The biggest yearly meeting for blood disease research has come and gone, and yet the stories to come out of the American Society of Hematology’s gathering in San Diego represent just a fraction of what happened in biopharma this week.
Even if ASH wasn’t on your radar, the CRISPR baby saga—the apparent birth of gene edited twin girls in China that shocked the life sciences world a few weeks ago—rolled on. One massive buyout was signed, and another even bigger one inched closer to being finalized. And then came the largest biotech IPO in history.
We’ve got those headlines and much more below. Let’s round it all up.
ASH Wrap
—ASH featured a slew of news regarding new and emerging treatments for multiple myeloma, a cancer of the bone marrow. Xconomy rounded up the news, including data for several emerging cell therapies and a regimen that could become the new standard of care for many patients with the disease.
—Xconomy also rounded up some of Wall Street’s winners and losers at ASH, highlighting news on treatments for sickle cell disease, lymphoma, beta-thalassemia and more.
CRISPR Baby Fallout
—Xconomy published a conversation with Stanford University professor and gene-editing specialist Matthew Porteus, who has become a key person in the CRISPR baby story.
Chinese researcher He Jiankui, who claimed nearly two weeks ago that his work led to twin girls born immune to HIV infection, told Porteus in February about his plans. Porteus was an organizer of the international meeting last week where He first publicly discussed his claims, and Porteus was one of two people to question He on stage, prodding him to admit there could be a third CRISPR baby on the way.
—Atlantic science writer Ed Yong described He’s claims—which remain unverified—as a “15-part farce.”
—STAT reported this week that one of He’s lab mates about a year ago asked a University of Pennsylvania researcher for help disabling in human embryos the PCSK9 gene, which is related to high cholesterol.
—Amid rumors sparked by reports in China that He has been detained by authorities, who earlier promised a full investigation of his potentially illegal work, the fallout continues. Scientists pored over He’s data and worried that his work was sloppy and potentially dangerous for the girls whose CCR5 gene he tried to alter.
Big Deals & Record IPOs
—Despite the commercial struggles a new class of cancer drugs known as “PARP inhibitors” have had, GlaxoSmithKline (NYSE: [[ticker:GSK]]) bought in this week, paying $5.1 billion for Waltham, MA-based Tesaro (NASDAQ: [[ticker:TSRO]]) and its PARP blocker niraparib (Zejula). The buyout pits GSK against British pharma rival AstraZeneca (NYSE: [[ticker:AZN]]), whose PARP inhibitor olaparib (Lynparza) is the market leader.
—Shire (NASDAQ: [[ticker:SHPG]]) shareholders overwhelmingly approved Takeda Pharmaceutical’s $62 billion buyout of the rare disease drug developer. The companies now say they expect to complete the acquisition on Jan. 8.
—Moderna (NASDAQ: [[ticker:MRNA]]), the high-flying messenger RNA drug developer known for raising billions in financings and drug partnerships, hit Wall Street Friday morning after bagging $604 million in the biggest biotech IPO ever. Now Moderna will have to prove that its clinical data have the substance that can match the hype of its lofty valuation.
—Grail, which is racing to develop a test that can detect the early stages of cancer in otherwise healthy people, might be shifting its financial goals from a Hong Kong IPO to one in the U.S. instead, Bloomberg reported.
Data Dumps
—Shares of San Diego-based Conatus Pharmaceuticals (NASDAQ: [[ticker:CNAT]]) plunged more than 55 percent after the company’s experimental drug emricasan failed its second Phase 2 trial in the fatty liver disease known as NASH. Its first study failed in March; there are two others underway.
—The prospects of AbbVie’s (NYSE: [[ticker:ABBV]]) $5.8 billion buyout of Stemcentrx continued to dim. AbbVie halted a Phase 3 lung cancer study of Rova-T, the crown jewel of the Stemcentrx deal, after a data monitoring board found that patients were living longer on chemotherapy than AbbVie’s drug. Rova-T already failed a mid-stage study in lung cancer. Two other studies in lung and neuroendocrine cancers are ongoing.
—Biohaven Pharmaceuticals (NYSE: [[ticker:BHVN]]) posted positive Phase 3 data of a newer formulation—a rapidly dissolving tablet—of experimental migraine pill rimegepant. Evercore ISI analyst Umer Raffat wrote that the data set the Biohaven drug “at parity” with a rival treatment from Allergan (NASDAQ: [[ticker:AGN]]), ubrogepant, which, like rimegepant, should head to an FDA review next year. Biohaven shares climbed 18 percent.
—Supernus Pharmaceuticals (NASDAQ: [[ticker:SUPN]]) said its experimental drug SPN-812 succeeded in two Phase 3 studies in children with attention deficit hyperactive disorder and the company expects to file for FDA approval next year. But analysts noted that the drug’s effect in the studies was not much different than that of a widely available generic, raising questions about the commercial viability of the SuperNus compound.
FDA News
—The FDA has begun a review of
