Aim­mune's peanut al­ler­gy im­munother­a­py clears Eu­ro­pean PhI­II with fly­ing col­ors, paving way for first US, EU ther­a­peu­tic ap­proval

Days af­ter the FDA fi­nal­ly ac­cept­ed the ap­pli­ca­tion to re­view its peanut al­ler­gy im­munother­a­py, Aim­mune laid out pos­i­tive piv­otal study da­ta from its Eu­ro­pean Phase III study on Mon­day, paving the way for an EMA mar­ket­ing ap­pli­ca­tion by mid-2019.

The drug de­vel­op­er, which has es­sen­tial­ly leapfrogged its arch ri­val DBV Tech­nolo­gies $DB­VT in the Unit­ed States, said its drug — AR101 — helped shore up tol­er­ance in peanut-al­ler­gic sub­jects in a 175-pa­tient tri­al called ARTEMIS. Pa­tients un­der­went ap­prox­i­mate­ly six months of dose es­ca­la­tion and then three months at a dai­ly ther­a­peu­tic dose of AR101 at 300 mg or place­bo, fol­lowed by a place­bo-con­trolled food chal­lenge.

AR101 met the main goal of sig­nif­i­cant­ly im­prov­ing pa­tients’ abil­i­ty to tol­er­ate a 1,000-mg sin­gle dose of peanut pro­tein (p<0.00001) — which cor­re­lates to at least three or four peanuts — ver­sus those who got the place­bo. Over­all, the me­di­an tol­er­at­ed dose of peanut pro­tein for AR101-treat­ed pa­tients im­proved 100-fold, from 10 mg at base­line to 1,000 mg at ex­it, the com­pa­ny said.

Jayson Dal­las

“This lev­el of pro­tec­tion pro­vides am­ple buffer be­yond a typ­i­cal bite of a peanut-con­tain­ing food in the re­al world,” said Aim­mune chief Jayson Dal­las in a state­ment. “AR101 has the po­ten­tial to be­come the first ap­proved ther­a­py for peanut al­ler­gy in both the Unit­ed States and Eu­rope, where up to two per­cent of chil­dren in many coun­tries are af­fect­ed.”

The ARTEMIS find­ings echo the re­sults from the high­est lev­el test­ed in Aim­mune’s land­mark Phase III PAL­ISADE tri­al, which formed an in­te­gral por­tion of Aim­mune’s US ap­pli­ca­tion. PAL­ISADE da­ta showed 50.3% of AR101-treat­ed pa­tients tol­er­at­ed a sin­gle high­est dose of 1,000 mg of peanut pro­tein af­ter ap­prox­i­mate­ly six months of dose es­ca­la­tion fol­lowed by six months at a dai­ly ther­a­peu­tic dose of 300 mg, com­pared to 2.4% of place­bo pa­tients (p<0.00001).

Daniel Adel­man

“The ARTEMIS da­ta demon­strate that most pa­tients ex­ceed­ed what we con­sid­er to be pro­tec­tive lev­els well be­fore a full year of treat­ment…these da­ta build up­on the in­sights gained through­out the en­tire AR101 pro­gram re­gard­ing the de­sen­si­ti­za­tion process and the abil­i­ty of our pa­tients to tol­er­ate rel­a­tive­ly large chal­lenge-dos­es of peanut pro­tein,” Aim­mune’s CMO Daniel Adel­man said.

The FDA is ex­pect­ed to make a de­ci­sion on AR101 by Jan­u­ary 2020, Cal­i­for­nia-based Aim­mune said last week.

