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.

Qual­i­ty Con­trol in Cell and Gene Ther­a­py – What’s Re­al­ly at Stake?

In early 2021, Bluebird Bio was forced to suspend clinical trials of its gene therapy for sickle cell disease after two patients in the trial developed cancer. As company scientists rushed to assess whether there was any causal link between the therapy and the cancer cases, Bluebird’s stock value plummeted – as did those of multiple other biopharma companies developing similar therapies.

While investigations concluded that the gene therapy was unlikely to have caused cancer, investors and the public may be more skittish regarding the safety of gene and cell therapies after this episode. This recent example highlights how delicate the fields of cell and gene therapy remain today, even as they show great promise.

Law pro­fes­sors call for FDA to dis­close all safe­ty and ef­fi­ca­cy da­ta for drugs

Back in early 2018 when Scott Gottlieb led the FDA, there was a moment when the agency seemed poised to release redacted complete response letters and other previously undisclosed data. But that initiative never gained steam.

Now, a growing chorus of researchers are finding that a dearth of public data on clinical trials and pharmaceuticals means industry and the FDA cannot be held accountable, two law professors from Yale and New York University write in an article published Wednesday in the California Law Review.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Covid-19 man­u­fac­tur­ing roundup: Mary­land looks to grow biotech ca­pac­i­ty with $400M check; Rus­sia lands sec­ond Sput­nik V part­ner this week

A Maryland real estate project has added three new biotech-focused manufacturing and research buildings to an office park to keep up with demand created by the pandemic, the Washington Business Journal reported.

The Milestone Business Park — located off of I-270 in Germantown, MD — will see the new buildings and a total of 532,000 square feet as the campus rebrands to Milestone Innovation Park.

Novavax CEO Stanley Erck at the White House in 2020 (Andrew Harnik, AP Images)

As fears mount over J&J and As­traZeneca, No­vavax en­ters a shaky spot­light

As concerns rise around the J&J and AstraZeneca vaccines, global attention is increasingly turning to the little, 33-year-old, productless, bankruptcy-flirting biotech that could: Novavax.

In the now 16-month race to develop and deploy Covid-19 vaccines, Novavax has at times seemed like the pandemic’s most unsuspecting frontrunner and at times like an overhyped also-ran. Although they started the pandemic with only enough cash to last 6 months, they leveraged old connections and believers into $2 billion and emerged last summer with data experts said surpassed Pfizer and Moderna. They unveiled plans to quickly scale to 2 billion doses. Then they couldn’t even make enough material to run their US trial and watched four other companies beat them to the finish line.

FDA of­fers scathing re­view of Emer­gent plan­t's san­i­tary con­di­tions, em­ploy­ee train­ing af­ter halt­ing pro­duc­tion

The FDA wrapped up its inspection of Emergent’s troubled vaccine manufacturing plant in Baltimore on Tuesday, after halting production there on Monday. By Wednesday morning, the agency already released a series of scathing observations on the cross contamination, sanitary issues and lack of staff training that caused the contract manufacturer to dispose of millions of AstraZeneca and J&J vaccine doses.

Brad Bolzon (Versant)

Ver­sant pulls the wraps off of near­ly $1B in 3 new funds out to build the next fleet of biotech star­tups. And this new gen­er­a­tion is built for speed

Brad Bolzon has an apology to offer by way of introducing a set of 3 new funds that together pack a $950 million wallop in new biotech creation and growth.

“I want to apologize,” says the Versant chairman and managing partner, laughing a little in the intro, “that we don’t have anything fancy or flashy to tell you about our new fund. Same team, around the same amount of capital, same investment strategy. If it ain’t broke, don’t fix it.”

But then there’s the flip side, where everything has changed. Or at least speeded into a relative blur. Here’s Bolzon:

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Bay­er plots a ma­jor facelift at Berke­ley cam­pus, un­cork­ing a 30-year, $1.2B plan to dri­ve cell and gene ther­a­pies

Bayer first set roots in Berkeley back in 1974, when it was still operating as Miles Labs. The site has pumped out three hemophilia A treatments for distribution worldwide; but now, as the pharma continues its cell and gene therapy push, it has something bigger in mind.

Bayer is planning a 30-year revamp at the campus, which includes 918,000 square feet in new buildings and double the jobs, according to a report by the Bay Area Council Economic Institute.

LLS backs 5 new can­cer drug projects with up to $50M; Trodelvy con­tin­ues to im­press with more TNBC da­ta

The Leukemia and Lymphoma Society has tapped 5 new early-stage projects to back with up to $10 million each in fresh investments. The 5 biotechs are:

— Caribou, headed by Rachel Haurwitz and co-founded by Jennifer Doudna, is working on next-gen, off-the-shelf CAR-Ts to replace the patient-derived cells now in use.

— The LLS supported NexImmune’s IPO, helping fund its work on nanoparticles that can gin up an immune response directed at cancer cells. The biotech has 2 projects now in Phase I trials.

Jenny Rooke (Genoa Ventures)

Ear­ly Zymer­gen in­vestor Jen­ny Rooke re­flects on 'chimeras' in biotech, what it takes to spot a $500M gem

When Jenny Rooke first heard of Zymergen back in 2014, she knew she was looking at something different and exciting. The Emeryville, CA biotech held the promise of blending biology and technology to solve a huge unmet need for cost-effective chemicals — of all things — and a stellar founding team to boot.

But back then, West Coast venture capitalists didn’t see in Zymergen the one thing they were looking for in a winning biotech: therapeutic potential. Rooke, however, saw an opportunity and made her bets. Seven years later, that bet is paying off in a big way.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 107,400+ biopharma pros reading Endpoints daily — and it's free.