East Coast Life Sciences Roundup: Orphan Drugs, Financings, More

Xconomy New York — 

Here’s our roundup of this week’s news from the Boston and New York life sciences communities.

— It was a big week for biotech companies focused on developing treatments for very rare diseases, sometimes called “ultra-orphan” diseases.  Two Food & Drug Administration advisory panels of outside experts met this week to consider two such ultra-orphan drugs, and gave both a resounding seal of approval despite safety concerns. NPS Pharmaceuticals  (NASDAQ: [[NSPA]]), in Bedminster, NJ won two unanimous votes from the 12 panel members on Tuesday for teduglutide (Gattex), which helps people with small bowel disease absorb nutrients so that they can be weaned off intravenous feeding. The following day a different panel voted 14-2 to endorse Cambridge, MA-based Aegerion Pharmaceuticals’  (NASDAQ: AEGR) lomitapide, for a very rare genetic condition that causes cholesterol level to soar, leading to almost certain death from heart disease by age 30. Although both drugs have serious side effects, the panels ruled that the benefits outweighed the risks as long as patients taking teduglutide or lomitapide are closely monitored. The FDA is scheduled to issue its decision on both drugs before year-end.

—A new medical device company based in New York, Cibiem,  launched on Wednesday with $10 million from a Series A round of financing. The firm came out of Coridea, a medical device incubator in New York that has successfully launched six companies since starting up in 2003. Cibiem is developing a minimally invasive catheter-based system that targets the carotid body, a small cluster of nerve cells at the base of the throat that play a role in a broad range of diseases. The financing was led by life sciences venture investors SV Life Sciences  and Third Rock Ventures. This is another example of Third Rock’s focus on risky bets on emerging technologies, as the carotid body has only emerged in recent years as a therapy target for hypertension and other cardiovascular diseases.

—Another successful financing deal was reported by Healthrageous, a two-year-old Boston firm that provides health management tools. It raised $6.5 million in series B financing on Tuesday. The company said the funding was a validation of its shift in focus from both healthy and sick people to specifically target only those suffering from chronic diseases, such as diabetes or hypertension. The funding round was led by North Bridge Venture Partners, Long River Ventures and Egan-Managed Capital. Healthrageous CEO Rick Lee said the money would be used to scale up the company’s health-tracking devices that are offered to individuals through insurance companies and employers, to help them perform such tasks as track their blood sugar levels and weight. Healthrageous is one of a number of startups seeking to capitalize on the “gamification” of healthcare, when technology and social media are used to encourage people to adopt healthy habits.

—Despite these successful financings, venture capital interest in healthcare was lackluster in the third quarter, according to a report  issued Wednesday by New York financial data firm CB Insights. As my colleague Bruce Bigelow reported, “The almost $1.6 billion that was invested in 134 deals represented a 7 percent decline from the almost $1.7 billion invested in 143 deals during the same quarter a year ago, although it was a slight uptick from the previous quarter, when VCs put more than $1.5 billion in 142 companies.” Overall, the report found that VCs invested $847 million in 84 Massachusetts companies of all kinds, while 86 New York companies received $489 million.