On June 4, 2019 VBI Vaccines Inc. (NASDAQ: VBIV) ("VBI"), a commercial-stage biopharmaceutical company developing next-generation infectious disease and immuno-oncology vaccines, reported a poster on Sunday, June 2, 2019, at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting that exhibited expanded clinical data from Part A of the Phase 1/2a study of VBI-1901 in recurrent Glioblastoma (GBM) patients (Press release, VBI Vaccines, JUN 4, 2019, View Source [SID1234536896]).

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The poster presented data on a total of 18 patients enrolled in Part A of the study, which was a multi-center, open-label, dose-escalation study across three dose cohorts of VBI’s vaccine immunotherapeutic, VBI-1901 – 0.4 µg, 2.0 µg, and 10.0 µg. Part A was designed to evaluate the safety and tolerability of VBI-1901, and to define the optimal immunogenic dose level to test in the Part B extension phase of the study, which is expected to initiate enrollment mid-year 2019. Part B of the trial will further assess immunologic responses and potential correlations with tumor and clinical responses.

Andrew B. Lassman, M.D., Chief of Neuro-oncology at Columbia University Irving Medical Center and Associate Director for Clinical Infrastructure at Herbert Irving Comprehensive Cancer Center, and principal investigator of the study commented, "Though early, the data we’ve seen to-date in this Phase 1/2a study of VBI-1901 are intriguing, yet of course require confirmation in later phase and additional trials. The patients in this study, and more generally in the recurrent GBM setting, are immunocompromised and have very few effective treatment options available to them. Any treatment that could demonstrate even some benefit would be incredibly meaningful for these patients and their families. I look forward to seeing additional data from Part B of the study."

Highlights from Poster Presentation (Poster #237, Abstract #2048)

Safety:

The vaccine immunotherapeutic was well-tolerated at all doses, with no safety signals observed
Grade 2, 3, or 4 adverse events occurred in 66%, 22%, and 11% of participants, respectively – none were related to the vaccine immunotherapeutic

Immunogenicity and Tumor/Clinical Responses:

Six (6) patients immunologically responded to VBI-1901, with evidence of robust boosting of cytomegalovirus (CMV)-specific immune responses against both glycoprotein B (gB) and pp65 antigens.
Median progression-free survival (PFS) was longer among responders (14.5 weeks) vs. non-responders (6 weeks).
Three out of six (3/6) patients in the high-dose, 10 µg, cohort had evidence of stable disease (SD) by magnetic resonance imaging (MRI), compared to one out of six (1/6) in the low-dose cohort and zero out of six (0/6) in the intermediate-dose cohort.

"The tumor responses seen in three of the six patients in the high-dose cohort are promising, with all three having immunologic responses to VBI-1901 as well," said David E. Anderson, Ph.D., VBI’s Chief Scientific Officer. "In Part B of the study, we are narrowing the enrollment criteria to ensure a more homogenous patient population that may better assess the potential correlation between immunogenicity and tumor and clinical responses to VBI-1901. Enrollment of 10 patients in Part B, all first-recurrent GBM patients, is expected to initiate mid-year this year, 2019."

The full poster can be viewed on the "Events/Presentations" page in the Investor Section of the VBI Vaccines website.

VBI Press Release

About the Phase 1/2a Study Design

VBI’s two-part Phase 1/2a study is a multi-center, open-label, dose-escalation study of VBI-1901 in up to 28 patients with recurrent GBM:

Part A: Dose-escalation phase to define the safety, tolerability, and optimal dose level of VBI-1901 in recurrent GBM patients. This phase enrolled 18 patients across three dose cohorts.
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 is administered intradermally and is 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 receive the vaccine immunotherapeutic every four weeks until tumor progression.

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

On June 4, 2019 Sierra Oncology, Inc. (SRRA), a late-stage drug development company focused on advancing targeted therapeutics for the treatment of patients with significant unmet needs in hematology and oncology, reported that it has obtained regulatory clarity with the U.S. Food and Drug Administration (FDA) concerning the design of a Phase 3 clinical trial for momelotinib intended to support potential registration of this differentiated drug candidate for the treatment of previously JAK inhibitor treated myelofibrosis patients (Press release, Sierra Oncology, JUN 4, 2019, View Source [SID1234536864]). Following receipt of this clarity, Sierra also announced the design of the MOMENTUM Phase 3 clinical trial in myelofibrosis, planned for launch in Q4 2019.

