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.

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.

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.