To be cost-ef­fec­tive, Bio­gen should slash Spin­raza price, and No­var­tis can­not jus­ti­fy a $4M-$5M price tag for Zol­gens­ma — ICER

As treat­ments for rare dis­eases and gene ther­a­pies make strides on the US mar­ket, the is­sue of pric­ing is once again cen­ter stage. Bio­gen should cut the price of its spinal mus­cu­lar at­ro­phy (SMA) treat­ment Spin­raza and the price for No­var­tis’ one-shot Zol­gens­ma — which is ex­pect­ed to win FDA ap­proval by May — is jus­ti­fi­able up to $1.5 mil­lion, Boston-based ICER con­clud­ed on Wednes­day.

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 be­com­ing in­flu­en­tial with pay­ers.

Spin­raza was ap­proved by the FDA amidst much fan­fare in 2016 as the first and on­ly dis­ease-mod­i­fy­ing treat­ment for SMA, a rare and but lead­ing ge­net­ic cause of in­fant deaths. But the price tag of $750,000 for the first year of ther­a­py (and a $375,000 there­after) sparked heavy crit­i­cism, even though many pay­ers even­tu­al­ly agreed to re­im­burse the treat­ment. It gen­er­at­ed $1.7 bil­lion in 2018 sales for Bio­gen.

No­var­tis’ Zol­gens­ma is cur­rent­ly un­der FDA re­view and the agency is ex­pect­ed to an­nounce its de­ci­sion in the com­ing months. The Swiss drug­mak­er has sug­gest­ed a price of $4 mil­lion for the gene re­place­ment ther­a­py, which it ac­quired via its $8.7 bil­lion takeover of AveX­is, may be jus­ti­fied.

David Rind

“(T)he cur­rent price of Spin­raza far ex­ceeds com­mon thresh­olds for cost-ef­fec­tive­ness. The price of Zol­gens­ma is not yet known, but there has been pub­lic dis­cus­sion of prices above com­mon­ly ac­cept­ed cost-ef­fec­tive­ness thresh­olds as well. These treat­ments will be cov­ered by US in­sur­ers re­gard­less of the pric­ing, but the rip­ple ef­fect of pric­ing de­ci­sions like these threat­ens the over­all af­ford­abil­i­ty and sus­tain­abil­i­ty of the US health sys­tem,” ICER’s chief med­ical of­fi­cer David Rind said in a state­ment.

In the ini­tial draft ICER rec­om­men­da­tions is­sued late last year, ICER had sug­gest­ed Zol­gens­ma — priced at $2 mil­lion — could be more cost-ef­fec­tive in the long run ver­sus Spin­raza.

On Wednes­day, ICER’s fi­nal re­port sug­gest­ed al­though Spin­raza’s price “should be far low­er than it is, and the price for Zol­gens­ma should be low­er than the hy­po­thet­i­cal $4-5 mil­lion price the man­u­fac­tur­er has sug­gest­ed could be jus­ti­fied.”

ICER con­duct­ed its analy­ses us­ing two mea­sures: 1) QALYs, or qual­i­ty-ad­just­ed life-years, are a mea­sure of the state of health of a per­son or group in which the ben­e­fits — in terms of length of life — are ad­just­ed to re­flect the qual­i­ty of life. Es­sen­tial­ly, one QALY is equal to one year of life in per­fect health. 2) Life years gained (LYG), which ex­press­es the ad­di­tion­al num­ber of years of life that a per­son lives as a re­sult of re­ceiv­ing a treat­ment.

His­tor­i­cal­ly, life ex­pectan­cy in the most com­mon and se­vere form of SMA (type I) is less than two years. Sur­vival de­pends on res­pi­ra­to­ry func­tion, and many in­fants and chil­dren even­tu­al­ly re­quire per­ma­nent ven­ti­la­tion.

Us­ing the QALY mea­sure for cost-ef­fec­tive­ness, Spin­raza’s val­ue was as­sessed in the presymp­to­matic pop­u­la­tion, as there are da­ta sup­port­ing its ef­fec­tive­ness in these types of pa­tients. The drug’s price should be be­tween $72,000-$130,000 for the first year of treat­ment and be­tween $36,000-$65,000 for each suc­ces­sive year. Un­der the LYG bench­mark, Spin­raza would need to be priced be­tween $83,000-$145,000 dur­ing the ini­tial year and $41,000-$72,000 for each suc­ces­sive year, ICER said.

“As the re­port notes, there is a sig­nif­i­cant dif­fer­ence in ro­bust­ness and qual­i­ty of ev­i­dence for Spin­raza as com­pared to Zol­gens­ma. The analy­sis, how­ev­er, fails to ac­count for those dif­fer­ences. Spin­raza is the stan­dard of care in SMA and has ben­e­fit­ted the lives of more than 6,600 peo­ple. In con­trast, Zol­gens­ma is an ex­per­i­men­tal ther­a­py which has re­port­ed re­sults to date for on­ly 15 pa­tients fol­lowed for up to 2.5 years, sev­en of whom are re­port­ed to have sub­se­quent­ly ini­ti­at­ed treat­ment with Spin­raza,” a Bio­gen spokesper­son said in an emailed state­ment.

Mean­while, ICER did not vote on the long-term val­ue of Zol­gens­ma, as its fi­nal price is not yet known. It con­duct­ed its as­sess­ment for the SMA type I pop­u­la­tion as this is the on­ly pop­u­la­tion in which it has been clin­i­cal­ly eval­u­at­ed. Us­ing QALY, the ther­a­py’s price for type I SMA would need to be be­tween $310,000-$900,000 per shot, while un­der the LYG bench­mark it would need to be be­tween $710,000-$1.5 mil­lion per treat­ment, ICER con­clud­ed.

