Ver­tex's cys­tic fi­bro­sis drugs work, but they're too ex­pen­sive, ICER finds

Ear­li­er this month, Resh­ma Ke­wal­ra­mani took over the reins as Ver­tex Phar­ma­ceu­ti­cals’ CEO — she will now be tasked with grap­pling with crit­i­cism from cost-ef­fec­tive­ness watch­dog ICER, which on Mon­day is­sued a re­port sug­gest­ing that de­spite the health gains of­fered by its suite of cys­tic fi­bro­sis drugs, their prices are far too high to be sus­tain­able for pa­tients and health care sys­tems.

David Rind

“De­spite be­ing trans­for­ma­tive ther­a­pies, the prices set by the man­u­fac­tur­er – cost­ing many mil­lions of dol­lars over the life­time of an av­er­age pa­tient – are out of pro­por­tion to their sub­stan­tial ben­e­fits,” said David Rind, ICER’s chief med­ical of­fi­cer in a state­ment.

The cys­tic fi­bro­sis drugs made by Ver­tex are the first treat­ments that ad­dress the un­der­ly­ing ge­net­ic caus­es of CF, which is char­ac­ter­ized by thick sticky mu­cus in the lungs, di­ges­tive sys­tem and oth­er or­gans that re­duces life ex­pectan­cy. Ver­tex’s med­i­cines tar­get the cys­tic fi­bro­sis trans­mem­brane con­duc­tance reg­u­la­tor (CFTR) gene and are en­gi­neered to cor­rect the mal­func­tion­ing pro­tein it makes.

A lit­tle over 300 dif­fer­ent mu­ta­tions are known to cause CF, and pa­tients typ­i­cal­ly car­ry path­o­gen­ic mu­ta­tions in both copies of the CFTR gene — the most com­mon such mu­ta­tion is F508del. The com­pa­ny’s Orkam­bi and Symke­vi fo­cus on the more com­mon F508del mu­ta­tion. Ver­tex’s first-ever ap­proved CF drug is Ka­ly­de­co. Its triplet reg­i­men, Trikaf­ta, which is de­signed to treat 90% of all CF pa­tients, was ap­proved by the FDA a spec­tac­u­lar five months ahead of its ex­pect­ed de­ci­sion date in Oc­to­ber.

“When a man­u­fac­tur­er has a mo­nop­oly on treat­ments and is aware that in­sur­ers will be un­able to refuse cov­er­age, the lack of usu­al coun­ter­bal­anc­ing forces can lead to ex­ces­sive prices. Pa­tients who re­ceive the treat­ments will ben­e­fit, but un­aligned prices will cause sig­nif­i­cant neg­a­tive health con­se­quences for many un­seen in­di­vid­u­als – those through­out so­ci­ety who will ex­pe­ri­ence fi­nan­cial tox­i­c­i­ty and may have to de­lay care, forego care, or even aban­don in­sur­ance as their out of pock­et costs and pre­mi­ums are dri­ven up­ward,” said Rind.

There was no doubt that all three drugs, most no­tably Trikaf­ta, of­fer a clin­i­cal ben­e­fit over stan­dard sup­port­ive CF care — ICER’s analy­sis sug­gest­ed that dis­counts of up to 77% would be im­per­a­tive to align prices with the drugs’ clin­i­cal val­ue.

Source: ICER, 2020

Click on the im­age to see the full-sized ver­sion

The in­sti­tute worked up a health ben­e­fit price bench­mark (HBPB) — a price range in­di­cat­ing the high­est price a man­u­fac­tur­er should charge for a treat­ment, based on the amount of im­prove­ment in over­all health pa­tients achieve on that ther­a­py. Each of the four ther­a­pies, ac­cord­ing to this analy­sis, re­quires a sub­stan­tial dis­count, with­out which Ver­tex risks caus­ing “dis­pro­por­tion­ate­ly greater loss­es in health among oth­er pa­tients in the health sys­tem due to ris­ing over­all costs of health care and health in­sur­ance.”

