Ascenta Therapeutics

Based: San Diego
Founded: 2003
Website: www.ascenta.com [1]

The Scoop: "Ascenta is gaining serious ground in the field of apoptosis and has a clearly thought-out strategy for a full range of pipeline candidates."

What makes it Fierce: Ascenta Therapeutics has come a long way on a lean and mean development machine. But with some mid-stage studies now under way for a lead cancer therapy, the company is transforming itself into a bigger biotech player with more therapies in the pipeline.

Launched four years ago by three key scientists who were then at the University of Michigan--Marc Lippman, Shaomeng Wang and Dajun Yang--Ascenta started out as a virtual developer with five employees and a small office. To keep costs low, Ascenta has tapped its founders' contacts in China to help develop a low-cost facility there to advance its discovery work. Just months ago, though, Ascenta announced its $50 million C Series [2], much of which will be used to advance AT-101, its lead oncology candidate, through Phase II clinical trials. AT-101 is an oral cancer therapy that inhibits the Bcl-2 family of proteins, directly and selectively triggering apoptosis, or programmed cell death in cancer cells.

"By the end of the year, we'll have the potential to see some significant data around indications," says Mark Benedyk, vice president of business development for Ascenta, who adds that an initial approval for AT-101 could come as early as 2010.

Strategically, says Benedyk, it's one of the new kinds of targeted agents that payers and doctors are looking for. As an oral therapy, it may compete effectively against some of the expensive monoclonal infusion therapies that have recently hit the market.

The most recent investment round gives the company enough runway to analyze mid-stage data for major cancer indications on AT-101. And there's room to extend the Series C by another $5 million to add to the runway. Once some significant Phase II data is on hand, says Benedyk, executives can ponder options like a partnership deal, IPO or a buyout.

What to look for: They're not just going to sit back and wait for the next big batch of data. The company is already looking to line up partners for their preclinical stage programs. The company has bulked up to 53 employees at this stage, and for the first time they're taking clinical research outside of the company and hiring a CRO to mount an upcoming trial. Ascenta is also looking for a Phase I program to in-license to fill a gap in the pipeline. Ideally, Ascenta will soon have a Phase IIb lead candidate, a new Phase I program, a partnered preclinical program and another preclinical program they're going to keep in-house.