Bio Roundup: Pfizer’s Prices, Novartis Cuts, Gene Therapy Guide & More

Xconomy National — 

Drug price changes typically happen either at the start of the calendar year or the beginning of the third quarter. Such changes are routine for many companies, but Pfizer took an unusual step this week by rolling back scheduled price increases on 40 of its drugs.

The change of plan happened after Pfizer (NYSE: PFE) CEO Ian Read spoke by phone with President Trump, according to Reuters. An unnamed source told the news service that the president said on the call the price hikes threw a wrench in his administration’s plans to tackle drug prices. In May, Trump and Health and Human Services Secretary Alex Azar unveiled a “blueprint” plan to rein in the rising cost of prescription drugs.

Consumers might want to refrain from applauding Pfizer’s actions, however, because the price rollback is temporary. Pfizer merely deferred the increases until the end of the year, or when the president’s blueprint takes effect—whichever comes first, Reuters reported. Sen. Ron Wyden (D-OR) expressed concerns about a “secret, sweetheart deal,” and he wrote a letter to HHS calling on the Trump administration and Pfizer to release details of the call. Meanwhile, other drug companies seem content to let Pfizer stay in the spotlight as they quietly press ahead with their own planned price increases. A Bloomberg analysis found 10 biopharma companies raised prices on at least 20 brand name drugs in the first 10 days of July. The analysis found that the price of one drug spiked more than 700 percent.

In other news this week, a drug for a rare blood disease is on its way to the FDA, yet another pharma giant pulled out of antibiotics R&D, and a competitor emerged for an FDA-approved treatment for a form of epilepsy. Let’s get to those stories and more in this week’s roundup of the top biotech and pharma headlines.


—Acceleron Pharma (NASDAQ: XLRN) and partner Celgene (NASDAQ: CELG) reported success for their drug, luspatercept, in a late-stage study testing it in patients who have the rare blood disorder beta-thalassemia. Based on the results, the companies say they plan to file for FDA approval in the first half of 2019.

—An experimental drug from Emeryville, CA-based Zogenix (NASDAQ: ZGNX) succeeded in the second of two Phase 3 trials for a rare form of epilepsy called Dravet Syndrome. The drug, ZX008, could soon compete with GW Pharmaceuticals’ (NASDAQ: GWPH) marijuana derivative cannabidiol (Elpidiolex), which the FDA just approved for Dravet patients in June.

Non-Hodgkin lymphoma drug pixantrone (Pixuvri) failed a late-stage study, threatening the conditional approval that CTI BioPharma (NASDAQ: CTIC) had received for the treatment in Europe and casting doubt on its prospects with the FDA.

—Seven months after Alzheimer’s disease drug BAN2401 failed a 12-month Phase 2 study, partners Biogen (NASDAQ: BIIB) and Eisai reported new, encouraging data following 18 months of treatment at the highest dose. Those results will formally be presented later this month at the Alzheimer’s Association International Conference in Chicago.

—Researchers at the University of California, San Francisco have developed new CRISPR gene-editing technology that doesn’t require virus-based methods to engineer human T cells. They say the technique, published in Nature, could produce T-cell therapeutics faster, cheaper, and make the cells more versatile. Here’s more from The New York Times.

—Science Translational Medicine published data from a mid-stage trial showing an experimental drug from resTORbio’s (NASDAQ: TORC) reduced the frequency of all infections, including lung infections, in patients over 65 years old. resTORbio is running a second Phase 2 trial of the drug, RTB101, with results expected later this year.


—European Union regulators approved inotersen (Tegsedi) to treat the rare genetic disease transthyretin amyloidosis. Inotersen, from Akcea Therapeutics (NASDAQ: AKCA), and rival drug patisiran, from Alnylam Pharmaceuticals (NASDAQ: ALNY), face FDA approval decisions in coming months.

—Pfizer will reorganize into three divisions and split off its consumer healthcare business, which the New York company has been trying to sell for some time.

—Despite government programs to encourage new antibiotic development, Novartis (NYSE: NVS) is exiting the anti-infective business and cutting 140 jobs in the Bay Area. The move comes two years after the company issued a “call to action” to combat drug-resistant “superbugs.”


—Antibody drug developer Visterra of Waltham, MA, agreed to be acquired by Otsuka Pharmaceutical for $430 million in cash.

—Durham, NC-based BioCryst Pharmaceuticals (NASDAQ: BCRX) terminated merger plans with Idera Pharmaceuticals (NASDAQ: IDRA) after its shareholders overwhelmingly voted down the plan.

—Dermavant Sciences of Durham, is paying GlaxoSmithKline (NYSE: GSK) £150 million upfront and potentially £100 million more for rights to tapinarof, an experimental drug to treat psoriasis and atopic dermatitis.

—Compass Therapeutics of Cambridge, MA, nabbed a final tranche of its $132 million Series A round, which has been three years in the making.

—Cell therapy developer Nohla Therapeutics announced a second closing of its Series B round of financing, bringing the Seattle biotech’s funding total to $56 million.

—Vaccinex, of Rochester, NY, filed the paperwork for an IPO to finance research and development of its lead antibody drug as a treatment for both lung cancer and Huntington’s disease. Meanwhile, Renaissance Capital counts five biotech IPOs expected to price next week.


—The FDA released a broad set of guidelines for the nascent gene therapy sector, with specific guidance for hemophilia, rare disease, and retinal disorders. The six documents include clinical trial design, manufacturing issues, and other technical recommendations. Regulatory Focus has an overview of the FDA’s documents here.

—The Federal Trade Commission gave regulatory clearance for Takeda Pharmaceutical’s acquisition of Shire (NASDAQ: SHPG), moving the proposed $62 billion deal one step closer to closing.


—This week we profiled the Xconomy Awards startup finalists, a crop that includes developers of microbiome drugs, gene therapies, cell therapies, and more.

Ben Fidler and Alex Lash contributed to this report.

Photo by Flickr user ollagrafik via a Creative Commons license