Five drugs, in­clud­ing two No­var­tis ther­a­pies, win EMA en­dorse­ment

As is cus­tom, an EMA pan­el on Fri­day is­sued its week­ly rec­om­men­da­tions on mar­ket­ing ap­pli­ca­tions sub­mit­ted by drug de­vel­op­ers. This week, the agency backed the use of five new ther­a­pies — in­clud­ing two No­var­tis drugs — but is­sued no neg­a­tive re­views.

No­var­tis’ S1P drug for re­laps­ing forms of mul­ti­ple scle­ro­sis (MS) drug, Mayzent (known chem­i­cal­ly as sipon­i­mod), which was ap­proved by the FDA in March — has been giv­en the nod by the EMA. The Swiss drug­mak­er al­ready sells its oth­er MS drug, Gilenya, in both re­gions.

The com­pa­ny has al­so se­cured a pos­i­tive EMA rec­om­men­da­tion for Is­tur­isa — known chem­i­cal­ly as os­ilo­dro­stat — for use in Cush­ing’s syn­drome, a dis­ease char­ac­ter­ized by the ex­ag­ger­at­ed pro­duc­tion of the hor­mone cor­ti­sol. Ear­li­er this year, No­var­tis palmed off three en­docrine drugs, in­clud­ing os­ilo­dro­stat, to Italy’s Recor­dati for $390 mil­lion up­front. It al­so stands to make more in mile­stone pay­ments tied to os­ilo­dro­stat.

Fel­low Swiss drug gi­ant Roche al­so scored the EMA en­dorse­ment for its lat­est an­ti­body-drug con­ju­gate (ADC), Po­livy. The drug, known chem­i­cal­ly as po­latuzum­ab ve­dotin, has al­ready won US ap­proval in pa­tients with dif­fuse large B-cell lym­phoma as part of a reg­i­men that in­cludes the chemother­a­py ben­damus­tine and a ver­sion of its ag­ing block­buster can­cer drug, Rit­ux­an. The com­bi­na­tion is rem­i­nis­cent of Roche’s first cleared ADC reg­i­men, Kad­cy­la, which in­cludes its best­selling can­cer drug Her­ceptin and a chemother­a­py agent.

Jazz Phar­ma­ceu­ti­cals, which scored US ap­proval for its wake-pro­mot­ing drug sol­ri­amfe­tol, chris­tened Sunosi, in March — now has the EMA’s bless­ing. Like Xyrem — the block­buster nar­colep­sy drug that ac­counts for the bulk of Jazz’s sales — the dual-act­ing dopamine and nor­ep­i­neph­rine re­up­take in­hibitor is de­signed to treat ex­ces­sive day­time sleepi­ness for pa­tients with nar­colep­sy.

Rigel’s Tavlesse, known chem­i­cal­ly as fos­ta­ma­tinib, was ap­proved last year by the FDA as a sec­ond-line ther­a­py for throm­bo­cy­tope­nia in adult pa­tients with chron­ic im­mune throm­bo­cy­tope­nia. The drug, which is de­signed to block spleen ty­ro­sine ki­nase (SYK), and re­duces an­ti­body-me­di­at­ed de­struc­tion of platelets, now has the EMA’s back­ing.

The EMA al­so backed the ap­proval of two gener­ic med­i­cines and rec­om­mend­ed ex­pand­ing the la­bels of Roche’s Kad­cy­la and Cel­gene’s Revlim­id. The Eu­ro­pean Com­mis­sion typ­i­cal­ly is­sues its de­ci­sions about two months fol­low­ing its com­mit­tee’s rec­om­men­da­tions.

The use of two ap­proved drugs was al­so re­strict­ed by the agency’s hu­man med­i­cines com­mit­tee (CHMP).

The com­mit­tee en­dorsed the de­ci­sion of the EMA’s safe­ty pan­el, con­clud­ing that the use of Pfiz­er’s block­buster JAK in­hibitor Xel­janz could in­crease the risk of blood clots in the lungs and in deep veins in pa­tients who are al­ready at high risk. Af­ter re­view­ing all the da­ta, in­clud­ing the on­go­ing study A3921133 in pa­tients with rheuma­toid arthri­tis, the agency said it was cau­tion­ing the use of the drug in all pa­tients at high risk of blood clots.

In Feb­ru­ary, Pfiz­er oust­ed the 10 mg dose from the A3921133 study, switch­ing pa­tients to the small­er 5 mg dose af­ter doc­u­ment­ing “a sta­tis­ti­cal­ly and clin­i­cal­ly im­por­tant dif­fer­ence in the oc­cur­rence of pul­monary em­bolism” as well as an im­bal­ance in mor­tal­i­ty rate.

