Myelofibrosis is a rare hematologic neoplasm categorized under myeloproliferative neoplasms, with over 19,000 cases in the US, according to DelveInsight. JAK inhibitors have emerged as the centerpiece of pharmacologic therapy for patients with myelofibrosis, providing unprecedented benefits in terms of spleen shrinkage, symptom improvement, and improved quality of life. Different mechanisms other than JAK inhibitors under investigation are telomerase inhibitor (imetelstat), BCL-XL/BCL-2 inhibitor (navitoclax), MDM2 protein inhibitor (navtemadlin), BET inhibitor (pelabresib), and others which may cover the patient need and provide an alternative treatment for the patients.

The EHA 2024 conference will spotlight groundbreaking advancements in this field, with leading pharmaceutical companies such as Abbvie, Keros Therapeutics, MorphoSys, Disc Medicine, Incyte Corporation, and others set to present new data and final analyses, that are expected to spark significant interest and discussion.

Updated results from the ongoing Phase Ib study of DISC-0974, a first-in-class monoclonal antibody against hemojuvelin, in patients with myelofibrosis and anemia, including a larger data set and longer follow-up, to be discussed in a poster, will be a significant focus of the EHA 2024 congress. Disc Medicine anticipates releasing the final Phase Ib data in the second half of 2024. 

Combination therapies are being explored to improve outcomes in myelofibrosis patients. Two Phase III trials showed that adding drugs to ruxolitinib significantly increased spleen volume reduction compared to ruxolitinib alone. The BET inhibitor, pelabresib was included in the MANIFEST-2, while the BCL2 inhibitor, navitoclax was added in TRANSFORM-1 study, leading to a doubling in patients achieving ≥35% spleen volume reduction at 24 weeks. Additional updated findings from the both these studies, are eagerly awaited to be presented at the conference.

With numerous other findings set to be revealed, this session promises substantial progress in the understanding and treatment of myelofibrosis. On this note, DelveInsight has compiled a list of top abstracts within the myelofibrosis field, poised to potentially revolutionize patient care. These abstracts hold promise for significant advancements, aiming to positively impact the lives of patients affected by myelofibrosis.

Myelofibrosis Key Abstracts