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CTI Biopharma Corp.

Partner : Baxter

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Pacritinib

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 February 2016, the FDA placed pacritinib's investigational new drug application (IND) on a full clinical hold and CTI BioPharma subsequently withdrew its NDA.
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Source: press release, 2/16/16. http://investors.ctibiopharma.com/phoenix.zhtml?c=92775&p=irol-newsArtic...

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CTI BioPharma Provides Update On Clinical Hold Of Investigational Agent Pacritinib And New Drug Application In U.S.
SEATTLE, Feb. 9, 2016 /PRNewswire/ -- CTI BioPharma Corp. (CTI BioPharma) (NASDAQ and MTA: CTIC) today provided an update regarding the clinical studies being conducted under the Company's Investigational New Drug ("IND") application for pacritinib. Following the issuance of the Company's February 8, 2016, press release describing the partial clinical hold issued by the U.S. Food and Drug Administration (FDA) regarding those clinical studies, the Company received an oral communication from the FDA followed by a letter notifying the Company that the Company's IND for pacritinib has been placed on full clinical hold. The Company has withdrawn its New Drug Application (NDA) until the Company has had a chance to review the safety and efficacy data from the PERSIST-2 Phase 3 clinical trial and decide next steps.

The FDA's February 8, 2016, letter notes the interim overall survival results from PERSIST-2 show a detrimental effect on survival consistent with the results from PERSIST-1. The deaths in PERSIST-2 in pacritinib-treated patients include intracranial hemorrhage, cardiac failure and cardiac arrest. The FDA made recommendations that supersede the recommendations made by the FDA in connection with the partial clinical hold imposed by the FDA on February 4, 2016. The current recommendations include conducting dose exploration studies for pacritinib in patients with myelofibrosis, submitting final study reports and datasets for PERSIST-1 and PERSIST-2, providing certain notifications, revising relevant statements in the related Investigator's Brochure and informed consent documents and making certain modifications to protocols. In addition, the FDA recommended that the Company request a meeting prior to submitting a response to full clinical hold.

Under the full clinical hold, all patients currently on pacritinib must discontinue pacritinib immediately and no patients can be enrolled or start pacritinib as initial or crossover treatment.

All clinical investigators worldwide have been delivered a notice of the full clinical hold.
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Source: press release, 2/09/16. http://investors.ctibiopharma.com/phoenix.zhtml?c=92775&p=irol-newsArtic...

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CTI BioPharma And Baxalta Complete Submission Of New Drug Application For Pacritinib For Unmet Medical Need In Myelofibrosis

SEATTLE and BANNOCKBURN, Ill., Jan. 5, 2016 /PRNewswire/ -- CTI BioPharma Corp. (CTI BioPharma) (NASDAQ and MTA: CTIC) and Baxalta Incorporated (Baxalta) (NYSE: BXLT) today announced the completion of the rolling submission of the 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 are requesting 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) – a specific patient population for which there is an existing unmet medical need. The Companies are seeking accelerated approval and have requested a Priority Review of the application.

Pacritinib is an investigational treatment being developed for patients with myelofibrosis regardless of their platelet counts. If approved, pacritinib would be the first JAK2 inhibitor indicated for the treatment of patients with myelofibrosis and baseline platelet counts of less <50,000/μL.

"We are pleased to have completed the rolling submission and look forward to working with the FDA during the review process with the goal of bringing this important treatment to people living with myelofibrosis, including those with low platelet counts," said James Bianco, M.D., president and chief executive officer of CTI BioPharma.

Myelofibrosis is a rare, but serious and life-threatening chronic leukemia that disrupts the normal production of blood cells and results in scarring of the bone marrow, limiting the ability to produce new blood cells and prompting the spleen and other organs to take over this function. The disease often leads to an enlarged spleen and lower than normal counts of blood cells – including red blood cells and platelets, which are essential for blood clotting.