The EMA pan­el al­so banned the use of the 10 mg twice dai­ly in pa­tients with ul­cer­a­tive col­i­tis who are at high risk of blood clots, un­less there is no suit­able al­ter­na­tive treat­ment. Fur­ther, the EMA is rec­om­mend­ing that, due to an in­creased risk of in­fec­tions, pa­tients old­er than 65 years of age should be treat­ed with Xel­janz on­ly when there is no al­ter­na­tive treat­ment, it said. These rec­om­men­da­tions fol­low a lim­it im­posed by the EMA safe­ty com­mit­tee in May.

Mean­while, Sanofi’s re­laps­ing-re­mit­ting mul­ti­ple scle­ro­sis drug Lem­tra­da was al­so re­strict­ed in a rec­om­men­da­tion by the CHMP. Re­ports of im­mune-me­di­at­ed con­di­tions and prob­lems with the heart and blood ves­sels with the med­i­cine, in­clud­ing fa­tal cas­es, trig­gered a re­view in April by the agency’s safe­ty pan­el and a rec­om­men­da­tion for lim­it­ed use.

Regeneron CEO Leonard Schleifer speaks at a meeting with President Donald Trump, members of the Coronavirus Task Force, and pharmaceutical executives in the Cabinet Room of the White House (AP Photo/Andrew Harnik)

OWS shifts spot­light to drugs to fight Covid-19, hand­ing Re­gen­eron $450M to be­gin large scale man­u­fac­tur­ing in the US

The US government is on a spending spree. And after committing billions to vaccines defense operations are now doling out more of the big bucks through Operation Warp Speed to back a rapid flip of a drug into the market to stop Covid-19 from ravaging patients — possibly inside of 2 months.

The beneficiary this morning is Regeneron, the big biotech engaged in a frenzied race to develop an antibody cocktail called REGN-COV2 that just started a late-stage program to prove its worth in fighting the virus. BARDA and the Department of Defense are awarding Regeneron a $450 million contract to cover bulk delivery of the cocktail starting as early as late summer, with money added for fill/finish and storage activities.

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UP­DAT­ED: Bio­gen shares spike as ex­ecs com­plete a de­layed pitch for their con­tro­ver­sial Alzheimer's drug — the next move be­longs to the FDA

Biogen is stepping out onto the high wire today, reporting that the team working on the controversial Alzheimer’s drug aducanumab has now completed their submission to the FDA. And they want the agency to bless it with a priority review that would cut the agency’s decision-making time to a mere 6 months.

The news drove a 10% spike in Biogen’s stock $BIIB ahead of the bell.

Part of that spike can be attributed to a relief rally. Biogen execs rattled backers and a host of analysts earlier in the year when they unexpectedly delayed their filing to the third quarter. That delay provoked all manner of speculation after CEO Michel Vounatsos and R&D chief Al Sandrock failed to persuade influential observers that the pandemic and other factors had slowed the timeline for filing. Actually making the pitch at least satisfies skeptics that the FDA was not likely pushing back as Biogen was pushing in. From the start, Biogen execs claimed that they were doing everything in cooperation with the FDA, saying that regulators had signaled their interest in reviewing the submission.

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Daniel O'Day, Gilead CEO (Kevin Dietsch/UPI/Bloomberg via Getty Images)

A new study points to $6.5B in pub­lic sup­port build­ing the sci­en­tif­ic foun­da­tion of Gilead­'s remde­sivir. Should that be re­flect­ed in the price?

By drug R&D standards, Gilead’s move to repurpose remdesivir for Covid-19 and grab an emergency use authorization was a remarkably easy, low-cost layup that required modest efficacy and a clean safety profile from just a small group of patients.

The drug OK also arrived after Gilead had paid much of the freight on getting it positioned to move fast.

In a study by Fred Ledley, director of the Center for Integration of Science and Industry at Bentley University in Waltham, MA, researchers concluded that the NIH had invested only $46.5 million in the research devoted to the drug ahead of the pandemic, a small sum compared to the more than $1 billion Gilead expected to spend getting it out this year, all on top of what it had already cost in R&D expenses.

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FDA bars the door — for now — against Mer­ck’s star can­cer drug af­ter Roche beat them to the punch

Merck has been handed a rare setback at the FDA.

After filing for the accelerated approval of a combination of their star PD-1 drug Keytruda with Eisai’s Lenvima as a first-line treatment for unresectable hepatocellular carcinoma, the FDA nixed the move, handing out a CRL because Roche beat them to the punch on the same indication by a matter of weeks.

According to Merck:

Ahead of the Prescription Drug User Fee Act action dates of Merck’s and Eisai’s applications, another combination therapy was approved based on a randomized, controlled trial that demonstrated overall survival. Consequently, the CRL stated that Merck’s and Eisai’s applications do not provide evidence that Keytruda in combination with Lenvima represents a meaningful advantage over available therapies for the treatment of unresectable or metastatic HCC with no prior systemic therapy for advanced disease. Since the applications for KEYNOTE-524/Study 116 no longer meet the criteria for accelerated approval, both companies plan to work with the FDA to take appropriate next steps, which include conducting a well-controlled clinical trial that demonstrates substantial evidence of effectiveness and the clinical benefit of the combination.

