J&J ex­ecs be­lieve they have found their next big can­cer drug.

Ear­ly Mon­day the phar­ma gi­ant an­nounced that it was pay­ing Bel­gium’s ar­genx $500 mil­lion — $300 mil­lion as an up­front and $200 mil­lion for eq­ui­ty — to grab the world­wide com­mer­cial rights to cusat­uzum­ab (ARGX-110) to treat acute myeloid leukemia and high-risk myelodys­plas­tic syn­dromes. The deal al­so comes with $1.3 bil­lion in mile­stones.

The deal adds to J&J’s grow­ing im­muno-on­col­o­gy group, adding an an­ti-CD70 check­point strat­e­gy to the mix. Ar­genx’s share price $ARGX spiked 10% on the news in pre-mar­ket trad­ing.

Yus­ri El­sayed

The Bel­gian biotech flagged stel­lar — but very ear­ly-stage — da­ta for this drug in the lead-up to ASH last week. In the Phase I por­tion of the study re­searchers tracked a 92% re­sponse rate and a stun­ning 42% com­plete re­sponse with min­i­mal resid­ual re­sponse among 12 pa­tients.

That’s all very promis­ing, but it al­so cov­ers on­ly 12 pa­tients. J&J will al­so now co-de­vel­op the drug with ar­genx, throw­ing its am­bi­tious on­col­o­gy R&D group in­to the fray.

In the deal — which J&J’s af­fil­i­ate Cilag inked — ar­genx is keep­ing co-pro­mo­tion rights in the US.

This new deal fol­lows a re­cent J&J deal with Chi­na’s Leg­end Biotech on their BC­MA CAR-T, in which J&J pitched in $350 mil­lion in cash. That fol­lowed an­oth­er star turn, which was al­so com­plete­ly un­ex­pect­ed, at AS­CO in 2017. We’re sched­uled to see more da­ta on that drug lat­er to­day.

J&J has done a string of big can­cer drug deals with block­buster up­fronts like this. One of them — apa­lu­tamide (ARN-509) for pre-metasta­t­ic prostate can­cer — is now a star late-stage ther­a­py. J&J picked up this drug with its $1 bil­lion deal for Aragon. These new drugs were part of one leg of the com­pa­ny’s three-leg strat­e­gy for grow­ing rev­enue, with a promise that it can im­prove sig­nif­i­cant­ly on ex­ist­ing drugs — like Ste­lara, In­vokana and Xarel­to — while beef­ing up on a new core fo­cus on pul­monary ar­te­r­i­al hy­per­ten­sion through the Acte­lion buy­out.

“We be­lieve CD70 is an im­por­tant tar­get in the bi­ol­o­gy of se­lect can­cers, and we are ea­ger to ac­cel­er­ate the de­vel­op­ment of this in­no­v­a­tive an­ti­body to­geth­er with ar­genx,” said Yus­ri El­sayed, vice pres­i­dent, hema­to­log­ic ma­lig­nan­cies dis­ease area leader, Janssen Re­search & De­vel­op­ment. “Phase 1/2 da­ta in acute myeloid leukemia showed the ac­tiv­i­ty of cusat­uzum­ab, and we hope to trans­late these find­ings to im­prove out­comes for pa­tients with myeloid ma­lig­nan­cies.”

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.