On January 17, 2018 RXi Pharmaceuticals Corporation (NASDAQ: RXII) a clinical-stage company developing a new class of RNAi-based therapeutics reported that it will give an oral presentation highlighting the company’s proprietary therapeutic platform at the Immuno-Oncology Frontiers World conference (Press release, RXi Pharmaceuticals, JAN 17, 2018, View Source [SID1234523194]). This exclusive Immuno-oncology (I-O) partnering event provides direct access to decision makers and KOL’s working towards the next oncology blockbuster. Also, this is the only I-O event co-located with the leading Cell and Gene Therapy global meeting; providing access to the most commercially advanced technical expertise specifically for cellular immunotherapies. The conference will take place January 22-25, 2018 at the Hyatt Regency in Miami, Florida.

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Title: Targeting Immune Checkpoints Using Self-Delivering RNAi (sd-rxRNA) Technology
Session: Stream 2- Emerging Science: Reprogramming the Tumor Microenvironment – Conquering Immune Suppressive Molecular Pathways
Date and Time: Tuesday, January 23, 2018, at 11:50 am ET
Presenter: James Cardia, Ph.D., Director of Business Development and Intellectual Property

The presentation will be available on the Company’s website approximately 1 hour after the presentation at www.rxipharma.com.

Logo – View Source

About RXi’s self-delivering RNAi (sd-rxRNA) technology platform

sd-rxRNA, RXi’s proprietary self-delivering RNAi platform, is a single chemically modified compound with delivery and therapeutic properties built directly into the compound itself. The compound is asymmetrical with a phosphorothioate backbone and contains chemical modifications that provide for efficient cellular uptake and gene silencing. These compounds are potent, stable and specific, and demonstrated to be safe and active in a clinical setting.

RXi’s novel sd-rxRNA technology differs from natural and most synthetic RNA interference (RNAi) molecules in that they are chemically modified to allow for efficient internalization of the compounds by cells and silencing of the targeted genes. Importantly, unlike other naked siRNA compounds, delivery of sd-rxRNAs are not limited to a specific cell type. For local delivery and ex vivo cell-based therapeutic applications, our compounds do not require delivery vehicles. This is a major advantage since delivery vehicles can have related toxicity that affects cell viability. sd-rxRNA has demonstrated nearly 100 percent transfection efficiency with high cell viability in numerous cell types.

On January 17, 2018 VBI Vaccines Inc. (Nasdaq:VBIV) (TSX: VBV) (VBI), a commercial-stage biopharmaceutical company developing next-generation infectious disease and immuno-oncology vaccines, reported that the first patient has been dosed in a Phase 1/2a clinical study of VBI-1901 for the treatment of recurrent glioblastoma multiforme (rGBM) (Press release, VBI Vaccines, JAN 17, 2018, View Source [SID1234523238]). The multi-center, open-label, two-part study will enroll up to 28 patients and is designed to evaluate safety, tolerability, and the optimal therapeutic dose level of VBI-1901.

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"We are excited to announce that the first patient has been dosed in this initial clinical study of VBI-1901, our first clinical study in immuno-oncology," said Jeff Baxter, President and CEO of VBI. "Recurrent GBM is a devastating CMV-associated tumor with few effective treatment options. We developed VBI-1901 to target two highly immunogenic CMV antigens, and, based on preclinical studies, we believe it has the potential to induce a strong anti-tumor immune response in these patients."

About the Phase I/IIa Study Design

This two-part Phase 1/2a study is a multi-center, open-label, dose-escalation study of VBI-1901 in approximately 28 patients with rGBM:

● Part A: Dose-escalation phase to define the safety, tolerability, and optimal dose level of VBI-1901 in rGBM patients. This phase is expected to enroll up to 18 patients.

● Part B: A subsequent extension of the optimal dose level, as defined in the dose escalation phase. This phase is expected to enroll an expanded cohort of approximately 10 additional patients.

VBI-1901 will be administered intradermally and will be adjuvanted with granulocyte-macrophage colony-stimulating factor (GM-CSF), a potent adjuvant that mobilizes dendritic cell function. Patients in both phases of the study will continue to receive vaccine every four weeks until tumor progression.

The study will take place at two leading sites in the U.S. – NewYork Presbyterian-Columbia University Medical Center in New York City, New York, and Inova Health System in Falls Church, Virginia. Andrew Lassman, MD, the John Harris Associate Professor of Neurology and Chief of Neuro-oncology at Columbia University, has been named as the lead investigator.

Additional information, including a detailed description of the study design, eligibility criteria, and investigator sites, is available at ClinicalTrials.gov using identifier NCT03382977.

About VBI-1901 and GBM

VBI-1901 is a novel immunotherapy developed using VBI’s eVLP technology to target two highly immunogenic cytomegalovirus (CMV) antigens, gB and pp65. Scientific literature suggests CMV infection is prevalent in multiple solid tumors, and recent research has demonstrated that an anti-CMV dendritic cell vaccination regimen may extend overall survival in patients with GBM. Additionally, recent preclinical studies confirmed that VBI-1901 may be a potent, "off-the-shelf" therapeutic vaccine.

