Happy new year, everyone. Are you all strapped in? Tray tables in the locked and upright position? As Bette Davis once said, “It’s going to be a bumpy night.”
The news from Washington is already coming fast and furious. The effort to repeal Obamacare is underway, spawning more perspectives and opinions than a postmodern Japanese samurai-noir flick. Support for Planned Parenthood could be at stake, too.
With massive changes looming for the FDA, thanks to last year’s 21st Century Cures Act and this year’s Republican control of the White House and Congress, a top FDA drug evaluator touted the agency’s 2016 approval record—roughly half the annual volume the agency approved in 2014 and 2015. It’s been a month since incoming President Donald Trump’s transition team floated libertarian Jim O’Neill as a potential FDA chief. Other nominees have cropped up since, but there’s still no official word of a nominee.
Ripples from one of FDA’s 2016 approvals—the Duchenne muscular dystrophy drug eteplirsen (Exondys 51)—washed into the new year. As our Ben Fidler reports, another treatment for a rare disease has gained approval, but patients with the disease, spinal muscular atrophy, and their families remain uneasy about getting access to it.
Meanwhile, another rare disease drug developer, Alexion, admitted that its former executives—now spending more time with their families—pressured employees to fiddle with sales reports. (As if its lead product Soliris’s $500,000 annual cost wasn’t enough.)
There was plenty of other drug-price news, as we’ll see, as well as high profile personnel moves and funds sloshing around. Let’s get to the first roundup of 2017.
—Several reports about drug price hikes emerged this week, underscoring the importance of the subject to the American public (not to mention the drug industry). After its CEO Brent Saunders pledged last year to put limits on its increases, Allergan (NYSE: AGN) reportedly kept to his word by keeping hikes on nine drugs under 10 percent. Outgoing Vice-President Joe Biden said he would form a nonprofit to work on cancer—and press for affordable drugs—when he leaves office.
—Over the holiday break, Biogen (NASDAQ: BIIB) priced the first-ever approved spinal muscular atrophy drug, nusinersen (Spinraza), at $750,000 for the first year of treatment and $375,000 for each year thereafter. The price underscores the increasing costs of rare disease drugs. SMA patients and their families are anxious about getting access to the drug, despite the FDA’s enthusiastic recommendation. It remains to be seen if insurers will push back on nusinersen the way they have with another recently approved rare disease therapy, eteplirsen (Exondys 51).
—A district court judge banned Regeneron Pharmaceuticals (NASDAQ: REGN) and partner Sanofi from selling their cholesterol lowering drug, alirocumab (Praluent), in the U.S., ruling that it infringes on a patent Amgen (NASDAQ: AMGN) has on rival evolocumab (Repatha). The ruling won’t take effect for 30 days, however, during which time Regeneron and Sanofi can appeal or settle with Amgen. Both drugs have struggled mightily with sales while waiting for the results of huge studies showing whether they lead to better health outcomes. But should the decision hold up and the outcomes studies prove positive—data are expected this year—Amgen could see a huge windfall. Here’s more from the Wall Street Journal.
—Reports emerged last month, and cancer diagnostic firm Grail confirmed yesterday it wants to raise more than $1 billion to develop a blood test that screens for cancer in people who otherwise seem healthy. Grail is among several companies developing so-called liquid biopsies. Some are using the tests to track patients who have already been diagnosed with cancer. Screening healthy patients poses a different set of risks.
—Former National Institutes of Health director Harold Varmus and onetime Vertex Pharmaceuticals and Biogen executive Vicki Sato are co-chairing a advisory committee for LifeSci NYC, the $500 million government plan to spur life sciences in New York. Xconomy spoke with Varmus and Sato last week.
—Former Biogen CEO George Scangos has returned to lead a new San Francisco, CA-based startup, Vir Biotechnology, that aims to battle infectious diseases. Arch Venture Partners has committed $150 million to the firm. Forbes has more here.
—Cambridge, MA-based Neon Therapeutics raised $70 million in a Series B round that it will use to continue its work on personalized cancer vaccines.
—The Bill & Melinda Gates Foundation put $140 million in grant and equity funding into Intarcia Therapeutics to develop an implantable drug-delivery device for HIV.
—Synthego raised $41 million to bolster its business of providing synthetic parts of the CRISPR-Cas9 genome editing tool. CRISPR-Cas9 pioneer Jennifer Doudna, a UC Berkeley professor, was among the investors.
…AND FUNDS FOR THOSE WHO FUND
—Versant Ventures closed its sixth fund at $400 million. The San Francisco-based firm has incubator and laboratory spaces in San Diego, Vancouver, Toronto, Montreal, and New York, and it plans to open a new outpost in Basel, Switzerland.
—Hatteras Venture Partners closed its fifth fund at $150 million. The Durham, NC-based venture capital firm previously told Xconomy that in addition to seeking early stage biotech and medical device startups, this fund would also pursue health IT investments.
—Digitalis Ventures said it had begun investing its inaugural $100 million fund in healthcare companies that have a big data foundation.
DEALS AND POSSIBLE DEALS
—Carlsbad, CA-based Ionis Pharmaceuticals (NASDAQ: IONS) got $75 million up front from Novartis in a deal that gives the Swiss firm an option to license two experimental heart disease drugs after they complete Phase 2 trials.
—PvP Biologics, a University of Washington spinout, inked a $35 million deal with Takeda Pharmaceutical for its celiac disease treatment.
—Cambridge startup RaNA Therapeutics cut a deal to grab rights to the messenger RNA therapeutics division that Shire (NASDAQ: SHPG) shuttered in 2016. The move puts RaNA into an emerging group of mRNA drug developers, among them nearby Moderna Therapeutics.
—The first test of the biotech IPO market in 2017 could come from Cambridge, MA-based Jounce Therapeutics, which outlined plans to go public to fund its cancer drug work.
YOU SAY GOODBYE, I SAY HELLO
—David Epstein, the former CEO of Novartis’s U.S. pharma business, joined venture firm Flagship Pioneering as an executive partner. His first post is as chairman of Flagship startup Rubius Therapeutics, which launched in late 2015.
—Craig Venter ceded the CEO chair of Human Longevity to Cynthia Collins, who was previously at GE Healthcare. Venter, who recently had prostate cancer surgery, will remain executive chairman.
TRIALS AND TRIBULATIONS
—Cempra (NASDAQ: CEMP) failed to get FDA approval for its pneumonia antibiotic. The regulator told the Chapel Hill, NC-based biotech it must conduct another trial enrolling more than 900 patients to address liver-damage risks associated with the drug.
Ben Fidler and Frank Vinluan contributed to this report.