Thumbs Up/Thumbs Down: The UN fum­bles bad­ly over ac­cess to drugs while de­vel­op­ers think again about R&D strate­gies

 

End­points as­sess­es the big bio­phar­ma R&D sto­ries of the week, with a lit­tle added com­men­tary on what they mean for the in­dus­try.

  The UN has a ter­ri­ble idea for re­solv­ing the drug pric­ing is­sue

The brouha­ha over drug prices this year has now helped stir up one of the worst pro­pos­als we’ve heard of so far. This one comes from the UN, which we oc­ca­sion­al­ly for­get ex­ists at all. The prob­lem with drugs, says the UN, is that all that nasty IP used by bio­phar­ma com­pa­nies to con­trol these prod­ucts is al­low­ing them to price ther­a­pies be­yond the reach of the poor. The US and oth­er coun­tries should do what they can to de­mand ready ac­cess to the drugs tax­pay­ers help pro­vide re­search sup­port for, says the UN. We beg to dif­fer. Any so­lu­tion re­gard­ing drug pric­ing is go­ing to have to be mar­ket-based, un­less you just want to de­stroy the mar­ket. If you de­stroy the mar­ket, there aren’t go­ing to be many drug prices to fight over, be­cause we’ll have far few­er ther­a­pies. There are mar­ket-based ini­tia­tives — let’s all ap­plaud Brent Saun­ders on that score — which can go a long way to re­solv­ing this de­bate. Man­u­fac­tur­ers, we note, have al­so been go­ing a long way to de­vel­op pric­ing struc­tures for each econ­o­my. As for the UN, it might want to find an­oth­er cri­sis to ad­dress. They’re al­ready ir­rel­e­vant on the drug pric­ing de­bate.

New rev­e­la­tions from ge­net­ics re­search help dri­ve a new ap­proach to R&D

Ei­sai’s new re­search group in An­dover, MA, which we cov­ered this week, il­lus­trates once again how drug de­vel­op­ment has been un­der­go­ing a ma­jor re­vamp. Groups like this promise to start with a clean slate and build new drugs from the ground up, do­ing more with fresh rev­e­la­tions about ge­net­ics to crack some very dif­fi­cult tar­gets like Alzheimer’s. We may even­tu­al­ly find our­selves with a few bad-to-mediocre drugs for this dis­ease, plucked out of the cur­rent late-stage crop, but a clean-slate ap­proach of­fers a much bet­ter shot at mak­ing a mean­ing­ful dif­fer­ence for pa­tients. De­nali, run by some ex-Genen­tech ex­ecs in San Fran­cis­co, is on the same track. Yes, this will all re­quire years of ex­plo­ration and plen­ty of mis­steps along the way, but the ul­ti­mate pay­off will like­ly be well worth the wait.

In the ab­sence of news on eteplirsen, we’re left with tea leaves

In the ab­sence of an FDA de­ci­sion, we’re all left read­ing tea leaves when it comes to cov­er­ing Sarep­ta. This week it was the news about Ron Farkas’ de­par­ture from the FDA that pro­vid­ed the ba­sis for a fresh round of spec­u­la­tion about their Duchenne mus­cu­lar dy­s­tro­phy drug. Ad­vo­cates who booed Farkas’ harsh crit­i­cism of Sarep­ta’s case for this drug saw it as a big win. Skep­tics didn’t see how one reg­u­la­tor’s ex­it could make much of a dif­fer­ence. We’ve been wait­ing for the FDA to make up its mind on this drug, and the tiny study used to back up its case for an ac­cel­er­at­ed ap­proval, all year long. It’s time for the FDA to stop dither­ing and make a de­ci­sion. Pa­tients need to know where the agency stands on this.

Stat will need to back up its next slap­down in biotech

I’ve been cov­er­ing bio­phar­ma for a long time, and jumped in­to jour­nal­ism close to four decades ago. If there’s one thing I’ve learned, if you want to re­al­ly ex­pose a bad­ly run com­pa­ny, you had bet­ter be able to es­tab­lish the facts. Stat, an on­line pub­li­ca­tion which has done much wor­thy work, claimed it had the goods on Mod­er­na, but woe­ful­ly failed to de­liv­er. The open­ing para­graphs of that sto­ry promised ex­plo­sives, and it de­liv­ered a few fire­crack­ers. And most, if not all, of those fire­crack­ers were thrown by anony­mous crit­ics. Stat un­for­tu­nate­ly dis­pens­es with stan­dards when it wants to stir up a con­tro­ver­sy. Next time, they should do the hard work, in the way that the Wall Street Jour­nal demon­strat­ed with its cov­er­age of Ther­a­nos. We’re wait­ing.

Un­pack­ing the Aduhelm de­ci­sion, Ver­tex's half full glass, a $525M J&J breakup, and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

By now you have surely read about the FDA’s controversial approval of Biogen’s Alzheimer’s drug and all its reverberations. But I’d still recommend checking out the meaty recap below to make sure you didn’t miss all the angles that the Endpoints team has covered. If you’d rather look ahead, look no further than our three-day virtual panels next week at BIO, where we will discuss what the new normal means for every part of the industry.

