On August 9, 2019 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 second quarter ending June 30, 2019, and provided an update on the Company’s recent and future developments (Press release, VBI Vaccines, AUG 9, 2019, View Source [SID1234538551]).

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"In Q2 2019, we presented positive data on two of our lead programs – the successful PROTECT Phase 3 data for Sci-B-Vac, our trivalent hepatitis B vaccine, and a poster presentation at ASCO (Free ASCO Whitepaper) illustrating encouraging data from Part A of the ongoing Phase 1/2a clinical study of VBI-1901 in recurrent GBM patients," said Jeff Baxter, VBI’s president and CEO. "We continue to execute our corporate objectives and, based on the recent achievements and announcements, we believe the potential of the VBI pipeline is as strong as ever. We remain excited about VBI’s future, especially in light of the expected near-term milestones across all four lead programs."

Recent Highlights and Upcoming Milestones

Sci-B-Vac: Trivalent Prophylactic Hepatitis B Vaccine

Positive top-line data from PROTECT were announced in June 2019. The study, which enrolled a total of 1,607 adults, of which approximately 80% were age ≥ 45 years, successfully met both of its co-primary endpoints:

Non-inferiority of seroprotection rates (SPR) of Sci-B-Vac compared with Engerix-B in all subjects age ≥ 18 years
Superiority of SPR of Sci-B-Vac compared with Engerix-B in subjects age ≥ 45 years
Moreover, the SPR of Sci-B-Vac compared with Engerix-B was statistically significantly higher at each time point on a per-visit basis, and in all key subgroup analyses of adults age ≥ 18 years, including by age, gender, body mass index (BMI), diabetic status, and smoking status.

"The positive results from PROTECT reaffirm the robust safety and efficacy data that we have for Sci-B-Vac from previous clinical studies, and they further support the meaningful commercial opportunity for the vaccine," said Jeff Baxter. "We believe that, if approved, Sci-B-Vac will be highly-competitive and well-positioned within the North American and European hepatitis B markets, differentiating on efficacy, safety, and cost. Where faster seroprotection and cost may drive use in the young and otherwise healthy populations, superior seroprotection rates and safety are likely to drive use in the older and immunocompromised populations. Pending successful completion of the CONSTANT Phase 3 study, we anticipate beginning the submissions of applications for regulatory approvals in the U.S., Europe, and Canada mid-year 2020."

Top-line data from the CONSTANT Phase 3 study, a 4-arm lot-to-lot consistency study in approximately 2,850 subjects, is expected around year-end 2019.

VBI-1901 – Glioblastoma (GBM) Immunotherapeutic

Immunogenicity data and tumor/clinical responses from Part A of the ongoing Phase 1/2a study in recurrent GBM patients were presented at ASCO (Free ASCO Whitepaper) in June 2019. This data showed three out of six (3/6) patients in the high-dose, 10 μg, cohort had evidence of stable disease by magnetic resonance imaging (MRI) and median progression-free survival (PFS) was longer among vaccine responders (14.5 weeks) compared with non-responders (6 weeks).

Based on safety and immunogenicity, the highest dose tested in Part A, 10 µg, has been selected as the optimal dose level to test in Part B of the study. Part B of the study is a subsequent extension phase with narrower enrollment criteria, designed to more clearly assess immunologic responses and potential correlations with early clinical benefit. Enrollment of 10 first-recurrence GBM patients in Part B initiated in July 2019.

VBI-2601 – Hepatitis B Immunotherapeutic

As part of the collaboration with Brii Biosciences, work is underway to prepare regulatory filings to enable the expected initiation of enrollment in the Phase 2 study in subjects with chronic hepatitis B in the fourth quarter of 2019, which would thus provide an expected initial human proof-of-concept readout in the second half of 2020.

