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The Readout Damian Garde

Awaiting Legend's new data at ASH

Legend Biotech, a Chinese startup, dazzled the biotech world roughly a year and a half ago with impressive CAR-T data from 35 patients with multiple myeloma. Now, all eyes will be on Legend again, this time at next month’s meeting of the American Society of Hematology, where it will flesh out its initial findings with data from 22 more Chinese patients.

This time might be trickier, though, as Rebecca Robbins explains, with questions about how reliable and replicable the data are. Those questions loom large, not least of all because Johnson and Johnson has licensed the therapy in the U.S.

For more on ASH, be sure to sign up for our pop-up newsletter, "ASH in 30 Seconds." STAT's Adam Feuerstein will be on site, reporting back the most intriguing developments from San Diego.

Peanut allergy therapy effective, but uncomfortable

Peanut allergies can be severe, even lethal — and are increasing in incidence. That’s why Aimmune Therapeutics has been developing a treatment that desensitizes patients to peanut proteins. And phase 3 data published in the New England Journal of Medicine show severely allergic children taking the drug  were able to substantially improve their tolerance to peanuts. 

However, this desensitization process “was not easy on patients,” according to the NEJM review of the data. Although effective, Aimmune’s peanut allergy pills showed a concerning safety profile — with epinephrine shots necessary in about 14 percent of patients receiving the drug. They were, however, generally used to treat mild or moderate allergic reactions. 

“We delivered 112,000 doses of AR101 during the study and only saw one case of anaphylaxis. For an immune therapy intended to desensitize patients, this was a very successful study,” Aimmune Chief Medical Officer Dan Adelman told STAT. 

Read more. 

Squeezing cells to make room for medicine 

Delivering material into a cell can be a tricky endeavor. Boston-area startup SQZ Biotech says it can do this in an efficient, effective manner. Its technology delivers gene therapies or medicines straight into cells by squeezing them — forcing them through high-pressure tubes on microfluidic chips. This loosens up the membrane’s pores, so they’re able to soak in proteins, RNA, DNA, and other such minutiae. 

A new paper in PNAS showed that SQZ’s methodology was more effective than electroporation — in which a pulse of electricity is deployed to briefly open up the pores in cell membranes.

“The next thing is bringing it into humans,” MIT chemistry whiz and serial entrepreneur Robert Langer told STAT. “I’m looking forward to seeing what happens there, but I’m very optimistic because of what’s been done so far.”

Read more.

Who else is ready to hike prices again?

Pfizer is pushing forward with plans to hike the price of 41 of its drugs next year, despite pushback from the Trump administration and the (generally infuriated) general public. Over the summer, Pfizer said it would delay price increases — but only until the end of the year — after Trump chastised the company. Now, most of listed drugs will see bumps of around 5 percent.

Attention now shifts to other drug makers who followed Pfizer’s example in delaying the price hikes. Novartis, Merck, Roche, Sanofi, and Novo Nordisk all backtracked on price hike plans — but those, too, may have been temporary.

More reads

  • After five years, Verily shelves project to create glucose-sensing contacts. (STAT
  • From gene editing to AI, how will tech transform humanity? (New York Times)

Thanks for reading! More tomorrow,

Damian

Monday, November 19, 2018

STAT

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