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

Hal Barron, GSK

Break­ing the death spi­ral: Hal Bar­ron talks about trans­form­ing the mori­bund R&D cul­ture at GSK in a crit­i­cal year for the late-stage pipeline

Just ahead of GlaxoSmithKline’s Q2 update on Wednesday, science chief Hal Barron is making the rounds to talk up the pharma giant’s late-stage strategy as the top execs continue to woo back a deeply skeptical investor group while pushing through a whole new R&D culture.

And that’s not easy, Barron is quick to note. He told the Financial Times:

I think that culture, to some extent, is as hard, in fact even harder, than doing the science.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.

Michel Vounatsos, Getty Images

UP­DAT­ED: Stay tuned: Bio­gen’s num­bers are great — it’s their wor­ri­some fu­ture that leaves an­a­lysts skit­tish

Biogen came out with an upbeat assessment of their Q2 numbers today, discounting the arrival of a key rival for its blockbuster Spinraza franchise. But the top execs remain grimly determined to not say much anything new about the sore points that have dragged down its stock, including the future of its big investment in Alzheimer’s or how it plans to invest the considerable cash that the big biotech continues to reap.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.

Why wait? Cel­gene re­struc­tures a big Jounce pact — ze­ro­ing in on new I/O path­way with $530M deal and bump­ing ICOS

Celgene’s business team isn’t waiting for the big merger with Bristol-Myers Squibb to go through before syncing its strategy with the new mother ship.

Tuesday evening the big biotech unveiled a $530 million deal — $50 million in upfront cash — to amend their alliance with Jounce Therapeutics $JNCE to gain worldwide rights to JTX-8064, an antibody that targets the LILRB2 receptor on macrophages. Their old, $2.6 billion deal is being scrapped, leaving Jounce with a pipeline that includes the lead drug, the ICOS-targeting vopratelimab.

PACT Phar­ma says it's per­fect­ed the tech to se­lect neoanti­gens for per­son­al­ized ther­a­py — now on­to the clin­ic

At PACT Pharma, the lofty goal to unleash a “tsunami” of T cells personalized for each patient has hinged on the ability to correctly identify the neoantigens that form something of a fingerprint for each tumor, and extract the small group of T cells primed to attack the cancer. It still has a long way to go testing a treatment in humans, but the biotech says it has nailed that highly technical piece of the process.

UP­DAT­ED: My­ovan­t's uter­ine fi­broid drug looks com­pet­i­tive in PhI­II — but can they van­quish mighty Ab­b­Vie?

Vivek Ramaswamy’s Myovant $MYOV has closely matched its positive first round of Phase III data for their uterine fibroid drug relugolix, setting up a head-to-head rivalry with pharma giant AbbVie as the little biotech steers to the market with a planned filing in Q4.

Here’s how Myovant plans to prevail over the AbbVie $ABBV empire.

In the study, 71.2% of women receiving once-daily relugolix combination therapy achieved the clinical response they were looking for, compared to only 14.7% in the control arm. The data comfortably reflected the same outcomes in the first Phase III — 73.4% of women receiving once-daily oral relugolix combination therapy achieved the responder criteria compared with 18.9% of women receiving placebo — which will reassure regulators that they are getting the carefully randomized data that qualifies for the FDA’s gold standard for success.

Lit­tle Mar­i­nus sees its shares eclipsed as the Sage ri­val fails to com­pare on PPD in PhII

The executive team at Sage $SAGE have skirted another potential pitfall on its way to racking up a big future for its depression drug Zulresso.

Little Marinus Pharmaceuticals $MRNS had sought to challenge the Sage drug with an IV formulation — followed by an oral version — of ganaxolone for postpartum depression. But researchers say their Phase II study failed to positively differentiate itself from a placebo at 28 days — leaving them to hold up “clinically meaningful” data within the first day of administration compared to the control arm.

Roche cuts loose Tam­i­flu OTC rights, hand­ing Sanofi the keys as the phar­ma gi­ant dou­bles down on Xofluza

Roche set out to make a better flu medicine than Tamiflu as that franchise was headed to a generic showdown. Now they’ll see just how well Xofluza stacks up against the mainstay drug after handing off over-the-counter rights in the US to Sanofi.

Sanofi $SNY says it will now step in to negotiate a deal with the FDA to steer Tamiflu into the OTC market, a role that could well involve new studies to ease passage of the drug out of doctor’s hands and into the consumer end of the market. And the French pharma giant will have first dibs over “selected” OTC markets around the world as they push ahead.

Aca­dia is mak­ing the best of it, but their lat­est PhI­II Nu­plazid study is a bust

Acadia’s late-stage program to widen the commercial prospects for Nuplazid has hit a wall. The biotech reported that their Phase III ENHANCE trial flat failed. And while they $ACAD did their best to cherry pick positive data wherever they can be found, this is a clear setback for the biotech.

With close to 400 patients enrolled, researchers said the drug flunked the primary endpoint as an adjunctive therapy for patients with an inadequate response to antipsychotic therapy. The p-value was an ugly 0.0940 on the Positive and Negative Syndrome Scale, which the company called out as a positive trend.

Their shares slid 12% on the news, good for a $426 million hit on a $3.7 billion market cap at close.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.

Some Big Phar­mas stepped up their game on da­ta trans­paren­cy — but which flunked the test?

The nonprofit Bioethics International has come out with their latest scorecard on data transparency among the big biopharmas in the industry — flagging a few standouts while spotlighting some laggards who are continuing to underperform.

Now in its third year, the nonprofit created a new set of standards with Yale School of Medicine and Stanford Law School to evaluate the track record on trial registration, results reporting, publication and data-sharing practice.