Bio Roundup: $74B for Celgene, Two CAR-T Tales, Ready for SF & More

Xconomy National — 

In 2018, my Exome colleagues and I published hundreds of stories about health, medicine, the biopharma industry, government policy, and more. You’ll find a few of our favorite stories in this review of some of the year’s best from across the Xconomy network. Topics ranged from the worries over CRISPR genome editing and Wild West biohacking, to Nobel Prize-fueled drug approvals and the headaches around drug prices (even for drugs meant to cure headaches).

What will make our Best of 2019 list? You can help with that by sending us frequent feedback via email (first initial-last and Twitter. Why not start right away? This week, we have coverage of the proposed $74 billion buyout that is sending shockwaves around the biopharma world, and we take a side-by-side look at the different fates of the first two live cell therapies known as CAR-T to gain approval, now that they’ve both been on the market for more than a year.

Typical for the days leading up to the annual J.P. Morgan healthcare conference, there’s plenty more news to digest. My colleagues and I will be roaming the halls of the conference and the surrounding San Francisco sidewalks next week. If you see us there, give us your feedback in person—and perhaps in between raindrops. Meanwhile, let’s get to the roundup.


—In one of the biggest pharma blockbusters ever, Bristol-Myers Squibb (NYSE: BMY) agreed to buy Celgene (NASDAQ: CELG) for $74 billion to create an oncology behemoth, a daring bet on the many creative alliances Celgene has forged with smaller biotechs. Both companies have seen their share prices plunge the past year due to various pipeline failures.

—It’s a tale of two CAR-Ts. Yescarta and Kymriah, the first cutting-edge live cell therapies to gain historic approval in 2017, have had different fates so far. Robust real-world use of Yescarta has given doctors hope that dangerous CAR-T side effects can be managed effectively. Hard to tell with Kymriah, however, because it has been dogged by manufacturing problems.

— Verily, formerly known as Google Life Sciences, raised $1 billion from sources that include private equity firm Silver Lake and the Ontario Teachers’ Pension Plan. It remains a subsidiary of Alphabet, Google’s parent company. The firm is working on a multiyear health study called Baseline and other experimental health projects.

Atlas Venture raised a new $250 million fund to invest in its portfolio companies that have progressed to a Series B round and beyond. The fund complements Atlas’s existing funds, which it uses to seed new biotechs.

—MyoKardia (NASDAQ: MYOK) and Sanofi (NYSE: SNY) ended a 2014 alliance, which means the South San Francisco, CA, company reclaims rights to a group of heart disease drugs that it will develop on its own. CEO Tassos Gianakakos said the move will help MyoKardia build a company “for the long haul.” Shares fell roughly 20 percent.


—Despite growing outcry against the high cost of drugs, several drug companies stuck to tradition and raised the prices of hundreds of medicines on January 1. List price increases were generally less than 10 percent, but Allergan led the way with hikes of 9.5 percent, according to the Wall Street Journal. Expect more increases this month.

—In late December, more opposition emerged to the Trump administration’s proposal to lower Medicare drug costs by tying them to international prices. First announced in October, the plan quickly drew fire from the drug industry. Now the American Hospital Association, the American Medical Association, and others say it could reduce access to drugs and increase administrative burden. FiercePharma has an overview.

—FDA commissioner Scott Gottlieb said this week that the government shutdown “could lead to delays in FDA decision-making” on drug and device approvals and stymie other agency activities. Regulatory Focus has more.


—AbbVie (NYSE: ABBV) paid $105 million upfront to Tizona Therapeutics for development rights to the South San Francisco, CA, biotech’s lead drug, an immunotherapy that targets the cancer protein CD39.

—Esperion Therapeutics (NASDAQ: ESPR) got $300 million up front from Daiichi Sankyo for rights in Europe to bempedoic acid, the Ann Arbor, MI, company’s experimental drug for high cholesterol. Esperion could file for approval of bempedoic acid in the U.S. and Europe this year and begin selling the drug in 2020.

