Big ambitions, and creative, risk-sharing deals—two concepts that often go hand in hand with biotech. They’re also the themes of two of the big stories on the East Coast this week. Those and plenty more below.
—Drug discovery is expensive, time-consuming business. Framingham, MA-based Berg Pharma thinks it’s found a way to make it quicker and cheaper. Backed by billionaire Carl Berg and based around a system that combines biological models, big data analytics, artificial intelligence, and a whole variety of omics, Berg has built itself into a roughly 200-person company with three divisions and some ambitious goals. Now the question is whether Berg can deliver on its promise of efficiency and create drugs that really make a difference. I spoke with Berg president and chief technology officer Niven Narain and adviser (and genomics leader) Eric Schadt about the venture, and the challenges that lie ahead.
—When Watertown, MA-based Forma Therapeutics introduced itself to Summit, NJ-based Celgene (NASDAQ: CELG) last year, it didn’t just add another drug discovery partner—it found what may be its future buyer as well. The two companies this week cut a big deal that could not only see Forma receive up to $600 million in new non-dilutive cash, but also potentially lead to it being acquired by Celgene down the road at a price that depends on the value Forma creates over time. I spoke with Forma CEO Steven Tregay about the creative, multi-stage deal structure, and the mechanisms in place to protect Forma should Celgene decide to walk away.
—Hayward, CA and Boston-based Intarcia Therapeutics burst on the scene a few years ago with a $210 million private financing round to develop an implantable drug/device combination therapy for type 2 diabetes. With that treatment, known as ITCA 650, currently going through a large Phase 3 clinical trial, Intarcia loaded up this week with another big helping of cash. It raised $200 million in equity financing led by new investor RA Capital and including help from Farallon Capital Management, Foresite Capital, Franklin Templeton, Fred Alger Management, New Leaf Venture Partners, Quilvest, and three other unspecified “large top-tier institutional investors” that took part in its last financing. Intarcia expects its Phase 3 trial to wrap up in early 2016. ITCA 650 is a tiny, implantable pump that steadily secretes diabetes drug exenatide to keep blood sugar levels in check.
—Foresite Capital poured $57 million into that Intarcia round, and it used cash from its latest fund—a $300 million fund launched this week—to do it. Foresite started up Foresite Capital II, a fund specifically earmarked for investments in established, late-stage biotechs and other healthcare companies. The fund follows up Foresite’s first effort, a $100 million fund it raised in January 2013 for the same purpose. A number of Foresite’s investments are, accordingly, well along in their development, like Epizyme (NASDAQ: EPZM), Karyopharm (NASDAQ: KPTI), and Orexigen Therapeutics (NASDAQ: OREX).
—Blueprint Health, the New York-based healthtech accelerator, unveiled its latest class of graduates this week, many of which have developed applications they’ll try to sell to hospitals. I attended the demo day Thursday at the City Winery in SoHo. Here’s my rundown of the new class.
—New York legislators gave the area’s life sciences scene a funding jump this week, committing up to $160 million of the state’s budget to initiatives in genomics and health tech. Lawmakers set $105 million of that funding for a partnership between the New York Genome Center and the University of Buffalo’s Center for Computational Research, a pact that enables the NYGC to tap into the CCR’s large-scale computing capabilities. The remaining $55 million will help complete the Statewide Health Information Network of New York, or SHIN-NY, a centralized system of electronic health records for the state.
—Lexington, MA-based Curis (NASDAQ: CRIS) got some good news from the FDA this week, as the agency lifted a partial clinical hold on the company’s experimental cancer drug, CUDC-427. With the hold lifted, Curis can now proceed with the early-stage trial that the FDA halted in November. The agency stopped the trial for safety precautions—the liver enzymes of one patient taking CUDC-427 spiked during treatment, and didn’t subside after discontinuing the drug. That patient died about a month later. Curis has amended the protocol for the trial, but hasn’t specified how as of yet.
—New York’s Weill Cornell Medical College has struck a three-year deal with Belgian drugmaker UCB to try to move forward three programs coming out of the university’s research in bone disorders, metabolic disease, and genetics. One project deals with ways to build up bones and treat people with disorders like osteoporosis. Another is looking into how the body becomes insulin resistant. And a third study centers around genetic variations that lead to immunological disorders like rheumatoid arthritis. Researchers from both organizations will work together on advancing the projects.
—An FDA advisory panel this week unanimously voted in support of approving Lexington-based Cubist Pharmaceuticals’ (NASDAQ: CBST) antibiotic for bacterial skin infections, tedizolid. The FDA will decide whether to approve the drug—to be sold under the name Sivextro—by June 20. Cubist acquired tedizolid when it bought Trius Therapeutics last year.
—Celgene snapped up 1.1 million more shares of Acceleron Pharma (NASDAQ: XLRN), giving the Cambridge-based company about $47.1 million in new cash. Celgene now has a 14.8 percent stake in Acceleron, which went public in September. Celgene had a 12.3 percent stake before Acceleron’s IPO. The two are partners on a couple of experimental drugs Acceleron is developing for anemia, multiple myeloma, and other diseases. Shares of Acceleron jumped about 15 percent on the news.
—The high-stakes PCSK9 race took center stage earlier this week at the annual meeting of the American College of Cardiology. New York-based Regeneron (NASDAQ: REGN) (and partner Sanofi), and Thousand Oaks, CA-based Amgen (NASDAQ: AMGN) both presented updated, similarly promising results from the ongoing studies of their prospective cholesterol-lowering PCSK9 blockers. The belief is that PCSK9 blockers can be a valuable option when combined with statins, or for those who don’t respond well to them—a potentially huge market. These drug candidates are proving they can effectively lower low-density lipoprotein (LDL) or “bad” cholesterol on their own and with statins, but it’s still an open question whether payers will pay up for PCSK9 blockers before seeing the results of ongoing long-term “outcomes” studies which should indicate whether these drugs lead to fewer heart attacks. Amgen looks to be in position to find out first—it reported the details of its latest arm of its Phase 3 study, and expects to file an application for approval by the end of the year.
—Cambridge-based Biogen Idec (NASDAQ: BIIB) won FDA approval of its long-acting hemophilia B drug, which the company will sell under the brand name Alprolix. The drug is to be administered once a week, compared to current treatments like Baxter International’s Advate, which requires an infusion every two or three days. Baxter is also developing a longer-lasting hemophilia B treatment, as is Novo Nordisk.
—Rockland, MA-based EMD Serono, the U.S. drugmaking arm of Merck KGaA, has teamed with The Broad Institute of MIT and Harvard and Pfizer to try to unearth new treatments for lupus. The Broad Institute will analyze genetic samples from lupus patients and use computer models to attempt to unearth new drug targets and find biomarkers and molecular drivers of the disease. EMD and Pfizer will get access to all the data and analysis, and can send over their own scientists to chip in on the work.
—Watertown, MA-based EnVivo Pharmaceuticals, the Alzheimer’s and schizophrenia drug developer that ex-Millennium chief Deborah Dunsire took over last year, has changed its name to Forum Pharmaceuticals. The newly-named Forum said the change doesn’t represent a change in organizational structure or to its investor base.