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Roger Tung, Concert Pharmaceuticals CEO (Concert)

Con­cert gets BTD for alope­cia drug, set­ting up a late-stage show­down with gi­ant ri­val Pfiz­er

Concert Pharmaceuticals’ path to developing a drug that treats alopecia areata has been bumpy, but the pharma company scored a win Wednesday.

The FDA granted Concert a Breakthrough Therapy Designation (BTD) for its oral Janus kinase inhibitor, named CTP-543, paving the way for a Phase III study of the drug to begin in the fourth quarter of 2020. The news follows positive Phase II results from last September, which saw the drug meet its primary endpoint in both 8 mg and 12 mg twice-daily doses.

Covid-19 roundup: Mod­er­na sticks to Ju­ly for its Phase III as ru­mors swirl; Fol­low­ing US lead, EU buys up Covid-19 treat­ments

The Phase III might be delayed from its original early July goal, but Moderna says it will still kick off the pivotal study for what could ultimately be the first Covid-19 vaccine before the end of the month.

A day after Reuters reported that squabbling between the Cambridge biotech and government regulators had held up the trial by about two weeks, Moderna released a statement saying that they had completed enrollment of their 650-person Phase II trial and were on track to begin Phase III by the end of the month. The protocol for that study, which is meant to prove whether or not the vaccine can prevent people from becoming sick, has been finalized, they said.

GSK sets the stage for a toe-to-toe mar­ket show­down with Gilead­'s HIV cham­pi­on Tru­va­da

ViiV Healthcare and majority owner GlaxoSmithKline have cleared another important hurdle on a long-running quest to challenge Gilead’s dominance in preventative HIV treatments.

The final analysis of a new study shows the GSK subsidiary’s long-lasting injection, cabotegravir, proved 66% more effective in HIV prevention than Gilead’s breakthrough Truvada pill. And they now intend to carve away some of the blockbuster revenue that Gilead has enjoyed for years.

Stephen Hahn, AP

Trump and Navar­ro press again for hy­drox­y­chloro­quine. Can the FDA stay in­de­pen­dent?

Tuesday morning, economist and Trump advisor Peter Navarro walked onto the White House driveway and promptly brought a political cloud back onto the FDA.

Speaking to a White House pool reporter, Navarro said that four Detroit doctors were, based on a single disputed study, filing for the FDA to again issue an emergency authorization for hydroxychloroquine, the anti-malarial pill that President Trump hyped for months as a Covid-19 treatment over the objections of his own scientists. Then, while avoiding directly calling for the FDA to OK the drug, blasted the agency. He said its decision to pull an earlier authorization “was based on bad science” and “had a tremendously negative effect” on doctors and patients.

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Donald and Melania Trump watch the smoke of fireworks from the South Lawn of the White House on July 4, 2020 (via Getty)

Which drug de­vel­op­ers of­fer Trump a quick, game-chang­ing ‘so­lu­tion’ as the pan­dem­ic roars back? Eli Lil­ly and Ab­Cellera look to break out of the pack

We are unleashing our nation’s scientific brilliance and will likely have a therapeutic and/or vaccine solution long before the end of the year.

— Donald Trump, July 4

Next week administration officials plan to promote a new study they say shows promising results on therapeutics, the officials said. They wouldn’t describe the study in any further detail because, they said, its disclosure would be “market-moving.”

— NBC News, July 3

Something’s cooking. And it’s not just July 4 leftovers involving stale buns and uneaten hot dogs.

Over the long weekend observers picked up signs that the focus in the Trump administration may swiftly shift from the bright spotlight on vaccines being promised this fall, around the time of the election, to include drugs that could possibly keep patients out of the hospital and take the political sting out of the soaring Covid-19 numbers causing embarrassment in states that swiftly reopened — as Trump cheered along.

So far, Gilead has been the chief beneficiary of the drive on drugs, swiftly offering enough early data to get remdesivir an emergency authorization and into the hands of the US government. But their drug, while helpful in cutting stays, is known for a limited, modest effect. And that won’t tamp down on the hurricane of criticism that’s been tearing at the White House, and buffeting the president’s most stalwart core defenders as the economy suffers.

We’ve had positive early-stage vaccine data, most recently from Pfizer and BioNTech, playing catchup on an mRNA race led by Moderna — where every little sign of potential trouble is magnified into a lethal threat, just as every advance excites a frenzy of support. But that race still has months to play out, with more Phase I data due ahead of the mid-stage numbers looming ahead. A vaccine may not be available in large enough quantities until well into 2021, which is still wildly ambitious.

So what about a drug solution?

Trump’s initial support for a panacea focused on hydroxychloroquine. But that fizzled in the face of data underscoring its ineffectiveness — killing trials that aren’t likely to be restarted because of a recent population-based study offering some support. And there are a number of existing drugs being repurposed to see how they help hospitalized patients.

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