With eyes on first allogeneic CAR-T approval, Precision touts 100% response in ‘small area of interest’ inhabitants – Endpoints News

Pre­ci­sion Bio­Sciences has grand reg­u­la­to­ry am­bi­tions for its lead pro­gram: first al­lo­gene­ic CAR-T ther­a­py on the mar­ket.

En route to meet­ing with the FDA lat­er this year on po­ten­tial reg­is­tra­tion strat­e­gy, the North Car­oli­na biotech is out with new in­ter­im Phase I/IIa clin­i­cal da­ta on its prized pos­ses­sion, dubbed PB­CAR0191. The an­ti-CD19 can­di­date is be­ing test­ed in pa­tients with re­lapsed or re­frac­to­ry ag­gres­sive lym­phomas.

All 11 evalu­able CAR-T re­lapsed pa­tients re­spond­ed to the ther­a­py as of a May 31 da­ta cut­off, the biotech said Wednes­day morn­ing. Eight of the 11 pa­tients had a com­plete re­sponse at day 28. The da­ta come from six pa­tients on dose lev­el 3 and an­oth­er six in a co­hort added this year at dose lev­el 4. One of the 12 pa­tients died at day 23 due to “sus­pect­ed flu­dara­bine-as­so­ci­at­ed neu­ro­tox­i­c­i­ty,” the biotech said.

“Is this a small niche of pa­tients? Why fo­cus on the re­lapsed CAR-T pop­u­la­tion? The short­est an­swer I can pro­vide is pa­tients need this ur­gent­ly and the time has nev­er been more pre­car­i­ous,” CEO Michael Amoroso said on an in­vestor call. “The CAR-T re­lapsed pop­u­la­tion is ex­pect­ed to grow four- to five-fold over the next few years.”

Three of six evalu­able pa­tients re­spond­ed for at least six months, and six of the 11 pa­tients were still re­spond­ing as of the cut­off date. Sev­en of 10 evalu­able pa­tients had a pro­gres­sion free sur­vival of at least two months, the biotech said.

Pre­ci­sion ex­pects to start con­ver­sa­tions with the FDA in the third quar­ter and gen­er­ate da­ta on an­oth­er “three, four or five pa­tients,” Amoroso said on the in­vestor call. The com­pa­ny’s stock $DTIL was up about 9.5% be­fore the open­ing bell Wednes­day morn­ing.

Where­as au­tol­o­gous CAR-T ther­a­pies take cells di­rect­ly from the pa­tient, Pre­ci­sion’s ther­a­py takes T cells from donors and us­es gene edit­ing to mod­i­fy them. Au­tol­o­gous CAR-T ther­a­pies have racked up mul­ti­ple ap­provals in re­cent years — Gilead and Kite’s Yescar­ta and Tecar­tus, No­var­tis’ Kym­ri­ah, Bris­tol My­ers Squibb’s Breyanzi and Leg­end-J&J’s Carvyk­ti — but al­lo­gene­ic CAR-T has not se­cured reg­u­la­to­ry vic­to­ry yet.

Amoroso was Kite’s chief com­mer­cial of­fi­cer dur­ing the ear­ly days of the Yescar­ta mar­ket roll­out and joined Pre­ci­sion last au­tumn.

Fur­ther down the pipeline, Pre­ci­sion’s sec­ond gen­er­a­tion an­ti-CD19 al­lo­gene­ic CAR-T is ex­pect­ed to re­sume dos­ing in the third quar­ter. The com­pa­ny pre­vi­ous­ly paused dos­ing to op­ti­mize man­u­fac­tur­ing in the first quar­ter. New clin­i­cal tri­al ma­te­r­i­al is slat­ed to be re­leased soon so the dose lev­el 2 group can be­gin, the biotech said.

And the com­pa­ny is al­so at­tempt­ing to com­pete against Carvyk­ti, a re­cent­ly ap­proved CAR-T for pa­tients with re­lapsed or re­frac­to­ry mul­ti­ple myelo­ma. Pre­ci­sion is bet­ting it can best the biotech-Big Phar­ma duo by com­bin­ing its al­lo­gene­ic CAR-T, named PB­CAR269A, with Spring­Works Ther­a­peu­tics’ nirogace­s­tat. A Phase I/IIa study is mov­ing in­to dose lev­el 3.

The fo­cus on hu­man ther­a­pies comes af­ter a spin­out of a food and agri­cul­ture busi­ness, named Elo Life Sys­tems, at the end of 2021.