On March 9, 2018 MiNA Therapeutics, the pioneer in RNA activation therapeutics, reported the publication of pre-clinical data on its MTL-CEBPA program in which the compound was shown to promote disease reversal in several models of liver disease and to reduce tumour burden in a model of liver cancer. MTL-CEBPA consists of CEBPA-51 small activating RNAs encapsulated in SMARTICLES nanoparticles (Press release, , SEP 9, 2018, View Source [SID1234524616]). It is the first development candidate to emerge from MiNA’s RNA activation platform and is currently being evaluated in a Phase I clinical study in patients with liver cancer. The data underscore the potential of this compound to address both severe liver disease indications as well as earlier disease stages to enhance chances of survival.

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"Gene activation of CEBPA using saRNA: Preclinical studies of the first in human saRNA drug candidate for liver cancer"

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"The combination of beneficial effects observed across a range of liver disease models supports a unique and promising role for MTL-CEBPA therapy in the treatment of liver diseases and liver cancer," commented Robert Habib, CEO of MiNA Therapeutics. "This exciting pre-clinical data highlights the potential of saRNAs to up-regulate gene expression and treat disease in radically new ways compared to conventional medicines."

In the publication, researchers investigated the potential benefits of MTL-CEBPA in a set of in vivo severe liver disease models representing advanced liver cirrhosis, non-alcoholic steatohepatitis (NASH) and liver cancer. Overall, MTL-CEBPA was shown to restore the expression of CEBPA, a master regulator of liver function. In the advanced liver cirrhosis model, MTL-CEBPA significantly reversed liver fibrosis and liver dysfunction and enhanced survival. In a model of NASH, MTL-CEBPA reversed liver steatosis. Improved liver function as well as a large reduction in tumour burden was also seen in a model of primary liver cancer. Together, these findings validate the beneficial role of up-regulating CEBPA expression in liver disease and liver cancer and are consistent with externally published data using genetic models of liver disease.

The publication titled "Gene activation of CEBPA using saRNA: Preclinical studies of the first in human saRNA drug candidate for liver cancer", was published in the latest issue of Oncogene by researchers at MiNA Therapeutics in collaboration with several well-regarded academic institutions including scientists at Imperial College London and National Taiwan University Hospital. The paper is available on the Company’s website in the publications section under "Media".

On March 9, 2018 CTI BioPharma Corp. (CTI BioPharma) (NASDAQ: CTIC) reported that results from the Phase 3 PERSIST-2 clinical trial of pacritinib (an investigational JAK2 inhibitor) have been published online in JAMA Oncology (Press release, CTI BioPharma, MAR 9, 2018, View Source;p=RssLanding&cat=news&id=2337281 [SID1234524602]). The randomized, international, multicenter study compared the efficacy and safety of pacritinib at two dose levels, compared with best available therapy (BAT), which included ruxolitinib (a JAK1/JAK2 inhibitor), in patients with myelofibrosis and thrombocytopenia (defined as platelet counts ≤100 x 109/L). The publication can be accessed at: View Source

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In the intent-to-treat patient population of the study, the combined pacritinib arms (400mg once daily and 200mg twice daily dosing, 149 patients total) demonstrated a significant improvement of 35% or more in spleen volume reduction (SVR) at 24 weeks of treatment in 27 patients (18%) compared to 2 patients (3%) out of 72 patients in the BAT arm, which included treatment with ruxolitinib (P=0.001). The combined pacritinib treatment arms also demonstrated a greater than 50% reduction in total symptom score (TSS) in 37 patients (25%), compared to 10 patients (14%) in the BAT arm (P=0.079). Additionally, an exploratory analysis of the 74 patients who received pacritinib 200mg twice daily showed an improvement of 35% or more reduction of SVR in 16 patients (22%; P=0.001 vs BAT) and 50% or greater reduction of TSS in 24 patients (32%; P=0.01 vs BAT).

Pacritinib was generally well tolerated. The most commonly reported (≥15%) nonhematologic adverse events with pacritinib were gastrointestinal events, fatigue, peripheral edema, and dizziness, and those with BAT (including 19 patients with watchful-waiting only) were abdominal pain, fatigue, diarrhea, and peripheral edema. The majority of common nonhematologic adverse events were grade 1 or 2 in severity. Diarrhea was the most frequently observed adverse event with pacritinib (53% grade 1/2; 4% grade 3) most often occurring during weeks 1 to 8. The incidence of diarrhea was lower with pacritinib twice daily dosing compared to once daily dosing (48% vs 67%, respectively). Diarrhea was manageable with standard antidiarrheal agents (e.g., loperamide) and generally resolved within 1 to 2 weeks. The rate of on-study death was lowest with pacritinib twice daily (6%) compared to BAT (9%) and pacritinib once daily (14%).

Cardiac events were reported at similar rates in all arms (32%, pacritinib once daily or twice daily; 28%, BAT) and were most commonly peripheral edema in all arms. Grade 3 or 4 cardiac events were reported in 13 patients (13%) treated with pacritinib once daily, 7 patients (7%) treated with pacritinib twice daily, and 9 patients (9%) treated with BAT.