Glioblastoma is among the most common and aggressive malignant primary brain tumors in humans. In the U.S. alone, 12,000 new cases are diagnosed each year. The current standard of care for treating GBM is surgical resection, followed by radiation and chemotherapy. Even with aggressive treatment, GBM progresses rapidly and is exceptionally lethal, with median patient survival of less than 16 months.

To learn more about VBI-1901, visit: View Source

On January 17, 2018 CTI BioPharma Corp. (NASDAQ and MTA:CTIC) reported that it expects to receive a $10 million milestone payment in February, 2018 from Teva Pharmaceutical Industries Ltd. related to the achievement of a milestone for U.S. Food and Drug Administration approval of TRISENOX (arsenic trioxide) for first line treatment of acute promyelocytic leukemia (Press release, CTI BioPharma, JAN 17, 2018, View Source;p=RssLanding&cat=news&id=2327170 [SID1234523276]). The milestone will be paid pursuant to an acquisition agreement for TRISENOX previously entered into with Teva under which CTI BioPharma is eligible to receive up to an additional $50 million in payments upon achievement by Teva of specified sales and development milestones related to TRISENOX.

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On January 17, 2018 NanOlogy reproted that it will Present at Drug Development & Delivery Networking Summit (Press release, NanOlogy, JAN 17, 2018, View Source [SID1234523294]).

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Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Event: Drug Development & Delivery Networking Summit
Dates: March 8, 2018
Location: Parsippany, NJ
Event Website: View Source

On January 17, 2018 Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development and commercialization of innovative and effective therapeutics for the treatment of cancer, reported initiation of patient dosing in a Phase 1 trial of CA-4948, an orally available small molecule inhibitor of the IRAK4 kinase, for treatment of patients with lymphoma (Press release, Curis, JAN 17, 2018, View Source [SID1234523297]). CA-4948 was discovered at Aurigene and is the second licensed program from the Curis-Aurigene collaboration to enter the clinic.

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The Phase 1 study is designed to evaluate the safety, tolerability, and pharmacokinetic profile of CA-4948; identify any dose-limiting toxicities; and establish the recommended Phase 2 dose for the treatment of patients with lymphomas. The dose escalation stage of the trial will enroll patients with relapsed/refractory non-Hodgkin’s lymphoma, and the expansion stage will focus on specific populations of patients with lymphomas harboring alterations in the MYD88 gene or Toll-like receptor (TLR) signaling pathway.

"We are pleased to announce the advancement of our clinical pipeline with the initiation of the Phase 1 trial for this selective IRAK kinase inhibitor in patients with lymphomas," commented Ali Fattaey, Ph.D., Curis’s president and CEO. "Given the prevalence of activating mutations in the MYD88 gene and the TLR pathway, IRAK4 represents a significant target for the precision treatment of patients with different hematologic malignancies. In addition to its preclinical anti-tumor activity in MYD88-mutated lymphomas, we have observed encouraging effects of CA-4948 in animal models of acute myeloid leukemia (AML), myelodysplastic syndromes (MDS) as well as non-oncology inflammatory disease models. We look forward to working with our partner Aurigene to develop CA-4948 and explore clinical opportunities for these conditions in the near future."

"We are excited to have enrolled the first patient in this lymphoma clinical trial and look forward to further investigating CA-4948 as a potential new treatment option for patients with hematologic malignancies," said Mathew Lunning, DO, University of Nebraska Medical Center, an investigator for the study.

"We are delighted with our collaboration that has led to the advancement of the second program into the clinic, an IRAK4 targeting molecule that came out of Aurigene’s discovery efforts over many years," said CSN Murthy, Aurigene’s CEO. "Our investment into Curis exhibits our belief and commitment for this program and beyond as we work with Curis to focus our collective resources to advance exciting drug candidates."

About CA-4948, a Small-Molecule Inhibitor of IRAK4 Kinase

Innate immune responses orchestrated through Toll-like receptors are important mediators of the body’s initial defense against infections, while their dysregulation is associated with certain inflammatory conditions. Toll-like receptor signaling through the adaptor protein MYD88 results in the activation of IRAK4, initiating a signaling cascade that induces cytokine and survival factor expression. MYD88 gene mutations occur in approximately 30 percent of activated B-cell subtype of diffuse large B-cell lymphomas (DLBCL)1,2 and in over 90 percent of cases of the B-cell malignancy Waldenstrom’s macroglobulinemia (WM).3 IRAK4 has been validated as a target in DLBCL and WM disease setting, and its inhibition by CA-4948 has been shown to provide potent in vivo anti-tumor activity in animal models.4,5 IRAK4 inhibitors are also in clinical testing for treatment of patients with rheumatoid arthritis.

1Nature. 2011; 470(7332):115–119
2Immunology and Cell Biology. 2011; 89(6):659–660
3N Engl J Med. 2012; 367(9):826–833
4Cancer Res. 2017; 77(13 Suppl): Abstract 1168
5Blood. 2015;126(23):4004–4004