In early 2017, Scott Gottlieb was considered the most moderate of the potential candidates to head the Food and Drug Administration, which is the most powerful regulator of medical products in the world.
It wasn’t a high bar to clear. Libertarians who had questioned some of the basic premises of the FDA were in the mix, and even their laissez-faire attitudes had some advocates of a speedier agency worried, as Xconomy reported in early 2017.
Still, Gottlieb had spent time in the George W. Bush FDA, then years as a life-sciences investor and conservative op-ed writer. His potential financial conflicts, combined with President Trump’s vow to slash FDA rules, made Democrats and others shy away.
Other than the age-old “spending more time with family” (Gottlieb and his wife have three kids under 10, including twins), no clear reason has emerged as of late Thursday. He took on the tobacco-vape industry and, in denouncing anti-vaccine activism, has clashed with a movement that President Trump himself has encouraged. But there’s no indication those stances led to his departure. The agency under Gottlieb won biopharma industry kudos for continuing Obama-era reforms to speed reviews and clarify regulation of new kinds of medicines, like gene therapy. But it also moved for new rules against Big Pharma tactics that block generic competition.
The announcement sent biopharma indices south. The drug industry and its investors hate uncertainty. And now that there’s yet another personnel hole that the Trump administration needs to fill, there’s uncertainty in buckets.
Any interest? You can apply now. Or you can apply yourself to the rest of this week’s roundup.
LAW, POLICY, AND PRICES
—Amid several US measles outbreaks, the Senate’s health committee held a hearing on vaccines. The opening statement from chair Lamar Alexander (R-TN) echoed Gottlieb’s 2017 confirmation-hearing remarks before the same committee. Saying “vaccines save lives,” Alexander lit into antivaccine activists and the information they spread online: “Just because you found it on the Internet doesn’t make it true.”
—Purdue Pharma is exploring bankruptcy to manage mounting lawsuits that claim the drug maker contributed to the nation’s opioid crisis, Reuters reported.
—Amid new generic competition and scrutiny of rising drug prices, Eli Lilly (NYSE: LLY) announced an authorized generic version of its fast-acting insulin Humalog that will sell for half the price.
—A bipartisan Senate bill emerged this week to clear up the so-called “patent thickets” that, as Gottlieb has previously said, large drug makers accumulate only to deter biosimilar versions of their protein-based drugs.
—The Wall Street Journal reports that Martin Shkreli is running his old drug company from his prison cell in New Jersey.
DEALS, DEALS, DEALS
—Biogen (NASDAQ: BIIB) will pay $800 million to buy Nightstar Therapeutics (NASDAQ: NITE), a developer of gene therapies for rare eye diseases. The acquisition is part of a wave of gene therapy deals and comes weeks after Biogen dumped a partnership with AGTC (NASDAQ: AGTC), a Nightstar rival.
—Drama surrounding Bristol-Myers Squibb’s (NYSE: BMY) proposed buyout of Celgene (NASDAQ: CELG) continued. Bristol defended the deal amidst mounting opposition, and its executives have met with institutional investors several times the past few weeks, CNBC reported. Meanwhile, the Loncar Cancer Immunotherapy ETF became the third investor to come out against the deal, following Wellington Management and Starboard Value.
—Paris-based Seventure Partners closed a $224 million fund to invest in microbiome therapies, nutrition, and digital health.
—Precision BioSciences of Durham, NC, filed for an IPO to fund human tests of a cancer cell therapy developed with its proprietary gene-editing technology.
—Beam Therapeutics of Cambridge, MA, raised a $135 million Series B round as it pushes ahead with medicines that use base editing, a more precise form of CRISPR gene editing technology.
—San Diego startup Erasca, formed by Ignyta founder Jonathan Lim, added another $22 million to its Series A and provided more insight into its plans to develop targeted cancer drugs with the help of artificial intelligence.
—Pacira Pharmaceuticals of Fremont, CA, is paying $120 million upfront for Myoscience, a maker of a painkiller delivery device. Pacira could pay an additional $100 million.
—Shionogi & Co. will pay Akili Interactive $20 million upfront for rights to sell its digital medicines for ADHD (AKL-T01) and autism spectrum disorder (AKL-T02) in Japan and Taiwan.
CLINICAL YEAS & NAYS
—The FDA approved the Johnson & Johnson (NYSE: JNJ) drug esketamine (Spravato), making the fast-acting nasal spray the first new type of medicine for treatment-resistant depression in decades. The approval, however, comes with a safety warning and restrictions. As a close relation to “Special K,” the addictive club drug, esketamine must be used in a doctor’s office or medical facility. The drug has a wholesale cost of $590 to $885 per dose. Here’s more from Stat.
—U.K. clinicians reported that an HIV patient has gone 18 months without signs of the virus in his blood. This marks the second time—and the first in a decade—that a stem cell transplant appears to have cleared the virus from an HIV patient. (Clinicians are hesitant to use the word “cured.”) The results were published in Nature.
—Alnylam Pharmaceuticals (NASDAQ: ALNY) reported that givosiran, an RNA interference medicine for the rare genetic disease acute hepatic porphyria, succeeded in a Phase 3 trial, but side effects clouded the results, and shares ticked down 4 percent. Alnylam said it would complete U.S. and Europe approval applications by mid-2019.
—Vertex Pharmaceuticals (NASDAQ: VRTX) reported that a cystic fibrosis treatment cocktail hit the main goals in a Phase 3 study. It’s the second triple combination from the company to succeed in clinical testing, and a remarkable turnaround from the company’s days relying on a doomed hepatitis C franchise. The Boston drug maker says it will decide in the second quarter which of the two combinations it will submit for FDA approval.
—Weeks before an expected FDA approval decision, the agency sent Evoke Pharmaceuticals (NASDAQ: EVOK) a letter flagging problems with Gimoti, a formulation of a diabetic gastroparesis drug, and the nasal-spray device that administers it. Shares of thinly traded Evoke plunged more than 120 percent.
—The Syndax Pharmaceuticals (NASDAQ: SNDX) drug entinostat failed early-stage studies in breast and ovarian cancer. The studies were testing the Waltham, MA, company’s drug in combination with Genentech’s atezolizumab (Tecentriq) and Pfizer’s avelumab (Bavencio).
—-Amag Pharmaceuticals (NASDAQ: AMAG) drug hydroxyprogesterone caproate (Makena) failed a post-marketing study needed to retain approval for reducing the risk of spontaneous preterm birth. The FDA awarded the injectable drug accelerated approval in 2011. But on Friday, Waltham, MA-based Amag said its drug failed to outperform a placebo in the post-marketing study. Amag attributed those results to the enrollment of primarily Eastern European patients, a demographic that’s different than the one in the study used for the drug’s initial approval.
PEOPLE ON THE MOVE
—Following recent positive Phase 2 data for its type 2 diabetes treatment, Zafgen (NASDAQ: ZFGN) has hired a top Biogen executive, Priya Singhal, as head of R&D.
—Alessandro Riva, EVP of oncology therapeutics for Gilead Sciences (NASDAQ: GILD), is leaving to become CEO of a Glenmark Pharmaceuticals spinout.
—As DBV Technologies (NASDAQ: DBVT) prepares to refile for FDA approval of its peanut allergy treatment, the French company is losing chief operating officer Charles Ruban and chief development officer Laurent Martin.
Ben Fidler and Frank Vinluan contributed to this report.
Photo courtesy of the US Department of Agriculture.