Source: ICER, 2020

Click on the im­age to see the full-sized ver­sion

Al­though these drugs were de­vel­oped with sig­nif­i­cant fi­nan­cial and sci­en­tif­ic sup­port from the Cys­tic Fi­bro­sis Foun­da­tion, a rep­re­sen­ta­tive from the group sug­gest­ed that it was not in­clud­ed in Ver­tex’s dis­cus­sions around how the prod­ucts would be priced, ICER said.

Akin to NICE in the UK, ICER is an in­de­pen­dent body that an­a­lyzes the cost-ef­fec­tive­ness of drugs and oth­er med­ical ser­vices in the Unit­ed States. Un­like NICE, though, ICER is not gov­ern­ment-af­fil­i­at­ed, but its de­ter­mi­na­tions are in­creas­ing­ly gain­ing trac­tion with pay­ers and pol­i­cy­mak­ers. Man­u­fac­tur­ers fre­quent­ly at­tack the method­olo­gies the in­sti­tute em­ploys in its analy­ses.

So­cial im­age: Resh­ma Ke­wal­ra­mani, Ver­tex CEO via YouTube

Jude Samulski, Marianne De Backer

Bay­er buys a biotech ‘race horse’ with a $4B deal — $2B in cash — aimed at go­ing big in­to gene ther­a­py

In the latest sign that Big Pharma wants a leading place in the push to develop a new generation of cell and gene therapies, Bayer is stepping up today with a $2 billion cash deal to buy out one of the fast-moving pioneers in the field, while adding up to $2 billion more in milestones if the new pharma subsidiary can deliver the goods.

As part of a continuing series of deals engineered by Bayer BD chief Marianne De Backer, the pharma player has snapped up Asklepios, more commonly referred to in more casual fashion as AskBio. And they are paying top dollar for a Research Triangle Park-based company that raised $225 million a little more than a year ago to back the brainchild of Jude Samulski, the gene therapy pioneer out of the University of North Carolina Gene Therapy Center.

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Patrick Soon-Shiong at the JP Morgan Healthcare Conference, Jan. 13, 2020 (David Paul Morris/Bloomberg via Getty Images)

Af­ter falling be­hind the lead­ers, dissed by some ex­perts, biotech show­man Patrick Soon-Sh­iong fi­nal­ly gets his Covid-19 vac­cine ready for a tri­al. But can it live up to the hype?

In January, when dozens of scientists rushed to start making a vaccine for the then-novel coronavirus, they were joined by an unlikely compatriot: Patrick Soon-Shiong, the billionaire doctor most famous for making big, controversial promises on cancer research.

Soon-Shiong had spent the last 4 years on his “Cancer Moonshot,” but part of his project meant buying a small Seattle biotech that specialized in making common-cold vectors, called adenoviruses, to train the immune system. The billionaire had been using those vectors for oncology, but the company had also developed vaccine candidates for H1N1, Lassa fever and other viruses. When the outbreak began, he pivoted.

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Once re­ject­ed, Kala's dry eye drug now gains en­try to a field where No­var­tis is groom­ing its own block­buster

When the FDA slapped a rejection on Kala Pharma’s dry eye drug last August, the biotech’s execs promised investors that a third Phase III study — they had already started at that point — would reverse their fortune.

Today they made good on that promise, clinching an approval for Eysuvis, an ocular corticosteroid being positioned as a first-line, short term treatment of dry eye disease.

Boasting a technology invented by Bob Langer out of MIT, Eysuvis is a corticosteroid, loteprednol etabonate, delivered by mucus-penetrating particles. It promises to enhance penetration into target tissue on the ocular surface, achieving an effect quicker than systemic corticosteroids and stronger than over-the-counter eye drops.

