in the tumor each drug was injected and where it went after that.
It’s an efficient way to run combination studies—rather than testing a two-drug cocktail in one patient, one half of it in another patient and the other half in a third patient, CIVO allows for the simultaneous microdosing of all three into a single tumor, Klinghoffer said. Investigators can then gauge how the combo fared against its components as single agents in different parts of the tumor.
Presage and Bristol-Myers Squibb kept the details of their partnership under wraps.
"At Bristol-Myers Squibb we are seeking a more precise understanding of how treatments impact each patient to help inform tailored therapeutic strategies that will offer the greatest possible benefit,” said Jonathan Leith, head of clinical mechanisms at Bristol-Myers Squibb, in a statement. “This collaboration may provide important insights about how our compounds work mechanistically in combination studies and help us bring forward more effective options for patients with cancer.”
This is the third time Presage has partnered to use CIVO to study responses in tumor cells and the tumor microenvironment to new drugs in a phase 0 clinical trial. Celgene, which chipped into the company's $6 million series D last summer, was the first. And Presage can’t divulge which company was second—though that might change over the coming months.
As for the future, Presage is "absolutely" looking for more partners.
"We're gaining traction in the biopharma industry, as we always hoped we would," Klinghoffer said. "We have another marquee cancer drug development firm that understands that we have to start doing something differently in terms of evaluating early-stage assets and combinations because the conventional approach simply isn't working."