Ascent Therapeutics’ Blood Cancer Drug Could Rival Genzyme’s Mozobil

9/16/09

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HealthCare Ventures, Novartis Option Fund, and TVM Capital, no less—before the financial markets collapsed last September. And though Jones declined to say how long Novartis has to exercise its option with Ascent, he noted that it could bring his startup more than $200 million in potential milestone payments.

Aside from financial issues, Ascent appears to have made good on identifying drug candidates by the end of this year, as Jones told me the firm planned to do when it came out of stealth mode last October. He said that in addition to the drug identified for stem cell mobilization, the firm has also engineered one of its proprietary molecules to treat an undisclosed cardiovascular condition and another to treat a gastrointestinal disorder.

So-called “pepducins,” which were discovered in a lab at Tufts Medical Center in the 1990s, are synthesized amino acid chains attached to fatty molecules. The drugs are intended to home in on signaling proteins called G-protein coupled receptors (GPCRs) that span the surfaces of cells and are the main targets for many big moneymakers such as Eli Lilly’s schizophrenia drug olanzapine (Zyprexa) and the heartburn pill ranitidine (Zantac). Ascent’s advantage, Jones said, is its ability to engineer customized pepducins that can target the interior side of the GPCR molecule, while most other drugs are thought to bond to the part that sticks out of the cell’s surface.

Despite its progress, the firm no longer plans to use its first-round financing to launch human clinical trials sometime in 2010, Jones said. Instead, it will build out its technology platform in hopes of attracting partnership deals with drug companies. However, if Novartis decides to exercise its option with Ascent, Jones said the company would be in a position to enter an initial clinical trial by late next year.

Jones sees a lot of potential for the firm’s lead drug for cancer patients beyond stem cell mobilization. (Cambridge-based biotech powerhouse Genzyme (NASDAQ: GENZ) has garnered FDA and European approval for that use of plerixafor in combination with granulocyte-colony stimulating factor.) Jones said that he believes that his firm’s drug candidate could also be used to free cancer cells from the bone marrow into the bloodstream, where they are more easily destroyed by chemotherapy drugs. This would be useful for treating leukemia and other malignancies of the bone marrow.

So, like other platform companies, Ascent has a plethora of potential uses of its science. Yet the key for the company now is to find another big pharma company or two to buy into that potential.

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