“The val­ue mea­sures and thresh­olds em­ployed by ICER in this re­port are de­signed around the sta­tus quo of chron­ic care man­age­ment and can­not pos­si­bly cap­ture the full ben­e­fits of dis­ease-mod­i­fy­ing treat­ments de­liv­ered as a one-time ad­min­is­tra­tion,” a No­var­tis spokesper­son said in an emailed state­ment.

ICER al­so rec­om­mend­ed pay­ers en­dorsed the con­cept of val­ue-based con­tracts, a sys­tem in which a re­im­burse­ment for a sub­stan­tial por­tion of the treat­ment is not passed on to the drug­mak­er, should pa­tients not re­ceive ad­e­quate clin­i­cal ben­e­fit.

Im­age: Shut­ter­stock

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.

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)

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

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.

Endpoints News writer Nicole DeFeudis has posted a snapshot of all the companies, universities and hospital-based groups now racing through the clinic, ranking them according to their place in the pipeline as well as the latest remarks available on timelines. And we’ll keep this lineup updated right through the end of the year, as the checkered flags start to fall, possibly as early as October.

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Bayer's Marianne De Backer with Endpoints founder John Carroll, Endpoints@JPM20 (Jeff Rumans for Endpoints News)

UP­DAT­ED: Hunt­ing a block­buster, Bay­er forges an $875M-plus M&A deal to ac­quire women’s health biotech

Bayer has dropped $425 million in cash on its latest women’s health bet, bringing a UK biotech and its non-hormonal menopause treatment into the fold.

KaNDy Therapeutics had its roots in GlaxoSmithKline, which spun out several neuroscience drugs into NeRRe Therapeutics back in 2012. Five years later the team created a new biotech to focus solely on NT-814 — which they considered “one of the few true innovations in women’s health in more than two decades.”

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Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.

Eisai moves to 200 Metro Blvd. by late 2021 (ON3)

Ei­sai is cre­at­ing a new US cor­po­rate, R&D HQ in Roche’s old Nut­ley, NJ cam­pus

Eight years after Roche pulled up stakes from Nutley, NJ in a major R&D reorganization, Japan’s Eisai is moving its US corporate and research hub into their old campus.

Now the ON3 property, Eisai — a longtime Biogen partner focused on neurodegenerative disorders like Alzheimer’s — will bring together a staff of up to 1,200 employees. And execs are pitching the move to the New Jersey campus as a cultural game-changer.

DFC CEO Adam Boehler and Kodak CEO Jim Continenza (Kodak)

Covid-19 roundup: Cure­Vac beefs up its uni­corn IPO dreams as bil­lion­aire own­er takes this Covid-19 mR­NA play­er on a forced march to Nas­daq; Ko­dak's $765M deal is put on hold

When CureVac initially jotted down $100 million for its IPO raise a couple of weeks ago, it seemed small. The German mRNA player, after all, had jumped into a Covid-19 race that swelled the sails of Moderna and BioNTech by tens of billions. And after raising $640 million in a slate of deals, $100 million in a hot market like this seemed like a pittance in the bigger scheme of things.

Today, we got a look at a figure that probably comes closer to the game-changing number the top execs probably have in mind. Selling 15.3 million shares at the high end of their $14 to $16 range would net a $243 million bounty. Majority owner Dietmar Hopp is putting in another €100 million, bringing the total to around $350 million. And what are the chances they want to do even better than that?

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Ugur Sahin, BioNTech CEO

Covid-19 roundup: Pfiz­er-backed BioN­Tech plans to seek FDA OK for a new vac­cine 'as ear­ly as' Oc­to­ber — ahead of the elec­tion

BioNTech execs say they’re on track to get their late-stage data on a Covid-19 vaccine — partnered with Pfizer — into the hands of regulators as early as October.

In their Q2 release Tuesday morning, the biotech reported that investigators could have late-stage data as early as October, and they won’t be wasting any time in hustling that over to the FDA.

“I am incredibly proud of our team, who has worked tirelessly to initiate our BNT162 Phase 2b/3 trial in record time and put us in a position to seek regulatory review as early as October of this year, if our trials are successful,” said Ugur Sahin, BioNTech’s CEO and co-founder.

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Eric Shaff (Seres)

UP­DAT­ED: Af­ter a 4-year so­journ, strug­gling mi­cro­bio­me pi­o­neer Seres claims a break­out PhI­II come­back. And shares re­spond in fren­zied spike

Almost exactly 4 years ago, Seres Therapeutics $MCRB experienced one of those soul-crunching failures that can raise big questions about a biotech’s future. Out front in their pursuit of a gut punch to C. difficile infection (CDI), the Phase II test was a flat failure, and investors wiped out a billion dollars of equity value that never returned in the years that followed.

Seres, though, pressed ahead, changing out CEOs a year ago — bidding Merck vet Roger Pomerantz farewell from the C suite — and pushing through a Phase III, hoping that amping up the dosage would make the key difference. And this morning, they unveiled a claim that they had aced the Phase III and positioned themselves for a run at a landmark FDA OK.

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Michel Vounatsos, Biogen CEO (via YouTube)

Bio­gen scores a pri­or­i­ty re­view for its Alzheimer's drug ad­u­canum­ab, mov­ing one gi­ant leap for­ward in its con­tro­ver­sial quest

Biogen scored a big win at the FDA today as regulators accepted their application for the controversial Alzheimer’s drug aducanumab and gave it a priority review.

The PDUFA date is March 7, 2021.

Significantly, Biogen says it did not use its priority review voucher to win special treatment at the FDA. The agency handed that out gratis.

That’s the ideal scenario Biogen was looking for as disappointed analysts wondered aloud about the delayed application earlier in the year.

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