Today, Sanofi announced that its JAK2 inhibitor met the primary endpoint in both dose groups during a Phase III study evaluating the drug for treatment of myelofibrosis.
The pivotal study, JAKARTA, evaluated once-daily, oral SAR302503 compared to placebo. The study consisted of 289 patients with intermediate-2 or high-risk primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis, which were randomly assigned a 400mg dose of SAR302503, 500mg dose of SAR302503, or placebo for 24 weeks. The primary endpoint was the proportion of patients with a reduction in spleen volume ≥ 35 percent after 24 weeks of treatment. According to the company, the primary endpoint was met and full results will be presented at an upcoming medical congress.
“Patients with myelofibrosis in advanced stages are desperately ill and in need of treatments that will improve their outcomes. I am pleased with the results of JAKARTA and would like to thank the patients and the investigators in this trial,” Debasish Roychowdhury, MD, Senior Vice President and Head, Sanofi Oncology stated. “Since Sanofi’s acquisition of the molecule, SAR302503 has moved from Phase I to the completion of pivotal Phase III studies in less than three years, and now we are planning regulatory filings with authorities to make this medicine available for patients.”
Myelofibrosis (MF) is a rare, serious blood disease. MF is characterized by an abnormal blood cell production and fibrosis (scarring) within the bone marrow. Scarring of the bone marrow interferes with blood cell production, which causes the spleen and liver to produce and store extra blood cells, causing an enlarged spleen. For patients with intermediate-2 and high-risk patients, median survival is approximately two and a half years and for MF patients overall is approximately six years.
Last Updated: 5/17/13; 2:20PM EST