David Zaccardelli, Verona CEO

Two years af­ter flop, Verona gets a win as it preps for piv­otal Phase III tri­als

Two years af­ter the lead for­mu­la­tion of Verona’s lead COPD drug whiffed in a Phase II study that knocked back their stock, an­oth­er for­mu­la­tion has hit across the board.

Verona said Tues­day that all four dos­es of an in­haler form of its com­pound en­sifen­trine sig­nif­i­cant­ly im­proved lung func­tion in a 40-per­son Phase II tri­al of the chron­ic con­di­tion. Most dos­es of the drug hit on all pri­ma­ry and sec­ondary end­points, with the ef­fect in­creas­ing with the dose.

On the pri­ma­ry end­point of forced ex­pi­ra­to­ry vol­ume (FEV1) — a stan­dard mea­sure of lung func­tion that tests how much breath one can force­ful­ly ex­hale in 1 sec­ond — pa­tients who re­ceived the high dose of the drug were able to ex­hale 326 more mL af­ter 7 days of treat­ment, when ad­just­ed for place­bo. On the low dose, that fig­ure was 205 mL. Both had P val­ues <0.0001.

Verona’s stock $VR­NA ticked up in re­sponse, ris­ing 11% from $7.90 to $8.78.

The com­pa­ny has no im­me­di­ate plans, though, on bring­ing the in­haler for­mu­la­tion in­to Phase III, a spokesper­son said. In­stead, ex­ec­u­tives are say­ing that it boosts the case for the neb­u­lized form — the one that failed in 2019 but has hit on oth­er stud­ies, in­clud­ing a much larg­er one — as they push in­to two large, pan­dem­ic-de­layed Phase III tri­als.

“We are very en­cour­aged by these com­pelling da­ta, which are con­sis­tent with re­sults from Phase 2 clin­i­cal tri­als with our neb­u­lized and DPI for­mu­la­tions of en­sifen­trine,” CEO David Za­c­cardel­li said in a state­ment. “All three in­haled for­mu­la­tions have demon­strat­ed sig­nif­i­cant im­prove­ments in lung func­tion in COPD pa­tients, sup­port­ing the broad util­i­ty of en­sifen­trine de­liv­ered via neb­u­liz­ers and hand­held in­halers.”

Verona, found­ed in 2005 and built sole­ly around en­sifen­trine, ar­gues that the mol­e­cule is the first that can both widen pa­tients’ air­ways and re­duce in­flam­ma­tion. In March 2018, they an­nounced that, in a Phase IIb study of 400 pa­tients, the neb­u­liz­er form beat place­bo at im­prov­ing lung func­tion.

In 2019, the drug failed to sig­nif­i­cant­ly im­prove lung func­tion in a three-day study when giv­en on top of cur­rent COPD drugs. Last Jan­u­ary, 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.

Neb­u­liz­ers are elec­tric de­vices that re­lease pul­monary drugs as a mist and can of­fer some ad­van­tages over tra­di­tion­al in­halers.

The Verona spokesper­son said they don’t plan on adding the in­haler for­mu­la­tion to the ex­ist­ing tri­als. In­stead, they could look to ap­ply it down the road to oth­er in­di­ca­tions, such as cys­tic fi­bro­sis, or part­ner it out.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Lat­est on ul­tra-rare dis­ease ap­proval; Pos­i­tive, if mixed, signs for Bio­gen's ALS drug; Clay Sie­gall finds a new job; and more

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Over the last four years, we’ve honored 80 women whose extraordinary accomplishments have changed the game in biopharma R&D. You can now nominate someone to be highlighted in this year’s special report. Details are here.

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FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

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FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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Sijmen de Vries, Pharming CEO

FDA ap­proves Pharm­ing drug for ul­tra-rare im­mun­od­e­fi­cien­cy dis­ease

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.

No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

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Ly­me vac­cine test com­ple­tion is pushed back by a year as Pfiz­er, Val­ne­va say they'll ad­just tri­al

Valneva and Pfizer have adjusted the end date for the Phase III study of their investigational Lyme disease vaccine, pushing it back by a year after issues at a contract researcher led to thousands of US patients being dropped from the test.

In a March 20 update to clinicaltrials.gov, Valneva and Pfizer moved the primary completion date on the trial, called VALOR, from the end of 2024 to the end of 2025.

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Zhi Hong, Brii Biosciences CEO

Brii Bio­sciences stops man­u­fac­tur­ing Covid-19 an­ti­body com­bo, plans to with­draw EUA re­quest

Brii Biosciences said it will stop manufacturing its Covid-19 antibody combination, sold in China, and is working to withdraw its emergency use authorization request in the US, which it started in October 2021.

The Beijing and North Carolina biotech commercially launched the treatment in China last July but is now axing the work and reverting resources to other “high-priority programs,” per a Friday update. The focus now is namely hepatitis B viral infection, postpartum depression and major depressive disorders.

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