Ul­tragenyx, Ky­owa Hakko Kirin plan a date with the FDA af­ter pos­i­tive PhI­II for X-linked hy­pophos­phatemia

Emil Kakkis

Shares of Ul­tragenyx surged about 10% af­ter the biotech re­port­ed clear­ly pos­i­tive Phase III da­ta for their Ky­owa Hakko Kirin-part­nered drug for X-linked hy­pophos­phatemia. And now they plan to hus­tle that da­ta pack­age to reg­u­la­tors at the FDA and EMA.

Re­searchers re­cruit­ed 134 pa­tients for the study, and found that 94% of the pa­tients treat­ed with buro­sum­ab achieved nor­mal serum phos­pho­rus lev­els, com­pared to on­ly 8% in the place­bo arm. The drug al­so hit on a cou­ple of sec­ondary end­points, rat­ing im­prove­ments in stiff­ness and phys­i­cal func­tion, but missed sta­tis­ti­cal sig­nif­i­cance on the pain score.

Ky­owa Hakko Kirin dis­cov­ered this an­ti­body, which tar­gets phos­pha­turic hor­mone fi­brob­last growth fac­tor 23, or FGF23, which lim­its serum lev­els of phos­pho­rus and ac­tive vi­t­a­min D. The drug is al­so in de­vel­op­ment for tu­mor-in­duced os­teo­ma­la­cia, which is al­so char­ac­ter­ized by ex­cess lev­els of FGF23.

These two com­pa­nies struck a de­vel­op­ment deal for this drug back in 2013, with Ul­tragenyx lead­ing de­vel­op­ment ef­forts in the XLH in­di­ca­tion and a split on the de­vel­op­ment costs. If the drug is ap­proved, Ul­tragenyx and KHK will share com­mer­cial re­spon­si­bil­i­ties and prof­its in the US and Cana­da. KHK will com­mer­cial­ize the ther­a­py alone in the EU while Ul­tragenyx will de­vel­op and com­mer­cial­ize KRN23 in Mex­i­co, Cen­tral and South Amer­i­ca.

“These da­ta demon­strate a clin­i­cal im­prove­ment in pa­tients treat­ed with buro­sum­ab and sup­port the po­ten­tial for treat­ment of adults,” said Ul­tragenyx CEO Emil Kakkis in a state­ment. “When com­bined with a fa­vor­able safe­ty pro­file and a strong serum phos­pho­rus re­sponse, we be­lieve these clin­i­cal da­ta should sup­port reg­u­la­to­ry sub­mis­sions in adults with XLH, and we look for­ward to dis­cussing our fil­ing plans with the U.S. FDA.”

Dit­to the ex­ec­u­tive crew at part­ner Ky­owa Hakko Kirin.

“This study pro­vides valu­able ad­di­tion­al place­bo con­trolled da­ta to that al­ready ob­tained from the glob­al clin­i­cal de­vel­op­ment pro­gram for pe­di­atric and adult pa­tients with XLH.” said Mit­suo Satoh, VP and R&D chief at Ky­owa Hakko Kirin. “I be­lieve buro­sum­ab has the po­ten­tial to be an ef­fec­tive treat­ment op­tion for pa­tients with XLH.”

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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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