East Coast Biotech Roundup: Bluebird, Moderna, Navitor, & More

Xconomy Boston — 

New gene therapy data. New startups. More IPOs. Even more deals. Plenty of news to catch up on in this week’s roundup, all before your deputy biotech editor heads out of town for a vacation. Have a great week everyone, see you in July. But before that, here are your East Coast headlines:

—Gene therapy has seen more than its share of ups and downs over the past few decades. This past week, Cambridge, MA-based Bluebird Bio (NASDAQ: BLUE) provided a small, yet encouraging glimpse into its potential. Bluebird reported data from two beta-thalassemia patients dosed with its gene therapy, Lenti-Globin. Both of them were able to kick the chronic blood transfusions they’ve been getting most of their lives in a matter of weeks, and at least in the short term (3.5 months and 6.5 months respectively), their responses have held up. Bluebird’s shares have soared about 40 percent this week as a result. There are many hurdles still to come for Bluebird, of course, but I spoke with executives about the data, and what it means both for Bluebird and the gene therapy field.

—Cambridge-based Moderna Therapeutics has amassed a huge war chest of cash over the past few years. Now it’s putting that money to work—on an army of spinouts that will develop its messenger RNA (mRNA) drugs. Moderna initially aimed to be both a platform company and a drug developer. But CEO Stephane Bancel told me recently that the company shifted its strategy after cutting a big deal with AstraZeneca in late 2012, and now no longer plans to develop and sell its own drugs. You can read more about why Moderna made that decision here.

—David Sabatini helped discover the kinase mTOR, or “mammalian target of rapamycin,” two decades ago. Now the work on the molecule at his lab at the Whitehead Institute is the foundation for a Cambridge-based startup called Navitor Pharmaceuticals, which just raised $23.5 million from Polaris Partners, Atlas Venture, and the venture arms of GlaxoSmithKline (SR One) and Johnson & Johnson (Johnson & Johnson Development Corp.). Led by former Sirtris Pharmaceuticals CEO George Vlasuk, Navitor is looking to create drugs that target proteins in a complex mTOR is a part of called mTORC1—which gets thrown out of whack in a whole variety of diseases. I spoke with Vlasuk this week about the effort.

—Cambridge-based Zafgen (NASDAQ: ZFGN) and its fat-burning drug prospect beloranib got a good reception from Wall Street this week. The company, incubated by Atlas Venture almost 10 years ago, priced its IPO at $16 per share, the top of its projected range, and sold 6 million shares—1 million more than it originally planned to sell. All told, Zafgen raised $96 million before discounts due to underwriters. Atlas (35.6 percent stake) and Third Rock Ventures (35.4 percent) were Zafgen’s two most significant shareholders prior to the IPO. Zafgen ended its first trading day up about 23 percent from the IPO price.

—Sperm banks only screen their donors for a handful of the numerous potential genetic diseases that can be passed on to a baby. A New York- and Boston-based startup called GenePeeks is looking to increase peoples’ odds of having a healthy child with a product called “Matchright,” which combines DNA sequencing tools with a computerized platform to asses the risk of passing on one of around 500 genetic diseases via a sperm donor pregnancy. GenePeeks does this by combining the genetic information of a client and a sperm donor to create thousands of “digital babies,” or virtual genomes of hypothetical children. The company’s plan is to start with sperm bank clients before ultimately providing an alternative to conventional carrier screening for the wider population of couples who are considering having children. But the road to get there will be tough and fraught with competition: I spoke with CEO Anne Morriss and chief scientific advisor Lee Silver about the journey.

—Sanofi this week returned the worldwide rights to prospective cancer drug MM-121 to Cambridge-based Merrimack Pharmaceuticals (NASDAQ: MACK) after the company reported top-line results from the last of a series of mid-stage trials testing the drug in a variety of cancers. MM-121 has failed to meet its main goal in a few of those studies, but Merrimack, for its part, has said that it didn’t expect all of these trials to hit—just that they would help it find out if MM-121 works, and if so, which patients might respond best to the drug. Still, Sanofi’s decision leaves Merrimack without a strategic partner to take the drug into Phase 3 testing.

—It seemed only a matter of time until Cambridge-based Sage Therapeutics would take itself public, and sure enough, this week the company outlined plans to raise up to $69 million through an IPO to develop its drugs for rare central nervous system disorders. Third Rock, which started the company up in 2010, holds 58.5 percent of Sage’s stock. The company will trade under the ticker symbol “SAGE” if it completes the offering.

