Fol­low­ing PhI­II break­down, As­traZeneca cruis­es to­ward an FDA de­ci­sion for its BTK block­buster hope­ful acal­abru­ti­nib

Just when As­traZeneca would pre­fer to turn away from its Phase III break­down with dur­val­um­ab and treme­li­mum­ab, along comes acal­abru­ti­nib.

Yes­ter­day, their BTK in­hibitor was grant­ed a break­through ther­a­py des­ig­na­tion at the FDA, and reg­u­la­tors im­me­di­ate­ly backed it up to­day with a pri­or­i­ty re­view des­ig­na­tion that will speed up the over­sight process and quite like­ly of­fer an­oth­er can­cer drug ap­proval some­time in Jan­u­ary 2018.

Peak sales es­ti­mates for this drug scrape the $1 bil­lion mark — earn­ing it a spot on the top 10 or­phan drugs in the late-stage pipeline, ac­cord­ing to Eval­u­atePhar­ma stats for 2022 — which would make it a valu­able ad­di­tion to the port­fo­lio.

As­traZeneca paid $4 bil­lion to buy a ma­jor­i­ty stake in Ac­er­ta less than 2 years ago just so the phar­ma gi­ant could land this drug. At the time, CEO Pas­cal So­ri­ot con­fi­dent­ly pre­dict­ed that the drug was a $5 bil­lion an­nu­al earn­er in the mak­ing. And he was hap­py to say that would be on top of the $45 bil­lion in 2023 rev­enue he had promised in­vestors in his cam­paign to fend off a buy­out at­tempt by Pfiz­er.

Af­ter a se­ries of prat­falls in the clin­ic, though, it’s un­like­ly any­one in the com­pa­ny would be so bold with pre­dic­tions like that again. Nev­er­the­less, acal­abru­ti­nib re­mains a key part of its late-stage pipeline, and one of its best bet for some near-term good news on the drug ap­proval front.

As­traZeneca lined up this ap­pli­ca­tion with Phase II da­ta as a sec­ond-line ther­a­py for re­lapsed/re­frac­to­ry man­tle cell lym­phoma, in keep­ing with the fast-for­ward strat­e­gy to ac­cel­er­at­ed ap­provals that the FDA has been en­cour­ag­ing.

As­traZeneca has pushed ahead on a slate of can­cer drugs in some big fields, mak­ing this its one ma­jor bright spot as the com­pa­ny con­tin­ues the years-long pur­suit of a turn­around. Lyn­parza was the first PARP ap­proved, and has moved along with sig­nif­i­cant new da­ta over the last year. The phar­ma gi­ant has built up some megablock­buster hopes for Tagris­so as well as Imfinzi (dur­val­um­ab) on the PD-L1 side.

Pas­cal So­ri­ot’s big bet on leap­ing ahead on the check­point front with com­bos has run in­to a brick wall, for now, but the com­pa­ny re­tains block­buster hopes that it can be turned around. Adding acal­abru­ti­nib and go­ing af­ter the Im­bru­vi­ca (ibru­ti­nib) fran­chise — the big can­cer drug from J&J and Ab­b­Vie, would be a big help — and a wel­come dis­trac­tion.

Ac­er­ta CEO Flavia Borelli­ni said: “We be­lieve acal­abru­ti­nib has the po­ten­tial to be a very im­por­tant treat­ment op­tion for pa­tients with this life-threat­en­ing blood can­cer. The FDA’s NDA ac­cep­tance ex­em­pli­fies our progress in the acal­abru­ti­nib de­vel­op­ment pro­gramme and con­tin­ues our mo­men­tum as we seek to trans­form care for peo­ple with haema­to­log­ic ma­lig­nan­cies.”

Im­age: Pas­cal So­ri­ot AP Im­ages

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

UP­DAT­ED: Es­ti­mat­ing a US price tag of $5K per course, remde­sivir is set to make bil­lions for Gilead, says key an­a­lyst

Data on remdesivir — the first drug shown to benefit Covid-19 patients in a randomized, controlled trial setting — may be murky, but its maker Gilead could reap billions from the sales of the failed Ebola therapy, according to an estimate by a prominent Wall Street analyst. However, the forecast, which is based on a $5,000-per-course US price tag, triggered the ire of one top drug price expert.

FDA de­lays de­ci­sion on No­var­tis’ po­ten­tial block­buster MS drug, wip­ing away pri­or­i­ty re­view

So much for a speedy review.

In February, Novartis announced that an application for their much-touted multiple sclerosis drug ofatumumab had been accepted and, with the drug company cashing in on one of their priority review vouchers, the agency was due for a decision by June.

But with June less than 48 hours old, Novartis announced the agency has extended their review, pushing back the timeline for approval or rejection to September. The Swiss pharma filed the application in December, meaning their new schedule will be nearly in line with the standard 10-month window period had they not used the priority voucher.

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Credit: AP Images

Covid-19 roundup: BAR­DA sup­ports Op­er­a­tion Warp Speed with big $628M con­tract to ser­vice Amer­i­ca's vac­cine pro­duc­tion needs

Another BARDA contract designed to service America’s Covid-19 vaccine needs has been deployed.

The White House-led initiative designed to bankroll development to bring a vaccine to the American public by this fall — Operation Warp Speed — has via BARDA handed a meaty contract to the maker of an FDA-licensed anthrax vaccine to open up its manufacturing apparatus to shore up production of Covid-19 vaccines.

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Why Mer­ck wait­ed, and what they now bring to the Covid-19 fight

Nicholas Kartsonis had been running clinical infectious disease research at Merck for almost 2 years when, in mid-January, he got a new assignment: searching the pharma giant’s vast libraries for something that could treat the novel coronavirus.

The outbreak was barely two weeks old when Kartsonis and a few dozen others got to work, first in small teams and then in a larger task force that sucked in more and more parts of the sprawling company as Covid-19 infected more and more of the globe. By late February, the group began formally searching for vaccine and antiviral candidates to license. Still, while other companies jumped out to announce their programs and, eventually and sometimes controversially, early glimpses at human data, Merck remained silent. They made only a brief announcement about a data collection partnership in April and mentioned vaguely a vaccine and antiviral search in their April 28 earnings call.

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A low-pro­file biotech bests Re­gen­eron in high-pro­file patent suit

For nearly a decade now, the low-profile Cambridge biotech Kymab has been battling in US, UK, Japanese and Australian courts with the biotech behemoth Regeneron.

Regeneron has turned itself into a $70 billion company off of a platform of transgenically humanized mice they can use to make antibodies for anything from Ebola to colorectal cancer. The technology took decades and billions to build, 20 years from the company’s founding to the first approved drug. And the company guards and touts it zealously, breaking their production process down into various branded components — Velocimmune, Velocigene, Velocimouse and four other Velocis — and sometimes suing would-be copycats. In 2014, most notably, they sued two Pfizer-backed entities for patent infringement.

Bull­ish biotech mar­ket pro­pels Pli­ant to $144M IPO — as No­var­tis pro­vides a $10M boost

After pharma partner Novartis boosted its IPO with a $10 million private placement, Pliant Therapeutics has wrapped its journey to the Nasdaq on a high note.

Pliant had penciled in a $86 million raise back in May. But as has become the norm in recent months, that initial number has turned out to be a mere placeholder, making way for the final haul of $144 million.

The South San Francisco biotech did so by pricing at $16, the high end of the range, while bringing the number of shares offered up to 9 million.