STAT+: Mixed study results for MorphoSys blood-cancer drug raise questions about approval

LONDON — An experimental blood cancer drug from MorphoSys hit its primary target in a pivotal trial, the company announced late Monday, but the drug faltered in addressing patient symptoms,…

Nov 21, 2023 - 18:00
STAT+: Mixed study results for MorphoSys blood-cancer drug raise questions about approval

LONDON — An experimental blood cancer drug from MorphoSys hit its primary target in a pivotal trial, the company announced late Monday, but the drug faltered in addressing patient symptoms, causing the company’s stock to fall in early Tuesday trading.

In the Phase 3 trial, the German company was testing its drug candidate pelabresib combined with the drug Jakafi against a Jakafi-placebo duo in patients with newly diagnosed myelofibrosis, a type of rare blood cancer. Jakafi is a standard myelofibrosis treatment.

The drug combination succeeded on its primary endpoint, with 66% of patients seeing at least a 35% reduction in spleen volume after 24 weeks versus 35% on the placebo regimen — an improvement the company in a release called “statistically significant and clinically meaningful.” Myelofibrosis is a rare bone marrow cancer that causes low blood cell counts and enlarged spleens, so reducing spleen volume is commonly used as the primary efficacy goal in clinical trials.

Continue to STAT+ to read the full story…

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