On November 9, 2018 Innovent Biologics, Inc. (Innovent) (HKEX: 1801), a world-class China-based biopharmaceutical company that develops and commercializes high quality drugs, reported its clinical research data on cohort E from a Phase Ib clinical trial at the International Association for the Study of Lung Cancer (IASLC) Asia Conference on Lung Cancer 2018 (#ACLC18) (Press release, Innovent Biologics, NOV 9, 2018, View Source [SID1234531216]). In this cohort of the Phase Ib clinical trial (NCT02937116), patients with first-line squamous non-small cell lung cancer (sNSCLC) were treated with sintilimab, a fully human anti-programmed cell death 1 (PD-1) monoclonal antibody, in combination with gemcitabine and cisplatin.

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The combination demonstrated an objective response rate (ORR) of 64.7% and disease control rate (DCR) of 100.0%, based on data from 17 patients with at least one radiological assessment among a total of 20 patients in this cohort. As of the data analysis cutoff on September 1, 2018, after median follow up of 6.6 months, the data of median duration of response (DOR) and median progression free survival (PFS) were not yet mature, the preliminary results of which were 6.0 months and 6.8 months, respectively. Twelve-month overall survival (OS) was 87.0%. The study shows evidence of anti-tumor efficacy and an acceptable safety profile.

Based on the efficacy and safety profile from this early phase clinical trial, we have initiated ORIENT-12, a randomized, double-blinded, multicenter, phase III study of sintilimab versus placebo, both in combination with gemcitabine and platinum-based chemotherapy as first-line treatment for advanced or recurrent sNSCLC in China. Patient recruitment for this study is currently under way and Innovent plans to enroll 348 patients. The first patient dosing has been accomplished recently.

"Immune checkpoint inhibitor has brought hope to patients suffering from squamous non-small cell lung cancer, who are unfit for either target therapy due to its lack of driving gene mutation, or anti-angiogenic therapy in first line setting," said Professor Kejing Ying from Sir Run Run Shaw Hospital, Zhejiang University School of Medicine. She expressed her great expectation for the result from phase III clinical trial and the approval of sintilimab as first-line treatment for NSCLC.

"Lung cancer has the highest morbidity and mortality among malignant tumors in China. Despite breakthrough in recent years, treatment for squamous lung cancer is still limited, and the effect is unsatisfying," said Michael Yu, Founder, Chief Executive Officer and Chairman of Innovent, "At present, Innovent has launched phase III clinical study of squamous lung cancer. We hope these clinical trials will benefit squamous lung cancer patients as quickly as possible, bringing patients and their families hope for extended vitality."

About advanced or metastatic squamous non-small cell lung cancer (sNSCLC)

Lung cancer has the highest incidence and mortality among all malignancies in China. NSCLC accounts for about 80% to 85% of all lung cancer cases, and about 70% of NSCLC patients have locally advanced or metastatic disease at first diagnosis. In addition, many patients with early-stage NSCLC after undergoing potentially curative surgery eventually die from disease progression after disease recurrence or distant metastasis. Squamous NSCLC (sNSCLC) accounts for about 30% of all NSCLC patients in China. Due to its unique epidemiological, histological and molecular biological character, sNSCLC still lacks effective treatment modality, with platinum-based chemotherapy as the first-line treatment. However, platinum-based chemotherapy only obtains an objective response rate (ORR) of 30%, progression free survival (PFS) of 5.5 months and overall survival (OS) of 10.8 months, which is far from satisfying. The unmet medical needs in this patient population are high.

About ORIENT-12

ORIENT-12 is a randomized, double-blinded, multicenter, phase III study of sintilimab versus placebo, both in combination with gemcitabine and platinum-based chemotherapy as first-line treatment for advanced or recurrent sNSCLC in China. Patient recruitment for this study is currently under way and Innovent plans to enroll 348 patients. The first patient dosing has been accomplished recently.

About Sintilimab

Sintilimab is a fully human anti-PD-1 antibody. It binds to the PD-1 receptor on T cells, blocking the PD-L1 ligand from interacting with PD-1 to help restore T-cell response and immune response, thus destroying the tumor cells. Sintilimab is an anti-PD-1 monoclonal antibody jointly developed by Innovent and Eli Lilly and Company in China. National Medical Products Administration (NMPA, successor to CFDA) accepted the New Drug Application (NDA) submitted by Innovent for sintilimab on April 16, 2018, and granted it priority review status on April 23, 2018. The indication for the first new drug application is relapsed/refractory classical Hodgkin’s Lymphoma.

