[Updated 1/14/13, 1:31 pm. See below] Aileron Therapeutics said today that, as the result of successful preclinical studies, it secured the $12 million it needs to move its experimental treatment ALRN-5281 into initial clinical trials, in patients with rare endocrine disorders. According to Aileron, ALRN-5281 will be the first of a new class of drugs called stapled peptides to be tested in humans.
The seven-year-old startup, based in Cambridge, MA, made a big splash in 2010 when it signed a development deal that could be worth up to $1.1 billion with Swiss pharma giant Roche to develop stapled peptide drugs for various cancers. The pact significantly raised the profile of this relatively new drug platform in the process. Stapled peptides are fragments of proteins that are locked into a helical shape meant to help them last longer in the blood stream, and to penetrate the cell membrane rather than attach to the surface of the cell, as most protein-based drugs do.
So far, there has been a lot speculation about the potential for stapled peptides and a lot of academic research, but no proof yet that the fragments will work. Aileron’s compounds are based on technology developed by scientists from Harvard and Dana-Farber Cancer Institute in Boston.
[Paragraph added to include information gained from interview with CEO.] Aileron CEO Joseph A. Yanchik III told me in a phone interview that the company hopes to start the clinical trial early this year. He expects Aileron will make a range of announcements about its business, including other drugs in development, in the next six months. “We’ve started to talk a lot more openly about our work,” he said. In its lab research the company has found that stapled peptides may show efficacy against a number of different conditions, including inflammatory and infectious diseases. “Our industry is fundamentally starved for new innovations in drug development. There is a lot of interest in stapled peptides as a result,” Yanchik told me.
ALRN-5281 is not part of the Roche deal, which is focused on developing cancer treatments. Instead, it is a a long-acting drug that boosts the body’s production of growth hormone, designed to treat such conditions as adult growth hormone deficiency and HIV lipodystrophy.
The $12 million represents the second tranche of a Series D financing first announced in June 2009, and brings the total round to $42 million. Current investors Apple Tree Partners, Excel Venture Management, Lilly Ventures, Novartis Venture Funds, Roche Venture Fund, and SR One all participated in the round.
“These proceeds will allow us to advance ALRN-5281 through Phase 1 development and continue to develop our pipeline of stapled peptide drugs,” Yanchik said in a press release.