Ver­tex CEO to UK Prime Min­is­ter: You’re de­valu­ing lives, pass­ing up a great of­fer and threat­en­ing the coun­try’s biotech in­dus­try

Ver­tex has scored big in the US with its new drugs for cys­tic fi­bro­sis, but the biotech still has to slug it out for every inch of mar­ket ter­ri­to­ry in Eu­rope — where its price for Orkam­bi is of­ten seen as way too high for the val­ue it of­fers. And af­ter get­ting snubbed in the UK last week — again — af­ter a long-run­ning cam­paign to win a re­im­burse­ment deal from the NHS, CEO Jef­frey Lei­den has de­cid­ed to pub­licly chas­tise British Prime Min­is­ter There­sa May for the stand­off.

This lat­est blast oc­curred af­ter UK health of­fi­cials de­cid­ed last week that the price Ver­tex is de­mand­ing for Orkam­bi is “un­sup­port­able,” de­spite a pro-re­im­burse­ment cam­paign backed by thou­sands of pa­tients with the dis­ease.

That de­ci­sion, Lei­den tells May, demon­strates how the gov­ern­ment puts “a low­er val­ue on the life of a CF pa­tient than oth­er coun­tries around the world.” He as­serts that the UK’s de­ci­sion amounts to “shut­ting the door” on a new gen­er­a­tion of pre­ci­sion med­i­cines. And the UK, he adds, is pass­ing up a great of­fer.

We have pro­vid­ed the most in­no­v­a­tive of­fer in the world to the NHS, yet have seen no re­cep­tiv­i­ty from NHS Eng­land. In one of the most pros­per­ous coun­tries in the world, NHS Eng­land’s lev­el of in­ter­est in our of­fer rep­re­sents a lack of com­mit­ment to chil­dren and young peo­ple with this dev­as­tat­ing dis­ease….

For the UK to de­vel­op its sci­ence and in­no­va­tion ecosys­tem post-Brex­it, these pol­i­cy state­ments and plans must trans­late to im­proved pa­tient ac­cess to in­no­v­a­tive ther­a­pies, such as the new gen­er­a­tion of pre­ci­sion med­i­cines which work on dif­fer­ent geno­types of rare dis­eases. This re­quires ap­praisal process­es to keep pace with the sci­ence; cur­rent process­es have sig­nif­i­cant lim­i­ta­tions in how they cap­ture and val­ue the full med­ical, so­ci­etal, eco­nom­ic and in­no­va­tion ben­e­fits of such pre­ci­sion med­i­cines. With­out this, any fu­ture biotech in­vest­ment in the UK is at sig­nif­i­cant risk.

Ver­tex took the gloves off with re­cal­ci­trant Eu­ro­pean sin­gle-pay­er groups long ago. In France, the biotech re­cent­ly goad­ed the lo­cal pa­tient pop­u­la­tion with its de­ci­sion to scrap any plans to re­cruit French pa­tients in­to a study, in light of the gov­ern­ment’s re­fusal to cov­er their drug at what they felt was an ap­pro­pri­ate amount. 

We’ll see if lec­tur­ing the British prime min­is­ter works any bet­ter.

Ver­tex Let­ter to Prime Min­is­ter 6 Ju­ly 2018 by Am­ber on Scribd


Im­age: Jef­frey Lei­den. 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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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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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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Sanofi takes a $260M hit to ex­tri­cate it­self from a dis­as­trous al­liance with Lex­i­con

Sanofi spent $300 million in cash to get into a $1.7 billion alliance with Lexicon on their SGLT1/2 diabetes drug sotagliflozin. And now that the drug has been spurned by the FDA after burning through a program that provided mixed late-stage data and a late shot at a last-place finish, the French pharma giant is forking over another $260 million to get out of the deal.

Sanofi’s unhappiness was already apparent when the company — now under new CEO Paul Hudson — posted a statement back in July that they were dropping the deal. But it wasn’t that simple. 

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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

An early-stage update on Adverum Biotechnologies’ intravitreal gene therapy has triggered investor concern, after patients with wet age-related macular degeneration (AMD) saw their vision deteriorate, despite signs that the treatment is improving retinal anatomy.

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.