FDA Ad­Com backs Gilead­'s move to ex­pand De­scovy la­bel — but stops short of en­dors­ing broad PrEP use

Gilead is one step clos­er to un­lock­ing a po­ten­tial­ly block­buster new use of its two-drug HIV reg­i­men De­scovy — though prob­a­bly not all of it.

In a meet­ing late Wednes­day, the FDA’s An­timi­cro­bial Drugs Ad­vi­so­ry Com­mit­tee vot­ed 16 to 2 en­dors­ing De­scovy as a pre-ex­po­sure pro­phy­lax­is, or PrEP, in men and trans­gen­der women who have sex with men. On the ques­tion of ex­pand­ing the in­di­ca­tion to in­clude cis­gen­der women, though, a slight ma­jor­i­ty of ex­perts (10 ver­sus 8) vot­ed against it, cit­ing a lack of da­ta.

Di­ana Brainard

The rec­om­men­da­tions were large­ly in line with the agency’s in-house re­view, which point­ed out that an ap­proval cov­er­ing cis­gen­der women would re­quire a ques­tion­able ex­trap­o­la­tion of PK da­ta. While Gilead did pro­vide proof of sys­temic drug con­cen­tra­tions, the mea­sure­ments were large­ly un­help­ful in quan­tifi­able and in­ter­pret­ing whether there are ad­e­quate drug con­cen­tra­tions in cer­vi­co­v­agi­nal tis­sues.

In a state­ment, Di­ana Brainard — SVP of HIV and emerg­ing virus­es — praised the Ad­Com for a “thought­ful re­view and dis­cus­sion of the da­ta.” Though the com­pa­ny did not com­ment di­rect­ly on ex­pand­ing De­scovy’s use to cis­gen­der women, it had this to add:

Gilead rec­og­nizes the val­ue that De­scovy could bring to HIV pre­ven­tion ef­forts for the broad­est pos­si­ble at-risk pop­u­la­tion in the Unit­ed States and will con­tin­ue to work with FDA as the agency com­pletes its re­view of the ap­pli­ca­tion.

In terms of seiz­ing a big piece of the mar­ket, though, miss­ing out on the cis­gen­der women la­bel might not be that big of a deal af­ter all. Cis­gen­der women form less than 7% of PrEP use, ac­cord­ing to Jef­feries an­a­lyst Michael Yee.

SVB Leerink’s Ge­of­frey Porges not­ed just a few days ago that MSMs form the most im­por­tant pop­u­la­tion for De­scovy’s add-on OK, though “for Gilead’s pur­pos­es it is use­ful to have a broad la­bel with all rel­e­vant at-risk pop­u­la­tions in­clud­ed, and get­ting such an en­dorse­ment from the Ad­Com would give their De­scovy launch ad­di­tion­al mo­men­tum.”

His peak sales es­ti­mate for De­scovy stands at $3.8 bil­lion.

Ad­di­tion­al mo­men­tum mat­ters here be­cause De­scovy will like­ly be go­ing up against gener­ics of Gilead’s own Tru­va­da, with launch­es sched­uled for next Sep­tem­ber. De­scovy’s ad­van­tages over Tru­va­da — sub­sti­tut­ing TAF, or teno­fovir alafe­namide, for TDF (teno­fovir diso­prox­il fu­marate) in com­bi­na­tion with emtric­itabine — could ap­pear slight un­der the best of cir­cum­stances. It re­mains to be seen whether pay­ers will be will­ing to cov­er a new brand­ed drug over a gener­ic for safe­ty is­sues such as bone min­er­al den­si­ty.

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
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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Sanofi’s big week in­cludes a promis­ing PhI­II for an or­phan dis­ease drug, with plans for a pitch to the FDA

The biopharma R&D food chain is paying off with a plan at Sanofi to pitch regulators on a new drug for an orphan disease called cold agglutinin disease.

The pharma giant ushered out a statement Tuesday morning — after it spelled out plans to radically restructure the company, abandoning cardio and diabetes research altogether — saying that their C1s inhibitor sutimlimab had cleared the pivotal study.

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 an overweight 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.

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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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.

Chris Garabedian. Perceptive

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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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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