Pralu­ent Odyssey: Re­gen­eron/Sanofi gun for mar­ket break­out with first-ever mor­tal­i­ty ben­e­fit — and a big price dis­count

Few drug ri­val­ries have been car­ried out with more di­rect, hand-to-hand fight­ing than the scrap over the PC­SK9 mar­ket. Mar­gin­al as the mar­ket is now, the com­bined forces of Re­gen­eron and Sanofi al­lied against Am­gen have nev­er­the­less punched it out in court and the play­ing field in the de­vout be­lief that bil­lions are at stake. And all three have had to strug­gle with a pay­ers’ back­lash against the two drugs they’re sell­ing.

Now Re­gen­eron/Sanofi are launch­ing a full scale as­sault, armed with new da­ta from the 18,924-pa­tient Odyssey Out­comes study high­light­ing ground­break­ing ev­i­dence of a mor­tal­i­ty ben­e­fit for Pralu­ent that they be­lieve will give them an edge. And they’re fol­low­ing through by dra­mat­i­cal­ly mark­ing down the pub­lic price they are propos­ing to charge for pay­ers will­ing to fi­nal­ly dis­man­tle the hur­dles they’ve erect­ed to stop their mem­bers from tak­ing their cho­les­terol treat­ment.

Just as Am­gen won ku­dos for its work to con­vince pay­ers to do the same with mon­ey-back guar­an­tees, this new round marks the in­dus­try’s in­creas­ing will­ing­ness to ne­go­ti­ate over what it charges — a trend that is cre­at­ing a sea change over some drugs that have to sur­vive in a com­pet­i­tive scene.

First, here’s the long-await­ed Odyssey Out­comes da­ta, re­leased at the Amer­i­can Col­lege of Car­di­ol­o­gy’s sci­en­tif­ic meet­ing in Or­lan­do.

Re­searchers say they have con­clu­sive ev­i­dence that Pralu­ent not on­ly sig­nif­i­cant­ly re­duced the num­ber of car­dio in­ci­dents ex­pe­ri­enced by pa­tients on the LDL drug, with the vast ma­jor­i­ty al­so on high-in­ten­si­ty statins, they al­so have a clear re­duc­tion in deaths — the goal that elud­ed Am­gen when it float­ed their Fouri­er da­ta last sum­mer.

— Look­ing at MACE (Ma­jor Ad­verse Car­diac Events) among pa­tients with an LDL lev­el over 100 mg/dL, there was a drop of 3.4 points be­tween the place­bo arm and Pralu­ent rates — 14.9% to 11.5%, a risk re­duc­tion of 24%.

— For CV death the rate dropped from 4.2% to 2.9%.

— And for all-cause deaths the de­crease was 5.7% to 4.1%, with a 1.7 point drop; a 29% drop in risk.

To put it an­oth­er way, for every 1,000 pa­tients who met this pro­file and were on drug, 17 peo­ple would still be alive. And that, say the part­ners, should be the telling dif­fer­ence for pay­ers.

To dri­ve that mes­sage home, Sanofi to­day has agreed to cut their price of Pralu­ent to $4,460 to $7,975 a year for pay­ers who are will­ing to stop throw­ing up road­blocks to this drug, drop­ping to a price that on the low end is not much high­er than the $10-a-day cost of the old gen­er­a­tion of statins be­fore they went gener­ic. At the high end, it still marks a sharp drop from the $14,000 whole­sale price that had been the mar­ket stan­dard used to peg dis­counts against.

“Too many pa­tients in ur­gent need of ad­di­tion­al treat­ment op­tions on top of statins have faced tremen­dous hur­dles to gain ac­cess to this im­por­tant med­i­cine. We are pre­pared to change this by im­prov­ing ac­cess and af­ford­abil­i­ty, elim­i­nat­ing these bur­den­some bar­ri­ers for high-risk pa­tients in need,” said Sanofi CEO Olivi­er Brandi­court in a state­ment. “We will be­gin work­ing with pay­ers to en­sure that high-risk pa­tients have ap­pro­pri­ate ac­cess. This is the right thing to do for pa­tients.”

That was not an easy con­ces­sion for Sanofi, but these drugs are earn­ing on­ly a frac­tion of what an­a­lysts had ex­pect­ed.

At this point, there’s vir­tu­al­ly no ques­tion that these drugs work as billed, slash­ing LDL lev­els safe­ly. In fact, many an­a­lysts would al­so tell you that they are re­mark­ably sim­i­lar. Per­haps they were a lit­tle too sim­i­lar for a judge who once or­dered Re­gen­eron and Sanofi to yank their drug dur­ing their court bat­tle with Am­gen over the patents in­volved. That rul­ing was over­turned, but the le­gal fight con­tin­ues.

