As a promi­nent an­a­lyst pre­dicts (even­tu­al) drug price con­trols, bio­phar­ma braces for a blow while Trump balls his fist

Some­time this week, no one is sure when, Pres­i­dent Don­ald Trump is ex­pect­ed to give his long-await­ed speech on drug pric­ing. And a long line­up of key play­ers in the field has been scratch­ing their heads, anx­ious­ly try­ing to de­ter­mine just how far the na­tion’s most un­pre­dictable pres­i­dent could go.

Trump, of course, has been free with his pop­ulist brand of an­ti-in­dus­try rhetoric for more than a year now, af­ter es­tab­lish­ing the base­line with re­peat­ed as­ser­tions that phar­ma com­pa­nies have been get­ting away with mur­der for years. 

But there’s been lit­tle di­rect ac­tion, be­yond the work at the FDA to hus­tle up new gener­ic ap­provals while try­ing to use the pub­lic spot­light to whip up some fresh com­pe­ti­tion for the price gougers. 

FDA com­mis­sion Scott Got­tlieb like­ly tipped the ad­min­is­tra­tion’s hand on one point in a pre­view he of­fered on pric­ing last week, in­di­cat­ing that in­dus­try prac­tices in fight­ing a de­lay­ing ac­tion against gener­ics and biosim­i­lars will face some di­rect chal­lenges. But while sig­nif­i­cant, that by it­self won’t bend any longterm trends on pric­ing in fa­vor of pay­ers.

Mean­while, pub­lic anger over drug pric­ing is re­peat­ed­ly stoked with an un­end­ing se­ries of high pro­file as­saults on cer­tain play­ers that have used their pric­ing pow­er to jack up rev­enue from old drugs.

Just last night, Mallinck­rodt was back in the spot­light with Ac­thar, which has gone from $40 a vial in 2001 to $40,000, threat­en­ing the ba­sic po­lice and fire ser­vices pro­vid­ed by the town of Rock­ford, IL.

For­mer Rock­ford May­or Lar­ry Mor­ris­sey set the stage for a 60 Min­utes seg­ment with this quote: “‘Why is health­care so ex­pen­sive? Be­cause the fix is in. That’s the an­swer. That’s the short an­swer.”

And it’s not the kind of an­swer bio­phar­ma wants to hear right now, es­pe­cial­ly on 60 Min­utes.

The in­dus­try is still dis­grun­tled over the ad­min­is­tra­tion’s de­ci­sion to get phar­ma to cov­er part of the donut hole in Medicare drug prices. But they can live with that. There’s been a steady fo­cus at PhRMA on try­ing to shift the fo­cus from the man­u­fac­tur­ers to the in­sur­ers and PBMs that man­age the ben­e­fit. But it hasn’t worked. And re­cent­ly that tac­tic has pro­duced some painful­ly awk­ward sal­lies on so­cial me­dia that tends to at­tract as much spon­ta­neous laugh­ter as dis­cus­sion.

With pub­lic anger per­co­lat­ing at an un­prece­dent­ed rate — a ma­jor­i­ty told Kaiser Health that re­duc­ing drug pric­ing should be a top pri­or­i­ty of pol­i­cy mak­ers — some an­a­lysts be­lieve that what­ev­er Trump comes up with it won’t be enough to stop the one strat­e­gy that would make the bio­phar­ma in­dus­try reel: Price con­trols.

Se­nior Wells Far­go an­a­lyst David Maris put it in blunt terms a few days ago.

We be­lieve the US is on a longer-term arc to­ward price con­trols and low­er mar­gins, as there is an up­ris­ing that has been per­co­lat­ing for years against high drug prices. While the caus­es are many and the prob­lem much more com­pli­cat­ed and nu­anced than many oth­ers would lead read­ers to be­lieve, there is a re­bel­lion at hand. Ar­gu­ments about the cost to de­vel­op a drug or how oth­er sup­ply chain con­stituents are to blame are not ap­pre­ci­at­ed by the par­ent stand­ing at a phar­ma­cy counter aban­don­ing their child’s pre­scrip­tion be­cause they can­not af­ford it due to an out­ra­geous de­ducible or high co-pay. And ex­am­ples of un­con­scionable drug price in­creas­es have fur­ther hard­ened many con­sumer opin­ions. 

That day prob­a­bly won’t ar­rive this week. But if that’s the path we’re on, Trump is faced with ei­ther sat­is­fy­ing the anger with some grand com­pro­mis­es, or stok­ing it by pulling his punch­es.


