Platform: Stapled peptides
Company: Aileron Therapeutics
Therapeutic opportunities: Oncology, infectious disease, metabolic disease, immune/inflammatory diseases
Partnerships: Potential $1.1 billion agreement with Roche for up to 5 disease targets, including two for oncology, and inflammatory diseases; Multiple Myeloma Research Foundation.
Peptides are short-chain chemical compounds that, when linked together, become proteins. Yet peptides alone have their problems: They proved unable to penetrate cell walls well enough to deliver their therapeutic payload and to be unstable and short-lived.
Aileron's stapled peptides look to overcome that problem by being folded and locked ("stapled") into their biologically active helical shape, which mimics the structures found in natural protein-protein interactions.
Like small-molecule drugs, these stapled peptides can then penetrate a cell's outer surface rather than attach to surface targets, and they are not as limited to the kinds of targets with which they can bind, Chief Operating Officer and Chief Financial Officer Steven Kafka told FierceBiotech. They also keep their specificity and retain the natural targeting capability of therapeutic proteins with "very few limitations in their ability to address extracellular and intracellular targets."
Aileron's science was developed by a Harvard chemist and two biologists at Dana-Farber Cancer Institute. Cambridge, MA-based Aileron acquired exclusive rights to this technology and spent 5 years expanding the academic research into potential therapies. Its patience paid off with a $25 million upfront multitarget deal with Roche in August 2010 that focused on two cancer targets. An expansion of the agreement into inflammatory diseases came last November after Aileron succeeded in an in vivo proof-of-concept study of one of the Roche drugs. Its work with Roche has $1.1 billion potential.
Independent of the Roche agreement, Aileron is pursuing infectious and muscular degenerative disease programs designed to provide clear clinical proof-of-concept.