On November 9, 2018 Leap Therapeutics, Inc. (NASDAQ: LPTX) reported its clinical data from its ongoing Phase I/II study of DKN-01 in combination with paclitaxel in patients with advanced esophagogastric cancer at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 33rd Annual Meeting (SITC 2018) (Press release, Leap Therapeutics, NOV 9, 2018, View Source [SID1234531215]).

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The esophagogastric cancer clinical trial (P102) is a multipart study of DKN-01 as a monotherapy and in combination with paclitaxel or pembrolizumab. The arms evaluating DKN-01 plus paclitaxel enrolled fifty-nine patients who had received one to seven prior lines of therapy and were designed to assess the safety, pharmacokinetics and efficacy of the combination.

The combination of DKN-01 and paclitaxel generated a 46.7% overall response rate, 19.6 weeks median progression free survival, and 61.1 weeks median overall survival in fifteen evaluable patients as a second line therapy. In the benchmark RAINBOW study, paclitaxel, as a monotherapy in second line gastroesophageal junction or gastric cancer patients, generated a 16.1% overall response rate, 2.9 months of median progression free survival, and 7.4 months median overall survival.

In addition, in the subgroup of twelve evaluable patients with heavily pre-treated esophageal squamous cell carcinoma, the combination of DKN-01 and paclitaxel produced a 33.3% overall response rate, 13.7 weeks median progression free survival, and 31.0 weeks median overall survival.

"The combination of DKN-01 and paclitaxel has generated a promising signal in second-line esophagogastric cancer patients, with clinically meaningful response rate, progression-free survival and overall survival data. In addition, there were encouraging outcomes in patients with esophageal squamous cell carcinoma, who have a significant unmet medical need and no approved therapies," commented John H. Strickler, MD, Assistant Professor of Medicine, Duke University Cancer Institute. "As GI oncologists, we are looking to improve upon the standard single agent therapies, and the combination of DKN-01 and paclitaxel merits further exploration in randomized clinical trials."

Preclinical data also presented at SITC (Free SITC Whitepaper) 2018 described the mechanism of action of DKN-01 and immune mediated anti-tumor activity in nonclinical models. A murine version of DKN-01 (mDKN-01) demonstrated efficacy in a melanoma tumor model. However, mDKN-01 was unable to impede tumor growth in immunodeficient mice, indicating that a functioning immune system is required for antibody activity. Specifically, the activity of DKN-01 was shown to require NK cells but not T or B cells, representing activation of the innate immune system. In preclinical models, the innate immune system activity of DKN-01 complemented the activity of paclitaxel and anti-PD-1 antibodies.

"To improve outcomes for patients with esophagogastric cancer, we need to develop drugs with new mechanisms of actions. The effect of DKN-01 on the innate immune system provides a strong rationale for enhanced activity in combination with chemotherapy and checkpoint inhibitors," commented Victoria M. Villaflor, MD, Associate Professor of Medicine, Northwestern University. "The DKN-01 clinical data with paclitaxel and as previously presented with Keytruda reflects the clinical translation of these complementary mechanisms and should be studied further as an important new approach for treating esophagogastric cancer patients."

On November 9, 2018 Nektar Therapeutics (Nasdaq: NKTR) reported a presentation of new clinical and preclinical data for its I-O pipeline at the 2018 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting (Press release, Nektar Therapeutics, NOV 9, 2018, View Source [SID1234531214]).

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New clinical study results from the PIVOT-02 Phase 1/2 Study were shared in an oral presentation titled, "Immune monitoring after NKTR-214 plus nivolumab (PIVOT-02) in previously untreated patients with metastatic Stage IV melanoma" (Abstract #O4) by Adi Diab, MD, Assistant Professor, Department of Melanoma Medical Oncology, Division of Cancer Medicine, University of Texas MD Anderson Cancer Center during the Cytokines Reinvented Session on Friday, November 9th.

Additional preclinical data presented at the annual meeting showed that NKTR-214 may drive, sustain and expand anti-tumor response when combined with therapies with complementary mechanisms of action including NKTR-262, poly (ADP-ribose) polymerase (PARP) inhibitors, radiation therapy (RT) and other agents. When combined with these treatments, NKTR-214 showed the potential to lead to tumor clearance and tumor specific immunologic memory.

