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DART's 'Inspiration' in DMD Could Borrow the Halo Effect

By Randy Osborne
Staff Writer

Think "rare, pediatric diseases" and you tend to think "drug cocktail that changes over time" and "almost impossible to get enough investors, given the risk vs. return, not to mention the long development period."

It doesn't have to be that way, said Gene Williams, CEO of Cambridge, Mass-based DART Therapeutics Inc.

A biotech firm owned by patient foundations – the Nash Avery Foundation and Charley's Fund – DART hopes to turn Duchenne's muscular dystrophy (DMD) into a chronic, manageable illness, "as opposed to the horrible situation" that exists now, said Williams, formerly senior vice president at Cambridge peer Genzyme Corp. (now a unit of Sanofi SA).

"Most of the venture capital-backed companies are trying to compete for the best asset," he said. "We have a slightly different mission. We compete with the disease."

DART stands for "disease action research therapy," said Williams, calling his company's model "the ultimate partnership," bearing a strategy that uses a somewhat new, but not unheard of, model to move research along.

Inspiration Biopharmaceuticals Inc., also of Cambridge, is put together similarly to DART, and the firm's CEO, John Butler, is a former Genzyme staffer, like Williams. In April, Inspiration submitted a biologics license application to the FDA for IB1001, a recombinant Factor IX therapy for hemophilia B. (See BioWorld Today, April 18, 2012.)

Though not as advanced, DART – founded two years ago, after six years of work on the idea – has been busy doing "all the things a good biotech does," such as acquiring assets and conducting trials, Williams said.

About 18 months ago, the firm brought aboard the anti-fibrotic drug candidate known as HT-100 (a formulation of halofuginine); spun out a special-purpose entity, Halo Therapeutics LLC, to develop it; and in May raised $1.1 million from 12 not-for-profit foundations for a Phase II study. The drug has been granted orphan status in the U.S. as well as Europe.

DART also just completed a 92-patient validation study of a biomarker that measures muscle health directly in a noninvasive way. "If that biomarker works out as well as we hope it will in prospective studies, it could reduce the cost of doing trials for everyone," Williams said.

He cited another, offsite program in DMD that has been stalled for three years, unable to nail down funding. DART came up with a plan to get $15 million, $5 million of which foundations would contribute, with the remainder from investors.

Although the foundations would make out to some degree if the drug proves worthy, "investors get preferential return, and have some of the risk reduced," Williams said. "In this case, DART is just helping to structure it," he added.

DMD players are getting plenty of foundation support. An example is Prosensa Therapeutics BV, of Leiden, the Netherlands, which raised €23 million (then US$29.9 million) in a Series C round to enable it to move two new exon-skipping antisense oligonucleotide drugs into clinical development in DMD and to take forward preclinical programs in two other rare disease indications. (See BioWorld International, Feb. 1, 2012, and BioWorld Insight, Oct. 15, 2012.)

"Prosensa probably wouldn't exist if it weren't for all the funding support from the patient foundation community," Williams said. "If exon skipping works, as we all hope it does – and it's showing some early promise – we're still going to need to manage the inflammation, the fibrosis, help restore muscle strength."

Considering all the exons that must be skipped, "we're probably going to need to see 20 to 30 products develop, before we get DMD where we want it to be," he said.

A higher-profile, exon-skipping company is Sarepta Therapeutics Inc., also of Cambridge, which said recently that its eteplirsen hit the primary Phase IIb endpoint, an increase in novel dystrophin. (See BioWorld Today, Oct. 4, 2012.)

Williams noted that very few one-shot magic bullets exist like Cerezyme (imiglucerase), Genzyme's enzyme-replacement therapy for Gaucher disease. As a result, hybrids such as DART, which has 20 employees though "a handful" are full time, and Inspiration are "something we'll see a lot more of," he said.