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What does a clear ma­jor­i­ty of the bio­phar­ma in­dus­try think of the FDA ap­proval of ad­u­canum­ab? 'Hor­ri­fy­ing' 'Dan­ger­ous' 'Con­fus­ing' 'Dis­as­ter'

Over the years, we’ve become used to seeing a consensus emerge early in our industry polls at Endpoints News. And when we took the pulse of drug hunters on the heels of a controversial FDA approval for aducanumab this week, it became immediately apparent that the vast majority of our readers — heavily concentrated among biopharma staffers and execs — were incensed by what they had just witnessed.

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Aaron Kesselheim (Scott Eisen/AP Images for AIDS Healthcare Foundation)

Har­vard’s Aaron Kessel­heim re­signs from ex­pert pan­el in wake of ad­u­canum­ab OK, blast­ing FDA for ‘worst drug ap­proval de­ci­sion in re­cent U.S. his­to­ry'

A third member of the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee has resigned in the wake of Biogen’s controversial Aduhelm approval, slamming the agency as he left and further deepening the controversy surrounding the decision.

Harvard University professor Aaron Kesselheim quit in protest Thursday afternoon, calling the Aduhelm OK “probably the worst drug approval decision in recent U.S. history.” Kesselheim follows both Joel Perlmutter, a neurologist from Washington University in St. Louis, and David Knopman, a neurologist from the Mayo Clinic, out the door.

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David Knopman (Mayo Clinic via YouTube)

A sec­ond ad­comm mem­ber aban­dons his post in af­ter­math of con­tro­ver­sial ad­u­canum­ab de­ci­sion

As the fallout from the FDA’s approval of Alzheimer’s med aducanumab grows, a second member of the adcomm overseeing that drug’s review has walked away. But even with two experts now having resigned from that committee in protest, is there enough broad-level outrage to prevent another aducanumab from getting approved?

The FDA on Wednesday lost another member of its Peripheral and Central Nervous System Drugs Advisory Committee as Mayo Clinic neurologist David Knopman hit the exit over the agency’s decision to approve Biogen’s Alzheimer’s drug Aduhelm despite the committee’s near-unanimous vote against it.

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FDA au­tho­rizes about 10M J&J vac­cine dos­es, trash­es 60M more from trou­bled Emer­gent plant

The FDA on Friday released about 10 million doses of J&J’s vaccine for use, and disposed of another 60 million doses that were manufactured at the now-shuttered Emergent BioSolutions facility in Baltimore where cross-contamination occurred.

The agency said it’s not yet ready to allow the Emergent plant to be included in the J&J EUA, but that may occur soon. FDA came to the decision to authorize some of the doses after reviewing facility records and quality testing results.

Janet Woodcock, acting FDA commissioner, at Thursday's Senate Appropriations hearing (Bill Clark/CQ Roll Call via AP Images)

Sen­a­tors lam­bast new Alzheimer’s drug’s price but give Janet Wood­cock a free pass on the ap­proval de­ci­sion

Senate Finance Democrats took aim at Biogen’s pricey new Alzheimer’s drug on Thursday, but members on both sides of the aisle at a separate appropriations hearing didn’t question acting FDA commissioner Janet Woodcock on the approval.

“I was appalled that Biogen priced their Alzheimer’s drug approved by the FDA at $56,000 per year — I’m not going to debate whether this is effective or not, but it’s double the household median income for Michiganders over the age of 65,” Sen. Debbie Stabenow (D-MI) said at the finance hearing.

Reshma Kewalramani, Vertex CEO (BIO via YouTube)

UP­DAT­ED: Ver­tex strikes out on its lat­est big shot at a rare ge­net­ic dis­ease. But they're go­ing to keep on swing­ing

It’s been several months since Vertex culled one of its small molecules for alpha-1 antitrypsin deficiency (AATD), taking a big hit after evidence of liver damage surfaced in a key Phase II trial. Now we learned that the company has whiffed on its second shot, and there’s nothing left in the clinic to treat the rare genetic disease — but that won’t stop it from trying.

Despite avoiding the safety issues that plagued the last candidate, Vertex $VRTX is taking the axe to VX-864 after Phase II results revealed the magnitude of the drug’s response is “unlikely to translate into substantial clinical benefit.” As a result of the news, the company’s stock fell 12.5% after hours.

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FDA plans new stud­ies on ac­cel­er­at­ed ap­proval dis­clo­sures in bio­phar­ma ads

When people read biopharma companies’ websites about new drugs approved via the FDA’s accelerated pathway, like Biogen’s new Alzheimer’s drug, do they understand that these drugs may only be reasonably likely to predict clinical benefit and still require confirmatory studies?

That’s what the FDA’s Office of Prescription Drug Promotion wants to firm up as an agency analysis of direct-to-consumer websites for accelerated approval drugs previously found that only 21% of the disclosures used language directly from the label.

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As it ex­pands its foot­print, Mod­er­na reach­es deal to man­u­fac­ture Covid-19 vac­cine dos­es in Mid­dle East

While the UAE leads the world with the highest percentage of residents vaccinated, neighboring Saudi Arabia — home to nearly 35 million people — has lagged behind significantly. On Friday, Moderna announced that it has partnered with the Saudi pharmaceutical company Tabuk to manufacture its jab and future variant-specific boosters in the country.

Tabuk will hold marketing authorization for the vaccine in Saudi Arabia, and the agreement gives them the possibility of distributing future Moderna mRNA products.