Lars Fruergaard Jørgensen, Novo Nordisk CEO (Chris Ratcliffe/Bloomberg via Getty Images)

Two years af­ter scrap­ping three he­mo­phil­ia tri­als, No­vo Nordisk plots route to FDA with new PhI­II da­ta

No­vo Nordisk con­tin­ues to move for­ward with its once-dai­ly mon­o­clon­al an­ti­body for he­mo­phil­ia A or B treat­ment aim­ing to stop ex­ces­sive bleed­ing events be­fore they hap­pen, tout­ing new Phase III da­ta Sun­day.

The Dan­ish phar­ma put out word that a piv­otal tri­al for con­cizum­ab achieved its pri­ma­ry goal, re­duc­ing spon­ta­neous and trau­mat­ic bleeds by 86% in a pro­phy­lac­tic set­ting com­pared to a con­trol arm. No­vo Nordisk is de­vel­op­ing the can­di­date such that he­mo­phil­ia A or B pa­tients can take it re­gard­less of whether they de­vel­op im­mune re­spons­es to oth­er drugs.

“One of the most crit­i­cal com­pli­ca­tions in the treat­ment of he­mo­phil­ia is the de­vel­op­ment of in­hibitors, as they ren­der stan­dard re­place­ment ther­a­py in­ef­fec­tive and se­vere­ly lim­it treat­ment op­tions for he­mo­phil­ia B,” lead in­ves­ti­ga­tor Vic­tor Jiménez-Yuste said in a state­ment.

Sun­day’s news marks a re­bound for con­cizum­ab, com­ing a lit­tle over two years af­ter No­vo ran in­to sig­nif­i­cant set­backs in the drug’s de­vel­op­ment. In March 2020, No­vo Nordisk halt­ed two Phase III stud­ies and one Phase II tri­al af­ter three pa­tients ex­pe­ri­enced non-fa­tal blood clots. The FDA al­so placed the pro­gram on clin­i­cal hold.

But No­vo restart­ed de­vel­op­ment af­ter find­ing a “new path for­ward” that Au­gust, the com­pa­ny said at the time, which in­clud­ed changes to the dos­ing reg­i­mens and fre­quen­cy of ad­min­is­tra­tion. With the Phase III read­out now in hand, No­vo plans to sub­mit its FDA pitch be­fore the end of this year.

The strat­e­gy has been to de­vel­op a treat­ment that can stop spon­ta­neous bleed­ing in pa­tients whether or not they are al­ready tak­ing re­place­ment ther­a­py and to de­vel­op an im­mune re­sponse known as in­hibitors. Con­cizum­ab is an an­ti-tis­sue fac­tor path­way in­hibitor (TF­PI) an­ti­body ad­min­is­tered once a day in an EpiPen-style in­jec­tion.

Re­searchers test­ed the drug in 133 boys and men aged 12 and over (No­vo says males rep­re­sent about 88% of he­mo­phil­ia cas­es). Pa­tients were ran­dom­ized 2-to-1 to ei­ther a pro­phy­lax­is arm or a non-pro­phy­lax­is arm and mea­sured for at least 32 or 24 weeks, re­spec­tive­ly.

No­vo com­pared the num­ber of treat­ed bleed­ing episodes, known as ABR, be­tween these two co­horts for the pri­ma­ry analy­sis. In the pro­phy­lax­is arm, the es­ti­mat­ed av­er­age ABR was 1.7, while in the non-pro­phy­lax­is arm the av­er­age ABR was 11.8. Ad­di­tion­al­ly, the over­all me­di­an ABR of con­cizum­ab was ze­ro, com­pared to 9.8 for no pro­phy­lax­is.

Safe­ty proved much bet­ter this time around, with No­vo say­ing the drug did not pro­duce any throm­boem­bol­ic events af­ter the treat­ment was restart­ed.

Al­hough No­vo is go­ing the an­ti­body route, many of the big-name he­mo­phil­ia play­ers have tried their hand at gene ther­a­pies in­stead. These pro­grams from Pfiz­er/Sang­amo and Bio­Marin go­ing af­ter he­mo­phil­ia A each have their own is­sues, how­ev­er, with the for­mer on­ly be­ing re­leased from clin­i­cal hold in May and the lat­ter earn­ing a CRL in 2020.

No­vo, though, is os­ten­si­bly try­ing to com­pete with Roche and Spark Ther­a­peu­tics’ block­buster drug Hem­li­bra, which pulled in about $870 mil­lion glob­al­ly in the first quar­ter this year. Hem­li­bra is on­ly ap­proved for he­mo­phil­ia A pa­tients with fac­tor VI­II in­hibitors, where­as No­vo’s drug is de­signed for ei­ther he­mo­phil­ia A or he­mo­phil­ia B.

Not to be out­done, Roche put out new Hem­li­bra da­ta ear­ly Mon­day morn­ing as well, say­ing the drug demon­strat­ed ef­fec­tive bleed con­trol in mild or mod­er­ate he­mo­phil­ia A pa­tients who don’t have fac­tor VI­II in­hibitors. Two-thirds of the study’s 72 pa­tients saw no bleed­ing events at a me­di­an fol­low-up of 55.6 weeks.

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

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Silviu Itescu, Mesoblast CEO

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Marc Dunoyer, Alexion CEO (AstraZeneca via YouTube)

Up­dat­ed: As­traZeneca nabs a small rare dis­ease gene ther­a­py play­er for 667% pre­mi­um

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