Sanofi/Re­gen­eron’s one-two punch on PC­SK9 price and health da­ta wins a key con­vert as Ex­press Scripts cre­ates ex­clu­sive for­mu­la­ry deal

Just 7 weeks af­ter Sanofi $SNY and Re­gen­eron $REGN flagged piv­otal da­ta on a clear mor­tal­i­ty ben­e­fit to be had from its PC­SK9 car­dio drug Pralu­ent, they’re back with a chum­my pub­lic self­ie with Ex­press Scripts that swaps a new dis­count price in ex­change for a pledge to dis­man­tle any hur­dles that could pre­vent the PBM’s 20 mil­lion mem­bers from tak­ing ad­van­tage of it.

Len Schleifer

No­body’s talk­ing specifics here, but in March Sanofi/Re­gen­eron pledged to cut its price 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.

Salt­ing that deal with new mor­tal­i­ty ben­e­fits from longterm use, a goal that elud­ed ri­val Am­gen, ap­pears to be enough for at least one of the most promi­nent phar­ma­cy play­ers to see their way through to drop­ping the com­plex ap­proval process that made this drug a non­event on the bot­tom line. Ex­press Scripts is giv­ing Sanofi/Re­gen­eron an ex­clu­sive place on their for­mu­la­ry — take that, Am­gen — with a pledge to pass along the sav­ings to its mem­bers through low­er out-of-pock­et ex­pens­es.

The blunt Re­gen­eron CEO Len Schleifer said the deal was “de­signed to break the grid­lock so that Pralu­ent is fi­nal­ly able to reach pa­tients most in need. U.S. car­di­ol­o­gists have ex­pe­ri­enced un­prece­dent­ed chal­lenges in se­cur­ing ac­cess for Pralu­ent for pa­tients who were clear­ly ap­pro­pri­ate, but were de­nied cov­er­age. This agree­ment sets a new stan­dard in in­dus­try and pay­er col­lab­o­ra­tion that we hope will serve as a mod­el for how to make in­no­v­a­tive med­i­cines more ac­ces­si­ble and af­ford­able.” 

And ICER chief Steven Pear­son of­fered his en­dorse­ment, hap­py to see some re­al ne­go­ti­at­ing on price and ac­cess. He not­ed:

Fol­low­ing a decades-long trend to­ward dys­func­tion and fin­ger-point­ing, the US health care sys­tem is be­gin­ning to ad­dress its drug pric­ing prob­lem through the emer­gence of a ‘grand bar­gain.’ When a man­u­fac­tur­er is will­ing to re­spon­si­bly price an in­no­v­a­tive med­i­cine in line with its clin­i­cal ben­e­fits, pay­ers should rec­i­p­ro­cate by re­mov­ing the hur­dles that can pre­vent pa­tients from get­ting the drug.

Mike Suesser­man

Sanofi/Re­gen­eron and Am­gen have been duk­ing it out over who owns the patents on PC­SK9, but both have been es­sen­tial­ly side­lined by pay­ers’ per­sis­tent re­fusal to hit a green light on these drugs. With physi­cians star­ing at a pile of pa­per­work and re­peat­ed de­nials, these com­pa­nies nev­er had a chance at the orig­i­nal high price they want­ed to fetch. Re­al­i­ty dawned slow­ly, but the sun is com­ing up on a field that a host of an­a­lysts still res­olute­ly be­lieves is a block­buster mar­ket in the mak­ing.

“We’ve been out on the mar­ket close to three years,” says Mike Suesser­man, VP of the car­diometa­bol­ic and opthamol­o­gy busi­ness unit at Re­gen­eron. They’ve seen lots of feed­back along the way and iden­ti­fied what the crit­i­cal hur­dles are. This deal, he says, ad­dress­es those hur­dles.

“In that sense I think it’s a huge leap for­ward.”

You can bet that more such leaps for­ward are in the works as Am­gen looks to strike back on the phar­ma­cy front.

Inside FDA HQ (File photo)

The FDA just ap­proved the third Duchenne MD drug. And reg­u­la­tors still don’t know if any of them work

Last year Sarepta hit center stage with the FDA’s controversial reversal of its CRL for the company’s second Duchenne muscular dystrophy drug — after the biotech was ambushed by agency insiders ready to reject a second pitch based on the same disease biomarker used for the first approval for eteplirsen, without actual data on the efficacy of the drug.

On Wednesday the FDA approved the third Duchenne MD drug, based on the same biomarker. And regulators were ready to act yet again despite the lack of efficacy data.

