FDA quash­es block­buster dreams at Eli Lil­ly and In­cyte, of­fer­ing a se­vere­ly lim­it­ed ap­proval for Olu­mi­ant

Vi­sions of block­buster sales for the rheuma­toid arthri­tis drug baric­i­tinib were quashed by the FDA’s ap­proval to­day. Reg­u­la­tors came through with an OK for the low, 2 mg dose of the drug, to be mar­ket­ed as Olu­mi­ant, spurn­ing the 4 mg dose that they had been seek­ing and lim­it­ing the pa­tient pop­u­la­tion to TNF re­sis­tant cas­es.

The reg­u­la­to­ry OK al­so comes with a black box warn­ing for the drug’s side ef­fects, which in­cludes a star­tling sig­nal on throm­boem­bolism.

Once tapped as a top fran­chise play­er for Eli Lil­ly $LLY and its part­ners at In­cyte $IN­CY, the FDA hauled the drug back from lim­bo af­ter the ar­rival of Scott Got­tlieb as com­mis­sion­er at the FDA. The agency had ini­tial­ly re­ject­ed the drug, de­mand­ing a new study. But then reg­u­la­tors abrupt­ly re­versed them­selves — with­out ex­pla­na­tion — and let the mar­ket­ing ap­pli­ca­tion pro­ceed.

De­spite the com­mer­cial set­back, Lil­ly plans to come out swing­ing for as much mar­ket share as it can grab. The phar­ma gi­ant says it will of­fer Olu­mi­ant at a 60% dis­count to the price of the lead­ing TNF in­hibitor.

“We are pleased to pro­vide RA pa­tients in the U.S. an ef­fec­tive treat­ment op­tion with Olu­mi­ant, as peo­ple with RA who have had an in­ad­e­quate re­sponse to TNF in­hibitors are gen­er­al­ly con­sid­ered to be some of the most dif­fi­cult to treat RA pa­tients,” said Christi Shaw, pres­i­dent, Lil­ly Bio-Med­i­cines.

To­day’s de­ci­sion fol­lows an ex­pert pan­el vote which weighed heav­i­ly against the 4 mg dose af­ter agency ex­perts out­lined their con­cerns about the safe­ty pro­file. They al­so gave a thumbs up to the 2 mg dose, which is like­ly to be far less suc­cess­ful and not near­ly as com­mer­cial­ly vi­able as the 4 mg. Some an­a­lysts were al­so shak­ing their heads to­day at the re­stric­tion to pa­tients who were re­sis­tant to TNF in­hibitors.

“The Olu­mi­ant ap­proval was a bit worse than the pan­el’s rec­om­men­da­tion,” Mor­gan Stan­ley an­a­lyst David Risinger said, ac­cord­ing to a re­port from Reuters.

Elim­i­nat­ing the 4 mg dose, notes Leerink’s Ge­of­frey Porges, gives Pfiz­er’s Xel­janz a big edge with 6 years of safe­ty da­ta. He adds:

Ul­ti­mate­ly this la­bel means that Olu­mi­ant is like­ly to be re­strict­ed to sal­vage ther­a­py on­ly, and ex­pect fore­casts for the prod­uct to de­cline even fur­ther (Lil­ly con­sen­sus re­mains >$1bn). This doesn’t mean that Ab­b­Vie’s upadac­i­tinib and Gilead’s fil­go­tinib will au­to­mat­i­cal­ly get this la­belling. In fact, it is like­ly that one or both of them avoids the throm­bo­sis risk and gets mul­ti­ple dos­es ap­proved with su­pe­ri­or ef­fi­ca­cy. We be­lieve that the best of the JAKs is still fil­go­tinib, un­til proven oth­er­wise, but the class as a whole is like­ly to be sad­dled with much of the lan­guage in to­day’s Olu­mi­ant la­bel.

This marks the sec­ond big set­back for In­cyte this year, af­ter its IDO pro­gram foundered and scut­tled a whole line­up of com­bi­na­tion stud­ies with ma­jor in­dus­try play­ers.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

PhII Alzheimer's fail­ure deals new blow to Roche, AC Im­mune — but the tau hy­poth­e­sis is far from dead

The leading anti-tau antibody has failed its first Phase II testing, casting a shadow on a popular target (just trailing amyloid beta) for Alzheimer’s disease.

Roche and AC Immune are quick to acknowledge disappointment in the topline readout, which suggested that semorinemab did not reduce cognitive decline among patients with early Alzheimer’s disease, who are either just starting to have symptoms or have mild manifestations.

