Check­point in­hibitors like Keytru­da and Op­di­vo quick­ly achieved leg­endary sta­tus as break­through can­cer ther­a­pies with broad, block­buster roles to play on the mar­ket. But in a new study pub­lished in the New Eng­land Jour­nal of Med­i­cine, in­ves­ti­ga­tors have spot­light­ed rare cas­es in which check­point pa­tients have died or been af­flict­ed by heart trou­ble. And there’s ev­i­dence that a T-cell dri­ven re­ac­tion threat­ens a very small group of pa­tients tak­ing the drugs.

Dr. Javid J. Mosle­hi

Two melanoma pa­tients died from my­ocardi­tis af­ter tak­ing a com­bi­na­tion of Yer­voy and Op­di­vo, ac­cord­ing to the re­port. And 0.27% — a tiny sliv­er of the to­tal — de­vel­oped the con­di­tion, a po­ten­tial­ly fa­tal, T-cell–dri­ven drug re­ac­tion.

The re­searchers be­hind the re­port were care­ful to em­pha­size that no one should stop tak­ing the drugs, which have helped rev­o­lu­tion­ize melanoma ther­a­py in the past two years. But they do want physi­cians to be aware of the risk as they go about ex­plor­ing new ways to pre­vent fur­ther deaths.

Both pa­tients had ad­vance cas­es of melanoma when they were treat­ed, and nei­ther had a his­to­ry of heart prob­lems.

“We’re work­ing to de­vel­op treat­ments for it. Our job is not to say the drugs are bad, but to say, ‘How can we deal with it?’” Dr. Javid J. Mosle­hi, the di­rec­tor of car­dio-on­col­o­gy at Van­der­bilt School of Med­i­cine and the se­nior au­thor, told The New York Times.

The news marks an­oth­er po­ten­tial prob­lem for Op­di­vo af­ter Bris­tol-My­ers Squibb was sent reel­ing by the fail­ure of its front­line Phase III study for non-small cell lung can­cer. That out­come helped Mer­ck leap ahead by gain­ing a new ap­proval for us­ing their drug on pre­vi­ous­ly un­treat­ed pa­tients whose can­cer cells ex­press high lev­els of PD-L1.

Belgian biotech Confo Therapeutics has landed $183 million, plus potential royalties, in a drug-discovery deal with Daiichi Sankyo.

Early Thursday, Confo Therapeutics put out word of the deal that will be focused on small molecule antagonists to go after an undisclosed target that the company says is associated with CNS diseases.

Confo CEO Cedric Ververken told Endpoints News that Daiichi originally reached out to learn about the biotech’s technology. He added that Confo, founded in 2015, will use its platform to drug a GPCR target that Daiichi has struggled with internally.

When Vigil Neuroscience filed its IPO papers in late 2021, the biotech revealed that the FDA had just cleared its Phase I trial — but with a partial clinical hold that limited dosing to under a certain level.

More than a year later, the FDA has lifted the hold.

Vigil is now free to dose VGL101, an antibody targeting TREM2, at levels higher than 20 mg/kg in its ongoing and future clinical trials in patients with adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP), an inherited condition that affects the brain and spinal cord.

Microcap biotech Seelos Therapeutics is halting enrollment of its study in spinocerebellar ataxia type 3 (also known as Machado-Joseph disease) because of “financial considerations,” and in order to focus on other studies, the company said today, adding that the pause would be temporary.

The study will continue with the patients who have already enrolled, and the data from them will be used to decide whether to continue enrolling others in the future.