"The data presented at this year’s SITC (Free SITC Whitepaper) Annual Meeting showcase our pipeline of novel investigational I-O agents that target key components of the immune cycle in order to restore immune surveillance and harness the body’s immune system to fight cancer," said Jonathan Zalevsky, Ph.D., Senior Vice President and Chief Scientific Officer at Nektar Therapeutics. "Clinical data presented for NKTR-214 show that NKTR-214 plus nivolumab give deep and durable responses in first-line IO-naive Stage IV melanoma patients, including a high rate of complete responses. For our TLR agonist candidate, NKTR-262, we presented data demonstrating alteration of the tumor micro-environment, including activating the innate and adaptive arms of the immune system along with encouraging anti-tumor activity."

Highlights from the oral presentation on Stage IV 1L Melanoma patients include:

Clinical Efficacy (Response measured per RECIST 1.1 by central independent radiology review for efficacy-evaluable patients treated at the recommended Phase 2 dose and with >1 on treatment scan). Response and median time on study calculated from data cut as of October 1, 2018:

Confirmed best overall response rate (ORR) was 53% (20/38) in efficacy-evaluable patients, with a 24% (9/38) complete response (CR) rate. Median time of follow-up was 7.2 months and median time to response was 2 months. 85% (17/20) patients with responses have ongoing responses. DCR, also known as disease control rate (CR+ PR + SD) was 76%.
Amongst the 33 patients with known pre-treatment PD-L1 status, ORR in PD-L1 negative patients was 6/14 (43%) and in PD-L1 positive patients was 13/19 (68%). One patient with unknown PD-L1 baseline status experienced a CR.
Clinical Safety (1L Melanoma safety database as of October 1, 2018):

A total of 41 patients have been treated at the RP2D. The most common (>30%) treatment-related adverse events (TRAEs) were grade 1-2 flu-like symptoms (78%), rash (70.7%), fatigue (63.4%), pruritus (46.3%), nausea (43.9%), arthralgia (36.6%) and myalgia (31.7%). A total of 8/41 (19.5%) of patients experienced a Grade 3 (G3) or higher TRAE with 2/41 (4.9%) patients discontinuing treatment due to a TRAE. 2/41 (4.9%) of patients experienced a G3 or higher immune-mediated AE.
A decreased frequency of Grade 1-2 cytokine related AEs was observed with continuous dosing.
Biomarkers and Mechanism of Action:

Clear activation of the IL-2 pathway demonstrated by an increase in absolute lymphocyte count with activated and proliferating CD4, CD8 and NK cells in blood.
Combination demonstrated T cell infiltration and activation in the tumor microenvironment.
TCR repertoire analysis demonstrates the presence of newly trafficked clonal infiltrates after treatment with NKTR-214 plus nivolumab.
Opdivo is a programmed death-1 (PD-1) immune checkpoint inhibitor that is designed to uniquely harness the body’s own immune system to help restore anti-tumor immune response. By harnessing the body’s own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers. NKTR-214 is an investigational immuno-stimulatory therapy designed to expand and activate specific cancer-fighting T cells and natural killer (NK) cells directly in the tumor micro-environment and increase expression of cell-surface PD-1 on these immune cells. A Phase 3 trial evaluating NKTR-214 in combination with nivolumab versus nivolumab in first-line advanced melanoma patients is currently recruiting patients (NCT03635983).

A copy of Dr. Diab’s presentation of PIVOT-02 data is available on Nektar’s corporate website at View Source

Details of the preclinical poster presentations at SITC (Free SITC Whitepaper) are as follows and each will be available for download at the time of presentation at View Source

Poster/Abstract #P364: "Systemic anti-tumor immunity and immune memory formation by a novel TLR7/8 targeting agent NKTR-262 combined with CD122-biased immunostimulatory cytokine NKTR-214", Kivimae, S., et al.

NKTR-262 and NKTR-214 in combination showed efficacy in all tested preclinical tumor models, correlated with sustained systemic expansion of tumor antigen specific CD8+ T cells.
Combining NKTR-262 with NKTR-214 coordinates tumor antigen presentation and costimulatory signaling with tumor antigen recognizing CD8+ T cell expansion to produce a sustained systemic anti-tumor immune response.
Poster/Abstract #P378: "NKTR-214 (CD122-biased agonist) and NKTR-262 (TLR7/8 agonist) combination treatment pairs local innate immune activation with systemic CD8+ T cell expansion to enhance anti-tumor immunity", Rolig, A., et al.

