Ab­b­Vie’s promised rheuma­toid arthri­tis block­buster upadac­i­tinib hits its marks in an­oth­er PhI­II — this time with­out any deaths

Ab­b­Vie $AB­BV has post­ed an­oth­er pos­i­tive batch of Phase III da­ta on its JAK1 in­hibitor upadac­i­tinib, hit­ting the pri­ma­ry and key sec­ondary end­points in their piv­otal pro­gram for rheuma­toid arthri­tis. And this round of da­ta comes with­out any new deaths in the drug arm to rat­tle in­vestors and an­a­lysts.

Their drug — for­mer­ly ABT-494 — hand­i­ly outscored a place­bo, which was not un­ex­pect­ed. The ther­a­py bare­ly edged out Hu­mi­ra on ACR 20 at 12 weeks, but did much bet­ter on a range of end­points like ACR50 (45% ver­sus 29%) and clin­i­cal re­mis­sion rates (29% ver­sus 18%). On­ly 6% of the place­bo arm ex­pe­ri­enced clin­i­cal re­mis­sion.

Ab­b­Vie has been spot­light­ing this JAK1 pro­gram as one of its two top late-stage drug can­di­dates with a mega-block­buster fu­ture ahead for it. While their cash cow Hu­mi­ra has been freed of the threat of biosim­i­lar com­pe­ti­tion to 2023, ri­vals are muscling in on its ter­ri­to­ry. The on­ly way Ab­b­Vie achieves big­ger rev­enue on Hu­mi­ra each year is by in­creas­ing its price.

An­a­lysts fol­low­ing the drug breathed an au­di­ble sigh of re­lief at this lat­est post. Ge­of­frey Porges not­ed:

The to­tal clin­i­cal ex­pe­ri­ence of upadac­i­tinib has now in­creased sub­stan­tial­ly with­out demon­strat­ing a ma­jor risk for VTEs, which we be­lieve re­duces the risk of un­ex­pect­ed as­set fail­ure. Af­ter the re­cent fail­ure of Ro­va-T in third-line small cell lung can­cer, and the re­lat­ed drop in Ab­b­Vie’s stock val­ue of >20%, the risk of a sub­se­quent pipeline fail­ure now al­so ap­pears sub­stan­tial­ly re­duced.

It’s a busy field. A Sanofi/Re­gen­eron team won an ap­proval for their IL-6 drug sar­ilum­ab (Kevzara) last year, as Gilead push­es ahead on their JAK1 fil­go­tinib af­ter Ab­b­Vie aban­doned it in fa­vor of its in-house pro­gram for upadac­i­tinib. Eli Lil­ly is back up for a re­view of baric­i­tinib, mean­while, af­ter the FDA re­versed a de­ci­sion to re­ject the drug with­out more da­ta. But J&J was per­ma­nent­ly shunt­ed aside re­cent­ly af­ter the FDA spurned its ap­pli­ca­tion on sirukum­ab.

If it can gain a new ap­proval on this drug, Ab­b­Vie ex­ecs are promis­ing peak sales in the range of $6.5 bil­lion a year, even though it faces plen­ty of com­pe­ti­tion.

Ab­b­Vie star­tled in­vestors with news of the death of two pa­tients in one study. And more re­cent­ly in­ves­ti­ga­tors had to ex­plain that an­oth­er pa­tient in the low-dose arm died of a he­m­or­rhag­ic stroke caused by a rup­tured aneurysm while there was al­so a case of pul­monary em­bolism in the same group. That fol­lowed a re­port in Sep­tem­ber that a pa­tient tak­ing a 30 mg dose ex­pe­ri­enced heart fail­ure and pre­sumed pul­monary em­bolism. In the most re­cent cas­es, both pa­tients had al­ready ex­hib­it­ed risks for these con­di­tions, ac­cord­ing to Ab­b­Vie.

Ab­b­Vie has al­so been ham­mered for some as­ton­ish­ing­ly bad da­ta on Ro­va-T, a can­cer drug it ac­quired in a jaw-drop­ping, $10.2 bil­lion buy­out deal for Stem­cen­trx two years ago.

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Gilead re­leas­es an­oth­er round of murky remde­sivir re­sults

A month after the NIH declared the first trial on remdesivir in Covid-19 a success, Gilead is out with new results on their antiviral. But although the study met one of its primary endpoints, the data are likely to only add to a growing debate over how effective the drug actually is.

