As ne­go­ti­a­tions with Eng­land la­bor on, Scot­land re­jects rou­tine use of Ver­tex's cys­tic fi­bro­sis drugs

As the bat­tle be­tween Ver­tex and Eng­land’s Na­tion­al Health Ser­vice (NHS) con­tin­ues — the Scot­tish NHS has re­ject­ed two of the drug­mak­er’s cys­tic fi­bro­sis med­i­cines.

The US drug­mak­er has been locked in ne­go­ti­a­tion with NICE, which has re­fused to al­low the drug in to Eng­land’s NHS un­til Ver­tex $VRTX of­fers it a dis­count on the treat­ment’s price tag that would com­pel the agency to look fa­vor­ably up­on its cost-ef­fec­tive­ness. On Mon­day, the Scot­tish NHS spurned two of the com­pa­ny’s med­i­cines — Orkam­bi and Symke­vi — for the same rea­son, to the dis­may of thou­sands of pa­tients.

Each drug car­ries a list price of more than £100,000 per pa­tient per year. The UK has more than 10,400 cys­tic fi­bro­sis pa­tients – the largest CF pop­u­la­tion out­side the US, ac­cord­ing to the Cys­tic Fi­bro­sis Foun­da­tion.

Cam­paign­ers in the Unit­ed King­dom have been coax­ing the NHS to cov­er Ver­tex’s CF drugs but to no avail. To the hor­ror of UK cys­tic fi­bro­sis pa­tients, a Ver­tex ex­ec­u­tive dis­closed in March, that last year close to 8,000 packs (each con­tain­ing a 28-day sup­ply) of the com­pa­ny’s treat­ment, Orkam­bi, were de­stroyed af­ter cross­ing their ex­piry date. In a stand­off with UK par­lia­ment in March, Ver­tex chief Jeff Lei­den stood his ground, de­spite be­ing chas­tised by a pletho­ra of MPs for Ver­tex’s pric­ing strat­e­gy, busi­ness mod­el and ethics.

Jeff Lei­den Ver­tex

The cys­tic fi­bro­sis drugs made by Ver­tex are the first treat­ments that ad­dress the un­der­ly­ing ge­net­ic caus­es of cys­tic fi­bro­sis, which is char­ac­ter­ized by a thick sticky mu­cus in the lungs, di­ges­tive sys­tem and oth­er or­gans that re­duces life ex­pectan­cy. In its sec­ond-quar­ter re­sults — pub­lished in late Ju­ly — Ver­tex’s tri­fec­ta of CF med­i­cines com­bined gen­er­at­ed near­ly $1 bil­lion in sales, up 25% from the pre­ced­ing quar­ter. The FDA is al­so re­view­ing the Boston biotech’s three-drug cock­tail for CF, which is ex­pect­ed to treat 90% of CF pa­tients.

The com­pa­ny is dis­ap­point­ed that Orkam­bi and Symke­vi have not been rec­om­mend­ed for rou­tine use in Scot­land, a Ver­tex spokesper­son told End­points News, adding that since learn­ing about the de­ci­sion, Ver­tex is work­ing with the Scot­ting gov­ern­ment to al­low “broad ac­cess” for all el­i­gi­ble pa­tients — which the drug­mak­er hopes to set­tle in the com­ing weeks.

“Ver­tex will con­tin­ue to pro­vide ac­cess to our med­i­cines via the PACS Tier 2 process — which al­lows clin­i­cians to ap­ply for ac­cess for in­di­vid­ual pa­tients based on clin­i­cal need — and was put in­to place pri­or to Ver­tex’s sub­mis­sion in Scot­land. So far, about 65 pa­tients have been ap­proved to re­ceive these med­i­cines un­der this scheme,” the spokesper­son added.

Apart from its rul­ing on Ver­tex’s med­i­cines, Scot­land’s cost-ef­fec­tive­ness watch­dog — the Scot­tish Med­i­cines Con­sor­tium — has made the de­ci­sion to back Akcea Ther­a­peu­tics’ drug for hered­i­tary transthyretin-me­di­at­ed amy­loi­do­sis, Tegse­di; Ab­b­Vie’s blood can­cer drug, Ven­clyx­to; Ca­mu­rus’ treat­ment for opi­oid de­pen­dence, Bu­vi­dal, in a re­strict­ed ca­pac­i­ty; and Almi­rall’s ther­a­py for plaque pso­ri­a­sis, Ilumetri, for lim­it­ed use.