NKTR-214 and NKTR-262 resulted in systemic CD8+ T cell expansion, enhanced intratumoral CD8+ T cell effector function, and favorable myeloid polarization.
This robust anti-tumor immunity resulted in improved tumor regression and tumor-free survival in preclinical models.
Poster/Abstract #P368: "Combination of a Dipeptidyl Peptidase Inhibitor BXCL701 and Biased CD122 Agonist NKTR-214 with Anti-PD1 Provides Functional Immunological Memory through Inflammatory Cell Death", MacDougall, J., et al.

In a triple combination with NKTR-214 and an anti-PD-1 antibody, BXCL701 generated complete and durable responses in models with high densities of tumor associated. macrophages, validating the importance of engaging multiple cell types of the immune system required for complete anti-tumor response.
Poster/Abstract #P348: "Survival and immune modulation in homologous recombination deficient murine ovarian tumors using the PARP inhibitor, rucaparib and immune agonist, NKTR-214", Charych, D., et al.

NKTR-214 paired with rucaparib provided significantly increased survival and durable complete response than either treatment alone in murine models of ovarian cancer.
Preclinical results suggest the activity of this combination is through antigen priming of infiltrating memory T cells, increased NK cell recruitment and enhanced cytotoxicity of tumor infiltrates.
Poster/Abstract #P418: "Pre-clinical investigation of NKTR-255, a polymer-conjugated IL-15 with a potent NK cell dependent anti-tumor efficacy", Miyazaki, T., et al.

NKTR-255 demonstrated powerful immune stimulation of NK cells with dose-dependent effect in the proliferation and activation of NK cells and enhanced anti-metastatic activity in mouse lung metastasis models.
Results suggest NKTR-255 has high promise as a novel anti-tumor agent that boosts NK cell expansion and survival.
Poster/Abstract #P419: "NKTR-214 in combination with radiation produces a potent in situ vaccine in the syngeneic B78 melanoma model", Sondel, P., et al.

In a murine melanoma model study, the combination of NKTR-214 and radiation therapy (RT) resulted in significant tumor regression, higher rates of complete response, and stronger immunologic memory compared with radiation RT, RT plus IL-2, or NKTR-214 alone.
Poster/Abstract #P422: "A polymer-associated human IL-15 (NKTR-255) has optimized biological activity and unique mechanisms of action on CD8 T Cells and NK Cells", Robinson T., et al.

Preclinical findings highlighted the ability of NKTR-255 to impact different mechanisms of action on CD8 T cells and NK cells leading to novel therapeutic effects.
Poster/Abstract #P424: "NKTR-214, an engineered IL-2, selectively depletes intratumoral Tregs and expands immunotherapy-induced effector T cell responses", Sharma, M., et al.

NKTR-214 showed synergy with both checkpoint blockade and peptide-vaccination resulting in improved overall survival and cure of mice in models of colon carcinoma and melanoma.
The synergistic mechanism led to proliferation and tumor infiltration of effector CD8+ T cells while promoting selective intratumoral depletion of Tregs to establish effective anti-tumor immunity.
Poster/Abstract #P557: "Overcoming genetically-based resistance mechanisms to PD-1 blockade", Torrejón, D., et al.
[NOTE: These data were also presented in an oral presentation titled "Overcoming genetically-based resistance mechanisms to PD-1 blockade" by Davis Torrejón, M.D., UCLA Hematology-Oncology, during the Rapid Oral Abstract Presentations Session on Friday, November 9th.]

Genetic models of resistance to anti-PD-1 therapy were assessed using JAK1, JAK2, and B2M knockout (KO) models.
NKTR-214 overcame resistance to anti-PD-1 in the B2M-KO tumor model and significantly increased survival.
Analyst Call with Melanoma Specialists
Nektar will webcast an analyst and investor conference call with melanoma specialists and company management on Saturday, November 10, 2018 at 9:00 a.m. EST in Washington, D.C. during the 2018 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting. The event follows today’s oral presentation of new efficacy, safety and immune monitoring data from the first-line Stage IV metastatic melanoma patient cohort in the PIVOT-02 study of NKTR-214 in combination with nivolumab.

