Novartis announced data from the largest clinical trial of myelofibrosis patients treated with Jakavi (ruxolitinib), supporting the safety profile and efficacy benefit as measured in primary and secondary endpoints respectively. In an analysis of 1,144 patients treated with Jakavi to date in this ongoing expanded access study, 69 per cent of patients achieved >50 per cent reduction in spleen size from baseline and patients also experienced a clinically meaningful improvement in myelofibrosis symptom score, important treatment goals for patients with myelofibrosis.

Findings from the study were presented at the 56th Annual Meeting of the American Society of Hematology (ASH) in San Francisco, California.

"Results to date from the JUMP study reinforce the critical role that Jakavi plays in the treatment of myelofibrosis, a life-threatening and debilitating blood cancer with limited treatment options," said Haifa Kathrin Al-Ali, MD, University Hospital of Leipzig, Leipzig, Germany. "These data provide insight into the real world experience of more than 1,000 patients living with myelofibrosis and further validate the safety and efficacy of Jakavi as an important treatment for myelofibrosis."

Novartis research and development efforts, in collaboration with Incyte Corporation, include early-phase and post-marketing studies in myelofibrosis and other myeloproliferative neoplasms. More than 50 abstracts on ruxolitinib are being presented at ASH, including three oral presentations exploring combinations of ruxolitinib with various investigational compounds, evaluating the possibility of simultaneously targeting multiple cancer pathways that may be involved in the pathogenesis of myelofibrosis.

"Data presented at ASH demonstrate our ongoing commitment to the myelofibrosis community and reinforce the role of Jakavi as the current standard of care for these patients," said Alessandro Riva, MD, Global Head, Novartis Oncology Development and Medical Affairs. "It's exciting to see the depth of research in a rare blood cancer like myelofibrosis. Results from these studies help us to better understand and address the needs of patients and physicians."

The JUMP study is a phase IIIb, expanded-access trial for countries with no access to Jakavi outside of a clinical trial. The open-label, multicenter study analyzed 1,144 enrolled myelofibrosis patients who received daily starting doses of either 5 mg, 15 mg or 20 mg of Jakavi twice daily based on platelet counts at baseline. The primary endpoint is assessment of safety and tolerability of Jakavi. Additional analyses included changes in spleen size and symptom scores as measured by the FACT-Lymphoma Total Score (FACT-Lym TS). As of September 22, 2014, 2,138 patients were enrolled at more than 200 study sites in 25 countries, and the final analysis will be performed after all patients have completed 24 months of treatment or ended treatment due to commercial availability.

Overall, the safety and efficacy profile of Jakavi was consistent with previous studies. The most common Grade 3 or 4 hematologic adverse events (AEs) were anemia (33.0 per cent) and thrombocytopenia (12.5 per cent); however, they rarely led to discontinuation (2.6 per cent of discontinuation was due to anemia and 3.2 per cent of discontinuation was due to thrombocytopenia). The most common nonhematologic AEs were diarrhoea (14.5 per cent), fever (13.3 per cent), fatigue (12.9 per cent) and asthenia (12.5 per cent), which were primarily Grade 1 or 2.

Also separately presented for the first time are results from the new MPN Landmark Survey of physicians and patients with myeloproliferative neoplasms (MPNs), a group of related blood cancers, including myelofibrosis. The survey, conducted by Incyte Corporation, concluded that an essential goal in disease management should focus on reducing symptom burden and improving quality of life in order to enhance the overall health of patients with myelofibrosis.

The Landmark Survey found that a remarkable 81 per cent of patients with myelofibrosis reported their myelofibrosis-related symptoms reduced their quality of life. The vast majority of myelofibrosis patients (81 per cent) reported that fatigue was the most severe and common symptom they experienced. According to physicians surveyed, fatigue, abdominal discomfort and pain had the greatest impact on quality of life in their myelofibrosis patients. In fact, both patients and physicians agreed that fatigue is the most urgent symptom myelofibrosis patients would like to resolve.

The MPN Landmark Survey is the first large US-based survey to examine both physicians who treat MPNs and patients diagnosed with one of the three MPNs, including myelofibrosis, polycythemia vera (PV) and essential thrombocythemia (ET). The survey was completed by 457 physicians and 813 patients (MF=207; PV=380; ET=226) who filled out an online survey of 59 to 65 questions (depending on the MPN), conducted between May and July 2014. Participants were asked about the overall burden of disease and impact of symptoms on quality of life, productivity, and activities of daily living (ADLs). Symptom severity was determined using the MPN Symptom Assessment Form total symptom scores (MPN-SAF TSS), and descriptive analyses were conducted to identify gaps in perceptions of disease burden and patient-physician communication.

Myelofibrosis is a rare, life-threatening blood cancer, with approximately 1 in every 133,000 people estimated to be affected by the disease. Myelofibrosis develops when uncontrolled signalling in the JAK pathway which regulates blood cell production - causes the body to make blood cells that do not work properly, which scars the bone marrow and results in an enlarged spleen as well as other severe complications and debilitating symptoms.

Studies show that patients with myelofibrosis have a decreased life expectancy, with a median overall survival of 5.7 years. Although allogeneic stem cell transplantation may cure myelofibrosis, the procedure is associated with significant morbidity and transplant-related mortality and is available to less than 5 per cent of patients who are young and fit enough to undergo the procedure. Current myelofibrosis treatment strategies are aimed at reducing spleen size, relieving symptoms, improving quality of life and reducing the risk of complications.

Jakavi (ruxolitinib) is an oral inhibitor of the JAK 1 and JAK 2 tyrosine kinases and was approved by the European Commission in August 2012 for the treatment of disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis (also known as chronic idiopathic myelofibrosis), post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis. Jakavi is approved in more than 70 countries, including the European Union, Canada, Japan and some countries in Asia, Latin and South America. Additional worldwide regulatory filings are underway.
Novartis licenced ruxolitinib from Incyte Corporation for development and commercialisation outside the United States. Both the European Commission and the US Food and Drug Administration (US FDA) granted ruxolitinib orphan drug designation for myelofibrosis. Jakavi is marketed in the United States by Incyte Corporation under the name Jakafi for the treatment of patients with intermediate or high-risk myelofibrosis.

The recommended starting dose for Jakavi in patients with myelofibrosis is 15 mg twice daily for patients with a platelet count between 100,000 cubic millimeters (mm3) and 200,000 mm3, and 20 mg twice daily for patients with a platelet count of >200,000 mm3. Doses may be titrated based on safety and efficacy. There is limited information to recommend a starting dose for patients with platelet counts between 50,000/mm3 and <100,000/mm3. The maximum recommended starting dose in these patients is 5 mg twice daily, and patients should be titrated cautiously.

Jakavi is a registered trademark of Novartis AG in countries outside the United States. Jakafi is a registered trademark of Incyte Corporation. The safety and efficacy profile of Jakavi has not yet been established outside the approved indication.