Im­age: Pres­i­dent Don­ald Trump at a speech in Oc­to­ber, 2017. AP IM­AGES

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Fangliang Zhang (Imaginechina via AP Images)

The big mon­ey: Poised to make drug R&D his­to­ry, a Chi­na biotech un­veils uni­corn rac­ing am­bi­tions in a bid to raise $350M-plus on Nas­daq

Almost exactly three years after Shanghai-based Legend came out of nowhere to steal the show at ASCO with jaw-dropping data on their BCMA-targeted CAR-T for multiple myeloma, the little player with Big Pharma connections is taking a giant step toward making it big on Wall Street. And this time they want to seal the deal on a global rep after staking out a unicorn valuation in what’s turned out to be a bull market for biotech IPOs — in the middle of a pandemic.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Gilead re­leas­es an­oth­er round of murky remde­sivir re­sults

A month after the NIH declared the first trial on remdesivir in Covid-19 a success, Gilead is out with new results on their antiviral. But although the study met one of its primary endpoints, the data are likely to only add to a growing debate over how effective the drug actually is.

In a Phase III trial, patients given a 5-day dose of remdesivir were 65% more likely to show “clinical improvement” compared to an arm given standard-of-care. The trial, though, gave little indication for whether the drug had an impact on key endpoints such as survival or time-to-recovery. And in a surprising twist, a 10-day dosing arm of remdesivir didn’t lead to a statistically significant improvement over standard of care.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,900+ biopharma pros reading Endpoints daily — and it's free.

Iterum's fu­ture looks un­cer­tain, af­ter lead an­tibi­ot­ic fails con­sec­u­tive piv­otal stud­ies

While the market for antibiotics remains in tatters — unlike many of its bankrupt (or at the brink of bankruptcy) peers — Iterum is suffering not because its antibiotic isn’t selling, but because the compound has now failed back-to-back late-stage studies.

The experimental drug, sulopenem, was designed to tackle drug-resistant infections with an outpatient focus (in addition to hospitals), to avert those reimbursement challenges that incentivize hospitals to prescribe cheaper, generic broad-spectrum antibiotics.

Len Schleifer (left) and George Yancopoulos, Regeneron (Vimeo)

Eyes on he­mo­phil­ia prize, Re­gen­eron adds a $100M wa­ger on joint de­vel­op­ment cam­paign with In­tel­lia

When George Yancopoulos first signed up Intellia to be its CRISPR/Cas9 partner on gene editing projects 4 years ago, the upstart smartly ramped up its IPO at the same time. Today, Regeneron $REGN is coming back in, adding $100 million in an upfront fee and equity to significantly boot up a whole roster of new development projects.

And they’re highlighting some clinical hemophilia research plans in the process.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,900+ biopharma pros reading Endpoints daily — and it's free.

Covid-19 roundup: Did in­sid­ers cash in on pos­i­tive news re­port about Gilead be­fore pub­li­ca­tion?

A series of bullish trades on Gilead options just before the release of a favorable news story is raising questions among regulatory experts, Reuters reported.

On April 16, just hours before STAT published anecdotes from a Chicago hospital that served as one of the clinical sites to test Gilead’s remdesivir in Covid-19 patients, the California-based company’s shares were trading at around $75. Four large blocks of options were purchased for about $1.5 million each, betting that the stock would rise beyond that to as much as $87.5 by mid-August.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,900+ biopharma pros reading Endpoints daily — and it's free.

Jean-Jacques Bienaimé, BioMarin chairman and CEO

Bio­Marin holds the line on bleeds with 4-year val­rox up­date on he­mo­phil­ia A — but what's this about an­oth­er de­cline in Fac­tor 8 lev­els?

BioMarin has posted some top-line results for their 4-year followup on the most advanced gene therapy for hemophilia A — extending its streak on keeping a handful of patients free of bleeds and off Factor VIII therapy, but likely stirring fresh worries over a continued drop in Factor VIII levels.

We just don’t know how big a drop.

We’ll see more data when the results are presented at the World Federation of Hemophilia in a couple of weeks. But in a statement out Sunday night, BioMarin $BMRN reported that none of the patients required Factor VIII treatment, adding:

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,900+ biopharma pros reading Endpoints daily — and it's free.

IPOs abound in the time of coro­n­avirus, as For­ma Ther­a­peu­tics pen­cils in $150M Nas­daq de­but

The IPO engine is thriving, never mind the rampage of the coronavirus crisis on R&D timelines.

On Friday, along with synthetic lethality-focused biotech Repare Therapeutics, another Bristol Myers partner Forma Therapeutics also unveiled its plans to vault on to the Nasdaq — penciling in a target of $150 million.

The Watertown, Massachusetts-based company — which poached senior Genentech executive Frank Lee to take over the reins last year after more than a decade under founder Steve Tregay — raised a plump $100 million late last year, while shepherding its sickle cell disease (SCD) drug through an early-stage trial.

Roche nabs front­line OK for Avastin/Tecen­triq in com­mon liv­er can­cer, best­ing an old Bay­er drug

For the first time in 12 years, the FDA has approved a new frontline treatment for the most common form of liver cancer.

The agency okayed a combination of Roche’s anti-VEGF antibody Avastin and their immunotherapy Tecentriq for patients with unresectable or metastatic hepatocellular carcinoma (HCC). The approval comes two weeks after Roche and their big biotech sub Genentech published Phase III results showing the combo improved both progression-free survival and, crucially, helped patients live longer than the long-running standard-of-care, Bayer’s Nexavar.