No­var­tis shows a hole card in a high-stakes PhI­II pok­er game for MS drugs


No­var­tis float­ed a new, pos­i­tive da­ta point from its re­cent­ly com­plet­ed Phase III study of sipon­i­mod (BAF312), an S1P re­cep­tor drug that is be­ing ad­vanced for sec­ondary pro­gres­sive mul­ti­ple scle­ro­sis (SPMS).

In­ves­ti­ga­tors for the phar­ma gi­ant turned up at the an­nu­al meet­ing of the Eu­ro­pean Com­mit­tee for Treat­ment and Re­search in Mul­ti­ple Scle­ro­sis to say that they tracked a 21% re­duc­tion in the risk of dis­abil­i­ty pro­gres­sion in the drug arm, com­par­ing it to the place­bo group. They al­so mapped a “sig­nif­i­cant” im­prove­ment in pa­tients’ an­nu­al­ized re­lapse rate but failed to see the da­ta pan out for an im­prove­ment in a 25-foot walk­ing test, a clas­sic way to check for im­prove­ments in phys­i­cal func­tion.

The goal now is to run the num­bers by reg­u­la­tors, to see if they’re in­clined to ap­prove the drug now. But reg­u­la­tors are like­ly No­var­tis’s least con­cern in the high stakes game of MS drug de­vel­op­ment.

For No­var­tis, an ap­proval would help pro­tect its $3 bil­lion in threat­ened Gilenya rev­enue, which has been grow­ing fast. An­a­lysts are ex­pect­ing gener­ic ri­vals to crowd in­to the block­buster mar­ket in a lit­tle more than 2 years, and No­var­tis is lin­ing up a new prod­uct while Te­va has been mount­ing a fi­nal, los­ing de­fense against knock­offs of Co­pax­one.

Roche, mean­while, is ex­pect­ed to hit pay dirt with Ocre­vus (ocre­lizum­ab), which has been suc­cess­ful for both re­laps­ing/re­mit­ting MS — where the bulk of the mar­ket is — and pri­ma­ry pro­gres­sive MS pa­tients (PPMS). In PPMS, pa­tients track along a steady de­cline. Sec­ondary pro­gres­sive MS, or SPMS, is made up of the large seg­ment of RRMS pa­tients whose dis­ease de­vel­ops over time in­to a pro­gres­sive form of the dis­ease.

The progress at Roche is adding pres­sure on Bio­gen, which had to ac­knowl­edge in June that opicinum­ab failed a crit­i­cal Phase II study, bad­ly dam­ag­ing its stock price. Their fo­cus is turn­ing to MT-1303, an­oth­er S1P drug it in­li­censed from Mit­subishi Tan­abe last fall with plans to hus­tle it in­to a late-stage pro­gram. Bio­gen — which has a lead­ing mar­ket po­si­tion in MS — has had a hard time pla­cat­ing an­a­lysts who have grown in­creas­ing­ly rest­less with the ero­sion of rev­enue fore­casts for its flag­ship drug Tec­fidera.

One oth­er play­er in this S1P game is Cel­gene, which paid $7.2 bil­lion in cash to buy Re­cep­tos last year to get its hands on ozan­i­mond, now in its own Phase III au­toim­mune pro­gram.

In pre­pared text, Vas­ant Narasimhan, Glob­al Head Drug De­vel­op­ment and Chief Med­ical Of­fi­cer for No­var­tis, added, “there are very few avail­able treat­ment op­tions to de­lay dis­ease pro­gres­sion in SPMS, and there is a high un­met need for ef­fec­tive ther­a­pies with an ac­cept­able safe­ty pro­file for peo­ple with the con­di­tion.”

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.

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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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.