Im­munomedics must en­dure an­oth­er de­lay as FDA re­jects can­cer drug cit­ing man­u­fac­tur­ing is­sues

We all like to root for the un­der­dog. Fol­low­ing an arid spell stretch­ing over three decades that in 2017 led to the ouster of Im­munomedics founder/CSO and his CEO wife, the com­pa­ny (and an­a­lysts) were hope­ful the drug de­vel­op­er would fi­nal­ly win its first FDA nod for its treat­ment for a dif­fi­cult-to-treat form of breast can­cer. The man­age­ment shake­up, at the be­hest of an ac­tivist share­hold­er cam­paign, had re­ju­ve­nat­ed the com­pa­ny’s for­tunes by at­tract­ing in­vest­ment and ramp­ing up the drug’s de­vel­op­ment. But the FDA on Thurs­day hand­ed the biotech a re­jec­tion cit­ing man­u­fac­tur­ing is­sues, adding yet an­oth­er de­lay to the reg­u­la­to­ry time­line.

The New Jer­sey-based com­pa­ny’s shares plum­met­ed about 33.5% in ear­ly Fri­day trad­ing.

The drug — called IM­MU-132 or sac­i­tuzum­ab govite­can — is an an­ti­body drug con­ju­gate (ADC) or an armed an­ti­body which is de­signed to de­liv­er a spe­cif­ic pay­load of a chemother­a­py di­rect­ly to a tu­mor, to re­duce the tox­i­c­i­ty as­so­ci­at­ed with the con­ven­tion­al ad­min­is­tra­tion of chemother­a­py. The FDA grant­ed the drug pri­or­i­ty re­view on the ba­sis of da­ta from an on­go­ing sin­gle-arm Phase II study in pa­tients with metasta­t­ic triple-neg­a­tive breast can­cer (mTNBC) who have un­der­gone pri­or ther­a­py. Sac­i­tuzum­ab govite­can, which is al­so be­ing test­ed in a con­fir­ma­to­ry Phase III study in mTNBC pa­tients, was ex­pect­ed to win ac­cel­er­at­ed ap­proval on Jan­u­ary 18. An­a­lysts had sug­gest­ed avail­able mid-stage drug da­ta in­di­cat­ed it was a sig­nif­i­cant im­prove­ment over stan­dard-of-care and had as­signed it peak sales ex­pec­ta­tions of $1 bil­lion and more.

Michael Pehl

In its last up­date in De­cem­ber, Im­munomedics re­port­ed da­ta from the 108-pa­tient mid-stage study — as as­sessed by in­de­pen­dent re­view — demon­strat­ed the drug in­duced par­tial or com­plete tu­mor shrink­age in 34% of pa­tients, with a me­di­an du­ra­tion of re­sponse of about 9 months.

To be sure, the com­pa­ny was not ex­pect­ing the re­jec­tion. It hired a sales force back in No­vem­ber in an­tic­i­pa­tion of the Jan­u­ary ap­proval and told an­a­lysts in De­cem­ber that man­u­fac­tur­ing is­sues the US health reg­u­la­tor had flagged in Au­gust had been since ad­dressed.

In a De­cem­ber 20 note, Jef­feries an­a­lysts said that while man­u­fac­tur­ing is­sues could be a source of con­cern, they re­mained “con­fi­dent in ‘132 as the com­pa­ny be­lieves all is­sues have been re­solved and it has in­vest­ed heav­i­ly in the com­mer­cial in­fra­struc­ture pri­or to ap­proval. Ad­di­tion­al­ly, we would high­light that IM­MU has se­cured an agree­ment with Sam­sung Bi­o­log­ics as a sec­ondary man­u­fac­tur­er…pro­vid­ing risk mit­i­ga­tion if is­sues can­not be re­solved at cur­rent site.”

On Thurs­day, Im­munomedics un­der­scored the sac­i­tuzum­ab govite­can re­jec­tion had noth­ing to do with the drug’s clin­i­cal da­ta.

“The is­sues re­lat­ed to ap­prov­abil­i­ty in the CRL were ex­clu­sive­ly fo­cused on Chem­istry, Man­u­fac­tur­ing and Con­trol mat­ters and no new clin­i­cal or pre­clin­i­cal da­ta need to be gen­er­at­ed,” Im­munomedics chief Michael Pehl said in a state­ment, adding that the com­pa­ny in­tend­ed to re­quest a meet­ing with the FDA to bet­ter un­der­stand the agency’s re­quire­ments.

But with no light at the end of the gov­ern­ment shut­down tun­nel, who knows how long that will take.

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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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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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

As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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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.