As blue­bird makes com­mer­cial progress in Eu­rope, US ap­pli­ca­tion for Zyn­te­glo suf­fers de­lay

Af­ter shock­ing Wall Street with a $1.8 mil­lion price tag for its gene ther­a­py Zyn­te­glo in Eu­rope — blue­bird bio on Tues­day re­vealed that it now ex­pects to com­plete its US ap­pli­ca­tion to mar­ket the be­ta-tha­lassemia treat­ment in the Unit­ed States on­ly by the sec­ond half of the year, ver­sus its pre­vi­ous es­ti­mate of the first half of 2020.

The one-shot ther­a­py, al­so called Lenti­Glo­bin, is en­gi­neered to help pa­tients with the rare in­her­it­ed blood dis­or­der — who need reg­u­lar blood trans­fu­sions and have no match­ing donor for a stem cell trans­plant — be­come trans­fu­sion-in­de­pen­dent.

As part of its fourth-quar­ter re­sults, the Cam­bridge, Mass­a­chu­setts-based com­pa­ny said the de­lay in com­plet­ing its rolling BLA sub­mis­sion was re­lat­ed to “in­for­ma­tion re­gard­ing var­i­ous re­lease as­says,” but did not go in­to fur­ther de­tail. This holdup could po­ten­tial­ly de­fer ap­proval to mid-2021.

The com­pa­ny’s shares $BLUE slipped about 2% to $86.45 in Wednes­day pre­mar­ket trad­ing.

“In the US, we con­tin­ue to mod­el ze­ro com­mer­cial pa­tients in 2020 giv­en what we an­tic­i­pate will be a com­pli­cat­ed ne­go­ti­a­tion process with pay­ors. While the FDA reg­u­la­to­ry time­lines ap­pear to be de­layed some­what, we’re leav­ing our 2021 num­bers un­changed as we think they’re al­ready con­ser­v­a­tive at $53mm (ver­sus con­sen­sus of $70mm),” Stifel’s Ben­jamin Bur­nett wrote in a note.

When Eu­ro­pean reg­u­la­tors grant­ed the ther­a­py con­di­tion­al ap­proval in June, an­a­lysts were ex­pect­ing a $900,000 price. But chief Nick Leschly un­veiled a price twice that — $1.8 mil­lion, over five years — mak­ing Zyn­te­glo the sec­ond most ex­pen­sive ther­a­py on the plan­et, just be­hind No­var­tis’ SMA ther­a­py Zol­gens­ma that car­ries an eye-pop­ping price of $2.1 mil­lion.

Akin to No­var­tis, blue­bird has set up as an in­stall­ment plan, with the cost of Zyn­te­glo un­fold­ing over five years (€315,000 an­nu­al­ly) as doc­tors eval­u­ate whether it’s work­ing. “If not, they stop. It’s not rock­et sci­ence; it just makes sense,” Leschly said on a con­fer­ence call.

The ther­a­py is priced at a 15% dis­count to its clear “in­trin­sic” val­ue of $2.1 mil­lion — de­liv­er­ing 22 qual­i­ty-ad­just­ed life years, or QALYs — for the most suc­cess­ful cas­es, he added.

In or­der for Zyn­te­glo to work, stem cells are har­vest­ed from the pa­tient’s body, and the pa­tient is giv­en chemother­a­py to prime their bone mar­row to re­ceive the ther­a­py. A vi­ral vec­tor is then used to im­plant a healthy copy of the be­ta-glo­bin gene in­to the bone mar­row, stim­u­lat­ing the pro­duc­tion of red blood cells.

When it works, Zyn­te­glo has been shown to keep pa­tients trans­fu­sion-free for more than 4 years, the com­pa­ny said in an up­date at the Amer­i­can So­ci­ety of Hema­tol­ogy. Ear­li­er this year, the ther­a­py was launched in Ger­many.

The ther­a­py is al­so be­ing test­ed for use in sick­le cell dis­ease. A late-stage tri­al is ex­pect­ed to ini­ti­ate in the first half of 2020, while a sec­ond Phase III study fo­cus­ing on pe­di­atric pa­tients is ex­pect­ed to be­gin en­rolling some­time this year.

So­cial im­age: Nick Leschly, End­points JPM 20 Jeff Ru­mans

Once fu­ri­ous over No­var­tis’ da­ta ma­nip­u­la­tion scan­dal, the FDA now says it’s noth­ing they need to take ac­tion on

Back in the BP era — Before Pandemic — the FDA ripped Novartis for its decision to keep the agency in the dark about manipulated data used in its application for Zolgensma while its marketing application for the gene therapy was under review.

Civil and criminal sanctions were being discussed, the agency noted in a rare broadside at one of the world’s largest pharma companies. Notable lawmakers cheered the angry regulators on, urging the FDA to make an example of Novartis, which fielded Zolgensma at $2.1 million — the current record for a one-off therapy.

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Covid-19 roundup: GSK, Am­gen tai­lor R&D work to fit the coro­n­avirus age; Doud­na's ge­nomics crew launch­es di­ag­nos­tic lab

You can add Amgen and GSK to the list of deep-pocket drug R&D players who are tailoring their pipeline work to fit a new age of coronavirus.

Following in the footsteps of a lineup of big players like Eli Lilly — which has suspended patient recruitment for drug studies — Amgen and GSK have opted to take a more tailored approach. Amgen is intent on circling the wagons around key studies that are already fully enrolled, and GSK has the red light on new studies while the pandemic plays out.

