David Zaccardelli, Verona Pharma CEO

Verona’s COPD drug shines in PhI­II study, po­ten­tial­ly clear­ing path to FDA — shares jump

UK-based Verona Phar­ma’s COPD drug, en­sifen­trine, has suc­ceed­ed in its Phase III tri­al, paving the way for a pos­si­ble FDA ap­proval.

In Verona’s Phase III EN­HANCE-2 study, rough­ly 800 pa­tients with mod­er­ate to se­vere COPD re­ceived en­sifen­trine or place­bo through a neb­u­liz­er twice a day for 24 weeks. At 12 hours post-treat­ment on week 12, the place­bo-cor­rect­ed change in forced ex­pi­ra­to­ry vol­ume (FEV1) — a stan­dard mea­sure of lung func­tion that tests how much breath one can force­ful­ly ex­hale in one sec­ond — was 94 mL, lead­ing the tri­al to meet its pri­ma­ry end­point.

The da­ta took in­vestors’ breath away, as Verona shares $VR­NA jumped more than 70% in ear­ly Tues­day trad­ing.

Ap­prox­i­mate­ly 52% of pa­tients had re­ceived back­ground COPD ther­a­py, ei­ther a long-act­ing mus­carinic an­tag­o­nist (LAMA) or a long-act­ing be­ta-ag­o­nist (LA­BA). Ad­di­tion­al­ly, ap­prox­i­mate­ly 15% of all sub­jects re­ceived in­haled cor­ti­cos­teroids (ICS) with con­comi­tant LAMA or LA­BA. How­ev­er, the re­sults of these spe­cif­ic sub­groups were not shared.

An­to­nio Anzue­to

“These da­ta, along with re­sults from our on­go­ing Phase 3 tri­al, EN­HANCE-1, which is on track to be re­port­ed around the end of 2022, if sim­i­lar­ly pos­i­tive, are ex­pect­ed to sup­port the sub­mis­sion of a New Drug Ap­pli­ca­tion to the US Food and Drug Ad­min­is­tra­tion in the first half of 2023,” said Verona’s CEO David Za­c­cardel­li in a press state­ment.

The study al­so met its sec­ondary end­point of im­prov­ing lung func­tion. At week 12, the peak FEV1 in the 0 to 4 hours af­ter dos­ing with en­sifen­trine was high­er than the place­bo by 146 mL. The place­bo-cor­rect­ed morn­ing trough FEV1 at week 12 was 49 mL.

While the study achieved its pri­ma­ry and sec­ondary end­points, the drug failed to beat place­bo on mea­sures of COPD symp­toms and qual­i­ty of life.

An­to­nio Anzue­to, a pro­fes­sor of med­i­cine and chief of pul­monary at South Texas Vet­er­ans Health­care Sys­tem, said in a re­lease:

En­sifen­trine has demon­strat­ed clear im­prove­ments in lung func­tion in ad­di­tion to fa­vor­able safe­ty re­sults. I am ex­treme­ly ex­cit­ed by the clin­i­cal­ly mean­ing­ful 42% re­duc­tion in the rate of ex­ac­er­ba­tions ob­served over 24 weeks in these symp­to­matic pa­tients, many re­ceiv­ing back­ground ther­a­py.

Verona has tout­ed en­sifen­trine, or RPL554, as the first po­ten­tial ther­a­py for res­pi­ra­to­ry dis­eases that acts as both a bron­chodila­tor and an­ti-in­flam­ma­to­ry agent in a soli­tary com­pound. The biotech, found­ed in 2005 and built sole­ly around en­sifen­trine, says the mol­e­cule is the first that can both widen pa­tients’ air­ways and re­duce in­flam­ma­tion.

But it hasn’t al­ways been smooth sail­ing. In 2019, the drug failed to sig­nif­i­cant­ly im­prove lung func­tion in a three-day study when giv­en on top of cur­rent COPD drugs. Last Feb­ru­ary, though, a longer study found the neb­u­lized form did im­prove lung func­tion when giv­en on top of ex­ist­ing drugs, and Verona was able to raise near­ly $200 mil­lion to launch a pair of piv­otal Phase III stud­ies, test­ing the drug both alone and in com­bi­na­tion.

Mean­while, oth­er re­cent drugs for COPD in­clude As­traZeneca’s FDA-ap­proved three-in-one in­haler Breztri Aeros­phere, which helps in the main­te­nance of COPD.

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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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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On­corus lays off most of its re­main­ing team, warns of wind-down as it takes one last shot at deal­mak­ing

Despite cutting its headcount, pipeline and lease late last year, Oncorus is still struggling to stay afloat and is now on the brink of bankruptcy or dissolution, the company revealed late Thursday.

The Andover, MA-based biotech is letting “substantially all of Oncorus’ workforce” go, after the board of directors approved the layoffs. CEO Ted Ashburn, COO/chief of staff Stephen Harbin and CMO John Goldberg are among the 55 to depart.

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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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