Med­i­cines Co., Al­ny­lam pre­pare to throw the dice on a late-stage ef­fort to leapfrog PC­SK9 gi­ants

While Am­gen and its ri­vals at Re­gen­eron/Sanofi strug­gle to es­tab­lish a mar­ket for their pi­o­neer­ing PC­SK9 ther­a­pies — while grap­pling over a nasty law­suit over the IP — Al­ny­lam $AL­NY and The Med­i­cines Com­pa­ny $MD­CO are hatch­ing plans for a come-from-be­hind at­tempt to leapfrog the squab­bling fron­trun­ners.

Clive Mean­well, The Med­i­cines Com­pa­ny

This morn­ing the com­pa­nies an­nounced that the FDA has signed off on their Phase III pro­gram, which will en­roll a to­tal of 3,000 sub­jects with ath­er­o­scle­rot­ic car­dio­vas­cu­lar dis­ease and fa­mil­ial hy­per­c­ho­les­terolemia to test the abil­i­ty of in­clisir­an to slash LDL over 18 months. And they aren’t wait­ing for the re­sults to be­gin their car­dio out­comes study.

Re­searchers plan to sweep up 14,000 pa­tients for the out­comes study, look­ing to see if this new drug can im­press pay­ers with da­ta on a com­pos­ite of coro­nary heart dis­ease death, non-fa­tal my­ocar­dial in­farc­tion and fa­tal and non-fa­tal is­chemic stroke.

That’s a tricky propo­si­tion, though.

Am­gen hit marks for sev­er­al out­comes with their car­dio fol­low-up tri­al ear­li­er this year, but was slapped with a back­lash of skep­ti­cism from an­a­lysts and pay­ers who want­ed to see bet­ter da­ta to jus­ti­fy the price of the drug — par­tic­u­lar­ly on re­duc­ing mor­tal­i­ty. Am­gen’s mon­ey-back guar­an­tee for pa­tients on their drug who go on to ex­pe­ri­ence a heart at­tack failed to win over the doubt­ful, who were quick to do the math on costs and sav­ings.

Learn­ing from the pi­o­neers, The Med­i­cines Com­pa­ny and Al­ny­lam — which de­vel­oped the RNA drug — want to nail down the right da­ta and then an­gle in with a drug that may on­ly have to be giv­en two or three times each year. Af­ter that, the whole glob­al bat­tle for mar­ket share could get down to price.

Last fall re­searchers in the Phase II study re­port­ed that a sin­gle 300 mg in­jec­tion of their RNAi drug — now dubbed in­clisir­an — reg­is­tered a mean 43% re­duc­tion in bad LDL cho­les­terol af­ter 180 days, trig­ger­ing some ex­cit­ed buzz among the an­a­lysts fol­low­ing this pro­gram.

Not­ed Med­i­cines Co. CEO Clive Mean­well:

Al­though not re­quired for the NDA, we will al­so per­form an ag­gres­sive car­dio­vas­cu­lar out­comes tri­al in high-risk sub­jects with AS­CVD and/or risk-equiv­a­lents, such as di­a­betes. We be­lieve that pos­i­tive out­comes da­ta, with pri­ma­ry out­come clin­i­cal ef­fects greater than those re­port­ed for an­ti-PC­SK9 mon­o­clon­al an­ti­bod­ies, will dri­ve a high lev­el of com­pet­i­tive­ness in the world­wide mar­ket – which we ex­pect to be­come very large.

Sanofi and Re­gen­eron, mean­while, ex­pect to have their car­dio da­ta avail­able lat­er this year, hop­ing to trump Am­gen as the com­pa­nies fight it out on the patent front.

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.