Roche, Io­n­is her­ald a big win in ear­ly-stage Hunt­ing­ton’s dis­ease tri­al, demon­strate dis­ease mod­i­fy­ing po­ten­tial

Io­n­is $IONS and its part­ners at Roche have un­veiled the bio­mark­er da­ta from a Phase I/II study that re­cent­ly drove the phar­ma gi­ant to fol­low through with a $45 mil­lion pay­ment to op­tion rights to IO­N­IS-HTT Rx (RG6042).

Re­searchers said Thurs­day evening they tracked a mean re­duc­tion of 40% in mu­tant hunt­ingtin pro­tein at the two high­est dos­es of the ther­a­py. Some pa­tients saw up to 60% re­duc­tions. And that, they add, is a first for any Hunt­ing­ton’s drug. It’s that tox­ic pro­tein that lays waste to neu­rons in the brain, caus­ing a se­vere neu­rode­gen­er­a­tive dis­ease marked by de­clin­ing mus­cle co­or­di­na­tion and cog­ni­tive abil­i­ties, which typ­i­cal­ly slow­ly kills pa­tients over a course of 20 years.

If they’re right, the de­vel­op­ers are on track to the first po­ten­tial drug that could change the course of the dis­ease, though some steep hur­dles on safe­ty and ef­fi­ca­cy re­main to be cleared.

The re­search team add the dose de­pen­dent re­sponse demon­strat­ed that mu­tant pro­tein lev­els were still on the de­cline af­ter the last read­out, rais­ing ex­pec­ta­tions that they can do even bet­ter. An open-la­bel ex­ten­sion of the study is un­der­way. Roche has now tak­en con­trol of the drug and is re­spon­si­ble for mount­ing a piv­otal tri­al.

Sarah Tabrizi

Roche first signed on to work with Io­n­is on this drug back in 2013.

Io­n­is has been on an up­swing re­cent­ly, buoyed by roy­al­ties from Spin­raza. The biotech al­so has two drugs un­der re­view, in­clud­ing in­ot­ersen, which is wide­ly ex­pect­ed to get trounced on the mar­ket by Al­ny­lam’s ri­val patisir­an. Still, no one was hold­ing back on the cel­e­bra­tion to­day.

“For near­ly twen­ty years, I have seen many fam­i­lies dev­as­tat­ed from loss­es to this pro­gres­sive neu­rode­gen­er­a­tive dis­ease.  With IO­N­IS-HT­TRx (RG6042), the HD com­mu­ni­ty has new hope for a ther­a­py that can re­duce the cause of HD, and there­fore, may slow the pro­gres­sion and po­ten­tial­ly pre­vent the dis­ease in fu­ture gen­er­a­tions, which is tru­ly ground­break­ing,” said Dr Sarah Tabrizi, pro­fes­sor of clin­i­cal neu­rol­o­gy, di­rec­tor of the Uni­ver­si­ty Col­lege Lon­don’s Hunt­ing­ton’s Dis­ease Cen­tre and the glob­al lead in­ves­ti­ga­tor on the study. “I look for­ward to a longer-term, larg­er study that can es­tab­lish the ben­e­fit of re­duc­ing the tox­ic mu­tant hunt­ingtin pro­tein in peo­ple with HD.”

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.