Hillary Clin­ton's War on Phar­ma II; A call for com­mon sense re­port­ing on Alzheimer's R&D

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

Hillary Clin­ton want­ed that war with phar­ma, bad­ly

For all the back­lash around Mar­tin Shkre­li’s brief, dis­as­trous turn as CEO of Tur­ing, the Tweet from Hillary Clin­ton con­demn­ing his price goug­ing stands out for the wide­spread dam­age it did to biotech stocks. There are lots of rea­sons why the biotech stock in­dex­es have tanked over the past year. And it’s easy to see why that may have been over­due. But hav­ing a pres­i­den­tial can­di­date use Shkre­li to de­clare a “war on pharma” sets the stage for the bat­tle to con­tin­ue in DC af­ter the votes are count­ed. Try­ing to score cheap points with vot­ers in a pri­ma­ry is a bad way to es­tab­lish pol­i­cy. Yet that is what we are look­ing at if Clin­ton is elect­ed. The US — and the bad­ly frac­tured health sys­tem that needs re­form­ing — de­serve bet­ter. And biotech has enough prob­lems right now with­out get­ting caught up in the war. Let’s urge Clin­ton to take a more re­spon­si­ble stand if she wins the big elec­tion Tues­day.

The SEC sends an im­por­tant mes­sage on who biotech ac­coun­tants are work­ing for

We re­port­ed this week that the SEC wants to sanc­tion the PwC ac­coun­tant who let Steven Bur­rill go his mer­ry way for years be­fore he was caught – by oth­er means – loot­ing the biotech fund he man­aged. The SEC has al­ready dealt with Bur­rill, who was forced to re­im­burse funds used for per­son­al ex­pens­es, but en­forcers want to send a mes­sage here to oth­er ac­coun­tants do­ing the books for biotechs and all com­pa­nies. You have a pro­fes­sion­al re­spon­si­bil­i­ty to check out why ex­ecs are, say, ad­vanc­ing big­ger and big­ger sums for work not yet done. Sums that may nev­er have been war­rant­ed un­der any cir­cum­stances. And you can’t be waved away by the same ex­ecs who may have some­thing to hide. Bur­rill’s in­vestors de­served bet­ter. What­ev­er the in­di­vid­ual mer­its of these charges, let’s hope that mes­sage comes through loud and clear.

New in­ter­nal FDA doc­u­ments un­der­score Wood­cock’s un­in­formed de­ci­sion to back Sarep­ta drug

As I’ve said be­fore, there’s no rea­son to ex­pect the Sarep­ta con­tro­ver­sy to go away any­time soon. The lat­est doc­u­ment drop from the FDA on the in­ter­nal war over Janet Wood­cock’s in­sis­tence on an ap­proval now in­cludes ac­cu­sa­tions that she ig­nored in­ter­nal as­sess­ments on its Duchenne drug Ex­ondys 51 from se­nior of­fi­cials. This was noth­ing more than a gut-lev­el de­ci­sion which clear­ly de­vi­at­ed from agency stan­dards for an ap­proval. And FDA Com­mis­sion­er Robert Califf un­wise­ly de­cid­ed to let her have her way, open­ing up a new and dan­ger­ous path for oth­ers to fol­low. Let’s all con­tin­ue to hope this drug is as safe and ef­fi­ca­cious as the com­pa­ny and pa­tients ad­vo­cates in­sist it is. But ap­prov­ing an ex­per­i­men­tal drug like this was worse than a mis­take. It was bad pol­i­cy mak­ing at its worse.

Let’s re­main pro­fes­sion­al­ly skep­ti­cal about Alzheimer’s drug re­search

Look­ing over all the ways main­stream me­dia fawns over ear­ly-stage Alzheimer’s drug re­search has be­come some­thing of a hob­by of mine. And this week we were treat­ed to the full smor­gas­bord of gulli­bil­i­ty with a string of un­re­served re­views for the ear­ly an­i­mal and hu­man da­ta that’s be­come avail­able for Mer­ck’s late-stage BACE drug. Some of the UK pa­pers (hel­lo Dai­ly Mail) have a ten­den­cy to jump from the slight­est in­di­ca­tion of ef­fi­ca­cy in small groups to won­der­ing when the new cure will be ap­proved. We saw the same thing re­cent­ly when Bio­gen tout­ed its ear­ly da­ta on ad­u­canum­ab. But it’s a cru­el hoax on the pa­tients and their fam­i­lies. There are good rea­sons why Alzheimer’s has been an R&D dis­as­ter zone for more than a decade. Let’s wait for some con­vinc­ing da­ta be­fore herald­ing the next big break­through. We’re a long way from a cure. And that’s a hard truth. Alzheimer’s de­vel­op­ment has a long, long way to go, un­der the best of cir­cum­stances.

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.