No­var­tis, Bay­er, Long­wood back ge­nomics start­up to speed search for im­munother­a­py tar­gets

Near­ly a cen­tu­ry passed be­tween the first pro­to-im­munother­a­py at­tempts in can­cer — crude and ob­scure but nonethe­less with some sci­en­tif­ic ba­sis — and Jim Al­li­son’s first T cell pa­per. Thir­ty-plus years flipped be­tween the dis­cov­ery of CT­LA-4 as an off-switch and the ap­proval of Yer­voy. Twen­ty-two rolled be­tween PD-1’s iso­la­tion and Op­di­va and Keytru­da. 

Long­wood co-founder Lea Hachi­gian is bet­ting she can has­ten that. It’s a bet on new­ly es­tab­lished sin­gle-cell ge­nom­ic analy­sis tech and the abil­i­ty to crunch end­less troves of da­ta at a rate few oth­ers can, and in­vestors in­clud­ing Leaps by Bay­er and No­var­tis Ven­ture Fund just put $39 mil­lion be­hind it. They call it Im­mu­ni­tas. 

“We can ask ques­tions of the im­mune sys­tem we weren’t able to ask be­fore,” Hachi­gian told End­points News.

The idea is to bridge im­muno-on­col­o­gy and tar­get­ed ther­a­pies. They’ll an­a­lyze in­di­vid­ual hu­man im­mune cells to de­ter­mine what genes are dri­ving it to, say, at­tack or not at­tack a par­tic­u­lar can­cer, es­sen­tial­ly con­vert­ing broad ques­tions —  what genes or pro­teins are at play? — in­to com­pu­ta­tion­al ones. Long­wood first learned of the plat­form in the win­ter, Hachi­gian said, and the work has so far led to one pre­clin­i­cal an­ti­body for can­cer. 

Sin­gle-cell se­quenc­ing, while a dra­mat­ic leap over the tech­nol­o­gy Al­li­son had to work with (he ac­tu­al­ly re­lied on a UC lab to ver­i­fy his ear­ly anti­gen pa­per), has now been around for some time. It was Na­ture’s 2013 Method of the Year, two years af­ter Cold Spring Har­bor Lab re­searchers used it to cre­ate the first map of a can­cer genome. 

Im­mu­ni­tas claims to stand apart by virtue of be­ing able to an­a­lyze that da­ta. Sin­gle-cell se­quenc­ing can gen­er­ate mas­sive da­ta sets, in­clud­ing tens of thou­sands of RNA, and Hachi­gian said most re­searchers don’t have the ca­pa­bil­i­ty to sort through it all. They look for spe­cif­ic things, in­stead of look­ing at the re­sults in their en­tire­ty. 

What we typ­i­cal­ly see are re­al­ly in­ter­est­ing da­ta sets that are hard to glean any­thing from,” Hachi­gian said. “No one is work­ing on the tar­gets we are.”

So far, that’s one tar­get, a mon­o­clon­al an­ti­body that Im­mu­ni­tas is tight-lipped about. But the com­pa­ny ar­gues that the tar­gets it comes up will be su­pe­ri­or be­cause they’re based on an­a­lyz­ing hu­man cells — an in­creas­ing­ly com­mon ap­proach — rather than the mouse mod­els that have be­come fa­mous for pro­duc­ing can­cer com­pounds that fail over 90% of the time in hu­mans. 

Be­yond on­col­o­gy, Im­mu­ni­tas will even­tu­al­ly piv­ot to fo­cus on au­to-im­mune dis­eases as well. No word yet on what those might be.

For now, they’ll add to a grow­ing trove of Long­wood-backed im­munother­a­py biotechs, join­ing the new­ly fi­nanced Were­wolf Ther­a­peu­tics and Thomas Gajew­s­ki’s sum­mer­time start­up Pyx­is Ther­a­peu­tics.

How to cap­i­talise on a lean launch

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Rev­o­lu­tion Med shoots for $100M+ IPO — and di­vulges some se­crets about that Warp Dri­ve buy­out

Biotech investors who like to wager on the race to the front of the KRAS market now have a new team to consider.

Revolution Medicines, which extended its reach on RAS with a deal to acquire Warp Drive Bio about 18 months ago, filed their S-1 in search of $100 million-plus. And they gave up a few secrets in the process.

The main clinical claim to fame that Revolution has centers on the SHP2 inhibitor RMC-4630, partnered with Sanofi back in the summer of 2018 — just after John Reed was named the incoming R&D chief. We already knew that the pharma giant handed over $50 million in cash plus a commitment of hundreds of millions more to align itself with Revolution as it makes a fresh foray into oncology. Now we know that Sanofi is also footing 80% of Revolution’s R&D bill on the program, while setting up a smorgasbord of $235 million in development milestones and $285 million in commercial bonuses.

