On September 10, 2019 Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) reported that management will present at the Morgan Stanley Global Healthcare Conference on Wednesday, September 11, 2019 at 12:15 p.m. ET (Press release, Vertex Pharmaceuticals, SEP 10, 2019, View Source [SID1234539411]).

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The audio portion of management’s remarks can be accessed live through Vertex’s website, www.vrtx.com, in the "Investors" section under the "News and Events" page. A replay of the conference webcast will be archived on the company’s website.

On September 10, 2019 Omeros Corporation (Nasdaq: OMER) reported a new approach to cancer immunotherapy that targets inhibition of GPR174, a member of the family of G protein-coupled receptors (GPCRs), which can be combined with and significantly improve the tumor-killing effects of adenosine pathway inhibitors. GPR174-targeting immunotherapy is expected to be applicable to all solid tumors (e.g., breast, lung, pancreas, colon, brain, etc.) (Press release, Omeros, SEP 10, 2019, View Source [SID1234539410]).

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The new cancer-immunotherapy approach is based on a series of discoveries by Omeros related to GPR174, which include:

The identification of cancer-immunity pathways controlled by GPR174
The identification of phosphatidylserine (PS) as a potent natural ligand for GPR174
A collection of novel small-molecule inhibitors of GPR174
A dramatic and synergistic enhancement of "tumor-fighting" cytokine production by T cells following the combined inhibition of both GPR174 and the adenosine pathway (e.g., A2A and/or A2B), another key metabolic pathway that regulates tumor immunity
GPR174 and A2A/A2B Adenosine Receptors

Like GPR174, A2A/A2B adenosine receptors are GPCRs. Central components of the adenosine pathway, A2A/A2B receptors are being targeted for cancer immunotherapy by several companies. GPR174 and A2A/A2B receptors share certain features. First, each of them increases intracellular cyclic adenosine monophosphate (cAMP), which is well known for suppressing the type of immune response necessary for killing tumor cells. Second, these receptors are activated by molecules (i.e., PS and adenosine, respectively) that are highly enriched in the tumor microenvironment. Strikingly, in experiments with total human peripheral blood mononuclear cells (PBMCs) where PS and adenosine are naturally abundant, GPR174 inhibitors synergized with A2A/A2B inhibitors to increase T-cell responses dramatically. Findings include the following:

A2A/A2B inhibition alone yielded, on average, 2-fold increases in both interferon-gamma (IFN-γ) and tumor necrosis factor (TNF) and smaller increases in granulocyte-macrophage colony stimulating factor (GM-CSF) and interleukin-2 (IL-2)
GPR174 inhibition alone averaged increases of 2.8-fold for IFN-γ, 2.7-fold for TNF, 1.7-fold for IL-2, and 1.4-fold for GM-CSF
Combining a GPR174 inhibitor with A2A and/or A2B inhibition increased the average IFN-γ and TNF levels 8- to 9-fold and the average IL-2 and GM-CSF levels nearly 3-fold, with maximum increases reaching 25-fold for IFN-γ and TNF and over 4-fold for IL-2 and GM-CSF
"Our GPR174-related discoveries open a wholly new approach to targeting the tumor microenvironment for cancer immunotherapy," said Marc Gavin, Ph.D., Omeros’ director of immunology who previously pioneered research on cAMP signaling in regulatory T cells and initiated and led Amgen’s groundbreaking IL-2 mutein program. "The adenosine pathway has been a focus of immunotherapy drug development efforts across the pharmaceutical industry. We have now shown that a relatively modest boost in cytokine production by T cells observed with inhibition of the A2A or A2B receptors, singly or together, is synergistically augmented when combined with GPR174 inhibitors. Furthermore, the discovery that PS itself stimulates an immunosuppressive GPCR – namely GPR174 – on all lymphocytes represents a significant advancement in our understanding of how PS regulates tumor immunity. New animal data from our group support these collective findings, and I look forward to clinical studies with GPR174 inhibitors, which could potentially become a treatment for most or all solid malignancies."

