Avila Therapeutics can’t be accused of thinking too small. This Waltham, MA-based startup is on a quest to show it has the scientific chops to build a better drug than one of Boston’s anchor biotech companies—Vertex Pharmaceuticals.
So this is shaping up to be a big year for Avila (AH-vill-uh), as it plans to start clinical trials of a drug for hepatitis C, and another with potential for certain cancers and autoimmune diseases. The company raised $30 million last July, recruited an experienced biotech dealmaker as CEO, and has laid out some ambitious goals to put its money and people to work.
For those new to the Avila story, it’s built on chemistry. The company is developing conventional small-molecule oral pills with a special feature. The Avila drugs form covalent bonds with their targets on cells. These bonds are supposed to completely, and irreversibly, shut down the biological activity of the intended protein target they hit on cells. That’s different from conventional therapies that connect with their targets via ionic or van der Waal bonds, which puts them in a state of “dynamic equilibrium” in which they are bouncing on and off the target, says CEO Katrine Bosley.
Why does that matter? By completely shutting down the target, Avila thinks it has found a way to improve upon the trailblazing work of Vertex in hepatitis C, and it might be able to nail other targets pharma companies have long pursued, but couldn’t effectively block.
“It’s getting the attention of pharma companies. Pharma companies are always looking for new innovation from beyond what they do within their four walls, and covalent drugs haven’t been much explored in the industry,” Bosley says. “As we develop a data set that says we can develop very specific covalent drugs, it’s intrigued a lot of people. Scientists are scientists. If you can get a new result that can’t be achieved another way, they want to talk about that.”
I pressed Bosley for a bit more explanation of how its lead drug is differentiated from Vertex’s telaprevir. That drug, a protease inhibitor, has helped Vertex create an $8 billion stock market valuation, largely because clinical trials have shown it can basically double the traditional cure rates for patients with hepatitis C, while shortening the usual year-long treatment regimen by half. Analysts predict Vertex could exceed $2 billion in U.S. sales alone after a couple years on the market, and the worldwide potential is vast with an estimated 170 million people infected.
Bosley didn’t want to sound dismissive of what Vertex has done. “Obviously everybody is excited about Vertex’s telaprevir, it’s a great drug, they’ve done a beautiful job advancing the molecule and developing the clinical paradigm in treating hepatitis C,” Bosley says.
But Bosley clearly sees room for improvement, and Avila thinks it has found a way. One of the challenges with viruses is that they mutate, essentially switching