Cedric Francois, Apellis CEO (Optum via YouTube)

UP­DAT­ED: So­bi bets $250M cash, about $1B in mile­stones for rights to a C3 ther­a­py be­ing pushed through 5 piv­otal tri­als

A couple years after licensing Novimmune’s emapalumab and turning around a quick FDA OK, Stockholm-based Sobi is betting up to $1.2 billion for rights to another rare disease drug.

The company is shelling out $250 million upfront and adding up to $915 million in milestones for rights to develop and commercialize Apellis Pharmaceuticals’ drug pegcetacoplan outside the US. Together, the companies will see the systemic C3 therapy through five registrational trials in hematology, nephrology and neurology.

Christian Rommel (via Roche)

Bay­er fol­lows R&D deal spree by raid­ing Roche's can­cer group for its new re­search chief

The day after Bayer signed off on a $4 billion deal designed to put the company among the leaders in gene therapy development, the pharma giant has recruited a new chief for its R&D division. And they opted for an expert in the cancer field.

Christian Rommel, Roche’s head of discovery and early-stage oncology development, has been tapped to take over the job. Joerg Moeller, who got the top research post after early and late-stage development roles were combined 2 years ago, is hitting the exit “to pursue other career opportunities.”

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Ul­tragenyx slams the brakes on rare dis­ease study af­ter all 5 pa­tients are hit with a se­ri­ous set­back

Ultragenyx $RARE and its partners at GeneTx have been forced to halt early-stage study for a drug to treat rare cases of Angelman syndrome after all 5 of the patients being treated for the first time experienced a severe adverse event at the highest dose.

The patients in the study experienced “lower extremity weakness believed to be related to local inflammation due to GTX-102,” triggering a red light on dosing and enrollment.

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Albert Bourla, AP

UP­DAT­ED: Where's the Pfiz­er ef­fi­ca­cy read­out? CEO Bourla says 'soon,' but you're go­ing to have to wait for it

Pfizer CEO Albert Bourla had promised repeatedly that the pharma giant would know if its leading Covid-19 vaccine is effective by the end of this month — now just a few days away.

Instead, the company reported early Tuesday that it has yet to conduct any interim efficacy analyses. And it won’t now until sometime next month.

The news was included in a slide for their Q3 report.

In the morning Q3 call with analysts, Bourla says that they expect efficacy data “soon,” but noted that they wouldn’t be able to say anything until all the administrative work was done on the interim, which would take about a week. And he added that Pfizer isn’t going to say anything else about that hot topic until they have the data in hand.

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Charles Baum, Mirati CEO

UP­DAT­ED: Mi­rati plots a march to the FDA for its KRAS G12C drug, breath­ing down Am­gen’s neck with bet­ter da­ta

Mirati Therapeutics $MRTX took another closely-watched step toward a now clearly defined goal to file for an approval for its KRAS G12C cancer drug adagrasib (MRTX849), scoring a higher response rate than the last readout from the class-leading rival at Amgen but still leaving open a raft of important questions about its future.

Following a snapshot of the first handful of responses, where the drug scored a tumor response in 3 of 5 patients with non-small cell lung cancer, the response rate has now slid to 45% among a pooled group of 51 early-stage and Phase II patients, 43% — 6 of 14 — when looking solely at the Phase I/Ib. Those 14 patients had a median treatment duration of 8.2 months, with half still on therapy and 5 of 6 responders still in response.

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Yung Chyung, Scholar Rock CMO (Business Wire)

A dark horse en­trant in­to the spinal mus­cu­lar at­ro­phy field dou­bles its val­ue on some PhII da­ta

The last four years have seen a sudden explosion in treatments for spinal muscular atrophy, a neurodegenerative condition that once led patients — often young ones — with a grim prognosis and no options. The prognosis still isn’t rosy, but now there are three FDA-approved options, enough to make the choice of one difficult.

Now a fourth potential option has entered the mix. Today, Scholar Rock announced the results from a proof-of-concept testing their SMA drug by itself and in combination with Ionis’ Spinraza, showing that all patient cohorts improved on standard scales used for measuring motor function in people with SMA.