UPDATED: Upstart Ra sheds stealth status, targets new drug class

Posted under angioedema, Biotech Venture Capital, Blog, Clycomimetics, Companies, Diagnostics, Doug Treco, Funding, Medical Devices, Medical Supply, Morganthaler Ventures, New Enterprise Associates, Pharmaceuticals, Pipeline, Ra Pharmaceuticals, rare diseases, Startups, Universities, Videos by john

Cambridge, MA-based Ra Pharmaceuticals is stealth no more. Two years after it snagged an initial $10 million in venture cash, Ra has hauled in a second tranche of $8.6 million and picked its disease target: hereditary angioedema, a rare and lethal immune system disorder. And after maintaining strict silence as the biotech was being incubated at Morgenthaler Ventures, biotech vet Doug Treco today is going public, asserting that Ra is on the trail of a new drug class.

Ra's claim to fame will rest on Cyclomimetics, which Treco says will have the "diversity and specificity of antibodies, coupled with the beneficial properties of small molecules."

"The only FDA-approved treatment for the prevention of HAE attacks is delivered intravenously every 3 to 4 days and produced from human blood," Treco said in a statement. "Our synthetic Cyclomimetics are easily produced, and could offer a stable, highly potent option for patients suffering from HAE. In addition, Cyclomimetics have the potential to be orally-available, which would significantly increase the quality of life for patients with HAE." 

The inspiration for the biotech dates back 7 to 8 years, says Treco, when he was working in the lab of Nobel prize-winning scientist Jack Szostak, who's now chairman of Ra's scientific advisory group.

"We want to claim the middle ground between small molecules and proteins," Treco tells FierceBiotech this morning. "Proteins and monoclonals are outstanding drugs," he adds, characterized by low toxicity and capable of carrying out multiple functions. "But they can't get into cells, are expensive to manufacture and require injection." Small molecules, meanwhile, have great availability but lack specificity and often trigger off-target toxicity.

A new drug class involving a slightly larger molecule, though, could disrupt protein-protein interaction, offer great specificity and low toxicity. And Ra plans to create new peptides that can make it into cell membranes.

Treco says the biotech is on track to get into the clinic in about two years. And he's upbeat about the prospects of building "massive libraries" that can be used to develop a significant pipeline of therapies; some that it can keep, others that can be partnered. And he also expects to partner with drug developers who will come to Ra looking to solve some thorny problems. Ra has gathered together a staff of 19 and plans about seven more hires by the end of the year.

Treco is a familiar face in the rare disease space. He co-founded Transkaryotic, which Shire bought out in 2005 for $1.6 billion. Ra was initially incorporated in 2008 and gained a $27 million Series A commitment from New Enterprise Associates with Morgenthaler Ventures, Novartis Venture Funds and Amgen Ventures.

- here's the press release

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NEA turns to Europe in $30M round for RNA-drug developer Prosensa

Posted under Blog, Companies, Diagnostics, Duchenne muscular dystrophy, Funding, GlaxoSmithKline, Medical Devices, Medical Supply, New Enterprise Associates, Pharmaceuticals, Prosensa, RNA, Startups, Universities, Videos by John Carroll

The Dutch biotech Prosensa has rounded up 23 million euros--about $30 million--in a new round earmarked to back its work on RNA drug development for Duchenne muscular dystrophy and other rare diseases. New Enterprise Associates led the fundraising round, spearheading its first investment in Europe and signaling its interest in more global deals. NEA joined Abingworth, Life Sciences Partners, Gimv, Idinvest Partners and MedSciences Capital.

"Over the past few years, we have made substantial progress in our research and development pipeline," says Prosensa CEO Hans Schikan. "Our lead drug candidate for Duchenne muscular dystrophy is in Phase III clinical trials in partnership with GlaxoSmithKline. We have advanced the development of five additional compounds in DMD and have announced preclinical testing for a compound for DM1. This financing will help us to further strengthen our position in rare diseases and will allow us to deliver on our promise of accelerated development of treatments for patients in need."

The round marks an encouraging advance for RNA, a development field which has been plagued by some major league exits in the past two years. In Prosensa's case investigators have been focused on a novel approach to correcting the mutation in the dystrophin gene that triggers DMD. They're using antisense oligonucleotides to induce exon skipping, essentially jumping past the genetic alterations associated with the disease. GlaxoSmithKline ($GSK) partnered with Prosensa back in 2009, pledging up to $680 million in their pact.

"While NEA is one of the largest venture firms in the world, with more than $11 billion in capital under management, Prosensa is our first biopharma investment in Europe and signals our openness to selectively consider investing in truly exceptional innovation in Europe as well as in our traditional geographies in the U.S., China and India," says David Mott, the general partner for NEA, which is managing $11 billion.

- here's the press release

Related Article:
Glaxo forges $680M RNA pact with Prosensa