"Pacritinib has the potential to change the treatment paradigm for people with intermediate and high-risk myelofibrosis, particularly those patients with cytopenias," said David Meek, executive vice president, president of Oncology at Baxalta. "Together with CTI BioPharma, we are continuing to develop this potential new treatment for more people in need around the world."
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Source: press release, 1/05/16. http://investors.ctibiopharma.com/phoenix.zhtml?c=92775&p=irol-newsArtic...

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CTI BioPharma Initiates Rolling Submission of U.S. New Drug Application for Pacritinib for the Treatment of Patients with Myelofibrosis

-- Completion of NDA Submission Expected Before the End of 2015 --

SEATTLE, Nov. 23, 2015 /PRNewswire/ -- CTI BioPharma Corp. (CTI BioPharma) (NASDAQ and MTA: 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 Baxalta plan 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.
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Source: press release, 11/23/15. http://investors.ctibiopharma.com/phoenix.zhtml?c=92775&p=irol-newsArtic...

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CTI BioPharma To Submit NDA For Pacritinib In Q4 Based Primarily On Data From Single Pivotal Persist-1 Trial

- Decision Follows Pre-NDA Meeting with the FDA to Request Accelerated Approval for Patients with Myelofibrosis and Low Platelet Counts -

- Accelerated Approval to Be Requested Based on Single Trial, Potentially Reducing Time to Market -

SEATTLE, Sept. 23, 2015 /PRNewswire/ -- CTI BioPharma Corp. (CTI BioPharma) (NASDAQ and MTA: CTIC) today announced its plan to submit a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) following a productive pre-NDA meeting for pacritinib, an investigational oral kinase inhibitor with specificity for JAK2, FLT3, IRAK1 and CSF1R. The company expects to submit the NDA in the fourth quarter of 2015 and to request accelerated approval 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). The NDA will be based primarily on data from the PERSIST-1 Phase 3 trial – as well as data from Phase 1 and 2 studies of pacritinib – and additional information requested by the FDA, including a separate study report and datasets for the specific patient population with low platelet counts of less than 50,000 per microliter (<50,000/uL) for whom there are no approved drugs. 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.

Myelofibrosis is a rare and serious chronic blood cancer that can affect patients of all ages with a median age of 65 years, with an estimated prevalence in the United States of approximately 18,000 patients.

"This improved timing on the plan to submit an NDA – which is supported by data after completion of the PERSIST-1 trial – will allow us the potential for making pacritinib available to patients with low platelet counts earlier than expected," said James A. Bianco, M.D., President and CEO of CTI BioPharma. "We look forward to working with the FDA on the submission and review of this application."
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Source: press release, 9/23/15. http://investors.ctibiopharma.com/phoenix.zhtml?c=92775&p=irol-newsArtic...

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Nancy L. Boman, M.D., Ph.D., SVP, Clinical Development and Regulatory Affairs, gave guidance for the regulatory filing for Pacritinib. She stated, "We are delighted to now build on this positive momentum and anticipate completing enrollment in the ongoing second phase-III trial known as PERSIST-2 and intend to potentially commence the regulatory submission to the FDA and or the EMA as early as the end of this year. Baxter is responsible for the submission of the MAA in Europe."
Source: Q4 2014 earnings conference call, 3/12/15.

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Compound/DeviceSpecialtyIndicationCompound ClassTarget
PacritinibOncology HematologicMyelofibrosisJanus-associated Kinase InhibitorsJanus-associated Kinase 2 (JAK2)

Mechanism of action: Pacritinib is an orally bioavailable inhibitor of Janus kinase 2 (JAK2) and the JAK2 mutant JAK2V617F with potential antineoplastic activity. Pacritinib competes with JAK2 for ATP binding, which may result in inhibition of JAK2 activation, inhibition of the JAK-STAT signaling pathway, and so caspase-dependent apoptosis. JAK2 is the most common mutated gene in bcr-abl-negative myeloproliferative disorders; the JAK2V617F gain-of-function mutation involves a valine-to-phenylalanine modification at position 617. The JAK-STAT signaling pathway is a major mediator of cytokine activity.

Phase of Development: Filed

Event Type: Regulatory FDA: PDUFA DATE

Dates: 2017-01-05

Results: Discontinued