J&J's head-to-head PhI­II da­ta promise drug launch in­to crowd­ed MS mar­ket

J&J an­nounced pos­i­tive da­ta from a head-to-head Phase III tri­al squar­ing their S1P1 MS drug pones­i­mod against Sanofi’s Auba­gio, set­ting up an FDA fil­ing lat­er this year and a pos­si­ble 2020 launch in­to an in­creas­ing­ly crowd­ed space.

A J&J launch will fly against steep head­winds. No­var­tis’s Mayzent (sipon­i­mod), tabbed by CEO Vas Narasimhan as a po­ten­tial block­buster, gained FDA ap­proval in March based off a place­bo study. The same month, Cel­gene re-filed its FDA ap­pli­ca­tion for ozan­i­mod, lever­ag­ing da­ta from a head-to-head tri­al against Avonex. And in Au­gust, TG Ther­a­peu­tics an­nounced pos­i­tive re­sults from its Phase II study of ubli­tux­imab, and No­var­tis an­nounced pos­i­tive re­sults from their oth­er MS drug Arz­er­ra (ofa­tu­mum­ab) in its head-to-head tri­al against Auba­gio.

J&J says it plans on mov­ing for­ward with mar­ket­ing ap­pli­ca­tions in the US and Eu­rope, and de­spite the crowd­ed MS field, it would be the on­ly di­rect ri­val to No­var­tis’s S1P Gilenya and Mayzent. No­var­tis an­nounced to­day da­ta from their Phase III EX­PAND tri­al in­di­cat­ed Mayzent can help pa­tients re­tain mo­bil­i­ty for four years longer on av­er­age.

All of these drugs tar­get re­laps­ing forms of mul­ti­ple scle­ro­sis and will have to com­pete with Roche’s land­mark block­buster Ocre­vus (ocre­lizum­ab). It’ll be a lim­it­ed com­pe­ti­tion, how­ev­er; nei­ther No­var­tis, Cel­gene nor TG has shown that their drugs are al­so ef­fec­tive as pri­ma­ry pro­gres­sive treat­ments. That dual sta­tus is what helped pro­pel Ocre­vus to the top of the mar­ket.

J&J pur­chased pones­i­mod in their $30 bil­lion Acte­lion buy­out close to 3 years ago when they beat out Sanofi, whose man­age­ment had in­sult­ed Acte­lion CEO, Jean-Paul Clozel, with “the tenor and con­tent” of their ne­go­ti­a­tions.

The J&J study’s pri­ma­ry end­point was an­nu­al re­lapse rate (ARR), as it was for No­var­tis and Cel­gene. They found an ARR of just over 20% for pones­i­mod, com­pared with 29% from Auba­gio. The tri­al cov­ered 1,133 par­tic­i­pants over 108 weeks across 162 lo­ca­tion.  Da­ta from No­var­tis’s head-to-head tri­al and TG Ther­a­peu­tics’s Phase II are be­ing pre­sent­ed this week.

The J&J drug al­so fared well in sev­er­al sec­ondary end­points, in­clud­ing fa­tigue and cu­mu­la­tive num­ber of com­bined unique ac­tive le­sions found by MRI.

S1P drugs work by block­ing lym­pho­cyte im­mune cells from cross­ing the blood-brain bar­ri­er and dam­ag­ing myelin, the fat­ty sub­stance that sur­rounds nerve cells and that, in MS pa­tients, is at­tacked by the body’s own im­mune sys­tem. No­var­tis’s Mayzent and the Cel­gene are al­so S1P drugs. Arz­er­ra and the TG Ther­a­peu­tics com­pounds are an­ti-CD20 drugs that tar­get the CD20 re­cep­tor on the lym­pho­cytes while they are cir­cu­lat­ing in the blood and be­fore they cause in­flam­ma­tion in the ner­vous sys­tem. .

S1P drugs are al­so be­ing in­ves­ti­gat­ed as a treat­ment for pso­ri­a­sis.

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 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

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.