Bio Roundup: Pfizer’s Rare Results, U.K.’s CAR-T No, IPO Go-Go & More

Xconomy National — 

Before you head out for the final summer getaway, catch up on the week’s headlines. Pfizer upped the ante in the field of transthyretin amyloidosis (ATTR) treatment, where competition to treat the rare disease has grown increasingly complex.

We’ll start with what was—and wasn’t—in Pfizer’s data release Monday, top up with more ATTR news, then round up a slew of drug approvals, a slug of policy news, and a welter of financings and deals. We’ve labored to gather it all so you don’t have to. Have a great weekend, everyone.


—To start the week, Pfizer (NYSE: PFE) released more data from its Phase 3 study of tafamidis (Vyndaqel), a treatment for ATTR. While regarded as positive, Pfizer left key details out of the paper, prompting more questions about the drug’s competitive potential in various patient populations—such as people who have genetically inherited the disease versus those who develop it over time. Pfizer shares are down about two percent this week.

—Patisiran (Onpattro), a hereditary ATTR drug from Alnylam Pharmaceuticals (NASDAQ: ALNY), became the first-ever approved RNA interference medicine in Europe. The decision sets up a commercial battle with Pfizer’s tamafidis and with inotersen (Tegsedi), from Akcea Therapeutics (NASDAQ: AKCA), both already approved in Europe.

—In a move that could have fallout in the ATTR space, the FDA rejected volanesorsen (Waylivra), from Akcea and its parent company Ionis Pharmaceuticals (NASDAQ: AKCA), as a treatment for the rare familial chylomicronemia syndrome, or FCS. Volanesorsen is based on the same technology as inotersen, and both drugs have caused similar serious side effects.


—Tetraphase Pharmaceuticals (NASDAQ: TTPH) picked up an FDA nod for eravocycline (Xerava), an antibiotic that treats complicated intra-abdominal infections.

—Bayer won FDA approval of Jivi, a hemophilia A treatment taken once every five days. The crowded hemophilia A marketplace also includes Roche’s emicizumab (Hemlibra), and in the next few years could see gene therapies and an RNAi medicine as well.

—The European Medicines Agency approved axicabtagene ciloleucel (Yescarta), the CAR-T treatment from Gilead Sciences (NASDAQ: GILD), for two types of blood cancer. Days later, however, the The National Institute for Health and Care Excellence, the U.K’s drug pricing watchdog, rejected Yescarta, citing its high cost. Here’s more from Reuters.

—The FDA also approved a combination regimen of Johnson & Johnson’s (NYSE: JNJ) ibrutinib (Imbruvica) and rituximab for the rare blood cancer Waldenström’s macroglobulinemia.

—Late last week, the U.S. Senate backed a White House proposal to force drug makers to disclose prices in their advertising. Forbes columnist Arlene Weintraub asked good questions: Which prices should consumer see, and will it make a difference?

—The FDA launched a pilot program to allow more flexible clinical trial designs for drugs and devices. It’s part of the agency’s push for faster, more efficient development leading to more competition.


—Ann Arbor, MI-based biotech Esperion Pharmaceuticals (NASDAQ: ESPR) said a combination pill of its experimental bempedoic acid and the generic ezetimibe reduced the “bad” type of cholesterol up to 35 percent beyond standard-of-care statins. The data come from the latest in a series of late-stage studies from Esperion, which made the unusual move of announcing its drug’s price several months ago. Forbes has more on the new data.

—More cardio: A massive University of Oxford study of diabetics showed no evidence that omega-3 supplements changed risk of serious vascular disease compared with a placebo of olive oil. The results prompted Amarin (NASDAQ: AMRN) to comment on the differences between the Oxford study and the soon-to-report clinical trial for its own experimental omega-3 product.

—Pfizer stopped two Phase 2 studies of experimental Duchenne muscular dystrophy drug domagrozumab after the data “did not support a significant treatment effect.” Pfizer has one other ongoing Duchenne trial, for a gene therapy it acquired in 2016.

—Not quite clinical: The journal Science published a preclinical study in which researchers used CRISPR gene editing to help dogs produce dystrophin, the muscle-protecting protein that people with Duchenne muscular dystrophy lack. Exonics Therapeutics, of Cambridge, MA, is advancing the method towards human testing. Here’s more from the Wall Street Journal.


—In its annual industry report, MassBio found that Massachusetts biotechs have accounted for nearly half of U.S.-based biotech IPOs this year, and the $2.7 billion in proceeds so far are on pace to top last year’s $3.1 billion record total.

—Four more biotechs filed to go public this week: Y-mAbs Therapeutics of New York set a $92 million target to finance pivotal studies of two neuroblastoma drugs…Sutro Biopharma of South San Francisco, CA, is shooting for $75 million to push its biologics pipeline forward…Arvinas of New Haven, CT, is aiming for $100 million for its cancer drugs… and Alzheimer’s developer Alzheon is giving it a second shot this year, targeting $40 million after its $80 million try in March was quickly withdrawn.

—The ongoing biotech bull run has led to a surge in recruitment for biotech analysts. Major firms are luring top analysts with multi-million dollar paydays, the Wall Street Journal reports.

—Cambridge-based RootPath announced $7 million in seed funding to continue preclinical work on a cell therapy that it says will compete with current CAR-T treatments.

—San Jose, CA-based medical device company Outset Medical closed a $132 million Series D financing to ramp up commercialization of Tablo, its hemodialysis system.

—German biotech Affimed (NASDAQ: AFMD) inked a deal with Genentech to co-develop cancer drugs that harness natural killer cells. Affimed received $96 million upfront and its shares more than tripled.

—NovaQuest Capital Management made a $100 million investment in Dermavant Sciences, a skincare biotech owned by Vivek Ramaswamy’s Roivant Sciences. Another Roivant entity, Urovant Sciences, grabbed an experimental gene therapy for overactive bladder from Ion Channel Innovations.

—Vertex Pharmaceuticals (NASDAQ: VRTX) invested about $12 million in U.K. biotech Genomics plc as part of a deal to discover drug targets for precision medicines.

–Harbour Biomed raised $85 million in Series B financing to boost a pipeline that includes drugs for autoimmune diseases and cancer.

Ben Fidler and Frank Vinluan contributed to this report.

Photo courtesy Chris Potter via a Creative Commons license