No­var­tis' SMA gene-ther­a­py, if priced at $2M, could be more cost-ef­fec­tive than Bio­gen's Spin­raza — ICER

Bio­gen’s Spin­raza was ap­proved by the FDA amidst much fan­fare in 2016 as the first and on­ly dis­ease-mod­i­fy­ing treat­ment for SMA, a rare and of­ten fa­tal ge­net­ic mus­cu­lar dis­or­der. But the price tag of $750,000 for the first year of ther­a­py (and a low­er price there­after) prompt­ed heavy crit­i­cism, al­though many pay­ers even­tu­al­ly agreed to re­im­burse the treat­ment. How­ev­er, a re­port by the In­sti­tute for Clin­i­cal and Eco­nom­ic Re­view (ICER) on Thurs­day has sug­gest­ed No­var­tis’ ex­per­i­men­tal SMA gene ther­a­py, Zol­gens­ma, could be more cost-ef­fec­tive in the long run ver­sus Spin­raza.

Zol­gens­ma is cur­rent­ly un­der FDA re­view and the agency is ex­pect­ed to an­nounce its de­ci­sion on the drug in May 2019. In its re­view, ICER has as­sumed Zol­gens­ma will car­ry a list price of $2 mil­lion, al­though the Swiss drug­mak­er has sug­gest­ed a price of $4 mil­lion for the cu­ra­tive ther­a­py, which it ac­quired via its $8.7 bil­lion takeover of AveX­is, may be jus­ti­fied.

De­spite the lack of long-term da­ta on ei­ther ther­a­py, the non-prof­it es­ti­mat­ed in­cre­men­tal cost-ef­fec­tive­ness of Spin­raza is $728,000 per QALY in presymp­to­matic SMA pa­tients, while Zol­gens­ma has an in­cre­men­tal cost-ef­fec­tive­ness of $247,000 per QALY in pa­tients with symp­to­matic Type I SMA (on the ba­sis of a place­hold­er price of $2 mil­lion).

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.

“No­var­tis has es­ti­mat­ed that Zol­gens­ma would yield 13.3 QALYs in SMA Type I pa­tients, and could be priced to $4-5 mil­lion based on the re­la­tion­ship of 10-year cost of ap­proved drugs for ul­tra-rare dis­eases and their cor­re­spond­ing in­cre­men­tal QALYs gained. ICER es­ti­mates Zol­gens­ma pro­vides 11.33 QALYs, and us­es a place­hold­er price of $2 mil­lion. While ICER un­der­cut both of No­var­tis’ es­ti­mates, the high cost/QALY of Spin­raza in Type I SMA pa­tients of­fers No­var­tis bar­gain­ing pow­er with pay­ers if the com­pa­ny can ar­gue Spin­raza use could be re­duced or elim­i­nat­ed to off­set to­tal costs,” Leerink an­a­lysts wrote in a note on Fri­day.

Cur­rent­ly avail­able da­ta on Spin­raza and Zol­gens­ma show pro­longed sur­vival and im­proved mo­tor func­tion com­pared with his­tor­i­cal con­trols or place­bo, but “there re­mains con­sid­er­able un­cer­tain­ty in the gen­er­al­iz­abil­i­ty of the re­sults and in the long-term dura­bil­i­ty and tol­er­a­bil­i­ty of treat­ment. In par­tic­u­lar, for both in­ter­ven­tions, the nar­row el­i­gi­bil­i­ty cri­te­ria of tri­als and the lim­it­ed sam­ple size (es­pe­cial­ly for Zol­gens­ma) rais­es con­cerns about gen­er­al­iz­abil­i­ty of re­sults to the wider pop­u­la­tion of pa­tients with SMA,” the re­port added.

Bio­gen un­der­scored this dif­fer­ence in sam­ple size in its re­sponse to the re­port. “The draft ICER re­port is an in­com­plete rep­re­sen­ta­tion of Spin­raza’s val­ue to pa­tients and health care sys­tems. Fur­ther­more, to com­pare a treat­ment that has helped near­ly 6,000 pa­tients world­wide against an in­ves­ti­ga­tion­al treat­ment on da­ta from 15 pa­tients with an ar­ti­fi­cial­ly se­lect­ed price is mis­lead­ing and wrong,” a spokesper­son told End­points News.

The ICER re­port is not fi­nal and has been opened to the pub­lic for com­ment, which will be in­cor­po­rat­ed in­to an ev­i­dence re­port in Feb­ru­ary 2019. This re­port will be sub­ject to fur­ther de­lib­er­a­tion by one of ICER’s in­de­pen­dent ev­i­dence ap­praisal com­mit­tees in ear­ly March, af­ter which a fi­nal re­port will be re­leased by the end of the month.

“If con­firmed in a fi­nal re­port…these find­ings could of­fer sup­port for No­var­tis in pric­ing and ac­cess ne­go­ti­a­tions for Zol­gens­ma ahead of 2019 ap­proval,” Leerink an­a­lysts added.

In re­sponse to the re­port, No­var­tis said that the find­ings had af­firmed the com­pa­ny’s ini­tial as­sess­ment of the val­ue of their prod­uct. “While we have not yet de­ter­mined the price of our in­ves­ti­ga­tion­al prod­uct…once we de­ter­mine a price, we are com­mit­ted to flex­i­bly part­ner­ing with health­care stake­hold­ers to en­sure ac­cess,” a spokesper­son told End­points News. 

