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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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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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.

French can­cer-fo­cused mi­cro­bio­me play­er is flush with €18M Se­ries B in­jec­tion

Last year, the death of an immuno-compromised elderly patient in a fecal microbiota transplantation trial — due to a donation that contained a rare type of E. coli bacteria — sent shivers across the field. The incident marred an otherwise exploding field of drug development that backed replenishing the gut with good bacteria as a safe and effective means to fortify the immune system to fight disease.

Catalent CEO John Chiminski speaks at an Endpoints News event at the JP Morgan conference in San Francisco, January 2020 [Jeff Rumans, Endpoints News]

Catal­ent chief John Chimin­s­ki trig­gers a $315M-plus wa­ger on the boom­ing cell ther­a­py mar­ket now in the mak­ing

Last year Catalent CEO John Chiminski invested more than a billion dollars to expand the contract manufacturer ahead of an explosion of gene therapy trials and expected approvals. He’s starting this year with a $315 million wager on the booming cell therapy field.

Just ahead of the market open Monday, Chiminski $CTLT laid out to me the details of his decision to buy a transatlantic player that has been building up manufacturing capability in CAR-Ts, TCRs, TILs (tumor-infiltrating lymphocytes) and mesenchymal stem cell therapies.

In exchange for the $315 million in cash, he’s getting MaSTherCell Global and its 32,000 square-foot facility in Houston and a 25,000 square-foot facility in Gosselies, Belgium. The manufacturing operation is in the process of building out a 60,000-square-foot addition in Belgium set to go live next year, expanding beyond clinical supplies to the commercial realm.

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The pow­er of prece­dent: How a one-off de­ci­sion by Janet Wood­cock es­tab­lished an un­in­tend­ed fast path­way at the FDA — what will fol­low?

When CDER chief Janet Woodcock overruled a group of senior regulators at the FDA a little more than 3 years ago to allow Sarepta to start marketing eteplirsen for Duchenne muscular dystrophy, she reassured some of her colleagues at the agency that it was the only time the biotech would win an approval based on a slight elevation of dystrophin production — a so-called “surrogate endpoint” used to help guide their accelerated approval.

The next time Sarepta arrived with a Duchenne drug — she said in several meetings about eteplirsen at the FDA — the biotech would be required to proffer data from a clinical trial with a hard endpoint, according to a regulatory expert with a close understanding of the internal workings of the agency. The biotech would need to prove efficacy in something like the 6-minute walk test to determine if their second drug, golodirsen, was actually helping the boys who suffered from the disease.

Testing an unproven theory against the reality of the declining strength these boys typically face in confronting a rare, lethal disease, Woodcock told her colleagues, a new application with hard data in it would give her a chance to determine if eteplirsen was providing a safe benefit to DMD patients and families, and if the slight boost in dystrophin is an accurate marker for the benefit.

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Source: Inato.com

The Sanofi-part­nered start­up find­ing clin­i­cal tri­als for the 90%

Sanofi wanted help finding new trial sites, so a couple years ago they started talking with a startup down the street – who sent them to China.

The Paris-based startup, Inato, was building a platform to expand the pool of patients for clinical trials. Only instead of advertising or matching patients with trials, as other young companies have, Inato was trying to match pharma companies with the vast majority of hospitals that rarely, if ever, host a trial, even if they have patients better suited to a particular study than a marquee name hospital. Call them a trial platform for the 99% – or, by Inato’s calculations, the 90% of sites that are generally ignored in clinical research.

HIV vac­cine re­mains Holy Grail as Sanofi/GSK reg­i­men dis­ap­points in large study

An HIV vaccine regimen comprising experimental components supplied by Sanofi and GlaxoSmithKline has been declared ineffective, underscoring the difficulty of this undertaking and raising the stakes for a mosaic vaccine being tested by a J&J-led coalition.

The National Institute of Allergy and Infectious Diseases has stopped administering vaccines in the HVTN 702 trial after an interim review concluded that it didn’t prevent HIV. The Phase IIb/III trial enrolled 5,407 HIV-negative volunteers in South Africa.

Bio­Mo­tiv, Bris­tol-My­ers al­liance bears fruit with new com­pa­ny: An­teros Phar­ma

As a pharmaceutical accelerator that selects and incubates nascent technology from the halls of academia, BioMotiv is constantly in need of funding. Enter Bristol-Myers Squibb — who along with assimilating the arsenal of Celgene assets it acquired with the $74 billion acquisition of the big biotech company — is looking for fresh meat to bulk up its pipeline.

On Tuesday, BioMotiv and Bristol-Myers unveiled the first company borne out of their 2019 pact: Anteros Pharmaceuticals, which is focused on difficult-to-treat fibrotic and other inflammatory diseases, using technology in-licensed from Yale.

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George Yancopoulos, Columbia via Youtube

Re­gen­eron joins the hunt for an­ti­bod­ies to fight a brew­ing pan­dem­ic as re­searchers whip up new tri­als for an­tivi­rals

Count Regeneron as the latest big biopharma company to pitch in on finding a therapy to combat the Wuhan virus.

Health and Human Services said on Tuesday that they’ll be working with Regeneron to use its antibody platform tech — one of the best in the world — to hunt for a therapy to fight the lethal virus. This is an expansion of a joint project they set up a little more than 2 years ago to hunt for antibodies that worked against Ebola and 9 other viruses, including flu viruses.