AstraZeneca had hoped data from Phase III PROpel would lead to an all-comers indication in first-line, metastatic castration-resistant prostate cancer, but new indication is limited to BRCA mutations.

The latest data from a Phase I/II study shows that pegtibatinase can yield a rapid, sustained reduction in homocysteine, the protein behind the underlying cause of the rare disease.

Lexicon gets broad indication across heart failure for its dual SGLT1/2 inhibitor, which will be positioned against Lilly/BI’s Jardiance and AstraZeneca’s Farxiga.

When the first non-alcoholic steatohepatitis drugs reach market, the health cost watchdog says payers will be justified in setting step-therapy including weight-management efforts, while early prescribing should be by specialists.

Apellis’s pegcetacoplan – approved for two other indications – will not go forward in amyotrophic lateral sclerosis after a Phase II failure, two days after Wave Life made a similar call in the neurodegenerative disease.

Pointing to data showing benefit in patients who completed 72 weeks of therapy, PTC thinks vatiquinone still could be viable for pediatric FA patients. Restructuring won’t affect Upstaza.