—Biogen (NASDAQ: BIIB) announced two deals with small biotechs. It will work with C4 Therapeutics to co-develop drugs for neurological diseases like Alzheimer’s and Parkinson’s, promising the startup a total of $415 million in “upfront and potential future milestone payments.” It will also pay Skyhawk Therapeutics $74 million up front to work on small molecule drugs for spinal muscular atrophy, multiple sclerosis, and more. Here’s more on C4 and Skyhawk.

—Separately, C4 also expanded an ongoing partnership with Roche.

—Before the door closed on 2018, the biggest year for biotech IPOs in recent memory, three more companies got their paperwork over the transom: Stealth Biotherapeutics, TCR2 Therapeutics, and Harpoon Therapeutics.

—Cambridge, MA-based Tiburio Therapeutics emerged from biotech startup accelerator Cydan with $31 million in Series A financing to take its experimental peptide drug for pituitary tumors into Phase 2 testing.

—For $20 million, Merck (NYSE: MRK) has exercised an option to license NGM313, an experimental treatment for NASH and type 2 diabetes, from NGM Biopharmaceuticals.

—A Johnson & Johnson (NYSE: JNJ) subsidiary is paying Locus Biosciences $20 million upfront to use the Research Triangle Park, NC, company’s CRISPR-Cas3 technology to develop bacteriophage treatments for infections.

—Jazz Pharmaceuticals (NASDAQ: JAZZ) of Dublin, Ireland is paying Cambridge, MA-based Codiak Biosciences $56 million upfront to tap into Codiak’s oncology drug development program.

—Cabaletta Bio, a cell therapy startup spun out of the University of Pennsylvania, raised a $50 million Series B. Cabeletta announced a $38 million Series A in November.

—South San Francisco, CA-based Veracyte (NASDAQ: VCYT) will get backing from Johnson & Johnson to develop a nasal swab test for the early detection of lung cancer.

—Flex Pharma (NASDAQ: FLKS) will merge with privately held, Houston-based cancer drug developer Salarius Pharmaceuticals after a series of clinical setbacks and strategic restarts.

—Mersana Therapeutics (NASDAQ: MRSN) will discontinue development of experimental cancer drug XMT-1522, which has struggled with safety issues, and end a related alliance with Takeda Pharmaceutical. Mersana will put its resources behind another cancer drug, XMT-1536.

—Selecta Biosciences (NASDAQ: SELB) will ax 36 percent of its workforce in a restructuring.

—“Digital therapeutics” startup Pear Therapeutics raised $64 million, a Series C financing that comes less than a month after the FDA cleared the company’s mobile app as a treatment for opioid abuse disorder.


—Juno Therapeutics co-founders Steve Harr and Hans Bishop have teamed up again to start up a new Seattle-based cell therapy developer, Sana Biotechnology, backed by Arch Venture Partners and F-Prime Capital.

—San Antonio physician Steven Venticinque used a 3D printer in his garage and $15,000 of his own money to start a medical device company, Olifant Medical.

—Elizabeth Barrett, former head of Novartis Oncology, is joining New York-based UroGen Pharma (NASDAQ: URGN) as president and CEO.

—SQZ Biotechnologies of Watertown, MA, has named Oliver Rosen chief medical officer, a post he held previously at Deciphera Pharmaceuticals.

—San Diego-based Retrophin (NASDAQ: RTRX) has a new CEO, Eric Dube, replacing Stephen Aselage, who called Martin Shkreli a “Pied Piper” during Shkreli’s fraud trial.

—South San Francisco’s Cortexyme has named Michael Detke chief medical officer and Chris Lowe chief financial officer.

—Marie Kosco-Vilbois is the new chief scientific officer of Swiss firm AC Immune.

Ben Fidler, Frank Vinluan, and Corie Lok contributed to this report.

Photo by Bernard Spragg via Creative Commons license.