Gilead needs to stop wait­ing and start buy­ing; Let's em­brace Chi­na as a new bio­phar­ma pow­er

End­points as­sess­es the big bio­phar­ma R&D sto­ries of the week, with a lit­tle added com­men­tary on what they mean for the in­dus­try.

 Gilead needs to start buy­ing things — pron­to

Gilead once had one of the best rep­u­ta­tions in R&D. Its abil­i­ty to hus­tle through a pack­age of hep C cures was done at a jaw-drop­ping speed and de­liv­ered the goods with first-mover ad­van­tage in rev­o­lu­tion­iz­ing a mar­ket and dis­ease treat­ment. But what have you done for in­vestors late­ly, Gilead? A string of set­backs in the clin­ic has high­light­ed that Gilead isn’t im­mune from the high fail­ure rate in bio­phar­ma. And with­out a big, quick as­sist, its num­bers are go­ing to keep shrink­ing. It’s time to take that big stash of cash and, per­haps with the help of the Trump ad­min­is­tra­tion, get it back in­to cir­cu­la­tion with a cou­ple of ma­jor ac­qui­si­tions that can have an im­me­di­ate im­pact. Any sell­er will see Gilead com­ing from miles away, and will write down a mag­ic num­ber that would of­fend most buy­ers. In this mar­ket, buy­ers can’t be choosers when it comes to su­pe­ri­or deals. Gilead will have to pay top dol­lar. The com­pa­ny wait­ed too long, hop­ing that the pipeline would come to its res­cue. Wrong. Call up the BD team and get mov­ing.

 The Chi­nese are here. Let the com­pe­ti­tion be­gin

The news this week that Chi­nese re­searchers had sur­prised the world with the first use of CRISPR-Cas9 tech in a hu­man study un­der­scores a new re­al­i­ty in the glob­al in­dus­try. Chi­na is mov­ing to chal­lenge the West in drug de­vel­op­ment. And that is go­ing to take some get­ting used to. While the US and Eu­ro­pean bio­phar­ma camps have been op­er­at­ing in clear sight, co­or­di­nat­ing their work with high­ly vis­i­ble reg­u­la­to­ry bod­ies, the R&D at­mos­phere in Chi­na is much more cloudy.  So we’re not al­ways go­ing to get a heads up about R&D firsts like this. While the rest of the world has been fo­cused else­where, Chi­na has been build­ing a mod­ern, au­da­cious R&D in­dus­try of its own. Chi­nese sci­en­tists are among the best in the world, as a large num­ber of com­pa­nies have been find­ing out as they out­source more and more of their work to the Asian su­per pow­er. It’s time we spent more time track­ing the go­ings-on in Chi­nese re­search cir­cles. As one ex­ec re­cent­ly told me, some of these groups are able to work teams on a 24-hour sched­ule when it comes to solv­ing a prob­lem. It’s time we start­ed pay­ing clos­er at­ten­tion. That won’t be easy. But the tech ri­val­ry is re­al, and it’s here. Great!

 Let’s get some clar­i­ty on Trump’s ideas about health­care re­form

Right now, every­body that cov­ers bio­phar­ma is look­ing for clues — sol­id, luke­warm, half-baked, you name it — about where the Trump ad­min­is­tra­tion is head­ed on the in­dus­try. There had been some en­cour­age­ment that Rich Bag­ger, a po­lit­i­cal in­sid­er in the Chris Christie camp and se­nior Cel­gene ex­ec, was like­ly go­ing to be tapped for a se­nior spot in the health­care sec­tor af­ter head­ing up the tran­si­tion as ex­ec­u­tive di­rec­tor. Then seem­ing­ly min­utes lat­er he was re­port­ed to be head­ed back to the pri­vate sec­tor af­ter Christie was clear­ly shoved to the side­lines. We heard sig­nals from Trump dur­ing the cam­paign that he fa­vored the idea that Medicare should ne­go­ti­ate drug prices. And he al­so gave a thumbs-up to reim­por­ta­tion. But such po­lit­i­cal asides mean lit­tle for a new ad­min­is­tra­tion. He fa­vored FDA “re­form,” but we don’t know what that means in prac­tice. And the fu­ture of Oba­macare is still large­ly a mys­tery. We’ll get a much bet­ter idea of what Trump is think­ing when we see his picks for HHS and the rest of the cab­i­net. The soon­er the bet­ter.

De­vel­op­ment of the Next Gen­er­a­tion NKG2D CAR T-cell Man­u­fac­tur­ing Process

Celyad’s view on developing and delivering a CAR T-cell therapy with multi-tumor specificity combined with cell manufacturing success
Transitioning potential therapeutic assets from academia into the commercial environment is an exercise that is largely underappreciated by stakeholders, except for drug developers themselves. The promise of preclinical or early clinical results drives enthusiasm, but the pragmatic delivery of a therapy outside of small, local testing is most often a major challenge for drug developers especially, including among other things, the manufacturing challenges that surround the production of just-in-time and personalized autologous cell therapy products.

Paul Hudson, Getty Images

UP­DAT­ED: Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

“To be out of cardiovascular and diabetes is not easy for a company like ours with an incredibly proud history,” Hudson said on a call with reporters, according to the Wall Street Journal. “As tough a choice as that is, we’re making that choice.”

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Chris Garabedian. Xontogeny

Per­cep­tive teams up with Chris Garabe­di­an to open up a new, $210M biotech fund fo­cused on A rounds

Perceptive Advisors is one of those prolific biotech investor groups which has traditionally enjoyed zeroing in on clinical-stage investments and crossover rounds, a group that prefers more established drug development players with near-term payoff potential.

