East Coast Life Sciences Roundup: Sarepta, Vertex, Celsion, More

2/1/13Follow @cathyarnst

It was a roller coaster week for news on the East Coast, with two clinical trials ending with diametrically opposite results, Vertex explaining lackluster earnings, and Sarepta dealing with a weird Twitter hoax.

Vertex Pharmaceuticals  (NASDAQ: VRTX) reported its third quarterly loss in a row on Tuesday, all because sales of its once high-flying hepatitis C drug teleprevir (Incivek) fell by half during the three months, to $222.8 million from $456.8 million the prior year. Teleprevir was approved in 2011 and racked up more than $1 billion in sales in its first year, but many patients are now waiting for new drugs in the pipeline that may have fewer side effects. Vertex executives told investors in a conference call that it still expects to generate sales from the drug from patients who want to begin treatment before the new drugs are available, or are too sick to wait.

—Someone in Twitter land does not like Sarepta Therapeutics (NASDAQ: SRPT). On Wednesday the Cambridge, MA-based company’s stock fell from a high of $29.20 early in the day to $26.50 by 1 pm, a 10 percent drop, after someone using the Twitter handle @citreonresearc alleged that results announced last October for a Phase II trial of eteplirsen, Sarepta’s experimental treatment for Duchenne Muscular Dystrophy, had been doctored. By the end of the day that Twitter account had been suspended and Sarepta CEO Chris Garabedian issued a statement calling the tweets false and misleading, and saying the drug is on track.

—New York-based Keryx Biopharmaceuticals (NASDAQ: KERX) finally had success with a clinical trial, after its first two drug candidates failed. The drug, ferric citrate (Zerenex), met both its primary and secondary goals in a Phase III clinical trial for patients on kidney dialysis. Keryx’s stock price soared from $3.43 last Friday to $9.08 by the close on Thursday.

—On the flip side, Celsion (NASDAQ: CLSN), based in Lawrenceville, NJ, reported on Thursday that its lead drug candidate Thermodox, a proprietary reformulation of the chemo drug doxorubicin, did not even come close to meet its goals in Phase III clinical trial for treatment of primary liver cancer. That failure could endanger a development deal Celsion signed just last week with Zhejiang Hisun Pharmaceutical, a major Chinese drug company, which could be worth up to $100 million.

—Genzyme, the Cambridge, MA subsidiary of Sanofi (NYSE: SNY), got some good news on Tuesday when the FDA approved the drug mipomersen (Kynamro) as a treatment for a rare inherited disease that causes very high levels of cholesterol in the blood. Genzyme developed the drug with Isis Pharmaceuticals (NASDAQ: ISIS) of Carlsbad, CA, and Genzyme will now pay Isis a $25 million milestone payment.

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  • Not Born Yesterday

    “Does not like” SRPT? Actually the perpetrator of this fraud used offshore account to immediately cover numerous short holdings while the stock plummeted. Where O where is the SEC?