Arch-backed biotech hauls in a fast B round as its re­born an­ti-psy­chot­ic zooms to a PhII fin­ish line

Steve Paul has booked a $68 mil­lion clin­i­cal round for his sin­gle as­set an­ti-psy­chot­ic biotech now in a Phase II study for schiz­o­phre­nia. And with $110 mil­lion in to­tal an­nounced ven­ture back­ing in 7 months, this one — Karuna Ther­a­peu­tics with a com­bo drug styl­ish­ly dubbed KarXT — is shap­ing up as a pos­si­ble turn in­to the fast lane to Wall Street, 2018 style.

Paul, you may re­call, com­plet­ed a lengthy de­vel­op­ment stint at Eli Lil­ly, where he spent a con­sid­er­able amount of time beat­ing his head against the stone wall of Alzheimer’s as the phar­ma gi­ant burned through bil­lions in its mis­guid­ed ef­forts to come up with a block­buster drug for that dis­ease. One of those drugs he stud­ied was the mus­carinic re­cep­tor ag­o­nist xanome­line, an an­ti-psy­chot­ic which was shelved with many oth­ers due to some se­ri­ous side ef­fects. Karuna is com­bin­ing that old drug with tro­spi­um chlo­ride (Sanc­tura) to con­trol the side ef­fects, look­ing to fol­low up with clin­i­cal da­ta to back them up ahead of what would have to be an am­bi­tious piv­otal plan to get through to a mar­ket­ing ap­proval.

As the A round came through last Au­gust, the el­der and more ex­pe­ri­enced 35-year vet Paul el­bowed com­pa­ny founder An­drew Miller aside for the CEO post, with Miller stay­ing on as COO. And the com­pa­ny has laid out plans for get­ting this drug to the mar­ket for both schiz­o­phre­nia as well as Alzheimer’s, which we all know is no easy tar­get — even for an­ti-psy­chotics.

“We did see an ef­fect on mem­o­ry,” Paul told me last sum­mer, “sim­i­lar to Ari­cept. What was quite sur­pris­ing, we mea­sured psy­chot­ic symp­toms in a de­ment­ed el­der­ly sub­group that had psy­chosis. And what we saw was a nice dose-de­pen­dent re­duc­tion in para­noia, vo­cal out­bursts and so on, com­pared to place­bo.”

At that time the biotech was still vir­tu­al, with on­ly 6 FTEs and a cast of sup­port­ing play­ers in the out­sourc­ing world. Now there’s more than $100 mil­lion rid­ing on their abil­i­ty to re­pro­duce the good re­sults, with­out any re­peat of the bad an­ti­cholin­er­gic ad­verse ef­fects you might ex­pect to see in a mus­carinic acetyl­choline re­cep­tor ag­o­nist.

It shouldn’t take too long to find out what they have. The biotech’s ran­dom­ized Phase II, with 180 pa­tients, should wrap in ear­ly No­vem­ber, ac­cord­ing to clin­i­cal­tri­ 

ARCH’s Bob Nelsen led the lat­est round, with this big group com­ing in be­hind: Fi­deli­ty Man­age­ment & Re­search Com­pa­ny, Even­tide As­set Man­age­ment, Piv­otal bioVen­ture Part­ners, Part­ner Fund Man­age­ment, Well­come Trust, Sands Cap­i­tal, Alexan­dria Ven­ture In­vest­ments, and founder PureTech Health. 

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

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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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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.

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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