Bleeding events were reported at similar rates in all arms (36%, 42%, and 41% of patients treated with pacritinib once daily, twice daily, and BAT, respectively) and were most commonly epistaxis in all arms. Grade 3 or 4 bleeding events were reported in 7 patients (7%), 15 patients (14%), and 7 patients (7%) treated with pacritinib once daily, twice daily, and BAT, respectively.

"Pacritinib was shown to reduce both spleen volume and total symptom score, two very important clinical measures, in myelofibrosis patients with thrombocytopenia including those patients who received prior treatment with ruxolitinib," stated John Mascarenhas, M.D., Adult Leukemia Program, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai. "Clinical improvements in hemoglobin levels and reduction in transfusions were also seen in patients who received pacritinib, and pacritinib had a generally manageable safety profile."

Dr. Mascarenhas continued, "Myelofibrosis is a difficult and progressive disease, and patients with thrombocytopenia that have already received JAK2 inhibitor therapy have an especially poor prognosis. These study results indicate there is important potential clinical benefit of pacritinib for these patients."

The PERSIST-2 trial results were previously presented at the 58th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, in December 2016.

CTI BioPharma is currently enrolling patients in the PAC203 study, which is evaluating the safety and efficacy of three dosing schedules, including 200mg twice daily (BID), 100mg twice daily (BID), and 100mg once daily (QD).

On March 9, 2018 LabCorp (NYSE: LH), a leading global life sciences company, reported that its Covance Drug Development (Covance) business has formed a global immunology and immunotoxicology (I&I) unit dedicated to the specific needs of biologic drug development (Press release, Covance, SEP 9, 2018, View Source [SID1234524652]). This team brings together Covance’s operational expertise in flow cytometry, immunoassays and cell-based assays with its scientific expertise in immunotoxicology study design, direction and operation to provide a more comprehensive offering for large-molecule drugs.

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"With the formation of this team, we continue to demonstrate Covance’s commitment to strengthening our biologics solutions with key investments in scientific staff, technology and facilities"

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"With the formation of this team, we continue to demonstrate Covance’s commitment to strengthening our biologics solutions with key investments in scientific staff, technology and facilities," said John Ratliff, CEO of Covance. "Biologics make up more than one-third of biopharma’s pipeline. Integrating our scientific and operational know-how into a seamless group allows us to deliver even richer scientific insights and faster cycle times for clients’ biologic programs, which helps our clients bring cutting-edge new drugs to patients faster."

Covance’s immunology and immunotoxicology experts, overseen by Shawn Heidel, D.V.M., Ph.D., vice president and global head, Safety Assessment, Metabolism and Lead Optimization, support and advise in the development of biologic projects from pre-clinical evaluation to new drug regulatory submission and clinical study sample analysis. The new I&I unit’s comprehensive offering also includes a seamless bridge to bioanalysis for use in regulatory submissions and to Covance Phase I clinical research units for first-in-human and first-in-patient studies. As studies progress, the I&I team can complete its unique set of solutions by drawing on additional expertise from Covance’s clinical development and commercialization, translational biomarker, immuno-oncology and central laboratory teams, as well as LabCorp’s specialist scientific and technical capabilities. The formation of Covance’s I&I unit illustrates LabCorp’s continued focus on biologics, which extends through LabCorp Diagnostics’ expanding suite of biologic therapeutic drug monitoring tests.

Covance’s initial investment in I&I capabilities focused on increasing global capacity by expanding dedicated laboratory space, state-of-the-art instrumentation and scientific and operations teams. In 2017 alone, Covance more than doubled its I&I laboratory footprint, including additional space in three locations and establishing a new innovation laboratory for method development and validation. Staff size has also doubled, driven by an increase in the number of scientists with advanced degrees. Covance’s recent investments in technology and innovation capabilities have enabled the business to further scale production, as well as support the development and implementation of new services within the rapidly growing biologics market.

"Our growing I&I team is capitalizing on the global experience of our established facilities in the U.S., Germany and the U.K. to deliver comprehensive support for large-molecule development, from innovative testing methods to insightful data analysis and interpretation," said Steve Street, Ph.D., senior vice president of Covance Early Development. "In addition to study design, development and direction, the global innovation team within our I&I unit will focus on new assay development and validation, keeping Covance at the forefront of biologic research and development."

On March 9, 2018 VBL Therapeutics (Nasdaq:VBLT), a clinical-stage biotechnology company focused on the discovery, development and commercialization of first-in-class treatments for cancer, reported that it will host a conference call and live audio webcast on Thursday, March 15 at 8:30am Eastern Time to report fiscal year ended December 31, 2017 financial results (Press release, VBL Therapeutics, MAR 9, 2018, View Source [SID1234524634]).

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On March 9, 2018 -X4 Pharmaceuticals, a clinical stage biotechnology company developing novel CXCR4 inhibitor drugs to improve immune cell trafficking to treat cancer and rare disease, reported that Paula Ragan, PhD, President and Chief Executive Officer, will present at the Cowen and Company 38th Annual Health Care Conference in Boston on Wednesday, March 14, 2018, at 8:00 a.m. Eastern time (Press release, , SEP 9, 2018, View Source [SID1234524617]).

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