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In a stun­ning set­back, Amarin los­es big patent fight over Vas­cepa IP. And its high-fly­ing stock crash­es to earth

Amarin’s shares $AMRN were blitzed Monday evening, losing billions in value as reports spread that the company had lost its high-profile effort to keep its Vascepa patents protected from generic drugmakers.

Amarin had been fighting to keep key patents under lock and key — and away from generic rivals — for another 10 years, but District Court Judge Miranda Du in Las Vegas ruled against the biotech. She ruled that:
(A)ll the Asserted Claims are invalid as obvious under 35 U.S.C.§ 103. Thus, the Court finds in favor of Defendants on Plaintiff’s remaining infringementclaim, and in their favor on their counterclaims asserting the invalidity of the AssertedClaims under 35 U.S.C. § 103.

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Covid-19 roundup: J&J, BAR­DA set ear­ly 2021 fin­ish line for $1B vac­cine race; FDA al­lows emer­gency drug use, ahead of piv­otal da­ta

J&J has zeroed in on a Covid-19 vaccine candidate that it hopes to begin testing in humans by September this year — with the extraordinary goal of getting it ready for emergency use in early 2021. And together with BARDA, it’s committing $1 billion to make it happen.

That kind of accelerated timeline would fall on the fast side of NIAID director Anthony Fauci’s well-publicized prediction that it would be another 12 to 18 months before a vaccine can be available for public use. A Phase I trial of Moderna’s mRNA vaccine began two weeks ago, and both the biotech and fellow mRNA player CureVac have discussed similar, if not even faster, timelines for emergency use among healthcare workers.

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As­traZeneca says its block­buster Farx­i­ga proved to be a game-chang­er in CKD — wrap­ping PhI­II ear­ly

If the FDA can still hold up its end of the bargain, AstraZeneca is already on a short path to scooping up a cutting-edge win with a likely approval for their SGLT2 drug Farxiga in cutting the risk of heart failure. Now the pharma giant says it can point to solid evidence that the drug — initially restricted to diabetes — also works for chronic kidney disease, potentially adding a blockbuster indication for the franchise.

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It is 'kind of a proven tech­nol­o­gy': Hep B vac­cine mak­er joins glob­al hunt for coro­n­avirus vac­cine

Using lab-grown proteins that are engineered to mimic the architecture of viruses to induce an immune response, VBI Vaccines is joining the hunt for a coronavirus vaccine — harnessing technology that has initially been proved safe in early trials as a prophylactic for cytomegalovirus (CMV) infection.

Unlike the raft of the companies in the Covid-19 vaccine race — including Moderna, CureVac and J&J — VBI is taking a pan-coronavirus approach, by developing a vaccine that will encompass Covid-19, severe acute respiratory syndrome (SARS), and Middle East respiratory syndrome (MERS).

Can a pair of top AveX­is alum­ni steer a new gene ther­a­py up­start to R&D glo­ry? 3 VCs bet $60M on it

VCs love few things more than a proven executive team when it comes to launching a new company. And now a group of A-listers has turned to a pair of top execs out of AveXis to steer the latest gene therapy player into the clinic.

The biotech is Waltham, MA-based Affinia and the two execs are Sean Nolan and Rick Modi — the former CEO and CBO respectively of AveXis, the gene therapy pioneer that fetched $8.7 billion in a sale to Novartis. Nolan has now taken the chairman’s role at Affinia while Modi moves up to the CEO post at the company.

Un­de­terred by a pan­dem­ic, Gilde Health­care rais­es their largest fund yet

When Pieter van der Meer started raising the capital for Gilde Healthcare’s fifth fund in the waning months of 2019, he had his eyes on a different chain of events that could change the healthcare system and perhaps even play to his firm’s advantage: The US presidential election.

Since raising their third fund in 2011, the 34-year-old Dutch firm had focused on value-based care. They chose late-stage biotechs that came up with new devices and delivery systems for de-risked established compounds, and when they chose preclinical biotechs, they spoke with potential pharma partners, payers and regulators to ask where and at what prices the drug made sense. As the Democratic primary became a contest over how to lower healthcare costs, it looked like a strategy that could pay off.

Daniel O'Day (AP Images)

Gilead CEO Dan O'­Day of­fers a de­tailed ex­pla­na­tion on remde­sivir ac­cess — re­as­sur­ing an­a­lysts that Covid-19 da­ta are com­ing fast

After coming under heavy fire from consumer groups ready to pummel them for grabbing the FDA’s orphan status for remdesivir — reserved to encourage the development of rare disease therapies — Gilead CEO Daniel O’Day had some explaining to do about the company’s approach to providing access to this drug to patients suffering from Covid-19. And he set aside time over the weekend to patiently explain how they are making their potential pandemic drug available in a new program — one he feels can better be used to address a growing pack of infected patients desperately seeking remdesivir under compassionate use provisions.

In addition to trying to reassure patients that they will once again have an avenue to pursue access, O’Day also reassured some analysts who had been fretting that China’s quick comeback from the coronavirus outbreak could derail its ultra-fast schedule for testing the drug in patients. The data are still expected in a few weeks, he says in the letter, putting the readout in April.

O’Day emphasizes that Gilead intends to pursue a pricing approach that will make this drug widely available — if it proves effective and safe. But no one is quite sure just what the longterm value would be, given the work being done on a variety of vaccines that may be rolled out as early as this fall — at least to the most heavily threatened groups.

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