Funny thing happened on the East Coast this week. The so-called Blizzard of 2015 threw us New Yorkers into a panic before unexpectedly veering eastward in a post-Christmas miracle and giving the city little more than a dusting. We weren’t relieved; we were livid—at weathermen for scaring us, politicians for shutting the city down for seemingly no reason, and everyone else, just because (we’re never happy).
The storm lived up to the hype in New England, which was belted with up to three feet of snow. Still, at least it gave Bostonians something other than suspiciously deflated footballs to talk about for a few days. There was some biotech news, too: we’ve got it all rounded up below.
—Neurodegenerative disorders like Alzheimer’s and Parkinson’s have bedeviled scientists for decades, largely because there’s so much about the underlying biology that isn’t understood. Cambridge, MA-based Yumanity Therapeutics is trying to crack the code by moving up and down the evolutionary ladder, starting with insights it’s gleaning from a simple organism—yeast. I took an in-depth look at Yumanity’s strategy this week, and the unorthodox, team-first, financing-second way it’s pursuing that plan.
—The most significant clinical trial news of 2015, so far, has been a report from Cambridge-based Biogen Idec (NASDAQ: [[ticker:BIIB]]) about a drug it’s developing not just to slow the progression of multiple sclerosis, but reverse the nerve damage the disease causes. The results weren’t clear-cut; my colleague Alex Lash explained why, and what to look for as Biogen moves the closely watched drug, known as anti-LINGO-1, forward.
—Biogen’s new gene therapy unit was also in the news this week. It’s collaborating with two Italian institutions to develop gene therapies for hemophilia A and B—and using a different approach than its rivals. I spoke to Olivier Danos, the head of Biogen’s gene therapy division, about the thinking behind the deal. Separately, Biogen reported its 2014 financial results, which included $2.9 billion in revenue for its oral MS pill dimethyl fumarate (Tecfidera) and a 40 percent revenue jump from the prior year.
—Creating a novel diagnostic test is one thing. Successfully selling it, and convincing insurers to pay for it (and clinicians to adopt it) is another. Cambridge-based Exosome Diagnostics is at that critical point in its development—and, as CEO Thomas McLain told me, is in the middle of raising $25 million to fund its journey. I explored the challenges that lie ahead for the company.
—Public investors lined up this week to buy into serial biotech executive Christoph Westphal’s latest startup, Flex Pharma (NASDAQ: [[ticker:FLEX]]). It’s not a typical biotech; Flex is starting out developing a dietary supplement to treat muscle cramps made out of three items (extracts of ginger, cinnamon, and capsicum) you can find in your local supermarket. But Flex bagged $86 million in an upsized IPO nonetheless.
—Shares of New York-based Intercept Pharmaceuticals (NASDAQ: [[ticker:ICPT]]) surged more than 20 percent late Thursday after the FDA bestowed a breakthrough therapy designation on obeticholic acid, the company’s closely watched prospective treatment for nonalcoholic steatohepatitis. The designation is a tool to speed the agency’s review of a drug.
—The FDA gave Bedminster, NJ-based NPS Pharmaceuticals (NASDAQ: [[ticker:NPSP]]) and its acquirer, Shire, good news this past week, approving the company’s second drug—an engineered version of human parathyroid hormone known as Natpara—as a treatment for low blood calcium levels in people with hypoparathyroidism. Approval, however, came with a caveat: Natpara’s label came with a warning that rats given the drug in preclinical tests developed bone cancer. As a result, it’s only to be prescribed to people who haven’t responded to treatment with calcium and vitamin D. Shire agreed to buy NPS for $5.2 billion a few weeks ago.
—Tarrytown, NY-based Regeneron Pharmaceuticals (NASDAQ: [[ticker:REGN]]) and its partner Sanofi’s decision to pay BioMarin Pharmaceutical $67.5 million for a priority review voucher allowed it to jump ahead of rival Amgen this week in the race to bring the first in a new class of cholesterol-lowering drugs—which block the protein PCSK9—to market. The FDA accepted their application for a priority review of their drug, alirocumab (Praluent), and will decide on the drug by July 24; the agency is set to make a ruling on Amgen’s treatment, evolocumab, by August 27.
—New York’s Pfizer terminated the $70 million deal it had in place with Waltham, MA-based Repligen (NASDAQ: [[ticker:RGEN]]) to develop drugs for spinal muscular atrophy.
Photo courtesy of Flickr user Shane Ford via Creative Commons.