VBI-1501 – Prophylactic Cytomegalovirus (CMV) Vaccine

In July 2019, VBI received positive feedback from the FDA on the design of the proposed Phase 2 dose-ranging study that is expected to assess the safety and immunogenicity of dosages of VBI-1501 up to 20 µg with alum. Submission of the Investigational New Drug (IND) application for this study is expected around the end of 2019.

Enveloped Virus-Like Particle (eVLP) Platform

On August 8, 2019, at the Immuno-Oncology Summit 2019 in Boston, David E. Anderson, Ph.D., VBI’s Chief Scientific Officer, presented further data on the broad potential of the eVLP platform in immuno-oncology. The clinical and pre-clinical data presented showed that not only are eVLPs a highly potent antigen delivery mechanism, but the lipid bilayer surrounding the eVLPs also enables the functional activity of immunomodulatory molecules, such as CD40 ligand, which further enhances potency by activating both innate and adaptive immunity.

Second Quarter 2019 Financial Results

Cash Position: VBI ended the second quarter of 2019 with $30.1 million in cash and cash equivalents compared to $59.3 million as of December 31, 2018.
Net Cash Used in Operating Activities: Net cash used in operations for the six months ended June 30, 2019 was $26.3 million compared to $24.5 million for the same period in 2018.
Cash Used for Purchase of Property and Equipment: Cash used for the purchase of property and equipment was $2.9 million for the six months ended June 30, 2019 compared to $2.0 million for the same period in 2018. The increase is largely related to the purchase of manufacturing and information technology equipment at the manufacturing facility in Rehovot, Israel, as part of the modernization and capacity increase of the facility. In 2018, the manufacturing facility in Rehovot was temporarily closed for modernization and capacity increase – operations re-commenced in May 2019, and we anticipate a review of the facility by the Israeli Ministry of Health in the second half of 2019.
Revenue: Revenue in the second quarter of 2019 was $0.6 million, compared to $0.2 million for the same period in 2018. The increase was primarily due to R&D services revenues earned pursuant to the therapeutic hepatitis B collaboration and license agreement with Brii Biosciences.
Research and Development (R&D): R&D expenses were $7.4 million for the second quarter of 2019, compared to $10.9 million for the same period in 2018. The decrease was primarily due to the completion of the Phase 1 study assessing VBI-1501, and to the decrease in spending related to the advancement of the Phase 3 program for Sci-B-Vac and the Phase 1/2a clinical study for VBI-1901.
General and Administrative (G&A): G&A expenses were $3.2 million for the second quarter of 2019, compared to $4.0 million for the same period in 2018. The decrease was primarily due to a decrease in marketing and administrative expenses, as well as both an impairment loss on property and equipment and a reclassification of certain costs of revenues to G&A, as part of the temporary closure of the Rehovot facility, that were incurred in the three months ended June 30, 2018 that did not re-occur during the three months ended June 30, 2019.
Net Loss: Net loss and net loss per share for the second quarter of 2019 were $13.2 million and $0.13, respectively, compared to a net loss of $16.7 million and a net loss per share of $0.26 for the second quarter of 2018.

On August 9, 2019 Stemline Therapeutics, Inc. (Nasdaq: STML), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel oncology therapeutics, reported that Ivan Bergstein, M.D., Stemline’s CEO, will present at the 2019 Wedbush PacGrow Healthcare Conference on Tuesday, August 13, 2019 at 4:15 PM ET at the Parker New York Hotel (Press release, Stemline Therapeutics, AUG 9, 2019, View Source [SID1234538582]). A live webcast of the presentation can be viewed on the company’s website at www.stemline.com.

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About ELZONRIS
ELZONRIS (tagraxofusp-erzs), a CD123-directed cytotoxin, is approved by the U.S. Food and Drug Administration (FDA) and commercially available in the U.S. for the treatment of adult and pediatric patients, two years or older, with blastic plasmacytoid dendritic cell neoplasm (BPDCN). For full prescribing information in the U.S., visit www.ELZONRIS.com. In Europe, a marketing authorization application (MAA) is under review by the European Medicines Agency (EMA). ELZONRIS is also being evaluated in additional clinical trials in other indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF), and acute myeloid leukemia (AML).