Date and Time: Saturday, November 10, 2018 at 9:00 a.m. EST
Dial- in: 877-881-2183 (toll-free) or 970-315-0453 (enter access code 7865989)

Clinical investigators on the conference call will include Dr. Harriet Kluger, Professor of Medicine, Medical Oncology at the Yale Cancer Center and Dr. Adi Diab, Assistant Professor, Melanoma Medical Oncology at the University of Texas MD Anderson Cancer Center. Investors and analysts can also view slides and listen to the live audio webcast of the presentation at View Source The event will also be available for replay for two weeks on the company’s website, www.nektar.com.

About NKTR-214
NKTR-214 is a CD122-biased agonist designed to stimulate the patient’s own immune system to fight cancer. NKTR-214 is designed to grow specific cancer-killing T cells and natural killer (NK) cell populations in the body which fight cancer, which are known as endogenous tumor-infiltrating lymphocytes (TILs). NKTR-214 stimulates these cancer-killing immune cells in the body by targeting CD122 specific receptors found on the surface of these immune cells, known as CD8+ effector T cells and Natural Killer (NK) cells. CD122, which is also known as the Interleukin-2 receptor beta subunit, is a key signaling receptor that is known to increase proliferation of these effector T cells.1 In clinical and preclinical studies, treatment with NKTR-214 resulted in expansion of these cells and mobilization into the tumor micro-environment.2,3 NKTR-214 has an antibody-like dosing regimen similar to the existing checkpoint inhibitor class of approved medicines.

About NKTR-262
Cancer treatments that couple pharmacological activation of tumor antigen presentation with activation and expansion of CD8+ T and natural killer (NK) cells in the tumor environment have the potential to induce an effective anti-tumor immune response in patients. NKTR-262 is a novel small molecule agonist designed to activate toll-like receptors (TLRs). Intratumoral delivery of NKTR-262 promotes TLR activation to induce the development of antigen-specific immunity by initiating the process by which the immune system generates antigen-specific cytotoxic T cells to the patient’s specific tumor.4 NKTR-214 targets CD122 specific receptors found on the surface of these cancer-killing immune cells, known as CD8+ effector T cells. By first generating antigen-specific cytotoxic T cells with NKTR-262 and then growing these CD8+ effector T cells with NKTR-214, the patient’s entire immunity cycle can potentially be engaged to fight cancer. In preclinical studies, a single intratumoral dose of NKTR-262, administered in combination with NKTR-214, resulted in complete abscopal tumor regressions in multiple mouse syngeneic tumor models.5

About NKTR-255
NKTR-255 is a memory T cell stimulating cytokine designed to engage the IL-15 pathway to induce long-term T cell activation and improve the quality of T cell memory response to treat cancer. Through optimal engagement of the IL-15Rα/IL-2Rγ receptor complex, NKTR-255 stimulates proliferation and survival of CD8+ T cells, natural killer (NK) cells and enhances formation of long-term immunological memory which may lead to sustained anti-tumor immune response. Native rhIL-15 is rapidly cleared from the body and must be administered frequently and in high doses limiting its utility due to toxicity. NKTR-255 is designed with IL-15 receptor alpha specificity to optimize biological activity and is uniquely engineered to provide optimal exposure and an improved safety profile.

On November 9, 2018 Fortress Biotech, Inc. (NASDAQ: FBIO) ("Fortress"), a biopharmaceutical company dedicated to acquiring, developing and commercializing novel pharmaceutical and biotechnology products, reported financial results and recent corporate highlights for the third quarter ended September 30, 2018 (Press release, Fortress Biotech, NOV 9, 2018, View Source [SID1234531206]).

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Lindsay A. Rosenwald, M.D., Fortress’ Chairman, President and Chief Executive Officer, said, "Fortress and our subsidiaries continued to achieve important corporate and clinical milestones in the third quarter of 2018. Mustang Bio expanded its pipeline into gene therapy by securing an exclusive worldwide license for the development of a potentially first-in-class ex vivo lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency ("X-SCID") from St. Jude Children’s Research Hospital ("St. Jude"). Additionally, Checkpoint Therapeutics announced positive interim safety and efficacy data from its Phase 1/2 clinical trial of CK-101, a third-generation epidermal growth factor receptor ("EGFR") inhibitor being evaluated in advanced non-small cell lung cancer ("NSCLC"). Finally, Cyprium Therapeutics’ product candidate for patients diagnosed with classic Menkes disease, CUTX-101, was granted Fast Track Designation by the U.S. Food and Drug Administration ("FDA"). We plan to continue to acquire and develop compelling and, in some instances, potentially life-saving product candidates, which could lead to maximizing shareholder value."

