East Coast Biotech Roundup: Schenkein, PureTech/JDRF, Sage, & More

Xconomy Boston — 

(Updated, 7:42 a.m. ET) This week’s East Coast biotech news runs the gamut. A career profile, and disappointing (and encouraging) clinical trial results. A new partnership structure, a new financing round, and a big FDA panel setback—even a Chapter 11 case to boot. Those stories and more below:

—You might know that David Schenkein is the CEO of Cambridge, MA-based Agios Pharmaceuticals (NASDAQ: AGIO), or even that he used to run clinical development for the big cancer drugmaker, Genentech. But I doubt you know the story that brought him from all the way from a small apartment in Forest Hills, NY, to the head seat of Agios, one of the nation’s most closely-watched biotechs. I spoke with Schenkein at length recently, and profiled his rise through the industry ranks, as well as his lofty ambition to build Agios into the next Genentech.

—Boston-based PureTech Ventures secured a $5 million investment from New York-based JDRF (formerly known as the Juvenile Diabetes Research Foundation) to form T1D Innovations, a company-creation vehicle that will help spawn startups with innovative therapies for type 1 diabetes. While the focus of the deal is diabetes, the goal, really, is to help usher scientific ideas through their infancy so they get developed. PureTech partner David Steinberg told me about the model the firm is using to do this—making a non-profit entity an investor in a startup-creator without jeopardizing its tax-exempt status.

—Cambridge-based Sage Therapeutics raised a $20 million Series B round of equity financing to help get its first experimental CNS drugs into clinical trials. New investor Arch Venture Partners joined with founding backer Third Rock Ventures to provide the financing. I spoke with CEO Jeffrey Jonas, an ex-Shire executive, about the company’s plans.

—(Updated with new item) A week after suspending a late-stage study for its leukemia drug, ponatinib (Iclusig), due to safety concerns, Cambridge-based Ariad Pharmaceuticals (NASDAQ: ARIA) has scrapped the trial altogether. Ariad’s stock plummeted more than 60 percent last week after it revealed that patients taking its drug in the trial were experiencing blood clots at a higher-than-expected rate. Ariad had been hoping to prove that ponatinib can treat chronic myeloid leukemia more effectively than the standard of care, imatinib (Gleevec). It’s currently just a third-line treatment for CML.

—Bridgewater, NJ-based Savient Pharmaceuticals (NASDAQ: SVNT) completed its precipitous three-year plunge this week, fizzling into Chapter 11 protection with a plan to sell itself for about $55 million to St. Matthews, KY-based US World Meds. Savient’s stock was worth more than $20 per share back in 2010, when the FDA first approved its gout drug, pegloticase (Krystexxa). Unfortunately, legal papers from the case show that Savient miscalculated its market, sales flopped, and now it’s hoping a bankruptcy sale can save it from outright liquidation.

—New York-based Regeneron Pharmaceuticals (NASDAQ: REGN) and Sanofi this week produced the first late-stage data to date from their closely-watched cholesterol-lowering drug candidate, alirocumab. The results were encouraging: the drug, on average, lowered patients’ low-density lipoprotein (LDL) cholesterol levels more than Merck’s ezetimibe (Zetia). But Regeneron and Sanofi still have a long way to go. The results only counted 103 patients—the entire program will test over 23,000.

—Shares of Bedminster, NJ-based Amarin (NASDAQ: AMRN) fell off a cliff this week after an FDA advisory panel voted 9 to 2 against expanding approval for the company’s fish oil pill into people with mixed dyslipidemia—or, triglyceride levels between 200 milligrams/deciliter of blood and 500 mg/dl. The panel instead advised Amarin to complete its “Reduce-It” study first. That study essentially answers whether Amarin’s pill, sold as Vascepa, really leads to less heart attacks and strokes in that patient group. Reduce-It, however, won’t yield results until 2016. Amarin’s shares dropped more than 60 percent on the news.

—Burlington, MA-based Coronado Biosciences (NASDAQ: CNDO) also felt investors’ wrath this week. Its shares plunged more than 65 percent after its experimental drug for Crohn’s Disease, CNDO-201, flopped in a mid-stage clinical trial. Coronado’s drug candidate, which contains tiny eggs from a parasite found in pigs, missed both its primary and secondary goals in the study and didn’t fare any better than the placebo it was tested against.

—Bedminster-based Aerie Pharmaceuticals set a range for its coming IPO this week. The company, a developer of glaucoma drugs, plans to sell 5.25 million shares to investors at between $12 and $14 apiece, good for just over $68 million should it price in the middle of its projected range. Aerie aims to list on the Nasdaq under the ticker symbol “AERI.”