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"We have held productive discussions with regulators in the US and EU, presenting a holistic analysis of momelotinib’s compelling array of positive efficacy and safety data observed in the two previously completed SIMPLIFY Phase 3 clinical studies, along with our strategy to conduct an additional Phase 3 trial intended to support momelotinib’s potential registration," said Dr. Nick Glover, President and CEO of Sierra Oncology. "We have been exceedingly pleased with the collaborative nature of these discussions which have culminated in alignment on the path to potential registration for momelotinib. Moreover, we have received constructive input that ensures that the design of the MOMENTUM Phase 3 study has the potential to generate compelling and persuasive clinical data capable of satisfying regulatory requirements."

"Momelotinib has consistently demonstrated clinically relevant benefits on the three hallmarks of myelofibrosis: symptoms, anemia and spleen enlargement," noted Dr. Srdan Verstovsek, MD, PhD, Professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center, Houston, Texas. "I have been involved in the development of momelotinib for many years, and I am very pleased to be named Chief Investigator of the MOMENTUM Phase 3 study. In my opinion, a good proportion of myelofibrosis patients in the second line setting would be candidates for momelotinib treatment due to its potential ability to improve both quality of life and anemia in a significant number of patients. As a myelofibrosis clinician, I can attest that we desperately need more treatment options that offer an array of distinct benefits for our patients. I look forward to momelotinib potentially becoming an important addition to the armamentarium in the treatment of myelofibrosis."

"We have designed MOMENTUM in order to generate highly persuasive clinical data with the potential to convincingly demonstrate momelotinib’s meaningful benefits on symptoms, anemia and spleen in the population of patients previously treated with a JAK inhibitor, as supplemented by both top-line and post hoc analyses of the prior SIMPLIFY Phase 3 datasets," said Dr. Barbara Klencke, Chief Development Officer, of Sierra Oncology. "We have outlined a robust, tractable study that we plan to launch in Q4 2019 and that we anticipate will yield top-line clinical data in Q4 2021."

About MOMENTUM Phase 3 Clinical Trial:
A Randomized, Double-Blind, Phase 3 Study to Evaluate the Activity of Momelotinib (MMB) versus Danazol (DAN) in Symptomatic, Anemic Subjects with Primary Myelofibrosis (PMF), Post-Polycythemia Vera (PV) Myelofibrosis, or Post Essential Thrombocythemia (ET) Myelofibrosis who were Previously Treated with JAK Inhibitor Therapy.

Sierra plans to launch the MOMENTUM Phase 3 clinical trial in Q4 2019. The randomized double-blind trial is designed to enroll 180 myelofibrosis patients who are symptomatic and anemic and have been treated previously with a JAK inhibitor. Patients will be randomized 2:1 to receive either momelotinib or danazol. Danazol has been selected as an appropriate treatment comparator given its use to ameliorate anemia in myelofibrosis patients, as recommended by NCCN and ESMO (Free ESMO Whitepaper) guidelines. After 24 weeks of treatment, patients on danazol will be allowed to crossover to receive momelotinib.

The Primary Endpoint of the trial is the Total Symptom Score (TSS) response rate of momelotinib compared to danazol at Week 24 (99% power; p-value < 0.05). Secondary and exploratory endpoints include:

Transfusion Independence (TI) rate at Week 24 (key secondary: > 90% powered; p-value < 0.05),
Splenic response rate (SRR) at Week 24 (> 90% powered; p-value < 0.05),
Duration of TSS response to Week 48,
Other measures of anemia benefit, including Transfusion Dependence response rate and various measures of cumulative transfusion burden,
Patient Reported Outcome measures of fatigue and physical function.
About Dr. Srdan Verstovsek, Chief Investigator of the MOMENTUM Phase 3 trial:
Dr. Srdan Verstovsek is Chief, Section for Myeloproliferative Neoplasms, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston. Dr. Verstovsek is a world-renowned physician-scientist, and a leading global authority on the treatment of myelofibrosis. His clinical and translational research is focused on understanding the biology of and developing new therapies for myeloproliferative neoplasms (MPNs). He has been Principal investigator for more than 50 clinical trials testing novel therapies for patients with MPNs, and has published more than 400 peer-reviewed manuscripts. He is the recipient of numerous awards including the Celgene 2010 Young Investigator Award, 7th Annual Irwin H. Krakoff Award for Excellence in Clinical and the Distinguished Lecturer Award from the Society of Hematologic Oncology and the Otis W. and Pearl L. Walters Faculty Achievement Award in Clinical Research by MD Anderson Cancer Center. He was made a Member of The American Society for Clinical Investigation in recognition of his contributions as a physician-scientist.

