Let's re­think Clin­ton's war on phar­ma; The FDA can't or­der the ge­nie back in­to the mag­ic lamp

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

 Sur­vey says!

Ever since I first start­ed think­ing about part­ner­ing up with Ar­salan Arif on End­points News, I want­ed to do a quar­ter­ly sur­vey of a sub­stan­tial num­ber of biotech ex­ecs to keep my thumb on the in­dus­try’s pulse. Now, 4 months from our launch, we did the first E100 sur­vey that found the pulse is quite strong. Yes, there is some flut­ter­ing ev­i­dent when you bring up top­ics like fu­ture fi­nanc­ings, IPOs, ac­cess to tal­ent, and so on. But by and large, biotechs are ex­pand­ing their teams and plan­ning to go in­to 2017 with am­bi­tious plans. We’ll fol­low up with a spe­cial 2017 fore­cast in Jan­u­ary. No one knows this in­dus­try bet­ter than the E100 com­bined. And you won’t want to miss what they have to say in the fu­ture.

 Hillary Clin­ton’s war on phar­ma was no chance event.

The lat­est batch of emails from Wik­iLeaks in­cludes the back­ground dis­cus­sion that oc­curred among Hillary Clin­ton’s cam­paign staff around her at­tack of Mar­tin Shkre­li, who still wears the black hat in biotech af­ter he de­cid­ed to jack up the price of Dara­prim by more than 5,000%. Clin­ton called it price goug­ing in a Tweet, trig­ger­ing a rout in biotech shares as the im­pli­ca­tions over some kind of price con­trols sank in. “FYI—We have start­ed the war with Phar­ma!!” ex­claimed se­nior ad­vis­er Ann O’Leary. “Great!” replied Mandy Grun­wald. There are bet­ter ways to have this dis­cus­sion than sin­gling out one bad ac­tor to slime an in­dus­try. Biotech is not Mar­tin Shkre­li, and the price of re­al in­no­va­tion in drugs will not be cheap. But that’s not price goug­ing. It’s time we sep­a­rat­ed drug de­vel­op­ment and the price of new drugs from the no­to­ri­ous prac­tice of rou­tine price hikes — or overnight prof­i­teer­ing — on old ther­a­pies. Maybe Clin­ton could Tweet about it some time. There’s no ques­tion now that she’s the odds-on fa­vorite to win this elec­tion.

 Kite is fly­ing high as its pi­o­neer­ing bi­o­log­ics ap­pli­ca­tion heads to the FDA

When Kite Phar­ma’s Arie Bellde­grun took to the stage in New York ear­li­er this week to spear­head a dis­cus­sion on the com­pa­ny’s am­bi­tious plans to launch the world’s first CAR-T, you could hear plen­ty of grum­bling on the side­lines. The da­ta are ear­ly. We all know that 3 months of re­spons­es in their piv­otal study is a quick read. Some want to wait for 6 months. We’ll see how that dis­cus­sion plays out. But while the com­pe­ti­tion at Juno and No­var­tis has fall­en well be­hind, Kite con­tin­ues to ag­gres­sive­ly press for­ward, not will­ing to give an inch un­less reg­u­la­tors re­quire it. Win or lose, you have to ad­mire the spir­it and com­pe­tence be­hind that.

 The FDA can’t make the Sarep­ta fall­out just dis­ap­pear

Now that the FDA has ac­tu­al­ly ap­proved Sarep­ta’s eteplirsen for Duchenne mus­cu­lar dy­s­tro­phy, the agency is try­ing to blunt the fall­out. Step one, for se­nior FDA of­fi­cial John Jenk­ins, was to spell out all the bad things that the biotech had done and then es­sen­tial­ly in­sist that no oth­er biotech will win an OK un­der sim­i­lar cir­cum­stances. Whip up ad­vo­cates to the ex­tent they at­tack FDA re­view­ers? Bad idea, says Jenk­ins. Use sus­pect da­ta from a woe­ful­ly in­ad­e­quate study to in­sist on an ap­proval? Uh-uh. The list goes on and on. What­ev­er the FDA says now, though, will do noth­ing to dis­pel the lessons to be learned from eteplirsen. And the next time a biotech comes along with an an­gry mob of sup­port­ers be­hind it, the FDA – and Janet Wood­cock – will have no one to blame but them­selves. This sto­ry is just be­gin­ning.

CRISPR: Los­ing some of the glit­ter

It was in­evitable. The third big IPO in gene edit­ing didn’t play out all that well, for­mal­ly end­ing gene edit­ing’s rep as the hottest thing in biotech. Noth­ing, of course, stays hot. But CRISPR Ther­a­peu­tics had to be dis­ap­point­ed this week that its tim­ing was off. Ed­i­tas and In­tel­lia had both made it out in big IPOs, on­ly to see their shares fade over the year. Gene edit­ing is still a pre­clin­i­cal field, with lots of changes in the tech to come. De­vel­op­ment ef­forts will be slow and painstak­ing, and it’s ask­ing a lot of in­vestors to stick through every­thing that has to hap­pen be­fore we see any ac­tu­al prod­ucts come out of it. Rev­o­lu­tions nev­er come fast or easy in biotech. Now comes the hard part. But it’s al­so the most valu­able stage for what comes next.

Gilead miss­es, again.

Gilead has plen­ty of re­sources to do R&D right. So it was kind of odd­ly fun­ny to see its messy da­ta from a tiny mid-stage pro­gram on a slate of in­di­ca­tions for GS-4997, in­clud­ing a pos­i­tive snap­shot for NASH. The prob­lem, though, was that the pic­ture proved too fuzzy to make out any ex­act ideas about its po­ten­tial. Gilead needs to prove now that it can do some­thing dra­mat­i­cal­ly great. This lit­tle stum­ble comes at a par­tic­u­lar­ly bad time.

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 costs in com­pa­ny-wide re­org

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy reveal tomorrow 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, 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.

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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.”

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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.

Roche faces an­oth­er de­lay in strug­gle to nav­i­gate Spark deal past reg­u­la­tors — but this one is very short

Roche today issued the latest in a long string of delays of its $4.3 billion buyout of Philadelphia-based Spark Therapeutics. The delay comes as little surprise — it is their 10th in as many months — as their most recent delay was scheduled to expire before a key regulatory deadline.

But it is notable for its length: 6 days.

Previous extensions had moved the goalposts by about 3 weeks to a month, with the latest on November 22 expiring tomorrow. The new delay sets a deadline for next Monday, December 16, the same day by which the UK Competition and Markets Authority has to give its initial ruling on the deal. And they already reportedly have lined up an OK from the FTC staff – although that’s only one level of a multi-step process.

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KalVis­ta's di­a­bet­ic mac­u­lar ede­ma da­ta falls short — will Mer­ck walk away?

Merck’s 2017 bet on KalVista Pharmaceuticals may have soured, after the UK/US-based biotech’s lead drug failed a mid-stage study in patients with diabetic macular edema (DME).

Two doses of the intravitreal injection, KVD001, were tested against a placebo in a 129-patient trial. Patients who continued to experience significant inflammation and diminished visual acuity, despite anti-VEGF therapy, were recruited to the trial. Typically patients with DME — the most frequent cause of vision loss related to diabetes — are treated with anti-VEGF therapies such as Regeneron’s flagship Eylea or Roche’s Avastin and Lucentis.