Relevance of Findings

Omeros’ findings are also particularly relevant for patients resistant to checkpoint inhibitors, such as anti-PD-1 (e.g., Keytruda and Opdivo) and anti-CTLA-4 (Yervoy), and to emerging cellular therapies such as CAR-T cells and adoptive T-cell therapy. Checkpoint inhibitors are only effective in a minority of patients, and high levels of adenosine-generating molecules have been observed in non-responding patients. Furthermore, overcoming natural immunosuppression in solid tumors represents a major hurdle for cellular therapies. Because PS and adenosine are both products of cell stress and death in solid tumors, it is expected that patients resistant to checkpoint inhibitors or cellular therapies would benefit greatly from the combined inhibition of the GPR174 and adenosine pathways.

"Our team’s discovery of the GPR174-controlled cancer-immunity pathways and their interrelationships with the adenosine pathway have been methodically elucidated and defined," said Gregory A. Demopulos, M.D., Omeros’ chairman and chief executive officer. "Simply put, Omeros has discovered that there are two feet on the cAMP brake pedal restraining immunity against the tumor and, to enable effective tumor-killing activity, both GPR174 and the adenosine pathway must be inhibited. We are optimizing our small-molecule GPR174 inhibitors with the objective of moving orally available therapeutics into the clinic as rapidly as possible. We look forward to providing physicians and patients with a new and broadly applicable option in cancer immunotherapy."

Omeros is preparing a manuscript for publication detailing its GPR174-related discoveries and data and plans to present these same discoveries and data beginning this year at upcoming oncology international congresses.

Omeros is establishing an expansive and exclusive intellectual property position directed to GPR174 inhibitors as well as to inhibition of the GPR174 receptor, both alone and in combination with other cancer therapies, for the treatment of any tumor or malignancy.

On September 10, 2019 DelMar Pharmaceuticals, Inc. (Nasdaq: DMPI) ("DelMar" or the "Company"), a biopharmaceutical company focused on the development of new cancer therapies, reported its financial results for the year ended June 30, 2019 (Press release, DelMar Pharmaceuticals, SEP 10, 2019, View Source [SID1234539409]).

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"This has been a productive quarter and year for DelMar as we have achieved multiple data milestones for both of our Phase 2 GBM trials for VAL-083," commented Saiid Zarrabian, DelMar’s President and Chief Executive Officer. "Most importantly, based on recent budget refinements, we now believe we have sufficient capital to achieve release of topline data for two of our three currently enrolling MGMT-unmethylated GBM patient groups. These topline results include the recurrent setting GBM trial being conducted at the MD Anderson Cancer Center and the first line GBM trial being conducted at Sun Yat-sen University Cancer Center in China. We anticipate results from both of these trials to be announced during the fourth quarter of calendar year 2020 and will be supported by the Company’s recent financings completed in June and August 2019. Additionally, we have also relocated our headquarters to San Diego to position ourselves for better access to required expertise as we advance our trials."

RECENT CORPORATE UPDATES

September 2019 – Moved corporate headquarters to San Diego, California
August 2019 – Closed an underwritten public offering with net proceeds of approximately $6.7 million
August 2019 – Provided update on Phase 2 clinical study on first line therapy in newly diagnosed MGMT-unmethylated GBM patients being conducted at Sun Yat-sen University Cancer Center. At the time of the update, nine patients were assessed as having achieved complete response, seven were assessed with stable disease, and one was assessed with disease progression
July 2019 – Enrolled first patient in adjuvant (pre-temozolomide maintenance) arm of Phase 2 open label study of VAL-083 being conducted at MD Anderson Cancer Center (MDACC)
July 2019 – Provided enrollment update of Phase 2 open label study of VAL-083 in recurrent GBM patients with MGMT-unmethylated status. As of this announcement, 56 of the planned 83 patients had been enrolled in the study being conducted at MDACC
June 2019 – Closed on registered direct financing with net proceeds of approximately $3.2 million
May 2019 – Expanded Scientific Advisory Board to include leading neuro-oncologists, Dr. David Reardon from Dana-Farber Cancer Center, Dr. Timothy Cloughsey from David Geffen School of Medicine, and Dr. Nicholas Butowski from UCSF and the Brain Tumor Center
April 2019 – Announced formation of Scientific Advisory Board, which includes current members of the Company’s board of directors, Dr. Napoleone Ferrara and Dr. John de Groot from the MD Anderson Cancer Center
SUMMARY OF FINANCIAL RESULTS FOR FISCAL YEAR ENDED JUNE 30, 2019

