Kite Pharma wasn’t expecting a regulatory decision for its non-Hodgkin lymphoma treatment until November, but this week, the company got the FDA’s O.K.
The approval of axicabtagene ciloleucel (Yescarta) marks the second time in the two months that the FDA has given the nod to a CAR-T therapy, a treatment made from a patient’s own engineered cells. The first, tisagenlecleucel from Novartis (NYSE: NVS), was approved in August to treat a severe form of pediatric leukemia.
Gilead Sciences (NASDAQ: GILD), the Foster City, CA, company that acquired Kite, put a $373,000 price tag on the Kite therapy, a discount to the $475,000 price of Novartis’ treatment. But Novartis offered performance pricing: patients who don’t respond to the treatment within a month don’t have to pay. Gilead’s announcement offered no such pricing option, but alternative pricing isn’t completely off the table. The company later told Xconomy that it is talking with payers who have varying degrees of interest different pricing models.
In other news this week, two senators proposed a short-term Obamacare fix, Vir Biotechnology revealed a global strategy to tackle infectious disease, and Eli Lilly reached a deal to get into mRNA drug development. Let’s roundup the week’s biotech and health policy news.
IN THE CAPITAL
—Sens. Lamar Alexander (R-TN) and Patty Murray (D-WA) released their short-term fix to shore up Obamacare markets, which were sent into deeper crisis last week by President Trump’s executive orders to cut off federal insurance subsidies and TK . The White House officially came out against the bill, while 24 senators, split between the two parties, co-sponsored it.
—The same two senators presided over a hearing about high prescription drug prices. Alexander questioned the need for complicated rebates, which are negotiated between drug companies, insurers, and middlemen in secret. Other senators also touted the need for more transparency. The witness representing the drug industry took much of the heat.
—Politico reported that Alex Azar is the leading candidate to replace recently ousted Tom Price at the head of the Department of Health and Human Services. Azar worked at HHS under George W. Bush, then was a top executive at Eli Lilly.
LEGAL & ETHICAL QUESTIONS
—A federal judge struck down patents for blockbuster eye disease medicine Restasis, which owner Allergan (NYSE: AGN) has tried to protect from generic competition. The drug giant said in September it had transferred ownership to a sovereign Native American tribe and licensed back exclusive rights. The Texas-based judge said the patents never should have been granted. He also criticized Allergan’s strategy.
—The long-awaited lawsuit that Incyte (NASDAQ: INCY) has been threatening against former employees is moving forward, the Delaware News Journal reported. Incyte alleges its cancer immunotherapy secrets were used to build a competitor, Flexus Biosciences, which Bristol-Myers Squibb (NYSE: BMY) quickly bought for as much as $1.25 billion. Industry observer Derek Lowe opines that the parties will reach a settlement before the October 2018 trial start date.
—Buzzfeed reported on Bay Area biohacker Josiah Zayner, who publicly injected himself with a CRISPR-Cas9 gene editing solution to see what happens if he knocks out the protein that keeps muscle growth in check.
DATA GOOD, DATA BAD
—Johnson & Johnson (NYSE: JNJ) dropped two antibody drugs, sirukumab for rheumatoid arthritis and talacotuzumab for acute myeloid leukemia. Up for approval, sirukumab ran into questions about patient deaths. J&J did not say why it has ended a Phase 3 study for talacotuzumab.
—After hitting rock-bottom in late 2014, Exelixis (NASDAQ: EXEL) continued a run of success with positive Phase 3 results of its flagship drug cabozantinib in liver cancer. It plans to ask the FDA for approval early next year. Cabozantinib is already approved for certain patients with kidney cancer.
—Interim data from a Phase 2 study of non-small cell lung cancer drug entrecitinib from Ignyta (NASDAQ: RXDX) showed improved overall survival in patients who have a particular genetic mutation, boosting the company’s stock price 25 percent. The San Diego biotech went on to raise $125 million in a stock offering.
—Celgene (NASDAQ: CELG) is stopping work on Crohn’s disease drug mongersen at the recommendation of a data safety monitoring committee. The Summit, NJ drugmaker hadacquired the drug for $710 million to diversify its portfolio. Celgene said there were no safety issues, and it will wait until reviewing Phase 2 data from an ulcerative colitis study before deciding what to do next with the drug.
DEALS OF THE WEEK
—Gemini Therapeutics emerged from stealth with a $42.5 million Series A round that the Cambridge, MA, company will use to develop gene-based therapies to treat dry age-related macular degeneration.
—Eli Lilly (NYSE: LLY) paid CureVac $50 million up front in a partnership to develop up to five new cancer vaccines based on the German company’s messenger RNA technology.
—Vir Biotechnology of San Francisco unveiled $500 million in committed financing and a slate of biotech and university partnerships that it plans to use to develop new treatments for infectious diseases.
—Warp Drive Bio inked a deal with Swiss pharma giant Roche that pays the Boston biotech $87 million up front, and could pay up to $300 million more if the partnership yields new antibiotics.
STARTUPS AND FUNDS
—Illumina Ventures said its first fund has grown to $230 million after launching last year with $100 million from sequencing giant Illumina. Based in San Francisco, it has invested in seven companies to date.
—Stanford University spinout Forty Seven has tacked on $75 million in Series B cash to double its total venture haul. The firm is going after cancer, and as Xconomy reported in 2016, got a $30 million boost from California taxpayers because of its stem-cell work.
—A new system for single-cell genomic analysis is close to commercial launch. South San Francisco, CA-based Mission Bio, a spinout of UCSF, raised $10 million in Series A financing. It plans to bring an acute myeloid leukemia analysis to market this year.
—TARA Biosystems said it has raised a $9 million Series A to bring its “heart on a chip” testing products to market. Xconomy profiled the New York startup in 2014.
—TransEnterix (NYSE MKT: TRXC) is preparing to commercialize its Senhance Surgical Robotic System after the FDA cleared the Research Triangle Park, NC, company’s robot for colorectal and gynecological procedures.
—Cydan, an orphan drug accelerator based in Cambridge, raised $34 million to bring more rare disease drugs through preclinical development and potentially spin them out into independent companies.
—Gene therapy developer uniQure (NASDAQ: QURE) is switching the hemophilia B treatment it will test in a Phase 3 clinical trial after receiving a go-ahead from regulators. UniQure, split between the Netherlands and Lexington, MA, says the new therapy, nearly identical to the earlier treatment, has modifications that better prevent bleeding.
Alex Lash contributed to this report.
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