In a Phase III trial, patients given a 5-day dose of remdesivir were 65% more likely to show “clinical improvement” compared to an arm given standard-of-care. The trial, though, gave little indication for whether the drug had an impact on key endpoints such as survival or time-to-recovery. And in a surprising twist, a 10-day dosing arm of remdesivir didn’t lead to a statistically significant improvement over standard of care.

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Fangliang Zhang (Imaginechina via AP Images)

The big mon­ey: Poised to make drug R&D his­to­ry, a Chi­na biotech un­veils uni­corn rac­ing am­bi­tions in a bid to raise $350M-plus on Nas­daq

Almost exactly three years after Shanghai-based Legend came out of nowhere to steal the show at ASCO with jaw-dropping data on their BCMA-targeted CAR-T for multiple myeloma, the little player with Big Pharma connections is taking a giant step toward making it big on Wall Street. And this time they want to seal the deal on a global rep after staking out a unicorn valuation in what’s turned out to be a bull market for biotech IPOs — in the middle of a pandemic.

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Len Schleifer (left) and George Yancopoulos, Regeneron (Vimeo)

Eyes on he­mo­phil­ia prize, Re­gen­eron adds a $100M wa­ger on joint de­vel­op­ment cam­paign with In­tel­lia

When George Yancopoulos first signed up Intellia to be its CRISPR/Cas9 partner on gene editing projects 4 years ago, the upstart smartly ramped up its IPO at the same time. Today, Regeneron $REGN is coming back in, adding $100 million in an upfront fee and equity to significantly boot up a whole roster of new development projects.

And they’re highlighting some clinical hemophilia research plans in the process.

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Jean-Jacques Bienaimé, BioMarin chairman and CEO

Bio­Marin holds the line on bleeds with 4-year val­rox up­date on he­mo­phil­ia A — but what's this about an­oth­er de­cline in Fac­tor 8 lev­els?

BioMarin has posted some top-line results for their 4-year followup on the most advanced gene therapy for hemophilia A — extending its streak on keeping a handful of patients free of bleeds and off Factor VIII therapy, but likely stirring fresh worries over a continued drop in Factor VIII levels.

We just don’t know how big a drop.

We’ll see more data when the results are presented at the World Federation of Hemophilia in a couple of weeks. But in a statement out Sunday night, BioMarin $BMRN reported that none of the patients required Factor VIII treatment, adding:

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As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Iterum's fu­ture looks un­cer­tain, af­ter lead an­tibi­ot­ic fails con­sec­u­tive piv­otal stud­ies

While the market for antibiotics remains in tatters — unlike many of its bankrupt (or at the brink of bankruptcy) peers — Iterum is suffering not because its antibiotic isn’t selling, but because the compound has now failed back-to-back late-stage studies.

The experimental drug, sulopenem, was designed to tackle drug-resistant infections with an outpatient focus (in addition to hospitals), to avert those reimbursement challenges that incentivize hospitals to prescribe cheaper, generic broad-spectrum antibiotics.

Covid-19 roundup: Did in­sid­ers cash in on pos­i­tive news re­port about Gilead be­fore pub­li­ca­tion?

A series of bullish trades on Gilead options just before the release of a favorable news story is raising questions among regulatory experts, Reuters reported.

On April 16, just hours before STAT published anecdotes from a Chicago hospital that served as one of the clinical sites to test Gilead’s remdesivir in Covid-19 patients, the California-based company’s shares were trading at around $75. Four large blocks of options were purchased for about $1.5 million each, betting that the stock would rise beyond that to as much as $87.5 by mid-August.

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Roche nabs front­line OK for Avastin/Tecen­triq in com­mon liv­er can­cer, best­ing an old Bay­er drug

For the first time in 12 years, the FDA has approved a new frontline treatment for the most common form of liver cancer.

The agency okayed a combination of Roche’s anti-VEGF antibody Avastin and their immunotherapy Tecentriq for patients with unresectable or metastatic hepatocellular carcinoma (HCC). The approval comes two weeks after Roche and their big biotech sub Genentech published Phase III results showing the combo improved both progression-free survival and, crucially, helped patients live longer than the long-running standard-of-care, Bayer’s Nexavar.