Like NICE in the UK, ICER an­a­lyzes the ev­i­dence on the ef­fec­tive­ness and val­ue of drugs and oth­er med­ical ser­vices in the Unit­ed States, how­ev­er un­like NICE, it is not a gov­ern­ment-af­fil­i­at­ed body.

In Au­gust, NICE re­fused to rec­om­mend the use of Spin­raza in the UK — de­spite Bio­gen dis­count­ing its price — on the ba­sis that the long-term im­pact of the drug was un­clear for pa­tients, some of whom die by the age of 2. With­out that da­ta, the price of the drug couldn’t be jus­ti­fied, they said.

Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

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Part club, part guide, part land­lord: Arie Bellde­grun is blue­print­ing a string of be­spoke biotech com­plex­es in glob­al boom­towns — start­ing with Boston

The biotech industry is getting a landlord, unlike anything it’s ever known before.

Inspired by his recent experiences scrounging for space in Boston and the Bay Area, master biotech builder, investor, and global dealmaker Arie Belldegrun has organized a new venture to build a new, 250,000 square foot biopharma building in Boston’s Seaport district — home to Vertex and a number of up-and-coming biotech players.

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On a glob­al romp, Boehringer BD team picks up its third R&D al­liance for Ju­ly — this time fo­cused on IPF with $50M up­front

Boehringer Ingelheim’s BD team is on a global deal spree. The German pharma company just wrapped its third deal in 3 weeks, going back to Korea for its latest pipeline pact — this time focused on idiopathic pulmonary fibrosis.

They’re handing over $50 million to get their hands on BBT-877, an ATX inhibitor from Korea’s Bridge Biotherapeutics that was on display at a science conference in Dallas recently. There’s not a whole lot of data to evaluate the prospects here.

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Servi­er scoots out of an­oth­er col­lab­o­ra­tion with Macro­Gen­ics, writ­ing off their $40M

Servier is walking out on a partnership with MacroGenics $MGNX — for the second time.

After the market closed on Wednesday MacroGenics put out word that Servier is severing a deal — inked close to 7 years ago — to collaborate on the development of flotetuzumab and other Dual-Affinity Re-Targeting (DART) drugs in its pipeline.

MacroGenics CEO Scott Koenig shrugged off the departure of Servier, which paid $20 million to kick off the alliance and $20 million to option flotetuzumab — putting a heavily back-ended $1 billion-plus in additional biobuck money on the table for the anti-CD123/CD3 bispecific and its companion therapies.

Novotech CRO Ex­pands Chi­na Team as Biotech De­mand for Clin­i­cal Tri­als In­creas­es up to 79%

An increase in demand of up to 79% for clinical trials in China has prompted Novotech the Asia-Pacific CRO to rapidly expand the China team, appointing expert local clinical executives to their Shanghai and Hong Kong offices. The company is planning to expand their team by 30% over the next quarter.

Novotech China has seen considerable demand recently which is borne out by research from GlobalData:
A global migration of clinical research is occurring from high-income countries to low and middle-income countries with emerging economies. Over the period 2017 to 2018, for example, the number of clinical trial sites opened by biotech companies in Asia-Pacific increased by 35% compared to 8% in the rest of the world, with growth as high as 79% in China.
Novotech CEO Dr John Moller said China offers the largest population in the world, rapid economic growth, and an increasing willingness by government to invest in research and development.
Novotech’s 23 years of experience working in the region means we are the ideal CRO partner for USA biotechs wanting to tap the research expertise and opportunities that China offers.
There are over 22,000 active investigators in Greater China, with about 5,000 investigators with experience on at least 3 studies (source GlobalData).

Den­mark's Gen­mab hits the jack­pot with $500M+ US IPO as small­er biotechs rake in a com­bined $147M

Danish drugmaker Genmab A/S is off to the races with perhaps one of the biggest biotech public listings in decades, having reaped over $500 million on the Nasdaq, as it positions itself as a bonafide player in antibody-based cancer therapies.

The company, which has long served as J&J’s $JNJ key partner on the blockbuster multiple myeloma therapy Darzalex, has asserted it has been looking to launch its own proprietary product — one it owns at least half of — by 2025.

FDA over­rides ad­comm opin­ions a fifth of the time, study finds — but why?

For drugmakers, FDA advisory panels are often an apprehended barometer of regulators’ final decisions. While the experts’ endorsement or criticism often translate directly to final outcomes, the FDA sometimes stun observers by diverging from recommendations.

A new paper out of Milbank Quarterly put a number on that trend by analyzing 376 voting meetings and subsequent actions from 2008 through 2015, confirming the general impression that regulators tend to agree with the adcomms most of the time — with discordances in only 22% of the cases.

Norbert Bischofberger. Kronos

Backed by some of the biggest names in biotech, Nor­bert Bischof­berg­er gets his megaround for plat­form tech out of MIT

A little over a year ago when I reported on Norbert Bischofberger’s jump from the CSO job at giant Gilead to a tiny upstart called Kronos, I noted that with his connections in biotech finance, that $18 million launch round he was starting off with could just as easily have been $100 million or more.

With his first anniversary now behind him, Bischofberger has that mega-round in the bank.

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