In a stun­ning set­back, FDA spurns Eli Lil­ly’s mar­ket­ing ap­pli­ca­tion for baric­i­tinib, de­mands more da­ta


The FDA has re­ject­ed Eli Lil­ly’s block­buster con­tender baric­i­tinib, say­ing that the phar­ma gi­ant will need to gath­er sig­nif­i­cant­ly more da­ta be­fore reg­u­la­tors will re­con­sid­er their de­ci­sion.

Ac­cord­ing to a state­ment from Lil­ly, the phar­ma gi­ant will not on­ly need to pro­vide more clin­i­cal da­ta on the ap­pro­pri­ate dos­es to be used for rheuma­toid arthri­tis, reg­u­la­tors are al­so de­mand­ing more safe­ty da­ta, which could sig­nif­i­cant­ly de­lay any re­sponse to the CRL. Lil­ly says it dis­agreed with the FDA’s con­clu­sion and the re­jec­tion.

The kick­back marks a stun­ning set­back for Eli Lil­ly $LLY CEO Dave Ricks, who owes in­vestors a string of promised ap­provals fol­low­ing a long drought in the clin­ic. The phar­ma gi­ant is known for its care­ful, if slow, ap­proach to late-stage de­vel­op­ment. Baric­i­tinib has been tagged as one of the top block­busters in late-stage de­vel­op­ment and an im­por­tant new drug for Lil­ly.

Hervé Hop­penot, In­cyte CEO

The re­jec­tion is al­so a set­back for its biotech part­ner, In­cyte $IN­CY, which li­censed out the drug to Lil­ly 8 years ago for $90 mil­lion up­front. An­a­lysts have pegged this as a po­ten­tial block­buster ca­pa­ble of earn­ing up to $2 bil­lion in peak an­nu­al sales, but that’s an­oth­er num­ber that is like­ly to get a se­ri­ous re­view.

Ever­cor­eISI’s Umer Raf­fat be­lieves this could in­di­cate that the FDA may ul­ti­mate­ly ap­prove a low dose of the drug, which could be­dev­il the de­vel­op­ers’ plans. He not­ed:

We’re wait­ing to hear back from com­pa­ny … but my sum­ma­ry thoughts are along the lines of:  there is a clear de­lay here, but I think they should get ap­proved ul­ti­mate­ly (keep in mind baric­i­tinib ap­proved in Eu­rope now).  How­ev­er, if FDA is re­al­ly fo­cused so much on “ap­pro­pri­ate dose”, I won­der if 2 mg is ul­ti­mate­ly the best dose … and if that’s the case, then it’s not so clear that 2 mg is su­pe­ri­or to Hu­mi­ra (re­call the head to head su­pe­ri­or­i­ty of baric­it­nib vs Hu­mi­ra was us­ing 4 mg dose … and plen­ty of da­ta from oth­er tri­als sug­gest­ing 2 mg has low­er ra­di­ograph­ic pro­gres­sion than 4 mg).

Lil­ly chose to re­lease the news on the morn­ing of Good Fri­day, a bank hol­i­day, de­lay­ing the beat­ing these two com­pa­nies faced. In­vestors, though, weren’t in a bet­ter mood Mon­day morn­ing. Lil­ly’s shares dropped 5.6% while In­cyte’s shares slid 13%.

Lil­ly’s John Lech­leit­er – now chair­man of the board – ini­tial­ly promised in­vestors at least two new drug ap­provals per year be­gin­ning in 2013, but had to de­lay on de­liv­er­ing un­til 2014. The com­pa­ny has al­so had three straight late-stage fail­ures for solanezum­ab in Alzheimer’s dis­ease. But new drugs have been kick­ing in, help­ing Lil­ly to start to grow its num­bers af­ter be­ing pum­meled by gener­ics for years.

I queried a com­pa­ny spokesper­son on whether Lil­ly now ex­pects to keep its promise on sev­er­al new drug ap­provals in 2017. She fol­lowed up by point­ing out that Lil­ly most re­cent­ly promised 20 new ap­provals be­tween 2014 and 2023, and that’s ev­i­dent­ly not re­strict­ed to FDA OKs. Baric­i­tinib al­so gets to count, as it was ap­proved in Eu­rope — as Olu­mi­ant — where it will be sub­ject­ed to a sin­gle-pay­er re­view and sig­nif­i­cant­ly low­er re­im­burse­ments.

That log­ic isn’t like­ly to help with dis­ap­point­ed an­a­lysts.

The re­jec­tion pro­vides Re­gen­eron and its part­ners at Sanofi a chance to over­come its re­cent re­jec­tion on sar­ilum­ab — for man­u­fac­tur­ing rea­sons — and push ahead with a ri­val ther­a­py that many an­a­lysts be­lieve is the odds-on fa­vorite. It al­so marks an­oth­er ad­van­tage for Ab­b­Vie, which has been cir­cling the wag­ons around Hu­mi­ra as it mounts a de­fense against new ther­a­pies bit­ing off pieces of its multi­bil­lion dol­lar fran­chise ther­a­py.

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. ICER clear­ly prefers sar­ilum­ab on ef­fi­ca­cy — though we don’t know the price — giv­ing on­ly a mar­gin­al stamp of ap­proval to baric­i­tinib.


“We are dis­ap­point­ed with this ac­tion. We re­main con­fi­dent in the ben­e­fit/risk of baric­i­tinib as a new treat­ment op­tion for adults with mod­er­ate-to-se­vere RA,” said Christi Shaw, pres­i­dent of Lil­ly Bio-Med­i­cines. “We will con­tin­ue to work with the FDA to de­ter­mine a path for­ward and ul­ti­mate­ly bring baric­i­tinib to pa­tients in the U.S.”

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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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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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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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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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

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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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.