Targeted blood cancer therapy, pelabresib from MorphoSys enters the spotlight

Edited by ad hoc news Flagship & Bestseller Desk. Reviewed before publication on 06/15/2026 at 11:38 AM ET. Details in the imprint.

In the race to bring new options to patients with blood cancers, MorphoSys is betting heavily on its late-stage candidate pelabresib, a small-molecule BET inhibitor being studied for myelofibrosis in combination with ruxolitinib. According to the company, pelabresib is in Phase 3 development and aims to improve spleen volume reduction and symptom control in patients with intermediate- or high-risk disease. An official MorphoSys pipeline update describes pelabresib as the firm’s most advanced and strategically important oncology asset.

What pelabresib is designed to do in myelofibrosis

Pelabresib, also known by the development code CPI-0610, is an oral bromodomain and extra-terminal domain (BET) inhibitor intended to modulate gene expression programs that drive malignant cell growth and inflammatory signaling in myelofibrosis. The drug is being developed primarily as an add-on to the JAK inhibitor ruxolitinib, with the goal of achieving deeper and more durable spleen and symptom responses than JAK inhibition alone can typically deliver. Clinical data published in the journal Blood suggest that BET inhibition may also influence bone marrow fibrosis and anemia parameters, which are key unmet needs for many patients.

The pivotal MANIFEST-2 trial is a randomized, double-blind Phase 3 study evaluating pelabresib plus ruxolitinib versus ruxolitinib plus placebo in JAK inhibitor-naive myelofibrosis patients, with spleen volume reduction of at least 35 percent at week 24 as the primary endpoint. MorphoSys has highlighted that the study also tracks symptom scores, anemia-related outcomes and bone marrow morphology to understand whether BET inhibition can translate into structural improvement of the disease. In addition to MANIFEST-2, the earlier MANIFEST Phase 2 study explored various combinations and monotherapy settings, helping define dosing and identify patient subgroups most likely to benefit from the drug. Sector analyses from Evaluate Vantage have underscored that the clinical profile emerging from these studies will be crucial for pelabresib’s commercial prospects.

Strategically, pelabresib sits at the center of MorphoSys’s transition from a broader antibody platform company into a more focused oncology player with a few higher-value assets. The candidate is developed for global markets, but MorphoSys is headquartered in Planegg near Munich and listed in Frankfurt, making German and European regulatory and reimbursement decisions especially important for its future revenue stream. Shares of MorphoSys (DE0006632003) last traded on Xetra in euros, with investors watching closely for further updates on pelabresib’s Phase 3 data and partnering outlook.

This article was a.i.-assisted and editorially reviewed. Product information without warranty; prices and availability may change at short notice. Not investment advice and not a buy or sell recommendation. Trading involves risk up to and including the total loss of invested capital.