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Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Franz-Werner Haas, CureVac CEO

UP­DAT­ED: On the heels of a snap $1B raise, Cure­Vac out­lines plans to seek emer­gency OK for Covid-19 vac­cine -- shares rock­et up

CureVac is going from being one of the quietest players in the race to develop a new vaccine to fight the worst public health crisis in a century to a challenger for the multibillion-dollar market that awaits the first vaccines to make it over the finish line. Typically low-key at a time of brash comments and incredibly ambitious development timelines from the leaders, CureVac now is jumping straight into the spotlight.

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US gov­ern­ment re­port­ed­ly be­gins prepar­ing for Covid-19 chal­lenge tri­als. Are they eth­i­cal?

Controversial human challenge trials for potential Covid-19 vaccines reportedly have a new booster — the US government.

Scientists working for the government have begun manufacturing a strain of the novel coronavirus that could be used in such studies, Reuters reported Friday morning. The trials would enroll healthy volunteers to be vaccinated and then intentionally infected with a weakened coronavirus.

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Sanofi vet Kather­ine Bowdish named CEO of PIC Ther­a­peu­tics; As the world Terns: Liv­er dis­ease biotech makes ex­ec­u­tive changes

PIC Therapeutics hasn’t raised much money, yet. But the fledgling biotech has attracted a high-profile player to the helm.

The Boston-based biotech has handed the reins to Katherine Bowdish as its president and CEO. Bowdish will also join the board of directors of PIC. Bowdish joins from Sanofi where she served as VP and head of R&D strategy, as well as helping launch and lead Sanofi Sunrise, a venture investment and partnering vehicle at Sanofi. Before that, Bowdish held several exec roles at Permeon Biologics, Anaphore, Alexion Pharmaceuticals and Prolifaron (acquired by Alexion).

Martin Shkreli (Shutterstock)

Mar­tin Shkre­li con­tin­ued to or­ches­trate an­ti-com­pet­i­tive schemes for Dara­prim be­hind bars — FTC

Martin Shkreli didn’t just blog, read up on drug development news and run his biotech business with a contraband cell phone in prison. According to the FTC, he was also coordinating the anticompetitive scheme to shield Daraprim — the drug at the center of a price-gouging controversy that earned him the “Pharma Bro” nickname — from generic rivals.

Back in January the FTC, together with New York’s attorney general, launched a federal lawsuit against Shkreli, who’s now serving a 7-year sentence for defrauding investors in his hedge fund, alleging that he effectively created a drug monopoly. While Shkreli’s notorious move to raise the per tablet price of Daraprim from $17.50 to $750 was perfectly legal, the tactics he allegedly deployed to box out competitors weren’t.

Stéphane Bancel speaks to President Donald Trump at the White House meeting on March 2 (AP Images)

UP­DAT­ED: Mod­er­na of­fers steep dis­count in US sup­ply deal — but still takes the crown with close to $2.5B in vac­cine con­tracts

The US pre-order for Moderna’s Covid-19 vaccine is in.

Operation Warp Speed is reserving $1.525 billion for 100 million doses of Moderna’s Phase III mRNA candidate, rounding out to about $15 per dose — including $300 million in incentive payments for timely delivery. Given that Moderna has a two-dose regimen, it’s good for vaccinating 50 million people. The US government also has the option to purchase another 400 million doses for a total of $6.6 billion, or $16.5 per dose.

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A lab technician works during research on coronavirus at Johnson & Johnson subsidiary Janssen Pharmaceutical in Beerse, Belgium, Wednesday, June 17, 2020. (Virginia Mayo/AP Images)

UP­DAT­ED: End­points News ranks all 28 play­ers in the Covid-19 vac­cine race. Here's how it stacks up to­day

(This piece was last updated on August 14. Endpoints News will continue to track the latest developments through the FDA’s marketing decisions.)

The 28 players now in or close to the clinical race to get a Covid-19 vaccine over the finish line are angling for a piece of a multibillion-dollar market. And being first — or among the leaders — will play a big role in determining just how big a piece.

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Cal­lid­i­tas bets up to $102M on a biotech buy­out, snag­ging a once-failed PBC drug

After spending years developing its oral formulation of the corticosteroid budesonide, Sweden’s Calliditas now has its sights set on the primary biliary cholangitis field.

The company will buy out France-based Genkyotex, and it’s willing to bet up to €87 million ($102 million) that Genkyotex’s failed Phase II drug, GKT831, will do better in late-stage trials.

Under the current agreement, Calliditas $CALT will initially pay €20.3 million in cash for 62.7% of Genkyotex (or €2.80 a piece for 7,236,515 shares) in early October, then circle back for the rest of Genkyotex’s shares under the same terms. If nothing changes, the whole buyout will cost Calliditas €32.3 million, plus up to  €55 million in contingent rights.