—New York-based Pfizer (NYSE: PFE) jumped into the chimeric antigen receptor, or CAR-T, chase this week, cutting a deal with French biotech Cellectis to develop more than two dozen therapies designed to turn the immune system’s T cells into cancer killers. Pfizer has paid Cellectis $80 million up front, bought a 10 percent stake in the biotech at a 50 percent premium, and agreed to shell out as much as $185 million per drug in development. Novartis and big Seattle startup Juno Therapeutics lead the emerging field of CAR-T therapies, but Cellectis’ approach is different. Rather than taking cells from specific patients, augmenting them, and reinfusing them into those same patients, Cellectis’ plan is to use T-cells from a single donor in multiple patients. Like the rest of the CAR-T field, these efforts are very early—Cellectis doesn’t have a prospective CAR-T therapy in clinical trials as of yet.

—Summit, NJ-based Celgene (NASDAQ: CELG) and Patrick Soon-Shiong have cut another deal. Just a few months after teaming with Shiong’s Los Angeles-based NantBioscience to develop nanoparticle albumin-bound cancer drugs and putting $75 million into the company, Celgene has poured $25 million into another Shiong entity, NantHealth, a healthcare cloud-based IT company developing an operating system to help with cancer diagnosis and treatment. Celgene and Shiong first teamed up when the big cancer drugmaker bought Shiong’s Abraxis Bioscience for $2.9 billion in 2010. It got protein-bound paclitaxel (Abraxane) out of the deal.

—Celgene also showed more support for Agios Pharmaceuticals (NASDAQ: AGIO), exercising an option to grab worldwide rights to the Cambridge-based company’s blood cancer drug, AG-221. Agios is still eligible to receive up to $120 million in milestone payments and royalties on sales of AG-221, and has an option to help commercialize the drug in the U.S. Agios also provided updated data from an ongoing study of AG-221. I covered the initial results from that study in April.

—Boston-based Vertex Pharmaceuticals (NASDAQ: VRTX) continued to sharpen its focus on cystic fibrosis this week, licensing a prospective influenza drug called VX-787 to Johnson & Johnson (NYSE: JNJ) subsidiary Janssen Pharmaceuticals. Vertex received a $30 million cash payment up front, and is eligible to receive additional unspecified milestone and royalty payments. Vertex wrapped up a Phase 2a study of VX-787 last year.

—Sanofi and New York-based Regeneron Pharmaceuticals (NASDAQ: REGN) provided updated data from one of the six Phase 3 studies of their rheumatoid arthritis antibody drug prospect, sarilumab, at the European League Against Rheumatism’s annual meeting in France. The two companies released initial data from the study last fall, but added new numbers this week showing that the response rates seen in rheumatoid arthritis patients have largely held up over 52 weeks of treatment. Sarilumab is one of several drugs Sanofi and Regeneron are working on together, including dupilumab, a prospect for allergic asthma, and cholesterol-lowering drug alirocumab.

—Fresh off raising a massive $200 million private financing round, Hayward, CA- and Boston-based Intarcia Therapeutics presented the first data from a 60-patient Phase 3 study of ITCA 650, its drug/device combination therapy for type 2 diabetes, at the American Diabetes Association’s annual meeting in San Francisco. Intarcia said after six months, ITCA 650 helped lower the blood sugar levels of 78 percent of the patients in the study by at least 2 percent, 50 percent of them by 3 percent, and 22 percent of them by at least 4 percent. Intarcia expects to report full results from the study in September.

—Watertown, MA-based Selecta Biosciences made a flurry of moves this week. The startup nabbed about $9 million in grants from the Bill & Melinda Gates Foundation and the National Institutes of Health, cut a deal with China’s 3SBio to make a souped-up version of its gout drug, pegsiticase, and revealed new programs that it’s developing for hemophilia A, life-threatening allergies, and type-1 diabetes. Selecta came out of the lab of MIT professor Bob Langer a few years ago with a plan to develop polymer nanoparticle vaccines that are supposed to selectively target a subset of white blood cells, and thus elicit more powerful immune responses than traditional vaccines while limiting side effects.

—Shares of Monmouth Junction, NJ-based Insmed (NASDAQ: INSM) soared more than 40 percent after the FDA unexpectedly granted a “breakthrough therapy” designation to the company’s inhalable antibiotic, liposomal amikicin (Arikayce), as a treatment for nontuberculosis mycobacterial lung infections. The FDA decision, based on the Phase 2 results Insmed revealed earlier this year, means the drug will get a speedier review from the agency than it otherwise would have. Insmed’s drug prospect has seen its share of ups and downs over the years. It faced a clinical hold in 2011, and actually failed to meet its main goal in the Phase 2 study that led to this week’s FDA decision (Insmed said at the time that the drug succeeded by a different, secondary measure that it hadn’t expected to hit, yet found more significant).

—Cambridge-based BIND Therapeutics (NASDAQ: BIND) added another name to the list of partners tapping into its nanoparticle technology to develop drugs—this time, Roche, which will work with the company to create nanodrugs in therapeutic areas outside of oncology. Financial terms of the deal weren’t disclosed. Bind already has deals in place with Pfizer, AstraZeneca, and Amgen.