Edimer, Third Rock’s Bet on Rare Diseases, Aims to Begin First Human Tests

3/15/11

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dentures,” Kirby says.

XLHED is the most prevalent form of ectodermal dysplasias, of which there are more than 150 types. And apparently, there’s been some excitement among patients in Edimer’s experimental drug. At a 2009 meeting of a patient group called the National Foundation for Ectodermal Dysplasias in Williamsburg, VA, Kirby says that people gave him big applause for his company’s efforts to develop its treatment. Kirby also keeps a photo on his desk of kids with the disease who are holding up a “Thank You” sign.

Edimer’s drug is a protein-based compound that features a recombinant piece of the naturally occurring protein called EDA-A1. Patients with XLHED lack functional EDA-A1, which is important to the development of teeth, hair, and skin. The firm’s treatment, code named “EDI200,” has shown promise in replacing the function of the naturally occurring protein in mouse and dog models of the disease, according to the company. The firm hopes it can replicate some of those benefits in humans with the disorder.

Indeed, Edimer Pharmaceuticals was formed in summer 2009 to develop EDI200, so the group has a lot riding on the success of this drug. (There are early signs that the drug might have other applications in regenerative medicine in other areas of health.) Kirby says he came across the compound while serving as an entrepreneur-in-residence at Third Rock . The drug is based on the early research of Jürg Tschopp and Pascal Schneider at the University of Lausanne in Switzerland, and at one point the compound was the property of the Swiss biotech firm Apoxis. Yet Denmark-based drug developer TopoTarget bought Apoxis in 2007, primarily for what Kirby said were separate assets in Apoxis’ pipeline.

The XLHED treatment was in need of investment to continue its development. At Third Rock, Kirby had been working with venture partner Philip Reilly to scout for opportunities in the rare diseases market when the opportunity to invest in the XLHED compound arrived. Third Rock took the lead in a $22 million Series A announced in June 2009 to finance Edimer, and the new firm also attracted funding from VI Partners in Zug, Switzerland. Kirby says that the funding is enough to advance the drug to a point where the group can see some early evidence of its effects in humans.

In fact, Third Rock—co-founded by general partner Mark Levin, a former CEO of Millennium Pharmaceuticals (now part of Takeda)—has made the field of rare diseases therapies one of its areas of focus. Kirby says that the venture firm also invested in Bluebird Bio (formerly Genetix Pharmaceuticals), a developer of gene therapies for rare diseases, as part of its concerted effort to increase its stake in the field. While a bit dated, a story I wrote last year covers the growing interest in rare diseases among big drug companies.

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