We don’t know when (ex­act­ly) Lil­ly will an­nounce the FDA’s baric­i­tinib de­ci­sion, but watch out for the loom­ing pric­ing squab­ble

Some­time in the next few days, the FDA will be right on the PDU­FA de­ci­sion dead­line for baric­i­tinib. We just don’t know which day.

For some rea­son, Eli Lil­ly $LLY has de­cid­ed to keep the dead­line un­der wraps, as con­firmed yes­ter­day by a spokesper­son for the com­pa­ny. It could be tonight, ahead of a long hol­i­day week­end in the US. It could be to­mor­row. They could even wait un­til Mon­day, af­ter the de­ci­sion is for­mal, to roll out their an­nounce­ment.

While de­layed from three months ago to some time in mid-April, just about every an­a­lyst be­lieves that — bar­ring some sort of man­u­fac­tur­ing is­sue or some oth­er un­ex­pect­ed sna­fu — Lil­ly and its biotech part­ner In­cyte $IN­CY have this one in the bag. And it’s an­oth­er whop­per.

Af­ter be­ing treat­ed to a string of block­buster OKs for Ocre­vus and Dupix­ent, baric­i­tinib — which will be mar­ket­ed as Olu­mi­ant — is look­ing to dis­rupt the rheuma­toid arthri­tis mar­ket. To do that, Lil­ly has care­ful­ly man­aged a drug that an­a­lysts be­lieve could earn up to about $2 bil­lion in peak sales.

Lil­ly is well known in the in­dus­try for its ap­petite for huge stud­ies and pricey com­par­isons with the com­pe­ti­tion. In this case, it paid off with new da­ta that demon­strat­ed su­pe­ri­or­i­ty over Hu­mi­ra in treat­ing rheuma­toid arthri­tis. Hu­mi­ra is still un­der patent pro­tec­tion, and will re­main so for as long as Ab­b­Vie can fight off gener­ic com­pe­ti­tion to the biggest rev­enue pro­duc­er in the com­pa­ny and one of the biggest drugs on the plan­et.

So the next big thing, if in­deed the FDA has giv­en baric­i­tinib the green light, will be the price.

A new re­port from ICER took is­sue with the price for Hu­mi­ra, which it post­ed as a net of $40,415 a year. And ICER pro­vid­ed a thumbs up for baric­i­tinib and sar­ilum­ab, an­oth­er drug from Re­gen­eron and Sanofi that got held up by one of those man­u­fac­tur­ing sna­fus. ICER clear­ly prefers sar­ilum­ab on ef­fi­ca­cy, though we don’t know the price, giv­ing its mar­gin­al stamp of ap­proval to baric­i­tinib.

Not sur­pris­ing­ly, Ab­b­Vie took ex­cep­tion to the re­port, and Lil­ly may well end up do­ing the same. In this brave new world, an FDA ap­proval is a pre­lude to the price de­bate. And this one should be a doozie.

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.

BeiGene and Mus­tang nail down spe­cial FDA sta­tus for top drugs; Roche bags added cov­er­age for Hem­li­bra

→ BeiGene $BGNE is getting a boost in its drive to field a rival to Imbruvica. The FDA has offered an accelerated review to zanubrutinib, a BTK inhibitor that has posted positive results for mantle cell lymphoma. The PDUFA date lands on February 27, 2020. The drug scored breakthrough status at the beginning of the year.

→ BeiGene isn’t the only biopharma company to gain special regulatory status today. Mustang Bio $MBIO and St. Jude Children’s Research Hospital announced that MB-107, a lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease, has been granted Regenerative Medicine Advanced Therapy status.