Aileron Therapeutics, based in Cambridge, MA, is set to receive up to $1.1 billion worth of Big Pharma affirmation for all its hard work on a unique drug delivery technology after signing a deal with Roche. Roche appears to be impressed with Aileron's "stapled peptides" and its ability to reach inside cells to deliver therapeutic agents without falling apart.
"This is a significant commitment by a very smart pharmaceutical company betting they're finally going to unlock the power of peptides as a superclass of drugs," Joseph A. Yanchik III, Aileron's chief executive, told the New York Times.
Like drugs that use interfering RNA, stapled peptides are a class all by themselves, potentially reaching disease targets that other classes of drugs cannot. Peptides seem like they'd be a natural for drug delivery. The problem, though, is they lack the ability to enter cells and, once they're in, they break apart into fragments. Finding a way to keep peptides stable inside a cell has perplexed researchers for years.
Aileron has ensured that the peptides are folded, then "stapled" into the proper shape to bind to its target. Stapled peptides are produced by connecting, or "stapling," two amino acids by mimicking the structure found at the interface of many protein-protein interactions. The end result, a peptide that will bind to its target long enough to deliver its payload of therapeutics before breaking apart.
Roche likes the idea so much that it's giving Aileron $25 million upfront to develop the technology. If Aileron succeeds in five different disease targets, which neither company will identify, $1.1 billion awaits the Massachusetts company. The targeted diseases, Roche says, fall within the company's priorities of cancer, viruses, inflammation, metabolism and the central nervous system.
Roche appears optimistic about the potential to create an entirely new class of drugs targeting diseases that previously had eluded scientists. "One of the challenges the industry is facing is not to identify new targets, but to be able to reach the target that we would like to reach with the right therapeutic benefit, particularly inside the cell," Dr. Jean-Jacques Garaud, Roche's global head of pharmaceutical research and early development, told the New York Times.
- read the New York Times story
- and a report from Xconomy
- then watch a video on stapled peptides
- see another video on their targets