Ver­tex deal for Scot­land — no deal for Eng­land

Cys­tic fi­bro­sis (CF) drug mak­er Ver­tex Phar­ma­ceu­ti­cals — which is still locked in ne­go­ti­a­tion with NHS Eng­land to en­dorse the use of its med­i­cines — has suc­cess­ful­ly ne­go­ti­at­ed a deal with Scot­tish au­thor­i­ties.

A month ago, the Scot­tish Med­i­cines Con­sor­tium spurned two of the com­pa­ny’s med­i­cines — Orkam­bi and Symke­vi — cit­ing un­cer­tain­ty over their long-term ef­fi­ca­cy in re­la­tion to their cost.

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.

But on Thurs­day, Ver­tex an­nounced a deal had been reached with Scot­land. The price ne­go­ti­at­ed for the treat­ments is con­fi­den­tial, but as part of the five-year agree­ment Ver­tex will col­lect re­al-world ev­i­dence on the drugs that sup­port any fu­ture sub­mis­sions, the com­pa­ny said, adding that about 400 out of the 900 CF pa­tients in Scot­land car­ry the mu­ta­tions ad­dress­able by Orkam­bi or Symke­vi.

David Rams­den Cys­tic Fi­bro­sis Trust

“We cel­e­brate the news in Scot­land to­day, but our cam­paign must con­tin­ue to fo­cus on Ver­tex and all par­ties in Eng­land, Wales and North­ern Ire­land. Those in need of the drugs have al­ready wait­ed too long and we must en­sure that thou­sands more peo­ple are not sub­ject­ed to a post­code lot­tery. Scot­land’s suc­cess must now be repli­cat­ed across the UK with­out fur­ther dam­ag­ing de­lay,” Cys­tic Fi­bro­sis Trust chief David Rams­den said in a state­ment.

The cys­tic fi­bro­sis drugs made by Ver­tex $VRTX 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.

Jeff Lei­den Ver­tex

Ver­tex has been hag­gling with UK’s cost-ef­fec­tive­ness watch­dog NICE, which has re­fused to al­low the drug in­to Eng­land’s NHS un­til Ver­tex of­fers it a dis­count on the treat­ment’s price tag that would com­pel the agency to look fa­vor­ably up­on its cost-ef­fec­tive­ness. Ear­li­er this year, NHS Eng­land of­fered Ver­tex £500 mil­lion over five years and £l bil­lion over the next 10 years to ac­cess the com­pa­ny’s med­i­cines — but the US drug­mak­er re­ject­ed the of­fer. Ne­go­ti­a­tions are still on­go­ing.

To the hor­ror of UK cys­tic fi­bro­sis pa­tients, a Ver­tex ex­ec­u­tive dis­closed 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.

So­cial im­age: Ver­tex

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Deborah Dunsire. Lundbeck

Deb­o­rah Dun­sire is pay­ing $2B for a chance to leap di­rect­ly in­to a block­buster show­down with a few of the world's biggest phar­ma gi­ants

A year after taking the reins as CEO of Lundbeck, Deborah Dunsire is making a bold bid to beef up the Danish biotech’s portfolio of drugs in what will likely be a direct leap into an intense rivalry with a group of giants now carving up a growing market for new migraine drugs.

Bright and early European time the company announced that it will pay up to about $2 billion to buy Alder, a little biotech that is far along the path in developing a quarterly IV formulation for a CGRP drug aimed at cutting back the number of crippling migraines patients experience each month.

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Lisa M. DeAngelis, MSKCC

MSK picks brain can­cer ex­pert Lisa DeAn­ge­lis as its next CMO — fol­low­ing José Basel­ga’s con­tro­ver­sial ex­it

It’s official. Memorial Sloan Kettering has picked a brain cancer expert as its new physician-in-chief and CMO, replacing José Baselga, who left under a cloud after being singled out by The New York Times and ProPublica for failing to properly air his lucrative industry ties.

