On January 17, 2018 Onconova Therapeutics, Inc. (Nasdaq:ONTX), a Phase 3-stage biopharmaceutical company focused on discovering and developing small molecule drug candidates to treat cancer, reported that it is moving forward with its Phase 3 INSPIRE pivotal trial following the interim analysis, consistent with the DMC’s recommendation (Press release, Onconova, JAN 17, 2018, View Source [SID1234523237]). The DMC recommended continuation of the trial with a one-time expansion in enrollment, using a pre-planned sample size re-estimation, consistent with the Statistical Analysis Plan (SAP). The INSPIRE pivotal trial is studying intravenously-administered (IV) rigosertib in patients with higher-risk myelodysplastic syndromes (MDS) who have progressed on, failed to respond to, or relapsed after prior hypomethylating agent (HMA) therapy. The Company remains blinded to the interim analysis results.

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Guillermo Garcia-Manero, M.D, Professor and Chief of the MDS Section at the MD Anderson Cancer Center, a lead investigator on the INSPIRE study, commented, "Choices are very limited for higher risk MDS patients after failure of HMA therapy and no second-line therapy has ever been approved by the Health Authorities for these patients. These patients have a very short life-span and there is a tremendous unmet medical need. We remain highly supportive of Onconova’s efforts. After the interim analysis, continuation of the INSPIRE study is encouraging for patients."

The SAP for the INSPIRE trial featured an adaptive trial design, permitting several options following the interim analysis, which included continuation of the trial as planned, discontinuation of the trial for futility, trial expansion using pre-planned sample size re-estimation, and trial continuation for only the pre-defined treatment subgroup of patients classified as VHR based on the Revised International Prognostic Scoring System (IPSS-R).

The expanded INSPIRE study will continue to enroll eligible patients based on the current trial design of the overall ITT population and will increase enrollment by adding 135 patients to the original target to reach a total enrollment of 360 patients, with the aim of increasing the power of the trial. Due to the adaptive trial design and the DMC’s assessment, the INSPIRE trial will continue to analyze both the ITT and the VHR population for the primary endpoint of overall survival. The design of the trial with the expanded study enrollment will be identical to the current study design and will include the analysis of the overall survival endpoint in the ITT and the pre-specified VHR subgroup.

Steve Fruchtman, M.D., Chief Medical Officer of Onconova, added, "With no FDA approved therapies for many patients with higher-risk MDS who are refractory to HMAs, we are encouraged by these results and pleased to be at the forefront of advances in this treatment landscape. The DMC’s recommendation based on the pre-planned interim analysis includes the expansion of the INSPIRE trial and retains the analysis of survival in both the ITT and the VHR pre-defined subgroups. Patients with MDS who are refractory to HMAs have the highest unmet medical need due to an extremely poor prognosis following failure of HMA therapy. We look forward to completing enrollment and for the opportunity to analyze overall survival in the higher-risk MDS patients who have failed prior HMA therapy."

Currently, the INSPIRE study is active at approximately 175 trial sites in 22 countries across four continents, and has enrolled more than 170 patients. In Japan, patients have been enrolled to this study by SymBio Pharmaceuticals, our collaboration partner for Japan and Korea. Onconova believes that this trial is the most advanced study for a new therapeutic agent in this indication, and there are no FDA approved therapies specifically for MDS patients after failure of front-line HMAs.

The Company will host a conference call today, January 17th at 8:30 a.m. Eastern Time to discuss the interim results and answer any questions. Interested parties may access the call by dialing toll-free (855) 428-5741 from the US, or (210) 229-8823 internationally and using conference ID: 8899928.

The call will also be webcast live. Please click here to access the webcast.

A replay will be available at this link until April 30, 2018.

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