This year, the company plans to start clinical trials of its hepatitis C drug, along with tests of another drug designed to treat certain cancers and autoimmune diseases. It raised $30 million in July and recruited an experienced biotech deal maker as chief executive.
Avila’s drugs form covalent bonds with their targets on cells. That means the bonds are supposed to shut down completely, and irreversibly, the activity of the intended protein target they hit on cells. Conventional therapies connect with targets in ways that put them in a state of “dynamic equilibrium,’’ meaning they bounce on and off targets, says chief executive Katrine Bosley. By shutting down the target and thus halting the disease, Avila believes it has found a way to improve upon the trailblazing work of Vertex in hepatitis C, and it might be able to nail other targets pharma companies have long pursued, but couldn’t effectively block.
“Covalent drugs haven’t been much explored in the industry,’’ Bosley says. “As we develop a data set that says we can develop very specific covalent drugs, it’s intrigued a lot of people.’’
Vertex’s hepatitis C treatment, telaprevir, a protease inhibitor, has helped the company build an $8 billion stock market valuation, largely because clinical trials have shown it can basically double traditional cure rates, while shortening the usual yearlong treatment by half. Analysts predict Vertex could exceed $2 billion in US sales alone after a couple of years on the market.
One of the challenges is that viruses mutate, essentially switching around an amino acid or two, which is a sort of a survival mechanism, Bosley says. There are four or five slightly mutated versions of the hepatitis C protease, and the Vertex drug and others like it have varying degrees of potency when they encounter the mutants, she says.
The advantage of a covalent drug is that it doesn’t allow the virus to escape via mutation, Bosley says.
Plus, there are different genotypes of the hepatitis C virus. Telaprevir is most effective against genotype 1, while Avila’s treatment is thought to be effective against all genotypes, Bosley says.
The company is being incubated at the Boston office of Morgenthaler Ventures, where Morgenthaler entrepreneur in residence Doug Treco is leading Ra as its chief executive, according to the venture firm’s website.
Documents filed with regulators do not specify who invested in the company, but they do say the board of directors includes Treco, Morgenthaler partner Jim Broderick, Novartis Option Fund managing director Lauren Silverman, and New Enterprise Associates partner Ed Mathers.
Founded in 2008, Ra Pharmaceuticals appears to have money in the bank and a leader who has had considerable success in developing drugs. Treco served as senior vice president of research and development at the former Cambridge-based Transkaryotic, which he cofounded. It was acquired by the Irish drug maker Shire for $1.6 billion in 2005. He was part of a team at TKT that developed biotech drugs for rare diseases, such as the Hunter syndrome treatment idursulfase (Elaprase), which is now a core product for what has become the Massachusetts-based human genetic therapies unit of Shire.
This report was compiled by the editors of Xconomy, an online news website focused on the business of technology and innovation. For more New England coverage, visit www.Xconomy.com/boston. 
