Bio Roundup: New Drug Bonanza, Vertex’s Triple Play, CAR-T Race & More

Xconomy National — 

As far as singular weeks go for biotech, this was a big one. Three highly anticipated drugs were approved. One is the first injectable medication for eczema. Another might help fend off the recurrence of ovarian cancer. And a third offers hope for people with severe forms of multiple sclerosis. Another drug, a new combination therapy for cystic fibrosis, could follow this year after succeeding in two large tests. Each drug might help change how these diseases will be treated for years to come.

But each of these drugs are expensive therapies. Their prices once again shine the spotlight on the rising costs of new medicines, and heighten the tensions between drugmakers, insurers, and drug pricing hawks in Washington D.C. Approvals and drug pricing debates now go hand in hand, which is why it was only fitting that in the same week biotech could trumpet successful innovation, a group of congressmen proposed a broad bill to curb drug prices.

Those stories and much more below. Let’s get right to it.



—Some 20 Democratic U.S. senators banded together to propose the “Improving Access to Affordable Prescription Drugs Act.” The bill calls for a slew of reforms, like enabling Medicare to negotiate drug prices, calling for drugmakers to more transparently report their R&D costs, and enabling government authorities to monitor and take action on periodic price hikes.

—Scott Gottlieb, President Trump’s nominee to be the next FDA commissioner, will recuse himself for one year from making drug approval decisions regarding 20 companies he has had ties with, the New York Times reports here. The confirmation hearing for Gottlieb—a candidate the drug industry overwhelmingly favors—will take place next week.

—Trump’s plans to rally the warring factions of the GOP behind the American Health Care Act crashed and burned last Friday, so he’s moved on to other healthcare goals, like tasking son-in-law Jared Kushner and New Jersey Gov. Chris Christie with running a commission to solve the opioid crisis, the Washington Post reports.

—Meanwhile, the Trump administration wants to cut NIH funding by $1.23 billion this fiscal year alone, Bloomberg reports.


—The FDA approved dupilumab (Dupixent), an injectable biologic drug for atopic dermatitis, a form of eczema. Owners Regeneron Pharmaceuticals (NASDAQ: REGN) and Sanofi set a $37,000 per year list price for the drug, higher than analysts’ expectations.

—Genentech’s ocrelizumab (Ocrevus) won FDA approval to treat two forms of multiple sclerosis, including the most severe form of the disease that, up until now, had no approved treatment. Its list price is $65,000.

—The FDA’s approval of Tesaro (NASDAQ: TSRO) ovarian cancer drug niraparib (Zejula) marked the third approved treatment in a group of drugs called PARP inhibitors. Tesaro hasn’t disclose niraparib’s price yet, but the two other approved PARP blockers have a list price of $12,600 and $13,700 per month, respectively.


—Boston-based Vertex Pharmaceuticals (NASDAQ: VRTX) will file for approval of the cystic fibrosis combination therapy, ivacaftor-tezacaftor, after reporting positive results in two Phase 3 trials. Its strong clinical showing means Vertex’s strategy to build three-drug cocktails with ivacaftor-tezacaftor as the backbone—which could impact a vast majority of CF patients—is on the right track.

—In the race to bring the first CAR-T cancer immunotherapy to market, Swiss firm Novartis has leapfrogged rival Kite Pharma (NASDAQ: KITE). Novartis said the FDA has begun a priority review of a CAR-T product it’s been developing for acute lymphoblastic leukemia. Kite has yet to complete its application.

—Acorda Therapeutics (NASDAQ: ACOR), of Ardsley, NY, will soon file for approval of CVT-301, an inhalable form of the Parkinson’s drug levodopa, now that it has data from two long-term safety studies.


—Boston Scientific (NYSE: BSX) is expanding its minimally invasive heart valve portfolio through a deal to acquire Swiss company Symetis for $435 million cash.

—Tango Therapeutics launched with $55 million in funding from Third Rock Ventures, the Cambridge, MA-based venture capital firm where it has spent the last two years researching a way to use a cancer’s own genes to kill cancer cells.

—Cambridge, MA-based Solid Biosciences, a Duchenne muscular dystrophy startup formed by a father whose son was diagnosed with the disease, could soon become an IPO candidate after raising $50 million from a wide range of investors.

—Ionis Pharmaceuticals (NASDAQ: IONS) spun out Cambridge-based subsidiary Akcea Therapeutics with plans for an initial public stock offering. Akcea, which focuses on rare lipid disorders, also signed a separate $50 million private placement deal with partner Novartis (NYSE: NVS).

—The Combating Antibiotic Resistant Bacteria Biopharmaceutical Accelerator, a public-private partnership to invest in therapies to combat drug-resistant bacteria, announced $24 million in investments in 11 biotechs in the U.S. and the U.K. The Washington Post has more here.

—South San Francisco, CA-based Antiva Biosciences, a spinout of the University of California, San Diego, raised $22 million more to continue developing a treatment for human papilloma virus infections that can cause cancer.


—Eli Lilly (NYSE: LLY) announced plans to invest $850 million into its labs and manufacturing sites across the country, much of it earmarked for its diabetes drug operations.

—Lexington, MA-based Agenus (NASDAQ: AGEN) will cut 50 jobs in a restructuring, and R&D chief Robert Stein will retire.

The Boston Business Journal reported that a round of layoffs has begun at Ariad Pharmaceuticals following its recent acquisition by Takeda.

Frank Vinluan contributed to this report