Re­gen­eron/Sanofi are com­ing out with a dif­fer­en­ti­at­ed pro­file now, though, af­ter Am­gen hus­tled its Repatha out­comes da­ta out last year, trum­pet­ing sig­nif­i­cant re­duc­tions in car­dio events but no mor­tal­i­ty ben­e­fit. And pay­ers re­mained unim­pressed.

Why did Odyssey Out­comes suc­ceed on that mor­tal­i­ty score?

Bob Pordy, the vice pres­i­dent of car­dio­vas­cu­lar and me­tab­o­lism ther­a­peu­tics at Re­gen­eron, says you can mark that down to the fact they fo­cused on a group of ACS pa­tients who were by far at the high­est risk, with a sig­nif­i­cant­ly longer du­ra­tion of treat­ment in the study.

“Odyssey Out­comes stud­ied Pralu­ent in the high­est risk pa­tients,” echoes Jay Edel­berg, Sanofi’s head of car­dio de­vel­op­ment. “They re­main at high risk of re­cur­ring heart at­tacks. By study­ing these high­est risk pa­tients, we were able to demon­strate re­duc­tions in MACE and a re­duc­tion in mor­tal­i­ty.”

While most of the at­ten­tion in the cho­les­terol mar­ket has cen­tered on the two PC­SK9 drugs, the two key com­pa­nies aren’t alone here. Es­pe­ri­on CEO Tim Mayleben has made it clear that they plan to charge un­der $4,000 for their late-stage cho­les­terol pill, now in late-stage de­vel­op­ment. That price point may not look like so much of a bar­gain now. And The Med­i­cines Com­pa­ny has been putting all their eggs in one RNAi bas­ket on LDL cho­les­terol as well, which may al­so face al­tered prospects.

This kind of pub­lic dis­cus­sion about re­tail drug prices is a re­mark­able change for an in­dus­try that has ben­e­fit­ed enor­mous­ly by keep­ing their true prices as opaque as pos­si­ble. But now da­ta aren’t enough, as pay­ers find new ways to put some dis­tance be­tween them­selves and the mass mar­ket drugs that threat­en to eat in­to their mar­gins.

Now you al­so have to pub­licly com­pete on the stick­er price, on oc­ca­sion.

Im­age: Sanofi CEO Olivi­er Brandi­court on Jan­u­ary 31, 2018  Alek­sey Nikol­skyi/Sput­nik via AP

Tesla and SpaceX founder Elon Musk gestures to the audience after being recognized by President Trump following the successful launch of a Falcon 9 rocket at the Kennedy Space Center. (via Getty Images)

Tes­la chief Elon Musk teams up with Covid-19 play­er Cure­Vac to build 'R­NA mi­cro­fac­to­ries'

Elon Musk has joined the global tech crusade now underway to revolutionize vaccine manufacturing — now aimed at delivering billions of doses of a new mRNA vaccine to fight Covid-19. And he’s cutting right to the front.

In a late-night tweet Wednesday, the Tesla chief announced:

Tesla, as a side project, is building RNA microfactories for CureVac & possibly others

That’s not a lot to go on. But the tweet comes a year after Tesla’s German division in Grohmann and CureVac filed a patent on a “bioreactor for RNA in vitro transcription, a method for RNA in vitro transcription, a module for transcribing DNA into RNA and an automated apparatus for RNA manufacturing.” CureVac, in the meantime, has discussed a variety of plans to build microfactories that can speed up the whole process for a global supply chain.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 84,600+ biopharma pros reading Endpoints daily — and it's free.

George Yancopoulos (Regeneron)

Re­gen­eron co-founder George Yan­copou­los of­fers a com­bat­ive de­fense of the po­lice at a high school com­mence­ment. It didn’t go well

Typically, the commencement speech at Yorktown Central School District in Westchester — like most high schools — is an opportunity to encourage students to face the future with confidence and hope. Regeneron president and co-founder George Yancopoulos, though, went a different route.

In a fiery speech, the outspoken billionaire defended the police against the “prejudice and bias against law enforcement” that has erupted around the country in street protests from coast to coast. And for many who attended the commencement, Yancopoulos struck the wrong note at the wrong time, especially when he combatively challenged someone for interrupting his speech with a honk for “another act of cowardness.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 84,600+ biopharma pros reading Endpoints daily — and it's free.

Elias Zerhouni (Photo by Vincent Isore/IP3/Getty Images)

Elias Zer­houni dis­cuss­es ‘am­a­teur hour’ in DC, the de­struc­tion of in­fec­tious dis­ease R&D and how we need to prep for the next time

Elias Zerhouni favors blunt talk, and in a recent discussion with NPR, the ex-Sanofi R&D and ex-NIH chief had some tough points to make regarding the pandemic response.

Rather than interpret them, I thought it would be best to provide snippets straight from the interview.