Financial Results:

· As of September 30, 2018, Fortress’ consolidated cash, cash equivalents, short-term investments (certificates of deposit), cash deposits with clearing organizations and restricted cash totaled $136.1 million, compared to $168.3 million as of December 31, 2017, a decrease of $32.2 million year-to-date.
· Net revenue totaled $63.7 million for the third quarter of 2018, compared to $46.9 million for the third quarter of 2017. Total revenue as of September 30, 2018 includes $5.2 million of Fortress revenue, primarily from the sale of Journey Medical Corporation products, and $58.5 million of revenue from National Holdings Corporation1 ("National Holdings"). Total revenue as of September 30, 2017 included $2.5 million of Fortress revenue and $44.4 million of revenue from National Holdings.
· Research and development expenses were $16.1 million for the third quarter of 2018, of which $15.1 million was related to Fortress Companies. This compares to $15.9 million for the third quarter of 2017, of which $14.2 million was related to Fortress Companies. Non-cash, stock-based compensation expenses included in research and development were $1.8 million for the third quarter of 2018, compared to $1.6 million for the third quarter of 2017.
· Research and development expenses from license acquisitions were $3.7 million for the third quarter of 2018, compared to $0.3 million for the third quarter of 2017.
· General and administrative expenses were $12.2 million for the third quarter of 2018, of which $7.4 million was related to Fortress Companies. This compares to $15.1 million for the third quarter of 2017, of which $10.5 million was related to Fortress Companies. Non-cash, stock-based compensation expenses included in general and administrative expenses were $2.3 million for the third quarter of 2018, compared to $2.6 million for the third quarter of 2017.
· National Holdings’ operating expenses totaled $55.2 million for the third quarter of 2018, compared to $47.7 million for the third quarter of 2017.
· Net loss attributable to common stockholders was $16.6 million, or $0.37 per share, for the third quarter of 2018, compared to a net loss attributable to common stockholders of $27.1 million, or $0.67 per share, for the third quarter of 2017. For the first nine months of 2018, net loss attributable to common stockholders was $59.3 million or $1.36 per share, compared to $56.5 million or $1.39 per share in the first nine months of 2017.

On November 9, 2018 VBI Vaccines Inc. (NASDAQ: VBIV) ("VBI"), a commercial-stage biopharmaceutical company developing next-generation infectious disease and immuno-oncology vaccines, reported financial results for the third quarter ending September 30, 2018, and provided a corporate update (Press release, VBI Vaccines, NOV 9, 2018, View Source [SID1234531205]).

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"The last few months have seen meaningful progress across our portfolio, marked by achievements in our Sci-B-Vac Phase 3 studies, PROTECT and CONSTANT, and early data from our Phase 1/2a study of VBI-1901 in recurrent glioblastoma (GBM)," said Jeff Baxter, President and CEO. "We have now completed vaccination and enrollment in the PROTECT and CONSTANT studies, respectively, with an exceptionally low drop-out rate, and no safety signals observed or reported to-date. These accomplishments support the clean safety profile of Sci-B-Vac and reaffirm the timeline for top-line data from the PROTECT and CONSTANT trials, expected to read out mid-year 2019 and around the 2019 year-end, respectively."

Mr. Baxter continued, "Additionally, we are encouraged by the early immunologic data we’ve seen from our Phase 1/2a study of VBI-1901 in recurrent glioblastoma (GBM), which has shown evidence of robust boosting of CMV-specific immunity in some subjects in both the low-dose and intermediate-dose cohorts. With the addition of two top neuro-oncology sites, Dana-Farber Cancer Institute and Massachusetts General Hospital, we expect to complete enrollment in the high-dose cohort quickly. Thanks to the dedication and achievements of the entire VBI team, the fundamentals of the company remain strong and on-track as we head into a critical and exciting first half of 2019."

Recent Highlights and Upcoming Milestones

Formation of Three Scientific and Clinical Advisory Boards

● In September, VBI announced the creation of three Scientific and Clinical Advisory Boards (SABs) comprised of leading global experts in immunology and vaccinology. The SABs will work closely with VBI’s management team to develop and advance the Company’s hepatitis B, cytomegalovirus (CMV), and glioblastoma (GBM) vaccine programs.

