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

Bris­tol My­ers is clean­ing up the post-Cel­gene merg­er pipeline, and they’re sweep­ing out an ex­per­i­men­tal check­point in the process

Back during the lead up to the $74 billion buyout of Celgene, the big biotech’s leadership did a little housecleaning with a major pact it had forged with Jounce. Out went the $2.6 billion deal and a collaboration on ICOS and PD-1.

Celgene, though, also added a $530 million deal — $50 million up front — to get the worldwide rights to JTX-8064, a drug that targets the LILRB2 receptor on macrophages.

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GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

Fangliang Zhang, AP Images

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Amid a flurry of splashy pandemic IPOs, a J&J-partnered Chinese biotech has emerged with one of the largest public raises in biotech history.

Legend Biotech, the Nanjing-based CAR-T developer, has raised $424 million on NASDAQ. The biotech had originally filed for a still-hefty $350 million, based on a range of $18-$20, but managed to fetch $23 per share, allowing them to well-eclipse the massive raises from companies like Allogene, Juno, Galapagos, though they’ll still fall a few dollars short of Moderna’s record-setting $600 million raise from 2018.

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As it hap­pened: A bid­ding war for an an­tibi­ot­ic mak­er in a mar­ket that has rav­aged its peers

In a bewildering twist to the long-suffering market for antibiotics — there has actually been a bidding war for an antibiotic company: Tetraphase.

It all started back in March, when the maker of Xerava (an FDA approved therapy for complicated intra-abdominal infections) said it had received an offer from AcelRx for an all-stock deal valued at $14.4 million.

The offer was well-timed. Xerava was approved in 2018, four years after Tetraphase posted its first batch of pivotal trial data, and sales were nowhere near where they needed to be in order for the company to keep its head above water.

Drug man­u­fac­tur­ing gi­ant Lon­za taps Roche/phar­ma ‘rein­ven­tion’ vet as its new CEO

Lonza chairman Albert Baehny took his time headhunting a new CEO for the company, making it absolutely clear he wanted a Big Pharma or biotech CEO with a good long track record in the business for the top spot. In the end, he went with the gold standard, turning to Roche’s ranks to recruit Pierre-Alain Ruffieux for the job.

Ruffieux, a member of the pharma leadership team at Roche, spent close to 5 years at the company. But like a small army of manufacturing execs, he gained much of his experience at the other Big Pharma in Basel, remaining at Novartis for 12 years before expanding his horizons.

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Another Big Pharma is entering the Covid-19 antibody hunt.

AbbVie has announced a collaboration with the Netherlands’ Utrecht University and Erasmus Medical Center and the Chinese-Dutch biotech Harbour Biomed to develop a neutralizing antibody that can treat Covid-19. The antibody, called 47D11, was discovered by AbbVie’s three partners, and AbbVie will support early preclinical work, while preparing for later preclinical and clinical development. Researchers described the antibody in Nature Communications last month.

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

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