At June 30, 2019, the Company had cash and cash equivalents on hand of approximately $3.7 million. In August 2019, the Company completed an underwritten public offering for net proceeds of approximately $6.7 million. The proceeds from the August 2019 financing combined with cash and cash equivalents on hand at June 30, 2019 are expected to be sufficient to fund the Company’s planned operations into the fourth quarter of calendar year 2020.

For the year ended June 30, 2019, the Company reported a net loss of approximately $8.0 million, or $3.16 per share, compared to a net loss of approximately $11.1 million, or $5.42 per share, for the year ended June 30, 2018.

On September 10, 2019 Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE:CFBI), a biotechnology company with a pipeline of proprietary small molecule drugs that bind specifically to the A3 adenosine receptor (A3AR), addressing cancer, liver, and inflammatory diseases, reported it has entered into a collaboration agreement with Univo Pharmaceuticals (TASE:UNVO), a medical cannabis company, to identify and co-develop specific formulations of cannabis components for the treatment of cancer, inflammatory, autoimmune, and metabolic diseases (Press release, Can-Fite BioPharma, SEP 10, 2019, View Source [SID1234539408]).

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It is widely recognized that the identification of specific receptors and pathways through which CBD operates is expected to greatly enhance the development of CBD-based pharmaceuticals. Can-Fite is uniquely positioned to contribute its deep pharmaceutical development expertise to the CBD market, based on findings published in peer reviewed scientific journals demonstrating that CBDs bind to the Gi protein-coupled A3 adenosine receptor (A3AR), which is over-expressed in pathological cells. Can-Fite is a global leader in the research and development of drugs that target A3AR.

There are two main components to the collaboration agreement:

Discovery, development, and commercialization of medical cannabis-based therapeutics: Can-Fite and Univo will jointly collaborate in the discovery of, and Can-Fite will have a first right to express interest to clinically develop, cannabis and cannabis components for the treatment of cancer, inflammatory, autoimmune, and metabolic diseases.

Development and commercialization of CBD screening assays: Can-Fite will develop a screening assay to identify therapeutically active cannabis components, and once developed, Univo will market the assay on a ‘fee for service’ basis to other pharmaceutical companies worldwide.

According to Adroit Market Research, the medical cannabis market is projected to grow at a CAGR of 29% to $56.7 billion by 2026 and according to Zion Market Research, the global market for cell-based assays is estimated to reach $16.2 billion by 2025.

In this collaboration, Univo will provide Can-Fite with cannabis and cannabis components, as well as full access to its laboratories for both research and manufacturing. Can-Fite has agreed to pay Univo a total of $500,000 through two installments and is issuing to Univo approximately 19.9 million of its ordinary shares through a private placement, representing approximately 16.6% of Can-Fite’s ordinary shares outstanding after giving effect to the issuance. The companies will initially share ownership of intellectual property developed in this collaboration. Revenues derived from the collaboration will generally be shared between Can-Fite and Univo on the basis of each party’s contribution. Golan Bitton, Univo’s CEO, is being appointed to the Can-Fite board in connection with the collaboration agreement.

Dr. Pnina Fishman, Can-Fite CEO, commented, "This collaboration provides Can-Fite with new and very exciting business opportunities for utilizing our technology platform and expertise."

Golan Bitton, Univo’s CEO stated, "Can-Fite’s deep expertise in A3 adenosine receptors is a significant asset in our joint collaboration and we are pleased to have formed this alliance. Can-Fite has taken its drug candidates into Phase II and Phase III clinical studies and this makes Can-Fite highly suitable to take cannabis-based therapeutics into fully-fledged clinical development."