Momelotinib Analyst & Investor Conference Call
The company will be hosting an Analyst and Investor conference call at 6:00am ET on Wednesday, June 5, 2019, to discuss next steps for momelotinib.

Domestic (Toll Free- US): 1-800-239-9838
International (Toll): 1-323-794-2551
Conference ID: 8101895
Webcast Link: www.sierraoncology.com
Direct Link: View Source

Event registration and webcast information are available through the Sierra Oncology website at www.sierraoncology.com. An archive of the presentation will be accessible after the event through the Sierra Oncology website.

About Momelotinib
Momelotinib, Sierra’s lead drug candidate, is a potent, selective and orally-bioavailable JAK1, JAK2 & ACVR1 inhibitor with a differentiated therapeutic profile in myelofibrosis encompassing robust constitutional symptom improvements, a range of meaningful anemia benefits, including eliminating or reducing the need for frequent blood transfusions, and comparable spleen control to ruxolitinib. More than 1,200 subjects have received momelotinib since clinical studies began in 2009, including more than 800 subjects treated for myelofibrosis. Momelotinib is covered by patents anticipated to provide potential exclusivity to 2040 in the U.S.

On June 4, 2019 The Janssen Pharmaceutical Companies of Johnson & Johnson reported the submission of a Type II variation to the European Medicines Agency (EMA) seeking approval of ERLEADA (apalutamide) for the treatment of patients with metastatic hormone-sensitive prostate cancer (mHSPC), regardless of extent of disease or prior docetaxel treatment history (Press release, Johnson & Johnson, JUN 4, 2019, View Source [SID1234536881]). The submission is based on findings from the Phase 3 TITAN study which were presented at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and simultaneously published online in The New England Journal of Medicine.1,2

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The submission to the EMA follows the submission of supplemental registration dossiers to the U.S. Food and Drug Administration (FDA) on 29th April 2019 and to the Japanese Ministry of Health, Labour and Welfare (MHLW) on 31st May 2019 seeking approval of a new indication for apalutamide for the treatment of patients with mHSPC.3,4

"Today’s application seeking to expand the approval of apalutamide for the treatment of patients with mHSPC marks an important step in our continued focus and commitment to bring innovative medicines forward in the treatment of prostate cancer," said Dr. Joaquín Casariego, Janssen Therapeutic Area Lead Oncology for Europe, Middle East & Africa, Janssen-Cilag S.A. "We look forward to working with the EMA to expand access to this next-generation androgen receptor inhibitor for those patients who may benefit from this treatment in the future."

Results from the Phase 3 TITAN study showed patients with mHSPC, treated with apalutamide plus androgen deprivation therapy (ADT) significantly extended overall survival (OS) compared to placebo plus ADT with a 33 percent reduction in the risk of death (HR=0.67; 95% CI, 0.51-0.89; P=0.0053).2 In both study arms, median OS was not reached.2 Apalutamide plus ADT also significantly improved rPFS compared to placebo plus ADT with a 52 percent reduction in risk of radiographic progression or death compared to placebo plus ADT (HR=0.48; 95% CI, 0.39-0.60; P<0.0001).1 The median rPFS was 22.1 months for placebo plus ADT and not reached for apalutamide plus ADT.2 The two-year OS rates, after a median follow up of 22.7 months, were 82 percent for apalutamide plus ADT compared to 74 percent for placebo plus ADT.2

Adverse events (AEs) were generally consistent with the known apalutamide safety profile. The incidence of Grade 3/4 AEs for apalutamide plus ADT, versus placebo plus ADT were similar (42 percent vs 41 percent).2 The most common Grade ≥3 AEs for apalutamide plus ADT versus placebo plus ADT were hypertension (8.4 percent vs. 9.1 percent) and skin rash (6.3 percent vs. 0.6 percent).2 Additional reported Grade ≥3 AEs for apalutamide plus ADT versus placebo plus ADT were back pain (2.3 percent vs. 2.7 percent), blood alkaline phosphatase increased (0.4 percent vs. 2.5 percent) and anemia (1.7 percent vs. 3.2 percent).2 Treatment discontinuation due to AEs was 8 percent in the apalutamide arm compared to 5 percent in the placebo arm.1 Rash of any grade was more common among patients treated with apalutamide plus ADT, versus placebo plus ADT (27 percent vs 9 percent, respectively).2

