Tiny In­nate re­ports a PhI­Ib dis­as­ter, bat­ter­ing shares — and a NY con­gress­man

Reps. Chris Collins, R-NY, left, and Robert Pit­tenger, R-NC, leave a meet­ing of the House Re­pub­li­can Con­fer­ence in the Capi­tol on June 7, 2017. CQ Roll Call

Just about every­thing the lit­tle Aus­tralian biotech In­nate Im­munother­a­peu­tics tracked in its Phase IIb study of its ex­per­i­men­tal drug for mul­ti­ple scle­ro­sis points to a dis­as­ter. The da­ta on all 93 pa­tients in the study demon­strate a clear fail­ure of the drug to de­liv­er an im­prove­ment for pa­tients on any of “mul­ti­ple” end­points. The dropout rate in the drug arm was high. And the rate of se­ri­ous ad­verse events was high­er in the drug arm than in the con­trol group.

Rep. Chris Collins (R-NY)

In­nate (ASX Code: IIL) is go­ing back over the da­ta to see if the per pro­to­col re­sults on just the pa­tients who had com­plet­ed a siz­able por­tion of the study did any bet­ter. But in­ves­ti­ga­tors aren’t try­ing to en­cour­age any­one to be­lieve in MIS416, de­liv­ered by IV once a week for a year. They say there is no rea­son to ex­pect a change in out­comes.

All that is bad enough on its own. But even so, you may nev­er have heard about any of this but for one fact: A promi­nent US con­gress­man bet the ranch on this com­pa­ny’s stock — and ev­i­dent­ly lost vir­tu­al­ly all of it. In­nate’s stock al­most dis­ap­peared af­ter in­vestors got a look at the da­ta, falling to a few pen­nies a share.

Bloomberg re­porters, who have been cov­er­ing this sto­ry, did the math and con­clud­ed that New York Re­pub­li­can Chris Collins, In­nate’s biggest share­hold­er and a long­time be­liev­er, lost $16.7 mil­lion af­ter tak­ing the fli­er. And ev­i­dent­ly a num­ber of oth­er Re­pub­li­cans, in­clud­ing HHS Sec­re­tary Tom Price, al­so lost mon­ey as well.

HHS Sec­re­tary Tom Price

The con­nec­tions be­tween law­mak­ers in charge of health­care pol­i­cy and In­nate have now re­port­ed­ly in­spired an ethics in­ves­ti­ga­tion in­to the role Collins played in at­tract­ing in­vestors to the com­pa­ny. And in a sto­ry this morn­ing, The Buf­fa­lo News re­port­ed that the FDA had just grant­ed the biotech an IND to launch tri­als in the US, void­ing the con­gress­man’s claim that he had no con­flicts be­cause the biotech nev­er had busi­ness in front of US reg­u­la­tors.

The biggest les­son from all this, though, may be that any­one who bets a large por­tion of their wealth on a biotech long shot like this — based in Syd­ney — doesn’t know much about in­vest­ing, biotech or health­care. Collins, for his part, doesn’t agree. “For those that in­vest­ed in In­nate, in­clud­ing me, we all were so­phis­ti­cat­ed in­vestors who were aware of the in­her­ent risk,” Collins says in a pre­pared state­ment. He adds: “For every suc­cess­ful drug, there are count­less num­bers that fail. That’s how to­day’s sys­tem works.”

Si­mon Wilkin­son, In­nate Im­munother­a­peu­tics’ chief ex­ec­u­tive said: “These re­sults are a shock and def­i­nite­ly not what we were ex­pect­ing based on our pre­vi­ous clin­i­cal ex­pe­ri­ence with MIS416 and the re­port­ing of treat­ment ben­e­fits we have re­ceived from many com­pas­sion­ate use pa­tients over an ex­ten­sive 8-year pe­ri­od. These da­ta will be as dis­tress­ing to them as they will be for all the stake­hold­ers who were re­ly­ing on the out­come of this study.”

Wilkin­son can count his biggest in­vestor and biggest los­er as one of the most bad­ly shocked of all.

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.