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.

Ryan Watts, Denali CEO

Bio­gen hands De­nali $1B-plus in cash, $1B-plus in mile­stones to part­ner on late-stage Parkin­son’s drug

Biogen is handing over more than a billion dollars cash to partner with the up-and-coming neurosciences crew at Denali on a new therapy for Parkinson’s. And the big biotech is ready to pile on more than a billion dollars more in milestones — if the alliance is a success.

For Biogen $BIIB, the move on Denali’s small molecule inhibitors of LRRK2 puts them in line to collaborate on a late-stage program for DNL151, which is scheduled to start next year.

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Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen scores a pri­or­i­ty re­view for its Alzheimer's drug ad­u­canum­ab, mov­ing one gi­ant leap for­ward in its con­tro­ver­sial quest

Biogen scored a big win at the FDA today as regulators accepted their application for the controversial Alzheimer’s drug aducanumab and gave it a priority review.

The PDUFA date is March 7, 2021.

Significantly, Biogen says it did not use its priority review voucher to win special treatment at the FDA. The agency handed that out gratis.

That’s the ideal scenario Biogen was looking for as disappointed analysts wondered aloud about the delayed application earlier in the year.

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Levi Garraway, Roche CMO (Source: Genentech)

UP­DAT­ED: FDA hands out a quick OK for po­ten­tial SMA block­buster ris­diplam, giv­ing Genen­tech and Roche a chance to chal­lenge ri­vals on the price

US regulators handed Roche and Genentech a big win Friday afternoon, one that has market-shaping potential for its high-priced rivals from Novartis and Biogen.

The FDA has green-lit the companies’ spinal muscular atrophy drug risdiplam, which will be marketed as Evrysdi in the US, for use in patients two months and older. It’s the first SMA drug that can be taken orally, as Biogen’s Spinraza is injected into the spine while Novartis’ Zolgensma is a gene therapy.

Moncef Slaoui, Getty Images

When will it end? Big Phar­ma's top vac­cine ex­pert at OWS of­fers a speedy time­line for a Covid-19 vac­cine — ei­ther be­fore or right af­ter the elec­tion

Moncef Slaoui hasn’t started making plans for his summer vacation next year. But he offers high odds that all Americans will be able to do that in the not too distant future.

In an interview with a pair of sympathetic podcasters at the conservative American Enterprise Institute, Slaoui provides an education to listeners on how any drug or vaccine can be sped through trials. And he leaves the door wide open to the notion that the leading vaccine developers can demonstrate efficacy and safety in a compelling fashion as early as October — or as late as the end of this year.

Covid-19 roundup: Gates Foun­da­tion pours $150M in­to In­dia’s Serum In­sti­tute; Pfiz­er teams with Gilead on remde­sivir

By CEO and scion Adar Poonawalla’s estimation, the Serum Institute in India has already poured hundreds of millions of dollars into scaling up the unproven Covid-19 vaccine being developed by AstraZeneca and Oxford for use in low and middle income countries. It’s meant taking on a risk that other companies, including AstraZeneca, have mitigated with huge amounts of government funding.

Now, for the first time, Poonawalla is getting some outside help. The Gates Foundation has agreed to pay the institute $150 million to supply 100 million vaccines to India and other emerging economies next year, Reuters reported. That includes both the vaccine being developed by AstraZeneca and the one being developed by Novavax. Those vaccines will be available in 92 countries and be priced at $3 per dose.

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President Trump speaks with members of the media before boarding Marine One (AP Images)

'Oc­to­ber is com­ing,' and every­one still wants to know if a Covid-19 vac­cine will be whisked through the FDA ahead of the elec­tion

Right on the heels of a lengthy assurance from FDA commissioner Stephen Hahn that the agency will not rush through a quick approval for a Covid-19 vaccine, the President of the United States has some thoughts on timing he’d like to share.

In an exchange with Fox News’ Geraldo Rivera on Thursday, President Trump allowed that a vaccine could be ready to roll “sooner than the end of the year, could be much sooner.”

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UP­DAT­ED: No­vavax her­alds the lat­est pos­i­tive snap­shot of ear­ly-stage Covid-19 vac­cine — so why did its stock briefly crater?

High-flying Novavax $NVAX became the latest of the Covid-19 vaccine players to stake out a positive set of biomarker data from its early-stage look at its vaccine in humans.

Their adjuvanted Covid-19 vaccine was “well-tolerated and elicited robust antibody responses numerically superior to that seen in human convalescent sera,” the company noted. According to the biotech:

All subjects developed anti-spike IgG antibodies after a single dose of vaccine, many of them also developing wild-type virus neutralizing antibody responses, and after Dose 2, 100% of participants developed wild-type virus neutralizing antibody responses. Both anti-spike IgG and viral neutralization responses compared favorably to responses from patients with clinically significant COVID‑19 disease. Importantly, the IgG antibody response was highly correlated with neutralization titers, demonstrating that a significant proportion of antibodies were functional.

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Bio­haven adds near­ly $1B in Nurtec deals with Roy­al­ty Phar­ma, Sixth Street

Biohaven just added nearly $1 billion to their balance sheet.

On Friday morning, the neuroscience biotech announced a pair of creative agreements with Royalty Pharma and the investment firm Sixth Street to bolster the commercial launch of their new migraine drug, Nurtec. Biohaven will sell a sliver of its royalties on Nurtec and 3% of the royalties on their experimental migraine drug zavegepant to Royalty Pharma as part of a larger agreeement that will pay $450 million. At the same time, the company announced they took out a $500 million loan from Sixth Street.

Ab­b­Vie set­tles in­sur­ance fraud suit, agrees to tweak nurse am­bas­sador pro­gram; CStone aims for NSCLC OK with pos­i­tive PhI­II da­ta

AbbVie has resolved a California lawsuit alleging insurance fraud in the promotion of its cash cow Humira, paying $24 million to settle things with the state’s insurance regulator.

The settlement comes almost four years after a whistleblower first reported AbbVie’s practice of deploying registered nurses to visit patients at home or call them by phone to ensure that Humira prescriptions are filled. AbbVie was also charged with providing illegal kickbacks to doctors in an attempt to encourage them to prescribe Humira for a range of anti-inflammatory diseases.