Nick Leschly at Endpoints News' panel at the 2020 JP Morgan Healthcare Conference. Credit: Jeff Rumans

At #JPM20, two CEOs, two rad­i­cal­ly dif­fer­ent ther­a­pies, and a fight to chase down sick­le cell

SAN FRANCISCO – Few CEOs tell a story better than bluebird’s Nick Leschly.

He cuts a Jeff Bezos figure on stage at the Colonial Room, the JP Morgan presentation hall for A-list biotechs: lean and bald, fast-talking and vest-wearing. He explains in simple language, apologizing when he has to brush on the data. It helps that he has a good story to tell.

“We treated them one time,” Leschly tells a packed crowd, gesturing to the slide behind him. “Look what happened.”

The slide shows 9 horizontal bars studded with diamonds. Each bar, he explained, represented a sickle cell patient, and each diamond represented a severe medical event, such as a pain crisis. The diamonds stud one side – before the therapy – and vanish on the other, afterward.

“A 99% reduction in these events — this is a functional cure for sickle cell disease,” Leschly says. “This is unprecedented data.”

Upstairs and an hour later, Ted Love stands before a narrow conference room in his suit and polka-dot tie. Love, the CEO of Global Blood Therapeutics, is a 60-year-old physician. His voice trails off at the end of sentences, and the story he tells is less compelling. There are no cured patients.

“This is the first drug that addresses the root cause of sickle cell disease,” Love says, speaking in front of a slide showing a white pill bottle for GBT’s new drug Oxbryta. “Right in the label, it says that this drug inhibits polymerization.”

In the 60 years after scientists discovered the cause of sickle cell, almost no treatments emerged, even as the condition debilitated hundreds of thousands of Americans, most of them black or Hispanic. But the last few years have seen a resurgence of interest as new technologies have made the disease seem newly beatable.

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Hal Barron at Endpoints News' UKBIO 2019

UP­DAT­ED: Glax­o­SmithK­line's lat­est makeover in­cludes auc­tion­ing off der­ma­tol­ogy, adding new tech and lay­offs. But how many?

GSK execs announced Wednesday morning that they are setting up a 2-year program to prepare the company for its split in two — consumer healthcare and the R&D-focused outfit she and research chief Hal Barron promised to revamp and reenergize 2 years ago.

A key part of that effort is a new R&D reorganization in which the vaccines group — traditionally separate inside the global organization — will integrate its work with the pharma teams in order to orchestrate common research themes on both sides. And GSK is helping pay for the 2-year program with a plan to sell off non-core assets, starting with its dermatology drugs portfolio.

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The Peanut But­ter Cup com­pa­ny puts $200M more be­hind a treat­ment for peanut al­ler­gy

Aimmune’s patients won’t ever be able to eat Reese’s, but every time they open a capsule, they’ll be consuming a product Nestlé helped produce.

Since the immunotherapy company completed its Phase II studies, Nestlé has become one of its largest backers. They invested $145 million in 2016 and $98 million in 2018. And today, days after Aimmune won FDA approval for their peanut allergy drug Palforzia, Nestlé announced a $200 million equity investment. They will now have invested $473 million and command 19.9% of the company.

Miki Kapoor

UP­DAT­ED: Re­al-world da­ta get re­al dol­lars: GV, Bain help in­fuse $100M in­to Ve­r­ana Health as prag­mat­ic tri­al space heats up

As the application of real-world data in clinical practice and drug development becomes the reality for a growing number of physicians as well as drugmakers, GV and a marquee syndicate are betting some real dollars on one San Francisco player.

The crew formerly known as Google Ventures is leading a $100 million round for Verana Health, which has just acquired a smaller company specializing in large-scale data architecture solutions dubbed PYA Analytics. Bain Capital Ventures, Casdin Capital and Define Ventures also chipped in.

Bill Gates pitch­es in on coro­n­avirus R&D and con­tain­ment ef­forts with $100M com­mit­ment

The Gates Foundation has signed up to take a role in the growing public-private response to a brewing pandemic.

The nonprofit group said Wednesday it is prepared to spend up to $100 million to help spur new R&D into vaccines, diagnostics and therapies while reserving part of that money for detection and containment work.

“Multilateral organizations, national governments, the private sector and philanthropies must work together to slow the pace of the outbreak, help countries protect their most vulnerable citizens and accelerate the development of the tools to bring this epidemic under control,” said new Gates Foundation CEO Mark Suzman. “Our hope is that these resources will help catalyze a rapid and effective international response. This response should be guided by science, not fear, and it should build on the steps that the World Health Organization has taken to date.”

Ken Frazier, AP Images

UP­DAT­ED: It's not just about Keytru­da, Mer­ck in­sists, as it carves out as­sets worth $6.5B in­to new com­pa­ny

As sales of Merck’s flagship therapy Keytruda grow from strength to strength — the New Jersey drugmaker is spinning off its women’s health, legacy brands, and biosimilar drugs into a new company.

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UP­DAT­ED: Yes, Gilead CEO Daniel O’Day is ready to ink some deals. No, you won’t see him sweat it

A bevy of analysts turned out for Gilead’s Q4 call Tuesday night looking for some of the old sizzle about the future that used to excite them in the past. What they got was a lecture on steady improvement, sound judgment and proper dealmaking — along with a plateful of slightly disappointing numbers that left more than a few feeling a bit deflated by the end.

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Helén Tuvesson

Ac­tive Biotech is back, with one last bid for the failed MS drug Te­va dis­card­ed

Laquinimod was the drug that was going to save Teva.

Over 14 years, the Israeli pharma started the compound on at least 15 clinical trials, 6 of them Phase III. They wanted it to replace the blockbuster multiple sclerosis drug Copaxone. But it failed late-stage study after late-stage study, not only in MS but also Huntington’s. And in 2018, after the fourth major whiff in 18 months, Teva returned the drug and all its rights back to sender: Sweden’s Active Bio.

Chi­na re­searchers tout in vit­ro da­ta for Gilead­'s an­tivi­ral against Wuhan virus — which they are try­ing to patent

There’s no definitive proof yet that Gilead’s remdesivir works as a treatment for 2019-nCov, but researchers in China clearly consider it promising enough to have applied for a patent on its use to combat the coronavirus virus outbreak stemming from Wuhan.

Amid worldwide vigilance over what many fear is becoming a pandemic, scientists from the Wuhan Institute of Virology and National Engineering Research Center for the Emergency Drug said they have tested a total of seven drugs in vitro — and found remdesivir and the malaria treatment chloroquine most effective against the novel coronavirus.

Drug pric­ing re­form ex­pect­ed to be biggest drag on bio­phar­ma in 2020; Beam up­sizes IPO of­fer­ing

→ US President Trump’s State of the Union address may have visibly elicited the ire of Speaker Nancy Pelosi but he did emphasize again the need for bipartisan legislation to lower drug prices. A survey conducted by GlobalData showed that 49% of global industry respondents believe that drug pricing and reimbursement constraints will have the greatest negative impact on the pharmaceutical industry this year.