NewLink grabs the AACR spot­light with IDO path­way, Keytru­da com­bo -- shares blitzed

At­ten­dees, speak­ers and awardees dur­ing AACR’s 2017 Open­ing Cer­e­mo­ny © AACR/Todd Buchanan 2017

WASH­ING­TON, DC — Re­searchers at NewLink Ge­net­ics are in the spot­light at AACR this morn­ing with da­ta show­ing that their pair-up of the biotech’s IDO path­way in­hibitor in­dox­i­mod with Mer­ck’s Keytru­da sig­nif­i­cant­ly pushed up re­sponse rates over what the PD-1 check­point alone nor­mal­ly pro­duces for ad­vanced melanoma.

The his­tor­i­cal per­cent­age re­sponse rate for Keytru­da alone in this field is in the low 30s, but for all the 60 evalu­able pa­tients in the study the ORR was 52% — in­clud­ing hard-to-tread oc­u­lar melanoma cas­es. If you leave out the oc­u­lar (eye) pa­tients, the suc­cess ra­tio ris­es to 59%, with a dis­ease con­trol rate of 80%. And the re­searchers say the safe­ty pro­file for the com­bo looked sim­i­lar to Keytru­da alone, which will help the biotech start mak­ing the case that it can add a ther­a­py to a check­point with­out push­ing tox­i­c­i­ty, the way a CT­LA-4 drug like Yer­voy would.

A group of crit­ics on Twit­ter hit the da­ta quick­ly Tues­day morn­ing, though, ques­tion­ing the re­sults and dri­ving the stock down 22% in ear­ly trad­ing.

NewLink $NLNK is un­usu­al in this field. The biotech — which was ham­mered in 2016 with the fail­ure of its pan­cre­at­ic can­cer vac­cine — part­nered with Genen­tech in 2014 on GDC-0919, a spe­cif­ic IDO en­zy­mat­ic in­hibitor. Genen­tech has that pro­gram un­der its wing af­ter pay­ing $150 mil­lion up­front with more than a bil­lion dol­lars promised in mile­stones.

Charles Link

NewLink CEO Chuck Link de­scribes in­dox­i­mod as its whol­ly-owned IDO path­way in­hibitor, sep­a­rate from the en­zy­mat­ic spe­cif­ic group, which in­cludes In­cyte’s lead­ing epaca­do­stat, now head­ed in­to a full slate of late-stage com­bi­na­tions with Keytru­da (Mer­ck) and Op­di­vo (Bris­tol-My­ers).

“In­dox­i­mod is a drug we’re try­ing to ful­ly un­der­stand,” Link tells me. The drug acts on both den­drit­ic cells and T cells, he says, al­low­ing the T cells to repli­cate in fight­ing can­cer. It does not bind with the kind of speci­fici­ty that char­ac­ter­izes the en­zy­mat­ic class.

The next step is to push ahead in­to a reg­is­tra­tion study. The lat­est da­ta comes from a Phase II that does not have a con­trol arm, which will be reme­died in the late-stage tri­al. And Link says he ex­pects the study to get un­der­way lat­er this year.

Link wasn’t ready to pro­vide an es­ti­mate of when the da­ta will read out. That might de­pend on a va­ri­ety of things, he says, in­clud­ing some de­sign el­e­ments that might al­low for an ear­ly cut of the re­sults that could be tak­en to the FDA in search of an ac­cel­er­at­ed ap­proval.

Time will tell.

IDO in­hi­bi­tion came in­to full fo­cus dur­ing the last few days as In­cyte surged on its late-stage de­vel­op­ment plans. Be­hind the lead­ers you’ll find a host of ear­ly-stage ef­forts un­der­way, and Link be­lieves that there may be a fu­ture for com­bin­ing IDO path­way and IDO en­zyme in­hibitors.

What In­cyte, NewLink and all the oth­er play­ers are won­der­ing is if an IDO/PD-1 or IDO/PD-L1 check­point can do as well as a check­point match-up us­ing a CT­LA-4. Those CT­LA-4s are ef­fec­tive, but well known for a harsh tox­i­c­i­ty that makes them hard to tol­er­ate. And that would have ma­jor im­pli­ca­tions not on­ly for Yer­voy’s fu­ture, but al­so com­bos like As­traZeneca’s dur­val­um­ab and treme­li­mum­ab, a CT­LA-4 that oc­cu­pies a cen­tral role in the phar­ma gi­ant’s plans to leap from last place in­to the fore­front, pre­sum­ing they can get a rel­a­tive­ly quick OK as the fifth in a se­ries.

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.