Alex­ion clinch­es aHUS ap­proval for Ul­tomiris as the clock ticks on Soliris con­ver­sion

Alex­ion has racked up a sec­ond ap­proval for Ul­tomiris, the suc­ces­sor ther­a­py to Soliris, as its main­stay block­buster ther­a­py faces a patent re­view process that could dras­ti­cal­ly short­en its patent ex­clu­siv­i­ty.

John Orloff

The FDA OK for atyp­i­cal he­molyt­ic ure­mic syn­drome (aHUS) on Fri­day was wide­ly ex­pect­ed af­ter Alex­ion post­ed a full slate of pos­i­tive Phase III da­ta in Jan­u­ary. But reg­u­la­tors al­so flagged con­cerns about se­ri­ous meningo­coc­cal in­fec­tions, slap­ping a black box warn­ing on the la­bel and man­dat­ing a REMS.

The late-stage re­sults, which in­clud­ed on­ly adults, were “broad­ly com­pa­ra­ble to ex­ist­ing Soliris da­ta in sim­i­lar pa­tient pop­u­la­tions, but with added con­ve­nience of every 8 week dos­ing,” SVB Leerink an­a­lysts Ge­of­frey Porges not­ed at the time.

Among 56 en­rolled, 53.6% demon­strat­ed a com­plete re­sponse af­ter 26 weeks, mea­sur­ing up to a 56% his­toric re­sponse rate for Soliris.

While an­oth­er Phase III in pe­di­atric and ado­les­cent pa­tients is still on­go­ing, the com­pa­ny dis­closed that in the ini­tial treat­ment pe­ri­od 71% of chil­dren demon­strat­ed a com­plete re­sponse. Out of 16 pa­tients, 14 were eval­u­at­ed for that in­ter­im analy­sis. The FDA went ahead and cleared it for use by in­fants as young as one month old, mark­ing the first pe­di­atric ap­proval for the drug.

Drugs for aHUS — a chron­ic, pro­gres­sive ul­tra-rare dis­ease that can have dev­as­tat­ing ef­fects on the kid­neys — are mea­sured by their abil­i­ties to clear blood clots in small blood ves­sels through­out the body, or throm­bot­ic mi­croan­giopa­thy (TMA).

Ul­tomiris works by in­hibit­ing un­con­trolled com­ple­ment ac­ti­va­tion, specif­i­cal­ly block­ing the C5 path­way. It’s al­so ap­proved in the US and Eu­rope as a ther­a­py for parox­ys­mal noc­tur­nal he­mo­glo­bin­uria.

“The con­se­quences of un­con­trolled com­ple­ment ac­ti­va­tion, like or­gan fail­ure and po­ten­tial­ly death, cre­ate sig­nif­i­cant chal­lenges and un­cer­tain­ty for peo­ple and fam­i­lies fac­ing aHUS,” said John Orloff, head of R&D, in a state­ment. “Based on the Phase 3 da­ta, which demon­strat­ed clin­i­cal­ly mean­ing­ful ben­e­fits in peo­ple with aHUS, we be­lieve UL­TOMIRIS has the po­ten­tial to be­come the new stan­dard of care for this dev­as­tat­ing dis­ease.”

Con­vinc­ing physi­cians and pa­tients would be cru­cial. In Au­gust, the US Patent Tri­als Ap­peal Board (PT­AB) agreed to con­duct an in­ter partes re­view on Soliris af­ter Am­gen, which is push­ing a biosim­i­lar to the com­ple­ment in­hibitor, launched an ef­fort to over­turn patents on the ac­tive in­gre­di­ent.

If Am­gen pre­vails, Soliris’ ex­clu­siv­i­ty could ex­pire as ear­ly as 2022. Cred­it Su­isse’s Mar­tin Auster has pre­vi­ous­ly mod­eled a rough­ly 80% con­ver­sion to Ul­tomiris in three to four years across the PNH and aHUS fran­chis­es.

The ba­ton is now in the com­mer­cial team’s hands.

Porges ex­pects US con­ver­sion ef­forts for Ul­tomiris to be­gin im­me­di­ate­ly, with the EU and Japan to fol­low next year. From his note out late Fri­day:

We es­ti­mate 2020 US Ul­tomiris aHUS rev­enues of ~$140mm, in­creas­ing to ~$640mm in 2025, rep­re­sent­ing ~72% of com­bined US Soliris/Ul­tomiris aHUS sales. We con­tin­ue to re­main op­ti­mistic about the prospects for Alex­ion’s com­ple­ment fran­chise and be­lieve fur­ther in­di­ca­tions of rapid con­ver­sion across in­di­ca­tions and ge­o­gra­phies, as well as fur­ther life-cy­cle man­age­ment op­por­tu­ni­ties from the Achillion (ACHN, MP) ac­qui­si­tion (as­sum­ing FTC clear­ance), could in­crease Alex­ion’s growth out­look over both the near- and longer-term; and po­ten­tial­ly draw in­vestors back in­to the stock over the next 6-12 months.

Why it Works: Man­u­fac­tur­ing a Vac­cine in a Mul­ti-Prod­uct Fa­cil­i­ty.

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