Bio Roundup: Spinraza Woes, Science March, Baseline Launch & More

Xconomy National — 

Rumblings of a new Obamacare replacement surfaced this week, while scientists and their supporters prepared for Saturday’s—a.k.a. Earth Day’s—nationwide Marches for Science.

Our own reporting focused this week on the fallout from high drug prices, with a look at the frustration of families whose children have had trouble gaining access to the first drug ever approved for the rare spinal muscular atrophy.

Elsewhere, Google’s cousin Verily finally launched a much ballyhooed health study, Novartis grabbed more momentum in the race toward the first CAR-T cell therapy approval, and a lot more news happened around the Xconomy network, with reports from Seattle to San Antonio and Indianapolis to North Carolina. Let’s get to the roundup.


—The high price and limited access to nusinersen (Spinraza), the first approved drug for spinal muscular atrophy, has left families of patients exasperated. but data that Biogen (NASDAQ: BIIB) will present at the American Academy of Neurology meeting next week could loosen the restrictions. Xconomy heard from SMA families and clinicians about the logistical and insurance challenges that have slowed the nusinersen rollout.

—Phoenix Biotechnology, a small San Antonio firm, has spent more than a decade trying to develop a plant-based drug for cancer. Running out of time and money, the company is pinning its future on preclinical data showing the drug might have a chance to treat Alzheimer’s disease.

—After three years of planning, Verily, once known as Google Life Sciences, has begun a long-range health study called “Baseline” with at least 10,000 participants across four years, aiming to learn about the biological changes underpinning the shift from health to illness. People who have grown accustomed to instant health information from their wearables might be surprised; Verily is not going to share the day-to-day data it gathers via its homebrewed “Study Watch.”


—Looking ahead to the March for Science on Saturday, Prothena CEO Gene Kinney penned this op-ed about the need for biotech professionals to explain science better to folks who aren’t immersed in the field.

—Weeks after the GOP’s healthcare plan crashed without reaching a House vote, efforts have begun to revive the bill, Politico reported. The news comes as the Trump administration has threatened to revoke federal subsidies that help support the Affordable Care Act. Here’s more in the Washington Post.


—A rheumatoid arthritis drug from Eli Lilly (NYSE: LLY) and partner Incyte (NASDAQ: INCY) that was expected to win FDA approval was instead rejected, as the regulator asked the companies to provide more dosing and safety data.

—Ultragenyx (NASDAQ: RARE), of Novato, CA, and partner Kyowa Hakko Kirin aim to file for approval of burosumab, for the rare genetic disease X-linked hypophosphatemia, after the drug hit its main goal in a Phase 3 trial.

—Novartis’s CTL-019 grabbed a second FDA “breakthrough” designation, this time in the diffuse large B cell lymphoma. Novartis is racing Kite Pharma (NASDAQ: KITE) to get the first CAR-T cancer immunotherapy across the finish line. Here’s more from

—Two weeks after a round of bad news, Redwood City, CA-based OncoMed Pharmaceuticals (NASDAQ: OMED), disclosed another clinical trial failure this week, this time in lung cancer. The firm plans to begin “a comprehensive portfolio prioritization review immediately.”

—Even though an independent board said in February that an experimental immunotherapy for kidney cancer from Argos Therapeutics (NASDAQ: ARGS) was unlikely to work, the Durham, NC, company said this week it would press on with the Phase 3 trial. Argos said its own analysis suggests a longer follow-up time may yet show a beneficial effect.


—Research Triangle Park, NC-based G1 Therapeutics unveiled plans for an initial public offering to finance clinical trials for its experimental cancer therapies that target two enzymes, CDK 4 and 6, that are key to cellular division.

—Controversial former Dendreon CEO Mitch Gold is back on Wall Street, now that his latest startup, Seattle-based Alpine Immune Sciences, has cut a deal to merge with struggling Nivalis Therapeutics (NASDAQ: NVLS) in a bid to go public. The combined company will be run by Gold and take on the Alpine name.

—Boston-based ZappRx, a startup that helps patients order and refill specialty medicine prescriptions, raised $25 million in new funding and added GV, the venture capital arm of Google, to its base of investors.


—Waltham, MA-based Tesaro (NASDAQ: TSRO) disclosed that its recently approved cancer drug, a so-called PARP inhibitor named niraparib (Zejula), would cost $9,833 a month for a 200 mg dose. While lower than rival drugs, the actual price is a more complicated because patients start on a higher, more expensive dose, and many move to lower doses afterwards. Here’s more in Endpoints.

—In other PARP blocker news, AbbVie (NYSE: ABBV) said that its PARP inhibitor velaparib failed two Phase 3 trials in lung and breast cancer.

—UniQure (NASDAQ: QURE) will pull alipogene tiparvovec (Glybera), the Western world’s first ever approved gene therapy, from the market in October. Glybera was a flop—only one patient was treated with it commercially—but it also played an important role in the advancement of gene therapy as a field.

—The legal feud continued between Amgen (NASDAQ: AMGN) and partners Regeneron Pharmaceuticals (NASDQ: REGN) and Sanofi. Already battling over their cholesterol-lowering PCSK9 blockers, Amgen sued over patents granted to Regeneron and Sanofi for their newly approved eczema drug, dupilumab (Dupixent). Expecting the lawsuit, Regeneron and Sanofi had filed a pre-emptive legal strike against Amgen last month. Here’s more in FiercePharma.

Alex Lash and Frank Vinluan contributed to this report.