Appointment of Chief Financial Officer and Head of Business Development

● In August, VBI appointed Christopher McNulty as Chief Financial Officer and Head of Business Development, bringing with him a strong background in licensing transactions, capital markets and financing strategy, and public company corporate finance in the biopharmaceutical and medical device fields.

Sci-B-Vac for Hepatitis B

● The Company’s prophylactic Hepatitis B vaccine, Sci-B-Vac, is currently being evaluated in a global, pivotal Phase 3 clinical program, the results of which are intended to support future regulatory and marketing authorization submissions in the U.S., Europe, and Canada. The program consists of two concurrent Phase 3 studies:
● PROTECT Study: A head-to-head immunogenicity study against Engerix-B.

○ In October, VBI announced completion of vaccination, with the last subject receiving the final vaccination.
○ Patients in the PROTECT study were vaccinated with a low drop-out rate of 4.5%, reinforcing the safety and tolerability of the vaccine.
○ The independent Data and Safety Monitoring Board (DSMB) reviewed all PROTECT safety data available to-date and did not observe or report any safety signals.
○ Top-line data from the PROTECT study are expected mid-year 2019.
● CONSTANT Study: A lot-to-lot consistency study.
○ VBI completed enrollment in the CONSTANT study, with the last subject receiving the first vaccination in November.
○ Top-line data from the CONSTANT study are expected around the 2019 year-end.

VBI-1901 for Glioblastoma (GBM)

● The Phase 1/2a study of VBI-1901 in recurrent GBM is ongoing, with early immunologic data to be presented in a poster discussion at the Annual Scientific Meeting and Education Day of the Society for Neuro-Oncology (SNO) on November 16, 2018.
○ Early data from the low- and intermediate-dose cohorts of VBI-1901 are encouraging, with robust boosting of CMV-specific immunity, directed against multiple antigens, observed in some subjects in both cohorts.
○ In September 2018, VBI announced that the DSMB unanimously recommended continuation of the study without modification after review of all safety data from the fully-enrolled, intermediate-dose patient cohort.
○ Following the DSMB recommendation, VBI initiated patient enrollment in the high-dose cohort of Part A of the study, the dose-escalation phase.
○ VBI expects to complete enrollment in the high-dose study arm later in 2018, at which point a final, pre-specified DSMB review will occur before initiation of enrollment in Part B of the Phase 1/2a study.
○ VBI expects to report more extensive immunologic data and 6-month overall survival and progression-free survival from all dose cohorts in Part A of the Phase 1/2a in the first half of 2019.

VBI-1501 for Congenital Cytomegalovirus (CMV)

● Following the positive safety and immunogenicity data from the Phase 1 randomized, observer-blind, placebo-controlled study evaluating the safety and immunogenicity of VBI-1501 in May 2018, discussions are ongoing with regulatory bodies to determine the design of a Phase 2 dose-ranging study.
● Further details are expected to be announced by the end of 2018.

Third Quarter 2018 Financial Results

○ VBI ended the third quarter of 2018 with $26.0 million in cash and cash equivalents compared with $67.7 million as of December 31, 2017. Net cash used in operations for the nine months ended September 30, 2018 was $38.0 million.

○ Revenue for the third quarter of 2018 was $0.26 million and was primarily attributable to sales of Sci-B-Vac in Israel and in Europe on a named-patient basis. Revenue for the third quarter of 2017 was $0.19 million and was primarily attributable to sales of Sci-B-Vac in Israel.

○ Research and development expenses were $10.5 million for the third quarter of 2018, as compared to $5.2 million for the same period in 2017. The increase was primarily due to the continuation of the Phase 3 program for Sci-B-Vac and the Phase 1/2a clinical study for VBI-1901 in recurrent GBM patients.

○ General and administrative expenses were $3.5 million for the third quarter of 2018 as compared to $2.8 million for the same period in 2017. The increase was primarily due to allocation of expenses from costs of revenues related to the modernization and capacity increases of our manufacturing facility in Rehovot, Israel, human resources expenses, and stock-based compensation expenses.

○ Net loss and net loss per share for the third quarter of 2018 were $15.4 million and $0.24 respectively, compared to a net loss of $9.8 million and a net loss per share of $0.24 for the third quarter of 2017.