About BPDCN
BPDCN is an aggressive hematologic malignancy with historically poor outcomes and an area of unmet medical need. BPDCN typically presents in the bone marrow and/or skin and may also involve lymph nodes and viscera. The BPDCN cell of origin is the plasmacytoid dendritic cell (pDC) precursor. The diagnosis of BPDCN is based on the immunophenotypic diagnostic triad of CD123, CD4, and CD56, as well as other markers. For more information, please visit the BPDCN disease awareness website at www.bpdcninfo.com.

About CD123
CD123 is a cell surface target expressed on a wide range of myeloid tumors including blastic plasmacytoid dendritic cell neoplasm (BPDCN), certain myeloproliferative neoplasms (MPNs) including chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF), acute myeloid leukemia (AML) (and potentially enriched in certain AML subsets), myelodysplastic syndrome (MDS), and chronic myeloid leukemia (CML). CD123 has also been reported on certain lymphoid malignancies including multiple myeloma (MM), acute lymphoid leukemia (ALL), hairy cell leukemia (HCL), Hodgkin’s lymphoma (HL), and certain Non-Hodgkin’s lymphomas (NHL). In addition, CD123 has been detected on some solid tumors as well as autoimmune disorders including cutaneous lupus and scleroderma.

On August 9, 2019 AstraZeneca reported positive overall survival (OS) results from the Phase III FLAURA trial, a randomised, double-blinded, multi-centre trial of Tagrisso (osimertinib) in previously-untreated patients with locally-advanced or metastatic non-small cell lung cancer (NSCLC) whose tumours have epidermal growth factor receptor (EGFR) mutations (Press release, AstraZeneca, AUG 9, 2019, View Source [SID1234538494]).

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Tagrisso showed a statistically-significant and clinically-meaningful improvement in OS, a secondary endpoint in the FLAURA Phase III trial, compared with erlotinib or gefitinib both of which were previous standard-of-care (SoC) treatments in this setting. The FLAURA trial met its primary endpoint in July 2017, showing a statistically-significant and clinically-meaningful improvement in progression-free survival (PFS), increasing the time patients lived without disease progression or death from any cause. The safety and tolerability of Tagrisso was consistent with its established profile.

José Baselga, Executive Vice President, Oncology R&D said: "Today’s positive results show that Tagrisso provides an unprecedented survival outcome versus previous standard-of-care epidermal growth factor receptor tyrosine kinase inhibitors, reaffirming Tagrisso as the 1st-line standard-of-care for EGFR-mutated metastatic non-small cell lung cancer."

AstraZeneca plans to present the OS results from the FLAURA trial at a forthcoming medical meeting.

Tagrisso is currently approved in 74 countries, including the US, Japan and the EU, for 1st-line EGFRm metastatic NSCLC.

About lung cancer

Lung cancer is the leading cause of cancer death among both men and women, accounting for about one-fifth of all cancer deaths, more than breast, prostate and colorectal cancers combined.1 Lung cancer is broadly split into NSCLC and small cell lung cancer (SCLC), with 80-85% classified as NSCLC.2 Approximately 10-15% of NSCLC patients in the US and Europe, and 30-40% of patients in Asia have EGFR-mutated (EGFRm) NSCLC.3-5 These patients are particularly sensitive to treatment with EGFR-tyrosine kinase inhibitors (TKI) which block the cell-signalling pathways that drive the growth of tumour cells. Approximately 25% of patients with EGFRm NSCLC have brain metastases at diagnosis, increasing to approximately 40% within two years of diagnosis.6 The presence of brain metastases often reduces median survival to less than eight months.7

