Stephen Ubl, president and chief executive officer of PhRMA (Photographer: Andrew Harrer/Bloomberg via Getty Images)

Two sides of 'his­toric' drug pric­ing bill: Phar­ma in­dus­try blasts 'mis­take' while ad­vo­cates plan for 'first ever' gains

Both Con­gres­sion­al De­moc­rats and the phar­ma in­dus­try agree that pend­ing drug pric­ing leg­is­la­tion is “his­toric” — they just di­a­met­ri­cal­ly dis­agree on the mod­i­fi­er.

Trade in­dus­try group PhRMA’s CEO Stephen Ubl called it an “his­toric mis­take” on Wednes­day, joined by Eli Lil­ly CEO David Ricks, At­las Ven­ture part­ner Jean-Fran­cois Formela along with a hema­tol­ogy on­col­o­gy-lead­ing physi­cian and a metasta­t­ic breast can­cer sur­vivor in a press con­fer­ence.

Mean­while, Sen­ate De­moc­rats lined up their own press con­fer­ence for lat­er in the day, tap­ping pa­tient ad­vo­ca­cy lead­ers and AARP CEO Jo Ann Jenk­ins to of­fer its his­to­ry-mak­ing take.

“This leg­is­la­tion would be his­toric, and that’s not an ex­ag­ger­a­tion,” said David Mitchell, founder of Pa­tients for Af­ford­able Drugs and speak­er at the Hill event. “Why his­toric? For the first time ever, af­ter al­most 20 years of fight­ing, Medicare will be able to use its pur­chas­ing pow­er to ne­go­ti­ate low­er drug prices for Amer­i­can. For the first time ever, there are go­ing to be curbs on an­nu­al drug price in­creas­es to lim­it to no more than the rate of in­fla­tion. And for the first time ever, there’s go­ing to be an an­nu­al cope on what Medicare Part D ben­e­fi­cia­ries pay for our drugs an­nu­al­ly.”

While nei­ther side can claim vic­to­ry for now, the bill spon­sors have hint­ed that pas­sage should be ex­pect­ed as they can use rec­on­cil­i­a­tion and on­ly need a sim­ple ma­jor­i­ty to pass the bill. The House has al­so sig­nalled a will­ing­ness to re­turn from their sum­mer re­cess to pass the bill next month.

Sen­ate Dems and Re­pub­li­cans met last week with the Sen­ate par­lia­men­tar­i­an to iron out what pro­vi­sions could be in­clud­ed if they use this short-cut, but sen­a­tors who are usu­al­ly on the fence, like Sen. Joe Manchin (D-WV), are now on board.

PhRMA, not sur­pris­ing­ly, sees those pro­vi­sions dif­fer­ent­ly — with Ubl call­ing Medicare ne­go­ti­a­tions “non­sense.”

“An in­no­v­a­tive drug­mak­er has two choic­es un­der this bill — ac­cept the gov­ern­ment’s price or pay a 95% tax on the sale of that med­i­cine,” he said, adding, “That’s not ne­go­ti­a­tion, that’s gov­ern­ment price set­ting. Let’s be hon­est and call it what it is.”

PhRMA is al­so unit­ed in the view that the bill’s pro­vi­sions, in­clud­ing a cap on the num­ber of years phar­mas can in­de­pen­dent­ly set drug prices, will stymie in­no­va­tion, and keyed in on can­cer treat­ments as an ex­am­ple.

Ricks said the bill will af­fect “de­ci­sions we make about how to in­vest in in­no­v­a­tive med­i­cines and those in par­tic­u­lar for can­cer.”

He out­lined two spe­cif­ic ef­fects on a like­ly de­crease in drug de­vel­op­ment for rare can­cers with small­er pop­u­la­tions (which cost just as much to de­vel­op as those for larg­er tar­get au­di­ences) and slow­ing ear­ly stage can­cer drug de­vel­op­ment. Can­cer drugs are of­ten ap­proved for lat­er-stage use, then move ear­li­er over time and ad­di­tion­al re­search to get to ad­ju­vant us­es, he said.

“Man­u­fac­tur­ers and in­vestors won’t sup­port that type of se­quen­tial de­vel­op­ment (any­more),” he said.

An­oth­er point of con­tention is the im­pact on new drug de­vel­op­ment. The Con­gres­sion­al Bud­get Of­fice (CBO) es­ti­mat­ed the bill would re­duce drug­mak­ers’ 1,300 to­tal ap­provals by about 10 drugs over the next three decades.

Ubl said the CBO “just got it wrong.”

He point­ed in­stead to Uni­ver­si­ty of Chica­go re­search ear­li­er this year that es­ti­mat­ed 135 few­er drug ap­provals through 2039 amid its pro­ject­ed drop of $663 bil­lion in R&D spend­ing.

Ricks said, “I would be shocked if the im­pact of this bill doesn’t re­sult in 15 few­er med­i­cines from Eli Lil­ly and Co. alone. I think that would im­ply one every oth­er year that we can­celled be­cause of this. But right now, 40% of our port­fo­lio are small mol­e­cules. We’ll need to reeval­u­ate every sin­gle one of those projects for vi­a­bil­i­ty.”

He al­so pre­dict­ed the po­ten­tial for R&D in­no­va­tion to leave the US, point­ing to the fact that R&D moved from Eu­rope to the US be­cause “Eu­rope has the same harm­ful poli­cies as em­bed­ded in this bill” caus­ing in­vest­ments to shift.

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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Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

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.

Gilead names 'k­ing­pin­s' in coun­ter­feit HIV med law­suit

Gilead is mounting its counterfeit drug lawsuit, naming two “kingpins” and a complex network of conspirators who allegedly sold imitation bottles of its HIV meds, some of which ended up in US pharmacies.

The pharma giant on Wednesday provided an update on what it called a “large-scale, sophisticated counterfeiting conspiracy,” accusing two new defendants of “leading and orchestrating” a scheme to sell hundreds of millions of dollars in illegitimate drugs posing as meds such as Biktarvy and Descovy.

Tar­sus looks to raise aware­ness of eye­lid mite dis­ease in cam­paign aimed at eye­care spe­cial­ists

Eyelid mite disease may be “gross” but it’s also fairly common, affecting about 25 million people in the US.

Called demodex blepharitis, it’s a well-known condition among eyecare professionals, but they often don’t always realize how common it is. Tarsus Pharmaceuticals wants to change that with a new awareness campaign called “Look at the Lids.”

The campaign and website debut Thursday — just three weeks after Tarsus filed for FDA approval for a drug that treats the disease.

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