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Turn­ing the cor­ner on treat­ing the root cause of sick­le cell dis­ease

Early in my career, as a medical resident, I saw first-hand the enormous challenges faced by children and adults with sickle cell disease (SCD), a genetic blood disorder that historically has lacked adequate treatment options. People living with this life-long disease are mainly those with ancestors from sub-Saharan Africa, as well as people of Hispanic, South Asian, Southern European and Middle Eastern descent. These patients suffer from devastating physical symptoms, including progressive, eventually fatal, organ damage and excruciating pain. In addition, they encounter emotional, mental and social burdens – non-physical aspects of living with SCD that also take a serious toll on patients and their caregivers.

Olivier Brandicourt, AP Images

#JPM20 ex­clu­sive: Olivi­er Brandi­court fol­lows the Big Phar­ma CEO path to pri­vate eq­ui­ty, join­ing Black­stone ahead of a mam­moth fund de­but

Nick Galakatos Blackstone

Seven months after Olivier Brandicourt’s surprise “early retirement” from Sanofi, he’s back in the game, this time taking meetings at JP Morgan to discuss his new role at Blackstone, where he’s quietly begun work with Nick Galakatos and the life sciences crew.

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Video Re­play: End­points at #JPM20 — news­mak­ers on deal­mak­ing, pric­ing and man­u­fac­tur­ing

On Monday, we held our fourth annual #JPM event — and the team hit a key milestone that I’d like to share with the entire Endpoints News audience: We live-streamed the conversation and had nearly triple the number of executives watching online than we had in the sold-out crowd of 320.

For a media company on a mission to connect the biopharma world in bigger and better ways, we’re proud of how we were able to extend the reach of our franchise event. Paid subscribers were given access to the stream in real time, and now, two days later, we’re opening it up to everyone in this post.

Endpoints@JPM: (left to right) Steve Pearson, Nick Leschly, Bari Talente, Stephen Ubl, John Carroll

#JPM20: 'The NPV is al­ways wrong.' Take­da preps an­oth­er spin­out — this time on psych

Editor’s Note: Endpoints News is reporting live from #JPM20 after kicking things off with an action-packed event, which you can replay here. What follows is a stream of tidbits we have collected while wandering around Union Square in San Francisco. Check back in throughout the week for updates by John Carroll and Jason Mast.

SAN FRANCISCO — A year ago Takeda CEO Christophe Weber and R&D chief Andy Plump arrived at JP Morgan right on the heels of closing their big Shire buyout. Now they’re back after shaking up the portfolio, boosting R&D spending by about 50% to $4.5 billion and adjusting the pipeline — a task which isn’t quite finished yet.

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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Neon Ther­a­peu­tics makes one last re­treat, sell­ing it­self cheap in a bar­gain base­ment M&A deal

Crushed by weak data for what had been their lead drug, Neon Therapeutics is being bought for parts this morning.

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Mark Pruzanski

#JPM20: Af­ter a year of NASH col­laps­es, all eyes on two biotechs

SAN FRANCISCO – It’s not quite Dewey defeats Truman, but Goldman Sachs calling 2019 “The Year of NASH” may well go down in the annals of worst biotech predictions.

Goldman Sachs slapped the label on weeks before 2019’s JP Morgan conference, projecting that long-discussed treatments for the obesity-driven condition suspected to lurk in millions of Americans would begin to bear fruit and investors would move accordingly. That did not quite happen.

“If you look at 2019, it was just a string of disappointing news,” Pascal Prigent, CEO of NASH-focused biotech Genfit, told Endpoints News in an interview.

The Year of NASH, or nonalcoholic steatohepatitis, became a year of NASH failures. Gilead failed two large Phase III trials. CymaBay went from a $1 billion company to a $100 million company after they found their drug was killing patients’ liver cells. Cirius withdrew an $86 million IPO bid after a disastrous readout. Industry-wide, there were few acqusitions in a market often projected to be worth $35 billion.

Gilead, after dominating the NASH discussion at the 2019 JPM, gave one quick mention to the program in their 2020 presentation before pivoting to other drugs.

“As promising as some of the mechanisms looked in earlier stages, when push comes to shove in large study settings, they just haven’t proven out,” Mark Pruzanski, CEO of the NASH-focused biotech Intercept, told Endpoints in an interview.

As biotech turns from 2019, the failures have refocused eyes away from Gilead and back toward two startups, both facing key events in the coming months: Intercept, which first alerted investors to NASH at JPM 2014, and the France-based Genfit.

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