Trump needs to prove he's se­ri­ous about sci­ence; PTC's lat­est move is a jaw drop­per

End­points as­sess­es the big bio­phar­ma sto­ries of the week, with a lit­tle added com­men­tary on what they mean for the in­dus­try.

Two big steps back for the Trump ad­min­is­tra­tion

Elias Zer­houni

With Don­ald Trump in the White House, it’s al­ways one step for­ward and two steps back for bio­phar­ma. So last week we had Scott Got­tlieb’s nom for FDA com­mis­sion­er (a big step for­ward) fol­lowed by this week’s news that Trump’s bud­get plans to slash NIH spend­ing by close to $6 bil­lion, while dou­bling in­dus­try’s fees to the FDA (you guessed it.)

Both bud­get items are non­starters. Blast­ing the re­search bud­get at the NIH would gut the world’s most promis­ing dis­cov­ery ac­tiv­i­ties. In an in­ter­view with Forbes’  Matthew Her­p­er, for­mer NIH chief Elias Zer­houni not­ed that if you cut the NIH’s bud­get by 20%, you’d es­sen­tial­ly close the tap on new grants, a knock­out blow to a large group of in­ves­ti­ga­tors which would be es­pe­cial­ly dev­as­tat­ing for young sci­en­tists.

Com­ing as it does af­ter years of flat bud­get­ing, bro­ken on­ly re­cent­ly by an in­crease last year, Trump is play­ing ir­re­spon­si­ble pol­i­tics. The idea of sim­ply dou­bling in­dus­try fees is al­so a stick in the eye to drug de­vel­op­ers.

And the sur­vey says…

Speak­ing of Scott Got­tlieb.

We ran a snap poll over the week­end to get a bet­ter sense of what bio­phar­ma was think­ing in re­gards to his nom­i­na­tion. And the re­sponse was un­equiv­o­cal, with 87% giv­ing the move their ap­proval.

For Trump, it was a savvy choice. Rather than put in some­one who would like to de­stroy the agency, he went with a pick who would set out to make it bet­ter. On Thurs­day night, I met with a group of biotech ex­ecs in the Bay Area, and the same sup­port we saw in the poll per­me­at­ed the room. The FDA is a large agency and while some el­e­ments in it have en­thu­si­as­ti­cal­ly sought to ac­cel­er­ate ap­provals, it’s al­so clear that oth­ers on the in­side could do bet­ter.

Sev­er­al in the room, though, were open­ly skep­ti­cal that Got­tlieb or any new com­mis­sion­er can achieve a quick re­sponse in a fed­er­al agency like the FDA. One sug­gest­ed a time­line of one to two years. But I’ll take that. In the scheme of things, a two-year evo­lu­tion would be bet­ter than try­ing to shove overnight changes.

Steady and in­tel­li­gent will beat rash and crash every time.

PTC’s shame­ful de­ci­sion to buy de­flaza­cort and part­ner with Marathon is a telling move

For the past two years, PTC Ther­a­peu­tics has been stub­born­ly ham­mer­ing on the FDA’s doors in search of its ac­cep­tance of ataluren for re­view as a new ther­a­py for Duchenne mus­cu­lar dy­s­tro­phy. Not un­rea­son­ably the FDA has done what it could to bar en­try, re­fus­ing to ac­cept the in­ad­e­quate ap­pli­ca­tion. This drug, af­ter all, has now failed three straight stud­ies, prov­ing over and over that it’s a dud.

So now they’re tak­ing a back door in­to the mar­ket. And they seem con­tent to make a large num­ber of en­e­mies in the Duchenne com­mu­ni­ty to cap­i­tal­ize on the gam­bit. Their deal to buy Marathon’s rights to de­flaza­cort for $140 mil­lion — plus a stream of roy­al­ties —i demon­strates their con­tempt for pa­tients.

If PTC re­al­ly cared for pa­tients, they’d nev­er mar­ket de­flaza­cort. That would let Duchenne fam­i­lies con­tin­ue to buy the ex­act same old steroid from over­seas sources for a still hefty price of about $1,000 a year — high for a gener­ic. There’s no ques­tion about qual­i­ty or safe­ty. It’s the ex­act same thing.

Any price that PTC puts on de­flaza­cort in the US high­er than $1,000 a year will be too high. They’ll on­ly join the ranks of com­pa­nies brand­ed as price-goug­ing vil­lains. That’s why their stock plunged on Thurs­day. It was a reck­less and stu­pid move by a com­pa­ny prac­ticed in deny­ing re­al­i­ty. But shame­less­ness is PTC’s hall­mark. This is a com­pa­ny that sells a drug in Eu­rope that it has shown re­peat­ed­ly can’t help pa­tients. And it’s look­ing to gain pa­tient sup­port for the same drug in the US.

Reg­u­la­tors, mean­while, seem help­less to pre­vent any of this.

Some se­ri­ous biotech mon­ey is be­ing raised for po­ten­tial­ly break­through sci­ence

Ever since the 2008 fi­nan­cial cri­sis, the biotech ven­ture scene has emerged as a very sta­ble, grow­ing field. A line­up of well known VC groups staffed by ex­pe­ri­enced in­sid­ers have been pump­ing bil­lions of dol­lars in­to star­tups.

Now, all of a sud­den, we’re start­ing to see a few new play­ers jump in­to the game. Over the course of this week I pro­filed three new de­buts — a re­mark­able num­ber for biotech VCs. There was Bio­mat­ics, which has al­ready made its mark in the past eight months, Piv­otal bioVen­ture Part­ners and a new fund from Bill Maris, the ex-Google gu­ru. The Piv­otal mon­ey is com­ing from a Chi­nese group in­ter­est­ed in mak­ing glob­al bets.

What is ex­tra­or­di­nary is that all three of these new play­ers are keen to fund star­tups pur­su­ing break­through sci­ence. And that is help­ing seed an­oth­er big year for some as­ton­ish­ing plays. I il­lus­trat­ed that this week in my sto­ry on eGe­n­e­sis, which is out to make xenograft­ing a re­al­i­ty af­ter decades as a sci­fi sideshow.

It’s a great time to be cov­er­ing this busi­ness. Ob­vi­ous­ly quite a lot of this will nev­er make it in­to med­ical prac­tice. But some ra­zor edge sci­ence is get­ting fi­nanced and we’ll all ben­e­fit from that.

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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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

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.

Arde­lyx bags its first FDA OK for IBS, set­ting up a show­down with Al­ler­gan, Iron­wood

In the first of what it hopes will be a couple of major regulatory milestones for its new drug, Ardelyx has bagged an FDA approval to market Ibsrela (tenapanor) for irritable bowel syndrome.

The drug’s first application will be for IBS with constipation (IBS-C), inhibiting sodium-hydrogen exchanger NHE3 in the GI tract in such a way as to increase bowel movements and decrease abdominal pain. This comes on the heels of two successful Phase III trials.