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.

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

Land­mark Amy­lyx OK spurs de­bate; Some... pos­i­tive? Alzheimer's da­ta; Can­cer tri­al bot­tle­neck; Sanofi's CRISPR bet; 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.

After brief stops in Paris and Boston, John Carroll and the Endpoints crew are staying on the road in October with their return for a live/streaming EUBIO22 in London. The hybrid event fireside chats and panels on mRNA, oncology and the crazy public market. We hope you can join him there.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

Up­dat­ed: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

Up­dat­ed: Al­ny­lam re­in­forces APOL­LO-B patisir­an da­ta be­fore head­ing to the FDA

Weeks after uncorking some mostly positive data for patisiran in transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy, Alnylam is bolstering its package with new exploratory and subgroup data before shipping it off to regulators.

The RNAi drug maintained “generally consistent” benefits in efficacy and quality of life across several prespecified subgroups at month 12, Alnylam announced on Friday afternoon, including age, baseline tafamidis use, ATTR amyloidosis type, baseline six-minute walk test score and others.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

#AAO22: J&J’s first look at com­mon eye dis­ease port­fo­lio pads the case for PhII of gene ther­a­py

CHICAGO — While the later-stage drug developers in the geographic atrophy field are near the finish line, Johnson & Johnson’s Janssen is taking a more deliberate route, with a treatment that it hopes to be a one-time fix.

The Big Pharma will take its Hemera Biosciences-acquired gene therapy into a Phase II study later this year in patients with GA, a common form of age-related macular degeneration that impacts about five million people worldwide. To get there, Janssen touted early-stage safety data at the American Academy of Ophthalmology annual conference Saturday morning, half a day after competitors Apellis and Iveric Bio revealed their own more-detailed Phase III analyses.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

Jerome Durso, Intercept Pharmaceuticals CEO

In­ter­cep­t's OCA fails a PhI­II NASH tri­al, rais­ing fresh doubts about its years­long quest for an OK

Intercept Pharmaceuticals has run into another big setback in its yearslong quest to win an approval for OCA in NASH. The biotech put out word Friday morning that its Phase III REVERSE study failed the primary endpoint for the liver disease, sending its share price into a tailspin.

There was no significant improvement in fibrosis among the patients suffering from cirrhosis who were treated with obeticholic acid, with investigators hunting for a minimum 1-stage histological improvement in the disease after 18 months of therapy.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

#AAO22 conference in Chicago (Photo credit: Associate editor Kyle LaHucik)

#AAO22: Iver­ic Bio, un­de­terred by loom­ing PDU­FA for com­peti­tor, touts sub­group da­ta on GA drug

CHICAGO — While its competitor is on the cusp of likely securing the first FDA nod, Iveric Bio is trudging ahead with its potential treatment for geographic atrophy, an advanced form of AMD, and has new data to support its upcoming NDA filing.

The biotech said its drug was more favorable than sham across all subgroups in the second Phase III study of the investigational complement C5 protein inhibitor, performing similarly to a previous, smaller late-stage trial.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

#AAO22 conference in Chicago (Photo credit: Associate editor Kyle LaHucik)

#AAO22: In bid for first FDA nod in ge­o­graph­ic at­ro­phy, Apel­lis claims an­oth­er first in eye dis­ease field

CHICAGO — Eight weeks before patients and industry find out if the FDA approves the first treatment for geographic atrophy, an advanced form of age-related macular degeneration, the biotech behind the drug is out with some new data on a secondary endpoint.

In what study investigator Charles Wykoff called the “first direct evidence of function preservation by slowing GA growth” in an investigational treatment, Apellis Pharmaceuticals’ drug pegcetacoplan led to less loss of retinal sensitivity versus sham  within 250 microns on either side of the GA lesion over 24 months.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.