His replacement, who now will be in charge of MSK’s cutting-edge research work as well as the cancer care delivered by hundreds of practitioners, is Lisa M. DeAngelis. DeAngelis had been chair of the neurology department and co-founder of MSK’s brain tumor center and was moved in to the acting CMO role in the wake of Baselga’s departure.

Penn team adapts CAR-T tech, reengi­neer­ing mouse cells to treat car­diac fi­bro­sis

After establishing itself as one of the pioneer research centers in the world for CAR-T cancer therapies, creating new attack vehicles to eradicate cancer cells, a team at Penn Medicine has begun the tricky transition of using the basic technology for heart repair work.

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Tal Zaks. Moderna

The mR­NA uni­corn Mod­er­na has more ear­ly-stage hu­man da­ta it wants to show off — reach­ing new peaks in prov­ing the po­ten­tial

The whole messenger RNA field has attracted billions of dollars in public and private investor cash gambled on the prospect of getting in on the ground floor. And this morning Boston-based Moderna, one of the leaders in the field, wants to show off a few more of the cards it has to play to prove to you that they’re really in the game.

The whole hand, of course, has yet to be dealt. And there’s no telling who gets to walk with a share of the pot. But any cards on display at this point — especially after being accused of keeping its deck under lock and key — will attract plenty of attention from some very wary, and wired, observers.

“In terms of the complexity and unmet need,” says Tal Zaks, the chief medical officer, “this is peak for what we’ve accomplished.”

Moderna has two Phase I studies it wants to talk about now.

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It's not per­fect, but it's a good start: FDA pan­elists large­ly en­dorse Aim­mune's peanut al­ler­gy ther­a­py

Two days after a fairly benign review from FDA staff, an independent panel of experts largely endorsed the efficacy and safety of Aimmune’s peanut allergy therapy, laying the groundwork for approval with a risk evaluation and mitigation strategy (REMS).

Traditionally, peanut allergies are managed by avoidance, but the threat of accidental exposure cannot be nullified. Some allergists have devised a way to dose patients off-label with peanut protein derived from supermarket products to wean them off their allergies. But the idea behind Aimmune’s product was to standardize the peanut protein, and track the process of desensitization — so when accidental exposure in the real world invariably occurs, patients are less likely to experience a life-threatening allergic reaction.

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Rit­ter bombs fi­nal PhI­II for sole lac­tose in­tol­er­ance drug — shares plum­met

More than two years ago Ritter Pharmaceuticals managed to find enough silver lining in its Phase IIb/III study — after missing the top-line mark — to propel its lactose intolerance toward a confirmatory trial. But as it turned out, the enthusiasm only set the biotech and its investors up to be sorely disappointed.

This time around there’s little left to salvage. Not only did RP-G28 fail to beat placebo in reducing lactose intolerance symptoms, patients in the treatment group actually averaged a smaller improvement. On a composite score measuring symptoms like abdominal pain, cramping, bloating and gas, patients given the drug had a mean reduction of 3.159 while the placebo cohort saw a 3.420 drop on average (one-sided p-value = 0.0106).

Ear­ly snap­shot of Ad­verum's eye gene ther­a­py sparks con­cern about vi­sion loss

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Adverum, on Wednesday, unveiled 24-week data from the OPTIC trial of its experimental therapy, ADVM-022, in six patients who have been administered with one dose of the therapy. On average, patients in the trial had severe disease with an average of 6.2 anti-VEGF injections in the eight months prior to screening and an average annualized injection frequency of 9.3 injections.

Alex Ar­faei trades his an­a­lyst's post for a new role as biotech VC; Sanofi vet heads to Vi­for

Too often, Alex Arfaei arrived too late. 

An analyst at BMO Capital Markets, he’d meet with biotech or pharmaceutical heads for their IPO or secondary funding and his brain, trained on a biology degree and six years at Merck and Endo, would spring with questions: Why this biomarker? Why this design? Why not this endpoint? Not that he could do anything about it. These execs were coming for clinical money; their decisions had been made and finalized long ago.