DBV $DB­VT re­scind­ed an ap­pli­ca­tion to mar­ket its peanut al­ler­gy patch on De­cem­ber 20 in re­sponse to FDA con­cerns about the state of man­u­fac­tur­ing and qual­i­ty con­trol da­ta sub­mit­ted, and the fol­low­ing day, Aim­mune $AIMT sub­mit­ted its ap­pli­ca­tion for AR101, in ef­fect leapfrog­ging its com­pe­ti­tion for a first-mover shot at cap­tur­ing the so far un­tapped mar­ket, which is ex­pect­ed to grow to $4.5 bil­lion in 2027 glob­al­ly, ac­cord­ing to Glob­al­Da­ta. The US shut­down be­gan on De­cem­ber 22. By mid-Jan­u­ary, Aim­mune said the health reg­u­la­tor had no­ti­fied them that it would not be able to re­view the ap­pli­ca­tion un­til the lapse in ap­pro­pri­a­tions end­ed. Ten days lat­er, on Jan­u­ary 25th, the gov­ern­ment re­opened, and the AR101 ap­pli­ca­tion was back in con­tention.

Aim­mune’s in­ves­ti­ga­tion­al egg al­ler­gy prod­uct — AR201 — is ex­pect­ed to be eval­u­at­ed in a Phase II study mid-2019. The con­di­tion af­fects an es­ti­mat­ed 6 mil­lion peo­ple in the US, Eu­rope, Japan and Chi­na.

Nick Leschly via Getty

UP­DAT­ED: Blue­bird shares sink as an­a­lysts puz­zle out $1.8M stick­er shock and an un­ex­pect­ed de­lay

Blue­bird bio $BLUE has un­veiled its price for the new­ly ap­proved gene ther­a­py Zyn­te­glo (Lenti­Glo­bin), which came as a big sur­prise. And it wasn’t the on­ly un­ex­pect­ed twist in to­day’s sto­ry.

With some an­a­lysts bet­ting on a $900,000 price for the β-tha­lassemia treat­ment in Eu­rope, where reg­u­la­tors pro­vid­ed a con­di­tion­al ear­ly OK, blue­bird CEO Nick Leschly said Fri­day morn­ing that the pa­tients who are suc­cess­ful­ly treat­ed with their drug over 5 years will be charged twice that — $1.8 mil­lion — on the con­ti­nent. That makes this drug the sec­ond most ex­pen­sive ther­a­py on the plan­et, just be­hind No­var­tis’ new­ly ap­proved Zol­gens­ma at $2.1 mil­lion, with an­a­lysts still wait­ing to see what kind of pre­mi­um can be had in the US.


Glob­al Blood Ther­a­peu­tics poised to sub­mit ap­pli­ca­tion for ac­cel­er­at­ed ap­proval, with new piv­otal da­ta on its sick­le cell dis­ease drug

Global Blood Therapeutics is set to submit an application for accelerated approval in the second-half of this year, after unveiling fresh data from a late-stage trial that showed just over half the patients given the highest dose of its experimental sickle cell disease drug experienced a statistically significant improvement in oxygen-wielding hemoglobin, meeting the study's main goal.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,000+ biopharma pros reading Endpoints daily — and it's free.

Gene ther­a­pies seize the top of the list of the most ex­pen­sive drugs on the plan­et — and that trend has just be­gun

Anyone looking for a few simple reasons why the gene therapy field has caught fire with the pharma giants need only look at the new list of the 10 most expensive therapies from GoodRx.

Two recently approved gene therapies sit atop this list, with Novartis’ Zolgensma crowned the king of the priciest drugs at $2.1 million. Right below is Luxturna, the $850,000 pioneer from Spark, which Roche is pushing hard to acquire as it adds a gene therapy group to the global mix.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,000+ biopharma pros reading Endpoints daily — and it's free.

News­mak­ers at #EHA19: Re­gen­eron, Ar­Qule track progress on re­sponse rates

Re­gen­eron’s close­ly-watched bis­pe­cif­ic con­tin­ues to ring up high re­sponse rates

Re­gen­eron’s high-pro­file bis­pe­cif­ic REGN1979 is back in the spot­light at the Eu­ro­pean Hema­tol­ogy As­so­ci­a­tion sci­en­tif­ic con­fab. And while the stel­lar num­bers we saw at ASH have erod­ed some­what as more blood can­cer pa­tients are eval­u­at­ed, the re­sponse rates for this CD3/CD20 drug re­main high.

