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.

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma.

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

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UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

A rumor that Amgen is closing in on buyout deal for Alexion has sparked a guessing game on just what kind of M&A strategy Amgen is pursuing and how much Alexion is worth.

Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.

FDA asks why No­var­tis took two months to launch for­mal in­ter­nal probe, af­ter AveX­is flagged da­ta ma­nip­u­la­tion

And the plot thickens. Novartis $NVS officials are reportedly now scrambling to explain to the FDA why it took them two months to open an internal investigation into data discrepancies for their $2.1 million gene-therapy for spinal muscular dystrophy — the world’s most expensive drug.

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Build­ing on suc­cess­ful PD-1 pact, Eli Lil­ly li­cens­es di­a­betes drug to Chi­nese part­ners at In­novent

Eli Lilly is expanding its partnership with China’s Innovent in a deal involving a diabetes drug sitting in its Phase I reserves.

The two companies had jointly developed one of China’s first homegrown PD-1 agents, scoring an approval for Tyvyt (sintilimab) late last year for relapsed/refractory classical Hodgkin’s lymphoma. This time around, Lilly is out-licensing a piece of its diabetes pipeline, a leading franchise that has historically produced the top-selling Trulicity and Humalog.

Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.