Richard Paulson, Karyopharm CEO

An­a­lysts ques­tion Karyophar­m's PhI­II en­dome­tri­al can­cer da­ta as biotech will charge ahead to FDA

Karyopharm Ther­a­peu­tics tout­ed on Tues­day Phase III da­ta for a can­cer pro­gram it de­scribed as pos­i­tive, but which Wall Street viewed in a more neg­a­tive light.

The biotech re­port­ed topline re­sults for Xpovio’s piv­otal study in en­dome­tri­al can­cer, say­ing the drug re­duced the risk of dis­ease pro­gres­sion or death by 30% com­pared to place­bo. Karyopharm not­ed the pri­ma­ry end­point of me­di­an pro­gres­sion-free sur­vival came in at 5.7 months for the ac­tive arm and 3.8 months in place­bo.

But while the da­ta were tech­ni­cal­ly sta­tis­ti­cal­ly sig­nif­i­cant, the p-val­ue came in just un­der the 0.05 thresh­old typ­i­cal­ly used to de­ter­mine such mat­ters at p=0.0486. Karyopharm al­so re­leased no ad­di­tion­al da­ta on over­all sur­vival, ap­pear­ing to con­fuse an­a­lysts on the com­pa­ny’s fourth-quar­ter earn­ings call Tues­day morn­ing.

As a re­sult, Karyopharm shares were down about 21% in ear­ly Tues­day trad­ing.

Com­pa­ny ex­ecs faced sev­er­al ques­tions dur­ing the call ask­ing to clar­i­fy some of the da­ta points. An­a­lysts sought more an­swers on the spe­cif­ic num­ber of sur­vival events and dis­con­tin­u­a­tion rates in each arm of the study, whether the re­port­ed da­ta in­volved the “in­tent-to-treat” pop­u­la­tion and if the FDA agrees with PFS as an ap­prov­able end­point.

They al­so seemed be­fud­dled by Karyopharm’s de­ci­sion to re­port da­ta from a pre-spec­i­fied sub­group de­spite the lin­ger­ing con­cerns over the press re­lease da­ta. On the call, Karyopharm not­ed that while most of the an­swers would be pro­vid­ed at an up­com­ing med­ical con­fer­ence, all of Tues­day’s da­ta came from the ITT group and that reg­u­la­tors view PFS as the pri­ma­ry.

The sub­group in ques­tion dealt with 103 pa­tients who have “wild-type p53.” Karyopharm re­port­ed that in these pa­tients who re­ceived Xpovio, the re­duc­tion in the risk of dis­ease pro­gres­sion or death was 62% com­pared to place­bo, good for a p-val­ue of p=0.0006. Me­di­an PFS in this group was 13.7 months against 3.7 months in the con­trol group.

De­spite the an­a­lysts’ ques­tions, Karyopharm plans for an sN­DA sub­mis­sion in the first half of this year, and pro­vid­ed the same time­line for the med­ical con­fer­ence re­veal. The biotech is go­ing for the front-line main­te­nance set­ting in en­dome­tri­al can­cer.

Tues­day is not the first time Xpovio has faced con­tro­ver­sy. When Karyopharm was ini­tial­ly seek­ing ap­proval for re­lapsed or re­frac­to­ry mul­ti­ple myelo­ma a few years ago, the FDA ap­peared skep­ti­cal of both its safe­ty and ef­fi­ca­cy, and an ad­comm rec­om­mend­ed reg­u­la­tors re­ject it in Feb­ru­ary 2019. But that Ju­ly, the drug won ac­cel­er­at­ed ap­proval in the in­di­ca­tion.

Phase III re­sults re­port­ed in March 2020 con­firmed Xpovio’s clin­i­cal ben­e­fit in such pa­tients, and the drug has since been OK’ed in late-line dif­fuse large B-cell lym­phoma and an ear­li­er-line mul­ti­ple myelo­ma set­ting. Karyopharm is con­tin­u­ing to re­search the drug in these can­cers and myelofi­bro­sis, both as a monother­a­py and in com­bi­na­tion with oth­er agents.

Image courtesy of The Janssen Pharmaceutical Companies of Johnson & Johnson.

Pro­tect­ing the glob­al phar­ma­ceu­ti­cal in­no­va­tion ecosys­tem – what’s at stake?

We are living in a new era of healthcare that is rapidly advancing progress impacting patient outcomes and experiences. We’ve seen a remarkable pace of transformational innovation, applied research, and advanced clinical development over the last decade.

Despite this tremendous progress, there is much more work to be done, and patients are counting on us – now more than ever – to continue that momentum. At the heart of our industry is a focus on developing and delivering medicines for some of the world’s most challenging diseases, including those that have few or no effective treatments today.

Roger Perl­mut­ter lines up deals, fresh fund­ing at Eikon; Sec­ond RSV vac­cine ap­proved; Sev­er­al biotechs flash­ing red; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

As you come back to our website this weekend for ASCO news, don’t forget to check out our updated event lineup at BIO, which will cover everything from the current state of VC investing in biotech to top pharma R&D chiefs discussing how to make pipeline decisions.