But now they’re partnering with Xontogeny chief and longtime biotech entrepreneur Chris Garabedian on a $210 million fund — with money contributed by institutional investors and family funds — to go into the launch space with their first early-stage VC fund. Dubbed the Perceptive Xontogeny Venture Fund, LP, or just PXV Fund, they plan to favor upstarts that Garabedian is fostering in his incubator. But they’ll also plan to reach outside that inner circle for more A rounds to back, with plans to dominate initial funding with $10 million to $20 million per newborn biotech.

Roger Perlmutter, Merck

#ASH19: Here’s why Mer­ck is pay­ing $2.7B to­day to grab Ar­Qule and its next-gen BTK drug, lin­ing up Eli Lil­ly ri­val­ry

Just a few months after making a splash at the European Hematology Association scientific confab with an early snapshot of positive data for their BTK inhibitor ARQ 531, ArQule has won a $2.7 billion buyout deal from Merck.

Merck is scooping up a next-gen BTK drug — which is making a splash at ASH today — from ArQule in an M&A pact set at $20 a share $ARQL. That’s more than twice Friday’s $9.66 close. And Merck R&D chief Roger Perlmutter heralded a deal that nets “multiple clinical-stage oral kinase inhibitors.”

This is the second biotech buyout pact today, marking a brisk tempo of M&A deals in the lead-up to the big JP Morgan gathering in mid-January. It’s no surprise the acquisitions are both for cancer drugs, where Sanofi will try to make its mark while Merck beefs up a stellar oncology franchise. And bolt-ons are all the rage at the major pharma players, which you could also see in Novartis’ recent $9.7 billion MedCo buyout.

ArQule — which comes out on top after their original lead drug foundered in Phase III — highlighted early data on ‘531 at EHA from a group of 6 chronic lymphocytic leukemia patients who got the 65 mg dose. Four of them experienced a partial response — a big advance for a company that failed with earlier attempts.

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US biosim­i­lar launch­es about to turn a cor­ner

The US biosimilar industry has lingered in the shadow of the European market since the US pathway for approvals was initiated in 2009.

Ten years later (or less than five years since the first FDA approval of a biosimilar), and just 42% (11 out of 26) of FDA-approved biosimilars have launched. But in the next three months (see chart below), a clutch of new biosimilars will hit the market, including new ones in oncology, hinting at a wave of uptake.

Left top to right: Mark Timney, Alex Denner, Vas Narasimhan. (The Medicines Company, Getty, AP/Endpoints News)

In a play-by-play of the $9.7B Med­Co buy­out, No­var­tis ad­mits it over­paid while of­fer­ing a huge wind­fall to ex­ecs

A month into his tenure at The Medicines Company, new CEO Mark Timney reached out to then-Novartis pharma chief Paul Hudson: Any interest in a partnership?

No, Hudson told him. Not now, at least.

Ten months later, Hudson had left to run Sanofi and Novartis CEO Vas Narasimhan was paying $9.7 billion for the one-drug biotech – the largest in the string of acquisitions Narasimhan has signed since his 2017 appointment.

The deal was the product of an activist investor and his controversial partner working through nearly a year of cat-and-mouse negotiations to secure a deal with Big Pharma’s most expansionist executive. It represented a huge bet in a cardiovascular field that already saw two major busts in recent years and brought massive returns for two of the industry’s most eye-raising names.

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Paul Hudson. Sanofi

New Sanofi CEO Hud­son adds next-gen can­cer drug tech to the R&D quest, buy­ing Syn­thorx for $2.5B

When Paul Hudson lays out his R&D vision for Sanofi tomorrow, he will have a new slate of interleukin therapies and a synthetic biology platform to boast about.

The French pharma giant announced early Monday that it is snagging San Diego biotech Synthorx in a $2.5 billion deal. That marks an affordable bolt-on for Sanofi but a considerable return for Synthorx backers, including Avalon, RA Capital and OrbiMed: At $68 per share, the price represents a 172% premium to Friday’s closing.

Synthorx’s take on alternative IL-2 drugs for both cancer and autoimmune disorders — enabled by a synthetic DNA base pair pioneered by Scripps professor Floyd Romesberg — “fits perfectly” with the kind of innovation that he wants at Sanofi, Hudson said.

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Game on: Re­gen­eron's BC­MA bis­pe­cif­ic makes clin­i­cal da­ta de­but, kick­ing off mul­ti­ple myelo­ma matchup with Bris­tol-My­ers

As J&J attempts to jostle past Bristol-Myers Squibb and bluebird for a landmark approval of its anti-BCMA CAR-T — and while GlaxoSmithKline maps a quick path to the FDA riding on its own BCMA-targeting antibody-drug conjugates — the bispecifics are arriving on the scene to stake a claim for a market that could cross $10 billion per year.

The main rivalry in multiple myeloma is shaping up to be one between Regeneron and Bristol-Myers, which picked up a bispecific antibody to BCMA through its recently closed $74 billion takeover of Celgene. Both presented promising first-in-human data at the ASH 2019 meeting.

FDA lifts hold on Abeon­a's but­ter­fly dis­ease ther­a­py, paving way for piv­otal study

It’s been a difficult few years for gene and cell therapy startup Abeona Therapeutics. Its newly crowned chief Carsten Thiel was forced out last year following accusations of unspecified “personal misconduct,” and this September, the FDA imposed a clinical hold on its therapy for a form of “butterfly” disease. But things are beginning to perk up. On Monday, the company said the regulator had lifted its hold and the experimental therapy is now set to be evaluated in a late-stage study.