Blood­ied Cel­gene posts promis­ing ozan­i­mod PhI­II MS da­ta, but is it re­al­ly a $6 bil­lion drug?

Cel­gene pumped out its Phase III da­ta on its block­buster con­tender ozan­i­mod over the week­end, of­fer­ing some en­cour­ag­ing com­par­isons with Avonex in treat­ing pa­tients with re­laps­ing mul­ti­ple scle­ro­sis. But some of these num­bers will like­ly trig­ger some sec­ond guess­ing about the drug’s peak sales abil­i­ty af­ter Cel­gene paid $7.2 bil­lion to get its hands on the drug.

In­ves­ti­ga­tors re­cruit­ed 1,346 pa­tients for the SUN­BEAM tri­al, post­ing sta­tis­ti­cal­ly sig­nif­i­cant scores for the an­nu­al­ized re­lapse rate. But in a pooled analy­sis of SUN­BEAM and RA­DI­ANCE Part B stud­ies, their drug did not hit the goal for the time to 3-month con­firmed dis­abil­i­ty pro­gres­sion.

An­a­lysts have been do­ing some cross-tri­al com­par­isons with No­var­tis’ Gilenya, which is com­ing off patent pro­tec­tion at the end of 2019, and con­clud­ed that Cel­gene looks like it could leap out on­to the mar­ket with a sim­i­lar ef­fi­ca­cy pro­file but bet­ter safe­ty fea­tures. If so, Cel­gene could gain a sig­nif­i­cant edge in the ri­val­ry to come for this drug, which Cel­gene has said is worth $4 bil­lion to $6 bil­lion a year in peak sales.

Ge­of­frey Porges

Sev­er­al an­a­lysts were will­ing to give Cel­gene — long a dar­ling of the biotech in­vestor crowd — a thumb’s up for the da­ta, which the big biotech bad­ly needs af­ter its mis­steps over the last two weeks, cut­ting longterm fore­casts and trig­ger­ing an 18% drop in the share price $CELG. Just be­fore that, Cel­gene was forced to con­cede that its pro­gram for mon­gersen had im­plod­ed in Phase III.

“The posters and pre­sen­ta­tions sug­gest that ozan­i­mod in­deed has a dif­fer­en­ti­at­ed safe­ty and ef­fi­ca­cy pro­file to oth­er wide­ly used med­i­cines in the MS cat­e­go­ry,” not­ed Leerink’s Ge­of­frey Porges, who says an OK in MS along with in­flam­ma­to­ry bow­el dis­ease and more could cre­ate $2.9 bil­lion in sales by 2022. “This prod­uct, along with lus­pa­ter­cept, has be­come the stan­dard bear­er for Cel­gene’s late stage pipeline, and in our view the da­ta at the meet­ing jus­ti­fy some re­cov­ery in sen­ti­ment about the com­pa­ny’s port­fo­lio and out­look.”

Sun­Trust’s Yatin Sune­ja did some ba­sic math and came out up­beat about Cel­gene’s mar­ket prospects.

Yatin Sune­ja

Ef­fi­ca­cy high­lights (pri­mar­i­ly fo­cused on ozan­i­mod 1mg) in­clud­ed (1) 48% ARR re­duc­tion vs. Avonex (pri­ma­ry end­point; broad­ly in-line with Gilenya’s 52% re­duc­tion in TRANS­FORMS, in our view), (2) T1 GdE le­sion re­duc­tion at month 12 of 63% vs. Avonex (which we be­lieve is bet­ter than Gilenya’s 55% re­duc­tion in TRANS­FORMS) and (3) 33% re­duc­tion in whole brain vol­ume loss vs. Avonex (at least in-line with Gilenya’s 32% re­duc­tion in TRANS­FORMS). While the tri­al was not pow­ered for 3-month con­firmed dis­abil­i­ty pro­gres­sion (an ex­plorato­ry end­point), there was a 31% re­duc­tion vs. Avonex.

Baird’s Bri­an Sko­r­ney has been fol­low­ing the pro­gram, and I asked for his take on the re­sults. His re­sponse:

I think the ozan­i­mod da­ta had no sur­pris­es, which giv­en the last two weeks of Cel­gene sur­pris­es is a rel­a­tive­ly good thing. I think every­one ex­pect­ed an ef­fi­ca­cy pro­file that looks on par with Gilenya and a safe­ty pro­file that looks bet­ter than Gilenya and that is what we saw. It def­i­nite­ly ap­pears to have a bet­ter car­diac pro­file. We will see how the FDA la­bels around that but with­out ini­tial dose mon­i­tor­ing, it seems like a more com­pelling oral to start pa­tients on than Gilenya. Every­thing is a cross tri­al com­par­i­son but the per­spec­tive is that Avonex is re­al­ly safe and ozan­i­mod seemed to match it pret­ty nice­ly, so com­pared to Gilenya, it will be per­ceived as look­ing safe. Things that peo­ple are con­cerned about with Gilenya, like liv­er tox and in­fec­tion risk, all look bet­ter here. I don’t think this is a par­a­digm shift­ing drug though, the way Ocre­vus  ap­pears to be.

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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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive PhI­II 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.

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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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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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

UP­DAT­ED: 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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Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.