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

UP­DAT­ED: Roche bags 'break­through' an­ti-fi­bro­sis drug in $1.4B biotech buy­out deal

Roche is snapping up a “breakthrough” anti-fibrotic drug in a $1.4 billion buyout.

The pharma giant announced Friday that it is acquiring Promedior, primarily to get its hands on PRM-151, a recombinant form of human pentraxin-2 (PTX-2) protein that has nailed down mid-stage clinical data on idiopathic pulmonary fibrosis and demonstrating its potential for a range of fibrotic conditions.

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Amarin emerges from an ex­pert pan­el re­view with a clear en­dorse­ment for Vas­cepa and high odds of suc­cess when the FDA weighs in for­mal­ly

Several FDA experts who gathered Thursday to consider the landmark approval of Vascepa to reduce cardio events in an at-risk population voiced their unease about various aspects of the efficacy and safety data, or ultimately the population it should be used to treat. But the overwhelming belief that the data pointed to the drug’s benefit and clearly outweighed risks carried the day for Amarin.

The panel voted unanimously (16 to 0) to support the company’s positive data presentation — backing an OK for expanding the label to include reducing cardio risk. The vote points Amarin $AMRN down a short path to a formal decision by the FDA, with the odds heavily in its favor. Chances are the rest of the questions about the future of this drug will be hashed out in the label’s small print.

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No­var­tis spin­out’s first an­ti-ag­ing PhI­II is a flop, so now they’ll turn to Parkin­son’s chal­lenge as shares wilt

Novartis spinout resTORbio is grappling with the collapse of its lead clinical program this morning — an anti-aging R&D failure that will badly damage their rep in the field.

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No­var­tis scores its lat­est FDA OK — this time for a new sick­le cell dis­ease drug picked up in a $665M deal

Novartis’ decision to buy Oklahoma-based biotech Selexys 3 years ago for up to $665 million has paid off with an FDA approval today.

Blessed with the FDA’s breakthrough drug designation for a speedy review, the pharma giant has pinned down an approval for crizanlizumab, a new therapy designed to reduce the frequency of painful incidents of vaso-occlusive crises among sickle cell disease patients 16 or older.

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As­traZeneca gains EU nod for di­a­betes triple; Am­gen and Duke launch re­al-world PC­SK9 ob­ser­va­tion­al study

→ Weeks after winning EU approval to start marketing dapagliflozin as Forxiga, AstraZeneca has racked up another OK for a triplet combo involving the SGLT2 diabetes drug. Named Qtrilmet, the pill combines Forxiga with the DPP-4 inhibitor Onglyza (saxagliptin) and the bedrock drug metformin in a modified-release format. That 3-in-1 approach proved superior in reducing average blood glucose levels to a number of other dual combinations across 5 Phase III trials, including Forxiga plus metformin, Onglyza with metformin, or glimepiride with metformin.

Five drugs, in­clud­ing two No­var­tis ther­a­pies, win EMA en­dorse­ment

As is custom, an EMA panel on Friday issued its weekly recommendations on marketing applications submitted by drug developers. This week, the agency backed the use of five new therapies — including two Novartis drugs — but issued no negative reviews.

Novartis’ S1P drug for relapsing forms of multiple sclerosis (MS) drug, Mayzent (known chemically as siponimod), which was approved by the FDA in March — has been given the nod by the EMA. The Swiss drugmaker already sells its other MS drug, Gilenya, in both regions.

Atom­wise's X-37 spin­out gets $14.5 mil­lion to launch AI dis­cov­ery ef­forts

The folks behind Atomwise’s spinout X-37 like to think in cosmological metaphors, and you can think of their AI drug development model as probes sent into space from a central station. That station just got $14.5 million in Series A funding from DCVC Bio, Alpha Intelligence Capital and Hemi Ventures to back those missions.

X-37 uses Atomwise’s AI platform to identify drug targets and – unlike the parent company, which largely sticks to computers  – bring those into a wet lab and preclinical testing.  In addition to AI professionals, it’s led in by part by drug developers from Velocity Pharmaceutical Development.

Ab­bott Lab­o­ra­to­ries CEO Miles White pass­es ba­ton down to suc­ces­sor; Lon­za CEO Marc Funk hits the ex­it

→ Abbott Laboratories has named a successor to CEO Miles White after he announced that he was stepping down in March after 21 years of service. Robert Ford, the company’s COO and president, will take the helm. Ford is known for his work in the $25 billion merger between St. Jude Medical into Abbott in January 2017. White will remain with the company as executive chairman of the board. 

→ After snapping up Novartis’ Swiss facility, Novartis Center of Excellence, in July, Lonza has announced that their CEO, Marc Funk, is hitting the exit for “personal reasons.” Funk has been the CEO of the company for less than a year — brought onto the company back in March. In the meantime, chairman Albert Baehny will serve as interim CEO. 

UCB adds on more pos­i­tive PhI­II da­ta for IL-17A/17F in­hibitor bimek­izum­ab, clear­ing a path to the FDA

A month after posting positive top-line data from their first Phase III trial of the IL-17A/17F inhibitor bimekizumab, Belgium’s UCB says they’ve added more upbeat results from their second late-stage test in moderate-to-severe plaque psoriasis.

That leaves the company on track for regulatory submissions in the middle of next year, says CMO Iris Loew-Friedrich.
Their drug beat out a placebo on the co-primaries — a 90% improvement in PASI 90 (the Psoriasis Area and Severity Index) and Investigator Global Assessment (IGA) response of clear or almost clear (IGA 0/1) at week 16, compared to placebo. Investigators also boasted of hitting some key secondaries.
UCB is angling to enter an increasingly crowded market space.
In their first of 3 Phase III studies for bimekizumab, researchers touted top-line wins on statistically significant results on clearing plaque psoriasis, including a victory over J&J’s IL-23 contender Stelara on key endpoints. The drug targets both IL-17A and IL-17F, a modification on the IL-17A strategy laid out for Taltz (Eli Lilly) and Cosentyx (Novartis). And the new group also includes J&J’s Tremfya and AbbVie’s Skyrizi.

Social image: UCB