ICER up­date on Duchenne drugs push­es up price es­ti­mates for Ex­ondys 51, Em­flaza ahead of Ju­ly pan­el re­view

In­creas­ing­ly in­flu­en­tial drug cost-ef­fec­tive­ness watch­dog ICER still doesn’t think that the drugs ap­proved to treat Duchenne mus­cu­lar dy­s­tro­phy have so far proved their val­ue to pa­tients. On Thurs­day, they used sig­nif­i­cant­ly high­er price es­ti­mates for two ther­a­pies in their mod­els, mak­ing them look even less at­trac­tive — and ac­knowl­edged that the es­ti­mates used in their draft re­port in May were in­cor­rect.

ICER’s up­dat­ed re­port on PTC Ther­a­peu­tics’ $PTCT steroid de­flaza­cort and Sarep­ta’s $SRPT Ex­ondys 51 ar­rives ahead of a meet­ing of ex­perts that will dis­cuss the body of ev­i­dence un­der­ly­ing the land­scape of ex­ist­ing and in­com­ing Duchenne mus­cu­lar dy­s­tro­phy (DMD) ther­a­pies. In their analy­sis, ICER has used a price that ex­ceeds $1 mil­lion a year for Ex­ondys 51 and $81,400 for Em­flaza — an old cor­ti­cos­teroid that many fam­i­lies once im­port­ed from over­seas at a cost of about $1,000 a year.

In the Unit­ed States, rough­ly 6,000 young boys suf­fer from the mus­cle wast­ing dis­ease — caused by the ab­sence of dy­s­trophin, a pro­tein that helps keep mus­cle cells in­tact. Symp­toms tend to kick in and pro­gres­sive­ly wors­en be­tween the ages of 3 to 5, typ­i­cal­ly caus­ing the pa­tient to be­come wheel­chair-bound by their ear­ly teens. Even­tu­al­ly, pa­tients suc­cumb to the dis­ease by their 30s. Cor­ti­cos­teroids, which work by di­min­ish­ing in­flam­ma­tion and lim­it­ing the im­mune sys­tem’s ac­tiv­i­ty, are com­mon­ly used to treat DMD.

ICER eval­u­at­ed the ef­fi­ca­cy, safe­ty and cost-ef­fec­tive­ness of four treat­ments in its ev­i­dence re­port. It looked at the steroids de­flaza­cort (sold as Em­flaza) and pred­nisone — as well as the ex­on-skip­ping drugs: the ap­proved eteplirsen (mar­ket­ed as Ex­ondys 51) and the ex­per­i­men­tal golodirsen (both come from Sarep­ta, and each treat­ment is de­signed to treat a dif­fer­ent sub­set of DMD pa­tients). As it con­clud­ed in its draft re­port in May, ICER re­it­er­at­ed that the ev­i­dence sup­port­ing each of the four treat­ments is lack­ing, and their im­pact on pa­tients un­clear.

In the ev­i­dence re­port pub­lished on Thurs­day — which pre­cedes an ad­vi­so­ry pan­el meet­ing that will make its rec­om­men­da­tions to ICER on Ju­ly 25 — used high­er an­nu­al cost es­ti­mates for Ex­ondys 51 and Em­flaza to con­duct var­i­ous cost-ef­fec­tive­ness cal­cu­la­tions.

DMD drug dos­ing is based on weight, which typ­i­cal­ly varies among pa­tients. Akin to its May re­port, ICER used an­nu­al cost es­ti­mates for a 40 kg pa­tient to cal­cu­late cost-ef­fec­tive­ness in its lat­est up­date, putting a fresh spot­light on the ex­ist­ing con­tro­ver­sy sur­round­ing DMD drugs.

In the ev­i­dence re­port, ICER pre­sent­ed this chart:

Source: ICER, Ju­ly 2019

Click on the im­age to see the full-sized ver­sion

In May, when ICER pub­lished its draft re­port, the non-prof­it pre­sent­ed this chart:

Source: ICER, May 2019

Click on the im­age to see the full-sized ver­sion

“We do not know what fac­tors ICER used in their mod­el­ing. We have not raised the price of the drug since launch and are not plan­ning any price in­creas­es,” a Sarep­ta spokesper­son told End­points News.

PTC con­curred. There has been no change in Em­flaza’s pric­ing or dis­counts, a spokesper­son told End­points News.

Lat­er on Fri­day, a spokesper­son for ICER clar­i­fied that the ta­ble in the ear­li­er draft re­port dis­played in­cor­rect an­nu­al costs for both Em­flaza and Ex­ondys 51. ICER’s fi­nal re­port is ex­pect­ed in Au­gust.

