The biotech world will be buzzing today about something called “stapled peptides.” That’s because Cambridge, MA-based Aileron Therapeutics, a pioneering developer of this new class of drugs, has secured $40 million in venture capital from an ensemble cast of investors that includes four of the world’s biggest drugmakers.
Aileron’s latest investment round was led by the venture arm of GlaxoSmithKline and a Boston-based venture fund, Excel Medical Ventures. They were joined by venture operations representing Novartis, Roche, Eli Lilly, as well as Apple Tree Partners, and two angel investors whose names aren’t being disclosed, says CEO Joe Yanchik. It’s a big leap onto the national stage for Aileron, which was founded in 2005 by the late Stanley Korsmeyer and Loren Walensky, a pair of biologists from Harvard Medical School and the Dana-Farber Cancer Institute, and Gregory Verdine, a Harvard University chemist. Aileron has now raised about $61 million since the beginning.
The new cash infusion will go toward developing novel kinds of peptide drugs that block targets inside cells that are unreachable by conventional small-molecule drugs or genetically engineered proteins, as I described in this feature story in November. A few other peptide drugs—small fragments of proteins—are already on the market, like Eli Lilly’s teriparatide (Forteo) for osteoporosis and exenatide (Byetta) for diabetes. But most of these drugs don’t work because they get chewed up by enzymes in the body before they can hit their target. Aileron’s key insight is to “staple” these peptides in a way that holds them together in a properly folded shape in the bloodstream, protecting them and preserving the unique structure that gives them ability to hit very specific cell targets.
The big break in the past year came in November, when Aileron scientists showed in a paper in Nature that drugs from this class could hit a novel target that triggers a self-destruct mechanism in cancer cells. Then, over the next few months, Aileron kept making more progress, showing these peptides could hit targets for metabolic diseases, inflammation, and infectious diseases, Yanchik says. What’s more, Aileron’s peptides can hit not just easier targets on the surface of cells, but are nimble enough to go even deeper, blocking the transcription of RNA in the nucleus of cells—which sounds a lot like RNA interference, one of the hottest concepts in biology today.
“We’re talking about a company that owns a new therapeutic modality. It’s like if one company had all of monoclonal antibodies, or all of RNA interference to itself, except without the delivery problems,” says one of Aileron’s investors, Campbell Murray, a managing director with Novartis Venture Funds in Cambridge, MA.
“We think we’re building something unique and special,” Yanchik says.
Aileron plans to use the money to expand its staff from 23 to about 35 to 40 people this year, Yanchik says. It will look to add depth in product development, and people with diverse skills in cancer, metabolic disease, and inflammation, he says. Financially, the money means the company has enough in the bank
