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.

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.

BeiGene and Mus­tang nail down spe­cial FDA sta­tus for top drugs; Roche bags added cov­er­age for Hem­li­bra

→ BeiGene $BGNE is getting a boost in its drive to field a rival to Imbruvica. The FDA has offered an accelerated review to zanubrutinib, a BTK inhibitor that has posted positive results for mantle cell lymphoma. The PDUFA date lands on February 27, 2020. The drug scored breakthrough status at the beginning of the year.

→ BeiGene isn’t the only biopharma company to gain special regulatory status today. Mustang Bio $MBIO and St. Jude Children’s Research Hospital announced that MB-107, a lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease, has been granted Regenerative Medicine Advanced Therapy status.

Trump ad­min­is­tra­tion re­vives bid to get drug list prices on TV ads

The Trump administration is not giving up just yet. On Wednesday, the HHS filed an appeal against a judge’s decision in July to overturn a ruling obligating drug manufacturers to disclose the list price of their therapies in television adverts — hours before it was stipulated to go into effect.

In May, the HHS published a final ruling requiring drugmakers to divulge the wholesale acquisition cost— of a 30-day supply of the drug — in tv ads in a bid to enhance price transparency in the United States. The pharmaceutical industry has vehemently opposed the rule, asserting that list prices are not what a typical patient in the United States pays for treatment — that number is typically determined by the type of (or lack thereof) insurance coverage, deductibles and out-of-pocket costs. Although there is truth to that claim, the move was considered symbolic in the Trump administration’s healthcare agenda to hold drugmakers accountable in a climate where skyrocketing drug prices have incensed Americans on both sides of the aisle.

Ver­sant-backed Chi­nook gets a $65M launch round for its dis­cov­ery quest in a resur­gent kid­ney field

Versant is once again stepping off the beaten track in biotech to see if they can blaze a trail of their own in a field that has looked too thorny to many investors for years.

The venture group and their partners at Apple Tree are bringing their latest creation out of stealth mode today. Born in Versant’s Inception Sciences’ Chinook Therapeutics is betting that its preclinical take on kidney disease can get an early lead among the companies starting up in the field.

Sir An­drew Dil­lon, NICE's first — and on­ly — chief ex­ec­u­tive to step down next year

Using a laptop borrowed from his former employer, South London’s St George’s Hospital, Sir Andrew Dillon set about establishing NICE — launched by the then health secretary Frank Dobson — in 1999.  On Thursday, the UK cost-effectiveness watchdog said its first and only chief executive — Dillon — is stepping down in March 2020.

Back in the day, decisions about which drugs and interventions were funded by the National Health Service (NHS) were made at the local level, but this ‘postcode prescribing’ system was fraught with skewed healthcare deployment making the structure unsustainable. A national system was deemed necessary — and NICE was formed to bridge that gap.