On the Trump administration response:

It was basically amateur hour. There is no central concept of operations for preparedness, for pandemics, period. This administration doesn’t want to or has no concept of what it takes to protect the American people and the world because it is codependent. You can’t close your borders and say, “OK, we’re going to be safe.” You’re not going to be able to do that in this world. So it’s a lack of vision, basically just a lack of understanding, of what it takes to protect the American people.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 84,600+ biopharma pros reading Endpoints daily — and it's free.

Dan Gold, MEI Pharma CEO

De­vel­op­ment part­ners at MEI, Helsinn dump a high-risk PhI­II AML study af­ter con­clud­ing it would fail sur­vival goal

Four years after Switzerland’s Helsinn put $25 million of cash on the table for an upfront and near-term milestone to take MEI Pharma’s drug pracinostat into a long-running Phase III trial for acute myeloid leukemia, the partners are walking away from a clinical pileup.

The drug — an HDAC inhibitor — failed to pass muster during a futility analysis, as researchers concluded that pracinostat combined with azacitidine wasn’t going to outperform the control group in the pivotal.

No­vavax snags Ben Machielse for CMC and pro­motes a trio of staffers; Mar­ty Du­vall lands an­oth­er CEO post at On­copep­tides

Novavax has been making waves recently by securing a $384 million commitment from CEPI to cover R&D and manufacturing for its Covid-19 vaccine while also spending $167 million on a 150,000 square-foot facility. The Maryland biotech continues to shore up its leadership team as well, bringing in Ben Machielse as their EVP of CMC just a couple weeks after nabbing AstraZeneca vet Filip Dubrovsky as their new CMO. Machielse was president and CEO of Vtesse from 2014-17, and before that, he also spent more than 11 years at MedImmune and was EVP of operations for the back half of his tenure.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 84,600+ biopharma pros reading Endpoints daily — and it's free.

No­var­tis los­es biosim­i­lar ap­peal as court up­holds a 31-year mo­nop­oly by Am­gen's En­brel

A new court ruling has strengthened Amgen’s grip on the IP estate around Enbrel, keeping biosimilars of the autoimmune and inflammatory drug at bay until 2029.

Novartis, the patent challenger, isn’t throwing in the towel yet. In a statement noting the failed appeal, its generics division Sandoz noted its reviewing options, “including potential appeal to US Supreme Court.”

It’s been almost four years since the FDA approved Erelzi, Sandoz’s copycat version of Enbrel. While sales of the Pfizer-partnered drug in the US — the market Amgen is in charge of — have dipped slightly during that time, it remains a solid megablockbuster with 2019 revenue slightly above $5 billion.

Pfiz­er shares surge on pos­i­tive im­pact of their mR­NA Covid-19 vac­cine — part­nered with BioN­Tech — in an ear­ly-stage study

Pfizer and their partners at the mRNA specialist BioNTech have published the first glimpse of biomarker data from an early-stage study spotlighting the “robust immunogenicity” triggered by their Covid-19 vaccine, which is one of the leaders in the race to vanquish the global pandemic.

Researchers selected 45 healthy volunteers 18-55 years of age for the study. They were randomized to receive 2 doses, separated by 21 days, of 10 µg, 30 µg, or 100 µg of BNT162b1, “a lipid nanoparticle-formulated, nucleoside-modified, mRNA vaccine that encodes trimerized SARS-CoV-2 spike glycoprotein RBD.” Their responses were compared against the effect of a natural, presumably protective defense offered by a regular infection.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 84,600+ biopharma pros reading Endpoints daily — and it's free.

An ex­pe­ri­enced biotech is stitched to­geth­er from transpa­cif­ic parts, with 265 staffers and a fo­cus on ‘new bi­ol­o­gy’

Over the past few years, different teams at a pair of US-based biotechs and in labs in Japan have labored to piece together a group of cancer drug programs, sharing a single corporate umbrella with research colleagues in Japan. But now their far-flung operations have been knit together into a single unit, creating a pipeline with 10 cancer drug development programs — going from early-stage right into Phase III — and a host of discovery projects managed by a collective staff of some 265 people.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 84,600+ biopharma pros reading Endpoints daily — and it's free.

Sec­ond death trig­gers hold on Astel­las' $3B gene ther­a­py biotech's lead pro­gram, rais­ing fresh con­cerns about AAV

Seven months after Astellas shelled out $3 billion to acquire the gene therapy player Audentes, the biotech company’s lead program has been put on hold following the death of 2 patients taking a high dose of their treatment. And there was another serious adverse event recorded in the study as well, with a total of 3 “older” patients in the study affected.

The incidents are derailing plans to file for a near-term approval, which had been expected right about now.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 84,600+ biopharma pros reading Endpoints daily — and it's free.