Ascent Therapeutics Climbs Out of Stealth Mode with “Pepducins”

Xconomy Boston — 

Ascent Therapeutics wants to be known as “The Pepducin Company.” The Cambridge, MA, biotech startup, which plans to publicly unveil its science and new executive team today, says it’s the first and only developer of pepducins—a new class of drugs aimed at modulating a family of cell surface receptors that are already lucrative therapeutic targets for the pharmaceutical industry.

Rick Jones, CEO of Ascent, puts his firm’s pepducins up among some of the more disruptive biotech discoveries to emerge in recent history. “This is a fabulous platform opportunity,” says Jones, who joined the firm in August. “This is like being at the first monoclonal antibody company or the first RNAi [RNA-interference] company.”

What are pepducins? They’re synthesized peptides, or amino acid chains, attached to lipids. While pepducins are a new class of drugs, they are designed to home in on cell surface receptors—called G protein coupled receptors (GPCRs)—which are the main targets of many marketed products. Take, for example, schizophrenia drug olanzapine (Zyprexa), which brought Indianapolis-based pharma giant Eli Lilly (NYSE:LLY) revenue of $4.76 billion last year. Ascent says on its Web site that GPCR-targeted drugs generate more than $40 billion in annual sales.

These cell surface receptors are involved in signaling between cells and play roles in many illnesses including cancer, cardiovascular disease, and inflammation. Pepducins target the receptors at the inside surface of the cell membrane, giving them the potential to interact with many GPCRs that are difficult to affect with traditional chemical drugs that aim for the receptors on the outside surfaces of cells.

Ascent has raised $19 million in its Series A venture capital round, which included investments from HealthCare Ventures, Novartis Option Fund, and TVM Capital. On top of its investment, Novartis Option Fund has purchased an option to develop certain uses of pepducins. Neither party, however, would tell me the amount of the option fee or which use or uses of pepducins the option deal addresses.

Ascent’s name has been out there for at least a couple of years, known mostly as the company where Michael Webb, former chairman of the Massachusetts Biotechnology Council, served as CEO. Prior to co-founding Ascent, Webb was the longtime chief executive of Lexington, MA-based Epix Pharmaceuticals (NASDAQ: EPIX), which also develops drugs to target GPCRs. Jones took over as CEO in August, and Webb has remained on the board of directors.

Jones, who was previously an executive at Belgian biotech Devgen, has begun to build out the rest of the management team. In October, Steve Hunt joined Ascent as senior VP of discovery research after serving as executive director of RNAi development at Pfizer’s local R&D beachhead in Cambridge.

The new crew at Ascent, while excited to be on the ground floor of the pepducin business, know there is work to do before pepducins can be made into drugs for humans. “We’ve learned a lot about some of the characteristics of the (pepducins) that are important,” Hunt says, “but there’s still quite a bit we’d like to know, particularly around how to develop them as therapeutics.”

Jones says that next year Ascent will identify its first pepducins to advance into preclinical animal studies, and the firm aims to start its first human clinical trials by mid-2010. Though the company hasn’t picked a therapeutic focus, the CEO notes that one area of particular interest is inflammation.

Pepducins were initially discovered in the late 1990s by researchers and Ascent co-founders Athan Kuliopulos and Lidija Covic of Tufts Medical Center, who published their early findings in this Nature article in 2002.