Se­cre­tive? Mod­er­na de­tails its pipeline strat­e­gy at JPM but at least one crit­ic bites back

Stephane Ban­cel, Mod­er­na CEO

Mod­er­na out­lined its pipeline strat­e­gy at JP Mor­gan this week, fo­cus­ing specif­i­cal­ly on five clin­i­cal-stage pro­grams for new vac­cines as a rel­a­tive­ly low-risk ap­proach to demon­strat­ing that their mes­sen­ger RNA tech­nol­o­gy and the plat­form work. But the kick­back didn’t take long to strike, as a per­sis­tent crit­ic at Stat picked at one trou­bled pro­gram while rais­ing ques­tions about the over­all val­ue of an en­try-lev­el ap­proach fo­cused on a high vol­ume, low-mar­gin busi­ness like vac­cines.

Af­ter rais­ing $1.9 bil­lion from a host of deep-pock­et in­vestors and part­ners, the uni­corn biotech has be­come the sub­ject of in­tense in­ter­est, par­tic­u­lar­ly in Boston biotech cir­cles. And Mod­er­na’s re­luc­tance to de­tail its work in the past has left the biotech with a rep among some re­porters as a se­cre­tive, pun­ish­ing place that has seen off a string of staffers who couldn’t main­tain the fren­zied pace.

Mod­er­na CEO Stephane Ban­cel, though, says that at­tack is un­war­rant­ed. His pre­sen­ta­tion at JP Mor­gan is in­tend­ed to shine a light on what the com­pa­ny’s top prospects are as it preps for its first clin­i­cal da­ta.

Ban­cel’s strat­e­gy is sim­ple: In­stead of fo­cus­ing on a sin­gle lead ef­fort, the pri­vate biotech chose to ramp up a full pipeline of clin­i­cal pro­grams. If one fal­ters, oth­ers can step up to make the case that mR­NA is on its way to de­liv­er­ing a new ap­proach to ther­a­peu­tic de­vel­op­ment with a vast ar­ray of po­ten­tial tar­gets.

“Peo­ple are go­ing to re­al­ize one day that mR­NA is a very good way to make med­i­cines,” Ban­cel says. “We are play­ing a very long game, a 10, 20-year game. We are a very in­tense com­pa­ny. We want to get those drugs for pa­tients.” And that means tack­ling dis­eases like Zi­ka with their tech­nol­o­gy.

Mod­er­na went af­ter two pan­dem­ic flu strains ear­ly, says Ban­cel, so in­ves­ti­ga­tors could ac­cu­rate­ly mea­sure an­ti­body re­spons­es among treat­ment naive pa­tients who had nev­er been ex­posed to the virus. In ad­di­tion to Zi­ka there’s an undis­closed pro­gram part­nered with Mer­ck that’s now in the clin­ic and sev­en pre­clin­i­cal pro­grams, in­clud­ing an­oth­er Mer­ck part­ner­ship on a per­son­al­ized can­cer vac­cine.

One of the com­pa­ny’s most promi­nent crit­ics, Stat’s Dami­an Garde, re­port­ed to­day that safe­ty is­sues hob­bled a ther­a­py for Crigler-Na­j­jar syn­drome — part­nered with Alex­ion — which Ban­cel promised a year ago would get in­to the clin­ic in 2016. That safe­ty is­sue, Garde added, raised fresh ques­tions about Mod­er­na’s high-stakes gam­ble on mR­NA, un­der­min­ing its $5 bil­lion val­u­a­tion it has en­joyed while rais­ing a moun­tain of cash.

Ban­cel tells me that a long-an­tic­i­pat­ed move to cash in on their val­u­a­tion through an IPO, though, won’t be hap­pen­ing this year.

“We will not go pub­lic this year,” he tells me. “This has been dis­cussed by the board. 2017 is not the right time. 2016 and ’17 is a crit­i­cal time for the com­pa­ny to move from dis­cov­ery to a de­vel­op­ment com­pa­ny with scale.”

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.