Gene therapy cancer risk materializes in pediatric cases

STAT reported on Nov. 9 that tumors possibly connected to pediatric gene therapies have emerged, a development the publication described as the materialization of a cancer risk that has long been debated in gene therapy. STAT's visible summary did not name the specific products involved, nor did it say how many tumor cases had been reported. The article's author wrote that the cases "make me sad," adding a one-word statement: "Babies."

The same roundup's headline said "Two heart trials come up empty," and the article explained that the trials were presented at the American Heart Association meeting. STAT argued that these failures should deflate the current enthusiasm for using AI to make drug development more predictable. The article did not give the names of the heart drugs or their sponsors in the portion available before the paywall.

The roundup's "need-to-know" section summarized announcements from Vir, Zura, Roivant and Argenx, each of which had released news that morning.

In the company updates that followed, Vir Biotechnology announced that a Phase 3 study of two experimental medicines given as monthly injections met its primary endpoint. The company said 44% of patients who received the regimen reached undetectable hepatitis D virus, compared with none of the patients assigned to delayed treatment with the same drugs. The two-drug regimen was given monthly.

Zura Bio reported that its investigational antibody tibulizumab did not improve symptoms of hidradenitis suppurativa relative to placebo in a mid-stage trial. Hidradenitis suppurativa is an inflammatory skin condition. The company did not provide further details in the summary.

Roivant said Lisraya, which was approved for dermatomyositis, generated $31 million in sales during its first five weeks on the market.

Argenx announced an agreement to acquire Unnatural Products, a privately held medicinal chemistry company that specializes in discovering protein-based drugs.

STAT's summary also noted that the Biotechnology Innovation Organization is suing to block a Medicare pilot program that would tie drug prices to those in peer countries. The article was authored by Meghana Keshavan and Brittany Trang and appears in STAT's biotech section. The piece appeared under the outlet's biotech coverage.