East Coast Biotech Roundup: Moderna, Aileron, Seres, & More

11/22/13Follow @benthefidler

[Updated, 9:08 am ET] The hours are quickly ticking down towards Turkey Day, so no surprise that many East Coast biotechs wanted to wrap up their big news announcements first. We’ve got all of those headlines below:

—Cambridge, MA-based Moderna Therapeutics turned heads in the biotech world when it got a $240 million check from AstraZeneca earlier this year as part of a deal to develop its messenger RNA technology. Now the privately-held biotech has added a big, $110 million round of equity financing from unspecified investors—some returning, and some new ones—to continue the charge. Moderna has now raised a total of $415 million through the AstraZeneca deal, equity financing, and government support. The new raise leaves Moderna with $340 million in the bank to both develop its own clinical programs, and its mRNA platform.

—Cambridge-based Aileron Therapeutics has long decided to make a stapled peptide drug to protect one of cancer biology’s hardest targets, the tumor suppressor protein called P53. This week, its Big Pharma investors—which include the venture arms of Roche, Eli Lilly, GlaxoSmithKline, and Novartis—teamed with Apple Tree Partners and Excel Venture Management to throw $30 million behind the ambitious effort. The $30 million Series E round bring Aileron’s total raised cash to about $100 million since its inception, and sets the stage for Aileron’s first clinical trial of the p53 peptide drug, ALRN-6924, which will take place next year. I spoke to Aileron CEO Joe Yanchik about what that trial will look like.

—Cambridge-based Seres Health is the latest startup to use the evolving understanding of the human microbiome to try to come up with new drugs. The startup came out of stealth mode this week with the help of a $10.5 million Series A round from Flagship Ventures, Enso Ventures, and others, planning to create microbiome-based treatments to combat infectious, metabolic, and inflammatory diseases. Seres says its first drug, SER-109, is already in its first clinical trial. The drug is designed to treat patients with C. difficile infections that are resistant to the antibiotic vancomycin.

—Busy week for Cranbury, NJ-based Amicus Therapeutics (NASDAQ: FOLD), which acquired Doylestown, PA-based Callidus Biopharma, raised $40 million in debt and equity financing, axed 14 percent of its workforce, and reacquired full rights to two Fabry Disease drugs it’s been developing with GlaxoSmithKline. The whirling dervish of strategic moves leaves Amicus with another drug in its portfolio and a focus on “next generation” enzyme-replacement therapies, but also a ways away from finding out if those therapies actually work.

—[Updated with new item] All is apparently not lost for Cambridge-based Ariad Pharmaceuticals (NASDAQ: ARIA). The European Medicines Agency said Friday that Ariad can continue to sell its cancer drug, ponatinib (Iclusig), in Europe—so long as prescribing doctors take extra precautions. Specifically, the EMA said that ponatinib shouldn’t be prescribed for people with a history of heart attacks or strokes “unless the benefits outweigh the risks.” Doctors also have to continue to monitor and manage patients’ cardiovascular status while they are on the drug. Ponatinib was pulled off the market in the U.S. in October because patients taking the drug were experiencing serious blood clots and other toxicities at a higher than expected rate. Ariad’s shares jumped about 30 percent in pre-market trading on Friday. The company has lost more than 75 percent of its market value since early October.

—LabCentral, the non-profit organization set up to provide lab space to Boston-area life sciences entrepreneurs, has officially opened its doors. The incubator is a 28,000-square-foot facility in an MIT-owned building at 700 Main St. in Kendall Square. It can house as many as 25 startups and 100 scientists and entrepreneurs.

—Bedford, MA-based Hologic (NASDAQ: HOLX) is Carl Icahn’s latest target. The activist investor bought a roughly 13 percent stake in Hologic and announced intentions to push for a board seat. Hologic promptly responded by adopting a poison pill to prevent a potential takeover.

—Cambridge-based Vertex Pharmaceuticals (NASDAQ: VRTX) agreed to sell the rights to the non-U.S. and Canada royalties on its hepatitis C drug, telaprevir (Incivo in Europe, Incivek in the U.S.) to marketing partner Janssen Pharmaceutica for $152 million in cash. The move is Vertex’s latest to shift more of its resources away from the fast fading hepatitis C program and towards its successful cystic fibrosis drug, ivacaftor (Kalydeco). Vertex recently announced plans to cut 15 percent of its workforce—positions related to telaprevir—after dismal sales of the drug in the third quarter.

—Xconomy’s National Biotech Editor Luke Timmerman examined the biotech startup class of 2013, and found that the list of new startups with a big idea, credible management, and at least $5 million in financing is noticeably shorter than it has been in previous years. Still, a few noteworthy East Coast names cropped up: in Cambridge, Jounce Therapeutics, Cydan, and NextCode Health. In New York, Tacurion Pharma and Loxo Oncology made the list of the year’s biggest biotech startups.

—Woburn, MA-based Aphios has spun off a subsidiary named Amylon to develop a drug candidate being developed for mild to moderate Alzheimer’s Disease and other central nervous system (CNS) disorders. The experimental drug, APH-1104, is supposed to work by dialing up alpha secretase, an enzyme implicated in the creation of beta amyloid plaques in the brain.

—An experimental hepatitis C drug co-developed by Watertown, MA-based Enanta Pharmaceuticals (NASDAQ: ENTA) and AbbVie (NYSE: ABBV) produced encouraging results in the first of six Phase III studies testing the drug with various other parts of an all oral regimen to treat the disorder. Results from the remaining five studies will be released in the coming months.

—Boston-based startup Topokine Therapeutics has begun a mid-stage clinical trial testing a topical gel it has developed to reduce excess submental (chin) fat. Topokine’s treatment is far behind a rival drug developed by Calabasas, CA-based Kythera Biopharmaceuticals’ (NASDAQ: KYTH) that just produced positive results in a Phase III study in September. Topokine’s hook, however, is its treatment is a gel—Kythera’s ATX-101 is an injection. Topokine is backed by Schooner Capital.

—Katrine Bosley, the former CEO of Bedford-based Avila Therapeutics and a entrepreneur-in-residence at the Broad Institute of MIT and Harvard, has become the chairman of the board of Cambridge-based Genocea Biosciences.

—Scientific entrepreneur Michael Gilman, formerly with Biogen Idec (NASDAQ: BIIB) and a startup Biogen acquired called Stromedix, announced on Twitter this week that he has become an entrepreneur-in-residence at Atlas Venture. The folks at BostInno called him a biotech “legend,” which has spawned a fair bit of jokes on social media.

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

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