Positive PhIII results in hand, Concert Pharma forges ahead with NDA and commercialization plans for hair loss drug – Endpoints News

Con­cert Phar­ma­ceu­ti­cals has been wait­ing to tout Section III da­ta amidst a sell-off of the majority of its pipeline to Ter­ran Bio­sciences and sev­er­al oth­er occasions for the biotech. And on Mon­day, the com­pa­ny rolled out the pink automobile­pet.

Con­cert un­veiled topline re­sults from its sec­ond Section III clin­i­cal tri­al, dubbed THRIVE-AA2, eval­u­at­ing its oral can­di­date cur­lease­ly referred to as CTP-543 in grownup pa­tients with mod­er­ate to se­vere alope­cia space­ta. The can­di­date is an in­hibitor of Janus ki­nas­es JAK1 and JAK2.

The pri­ma­ry ef­fi­ca­cy finish­level, the per­cent­age of pa­tients achiev­ing a cer­tain rating on an alope­cia sever­i­ty scale af­ter 24 weeks, was met with sta­tis­ti­cal sig­nif­i­cance in each the 8 mg twice-dai­ly and 12 mg twice-dai­ly dose teams rel­a­tive to position­bo. Deal with­ment of the can­di­date was al­so gen­er­al­ly well-tol­er­at­ed.

Ac­twine­ing to the com­pa­ny, these en­rolled have been re­quired to have not less than 50 per­cent scalp hair loss as mea­sured by the SALT scale (with a rating of 100 rep­re­despatched­ing com­plete scalp hair loss and ze­ro as no loss). The av­er­age base­line SALT rating throughout all pa­tients was ap­prox­i­mate­ly 87.9.

Sta­tis­ti­cal­ly sig­nif­i­cant professional­por­tions of pa­tients deal with­ed with ei­ther dose lev­els achieved a SALT rating of 20 or much less. Con­cert mentioned 38.3% hit the rating within the 12 mg twice-dai­ly dose group and 33% within the 8 mg twice-dai­ly dose group. The examine ul­ti­mate­ly web­ted a p-val­ue of p<0.0001 for each dos­es.

The pos­i­tive da­ta have been wel­come information to in­vestors, because the Mass­a­chu­setts biotech’s inventory value $CNCE rose over 6% since open­ing.

James Cas­sel­la

“Based mostly on the re­sults from our Section 3 professional­gram, we be­lieve CTP-543, if ap­proved, might be a best-in-class deal with­ment op­tion for alope­cia space­ta and have a dis­tinct ther­a­peu­tic professional­file with fea­tures which can be im­por­tant to pa­tients, in­clud­ing the per­cent­age of pa­tients with clin­i­cal­ly-mean­ing­ful hair re­progress, pa­tient sat­is­fac­tion lev­els, and hair re­progress charges,” mentioned Con­cert’s chief de­vel­op­ment of­fi­cer James Cas­sel­la.

On the again of the re­sults, Cas­sel­la mentioned the com­pa­ny is now look­ing to pre­pare an NDA sub­mis­sion to the FDA within the first half of subsequent 12 months in addition to plans to com­mer­cial­ize the drug.

Con­cert’s path to the FDA comes as Eli Lil­ly was the first across the line in June, as Olu­mi­ant (baric­i­tinib) web­ted FDA ap­proval in a once-dai­ly capsule in three dose lev­els, placing pres­positive on the biotech now to get ap­proval for its can­di­date.

Sec­ondary finish­factors in­clud­ed 47% of pa­tients within the 8 mg twice-dai­ly group and 52% of pa­tients within the 12 mg twice-dai­ly group say­ing they have been “sat­is­fied” or “very sat­is­fied” with the deal with­ment, com­pared to 2% of pa­tients within the place­bo group.

The can­di­date and the com­pa­ny it­self have been on a wild journey. In 2018, the drug’s da­ta ini­tial­ly didn’t maintain up in opposition to Pfiz­er’s can­di­date, however ex­ecs de­cid­ed to forge forward and obtained break­through ther­a­py des­ig­na­tion in 2020. CTP-543 is likely one of the com­pa­ny’s ma­jor hopes because the com­pa­ny hit a sig­nif­i­cant velocity bump last year when a Section II tri­al for its schiz­o­phre­nia professional­gram flopped, caus­ing the com­pa­ny to ax the pro­gram and its inventory to tank by 40%.