Pox­el, part­ner Sum­it­o­mo Dainip­pon taste suc­cess in first piv­otal test for oral di­a­betes drug

The French meta­bol­ic ex­perts at Pox­el and their Asian part­ners at Sum­it­o­mo Dainip­pon Phar­ma have hit a home run with the first late-stage Japan­ese test of their oral di­a­betes drug imeglim­in, which bodes well for the pair of Phase III tri­als ex­pect­ed to read­out lat­er this year and fu­els the block­buster ex­pec­ta­tions tagged on the mi­to­chon­dr­i­al ther­a­py.

The drug be­longs to a new chem­i­cal class of oral agents called ‘glim­ins’ that si­mul­ta­ne­ous­ly tar­get all three key or­gans in­volved in di­a­betes: the pan­creas, the liv­er and the mus­cles. Imeglim­in is de­signed to work on the two main de­fects seen in pa­tients with type II di­a­betes: by in­creas­ing in­sulin se­cre­tion in the pan­creas, in a glu­cose-de­pen­dent man­ner; and by de­creas­ing the ex­cess pro­duc­tion of glu­cose by the liv­er, while en­hanc­ing ‘in­sulin sen­si­tiv­i­ty’ in the mus­cles.

Thomas Kuhn, Pox­el

The TIMES 1 tri­al test­ed a 1000 mg dose of imeglim­in against a place­bo in 213 Japan­ese pa­tients with type 2 di­a­betes. The drug-in­duced a sta­tis­ti­cal­ly sig­nif­i­cant re­duc­tion (p<0.0001) in HbA1c — av­er­age blood sug­ar lev­els over a pe­ri­od of weeks/months — at week 24, meet­ing the main goal. The place­bo-cor­rect­ed mean change in HbA1c from base­line was – 0.87%, the com­pa­nies said.

The drug al­so met the main sec­ondary goal of re­duc­ing fast­ing plas­ma glu­cose, and analy­ses of oth­er sec­ondary end­points are on­go­ing.

With an ag­ing pop­u­la­tion and grow­ing rates of obe­si­ty, di­a­betes in Japan has bal­looned, with the num­ber of sus­pect­ed di­a­bet­ics hit­ting a record 10 mil­lion for the first time in 2016, ac­cord­ing to gov­ern­ment es­ti­mates cit­ed by Nikkei.

“The TIMES 1 re­sults con­firm the ro­bust ef­fi­ca­cy com­bined with fa­vor­able safe­ty ob­served in the Phase 2b tri­al in Japan and the po­ten­tial ben­e­fits that Imeglim­in can bring to type 2 di­a­betes pa­tients glob­al­ly,” said Pox­el chief Thomas Kuhn. “The TIMES 1 da­ta is the first ma­jor step to­wards fil­ing the Japan­ese New Drug Ap­pli­ca­tion in 2020. Japan rep­re­sents the sec­ond largest sin­gle mar­ket for type 2 di­a­betes and, Asia, in broad­er terms, is con­sid­ered the most im­por­tant ge­o­graph­ic lo­ca­tion with re­gards to treat­ing the di­a­betes pan­dem­ic in the fu­ture.

In 2016, Pox­el re­port­ed that a Phase IIb tri­al in Japan reg­is­tered he­mo­glo­bin A1c re­duc­tions of 0.52%, 0.94% and 1.00% for the 500 mg, 1000 mg and 1500 mg dose twice-dai­ly. In 2017, the Mer­ck Serono spin­out forked over the Asia rights to the treat­ment to Sum­it­o­mo Dainip­pon Phar­ma in a deal worth up to $300 mil­lion.

Da­ta from TIMES-2 and TIMES-3 are ex­pect­ed lat­er this year. Pox­el (EPA: $POX­EL) out-li­censed the rights to imeglim­in in the Unit­ed States and Eu­rope to Vivek Ra­maswamy’s Roivant Sci­ences last year, with this part­ner fund­ing the ma­jor­i­ty of the cost­ly Phase III pro­gram ex­pect­ed to com­mence this year.

“Imeglim­in has a nov­el mech­a­nism of ac­tion as a mi­to­chon­dr­i­al bioen­er­get­ics en­hancer, po­ten­tial­ly pro­vid­ing a unique po­si­tion in the com­pet­i­tive type II di­a­betes treat­ment land­scape,” Jef­feries an­a­lysts wrote in a note last month. They mod­eled a 60% prob­a­bil­i­ty of com­mer­cial suc­cess for imeglim­in in the US/EU and Asia, with an es­ti­mate of $3.1 bil­lion in glob­al peak sales, in­clud­ing $600 mil­lion in Japan.


Im­age: Shut­ter­stock

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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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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