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East Coast Biotech Roundup: Regeneron, Sage, Tara, Alnylam, & More

Xconomy Boston — 

The autumn chill has hit New York and Boston, which means one big thing for biotech: conference season is underway. Data are starting to pour in from a variety of scientific meetings, and much more is coming, as companies look to end the year or start 2015 with a bang. We’ve got those stories and more below:

—Tarrytown, NY-based Regeneron Pharmaceuticals (NASDAQ: REGN) and its partner Sanofi are ready to take their closely watched allergy drug, dupilumab, into Phase 3 testing after it hit its mark in another mid-stage trial—this time for patients with uncontrolled moderate-to-severe asthma. The drug, which blocks the interleukin 4 and interleukin 13 receptors, is also being developed for other disorders like eczema and chronic sinusitis with nasal polyposis.

—Cambridge, MA-based Sage Therapeutics (NASDAQ: SAGE) reported that 73 percent of the patients in its Phase 1/2 study responded to SAGE-547, its experimental treatment for super-refractory status epilepticus, a rare form of epilepsy. I talked to CEO Jeffrey Jonas about the findings, and what’s next for Sage.

—New York-based Tara Biosystems has become the latest startup with an idea to help make preclinical tests more reliable—a technology that turns stem cells into mature heart tissue that can be tested if it were an adult heart. I spoke with CEO Misti Ushio, a managing director of the VC firm that seeded Tara—Harris & Harris Group (NASDAQ: TINY)—about the technology and how it compares to others in the field.

—The Janssen Biotech group of New Brunswick, NJ-based Johnson & Johnson (NYSE: JNJ) cut a deal to grab worldwide rights to imetelstat, the myelofibrosis drug being developed by Menlo Park, CA-based Geron (NASDAQ: GERN). J&J paid Geron $35 million up front, and could shell out up to $900 million total if all goes well.

—Cambridge-based Blueprint Medicines raised a $50 million Series C round led by Partner Fund Management. Existing backers Third Rock Ventures, Casdin Capital, Fidelity Biosciences, Nextech Invest, and Biotechnology Value Fund contributed, as did new investors Wellington Management, RA Capital, Tavistock Life Sciences, Perceptive Advisors, Sabby Capital, Cowen Investments, and Redmile Group—several firms better known for backing public companies. Blueprint aims to begin its first clinical trials next year.

—New York-based Ophthotech (NASDAQ: OPHT) grabbed an option to license tivozanib from Cambridge-based Aveo Oncology (NASDAQ: AVEO). Ophthotech paid Aveo $500,000 up front to investigate tivozanib—once on the doorstep of FDA approval as a kidney cancer drug, before an advisory panel shot it down—as a potential treatment for eye diseases. If Ophthotech chooses to move forward, Aveo would get another $2 million fee and the chance to see some $95 million in various payments down the road. The deal is badly needed positive news for Aveo, which has lost partnerships with Astellas Pharma and Biogen Idec over the past couple years.

—Whitehouse Station, NJ-based Merck’s plans to upstage Gilead Sciences (NASDAQ: GILD) with a four-week regimen for hepatitis C fell short this week, as a triple-drug combination the company tested in a mid-stage trial posted cure rates below those that have been achieved by Gilead’s sofosbuvir (Sovalid) and Harvoni (a combination of sofosbuvir and another Gilead drug, ledipasvir) in longer regimens. Merck was testing two of its own drug candidates—grazoprevir and elbasvir—in combination with Gilead’s sofosbuvir in the study. The regimen posted better cure rates over a longer course of treatment.

—Cambridge-based Sarepta Therapeutics (NASDAQ: SRPT) began dosing the first of about 20 Duchenne Muscular Dystrophy patients in a new, open-label study testing its drug, eteplirsen, in people who either can’t walk or can’t complete a 6-minute walk test. It’s one of several studies Sarepta is conducting to gather enough data to support an FDA application for eteplirsen.

—Cambridge-based Alnylam Pharmaceuticals (NASDAQ: ALNY) reported data from a Phase 2 study of revusiran, its subcutaneous RNAi drug, in patients with transthyretin cardiac amyloidosis. Alnylam said the drug knocked down an average of 87.1 percent of patients’ disease-causing protein—transthyretin—after five weeks of treatment. Alnylam is conducting an open-label extension of the trial to get more safety data, and see whether knocking down transthyretin helps clear up patients’ disease—a much more clinically relevant study goal. It expects to begin a Phase 3 trial of revusiran by the end of the year.

Photo courtesy of flickr user Vivienne Gucwa via Creative Commons