So­cial im­age: Ver­tex, via com­pa­ny site

UP­DAT­ED: Clay Sie­gall’s $614M wa­ger on tu­ca­tinib pays off with solid­ly pos­i­tive piv­otal da­ta and a date with the FDA

Back at the beginning of 2018, Clay Siegall snagged a cancer drug called tucatinib with a $614 million cash deal to buy Cascadian. It paid off today with a solid set of mid-stage data for HER2 positive breast cancer that will in turn serve as the pivotal win Siegall needs to seek an accelerated approval in the push for a new triplet therapy.

And if all the cards keep falling in its favor, they’ll move from 1 drug on the market to 3 in 2020, which is shaping up as a landmark year as Seattle Genetics prepares for its 23rd anniversary on July 15.

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Med­ical an­i­ma­tion: Mak­ing it eas­i­er for the site and the pa­tient to un­der­stand

Medical animation has in recent years become an increasingly important tool for conveying niche information to a varied audience, particularly to those audiences without expertise in the specialist area. Science programmes today, for example, have moved from the piece-to-camera of the university professor explaining how a complex disease mechanism works, to actually showing the viewer first-hand what it might look like to shrink ourselves down to the size of an ant’s foot, and travel inside the human body to witness these processes in action. Effectively communicating a complex disease pathophysiology, or the novel mechanism of action of a new drug, can be complex. This is especially difficult when the audience domain knowledge is limited or non-existent. Medical animation can help with this communication challenge in several ways.
Improved accessibility to visualisation
Visualisation is a core component of our ability to understand a concept. Ask 10 people to visualise an apple, and each will come up with a slightly different image, some apples smaller than others, some more round, some with bites taken. Acceptable, you say, we can move on to the next part of the story. Now ask 10 people to visualise how HIV’s capsid protein gets arranged into the hexamers and pentamers that form the viral capsid that holds HIV’s genetic material. This request may pose a challenge even to someone with some virology knowledge, and it is that inability to effectively visualise what is going on that holds us back from fully understanding the rest of the story. So how does medical animation help us to overcome this visualisation challenge?

Pfiz­er gets some en­cour­ag­ing PhI­II news on a fran­chise sav­ior, but is a dos­ing ad­van­tage worth the $295M up­front?

Close to 3 years after Opko tried to defend itself as shares tumbled on the news that its long-acting growth hormone had failed to outperform a placebo, the Pfizer partner $PFE is back. And this time they’re pitching Phase III data that demonstrates their drug is non-inferior — or maybe a tad better — than their well-known but fading standard in the field.
The comparator drug here is Genotropin, which earned a marginal $142 million for Pfizer last year — down 9% from the year before. Approved 24 years ago, biosimilars are now in development that Pfizer would like to stay out in front of. The market leader here is Norditropin, a growth hormone from Novo Nordisk which uses the same basic ingredient as Genotropin which the pharma giant sells with a kid-friendly self-injectable pen. That would also present some big competition if the new therapy from Opko/Pfizer makes it to the market.
The new data, says researchers, underscore that a weekly injection of somatrogon performed as well or slightly better than Genotropin (somatropin) in young children with growth hormone deficiency. Investigators tracked height velocity at 10.12 cm/year, edging out the older drug’s 9.78 cm/year. That 0.33 difference may not prove compelling to payers, though, who have been known to overlook dosing advantages in favor of lower costs.
That message may have weighed on the stock reaction this morning, with a 30%-plus hike $OPK giving way to more marginal gains.
Back in late 2016, Opko had to defend itself against a devastating Phase III setback as their initial late-stage trial failed against a sugar pill. Opko later blamed that setback on outliers in the study, though it wasn’t able to expunge the failure.

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As­traZeneca's Farx­i­ga scores FDA nod to cut risk of hos­pi­tal­iza­tion for heart fail­ure in di­a­bet­ics

While the FDA recently spurned an application to allow AstraZeneca’s blockbuster drug Farxiga for type 1 diabetes that cannot be controlled by insulin, citing safety concerns — the US regulator has endorsed the use of the SGLT2 treatment to reduce the risk of hospitalisation for heart failure in patients with type-2 diabetes and established cardiovascular disease or multiple CV risk factors.