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FDA commissioner Stephen Hahn at the White House (AP Images)

Un­der fire, FDA to is­sue stricter guid­ance for Covid-19 vac­cine EUA this week — re­port

The FDA has been insisting for months that a Covid-19 vaccine had to be at least 50% effective – a measure of transparency meant to shore public trust in the agency and in a vaccine that had been brought forward at record speed and record political pressure. But now, with concerns of a Trump-driven authorization arriving before the election, the agency may be raising the bar.

The FDA is set to release new guidance that would raise safety and efficacy requirements for a vaccine EUA above earlier guidance and above the criteria used for convalescent plasma or hydroxychloroquine, The Washington Post reported. Experts say this significantly lowers the odds of an approval before the election on November 3, which Trump has promised despite vocal concerns from public health officials, and could help shore up public trust in the agency and any eventual vaccine.

News brief­ing: PureTech plans Nas­daq de­but with sec­ondary list­ing; GoodRx prices $725M IPO

London Stock Exchange-listed PureTech Health announced Wednesday that it’s looking to extend to Nasdaq. But due to its “strong cash position,” the biotech says it isn’t issuing any new shares in the potential secondary listing.

The company’s shares closed at £256.50 Tuesday on the London Stock Exchange. Its candidate LYT-100 is currently in Phase I development for various indications, including lymphatic flow disorders and fibrotic and inflammatory disorders. PureTech is expecting a Phase Ib readout in lymphatic flow disorders later this year, and is also planning to launch a Phase II study for the drug to treat respiratory conditions experienced after Covid-19.

Rep. Andy Harris (R-MD) (Tasos Katopodis/Pool via AP Images)

The mi­cro-cap that tapped a mask-skep­tic con­gress­man for their Covid DSMB is ap­ply­ing for an EUA. Their ev­i­dence? 21 pa­tients

NeuroRx, the tiny biotech that came under fire last week after Politico reported they selected a congressman and two other acquaintances of the CEO to supervise their Covid-19 drug trial, announced today that they will ask the FDA to authorize their drug based on the results of just 21 patients.

Such an application would test the agency’s standards of evidence for an EUA, which have already come under scrutiny after controversial authorizations for convalescent plasma and hydroxychloroquine. The only other company to announce their intention to file for an EUA, Eli Lilly, did so after results came back from a randomized control study testing their antibody in over 450 patients.

J&J re­leas­es PhI­II safe­ty blue­print for Covid-19 vac­cine tri­al. How does it stack up to Mod­er­na, Pfiz­er and As­traZeneca?

Along with the initiation of its Phase III Covid-19 vaccine study announced Wednesday morning, Johnson & Johnson also released its trial protocol, giving an inside look at how the company is conducting its late-stage research.

The move comes after the other three companies conducting Phase III’s in the US — Moderna, Pfizer and AstraZeneca — each disclosed their own trial blueprints within the last week. Though the release of such protocols is typically done after trials have been completed, drug developers had come under intense pressure after a brief safety scare in an AstraZeneca trial and amid growing concern of a politically motivated vaccine authorization.

Covid-19 roundup: J&J be­gins piv­otal Phase III tri­al for vac­cine; Con­tro­ver­sial hu­man chal­lenge tri­als to be­gin in Lon­don — re­port

Johnson & Johnson announced it’s beginning a pivotal Phase III trial for its Covid-19 candidate, JNJ-78436735 — the first single-dose vaccine in this stage.

The Phase III trial, dubbed ENSEMBLE, will enroll 60,000 patients worldwide, making it the largest Phase III study of a Covid-19 vaccine to date. J&J said the candidate achieved positive interim results in a Phase I/IIa study, which will be published “imminently.” There’s a possibility that the first batches will be ready for potential emergency use in early 2021, according to the biotech.

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Vas Narasimhan (AP Images)

UP­DAT­ED: Still held down by clin­i­cal hold, No­var­tis' Zol­gens­ma falls fur­ther be­hind Bio­gen and Roche as FDA asks for a new piv­otal study

Last October, the FDA slowed down Novartis’ quest to extend its gene therapy to older spinal muscular atrophy patients by slapping a partial hold on intrathecal administration. Almost a year later, the hold is still there, and regulators are adding another hurdle required for regulatory submission: a new pivotal confirmatory study.

The new requirement — which departs significantly from Novartis’ prior expectations — will likely stretch the path to registration beyond 2021, when analysts were expecting a BLA submission. That could mean more time for Biogen to reap Spinraza revenues and Roche to ramp up sales of Evrysdi in the absence of a rival.

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