Sarep­ta shares yo-yo in the red on an­oth­er glimpse of stel­lar re­sults for Duchenne MD gene ther­a­py — what gives?

There’s just no telling what a stock will do — even when you stack the deck in your fa­vor.

Tonight Sarep­ta $SRPT came out with a GREAT piece of news. Adding to the stel­lar, but very ear­ly glimpse of pos­i­tive da­ta for their gene ther­a­py for Duchenne mus­cu­lar dy­s­tro­phy, the biotech up­dat­ed their da­ta with a set of new num­bers that would ap­pear to add to the over­all glow of the pro­gram.


And then its stock took a dive, falling close to 10% in a mat­ter of min­utes af­ter the de­layed re­lease hit the wires. Then it bounced back, on­ly a few points in the red. What gives?

Is this sell the news? And why would that be? Shorts cov­er­ing their po­si­tions? Sev­er­al in­s­ta-re­ac­tions in the Twit­ter­verse sus­pect­ed that the fourth pa­tient did too well, see­ing a 182% in­crease in dy­s­trophin, the much-want­ed el­e­ment that cre­ates hav­oc for pa­tients, whose mus­cles waste and die with­out it.

As mea­sured by West­ern blot, pa­tient 4 showed ro­bust lev­els of mi­cro-dy­s­trophin, with a mean of 182.7% com­pared to nor­mal uti­liz­ing Sarep­ta’s method, or 222% com­pared to nor­mal pur­suant to Na­tion­wide Chil­dren’s quan­tifi­ca­tion of Sarep­ta’s method that ad­justs for fat and fi­brot­ic tis­sue.

Was that too much? Or too vari­able?

Baird’s Bri­an Sko­r­ney, a strong Sarep­ta bull, heard the dis­tur­bance in the force, but isn’t about to crum­ble now.

We ex­pect to de­bate con­cerns over noisy CK da­ta and im­pli­ca­tions of over­ex­pres­sion but see these as very mi­nor points on an oth­er­wise very im­pres­sive da­ta set and re­it­er­ate our Out­per­form.

I asked Sko­r­ney — one of the more thought­ful an­a­lysts in the bunch — about the red num­bers. His re­ply:

I think the ini­tial neg­a­tive re­ac­tion is due to the high ex­pres­sion lev­els that are rem­i­nis­cent of the con­cerns around vari­abil­i­ty with Bio­Marin’s Fac­tor VI­II pro­gram and some re­bound in CK lev­els in the first three pa­tients. I don’t re­al­ly ex­pect any af­ter­hours down move to be sus­tained to­mor­row though.

Sev­er­al an­a­lysts and re­porters help­ful­ly pro­vid­ed the em­bar­goed thumbs up, with quotes. Yet there, but for the grace of the biotech Gods, go I. I was tipped to the re­lease… but nev­er ac­tu­al­ly got it as of­fered.

Again, an­oth­er mys­tery for those of us who cov­er the field.

On the face of it, Sarep­ta would seem to have every­thing go­ing in its fa­vor. The gene ther­a­py is pro­vid­ing the nec­es­sary ev­i­dence of a sol­id bio­mark­er re­sponse. Even some signs in the sin­gle arm study — with­out the com­par­i­son da­ta that would help prove that it makes a dif­fer­ence  — that it’s help­ing pa­tients im­prove phys­i­cal­ly.

And this is a ter­ri­ble dis­ease. An ac­tu­al cure would be a god­send.

Sarep­ta has an un­for­tu­nate his­to­ry when it comes to his­tor­i­cal com­par­isons, though, which raised sig­nif­i­cant doubts in the agency that eteplirsen works.

Any­way, be­fore the post-mar­ket trad­ing closed Wednes­day evening, the stock had edged back in­to the green, so the de­fend­ers of the faith had held the line, briefly. By mid-morn­ing Thurs­day, the stock was back in the red again.

I’ll wait for some ad­di­tion­al com­men­tary. In the mean­time, caveat emp­tor.

You’re on your own, as al­ways.

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
Overview
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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In­scrip­ta push­es fund­ing to $260 mil­lion as they launch genome edit­ing plat­form

Inscripta presentations can begin with the advent of agriculture. Or even further back: The emergence of man.

“We’ve come a long way, starting with natural selection,” Inscripta microbial director Nandini Krishnamurthy told a session this year at SynBioBeta, the new annual conference for the synthetic biology field.

Behind her was a slide that’s recurred in company presentations, showing from left to right across the bottom the classic evolution-of-man chart (the ‘humorous’ kind that ends on a fat soda-drinker), a picture showing the development of corn from thin strand to bulbous Iowan, and then a squiggly protein close-up of “directed evolution.” Below that runs an arrow and a ticker of how long each takes, from 10^9 years to 1.

Re­pub­li­cans un­veil a drug price bill to ri­val the De­moc­rats — promis­ing low­er prices and more cures

Nancy Pelosi unveiled the Democrats’  drug pricing bill back in September and brought the fight straight to the industry with a proposal to empower the US government to negotiate prices for select drugs. Republicans, who decried the bill reeks of heavy-handed government intervention which will stifle innovation, now have a counterproposal they claim will result in cheaper drugs and incentivize R&D — further clouding the prospects of a bipartisan compromise that could land on Donald Trump’s desk.

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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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.

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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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.

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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