A pri­vate Cal­i­for­nia biotech is rais­ing some eye­brows on the fi­nal day of AACR22 as it re­port­ed in­ter­im da­ta for its ex­per­i­men­tal drug in com­bi­na­tion with Keytru­da.

Look­ing at 18 pa­tients with non-mus­cle-in­va­sive blad­der can­cer who are un­re­spon­sive to bacil­lus Cal­mette-Guérin (an FDA-ap­proved agent for blad­der car­ci­no­mas), CG On­col­o­gy found that 16 of the 18 achieved a com­plete re­sponse af­ter just three months. On top of this, 13 main­tained their re­sponse through six months, nine through nine months and eight af­ter a year — the study’s pri­ma­ry end­point.

That ini­tial three-month mark is good for an 89% CR rate. The two oth­er pa­tients did not re­spond to the ther­a­py at all, drop­ping out at three months and six months, re­spec­tive­ly, ac­cord­ing to a pre­sen­ta­tion from Mof­fitt Can­cer Cen­ter’s Roger Li.

Hap­py to share very promis­ing re­sults from CORE-1 tri­al com­bin­ing CG0070 and pem­bro for #BC­Gun­re­spon­sivedis­ease, pre­sent­ed by Dr. Uchio @ #AACR22! 14/16 CR, all 6 with durable re­sponse who reached 12mo! @garys­tein­bergmd @UroDocAsh @tbi­vala1 @pcvblack @Kua­nArthur @cgon­col­o­gy https://t.co/B2zPu­cUhYH

— Roger Li (@Uroger­liMD) April 12, 2022

CG On­col­o­gy’s Phase II study, like many ear­ly- and mid-stage tri­als in the on­col­o­gy space, is small and open-la­bel, and no firm con­clu­sions can be drawn just yet. The biotech is plan­ning on en­rolling 35 to­tal pa­tients for the study, and it’s pos­si­ble the re­sponse rate — and com­plete re­sponse rate — di­min­ish over time.

But the 89% fig­ure rep­re­sents a head-turn­ing num­ber nonethe­less, par­tic­u­lar­ly as Keytru­da monother­a­py achieved on­ly a 41% com­plete re­sponse rate in the study Mer­ck used to sup­port FDA ap­proval. The green light came back in Jan­u­ary 2020 for the same NIM­BC pa­tients who are BCG re­sis­tant and who are in­el­i­gi­ble for, or chose not to un­der­go, cys­tec­to­my.

CG On­col­o­gy al­so found no Grade 3 or high­er side ef­fects in the study so far, not­ing most of the ad­verse events in­clud­ed fre­quent or painful uri­na­tion, blood in the urine, blad­der spasms, fa­tigue and chills.

The ex­per­i­men­tal drug in ques­tion is known as CG0070, an on­colyt­ic im­munother­a­py us­ing a mod­i­fied ade­n­ovirus de­signed to de­stroy blad­der tu­mor cells through the de­fec­tive retinoblas­toma path­way, the biotech says. CG0070 con­tains a “GM-CSF trans­gene” that prompts an im­mune re­sponse af­ter the tu­mor cells rup­ture, re­leas­ing anti­gens and GM-CSF to be tar­get­ed.

It’s a sim­i­lar ap­proach to the one tak­en by Fer­Gene — both com­pa­nies used ade­n­ovirus vec­tors, but dif­fer­ent trans­genes to gar­ner the im­mune re­spons­es. CG On­col­o­gy will like­ly want to avoid Fer­Gene’s pit­falls, how­ev­er: Fer­Gene re­ceived a CRL on its drug due to sev­er­al man­u­fac­tur­ing and CMC is­sues, prompt­ing waves of lay­offs and C-suite de­par­tures.

CG On­col­o­gy has re­mained pri­vate since be­ing found­ed in 2010, and its most re­cent fundraise came in De­cem­ber 2020. The biotech put to­geth­er a $47 mil­lion Se­ries D to fund a Phase III NIM­BC monother­a­py study, as well as the Keytru­da com­bo tri­al.

In ad­di­tion to Mer­ck, CG On­col­o­gy has al­so part­nered with Bris­tol My­ers Squibb to pair CG0070 with Op­di­vo for mus­cle-in­va­sive blad­der can­cer and with Roche to at­tack sol­id tu­mors in com­bi­na­tion with Tecen­triq.

Dupixent, the blockbuster anti-inflammatory drug from Sanofi and Regeneron, has cleared a high-stakes Phase III study in chronic obstructive pulmonary disease, the companies announced Thursday morning.

If they hold up in a second, identical trial, the data pave the way for Dupixent to become the first biologic to treat patients whose COPD remains uncontrolled despite being on maximal standard-of-care inhaled therapy — the patient population studied in the pivotal program. The companies had spotlighted this as a key readout as they look to expand the Dupixent franchise and explore its full potential.

Novartis is calling off a UK-based trial for Leqvio in the primary prevention of cardiovascular events in patients with high cholesterol, the company confirmed on Wednesday.

The Swiss pharma giant made the decision after “careful evaluation,” a spokesperson told Endpoints News via email. The trial, dubbed ORION-17, was planned in partnership with England’s National Health Service (NHS) and was part of the company’s strategy to establish Leqvio as a standard of care in cardiovascular disease management.

89bio said its drug was better than placebo at lessening fibrosis without worsening nonalcoholic steatohepatitis, or NASH, in two of three dose groups.

The San Francisco biotech said it thinks the Phase IIb data pave the way for a potential Phase III, following in the footsteps of another biotech in its drug class, Akero Therapeutics. To fund a late-stage study, CEO Rohan Palekar told Endpoints News 89bio “would need to raise additional capital,” with the company having about $188 million at the end of last year.