In the hunt for a megablock­buster, Cel­gene plans FDA fil­ing in wake of PhI­II win for ozan­i­mod in MS

When Cel­gene bought out Re­cep­tos and its au­toim­mune drug ozan­i­mod back in 2015 for $7.2 bil­lion in cash, the big biotech bull­ish­ly fore­cast their drug would be worth $4 bil­lion to $6 bil­lion in peak sales. To­day, the ex­ec­u­tive crew start­ed to make good on that pro­jec­tion with a pos­i­tive read­out on the top-line da­ta from their Phase III mul­ti­ple scle­ro­sis study.

The com­pa­ny says it’s on track to file for an ap­proval of the S1P1 re­cep­tor an­tag­o­nist this year.

In­ves­ti­ga­tors weren’t hand­ing out any hard da­ta to­day. But the com­pa­ny says that ozan­i­mod de­liv­ered “sta­tis­ti­cal­ly sig­nif­i­cant and clin­i­cal­ly mean­ing­ful im­prove­ments com­pared to Avonex for the pri­ma­ry end­point of ARR (the an­nu­al­ized re­lapse rate) and the mea­sured sec­ondary end­points of the num­ber of gadolin­i­um-en­hanc­ing MRI le­sions and the num­ber of new or en­larg­ing T2 MRI le­sions at month 12.”

A sec­ond Phase III study in MS will read out in Q2.

This is a tough mar­ket, though. To get any­where close to $6 bil­lion, ef­fi­ca­cy alone won’t cut it. Cel­gene will al­so have to prove a sub­stan­tial safe­ty ad­van­tage with their drug.

Ge­of­frey Porges, Leerink

Just a few days ago Ge­of­frey Porges at Leerink out­lined the strat­e­gy for ozan­i­mod.

Re­sults from the two Ozan­i­mod phase III tri­als in mul­ti­ple scle­ro­sis are due in the first half of this year, with phase III ul­cer­a­tive col­i­tis da­ta ex­pect­ed in the first half of 2018. CELG al­ways as­sumed ozan­i­mod would get same class la­bel­ing match­ing oth­er S1P1 in­hibitors such as Gilenya, but al­so an­tic­i­pate that the phase III safe­ty da­ta would be in the la­bel so that CELG could dis­cuss the po­ten­tial dif­fer­ences with physi­cians and pay­ers. CELG stat­ed the com­pa­ny needs to see “re­al­ly good ef­fi­ca­cy” and a sim­i­lar lack of car­dio and liv­er tox­i­c­i­ty as shown in the phase II tri­al. About 15% of pa­tients dis­con­tin­ue Gilenya due to these tox­i­c­i­ty is­sues. CELG stat­ed that pric­ing will be low­er in UC when the drug is launched in that in­di­ca­tion, and the com­pa­ny plans to use dis­counts by in­di­ca­tion or oth­er meth­ods to match the ex­ist­ing pric­ing in the MS and IBD in­di­ca­tions (at a list price sim­i­lar to the ex­ist­ing $90,000 Gilenya price).

The com­pa­ny said to­day that the safe­ty pro­file of the drug is in line with their Phase II re­sults. And they sound­ed pumped about the prospects of their in­flam­ma­tion and im­munol­o­gy pipeline.

Scott Smith, Cel­gene

Jef­feries’ Bri­an Abra­hams gave the com­pa­ny a quick thumbs up, not­ing that there’s a long way to go be­fore this drug lives up to ex­pec­ta­tions.

We cur­rent­ly mod­el out-year MS sales of ozan­i­mod of ~$1B, giv­en the po­ten­tial Gilenya gener­i­ciza­tion, in­creas­ing com­pe­ti­tion, and grow­ing pric­ing pres­sure in the MS space. While it is not clear whether CELG would de­tail the agent on their own, the com­pa­ny has re­cent­ly shown more open­ness to us­ing ozan­i­mod as a cor­ner­stone to build out a broad­er neu­rol­o­gy com­mer­cial/de­vel­op­ment plat­form. In IBD, we as­sume peak sales ap­proach­ing ~$3.5B in the out-years, and while key ph.III UC da­ta is not ex­pect­ed un­til 2018, we could see some ini­tial Crohn’s dis­ease da­ta that could help clar­i­fy its pro­file and ex­pand­ed IBD po­ten­tial.

“Peo­ple liv­ing with mul­ti­ple scle­ro­sis need ad­di­tion­al ther­a­pies and we are pleased that oral ozan­i­mod showed mean­ing­ful im­prove­ments across pri­ma­ry and mea­sured sec­ondary end­points in this study,” said Scott Smith, pres­i­dent of Cel­gene In­flam­ma­tion and Im­munol­o­gy. “We look for­ward to da­ta from the con­fir­ma­to­ry phase III RA­DI­ANCE tri­al in the sec­ond quar­ter as we ad­vance to­ward planned reg­u­la­to­ry sub­mis­sions by year-end.”

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.