Bio Roundup: CAR-T’s Huge Week, Merck’s ‘Pib Choice & More

Xconomy National — 

[Corrected 9/5/17, 12:22 p.m. See below.] The far-out idea of reprogramming a patient’s immune cells to fight cancer, known as CAR-T, entered the mainstream this week, and two numbers highlight the tale. The first is $11.9 billion, the price Gilead Sciences is paying for Kite Pharma and its cancer-fighting cell therapy pipeline. It’s one of biopharma’s biggest deals in recent years and a massive bet that Gilead can build a cancer business, starting with, but not limited to, Kite’s nearest-term target of non-Hodgkin lymphoma.

The second number is $475,000, which Novartis intends to charge for its cell therapy for pediatric leukemia. The FDA approved it this week ahead of schedule. That wasn’t a surprise, thanks to a unanimous FDA advisory vote in July, but the cost of treatment, and Novartis’s unusual pledge not to collect cash if it doesn’t work initially, has kicked CAR-T squarely into the national debate over drug prices.

We also send our best wishes to Houston and other parts of southeast Texas. We’ll all pull together to help, even in these divisive times. If you’re wondering where to start, here’s a good guide.

A shout-out as well to our Texas editor Angela Shah, based in Houston and undaunted. She has been covering the local tech community during the storm. You can follow her coverage here.

For the CAR-T news and more, let’s get to the roundup.


—The FDA approved tisagenlecleucel (Kymriah), the first CAR-T cell therapy to pass regulatory muster. The treatment for kids with severe cases of acute lymphoblastic leukemia will cost $475,000, said its owner Novartis (NYSE: NVS). But Novartis pledged not to charge a dime to patients whose treatment doesn’t help within a month. The price was lower than industry analysts expected, but higher than some patient advocates felt was necessary.

—Why the one-month “no charge” threshold? Novartis said it wanted to stick to the data that the FDA used for its approval. But there is no rule saying drug prices must be set according to a drug’s label.

—Matthew Herper of Forbes described the Kymriah price tag as a Solomonic choice, as if Novartis had to forge a compromise that would not likely appease anyone. CEO Joe Jimenez told Herper that Novartis spent more than $1 billion bringing the complex cell therapy to market.

—The next CAR-T likely to gain approval will come from Gilead Sciences (NASDAQ: GILD). That’s because Foster City, CA-based Gilead is buying the CAR-T’s owner, Kite Pharma (NASDAQ: KITE) of Santa Monica, CA, for $11.9 billion. Gilead officials said the therapy, nicknamed axi-cel, would be the foundation of its nascent cancer business.

—The crowded field laid to waste one prospective CAR-T competitor this week. Johnson & Johnson (NYSE: JNJ) ended a partnership with Macrogenics (NASDAQ: MGNX) , of Rockville, MD, on an early-stage antibody drug, duvortuxizumab, that J&J paid $125 million to license in 2014. It was a candidate to treat some of the blood cancers that the first wave of CAR-T therapies have gone after. Macrogenics is scrapping the drug, citing neurological side effects and too much competition from CAR-T. [This paragraph has been changed to clarify the description of duvortuxizumab.]


—The FDA approved benznidazole, the first U.S. treatment for Chagas disease, a parasitic infection. Because it is the first drug approved in the U.S. for a neglected tropical disease, the developer, a consortium of for-profit and nonprofit entities, receives a valuable voucher. The consortium has pledged to put half the revenues from the voucher into its non-profit work. Martin Shkreli once had a plan to develop the same drug.

—Four years after the FDA rejected Aveo Oncology’s tivozanib (Fotivda), European regulators approved the treatment for kidney cancer patients. Aveo is eyeing a comeback in the U.S. as well, with plans to file for approval should a Phase 3 study succeed next year.

—The Medicines Co. won FDA approval of meropenem/vaborbactam (Vabomere) for complicated urinary tract infections. It has yet to announce a price.

—Merck’s (NYSE: MRK) cholesterol-lowering drug anacetrapib met its main goal in a 30,449-patient study called “Reveal,” but the New Jersey drug maker did not say if it would seek FDA approval. It could be the end of a class of drugs called CETP inhibitors, once seen as potential breakthrough heart medicines, if Merck doesn’t move anacetrapib forward.

—Eli Lilly (NYSE: LLY) of Indianapolis plans to reapply for FDA approval for rheumatoid arthritis drug baricitinib (Olumiant) by the end of January. Lilly said in July that the FDA’s safety concerns could take 18 months to address, but a new timeline emerged after discussions with the regulator.

—Ionis Pharmaceuticals (NASDAQ: IONS) subsidiary Akcea Therapeutics (NASDAQ: AKCA) filed for FDA approval of volanesorsen, a drug developed by Ionis for the metabolic disease familial chylomicronemia syndrome.


—San Diego-based Otonomy (NASDAQ: OTIC) scrapped development of an experimental drug for the hearing disorder Meniere’s disease after the treatment badly failed a Phase 3 trial. Otonomy shares plunged more than 82 percent.

—The FDA refused to review an experimental Parkinson’s disease drug from Acorda Therapeutics (NASDAQ: ACOR), citing manufacturing concerns and other questions. Shares fell more than 20 percent.

—InVivo Therapeutics (NASDAQ: NVIV) laid off 39 percent of staff in a shake-up that also cut the job of chief scientific officer Thomas Ulich.


—Summit, NJ-based Celgene (NASDAQ: CELG) is expanding its relationship with Forma Therapeutics by exercising a $195 million option to pursue drugs in more therapeutic areas. Celgene still holds another option to buy Watertown, MA-based Forma outright.

—Jazz Pharmaceuticals (NASDAQ: JAZZ) is paying ImmunoGen (NASDAQ: IMGN) $75 million up front to partner on three of the Waltham, MA, biotech’s experimental drugs for blood cancers.

—SetPoint Medical raised $30 million in Series D funding. The Valencia, CA, company will use the cash for Phase 2 tests of its implantable electrical-signal device to treat rheumatoid arthritis.

—Last Friday, Gene therapy developer RegenxBio (NASDAQ: RENX) said it would acquire Dimension Therapeutics (NASDAQ: DMTX) in a stock deal valued at $86 million. Cambridge-based Dimension was created by RegenxBio four years ago and its experimental treatments are all based on RegenxBio’s drug platform.

—Redwood City, CA-based Armo Biosciences added new investor Qiming Venture Partners in a $67 million Series C-1 financing that will help Armo test its experimental immunotherapy in several cancers.

—This week we unveiled the finalists for the Patient Partnership award, which includes a group of creative initiatives companies and academics are using to team with patient groups to better understand diseases.

Image of T cells attacking cancer via the National Cancer Institute.

Ben Fidler and Frank Vinluan contributed to this report.