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how to translate dosing from mice into humans, so Novo plans to do methodical work on getting the chemical formulations, delivery mode, dosing, dose schedules, and biomarker work all in alignment to give a drug its best shot in late-stage development, where the big money gets wagered.
Novo isn’t being too specific yet about what kind of projects it has on the front burner, but von Herrath did say he’s generally excited about therapies that can tamp down an excessive immune response in a local region, like that of the pancreas. The local-acting nature of the drug is critical, he says, because a drug that circulates throughout the bloodstream and tamps down the immune system could have the negative side effect of making people vulnerable to opportunistic infections. For Type 1 diabetes, a chronic disease often diagnosed in children, the safety profile of any new treatment has to squeaky-clean.
Novo has plenty of relationships in the biotech community, and von Herrath got to know quite a few people in industry through about 10 years of consulting while he was in academia. He says he learned a lot about not just the limits of what academia can do for drug development, but also the limits of what venture-backed biotech companies can do. He talked about how many companies with promising concepts for Type 1 diabetes, like ToleRx and Palo Alto, CA-based Bayhill Therapeutics, which hit the wall after struggling to deliver the kind of timely returns their investors wanted.
With oral insulins—an attractive concept that could reduce the need for regular injections—nobody in the pharma industry was able to figure out how to get the right dose into people after some promising animal experiments. The short-term investor mindset forced those programs to move ahead too far too fast, when a methodical approach might have been the best way to go, von Herrath says.
“Oral insulin works well in many animal models for tolerance, and it’s been known since the early 1990s,” von Herrath says. “The way it was translated, nobody knew what dose to use in humans. That’s a common issue. But instead of working the kinks out, and coming up with a rational plan, the first trial was a Hail Mary pass. People thought ‘we better do something rather than nothing.’ That’s a human impulse, I understand. They took a dose for humans that seemed like it wouldn’t do any harm, which is important in type 1. But that’s not necessarily the best dose to get efficacy.”
The need to do those “Hail Mary passes” might sound familiar to anyone working at a venture-backed biotech company that is expected to work wonders on a shoestring budget. Although Novo doesn’t have to worry about running out of money, it’s a Big Pharma company like many others with far-flung research operations, lots of different priorities, and a limited number of R&D successes to brag about. But von Herrath clearly exudes enthusiasm about his opportunity to take drug R&D programs further than he ever could in academia or as a consultant for cash-strapped biotech companies. “It’s something I’ve dreamed about for a long-time,” he says.