On September 10, 2019 VBI Vaccines Inc. (Nasdaq: VBIV) ("VBI"), a commercial-stage biopharmaceutical company developing next-generation infectious disease and immuno-oncology vaccines, reported a collaboration with GlaxoSmithKline (GSK) to clinically evaluate the combination of VBI-1901, VBI’s cancer vaccine immunotherapeutic, with GSK’s proprietary AS01B adjuvant system (Press release, VBI Vaccines, SEP 10, 2019, View Source [SID1234539406]). As part of the collaboration, VBI plans to add an additional study arm to Part B of the company’s ongoing, multi-center, open-label Phase 1/2a clinical study targeting recurrent glioblastoma (GBM), a cytomegalovirus (CMV)-associated tumor.

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"VBI-1901 has shown encouraging results in Part A of the ongoing Phase 1/2a clinical study in recurrent GBM patients and we are excited to be able to expand the scope of Part B to assess the candidate in combination with AS01B, a highly-innovative adjuvant system that has contributed to positive results in combination with the gE antigen in GSK’s shingles vaccine, Shingrix," said David E. Anderson, Ph.D., VBI’s Chief Scientific Officer. "VBI’s enveloped virus-like particle (eVLP) technology, the basis for VBI-1901, is highly versatile and has demonstrated clinical potency in both preventative and therapeutic settings. We believe that these two technologies may be an ideal match for next-generation vaccines, and we look forward to seeing the results of this collaboration."

"This is the first time we have partnered with a biopharma company to evaluate AS01B in such a clinical setting and the first time this adjuvant will be assessed in oncology for GBM patients. We have shown the ability of AS01B to boost T-cell mediated immunity and believe the combination of AS01B and VBI-1901 could have benefits for patients with glioblastoma, a rare but devastating cancer," said Emmanuel Hanon, Senior Vice President, Head of R&D at GSK Vaccines.

In Part A of the study, VBI-1901 adjuvanted with granulocyte-macrophage colony-stimulating factor (GM-CSF) was well-tolerated at all doses. Further, three out of six patients in the high-dose (10 µg) cohort demonstrated evidence of stable disease by magnetic resonance imaging (MRI), which correlated with vaccine-induced immune response. Based on this safety and immunogenicity data, the high-dose was identified as the optimal therapeutic dose to test in the Part B extension phase of the study.

Part B of the ongoing Phase 1/2a clinical study is now planned to be a two-arm, open-label study, enrolling 20 first-recurrent GBM patients to receive VBI-1901 in combination with either GM-CSF or AS01B as immunomodulatory adjuvants. Enrollment of the 10 patients in the VBI-1901 with GM-CSF arm was initiated at the end of July 2019. Initiation of enrollment of the 10 patients in the VBI-1901 with AS01B arm is expected later in the second half of 2019, subject to U.S. Food and Drug Administration (FDA) acceptance of the amended protocol.

VBI’s ongoing two-part study is being conducted at The Neurological Institute of New York Columbia University Medical Center, Dana-Farber Cancer Institute, and Massachusetts General Hospital.

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 38 patients with recurrent GBM:

Part A:

Dose-escalation phase that defined the safety, tolerability, and optimal dose level of VBI-1901 in recurrent GBM patients, with any number of prior recurrences
This phase enrolled 18 recurrent GBM patients across three dose cohorts of VBI-1901: 0.4 µg, 2.0 µg, and 10.0 µg
Enrollment completed in December 2018

Part B:

Subsequent extension of the optimal dose level, 10.0 µg, as defined in the Part A dose escalation phase
This phase will be a two-arm study, enrolling 10 patients in each arm, assessing VBI-1901 in combination with either GM-CSF or AS01B as immunomodulatory adjuvants
Part B will enroll first-recurrent GBM patients only

VBI-1901 is administered intradermally when adjuvanted with granulocyte-macrophage colony-stimulating factor (GM-CSF), and will be administered intramuscularly when adjuvanted with GSK proprietary AS01B adjuvant system. Patients in both phases of the study will receive the vaccine immunotherapeutic every four weeks until clinical progression.

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

About Glioblastoma (GBM)

Scientific literature suggests cytomegalovirus (CMV) infection is prevalent in multiple solid tumors, including GBM, gliomas, and breast cancer, among others. GBM is among the most common and aggressive malignant primary brain tumors. 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.