East Coast Biotech Roundup: Maraganore, Flex, NPS, Retrophin, & More

Xconomy Boston — 

It’s back to work, back to school, and back to the grind in biotech. One local company inched towards a potential FDA approval, yet its stock is down more than 10 percent. Another biotech came under fire for a big price hike on an old drug. And a new startup emerged with the backing of a well-known Boston biotech entrepreneur. Those stories and more below.

—John Maraganore is best known as the CEO of Alnylam Pharmaceuticals (NASDAQ: ALNY), the biggest developer of RNA interference drugs in the world. But it wasn’t too long ago that Maraganore was a young scientist forced to make the transition from the lab to the business world after the drug he designed—an anticoagulant called bivalirudin—failed a clinical trial. I profiled Maraganore’s rise through the biotech ranks, the critical moment that changed his life, and the ups and downs he faced on the way to leading Alnylam.

—Serial biotech executive Christoph Westphal raised $40 million from a diverse group of big-name investors to support his latest biotech startup, Boston-based Flex Pharma. Westphal’s Longwood Fund, Lightstone Ventures ,Bessemer Venture Partners, Patriots owner Bob Kraft (through the Kraft Group), and Celtics CEO and managing partner Wyc Grousbeck are among the investors in Flex, which aims to develop prescription drugs and medical foods to treat various types of muscle cramps.

—Bedminster, NJ-based NPS Pharmaceuticals (NASDAQ: NPSP) has inched closer to its second marketed drug, but not without lingering questions about its commercial potential. On one hand, an FDA advisory panel late Friday voted in favor of NPS’s engineered version of human parathyroid hormone, known as Natpara. On the other hand, the vote was close—8 to 5 in favor of the hormone—and several of the experts that voted positively admitted to being “on the fence,” according to a note from Leerink Partners analyst Joseph Schwartz. Schwartz added that if approved for hypoparathyroidism, the hormone would likely have a warning label about the risk of bone cancer. The FDA, which looks to its advisory panels for input, is set to make a decision on the NPS drug by Oct. 24, though Schwartz noted that ruling could be pushed back for discussions about the drug’s label. Shares of NPS were down more than 12 percent in early trading Monday.

—Waltham, MA-based Tesaro (NASDAQ: TSRO) filed an application with the FDA to approve rolapitant, its prospective treatment for chemotherapy-induced nausea and vomiting. If approved, rolapitant would compete with a number of other medications for chemo-induced nausea, like Merck’s aprepitant. You can read more about Tesaro and rolapitant here.

—Cambridge, MA-based Biogen Idec (NASDAQ: BIIB) and partner AbbVie (NYSE: ABBV) presented the full results from a Phase 3 study of daclizumab (Zinbryta), a once-monthly antibody drug for the most common form of multiple sclerosis. Biogen and AbbVie intend to seek regulatory approval of daclizumab next year. The drug would compete with a number of MS treatments, among them Biogen’s own blockbuster oral pill dimethyl fumarate (Tecfidera). AbbVie acquired rights to daclizumab when it bought Facet Biotech in 2010, edging out Biogen—a longtime partner of Facet on daclizumab—in the process.

—New York-based Retrophin (NASDAQ: RTRX) stoked a firestorm of criticism after raising the price of tiopronin (Thiola), a treatment for cystinuria (a disease that causes frequent cystine kidney stones), from $1.50 per pill to $30 apiece. Tiopronin is an old drug that has long been off patent. But Retrophin acquired the rights to the drug from a Texas company called Mission Pharmacal and has since said it plans to work on new formulations of it. Retrophin CEO Martin Shkreli showed up on a Reddit thread to defend the price increase, arguing, for instance, that the price increase is going to be passed on to insurers and that Mission had issues creating enough of a supply of the drug. You can read a recap of the controversy here from TheStreet.com and Derek Lowe.

—Berkeley Heights, NJ-based Edge Therapeutics secured $10 million in venture debt financing from Hercules Technology Growth Capital to continue developing EDG-1962, a reformulated version of nimodipine it’s developing to treat delayed complications from brain hemorrhages. Edge expects to produce top-line data from a mid-stage study of EDG-1962 next year. I profiled Edge and its plans in 2013.

—Cambridge, MA-based Akashi Therapeutics paid an undisclosed sum to acquire a potential Duchenne Muscular Dystrophy treatment from Williamsville, NY-based Tonus Therapeutics. GsMTx-4, currently in preclinical testing, is a peptide drug intended to address the imbalance of calcium levels in the muscles of patients with Duchenne. It was first discovered by researchers at SUNY Buffalo. Tonus would get certain unspecified milestone payments should the drug advance in clinical trials. You can read more about Akashi, a company formerly known as DART Therapeutics founded by a group of disease foundations, here.

—New York-based Ophthotech (NASDAQ: OPHT) bagged a $50 million milestone payment from Novartis after reaching an enrollment goal in its Phase 3 clinical program for Fovista, its prospective treatment for age-related macular degeneration. Ophthotech cut a $1 billion-plus deal with Novartis on the drug in May, and stands to receive another $80 million solely tied to enrollment milestones as part of the agreement.