CTI BioPharma Corp (NASDAQ:CTIC) announced positive progress of its lead clinical program in addition to several key business priorities for 2016.
“After a productive 2015, we have entered 2016 well capitalized and focused on preparing for the potential accelerated approval and launch of a new treatment option for people with intermediate and high-risk myelofibrosis with low platelet counts,” said James A. Bianco, M.D.,CTI BioPharma’s President and Chief Executive Officer. “With the recently completed NDA submission for pacritinib, interim data indicating the potential therapeutic utility for tosedostat in AML and high-risk MDS, and a strong financial position, we believe the stage is set for a transformational year for our company.”
Recent Progress Update
In January 2016, CTI BioPharma announced the completion of the 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. CTI BioPharma and Baxalta Incorporated (Baxalta) are seeking U.S. marketing approval of 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/µL).
In December 2015, researchers presented results from a new analysis of the pivotal Phase 3 trial, PERSIST-1, evaluating pacritinib versus best available therapy, excluding treatment with JAK2 inhibitors (BAT), in patients with myelofibrosis. Data examining patient outcomes across baseline demographic factors that are associated with prognosis showed that treatment with pacritinib resulted in consistent rates of spleen volume reduction and control of disease-related symptoms across all intermediate or high-risk myelofibrosis subgroups.
In December 2015, researchers presented an update on results from an investigator-sponsored Phase 2 trial of tosedostat – CTI BioPharma’s investigational oral selective aminopeptidase inhibitor – in elderly patients with either primary (de novo) acute myeloid leukemia (AML) or secondary AML. The data showed a complete response (CR) of 48.5 percent with tosedostat in combination with low dose cytarabine/Ara-C (LDAC) – 33 percent of these patients were still responding after a median of 506 days.
In November 2015, CTI BioPharma announced that the United Kingdom’s National Cancer Research Institute (NCRI) Haematological Oncology Clinical Studies Group has chosen to advance tosedostat to the second stage of a randomized clinical trial of low-dose cytarabine plus or minus tosedostat in older patients with AML or high risk myelodysplastic syndrome (MDS). The AML Less Intensive (LI-1) trial is designed as a “Pick-a-Winner” trial to be able to simultaneously test a number of promising agents added to standard therapy with low-dose cytarabine in older patients with AML or MDS who are unfit for standard aggressive induction therapy. Nine regimens have been tested in the Pick-a-Winner program, of which only four, including tosedostat, have passed the initial hurdle for progression (which requires evidence of an improvement in remission rate with acceptable safety). CTI BioPharma plans to discuss potential approval pathways with regulators in the U.S. and EU in 2016.
Corporate and Financial
In December 2015, the Company announced receipt of a $10 million milestone payment fromTeva Pharmaceutical Industries Ltd. related to the achievement of sales milestones for TRISENOX® (arsenic trioxide).
In September 2015, the Company completed a registered direct offering and in October andDecember 2015, the Company completed two underwritten public offerings resulting in aggregate net proceeds of approximately $115 million. CTI BioPharma’s current cash balance is anticipated to fund operations for at least the next two years based on our current budget expectations and development plans.
CTI BioPharma plans to provide 2016 financial guidance in its fourth quarter and year-end 2015 financial results announcement.
2016 Key Objectives
Seek U.S. regulatory approval and launch pacritinib. CTI BioPharma seeks U.S. marketing approval of pacritinib for the treatment of patients with intermediate and high-risk myelofibrosis with low platelet counts of <50,000/µL.
Complete enrollment and report PERSIST-2 topline results. CTI BioPharma expects to complete enrollment in the PERSIST-2 Phase 3 trial of pacritinib for patients with myelofibrosis whose platelet counts are less than or equal to 100,000 per microliter in the first quarter 2016 and unblind and report top-line data late in the fourth quarter of 2016.
Initiate trial in second indication for pacritinib. CTI BioPharma intends to advance a pacritinib development program in other hematologic malignancies in the fourth quarter of 2016.
Initiate registration-directed trial for tosedostat. CTI BioPharma plans to consult with the FDAand European Medicines Agency (EMA) regarding a registration-directed strategy for tosedostat, including the potential to utilize the results from the ongoing investigator-sponsored LI-1 trial to support registration and approval. If positive, these discussions could help enable the start of a pivotal program in 2016.
Secure ex-U.S. partner for tosedostat. CTI BioPharma intends to secure a partnership for the development and commercialization of tosedostat in certain territories outside the U.S.
Complete enrollment in PIX306. CTI BioPharma expects to complete enrollment in the ongoing PIX306 post-marketing commitment Phase 3 study of PIXUVRI in the fourth quarter of 2016. (Original Source)
Shares of CTI BioPharma Corp. closed last Friday at $1.2. CTIC has a 1-year high of $2.94 and a 1-year low of $0.80. The stock’s 50-day moving average is $1.19 and its 200-day moving average is $1.53.
On the ratings front, CTI BioPharma has been the subject of a number of recent research reports. In a report issued on December 10, Piper Jaffray analyst Charles Duncan reiterated a Buy rating on CTIC, with a price target of $8, which implies an upside of 566.7% from current levels. Separately, on November 9, WallachBeth Capital LLC’s Bob Ai downgraded the stock to Hold and has a price target of $1.60.
According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Charles Duncan and Bob Ai have a total average return of -1.3% and -12.3% respectively. Duncan has a success rate of 37.9% and is ranked #2582 out of 3610 analysts, while Ai has a success rate of 20.0% and is ranked #3076.