Ab­b­Vie shares tank on Ro­va-T flop, sud­den re­treat from ac­cel­er­at­ed ap­proval pitch

Ab­b­Vie’s stock $AB­BV is get­ting ham­mered this morn­ing af­ter its $10 bil­lion Ro­va-T pro­gram post­ed poor mid-stage re­sults for third-line small cell lung can­cer, forc­ing the com­pa­ny to scut­tle any plans to seek an ac­cel­er­at­ed ap­proval for the close­ly-watched drug af­ter talks with skep­ti­cal reg­u­la­tors.

Af­ter fan­ning hopes — amid con­sid­er­able skep­ti­cism in some cir­cles — with its pro­jec­tions of a mega-block­buster fu­ture for Ro­va-T, in­ves­ti­ga­tors out­lined a 16% ob­jec­tive re­sponse rate and me­di­an over­all sur­vival of 5.6 months for the drug.

Shares cratered on the terse re­lease, free falling 13% and wip­ing more than $20 bil­lion off its mar­ket cap, which start­ed the day at $178 bil­lion.

Michael Sev­eri­no

The mag­ni­tude of the ORR for the DLL3-high group was dis­mal, leav­ing the fu­ture of the drug in doubt. And by im­pli­ca­tion, some an­a­lysts are won­der­ing just how bad the da­ta are for the full group, ex­pect­ing the fi­nal read out to be even worse.

I queried Ab­b­Vie about the break­down, and a spokesper­son con­firmed the re­sults were for the sub­group.

The da­ta re­port­ed to­day were in pa­tients ex­press­ing high DLL3 com­pris­ing 74 per­cent of the over­all study pop­u­la­tion in TRIN­I­TY.

By fo­cus­ing on DLL3-ex­press­ing can­cers, the mar­ket was ex­pect­ing an ORR in the 40% range. Chemo com­bos are in the 20% area, an­a­lysts note, and IO com­bos are al­so demon­strat­ing ef­fi­ca­cy. So with an IRC as­sess­ment at 16%, Ab­b­Vie is well be­hind the 8 ball, with the FDA chill­ing talk of an ac­cel­er­at­ed ap­proval de­spite the fact that there are no ap­proved op­tions.

Se­vere ad­verse events were record­ed for throm­bo­cy­tope­nia (11%), pho­to­sen­si­tiv­i­ty re­ac­tion (7%) and pleur­al ef­fu­sion (5%).

Ab­b­Vie paid $5.8 bil­lion in cash for this drug, promis­ing up to $4 bil­lion more in mile­stones to ac­quire the lit­tle-known biotech uni­corn Stem­cen­trx. And with mon­ey like that on the ta­ble, ex­pec­ta­tions were run­ning high, fu­el­ing fore­casts that the phar­ma com­pa­ny could get past the even­tu­al loss of patent pro­tec­tion on Hu­mi­ra in style. Ab­b­Vie it­self pro­ject­ed peak sales at $5 bil­lion a year.

To­day, Ab­b­Vie is feel­ing the lash of dis­ap­point­ed an­a­lysts. Here’s Ge­of­frey Porges:

On­col­o­gy is the key growth busi­ness seg­ment for Ab­b­Vie af­ter the loss of ex­clu­siv­i­ty for Hu­mi­ra in 2023, and to­day’s re­sults and reg­u­la­to­ry de­ci­sion call in­to ques­tion the vi­a­bil­i­ty of the com­pa­ny’s cur­rent sol­id tu­mor strat­e­gy.

Com­bined with Bris­tol-My­ers’ Op­di­vo and Yer­voy, Ab­b­Vie was bet­ting that it had a win­ner in small cell lung can­cer, though its first cut of the ear­ly da­ta last year failed to im­press any­one out­side of the com­pa­ny. The drug drops a cy­to­tox­ic bomb right on DLL3-ex­press­ing can­cer cells com­mon in SCLC.

“We con­tin­ue to be­lieve Ro­va-T has po­ten­tial for pa­tients with small cell lung can­cer and oth­er DLL3-ex­press­ing can­cers,” said Mike Sev­eri­no, ex­ec­u­tive vice pres­i­dent of re­search and de­vel­op­ment and chief sci­en­tif­ic of­fi­cer, Ab­b­Vie. “Al­though the re­sults from the study were not what we hoped for, we look for­ward to re­ceiv­ing da­ta from the on­go­ing Phase 3 stud­ies in the first- and sec­ond-line set­tings and re­main com­mit­ted to de­vel­op­ing Ro­va-T for the treat­ment of pa­tients with small cell lung can­cer.”

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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So what hap­pened with No­var­tis' gene ther­a­py group? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

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Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Volker Wagner (L) and Jeff Legos

As Bay­er, No­var­tis stack up their ra­dio­phar­ma­ceu­ti­cal da­ta at #ES­MO21, a key de­bate takes shape

Ten years ago, a small Norwegian biotech by the name of Algeta showed up at ESMO — then the European Multidisciplinary Cancer Conference 2011 — and declared that its Bayer-partnered targeted radionuclide therapy, radium-223 chloride, boosted the overall survival of castration-resistant prostate cancer patients with symptomatic bone metastases.

In a Phase III study dubbed ALSYMPCA, patients who were treated with radium-223 chloride lived a median of 14 months compared to 11.2 months. The FDA would stamp an approval on it based on those data two years later, after Bayer snapped up Algeta and christened the drug Xofigo.

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Mi­rati tri­umphs again in KRAS-mu­tat­ed lung can­cer with a close­ly watched FDA fil­ing now in the cards

After a busy weekend at #ESMO21, which included a big readout for its KRAS drug adagrasib in colon cancer, Mirati Therapeutics is ready to keep the pressure on competitor Amgen with lung cancer data that will undergird an upcoming filing.

In topline results from a Phase II cohort of its KRYSTAL-1 study, adagrasib posted a response rate of 43% in second-line-or-later patients with metastatic non-small cell lung cancer containing a KRAS-G12C mutation, Mirati said Monday.

When ef­fi­ca­cy is bor­der­line: FDA needs to get more con­sis­tent on close-call drug ap­provals, agency-fund­ed re­search finds

In the exceedingly rare instances in which clinical efficacy is the only barrier to a new drug’s approval, new FDA-funded research from FDA and Stanford found that the agency does not have a consistent standard for defining “substantial evidence” when flexible criteria are used for an approval.

The research comes as the FDA is at a crossroads with its expedited-review pathways. The accelerated approval pathway is under fire as the agency recently signed off on a controversial new Alzheimer’s drug, with little precedent to explain its decision. Meanwhile, top officials like Rick Pazdur have called for a major push to simplify and clarify all of the various expedited pathways, which have grown to be must-haves for sponsors of nearly every newly approved drug.

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Ted White, Verrica CEO

Ver­ri­ca hits an­oth­er bump in the road with CMO re­lat­ed let­ter from FDA

The FDA has rejected Verrica’s new drug application for VP-102 again, with the company pinning the CRL on problems at a CMO that it was partnered with, the company announced Monday.

The FDA didn’t raise issues that directly relate to the manufacturing of VP-102, the company said, but raised “general quality issues” at the CMO’s facility. There were also no clinical concerns, it said, or need to collect more data.