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BIO Investor Forum Roundup: Immune Design, Sorbent Therapeutics, Epizyme

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in the year when it raised $36 million from Novartis Venture Funds, Sofinnova Ventures, Arch Venture Partners, CMEA Capital, and AgeChem. The company’s idea is to develop a type of polymer that doesn’t get absorbed into the bloodstream, and is made to soak up and remove fluids, and ions like sodiums or potassiums, that can cause complications in patients with heart failure and kidney failure. Sorbent estimates that 1 million heart failure patients a year get hospitalized with excess fluid buildup, and cheap diuretics often don’t work well enough.

One thing that caught my attention in CEO Detlef Albrecht’s presentation was the number 30. Sorbent’s current formulation requires patients to take 30 capsules a day to get enough of the compound into the patient’s system. That sounds like an awful lot to swallow. Afterwards, Albrecht said the company is experimenting with a powder formulation that could be ground up with meals, or various jelly-type substances which might be easier to take than a capsule. It’s likely the powder formulation will be the one to enter pivotal trials, Albrecht says.

—Cambridge, MA-based Epizyme has been one of the companies to watch in Boston for a while now, so I took some time to catch up with chief business officer Jason Rhodes. The company raised $54 million in its first couple years, and has continued to pull in even more cash through deals with GlaxoSmithKline, Eisai Pharmaceuticals, the Multiple Myeloma Research Foundation, and the Leukemia & Lymphoma Society. The company has grown now to about 40 employees, Rhodes says.

The money and brainpower of those organizations is going into Epizyme’s work to make cancer drugs based on the emerging science in epigenetics.This is the field that looks at how certain genes can be turned on or off without altering the underlying DNA. Instead of using a targeted cancer drug that’s made to hit one specific mutant protein, Epizyme’s idea is to make small molecule drugs that home in on a specific enzyme that basically makes it possible for multiple genes and proteins to spin out of control in cancer patients.

Specifically, Epizyme is going after a class of enzymes known as histone methyltransferases (HMTs). To hear Rhodes talk, he expects some big-name personalized cancer medicines of the future to come from this type of enzyme target.

“We worked through a 100-member target class of HMTs, and we prioritized about 20 of those members based on their very clear disease associations,” Rhodes says. “Plexxikon’s BRAF program, and Pfizer’s ALK program (crizotinib) are examples of late-stage targets that are like that. We saw about 20 targets that are like that, which are oncogenic, which really drive cancer in absolutely identifiable patient subgroups.”

Being a small company, Epizyme couldn’t really handle all the work of developing drugs against that many targets, so it did a “multi-target” partnership that handed over rights to a number of targets to Glaxo. But Epizyme made sure not to give away the store. One of its two lead targets, DOT1L, remains wholly-owned by Epizyme, Rhodes says.

The DOT1L program will “likely” be ready to enter clinical trials in 2012, Rhodes says. The drug is being positioned for patients with a rare disease known as mixed lineage leukemia. The drug is being developed with a companion diagnostic in mind from the start, to best select the patients with the DOT1L profile that makes them most likely to respond, Rhodes says. If the target can be validated in clinical trials—always a big if—then the company could get on a fast development path, Rhodes says. He pointed to a Journal of the American Medical Association article from June which said that from 2004 to 2010, the median size of a pivotal cancer drug trial in rare diseases—like mixed lineage leukemia—was a relatively manageable 95 patients.

“All of our programs are going after genetically defined cancers, they all will have companion diagnostics,” Rhodes says. “What it means is that when you go to the clinic, you are rapidly going into a relevant patient population. So it’s not a typical broad, slow Phase I, and then you get to a maximum tolerated dose (MTD) and you go into patients and wait, and wait. We should get relevant data quickly.”

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