In Europe, apalutamide is currently approved for use in adults with non-metastatic castration-resistant prostate cancer (nmCRPC) who are at high risk of developing metastatic disease.5 In the U.S. apalutamide is indicated for the treatment of nmCRPC.6

ENDS

About the TITAN Study1,2

TITAN is a Phase 3 randomised, placebo-controlled, double-blind study in men with mHSPC regardless of extent of disease or prior docetaxel treatment history. The study included 1,052 patients in intention-to-treat (ITT) population in 23 countries across 260 sites in North America, Latin America, South America, Europe and Asia Pacific. Patients with mHSPC were randomised 1:1 and received either apalutamide (240 mg) plus continuous androgen deprivation therapy (ADT) (n=525), or placebo plus ADT (n=527). The recruitment period for the study spanned from December 2015 to July 2017. The study included mHSPC patients with both low- and high-volume disease, those who were newly diagnosed, or those who had received prior definitive local therapy or prior treatment with up to six cycles of docetaxel or up to six months of ADT for mHSPC. Participants were treated until disease progression or the occurrence of unacceptable treatment-related toxicity. An independent data-monitoring committee was commissioned by the sponsor to monitor safety and efficacy before unblinding and make study conduct recommendations. Dual primary endpoints of the study were OS and rPFS. Secondary endpoints included time to cytotoxic chemotherapy, time to pain progression, time to chronic opioid use and time to skeletal-related event. Exploratory endpoints included time to PSA progression, time to second progression-free survival and time to symptomatic progression. For additional study information, visit ClinicalTrials.gov.

About ERLEADA

ERLEADA (apalutamide) is an androgen receptor (AR) inhibitor indicated for use in Europe for the treatment of patients with non-metastatic castration-resistant prostate cancer (nmCRPC) who are at high risk of developing metastatic disease.5 In the U.S. apalutamide is indicated for the treatment of nmCRPC.6

About Metastatic Hormone-Sensitive Prostate Cancer

Metastatic hormone-sensitive prostate cancer (mHSPC), also referred to as metastatic castration sensitive prostate cancer (mCSPC) refers to prostate cancer that still responds to androgen deprivation therapy (ADT) and has spread to other parts of the body.7 Patients with mHSPC tend to have a poor prognosis, with a median OS of less than five years, underscoring the need for new treatment options.8,9,10

On June 4, 2019 VBL Therapeutics (Nasdaq: VBLT), reported that Dr. Dror Harats, CEO will deliver a company presentation today at the 2019 BIO International Convention in Philadelphia (Press release, VBL Therapeutics, JUN 4, 2019, View Source [SID1234536865]). The presentation will include a discussion on the recent VB-111 progress, including data presented earlier this week at the 2019 ASCO (Free ASCO Whitepaper) Annual Meeting. The presentation will be also available to the investor audience by webcast.

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2019 BIO International Convention – Presentation Details

Date: June 4, 2019
Time: 3:30 pm EDT
Presentation Room: Theater 2
Location: Philadelphia Convention Center
Webcast: 2019 BIO International Convention Webcast

About VB-111 (ofranergene obadenovec)
VB-111, a potential first-in-class anticancer therapeutic candidate, is the Company’s lead oncology product currently being studied in the OVAL potential-registration Phase 3 pivotal trial for ovarian cancer (ClinicalTrials.gov Identifier: NCT03398655). VB-111 has received orphan drug designation in both the US and Europe, and fast track designation in the US for prolongation of survival in patients with rGBM. In addition, VB-111 successfully demonstrated proof-of-concept and survival benefit in Phase 2 clinical trials in radioiodine-refractory thyroid cancer and recurrent platinum-resistant ovarian cancer (NCT01711970). VB-111 has received an Orphan Designation for the treatment of ovarian cancer from the European Commission.

On June 4, 2019 Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE:CFBI), a biotechnology company with a pipeline of proprietary small molecule drugs that address cancer, liver and inflammatory diseases, reported that the Company’s VP of Business Development, Sari Fishman, is conducting one-on-one meetings with pharmaceutical companies for potential distribution and partnerships for the Company’s drug candidates, Piclidenoson and Namodenoson, at the BIO International Convention 2019 on June 3-6, 2019 in Philadelphia (Press release, Can-Fite BioPharma, JUN 4, 2019, View Source [SID1234536866]).

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Can-Fite’s near term milestones include announcing top line results from its Phase II study of Namodenoson in the treatment of NAFLD/NASH. The Company currently has out-licensing agreements in several territories and has received approximately $17 million in upfront and milestone payments to date.