CTI BioPharma Corp (NASDAQ:CTIC) announced the initiation of its rolling new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for pacritinib, an investigational oral kinase inhibitor with specificity for JAK2, FLT3, IRAK1 and CSF1R. As part of the application, CTI BioPharma and its partner, Baxalta Incorporated (Baxalta), are seeking accelerated approval and priority review for pacritinib for the treatment of patients with intermediate and high-risk myelofibrosis with low platelet counts of less than 50,000 per microliter (<50,000/uL). If approved for the requested indication, pacritinib would be the first JAK2 inhibitor approved for the treatment of patients with myelofibrosis with platelet counts of less than 50,000/uL – a specific patient population for which there are currently no approved drugs. The rolling NDA allows completed portions of an NDA to be submitted and reviewed by the FDA on an ongoing basis. CTI BioPharma and Baxaltaplan to complete the submission before the end of 2015.
Myelofibrosis (a type of myeloproliferative neoplasm) is a rare, but serious and life-threatening chronic bone marrow disorder caused by the accumulation of malignant bone marrow cells that triggers an inflammatory response and scars the bone marrow. Myelofibrosis is associated with significantly reduced quality of life and shortened survival, can affect patients of all ages (with a median affected age being 65 years) and an estimated prevalence in the United States of approximately 18,000 patients.
“We believe the initiation of the rolling NDA submission represents a major step forward toward potentially offering pacritinib as a next generation JAK2/FLT3 inhibitor to patients with this rare and chronic type of blood cancer,” said James A. Bianco, M.D., President and CEO of CTI BioPharma. “We are excited to have achieved this milestone and look forward to working with the FDA during the review process, with the goal of bringing this important treatment to market – which we hope will fill an unmet need for many patients whose lives are profoundly impacted by myelofibrosis.”
The submission includes data from the PERSIST-1 Phase 3 trial – as well as data from Phase 1 and 2 studies of pacritinib. Submission of an NDA after a single Phase 3 trial under accelerated approval, instead of waiting to complete two Phase 3 trials, could potentially reduce time to market by up to 14 months. In August 2014, pacritinib was granted Fast Track designation by the FDA for the treatment of intermediate and high-risk myelofibrosis including, but not limited to, patients with disease-related thrombocytopenia (low platelet counts); patients experiencing treatment emergent thrombocytopenia on another JAK2 therapy; or patients who are intolerant to or whose symptoms are not well controlled (or sub-optimally managed) on another JAK2 therapy. (Original Source)
Shares of CTI BioPharma Corp. closed last Friday at $1.10. CTIC has a 1-year high of $2.94 and a 1-year low of $1.07. The stock’s 50-day moving average is $1.45 and its 200-day moving average is $1.72.
On the ratings front, CTI BioPharma has been the subject of a number of recent research reports. In a report issued on November 9, WallachBeth Capital LLC analyst Bob Ai downgraded CTIC to Hold, with a price target of $1.60, which implies an upside of 45.5% from current levels. Separately, on November 2, Piper Jaffray’s Charles Duncan reiterated a Buy rating on the stock and has a price target of $8.
According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Bob Ai and Charles Duncan have a total average return of -7.6% and 9.0% respectively. Ai has a success rate of 33.3% and is ranked #3195 out of 3858 analysts, while Duncan has a success rate of 46.1% and is ranked #659.