East Coast Biotech Roundup: Voyager, Concert, Retrophin, & More

2/14/14Follow @benthefidler

(Updated, 9:05 am ET) The East Coast got buried in snow this week—again—but biotech activity was raging regardless. More IPOs priced (of course). A new high-profile startup emerged from stealth. A small acquisition wowed Wall Street. Those headlines and more below:

—Third Rock Ventures has had success so far with its first gene therapy portfolio company, Bluebird Bio (NASDAQ: BLUE). Now it’s back for seconds, having started up Cambridge, MA-based Voyager Therapeutics with a $45 million Series A round of equity financing. Voyager plans to target central nervous system disorders like amyotrophic lateral sclerosis, Friedreich’s Ataxia, and Parkinson’s Disease. But the startup is also trying to develop a proprietary library of engineered adeno-associated viruses that it could either use to develop its own gene therapies, or form licensing deals with pharmaceutical companies for diseases it isn’t interested in working on. I spoke with Third Rock co-founder and Voyager interim CEO Mark Levin about the new venture.

—The biotech IPO parade continued this week, as two more local companies took themselves public. Lexington, MA-based Concert Pharmaceuticals (NASDAQ: CNCE) upsized its offering to 6 million shares from 5 million and then priced its IPO at $14 per share, the high end of its projected range. Shares traded slightly up thereafter, as the company, which adds deuterium to drugs to boost their abilities, closed its first trading day at $14.18. Concert has partnerships in place with Summit, NJ-based Celgene (NASDAQ: CELG), Avanir Pharmaceuticals (NASDAQ: [[AVNR]]), and Jazz Pharmaceuticals (NASDAQ: JAZZ).

—Burlington, MA-based Flexion Therapeutics (NASDAQ: FLXN) also took itself public this week. Flexion sold 5 million shares at $13 apiece, pricing its offering right in the middle of its projected $12 to $14 per share range. Shares gained about 13.2 percent on Flexion’s first day of trading. The company is developing a group of drugs to treat osteoarthritis.

—New York-based Retrophin (NASDAQ: RTRX) was one of biotech’s biggest gainers this week, as Wall Street cheered the company’s $62.5 million acquisition of Fort Collins, CO-based Manchester Pharmaceuticals. In acquiring Manchester, Retrophin grabbed a drug that’s approved to treat gallstones but has also long been prescribed off-label for a rare, inherited disease known as cerebrotendinous xanthomatosis. The drug, a synthetic form of a natural bile acid called chenodeoxycholic acid (CDCA), is structurally similar to the obeticholic acid that Intercept Pharmaceuticals (NASDAQ: ICPT) is developing to treat nonalcoholic steatohepatitis and primary biliary cirrhosis. Retrophin plans to seek FDA approval to use CDCA to treat patients with cerebrotendinous xanthomatosis, and also develop it as a treatment for the two disease types Intercept is going after. Shares of Retrophin climbed more than 40 percent on the news.

—(Updated with new item) Astellas Pharma has finally cut bait with Cambridge-based Aveo Oncology (NASDSAQ: AVEO). The two companies announced Friday that they’ve ended their deal to co-develop Aveo’s troubled cancer drug, tivozanib, and are shutting down its only remaining study—a mid-stage trial testing the drug in patients with colorectal cancer.  Astellas cited “strategic reasons” based on the “clinical status” of the drug for its decision. Aveo will recoup full rights to tivozanib on Aug. 11.

—Shares of New York-based Ventrus Biosciences (NASDAQ: VTUS) cratered after the company said that a topical cream the company has been developing to treat anal fissures, VEN-307, flunked the second of two pivotal, Phase III trials. The drug fared no better than a placebo cream at reducing patients’ pain in the study, which Ventrus attributed to a higher than expected placebo response. While Ventrus still plans to meet with the FDA to discuss the next steps forward, investors aren’t waiting around. Shares plummeted about 63 percent following the news.

—Parsippany, NJ-based Medicines Co. (NASDAQ: MDCO) also felt the wrath of investors after an FDA advisory panel voted 7-2 not to approve its experimental anticoagulant cangrelor, citing issues with safety and the design of the company’s clinical trial, according to a report from Bloomberg News. The FDA looks to its advisory panels for input before choosing whether to approve a drug. The agency is scheduled to do so by April 30. Shares fell more than 12 percent after the panel rendered its decision.

—New Haven, CT-based Melinta Therapeutics, the company formerly known as Rib-X Pharmaceuticals, raised a $70 million Series C round to fund a Phase III study testing antibiotics for uncomplicated gonorrhea and skin infections. The round was led by existing investor Vatera Healthcare Partners.

—Ironwood Pharmaceuticals (NASDAQ: IRWD) raised another $175 million to continue to help commercialize its irritable bowel disease drug linaclotide (Linzess). Ironwood sold 13,725,500 shares at $12.75 apiece. Shares of the company closed at $14 on Thursday. Linaclotide generated about $119 million over its first full year of sales.

—Bayer has decided to license an early-stage drug discovery program from Waltham, MA-based X-Chem, triggering a milestone from the deal the two companies signed in 2012. X-Chem will get an unspecified upfront payment and continued research funding as a result. X-Chem also has a drug discovery deal in place with AstraZeneca. I profiled the company in September.

—George Golumbeski, Celgene’s senior vice president of business development, joined the board of Watertown, MA-based Enanta Pharmaceuticals (NASDAQ: ENTA). Golumbeski will be one of the speakers at Xconomy’s March 6 event, “New York’s Life Science Disruptors.”

Ben Fidler is Xconomy's Deputy Biotechnology Editor. You can e-mail him at bfidler@xconomy.com Follow @benthefidler

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