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.

Com­mu­ni­cat­ing the val­ue of pre­ci­sion med­i­cine

By Natasha Cowan, Content Marketing Manager at Blue Latitude Health.
Many stakeholders are confused by novel precision medicines, including patients and healthcare professionals. So, how can industry help them to navigate this complexity?

Precision medicine represents a new paradigm in healthcare. It embodies the shift from treating many patients with the same therapy, to having the tools to identify the best treatment for every patient.

(Image: Associated Press)

Amarin emerges from an ex­pert pan­el re­view with a clear en­dorse­ment for Vas­cepa and high odds of suc­cess when the FDA weighs in for­mal­ly

Several FDA experts who gathered Thursday to consider the landmark approval of Vascepa to reduce cardio events in an at-risk population voiced their unease about various aspects of the efficacy and safety data, or ultimately the population it should be used to treat. But the overwhelming belief that the data pointed to the drug’s benefit and clearly outweighed risks carried the day for Amarin.

The panel voted unanimously (16 to 0) to support the company’s positive data presentation — backing an OK for expanding the label to include reducing cardio risk. The vote points Amarin $AMRN down a short path to a formal decision by the FDA, with the odds heavily in its favor. Chances are the rest of the questions about the future of this drug will be hashed out in the label’s small print.

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What does $62B buy you these days? A lot, says Take­da ex­ecs as the phar­ma play­er promis­es a block­buster R&D fu­ture

First comes the $62 billion buyout. Then comes the asset auction and reorganization to pay down debt. Now comes the detailed pledge of a bigger, brighter future in drug development.

That’s where Takeda finds itself on R&D day today, about 11 months after closing on their Shire acquisition. R&D chief Andy Plump is joining CEO Christophe Weber and other top members of the team to outline a new set of priorities in the greatly expanded pipeline at Takeda, which has jumped into the top ranks of the world’s pharma giants in the wake of the Shire deal.

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BeiGene CEO John Oyler at an Endpoints event in Shanghai, October 2018 (Credit: Endpoints News/PharmCube)

UP­DAT­ED: Chi­na's BeiGene scores first-ever FDA ap­proval — but can they carve up J&J's block­buster fran­chise?

Weeks after Amgen took a $2.7 billion stake in BeiGene, the Beijing-based biotech has secured its first-ever FDA approval for zanubrutinib, a BTK inhibitor, months ahead of schedule.

BeiGene’s drug, branded as Brukinsa, has secured accelerated approval for adult patients with mantle cell lymphoma (MCL) — a typically aggressive, rare, form of blood cancer — who have received at least one prior therapy.

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Spe­cial re­port: Twen­ty ex­tra­or­di­nary women in bio­phar­ma R&D who worked their way to the top

What differentiates a woman leader in biopharma R&D from a man?

Not much, except there are fewer of them in senior posts. Data suggest women are not more risk-averse, family-oriented or less confident than their male counterparts — indeed the differences between the two sexes are negligible. But a glance at the top R&D positions in Big Pharma leaves little doubt that upward migration in the executive ranks of biopharma R&D is tough.

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GSK's asth­ma bi­o­log­ic Nu­cala scores in rare blood dis­or­der study

GlaxoSmithKline’s asthma drug Nucala, which received a resounding FDA rejection for use in chronic obstructive pulmonary disease (COPD) last year, has shown promise in a rare blood disorder.

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Mer­ck buys a fledg­ling neu­rode­gen­er­a­tive biotech spawned by an old GSK dis­cov­ery al­liance. What’s up with that?

Avalon Ventures chief Jay Lichter has a well-known yen for drug development programs picked up in academia. And what he found in Haoxing Xu’s lab at the University of Michigan pricked his interest enough to launch one of his umbrella biotechs in San Diego.

Xu’s work laid the foundation for Avalon to launch Calporta, which has been working on finding small molecule agonists of TRPML1 (transient receptor potential cation channel, mucolipin subfamily, member 1) for lysosomal storage disorders. And that pathway, they believe, points to new approaches on major market neurodegenerative diseases like Parkinson’s, ALS and Alzheimer’s.

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Carson Block. Muddy Waters via YouTube

Shorts ga­lore: Mud­dy Wa­ters sees slide for Pep­tiDream, tweets con­cerns about Fi­bro­Gen's new da­ta

The short seller Muddy Waters is taking aim at Japan’s most profitable biotech, projecting a slide for a company that has skyrocketed over the last four years. Meanwhile, the firm tweeted out an analysis accusing FibroGen of manipulating data to obscure safety concerns in their latest reveal, although some investors seem satisfied by the biotech’s explanation.

Muddy Waters shorted PeptiDream, a Japanese biotech-for-hire that leveraged its peptide library into partnerships with some of the world’s largest pharmaceutical companies, a 50% profit margin and $6 billion valuation. The firm noted that despite its esteem, PeptiDream has failed to bring a drug to market 13 years after its 2006 launch (although this is not especially rare for biotech).

Pin­cer move­ment: Cal­i­for­nia biotech gets $35M to suf­fo­cate can­cer in co­or­di­nat­ed at­tack

Having served in Afghanistan, the navy veteran leading California-based EpicentRx wants to leave no patient behind with his arsenal of anti-cancer drugs. On Thursday, the company was given a $35 million boost to further its mission.

The injection of funds will be used to shepherd its late-stage CD47 drug, RRx-001, to the FDA for marketing, and its oncolytic virus program into the clinic.

RRx-001, engineered as an agent that makes tumor cells more sensitive to therapy, is in a Phase III trial in combination with chemotherapy for use in third-line and beyond small cell lung cancer (SCLC). The drug has been granted orphan drug designation from FDA for SCLC, neuroendocrine cancer and glioblastoma.