So­cial im­age: Sarep­ta, AP Im­ages

Vlad Coric (Biohaven)

In an­oth­er dis­ap­point­ment for in­vestors, FDA slaps down Bio­haven’s re­vised ver­sion of an old ALS drug

Biohaven is at risk of making a habit of disappointing its investors. 

Late Friday the biotech $BHVN reported that the FDA had rejected its application for riluzole, an old drug that they had made over into a sublingual formulation that dissolves under the tongue. According to Biohaven, the FDA had a problem with the active ingredient used in a bioequivalence study back in 2017, which they got from the Canadian drugmaker Apotex.

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.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

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Chas­ing Roche's ag­ing block­buster fran­chise, Am­gen/Al­ler­gan roll out Avastin, Her­ceptin knock­offs at dis­count

Let the long battle for biosimilars in the cancer space begin.

Amgen has launched its Avastin and Herceptin copycats — licensed from the predecessors of Allergan — almost two years after the FDA had stamped its approval on Mvasi (bevacizumab-awwb) and three months after the Kanjinti OK (trastuzumab-anns). While the biotech had been fielding biosimilars in Europe, this marks their first foray in the US — and the first oncology biosimilars in the country.

Seer adds ex-FDA chief Mark Mc­Clel­lan to the board; Her­cules Cap­i­tal makes it of­fi­cial for new CEO Scott Bluestein

→ On the same day it announced a $17.5 million Series C, life sciences and health data company Seer unveiled that it had lured former FDA commissioner and ex-CMS administrator Mark McClellan on to its board. “Mark’s deep understanding of the health care ecosystem and visionary insights on policy reform will be crucial in informing our thinking as we work to bring our liquid biopsy and life sciences products to market,” said Seer chief and founder Omid Farokhzad in a statement.

Daniel O'Day

No­var­tis hands off 3 pre­clin­i­cal pro­grams to the an­tivi­ral R&D mas­ters at Gilead

Gilead CEO Daniel O’Day’s new task hunting up a CSO for the company isn’t stopping the industry’s dominant antiviral player from doing pipeline deals.

The big biotech today snapped up 3 preclinical antiviral programs from pharma giant Novartis, with drugs promising to treat human rhinovirus, influenza and herpes viruses. We don’t know what the upfront is, but the back end has $291 million in milestones baked in.

Vas Narasimhan, AP Images

On a hot streak, No­var­tis ex­ecs run the odds on their two most im­por­tant PhI­II read­outs. Which is 0.01% more like­ly to suc­ceed?

Novartis CEO Vas Narasimhan is living in the sweet spot right now.

The numbers are running a bit better than expected, the pipeline — which he assembled as development chief — is performing and the stock popped more than 4% on Thursday as the executive team ran through their assessment of Q2 performance.

Year-to-date the stock is up 28%, so the investors will be beaming. Anyone looking for chinks in their armor — and there are plenty giving it a shot — right now focus on payer acceptance of their $2.1 million gene therapy Zolgensma, where it’s early days. And CAR-T continues to underperform, but Novartis doesn’t appear to be suffering from it.

So what could go wrong?

Actually, not much. But Tim Anderson at Wolfe pressed Narasimhan and his development chief John Tsai to pick which of two looming Phase III readouts with blockbuster implication had the better odds of success.

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H1 analy­sis: The high-stakes ta­ble in the biotech deals casi­no is pay­ing out some record-set­ting win­nings

For years the big trend among dealmakers at the major players has been centered on ratcheting down upfront payments in favor of bigger milestones. Better known as biobucks for some. But with the top 15 companies competing for the kind of “transformative” pacts that can whip up some excitement on Wall Street, with some big biotechs like Regeneron now weighing in as well, cash is king at the high stakes table.

We asked Chris Dokomajilar, the head of DealForma, to crunch the numbers for us, looking over the top 20 deals for the past decade and breaking it all down into the top alliances already created in 2019. Gilead has clearly tipped the scales in terms of the coin of the bio-realm, with its record-setting $5 billion upfront to tie up to Galapagos’ entire pipeline.

Dokomajilar notes:

We’re going to need a ‘three comma club’ for the deals with over $1 billion in total upfront cash and equity. The $100 million-plus club is getting crowded at 164 deals in the last decade with new deals being added towards the top of the chart. 2019 already has 14 deals with at least $100 million in upfront cash and equity for a total year-to-date of over $9 billion. That beats last year’s $8 billion and sets a record.

Add upfronts and equity payments and you get $11.5 billion for the year, just shy of last year’s record-setting $11.8 billion.

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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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