Aim­mune wins 12-month re­view and ad­com for peanut al­ler­gy drug; Phase­Bio flies on ear­ly da­ta for Bril­in­ta re­ver­sal agent

→ Af­ter out­go­ing FDA com­mis­sion­er Scott Got­tlieb threw a span­ner in the works ear­li­er this year for peanut al­ler­gy drug de­vel­op­ers — Aim­mune $AIMT and DBV Tech­nolo­gies $DB­VT — sug­gest­ing in a se­ries of tweets that al­ler­genic prod­ucts are not cov­ered by user-fees, and con­se­quent­ly do not qual­i­fy for the PDU­FA process, Aim­mune on Mon­day of­fered some clar­i­ty re­gard­ing its mar­ket­ing ap­pli­ca­tion. The BLA for its prod­uct — AR101 — has been ac­cept­ed for re­view and will be mulled over by the FDA over 12 months. This pe­ri­od still keeps it ahead of arch ri­val DBV, which re­scind­ed its ap­pli­ca­tion last De­cem­ber — due to man­u­fac­tur­ing con­cerns — and plans to re­sub­mit in the third quar­ter. Aim­mune had sub­mit­ted its ap­pli­ca­tion to the FDA late last year — but the US gov­ern­ment shut­down stut­tered the reg­u­la­tor’s ac­cep­tance. “The FDA has in­formed Aim­mune that the BLA will be re­viewed…as mea­sured from the Jan­u­ary 2019 start date. As a con­se­quence, re­view of the BLA may take un­til late Jan­u­ary 2020. Aim­mune is cur­rent­ly en­gaged in dis­cus­sions with the FDA re­gard­ing the re­view time­line for the AR101 BLA. The FDA ex­pects to con­vene an ad­vi­so­ry com­mit­tee meet­ing to dis­cuss the ap­pli­ca­tion,” Aim­mune said in state­ment.

→ A tiny, re­cent­ly pub­lic biotech has re­port­ed ear­ly da­ta that might serve to re­vive its shaky for­tunes. Phase­Bio, which went pub­lic last Oc­to­ber, showed its ex­per­i­men­tal drug PB2452 was able to swift­ly and sus­tain­ably re­versed the ef­fects of As­traZeneca’s $AZN an­ti-clot­ting drug Bril­in­ta in a Phase I study — da­ta were un­veiled on Sun­day at the Amer­i­can Col­lege of Car­di­ol­o­gy (ACC) in par­al­lel to pub­li­ca­tion in the New Eng­land Jour­nal of Med­i­cine. The drug — in-li­censed from the British drug­mak­er — re­versed Bril­in­ta’s ef­fects with­in 5 min­utes af­ter ad­min­is­tra­tion and was sus­tained for more than 20 hours (p<0.001). The mon­o­clon­al an­ti­body is be­ing de­vel­oped to re­verse the an­tiplatelet ac­tiv­i­ty of Bril­in­ta in ma­jor bleed­ing and ur­gent surgery sit­u­a­tions. “Since the IPO, PHAS has trad­ed poor­ly as the sto­ry was per­ceived as ‘ear­ly’ and shares got caught up in the biotech sell-off last De­cem­ber. The well-re­ceived ACC pre­sen­ta­tion, and es­pe­cial­ly the sur­prise NE­JM pub­li­ca­tion, should right the ship, in our view, as in­vestors learn more about PB2452 and its sig­nif­i­cant mar­ket po­ten­tial. From here, PHAS plans to launch a P2a study to con­firm dos­ing in an old­er healthy-for-age pop­u­la­tion, with da­ta ex­pect­ed around mid-year,” Stifel an­a­lysts wrote in a note. Shares of the drug de­vel­op­er $PHAS shot up about 74.5% at $6.49 in ear­ly Mon­day trad­ing.

→ At the So­ci­ety of Gy­ne­co­log­ic On­col­o­gy 50th An­nu­al Meet­ing on Women’s Can­cer, Leap Ther­a­peu­tics of­fered an ear­ly pos­i­tive snap­shot of da­ta from an on­go­ing mid-stage study test­ing its drug, DKN-01, as a monother­a­py and in com­bi­na­tion with chemother­a­py pa­cli­tax­el in pa­tients with re­lapsed/re­frac­to­ry en­dometri­oid en­dome­tri­al can­cer (EEC) or en­dometri­oid ovar­i­an can­cer (EOC). Pa­tients, in­clud­ing those with car­ci­nosar­co­ma and Wnt path­way al­ter­ations, have ex­pe­ri­enced par­tial re­spons­es and durable clin­i­cal ben­e­fit in ei­ther arm of the study, the com­pa­ny said, adding that the com­plete da­ta set will be­come avail­able in the com­ing months. Shares of the Cam­bridge, Mass­a­chu­setts-based drug de­vel­op­er $LP­TX jumped about 19% at $2.67 in Mon­day morn­ing trad­ing.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.

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 with any issues.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.

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.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.

Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.