MiRagen Therapeutics – 2012 Fierce 15

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Bill Marshall, CEO of MiRagen Therapeutics

Based: Boulder, CO
Founded: 2007
CEO: Bill Marshall
Focus: MicroRNA drugs
Website: www.miragentherapeutics.com

The Scoop: MiRagen Therapeutics has claimed verdant territory in the emerging field of microRNA drug development, with a lead candidate (MGN-9103) in preclinical development that could combat heart failure while providing a weight-loss benefit and other positive metabolic effects. Servier, the second-largest drugmaker in France, grabbed certain rights to this program and two others as part of a $352 million partnership with the biotech announced last year. And recently the company drummed up additional support from venture investors to raise a $20 million Series B round in April, funding the company's expansion into research of orphan diseases and other indications beyond its core focus in cardiovascular and muscle ailments.

What Makes it Fierce?

MicroRNAs control expression of whole sets of genes involved in diseases, making them targets of high interest in academic and biopharma labs. MiRagen has explored multiple miRNA targets for cardiovascular and other diseases since forming in 2007, yet the past year for the Boulder-based biotech has been full of major highlights. The startup has made headlines on both the scientific and business fronts, cashing in on those years of biological research with academic collaborators from such schools as the University of Colorado and the University of Texas Southwestern (UTSW).

Last year, French drug powerhouse Servier became the first big-named pharma to place a sizable bet on MiRagen's cardiovascular disease programs. MiRagen captured $45 million in upfront and research funding for three years, with the potential to reap $352 million in regulatory and commercial milestones, according to the company. Servier picked up rights to MiRagen's two lead candidates, MGN-9103 and MGN-1374, and a third unnamed target in markets outside the U.S. and Japan. It leaves MiRagen with ample room to forge future alliances on those programs, too.

Though likely more than a year from human studies, MGN-9103 offers MiRagen and partner Servier a potential drug with applications in multiple blockbuster markets such as heart disease, diabetes and obesity. The candidate, which inhibits microRNA 208, first showed promise in preventing heart failure and then in April came a major publication in the journal Cell that not only showed that the compound could treat metabolic syndrome in mouse models, but uncovered the potential role of the heart in regulating metabolism.

"It's gone from something where we thought we would have some profound implications in heart failure to something that now has implications in heart failure but also diabetes, obesity and metabolic syndrome," co-founder and CEO Bill Marshall tells FierceBiotech.

MiRagen has a way to go before the dazzling effects of the drug in mice can be replicated in humans, yet the company could be on to one of the next important targets in both cardiovascular and metabolic diseases. Big Pharma outfits have urgent needs to find the next blockbuster cardio drugs, for instance, with generic drug threats to Pfizer's megaseller Lipitor and Sanofi ($SNY) and Bristol-Myers Squibb's Plavix. And the pharma stampede has chased new cardio targets such as PCSK9 in hopes of rescuing their fortunes.

Yet startups face daunting funding challenges in tackling the cardio market, as clinical trials for heart drugs often call for thousands of patients and hundreds of millions of dollars. Servier has agreed to cover clinical development of MiRagen's lead drugs through Phase II as part of the alliance inked last year, with the option to provide additional support in pivotal trials. In April, MiRagen found more support from a bevy of venture backers--including the VC unit of biopharma giant Amgen ($AMGN)--in a $20 million Series B round. The company previously raised $18 million in first-round funding.

With Servier supporting much of the startup's cardio research, MiRagen plans to use some of the fresh round of funding to do research on microRNA targets involved in rare diseases. As opposed to heart drugs, orphan drug development can be done with relatively small numbers of patients in clinical trials as well as offer relatively speedy and streamlined routes to market.

MiRagen and other microRNA drug developers such as San Diego-based Regulus Therapeutics face similar challenges in delivering drugs to desired tissues in the body. At MiRagen, Danish biotech Santaris Pharma serves as a partner in providing its locked nucleic acid (LNA) technology, which enables the nucleic acid drugs to remain stable in the bloodstream and reach target tissues without carrier molecules such as lipids. And MiRagen this year hired Art Levin, the former head of U.S. operations for Santaris, to be the startup's chief scientist, bringing his experience with Santaris' microRNA drug in mid-stage clinical development.

"MiRagen's novel microRNA-targeted therapeutics offer the potential to regulate entire families of genes, and therefore have enormous potential in the treatment of complex multifactorial disorders like cardiovascular disease," said Atlas Venture partner Bruce Booth, a co-founder and chairman of MiRagen. "As a pioneer in the field, MiRagen is well-positioned to create significant value both for patients and our shareholders through this new modality."

Investors: Amgen Ventures, Atlas Venture, Boulder Ventures, Broadview Ventures and Remeditex Ventures.

-- Ryan McBride (Email | Twitter)