House Speaker Nancy Pelosi (Jacquelyn Martin/AP Images)

Pelosi threat­ens to slide drug pric­ing re­form in­to Biden's bud­get bill, re­jects in­dus­try push­back that in­no­va­tion would suf­fer

Af­ter a re­port re­leased by a key House com­mit­tee showed a pro­ject­ed $1 tril­lion in buy­backs over the next 10 years by Big Phar­ma, Speak­er Nan­cy Pelosi said she wants to at­tach drug pric­ing re­form to the cur­rent bud­get bill, and slammed drug com­pa­nies for sug­gest­ing in­no­va­tion would be harmed as a re­sult.

If passed, the bill would em­pow­er Medicare to ne­go­ti­ate di­rect­ly with drug com­pa­nies to low­er prices, a long­stand­ing goal for De­moc­rats. The House re­port claims the pol­i­cy shift could save tax­pay­ers $456 bil­lion in the next 10 years.

“We have an ex­tra­or­di­nary op­por­tu­ni­ty to do this as we craft this rec­on­cil­i­a­tion bill,” Pelosi said on a con­fer­ence call with re­porters.

The House Over­sight Com­mit­tee re­leased a staff re­port that an­a­lyzed fi­nan­cial da­ta from the 14 largest drug com­pa­nies to eval­u­ate the amount of mon­ey they in­vest in R&D, and the im­pact that has on Medicare price ne­go­ti­a­tions.

From 2016 to 2020, the com­pa­nies spent $56 bil­lion more on stock buy­backs and div­i­dends — $577 bil­lion — than on R&D, the re­port found. The pro­ject­ed num­ber of spend­ing on buy­backs from 2020 to 2029 is $1.15 tril­lion for the same 14 com­pa­nies.

“The Com­mit­tee pre­vi­ous­ly re­leased six staff re­ports show­ing that the phar­ma­ceu­ti­cal in­dus­try has tar­get­ed the Unit­ed States for price in­creas­es for many years, while cut­ting prices in the rest of the world,” the re­port said. “The Unit­ed States is par­tic­u­lar­ly vul­ner­a­ble to these pric­ing tac­tics be­cause cur­rent law pro­hibits Medicare from ne­go­ti­at­ing di­rect­ly with drug com­pa­nies to low­er drug prices.”

The 14 com­pa­nies in­clud­ed the study are Ab­b­Vie, Am­gen, As­traZeneca, Bris­tol My­ers Squibb, Eli Lil­ly, Gilead, GSK, J&J, Mer­ck, No­var­tis, No­vo Nordisk, Pfiz­er, Roche and Sanofi. Be­tween 2016 and 2020, the top ex­ec­u­tives for these com­pa­nies brought home a com­bined salary of $3.2 bil­lion, and eight of those com­pa­nies spent less on R&D com­pared to buy­backs and div­i­dends, the re­port states. Am­gen’s spend­ing on buy­backs was six-fold, com­pared to R&D costs.

“This re­port finds that the world’s lead­ing drug com­pa­nies have used price in­creas­es to boost pay­outs to in­vestors and ex­ec­u­tives while spend­ing less on re­search and de­vel­op­ment,” Rep. Car­olyn Mal­oney (D-NY) said in a press re­lease. “The re­port al­so shows that in­dus­try claims about the po­ten­tial im­pact of pric­ing re­forms are overblown.”

In­dus­try lob­by­ists have played tight de­fense, ar­gu­ing that it would de­ter the cre­ation of new drugs, and hurt pa­tients in the end. Bri­an Newell, a spokesper­son for bio­phar­ma lob­by­ing group PhRMA, told Bloomberg that his or­ga­ni­za­tion was com­mit­ted to work­ing with pol­i­cy­mak­ers on com­mon­sense so­lu­tions that ad­dress the “re­al chal­lenges pa­tients face.”

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

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Land­mark Amy­lyx OK spurs de­bate; Some... pos­i­tive? Alzheimer's da­ta; Can­cer tri­al bot­tle­neck; Sanofi's CRISPR bet; 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.

After brief stops in Paris and Boston, John Carroll and the Endpoints crew are staying on the road in October with their return for a live/streaming EUBIO22 in London. The hybrid event fireside chats and panels on mRNA, oncology and the crazy public market. We hope you can join him there.

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Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

Up­dat­ed: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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#AAO22: J&J’s first look at com­mon eye dis­ease port­fo­lio pads the case for PhII of gene ther­a­py

CHICAGO — While the later-stage drug developers in the geographic atrophy field are near the finish line, Johnson & Johnson’s Janssen is taking a more deliberate route, with a treatment that it hopes to be a one-time fix.

The Big Pharma will take its Hemera Biosciences-acquired gene therapy into a Phase II study later this year in patients with GA, a common form of age-related macular degeneration that impacts about five million people worldwide. To get there, Janssen touted early-stage safety data at the American Academy of Ophthalmology annual conference Saturday morning, half a day after competitors Apellis and Iveric Bio revealed their own more-detailed Phase III analyses.

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Some­one old, some­one new: Mod­er­na pro­motes CTO, raids No­var­tis for re­place­ment amid pipeline push

Moderna CEO Stéphane Bancel made clear on the last quarterly call that “now is not the time to slow down.” On Thursday, he made a bit more room in the cockpit.

The company unveiled a new executive role on Thursday, promoting former chief technical operations and quality officer Juan Andres to president of strategic partnerships and enterprise expansion, and poaching a former Novartis exec to take his place.

#AAO22 conference in Chicago (Photo credit: Associate editor Kyle LaHucik)

#AAO22: In bid for first FDA nod in ge­o­graph­ic at­ro­phy, Apel­lis claims an­oth­er first in eye dis­ease field

CHICAGO — Eight weeks before patients and industry find out if the FDA approves the first treatment for geographic atrophy, an advanced form of age-related macular degeneration, the biotech behind the drug is out with some new data on a secondary endpoint.

In what study investigator Charles Wykoff called the “first direct evidence of function preservation by slowing GA growth” in an investigational treatment, Apellis Pharmaceuticals’ drug pegcetacoplan led to less loss of retinal sensitivity versus sham  within 250 microns on either side of the GA lesion over 24 months.

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BioCryst's new website for its HAE unbranded campaign encourages patients to take charge of treatment decisions.

BioCryst launch­es aware­ness cam­paign around man­age­ment of rare vas­cu­lar dis­ease

While hereditary angioedema (HAE) is rare, treatment options for the condition have become much more common. So BioCryst Pharmaceuticals is taking a new angle in its recently launched HAE awareness campaign encouraging patients to take control of their disease management.

“Hereditary angioedema (HAE) isn’t the author of your story — you are … #cHAEngetheplan. Not the goal,” the US campaign website advises.

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EMA makes new rec­om­men­da­tions for Im­bru­vi­ca, med­i­cines with ter­li­pressin over res­pi­ra­to­ry and car­dio­vas­cu­lar risks

The EMA is handing out a new recommendation surrounding J&J and AbbVie’s cancer drug Imbruvica and some medicines containing terlipressin.

On Friday, the EMA’s safety committee (PRAC) stated that it has taken new measures to reduce the risk of respiratory failure and sepsis when medicines containing terlipressin that are used to treat people with hepatorenal syndrome (HRS-1), particularly for patients with advanced acute-on-chronic liver disease or advanced kidney failure.