Autolus tips its hand, bags $220M as CAR-T showdown with Gilead looms – Endpoints News

The first batch of piv­otal da­ta on Au­to­lus Ther­a­peu­tics’ CAR-T is in, and ex­ecs are ready to plot a path to mar­ket.

With an over­all re­mis­sion rate of 70% at the in­ter­im analy­sis fea­tur­ing 50 pa­tients, the re­sults set the stage for a BLA fil­ing by the end of 2023, said CEO Chris­t­ian Itin.

Per­haps more im­por­tant­ly — giv­en that Au­to­lus’ drug, obe-cel, is go­ing af­ter an in­di­ca­tion that Gilead’s Tecar­tus is al­ready ap­proved for — the biotech high­light­ed “en­cour­ag­ing safe­ty da­ta” in the tri­al, with a low per­cent­age of pa­tients ex­pe­ri­enc­ing se­vere im­mune re­spons­es.

Au­to­lus sold $150 mil­lion worth of its stock im­me­di­ate­ly af­ter the read­out, as part of a pre­vi­ous­ly filed shelf reg­is­tra­tion. It is al­so col­lect­ing $70 mil­lion in mile­stone pay­ments from Black­stone per a $250 mil­lion col­lab­o­ra­tion and fi­nanc­ing deal they struck late last year.

The piv­otal Phase II tri­al, dubbed FE­LIX, en­rolled pa­tients with re­lapsed/re­frac­to­ry adult acute lym­phoblas­tic leukemia.

Jae Park, a prin­ci­pal in­ves­ti­ga­tor at Memo­r­i­al Sloan Ket­ter­ing Can­cer Cen­ter, called it an “un­der­served dis­ease set­ting” char­ac­ter­ized by “the ex­plo­sive growth of the leukemia and the poor con­di­tion of many pa­tients.”

Out of 50 pa­tients eval­u­at­ed for ef­fi­ca­cy, 70% went in­to ei­ther com­plete re­mis­sion or com­plete re­mis­sion with in­com­plete blood count re­cov­ery, as as­sessed by an in­de­pen­dent re­sponse re­view com­mit­tee. Among this group, obe-cel showed “com­pa­ra­ble ex­pan­sion and ini­tial per­sis­tence” to a pre­vi­ous Phase I study at me­di­an fol­low-up of 6.4 months, Au­to­lus re­port­ed, sug­gest­ing con­sis­tent da­ta.

When re­searchers look at all 92 pa­tients who have been dosed so far, 3% ex­pe­ri­enced grade 3 or high­er cy­tokine re­lease syn­drome, and 8% ex­pe­ri­enced grade 3 or high­er im­mune ef­fec­tor cell-as­so­ci­at­ed neu­ro­tox­i­c­i­ty syn­drome (ICANS), com­mon side ef­fects as­so­ci­at­ed with CAR-T ther­a­py. All told, 23% of pa­tients ex­pe­ri­enced any grade ICANS.

“We look for­ward to sup­ple­ment­ing this in­ter­im da­ta with longer fol­low up da­ta to more ful­ly ex­plore the clin­i­cal ben­e­fit of obe-cel,” said Itin, who leads a crew scat­tered across Lon­don, Mu­nich, Basel and Rockville, MD.

Shares of the com­pa­ny, though, plum­met­ed 27% to $2.17 in pre-mar­ket trad­ing, which Mizuho an­a­lyst Mara Gold­stein chalked up to the pub­lic of­fer­ing. She al­so not­ed over­all con­cerns about the com­mer­cial CAR-T mar­ket.

Obe-cel is de­signed to hit CD19, a fa­mil­iar tar­get in the CAR-T space, but Au­to­lus be­lieves that its en­gi­neer­ing ap­proach can lim­it T cell ac­ti­va­tion, keep­ing the im­mune sys­tem from go­ing hay­wire while al­so pre­vent­ing T cell ex­haus­tion.

In the r/r ALL set­ting, it will be go­ing up against Gilead’s Tecar­tus, which post­ed an ORR of 65% among 54 pa­tients at me­di­an fol­low-up of 12.3 months.

Au­to­lus plans to present more de­tails at a con­fer­ence in mid-2023.