IM­brave150: Roche’s reg­u­la­to­ry crew plans a glob­al roll­out of Tecen­triq com­bo for liv­er can­cer as PhI­II scores a hit

Just weeks after Bristol-Myers Squibb defended its failed pivotal study pitting Opdivo against Nexavar in liver cancer, Roche says it’s beat the frontline challenge with a combination of their PD-L1 Tecentriq with Avastin. And now they’re rolling their regulatory teams in the US, Europe and China in search of a new approval — badly needed to boost a trailing franchise effort.
Given their breakthrough and Big Pharma status as well as the use of two approved drugs, FDA approval may well prove to be something of a formality. And the Chinese have been clear that they want new drugs for liver cancer, where lethal disease rates are particularly high.
Researchers at their big biotech sub, Genentech, say that the combo beat Bayer’s Nexavar on both progression-free survival as well as overall survival — the first advance in this field in more than a decade. We won’t get the breakdown in months of life gained, but it’s a big win for Roche, which has lagged far, far behind Keytruda and Opdivo, the dominant PD-1s that have captured the bulk of the checkpoint market so far.
Researchers recruited hepatocellular carcinoma — the most common form of liver cancer — patients for the IMbrave150 study who weren’t eligible for surgery ahead of any systemic treatment of the disease.
Roche has a fairly low bar to beat, with modest survival benefit for Nexavar, approved for this indication 12 years ago. But they also plan to offer a combo therapy that could have significantly less toxicity, offering patients a much easier treatment regimen.
Cowen’s Steven Scala recently sized up the importance of IMbrave150, noting:

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Alex­ion clinch­es aHUS ap­proval for Ul­tomiris as the clock ticks on Soliris con­ver­sion

Alexion has racked up a second approval for Ultomiris, the successor therapy to Soliris, as its mainstay blockbuster therapy faces a patent review process that could drastically shorten its patent exclusivity.

The FDA OK for atypical hemolytic uremic syndrome (aHUS) on Friday was widely expected after Alexion posted a full slate of positive Phase III data in January. But regulators also flagged concerns about serious meningococcal infections, slapping a black box warning on the label and mandating a REMS.

FDA ap­proval lets Foamix set its maid­en ac­ne ther­a­py on course for US mar­ket launch

Months ago, Foamix leaned on its biggest shareholders — Perceptive Advisors and OrbiMed — to financially grease its wheels, ahead of the FDA decision date for its acne therapy. On Friday, that approval came in — and the topical formulation of the antibiotic minocycline is set for a January launch.

The therapy, Amzeeq (formerly known as FMX101), was approved to treat inflammatory lesions of non-nodular moderate-to-severe acne vulgaris in patients aged 9 and older.

Hal Barron, GSK's president of R&D and CSO, speaks to Endpoints News founder and editor John Carroll in London at Endpoints' #UKBIO19 summit on October 8, 2019

[Video] Cel­e­brat­ing tri­al fail­ures, chang­ing the cul­ture and al­ly­ing with Cal­i­for­nia dream­ers: R&D chief Hal Bar­ron talks about a new era at GSK

Last week I had a chance to sit down with Hal Barron at Endpoints’ #UKBIO19 summit to discuss his views on R&D at GSK, a topic that has been central to his life since he took the top research post close to 2 years ago. During the conversation, Barron talked about changing the culture at GSK, a move that involves several new approaches — one of which involves celebrating their setbacks as they shift resources to the most promising programs in the pipeline. Barron also discussed his new alliances in the Bay Area — including his collaboration pact with Lyell, which we covered here — frankly assesses the pluses and minuses of the UK drug development scene, and talks about his plans for making GSK a much more effective drug developer.

This is one discussion you won’t want to miss. Insider and Enterprise subscribers can log-in to watch the video.

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Christine Bunt, Robert Langer. Verseau

Armed with Langer tech and $50M, Verseau hails new check­point drugs un­leash­ing macrophages against can­cer

The rising popularity of CD47 has propelled the “don’t-eat-me” signal to household name status in the immuno-oncology world: By blocking that protein, the theory goes, one can stop cancer cells from fooling macrophages. But just as PD-(L)1 merely represents the most fruitful of all checkpoints regulating T cells, Verseau Therapeutics is convinced that CD47 is one of many regulators one can modulate to stir up or tame the immune system.