A to­tal of 13 out of 14 fol­lic­u­lar lym­phomas re­spond­ed to the drug, a 93% ORR, down from 100% at the last read­out. In 10 out of 14, there was a com­plete re­sponse. In dif­fuse large B-cell lym­phoma the re­sponse rate was 57% among pa­tients treat­ed at the 80 mg to 160 mg dose range. They were all com­plete re­spons­es. And 2 of these Cars were for pa­tients who had failed CAR-T ther­a­py.

Search­ing for the next block­buster to fol­low Darza­lex, J&J finds a $150M an­ti-CD38 drug from part­ner Gen­mab

Now that J&J and Genmab have thrust Darzalex onto the regulatory orbit for first-line use in multiple myeloma, the partners are lining up a deal for a next-gen follow-on to the leading CD38 drug.

Janssen — J&J’s biotech unit — has its eyes on HexaBody-CD38, a preclinical compound generated on Genmab’s tech platform designed to make drugs more potent via hexamerization.

Genmab is footing the bill on studies in multiple myeloma and diffuse large B-cell lymphoma; once it completes clinical proof of concept, Janssen has the option to license the drug for a $150 million exercise fee. There’s also $125 million worth of milestones in play.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,000+ biopharma pros reading Endpoints daily — and it's free.

J&J gains an en­thu­si­as­tic en­dorse­ment from Pres­i­dent Don­ald Trump for their big new drug Spra­va­to

Pres­i­dent Don­ald Trump has lit­tle love for Big Phar­ma, but there’s at least one new drug that just hit the mar­ket which he is en­am­ored with.

Trump, ev­i­dent­ly, has been read­ing up on J&J’s new an­ti-de­pres­sion drug, Spra­va­to. And the pres­i­dent — who of­ten likes to break out in­to a full-throat­ed at­tack on greedy drug­mak­ers — ap­par­ent­ly en­thused about the ther­a­py in a meet­ing with of­fi­cials of Vet­er­ans Af­fairs, which has long grap­pled with de­pres­sion among vet­er­ans.

Savara shares are crushed as PhI­II tri­al flunks pri­ma­ry, key sec­on­daries — but they can’t stop be­liev­ing

In­vestors are in no mood to hear biotechs tout the suc­cess of a “key” sec­ondary end­point when the piv­otal Phase III flunks the pri­ma­ry goal. Just ask Savara. 

The Texas biotech $SVRA went look­ing for a sil­ver lin­ing as com­pa­ny ex­ecs blunt­ly con­ced­ed that Mol­gradex, an in­haled for­mu­la­tion of re­com­bi­nant hu­man gran­u­lo­cyte-macrophage colony-stim­u­lat­ing fac­tor (GM-CSF), failed to spur sig­nif­i­cant­ly im­proved treat­ment out­comes for pa­tients with a rare res­pi­ra­to­ry dis­ease called au­toim­mune pul­monary alve­o­lar pro­teinosis, or aPAP.

As an­oth­er an­tibi­otics biotech sinks in­to a cri­sis, warn­ings of a sec­tor ‘col­lapse’

Another antibiotics company is scrambling to survive today, forcing the company’s founding CEO to exit in a reorganization that eliminates its research capabilities as the survivors look to improve on minuscule sales of their newly approved treatment. And the news — on top of an alarming series of failures — spurred at least one figure in the field to warn of a looming collapse of the antimicrobial resistance research field.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,000+ biopharma pros reading Endpoints daily — and it's free.

In a boost to Rit­ux­an fran­chise, Roche nabs quick ap­proval for po­latuzum­ab ve­dotin

Roche’s lat­est an­ti­body-drug con­ju­gate has crossed the FDA fin­ish line, gain­ing an ac­cel­er­at­ed ap­proval a full two months ahead of sched­ule.

Po­livy, or po­latuzum­ab ve­dotin, is a first-in-class drug tar­get­ing CD79b — a pro­tein promi­nent in B-cell non-Hodgkin lym­phoma. It will now be mar­ket­ed for dif­fuse large B-cell lym­phoma as part of a reg­i­men that al­so in­cludes the chemother­a­py ben­damus­tine and a ver­sion of rit­ux­imab (Rit­ux­an).