By this point, the nov­el­ty of the FDA’s break­through ther­a­py des­ig­na­tion has large­ly been worn away by a drum­beat of an­nounce­ments for new ad­di­tions to the pro­gram. But As­traZeneca and Am­gen just land­ed one that de­serves a clos­er look.

Bahi­ja Jal­lal

Reg­u­la­tors have hand­ed the big league part­ners VIP sta­tus for a se­vere asth­ma drug called teze­pelum­ab. As al­ways, BTD gives them ready ac­cess to FDA in­sid­ers who are now pledged to help push this through to a speedy con­clu­sion. And we know now that that promise is worth some­thing.

What makes this drug a stand­out is its abil­i­ty to tar­get thymic stro­mal lym­phopoi­etin — TSLP – an up­stream mod­u­la­tor of mul­ti­ple in­flam­ma­to­ry path­ways like IL-4, IL-5 and IL-13.

In a Phase IIb read­out, treat­ment-re­sis­tant pa­tients demon­strat­ed re­duc­tions of 61% to 71% in their an­nu­al ex­ac­er­ba­tion rate. And that has ex­ecs at both of the phar­ma com­pa­nies stoked that this drug could be worth bil­lions if it reach­es the mar­ket.

Bahi­ja Jal­lal, who runs As­traZeneca’s big sub­sidiary Med­Im­mune, has called the re­sults “un­prece­dent­ed,” and Leerink’s Sea­mus Fer­nan­dez pegged peak po­ten­tial sales at $4.5 bil­lion. That could ar­rive soon­er than lat­er, he added, de­pend­ing on the FDA’s will­ing­ness to let this study stand as a reg­is­tra­tional land­mark that would al­low for a con­fir­ma­to­ry Phase III to of­fer enough ev­i­dence wor­thy of ap­proval.

Asth­ma, though, is not can­cer. Late-stage pro­grams in the field of­ten run in­to years. And there’s no fi­nal word from the com­pa­nies on their Phase III pro­gram, or whether reg­u­la­tors will need to see da­ta from dose-rang­ing stud­ies be­fore they give their de­ci­sion. But the de­vel­op­ers’ chances of be­ing put on the in­side track just got bet­ter.

Both Am­gen and As­traZeneca — which has been stag­ger­ing through a set of fresh set­backs out­side of the on­col­o­gy group — could use a big OK. And while there are no guar­an­tees that reg­u­la­tors will play along here, they just took an im­por­tant step for­ward along the road to a po­ten­tial block­buster OK.

89bio said its drug was better than placebo at lessening fibrosis without worsening nonalcoholic steatohepatitis, or NASH, in two of three dose groups.

The San Francisco biotech said it thinks the Phase IIb data pave the way for a potential Phase III, following in the footsteps of another biotech in its drug class, Akero Therapeutics. To fund a late-stage study, CEO Rohan Palekar told Endpoints News 89bio “would need to raise additional capital,” with the company having about $188 million at the end of last year.

The National Institutes of Health has again declined to use so-called “march-in” rights to lower the price of Astellas and Pfizer’s prostate cancer drug Xtandi despite being invented at UCLA with grants from the US Army and NIH.

“Given the remaining patent life and the lengthy administrative process involved for a march-in proceeding, NIH does not believe that use of the march-in authority would be an effective means of lowering the price of the drug,” NIH told prostate cancer patients Robert Sachs and Clare Love, in a letter shared with Endpoints News. The institutes’ analyses found Xtandi “to be widely available to the public,” an indication that there was not a pressing need for the US to act.