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Roger Perlmutter, Eikon Therapeutics CEO

Roger Perl­mut­ter builds Eikon's pipeline with deal-mak­ing flur­ry, rais­ing $106M more

Eikon Therapeutics announced three business development deals on Thursday, effectively dropping in a pipeline of cancer drugs alongside more than $100 million in fresh funding.

The Hayward, CA-based company has become one of biotech’s richest startups since its 2019 founding, having raised nearly $775 million. It’s developing a massive, automated research approach built around Nobel Prize-winning microscope science to peer inside cells and watch proteins in action. After its Series B last year, PitchBook reported a $3.02 billion valuation. And while CEO Roger Perlmutter declined to comment on that figure, he said its first tranche of nearly $106 million in Series C funding is a “meaningful step-up to our Series B valuation.”

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Grail’s blood test charts path for di­ag­nos­ing pa­tients sus­pect­ed of hav­ing can­cer in large study: #AS­CO23

Grail’s vision is simple but bold. The blood testing company has long held that people are often diagnosed with cancer too late. If seemingly healthy people were screened for early signs of the disease before symptoms appear, they may be able to get more effective treatments that nip cancer in the bud.

That premise is the basis of Grail’s commercial blood test, Galleri, which searches for the genetic fingerprints of cancer in the blood. The test, launched in 2021, reaped $55 million in sales last year, but now the company is setting its sights on a new market: patients suspected of having cancer due to symptoms such as abdominal pain, rectal bleeding or unexplained weight loss. Rather than administering expensive scans or conducting invasive biopsies right away, Grail hopes doctors will consider a simple blood test.

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Novartis headquarters in Basel, Switzerland (Kyle LaHucik for Endpoints News)

No­var­tis’ Kisqali pre­vents breast can­cer from com­ing back for longer — but can it best Eli Lil­ly’s Verzenio? #AS­CO23

CHICAGO — Novartis’ CDK4/6 inhibitor Kisqali helped early breast cancer patients stay cancer-free for longer after surgery, according to interim study results presented at ASCO.

In a Phase III study, Kisqali was added on top of endocrine therapy — the current standard treatment for early breast cancer patients. Kisqali reduced the risk of disease relapse by 25% compared with endocrine therapy alone in patients with Stage II or III HR-positive, HER2-negative breast cancer.

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GSK pro­motes rou­tine im­mu­niza­tions for adults amid post-pan­dem­ic vac­cine back­slide

GSK launched a new initiative on Thursday and committed up to $1 million in grant funding to improve adult routine vaccination rates.

While the pandemic spotlight was trained on the race for novel Covid-19 vaccines, other routine vaccination rates plummeted, raising concerns that missed doses may put children and even some adults at risk of preventable diseases such as measles or shingles. The World Health Organization last year reported the largest drop in childhood vaccinations in roughly three decades.

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Tammie Denyse speaks up about Black women and breast cancer inequity in Gilead's first TikTok campaign. (Gilead Sciences)

Gilead joins Tik­Tok with on­col­o­gy aware­ness cam­paign fea­tur­ing di­verse group of can­cer ad­vo­cates

Gilead Sciences is taking over the opening page on TikTok for the next two weeks. A Gilead-sponsored video, featuring cancer advocates talking about equity and other issues, will show up as the landing page, called the “For You” page, for millions of TikTok watchers.

The cancer awareness campaign will begin on Monday and run for two weeks, a Gilead spokesperson told Endpoints News. The TikTok ad debut is timed around the ASCO medical conference, but the work is aimed more broadly at healthcare professionals, as well as people touched by cancer and people interested in advancing Black and general health equity.

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Stephen MacMillan, Hologic CEO (Photo by Riccardo Savi/Getty Images for Concordia Summit)

Il­lu­mi­na names Ho­log­ic CEO as new board mem­ber and chair

Illumina’s board appointed two new members, including Hologic CEO Stephen MacMillan as the non-executive chair, a move that followed a proxy fight that saw shareholders oust the company’s board chair.

The DNA sequencing company also appointed Scott Ullem, the CFO of Edwards Lifesciences, to the board, according to a company statement.

Illumina’s plans to add two new board members came as Carl Icahn waged a board proxy campaign culminating with shareholders electing his candidate, Andrew Teno, over board chair John Thompson. Illumina CEO Francis deSouza survived a threat to his board seat by securing more than twice the shareholder votes than his challenger. Another Illumina candidate, Robert Epstein, was also elected and remained on the board.

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Catal­ent makes ad­di­tion­al lay­offs at In­di­ana fa­cil­i­ty

Contract manufacturer Catalent is making more staff cuts at one of its locations in the US amid dramatic corrective actions it’s been taking over the past few months.

In an email to Endpoints News, a Catalent spokesperson confirmed the company is making “a number of personnel changes” at a manufacturing facility in Bloomington, IN. While a specific number was not given to Endpoints, several local media outlets, including Indiana Public Media and the Bloomington Herald-Times, have put the number of layoffs at 150. No postings have been made to the Indiana Department of Workforce Development’s WARN notice.

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