Incyte (INCY) announced results from pivotal Phase 3 trial of Jakafi (ruxolitinib) in polycythemia vera patients who are resistant to or intolerant of hydroxyurea. The results met the primary endpoints; achieving phlebotomy independence and reducing spleen size by 35 percent or more compared to best available therapy.
Jakafi® is approved to treat intermediate or high-risk myelofibrosis (MF), including primary MF, post–polycythemia vera MF and post–essential thrombocythemia MF.
The study was a global, randomized, open-label Phase III trial, called RESPONSE. It was conducted at 109 sites on 222 polycythemia vera patients who are resistant to or intolerant of hydroxyurea. Patients were randomized 1:1 to receive either ruxolitinib (10 mg twice-daily) or best available therapy. The dose was adjusted as needed throughout the trial.
Data from RESPONSE are expected to be presented at an upcoming scientific meeting and submitted to the U.S. Food and Drug Administration this year.
Polycythemia vera (PV) is a myeloproliferative neoplasm (MPN) characterized by an overproduction of red and white blood cells and platelets, which leads to an increased risk of thrombosis Erythrocytosis (elevated red blood cell mass) is the most prominent clinical manifestation of PV, distinguishing it from other myeloproliferative diseases. The disease occurs at all ages, but is often presents later in life, with a median age at diagnosis of 60 years.
Incidence: Approximately 100,000 patients in the United States are living with PV of which 25 percent develop resistance to or intolerance of hydroxyurea and are considered uncontrolled.
Although patients may be asymptomatic for many years, PV is associated with significant symptom burden, including fatigue, itching, night sweats, bone pain, fever and weight loss. Spleen enlargement is present in 30 percent to 40 percent of patients with PV. In patients who experience severe and burdensome symptoms, data show that the disease causes a significant and clinically meaningful erosion of quality of life.
Jakafi (ruxolitinib) is a Janus kinase inhibitor with selectivity for subtypes JAK 1 and JAK2 of this enzyme. The oral prescription drug is approved for intermediate or high-risk myelofibrosis, including primary MF, post–polycythemia vera MF and post–essential thrombocythemia MF. The approval was based on results from (COMFORT-I) and COMFORT-II trials. It is also being investigated for the treatment of cancers, including lymphomas and pancreatic cancer and for polycythemia vera and plaque psoriasis. The results of these trials demonstrated significant benefits by reducing spleen size, relieving debilitating symptoms, and improving overall survival.
Jakafi is marketed by Incyte in the United States and by Novartis as Jakavi® (ruxolitinib) outside the United States.
Prohost Comments: The news of a successful outcome of the pivotal Phase 3 trial of Jakafi (ruxolitinib) in polycythemia vera patients who are resistant to or intolerant of hydroxyurea is great for Incyte, its shareholders and for the PV patients who are resistant to the standard treatment. These patients who represent 25% of PV patients face a profound symptom burden and are at greater risk of cardiovascular complications and death. It is, indeed, good news.
We congratulate our subscribers who believed in our assessment of Incyte and of its drug Jakafi. To read our previous articles on Incyte click the following links:
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