Im­munomedics lays out its case for an ac­cel­er­at­ed OK of IM­MU-132 in triple-neg­a­tive breast can­cer

Aditya Bar­dia, Mass­a­chu­setts Gen­er­al Hos­pi­tal Can­cer Cen­ter

A lit­tle over a month af­ter Seat­tle Ge­net­ics $SGEN snapped up the glob­al rights to Im­munomedics lead can­cer drug IM­MU-132 (sac­i­tuzum­ab govite­can) in a $2 bil­lion deal, in­clud­ing $300 mil­lion in cash, the Mor­ris Plains, NJ-based biotech de­tailed the da­ta they plan to hus­tle to the FDA from its pos­i­tive mid-stage study for triple-neg­a­tive breast can­cer.

While the study was a fair­ly small, sin­gle-arm probe of ef­fi­ca­cy and safe­ty, the two part­ners are plan­ning the short­est route pos­si­ble to see if they can get an ac­cel­er­at­ed ap­proval while the ther­a­py nav­i­gates a Phase III piv­otal study.

In­ves­ti­ga­tors in the study tracked a me­di­an over­all sur­vival rate of 16.6 months and a pro­gres­sion-free sur­vival time of six months for the drug, which has the FDA’s break­through ther­a­py des­ig­na­tion for TNBC. Al­so, 30% of the 69 heav­i­ly pre­treat­ed pa­tients in the sin­gle-arm study achieved an ob­jec­tive re­sponse — two com­plete and 19 par­tial.

“These re­sults are very en­cour­ag­ing giv­en the im­pres­sive clin­i­cal ac­tiv­i­ty seen in a re­frac­to­ry set­ting, dura­bil­i­ty of re­spons­es, and the safe­ty pro­file,” not­ed lead au­thor Aditya Bar­dia, MD, MPH, at­tend­ing physi­cian at Mass­a­chu­setts Gen­er­al Hos­pi­tal Can­cer Cen­ter. “The av­er­age PFS is about 3.5 months for stan­dard agents, in­clud­ing cis­platin, capecitabine, nab-pa­cli­tax­el, and eribu­lin, as re­port­ed in ear­li­er metasta­t­ic TNBC tri­als.”

Three out of four of the pa­tients in the study who had pre­vi­ous­ly been treat­ed with a check­point — and failed — al­so achieved a par­tial re­sponse, a snap­shot of pos­si­ble com­bo po­ten­tial the biotech plans to fol­low up on.

These da­ta were laid out in the Jour­nal of Clin­i­cal On­col­o­gy. And Im­munomedics stock bumped up on the re­port, which fol­lows ear­li­er re­ports of the drug’s ef­fi­ca­cy.

The drug is an an­ti­body-drug con­ju­gate, or armed an­ti­body, de­signed for pre­cise de­liv­ery of SN-38, the ac­tive metabo­lite of irinote­can. As such, it fits com­fort­ably in Seat­tle Ge­net­ics sphere.

The drug didn’t kill any­one, but it’s not with­out safe­ty is­sues. Thir­ty-nine per­cent of the pa­tients suf­fered from neu­trope­nia, with 7% ex­pe­ri­enc­ing febrile neu­trope­nia and 13% af­flict­ed by se­vere di­ar­rhea.

Re­marked Im­munomedics CEO Cyn­thia L. Sul­li­van:

“We are work­ing dili­gent­ly with the FDA to make this valu­able prod­uct can­di­date avail­able to this group of pa­tients as soon as pos­si­ble. Blind­ed, in­de­pen­dent ra­di­o­log­i­cal as­sess­ments are on­go­ing, and so far show a high con­cor­dance with lo­cal to­mog­ra­phy find­ings.”

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 with any issues.

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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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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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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.

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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll