Cel­gene’s up­date on its big Crohn’s drug? Trust us, we’re do­ing fine

Cel­gene in­sists that an in­ter­im analy­sis of its close­ly-watched study for GED-0301 (mon­gersen) was “en­cour­ag­ing,” but it failed to show any of its cards to­day to back that up.

Scott Smith, Pres­i­dent of Glob­al In­flam­ma­tion and Im­munol­o­gy, Cel­gene

Cel­gene $CELG paid a whop­ping $710 mil­lion up front to in-li­cense this drug for Crohn’s dis­ease. High­light­ed as a key pro­gram in its at­tempt to build a fran­chise for in­flam­ma­tion and im­munol­o­gy, GED-0301 is one of the com­pa­ny’s biggest ex­per­i­men­tal as­sets. And at this stage, all that Cel­gene is will­ing to say is that their in­ves­ti­ga­tors tracked an im­prove­ment in some pa­tients.

An­a­lysts have been wait­ing for some clear signs re­lat­ed to the drug’s ef­fect on Crohn’s. In­ves­ti­ga­tors have tout­ed some re­mark­able ad­vances for pa­tients in an ear­ly study, but ex­perts al­so ques­tioned just how se­vere­ly af­flict­ed they were go­ing in­to the study. “For shares to move high­er,” BMO not­ed re­cent­ly, “the tri­al needs to demon­strate CDAI ben­e­fit (P3 pri­ma­ry end­point) in line with IGON-1 P2 tri­al and en­do­scop­ic im­prove­ment cor­re­lat­ed with ther­a­py du­ra­tion.”

From the state­ment:

Topline da­ta from CD-001 show that in a pro­por­tion of pa­tients treat­ed with oral GED-0301 there was en­do­scop­ic im­prove­ment (de­fined as a 25 per­cent im­prove­ment from base­line) and clin­i­cal re­sponse and re­mis­sion across all treat­ment groups at week 12. Find­ings to date re­veal no new safe­ty sig­nals and tol­er­a­bil­i­ty is con­sis­tent with ear­li­er stud­ies.

It’s not un­usu­al, though, to see a pro­por­tion of pa­tients im­prove in any arm of a study, in­clud­ing the place­bo group. The ques­tion is the rel­a­tive rate of im­prove­ment, and that’s some­thing that Cel­gene is not re­fer­ring to specif­i­cal­ly. What it will al­low is that the big biotech re­mains en­thu­si­as­tic about its prospects.

“Giv­en the high un­met need in Crohn’s dis­ease, we are pleased that oral GED-0301 showed both en­do­scop­ic im­prove­ments and clin­i­cal­ly mean­ing­ful re­spons­es and re­mis­sion at an ear­ly time­point in this study,” said Scott Smith, pres­i­dent of Cel­gene In­flam­ma­tion and Im­munol­o­gy. “These da­ta are par­tic­u­lar­ly en­cour­ag­ing for sev­er­al rea­sons, in­clud­ing the dif­fi­cult-to-treat pa­tient pop­u­la­tion eval­u­at­ed in the tri­al.”

An­a­lysts have vir­tu­al­ly noth­ing to go on. But some are try­ing to read this up­date as best they can. And not every­one is as en­cour­aged as Cel­gene pro­fess­es to be. Af­ter all, says Baird’s Bri­an Sko­r­ney, “it’s hard to be­lieve an un­am­bigu­ous suc­cess wouldn’t be de­scribed in some quan­ti­ta­tive de­tail.”

“In our view,” notes Leerink’s Ge­of­frey Porges, “while the drug could be ac­tive in some pa­tients, it seems that the im­pres­sive da­ta from the pre­vi­ous phase 2 tri­al will not trans­late to the phase 3 pa­tient pop­u­la­tion, or to the re­al world which main­ly con­sists of pa­tients with ac­tive, long­stand­ing, treat­ment re­sis­tant dis­ease, sim­i­lar to to­day’s en­doscopy tri­al pa­tients. Im­por­tant­ly, how­ev­er, the rel­a­tive­ly clean safe­ty pro­file re­port­ed from pre­vi­ous stud­ies was ap­par­ent­ly main­tained in this study. We spoke with Cel­gene this morn­ing, and the com­pa­ny stat­ed that there would be no changes to on­go­ing and planned phase 3 tri­als as a re­sult of this analy­sis.”

Bri­an Abra­hams at Jef­feries had this:

KOLs had in­di­cat­ed to us they be­lieved 1/3-1/2 of pts achiev­ing a 50% en­do­scop­ic im­prove­ment (SES-CD score re­duc­tion) by 12wks would sug­gest true ac­tiv­i­ty of the agent, which re­call is still un­clear based on the com­plex pri­or ph.IIa dataset. CELG had in­di­cat­ed that KOLs and reg­u­la­tors had told them a me­di­an 25% SES-CD score im­prove­ment for the whole group would be the bar for suc­cess. The PR’s state­ment that a pro­por­tion of pts had this 25% re­duc­tion ap­pears to sug­gest ac­tiv­i­ty fell short of both of these thresh­olds. How­ev­er, we note that giv­en the lack of sub­stan­tial his­tor­i­cal prece­dent, the bar for ad­e­quate sup­port­ive ac­tiv­i­ty on en­doscopy is not de­fin­i­tive, and based on our feed­back with the com­pa­ny ap­pears to be evolv­ing from their con­tin­ued dis­cus­sions with physi­cians and reg­u­la­tors (and may now be a por­tion of pts achiev­ing 25% re­duc­tions). His­tor­i­cal da­ta has sug­gest­ed in mod-to-se­vere Crohn’s pts tak­ing a place­bo, me­di­an SES-CD score im­proved by 6wks by a me­di­an of 14% and 1/4 of pts had 50% re­duc­tions, though this was from a small post-hoc analy­sis and may have been in less heav­i­ly pre-treat­ed pts.

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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