About Tagrisso

Tagrisso (osimertinib) is a third-generation, irreversible EGFR-TKI designed to inhibit both EGFR-sensitising and EGFR T790M-resistance mutations, with clinical activity against central nervous system metastases. Tagrisso 40mg and 80mg once-daily oral tablets have now received approval in more than 70 countries, including the US, Japan and the EU, for 1st-line EGFRm advanced NSCLC, and in more than 80 countries, including the US, Japan, China and the EU, for 2nd-line use in patients with EGFR T790M mutation-positive advanced NSCLC. Tagrisso is also being developed in the adjuvant setting (ADAURA trial), in the locally-advanced unresectable setting (LAURA), in combination with chemotherapy (FLAURA2) and with potential new medicines (SAVANNAH, ORCHARD).

About FLAURA

The FLAURA trial assessed the efficacy and safety of Tagrisso 80mg orally once daily vs. comparator EGFR-TKIs (either erlotinib [150mg orally, once daily] or gefitinib [250mg orally, once daily]) in previously-untreated patients with locally-advanced or metastatic EGFRm NSCLC. The trial was double-blinded and randomised, with 556 patients across 29 countries.

About AstraZeneca in lung cancer

AstraZeneca has a comprehensive portfolio of approved and potential new medicines in late-stage clinical development for the treatment of different forms of lung cancer spanning several stages of disease, lines of therapy and modes of action. We aim to address the unmet needs of patients with EGFR-mutated tumours as a genetic driver of disease, which occur in 10-15% of NSCLC patients in the US and EU and 30-40% of NSCLC patients in Asia, with our approved medicines Iressa (gefitinib) and Tagrisso, and ongoing Phase III trials ADAURA, LAURA, FLAURA and FLAURA2 as well as the Phase II combination trials SAVANNAH and ORCHARD.3-5

Our extensive late-stage Immuno-Oncology programme focuses on lung cancer patients without a known genetic mutation which represents up to 50% of all patients with lung cancer. Imfinzi (durvalumab), an anti-PDL1 antibody, is in development as monotherapy (Phase III trials ADJUVANT BR.31, PACIFIC-4, PACIFIC-5, and PEARL) and in combination with tremelimumab and/or chemotherapy (Phase III trials AEGEAN, PACIFIC-2, NEPTUNE, POSEIDON, ADRIATIC and CASPIAN).

About AstraZeneca in oncology

AstraZeneca has a deep-rooted heritage in oncology and offers a quickly-growing portfolio of new medicines that has the potential to transform patients’ lives and the Company’s future. With at least six new medicines to be launched between 2014 and 2020, and a broad pipeline of small molecules and biologics in development, we are committed to advance oncology as a key growth driver for AstraZeneca focused on lung, ovarian, breast and blood cancers. In addition to our core capabilities, we actively pursue innovative partnerships and investments that accelerate the delivery of our strategy as illustrated by our investment in Acerta Pharma in haematology.

By harnessing the power of four scientific platforms – Immuno-Oncology, Tumour Drivers and Resistance, DNA Damage Response and Antibody Drug Conjugates – and by championing the development of personalised combinations, AstraZeneca has the vision to redefine cancer treatment and one day eliminate cancer as a cause of death.

On August 9, 2019 CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a U.S. biopharmaceutical company focused on developing and commercializing therapeutics and pharmaceutical products in China, U.S., and throughout the world, reported financial results for the second quarter and six months ended June 30, 2019 and provided a review of recent accomplishments and anticipated milestones (Press release, CASI Pharmaceuticals, AUG 9, 2019, View Source [SID1234538536]).

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Wei-Wu He, Ph.D., CASI’s Chairman and Chief Executive Officer, commented, "Our financial results remained strong through the second quarter of 2019. We look forward to launching the first product from our hematology oncology pipeline, EVOMELA, this quarter and fully transitioning into a commercial organization. We are enthusiastic about our growth and momentum, and continue to pursue additional strategic assets for our pipeline, both in our hematology/oncology space as well as other high quality treatment solutions for patients."

Second Quarter and Recent Business Highlights

Acquired exclusive worldwide rights to commercialize anti-CD19 T-cell therapy in China and worldwide – On June 17, 2019, CASI signed a license agreement for exclusive worldwide commercialization rights to an autologous anti-CD19 T-cell therapy product (CNCT19) from Juventas Cell Therapy Ltd. CNCT19 targets CD-19, a B-cell surface protein widely expressed during all phases of B-cell development and is a validated target for B-cell driven hematological malignancies. CD19 antigen is the most frequently used biomarker in CAR-T cell therapy clinical trials for hematological malignancies.

Acquired exclusive worldwide rights to a novel anti-CD38 monoclonal antibody (CID-103) – On April 17, 2019, CASI acquired exclusive global rights to a novel anti-CD38 monoclonal antibody (CID-103). CID-103 is a human monoclonal antibody targeting a specific epitope of the CD38 cell surface antigen. CID-103 is at the IND/IMPD submission stage of development with initiation of the Phase 1 study targeted for early 2020.

EVOMELA (Melphalan Hydrochloride for Injection) expected to launch in China this quarter – The Company has made significant progress and commercial preparations for the launch of EVOMELA and expects commercialization this quarter. It will be the only approved melphalan product available in China.

Second Quarter and First Half 2019 Financial Results

Cash Position: As of June 30, 2019, CASI had cash and cash equivalents of $70.3 million compared to $99.7 million as of March 31, 2019. The decrease in cash is primarily due to the Black Belt and Juventas investments made during the three months ended June 30, 2019.

R&D Expenses: Research and development (R&D) expenses for the three and six months ended June 30, 2019, were $3.0 million and $5.6 million, respectively, compared to $1.7 million and $3.4 million for the same periods in 2018. The increase in R&D expenses primarily reflects costs associated with regulatory, consulting and manufacturing related services, as well as an increase in personnel costs due to growth in the number of employees.

G&A Expenses: General and administrative (G&A) expenses for the three and six months ended June 30, 2019, were $7.0 million and $12.7 million, respectively, compared to $4.0 million and $5.3 million for the same periods in 2018. The increase in G&A for both the three and six months ended June 30, 2019 was related to a combination of factors primarily resulting from the Company’s growth in China. These factors include an increase in salary, benefits and recruitment expense and facilities costs due to increases in head count, sales and marketing efforts to prepare for the anticipated launch of the Company’s first commercial product (EVOMELA), professional services fees (including audit and legal services), and an increase in non-cash stock compensation expense largely attributed to stock options issued to the President of CASI China and other new employees.

Net Loss: The Company reported a net loss for the three and six months ended June 30, 2019 of ($15.3) million, or ($0.16) per share, and ($23.4) million, or ($0.25) per share, respectively, compared to ($5.9) million, or ($0.07) per share, and ($9.5) million, or ($0.12) per share for the same periods in 2018. The larger net loss is primarily due to acquired in-process research and development expenses related to the Black Belt license, and the increase in both G&A and R&D expenses primarily due to the Company’s growth in China to support the Company’s first commercial product (EVOMELA).

Further information regarding the Company, including its Quarterly Report on Form 10-Q for the quarter ended June 30, 2019, can be found at www.casipharmaceuticals.com.

On August 9, 2019 Akebia Therapeutics, Inc. (Nasdaq: AKBA), a biopharmaceutical company focused on the development and commercialization of therapeutics for people living with kidney disease, reported that John P. Butler, President and Chief Executive Officer of Akebia, will participate in the following investor conferences (Press release, Akebia, AUG 9, 2019, View Source [SID1234538552]):

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The BTIG Biotechnology Conference 2019 on Monday, August 12, 2019 in New York City, New York (no formal presentation).
The Morgan Stanley 17th Annual Global Healthcare Conference on Monday, September 9, 2019 at 8:45 a.m. in New York City, New York.
Where applicable, a live webcast and replay of the Company’s presentation will be available on the Company’s website at www.akebia.com.